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Chen, Yu, und Ruiqing Yan. „From registration, protocol to report: are COVID-19-related RCTs in mainland China consistent? A systematic review of clinical trial registry and literature“. BMJ Open 12, Nr. 7 (Juli 2022): e058070. http://dx.doi.org/10.1136/bmjopen-2021-058070.
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ObjectiveTo provide a comprehensive review of registered COVID-19-related randomised controlled trials (RCTs) in mainland China and evaluate the transparency of reporting through comparison of registrations, protocols and full reports.DesignSystematic review of trial registrations and publications.Data sourcesInternational Clinical Trials Registry Platform, Chinese Clinical Trial Registry, ClinicalTrials.gov, the ISRCTN registry and EU Clinical Trial Register were accessed on 1 February 2022. Publications were searched in PubMed, Embase, Cochrane Library, Google Scholar, CNKI.net and Wanfangdata from 10 February 2022 to 12 February 2022.Eligibility criteriaEligible trials were COVID-19 related RCTs carried out in mainland China. Observational studies, non-randomised trials and single-arm trials were excluded.Data extraction and synthesisTwo reviewers independently extracted data from registrations, publications and performed risk of bias assessment for trial reports. Information provided by registrations and publications was compared. The findings were summarised with descriptive statistics.ResultsThe number of eligible studies was 415. From these studies 20 protocols and 77 RCT reports were published. Seven trials published both protocol and RCT full report. Between registrations and publications, discrepancy or omission was found in sample size (7, 35.0% for protocols and 47, 61.0% for reports, same below), trial setting (13, 65.0% and 43, 55.8%), inclusion criteria (12, 60.0% and 57, 74.0%), exclusion criteria (10, 50.0% and 54, 70.1%), masking method (9, 45.0% and 35, 45.5%) and primary outcome or time frame of primary outcome measurement (14, 70.0% and 51, 66.2%). Between protocols and full reports, 5 (71.4%) reports had discrepancy in primary outcome or time frame of primary outcome measurement.ConclusionsDiscrepancy among registrations, protocols and reports revealed compromised transparency in reporting of COVID-19-related RCTs in mainland China. The importance of trial registration should be further emphasised to enhance transparent RCT reporting.
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Zaghloul, Hadeel, Odette Chagoury, Sara Elhadad, Salma Hayder Ahmed, Noor Suleiman, Abdulla Al Naama, Katie El Nahas et al. „Clinical and metabolic characteristics of the Diabetes Intervention Accentuating Diet and Enhancing Metabolism (DIADEM-I) randomised clinical trial cohort“. BMJ Open 10, Nr. 12 (Dezember 2020): e041386. http://dx.doi.org/10.1136/bmjopen-2020-041386.
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ObjectivesDiabetes Intervention Accentuating Diet and Enhancing Metabolism-I (DIADEM-I) is the first randomised controlled trial (RCT) in the Middle East and North Africa (MENA) region testing the effectiveness of an intensive lifestyle intervention (ILI) for weight loss and diabetes remission. We report on the recruitment process and baseline characteristics of the DIADEM-I cohort based on origin (Middle East vs North Africa), and waist circumference.DesignDIADEM-I is an open-label randomised, controlled, parallel group RCT recruiting young individuals (18–50 years) with early type 2 diabetes (≤3 years since diagnosis) originating from MENA. Individuals from primary care were randomised to usual medical care or ILI (total dietary replacement phase using meal replacement products, followed by staged food reintroduction and physical activity support). The primary outcome is weight loss at 12 months. Other outcomes are glycaemic control and diabetes remission.SettingPrimary care, Qatar.Participants147 (73% men) randomised within DIADEM-I who were included in the final trial data analysis.Outcome measuresRecruitment metrics, and baseline clinical and metabolic characteristics.ResultsOf 1498 people prescreened, 267 (18%) were invited for screening and 209 (78%) consented. 173 (83%) were eligible. 15 (7%) withdrew before randomisation and the remaining 158 were randomised. Mean age was 42.1 (SD 5.6) years and mean body mass index was: 36.3 (5.5) kg/m2 (women) and 34.4 (5.4) kg/m2 (men). Mean diabetes duration was 1.8 (1.0) years and mean glycosylated haemoglobin (HbA1c) was 7.0% (1.30) (52.5 mmol/mol (SD 14.3)). Participants originated from 13 countries. Those from North Africa reported greater physical activity and had lower family history of diabetes. 90% of subjects were taking diabetes medications and 31% antihypertensives. Those with greater waist circumference had significantly higher insulin resistance and lower quality of life.ConclusionRecruitment of participants originating from the MENA region into the RCT was successful, and study participation was readily accepted. While DIADEM-I participants originated from 13 countries, there were few baseline differences amongst participants from Middle East versus North Africa, supporting generalisability of RCT results.Trial registration numberISRCTN20754766; NCT03225339
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Planas-Cerezales, Lurdes, Laura Fabbri und Laurence Pearmain. „Add-on therapy for pulmonary fibrosis, a forthcoming era with implications for practice: the BI 101550 and RELIEF trials“. Breathe 19, Nr. 3 (September 2023): 230090. http://dx.doi.org/10.1183/20734735.0090-2023.
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The therapeutic landscape for idiopathic pulmonary fibrosis (IPF) and progressive fibrosing interstitial lung disease (PFILD) is increasingly complex, with add-on antifibrotic options now in clinical trials, or available for patients progressing on first-line therapy in both conditions. Here, we review two recent trials of potential add-on therapeutic options, the BI 101550 and RELIEF trials. BI 101550 was a phase 2 randomised control trial (RCT) of a novel phosphodiesterase-4 inhibitor in patients with IPF, with a primary end-point of change in forced vital capacity (ΔFVC) (in mL) at 12 weeks. The RELIEF trial was a phase 2 RCT in patients with PFILD, with a primary end-point of ΔFVC (absolute % predicted) over 48 weeks. Whilst the BI 101550 and RELIEF trials showed positive results in their primary end-points, the strengths and weaknesses of both trials are discussed with importance for their interpretation and clinical impact. We review current clinical practice in IPF and PFILD and place the BI101550 and RELIEF trial results in context, highlighting advances and problems with antifibrotic therapies.Commentary on:Richeldi L,et al.Trial of a preferential phosphodiesterase 4B inhibitor for idiopathic pulmonary fibrosis. N Engl J Med 2022; 386: 2178–2187.Behr J,et al.Pirfenidone in patients with progressive fibrotic interstitial lung diseases other than idiopathic pulmonary fibrosis (RELIEF): a double-blind, randomised, placebo-controlled, phase 2b trial. Lancet Respir Med 2021; 9: 476–486.
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Keogan, Sheila, Shasha Li und Luke Clancy. „Allen Carr’s Easyway to Stop Smoking - A randomised clinical trial“. Tobacco Control 28, Nr. 4 (25.10.2018): 414–19. http://dx.doi.org/10.1136/tobaccocontrol-2018-054243.
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Objective To determine if Allen Carr’s Easyway to Stop Smoking (AC) was superior to Quit.ie in a randomised clinical trial (RCT). Setting Single centre, open RCT, general population based. Participants 300 adult smokers, 18 years plus, minimum 5 cigarettes daily, and English speaking. AC, 151 (females 44.4%) and Quit.ie, 149 (females 45.6%), mean age 44 years. outcomes for all 300 were analysed (intention-to-treat). Recruited through advertisement from July 2015 to February 2016. Intervention Randomly assigned to AC (n=151) and Quit.ie (n=149), matched for age, sex and education. Block randomisation, enrolment and follow-up at 1, 3, 6 and 12 months. Primary aim was to determine if AC had higher quit rates than Quit.ie service at 3 months. Secondary aims: quit rates at 1, 6 and 12 months and analysis of associated factors including weight. AC consisted of a 5-hour seminar, in a group setting. Quit.ie is an online portal for smoking cessation. Results AC had higher quit rates at 1, 3, 6 and 12 months. AC: 38%, (n=57), 27% (n=40), 23% (n=35), 22% (n=33) vs Quit.ie: 20% (n=30), 15% (n=22), 15% (n=23), 11% (n=17), respectively (all p values <0.05). Logistic regression AC vs Quit.ie, OR 2.26 (95% CI 1.22 to 4.21) p value=0.01. Weight gain 3.8 kg in AC vs 1.8 kg in Quit.ie (p value <0.05). Conclusions All AC quit rates were superior to Quit.ie, outcomes were comparable with established interventions. Trial registration number NCT12951013. Recruitment July 2015–February 2016.
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Jenkins, Valerie A., und Lesley J. Fallowfield. „For the Benefit of Others: Reasons Why Women with Breast Cancer Participate in RCTs“. Breast Care 10, Nr. 2 (2015): 88–93. http://dx.doi.org/10.1159/000376563.
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Background: Appreciation of the barriers and drivers affecting enrolment in randomised clinical trials (RCTs) is important for future trial design, communication and information provision. Methods: As part of an intervention to facilitate UK multidisciplinary team communication about RCTs, women with breast cancer who discussed trials with doctors or research nurses completed questionnaires examining i) clarity of trial information and ii) reasons for their trial decision. Results: 152 women completed the questionnaires; 113/152 (74%) consented to RCT enrolment. Patients' satisfaction with communication about the trial information was very good, irrespective of participation decisions. Acceptors' and decliners' responses to 9/16 statements concerning decisions about trial participation differed significantly. ‘Wanting to help with doctor's research' influenced 100% acceptors compared to 57% of decliners (p < 0.001). Decliners were more likely to be ‘worried about randomisation' (20 vs. 39%; p < 0.035) and to ‘want doctor to choose treatment rather than be randomised' (31 vs. 53%; p < 0.031). Primary reason for trial acceptance was altruism; ‘I feel that others with my illness will benefit from the results of the trial', 58/108 (54%). Conclusion: A majority of women accepted RCT entry citing altruistic motivations as the primary driver for participation. Trial design and setting (metastatic or adjuvant) had little impact on participation.
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Hamilton, E. P., G. H. Lyman, S. Kim und J. Peppercorn. „Availability of experimental therapy outside of randomized clinical trials in oncology“. Journal of Clinical Oncology 27, Nr. 15_suppl (20.05.2009): 6539. http://dx.doi.org/10.1200/jco.2009.27.15_suppl.6539.
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6539 Background: Investigational cancer therapies may be available outside of trials, or “off protocol” (OPRx), with implications for patient safety, trial accrual, and access to care. Previous studies suggest OPRx is prevalent in oncology, but there is little consensus on when it should or should not be considered. We evaluated the scope and impact of OPRx through assessment of availability of the experimental arms of recent randomized trials (RCT), and evaluation of study outcomes and accrual. Methods: We conducted a Medline search to identify all English language phase III RCT of medical interventions in oncology over a 2-year period ending April 17, 2008. We determined availability of experimental interventions based on FDA approval for any indication. We limited assessment of accrual (time to trial completion, patients/month) to studies with US sites. Significance of results was assessed by Fisher's exact test and unpaired t-test. Results: We identified 172 eligible RCT. The majority of RCT (108, 63%) evaluated drugs that were available OPRx at trial initiation, while an additional 19 (11%) trial drugs became available during the trial. 64 (55%) were available due to FDA approval for the same cancer in a different setting, 40 (35%) for a different cancer, and 12 (10%) for a non-cancer indication. 25% of trials were conducted at only US sites, 15% included US and international sites, and 60% were international only. Trials in which OPRx was available had slower time to completion compared to trials in which OPRx was unavailable (48 vs. 26 months, p = 0.04) and a trend towards slower accrual (14.0 vs. 40.7 patients/month, p = 0.06). For the majority of RCT (66%), there was at least one grade 3/4 toxicity that was greater in the experimental arm, for 47% the experimental interventions proved superior for 1 major outcome, and 27% demonstrated improvement in overall survival. These outcomes did not vary based on availability OPRx. Conclusions: The majority of recent oncology trials involve experimental regimens that are available outside of a trial. The safety and efficacy of novel interventions must be determined by trials but availability of OPRX may impact accrual. Guidelines are needed for OPRx in oncology. No significant financial relationships to disclose.
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Chen, Stephanie C., Cheryl McCullumsmith und Scott Y. H. Kim. „Disclosing the potential impact of placebo controls in antidepressant trials“. BJPsych Open 1, Nr. 1 (Juni 2015): 1–5. http://dx.doi.org/10.1192/bjpo.bp.115.000109.
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BackgroundAlthough placebo-control clinical trials that withhold effective treatments can be permissible, how best to inform participants of the placebo design has received little attention.AimsTo determine the effect of disclosing quantitative outcome estimates of individual treatment v. entering placebo-control randomised control trial (RCT) on willingness to enrol in such an RCT.MethodWe randomised 278 adult patients at a depression clinic to receive standard disclosure (n = 129) or enhanced (n = 149) quantitative outcome estimates (based on decision analysis) of individual treatment v. RCT, and assessed their willingness to enrol in the RCT.ResultsA greater proportion of those in the standard arm preferred enrolling in RCT (41.3% v. 23.8%, P = 0.002). Those in the standard arm preferred RCT more for direct benefit than altruism reasons, whereas the opposite was true in the enhanced arm.ConclusionsDisclosing the quantitative outcome implications of placebos may select for fewer but more altruistic participants.
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Alli, Babatunde Y., Sreenath Madathil, Simon D. Tran und Belinda Nicolau. „Protocol: carrageenan for the prevention of oral HPV infection – a feasibility randomised clinical trial“. BMJ Open 13, Nr. 7 (Juli 2023): e074498. http://dx.doi.org/10.1136/bmjopen-2023-074498.
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IntroductionHead and neck cancers (HNCs) are a significant health burden worldwide. Oral human papillomavirus (HPV) infection is a major risk factor for HNCs. Unfortunately, currently available prophylactic vaccines have limited coverage and potential for HPV type replacement. Carrageenan, a natural product extracted from marine red algae, has demonstrated potency as an HPV inhibitor and could offer a potential alternative to prevent HPV-related diseases, including oral HPV infection. However, there is a lack of clinical studies on the effect of carrageenan on oral HPV infections. As a first step to address this gap, we propose a randomised controlled trial (RCT) to evaluate the feasibility of conducting a larger multicentric RCT to investigate the effect of a carrageenan mouthwash on oral HPV infection.Methods and analysisWe will conduct a placebo-controlled triple-blinded feasibility RCT with two parallel arms, each arm consisting of 20 participants. Participants will complete a single in-person visit at baseline and conduct biweekly follow-ups from home by completing a web-based questionnaire and sending saliva self-samples via mail. During the 6-month period trial, participants will gargle with the mouthwash morning and night, and around sexual activities. The study will evaluate several factors including recruitment and retention rates, the feasibility of data collection procedures, compliance with study procedures, acceptability of RCT procedures and intervention and safety data on carrageenan use in the oral cavity. We will estimate the standard deviation of outcome measures, including time to the incidence of oral HPV infection and time to clearance of prevalent oral HPV infection. The trial primary outcome is whether to proceed to a definitive trial based on prespecified progression criteria.Ethics and disseminationThe protocol was approved by the McGill University institutional review board. Study results will be presented at scientific conferences and published in academic journals.Trial registration numberNCT05746988.
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Kho, Michelle E., Alexander J. Molloy, France J. Clarke, Julie C. Reid, Margaret S. Herridge, Timothy Karachi, Bram Rochwerg et al. „Multicentre pilot randomised clinical trial of early in-bed cycle ergometry with ventilated patients“. BMJ Open Respiratory Research 6, Nr. 1 (Februar 2019): e000383. http://dx.doi.org/10.1136/bmjresp-2018-000383.
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IntroductionAcute rehabilitation in critically ill patients can improve post-intensive care unit (post-ICU) physical function. In-bed cycling early in a patient’s ICU stay is a promising intervention. The objective of this study was to determine the feasibility of recruitment, intervention delivery and retention in a multi centre randomised clinical trial (RCT) of early in-bed cycling with mechanically ventilated (MV) patients.MethodsWe conducted a pilot RCT conducted in seven Canadian medical-surgical ICUs. We enrolled adults who could ambulate independently before ICU admission, within the first 4 days of invasive MV and first 7 days of ICU admission. Following informed consent, patients underwent concealed randomisation to either 30 min/day of in-bed cycling and routine physiotherapy (Cycling) or routine physiotherapy alone (Routine) for 5 days/week, until ICU discharge. Our feasibility outcome targets included: accrual of 1–2 patients/month/site; >80% cycling protocol delivery; >80% outcomes measured and >80% blinded outcome measures at hospital discharge. We report ascertainment rates for our primary outcome for the main trial (Physical Function ICU Test-scored (PFIT-s) at hospital discharge).ResultsBetween 3/2015 and 6/2016, we randomised 66 patients (36 Cycling, 30 Routine). Our consent rate was 84.6 % (66/78). Patient accrual was (mean (SD)) 1.1 (0.3) patients/month/site. Cycling occurred in 79.3% (146/184) of eligible sessions, with a median (IQR) session duration of 30.5 (30.0, 30.7) min. We recorded 43 (97.7%) PFIT-s scores at hospital discharge and 37 (86.0%) of these assessments were blinded.DiscussionOur pilot RCT suggests that a future multicentre RCT of early in-bed cycling for MV patients in the ICU is feasible.Trial registration numberNCT02377830.
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Watts, Stacey, Yogesh Apte, Thomas Holland, April Hatt, Alison Craswell, Frances Lin, Alexis Tabah et al. „Randomised, controlled, feasibility trial comparing vasopressor infusion administered via peripheral cannula versus central venous catheter for critically ill adults: A study protocol“. PLOS ONE 19, Nr. 5 (13.05.2024): e0295347. http://dx.doi.org/10.1371/journal.pone.0295347.
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Background When clinicians need to administer a vasopressor infusion, they are faced with the choice of administration via either peripheral intravenous catheter (PIVC) or central venous catheter (CVC). Vasopressor infusions have traditionally been administered via central venous catheters (CVC) rather than Peripheral Intra Venous Catheters (PIVC), primarily due to concerns of extravasation and resultant tissue injury. This practice is not guided by contemporary randomised controlled trial (RCT) evidence. Observational data suggests safety of vasopressor infusion via PIVC. To address this evidence gap, we have designed the “Vasopressors Infused via Peripheral or Central Access” (VIPCA) RCT. Methods The VIPCA trial is a single-centre, feasibility, parallel-group RCT. Eligible critically ill patients requiring a vasopressor infusion will be identified by emergency department (ED) or intensive care unit (ICU) staff and randomised to receive vasopressor infusion via either PIVC or CVC. Primary outcome is feasibility, a composite of recruitment rate, proportion of eligible patients randomised, protocol fidelity, retention and missing data. Primary clinical outcome is days alive and out of hospital up to day-30. Secondary outcomes will include safety and other clinical outcomes, and process and cost measures. Specific aspects of safety related to vasopressor infusions such as extravasation, leakage, device failure, tissue injury and infection will be assessed. Discussion VIPCA is a feasibility RCT whose outcomes will inform the feasibility and design of a multicentre Phase-3 trial comparing routes of vasopressor delivery. The exploratory economic analysis will provide input data for the full health economic analysis which will accompany any future Phase-3 RCT.
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Udayakumar, Suji, Sasha Thomson, Albiruni Ryan Abdul Razak und Kelvin K. Chan. „Do early phase trials predict clinical efficacy in subsequent phase III biomarker-enriched randomized trials?“ Journal of Clinical Oncology 40, Nr. 16_suppl (01.06.2022): 3152. http://dx.doi.org/10.1200/jco.2022.40.16_suppl.3152.
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3152 Background: Efficacy endpoints of randomized controlled trials (RCT) are commonly used as the basis of regulatory drug approvals. Recently, promising results in early phase trials have resulted in approval of biomarker-targeted therapies. We examined if early phase trial results were associated with efficacy in subsequent biomarker-enriched RCTs. Methods: All cancer drug RCTs conducted between January 2006 and March 2021 were identified through Clinicaltrials.gov. Trials were eligible if a biomarker was used to select a patient population for treatment with a targeted agent. Associated early phase trials were included if they matched the RCT in treatment setting and patient population. Trials pairs were compared using objective response rate (ORR) and progression-free survival (PFS). We assessed difference in endpoints using summary measures (e.g., average, range). We examined whether early phase trials results were associated with RCT results using logistic regression. Results: The search yielded 2,157 unique phase III RCTs and 27 RCTs met eligibility criteria pairing with associated early phase trials, where 17 RCTs met their primary endpoint. The most common biomarkers were EGFR+ (n = 8), HER2+ (n = 5) and PD-L1 (n = 5). Based on average difference of trial pairs, ORR was similar between trials (1.59%, 95% CI = -2.5-5.6, p = 0.50) and median PFS was slightly higher in early phase trials (1.95 months, 95% CI = 0.91-2.99, p < 0.05). On an individual pair basis, there was large range of variability in the difference between early phase trials and RCTs for ORR (range = -23.9-20.2%) and median PFS (range = -0.8-7.4 months). The probability of the RCT meeting its primary endpoint is 50% or 95%, when the early phase trial ORR is 41.2% (95% CI = 35.2-47.1%) or 77.7% (95% CI = 71.7-83.6%), respectively. Conclusions: Through comparison of early phase trials and subsequent phase III RCT, we found that, overall, ORR has minimal bias in early phase trials, and median PFS appears to be slightly overestimated. Substantial variability in results for trial pairs suggests that, on an individual basis, results in early phase trial can be inconsistent with results in subsequent RCT. Early phase trial results may be associated with RCTs meeting their primary endpoint when ORR is very high; however, caution must be exercised when using early phase trials as representative of RCTs for decision-making as the predictive ability of early phase trials is limited.
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Soomro, Naeem, Jan Lecouturier, Deborah D. Stocken, Jing Shen, Ann Marie Hynes, Holly F. Ainsworth, David Breen, Grenville Oades, David Rix und Michael Aitchison. „Surveillance versus ablation for incidentally diagnosed small renal tumours: the SURAB feasibility RCT“. Health Technology Assessment 21, Nr. 81 (Dezember 2017): 1–68. http://dx.doi.org/10.3310/hta21810.
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Background There is uncertainty around the appropriate management of small renal tumours. Treatments include partial nephrectomy, ablation and active surveillance. Objectives To explore the feasibility of a randomised trial of ablation versus active surveillance. Design Two-stage feasibility study: stage 1 – clinician survey and co-design work; and stage 2 – randomised feasibility study with qualitative and economic components. Methods Stage 1 – survey of radiologists and urologists, and development of patient information materials. Stage 2 – patients identified across eight UK centres with small renal tumours (< 4 cm) were randomised (1 : 1 ratio) to ablation or active surveillance in an unblinded manner. Randomisation was carried out by a central computer system. The primary objective was to determine willingness to participate and to randomise a target of 60 patients. The qualitative and economic data were collected separately. Results The trial was conducted across eight centres, with a site-specific period of recruitment ranging from 3 to 11 months. Of the 154 patients screened, 36 were eligible and were provided with study details. Seven agreed to be randomised and one patient was found ineligible following biopsy results. Six patients (17% of those eligible) were randomised: three patients received ablation and no serious adverse events were recorded. The 3- and 6-month data were collected for four (67%) and three (50%) out of the six patients, respectively. The qualitative substudy identified factors directly impacting on the recruitment of this trial. These included patient and clinician preferences, organisational factors (variation in clinical pathway) and standard treatment not included. The health economic questionnaire was designed and piloted; however, the sample size of recruited patients was insufficient to draw a conclusion on the feasibility of the health economics. Conclusions The trial did not meet the criteria for progression and the recruitment rate was lower than hypothesised, demonstrating that a full trial is presently not possible. The qualitative study identified factors that led to variation in recruitment across the sites. Implementation of organisational and operational measures can increase recruitment in any future trial. There was insufficient information to conduct a full economic analysis. Trial registration Current Controlled Trials ISRCTN31161700. Funding This project was funded by the National Institute for Health Research (NIHR) Health Technology Assessment programme and will be published in full in Health Technology Assessment; Vol. 21, No. 81. See the NIHR Journals Library website for further project information.
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Young, Amber E., Anna Davies, Sophie Bland, Sara Brookes und Jane M. Blazeby. „Systematic review of clinical outcome reporting in randomised controlled trials of burn care“. BMJ Open 9, Nr. 2 (Februar 2019): e025135. http://dx.doi.org/10.1136/bmjopen-2018-025135.
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IntroductionSystematic reviews collate trial data to provide evidence to support clinical decision-making. For effective synthesis, there must be consistency in outcome reporting. There is no agreed set of outcomes for reporting the effect of burn care interventions. Issues with outcome reporting have been identified, although not systematically investigated. This study gathers empirical evidence on any variation in outcome reporting and assesses the need for a core outcome set for burn care research.MethodsElectronic searches of four search engines were undertaken from January 2012 to December 2016 for randomised controlled trials (RCTs), using medical subject headings and free text terms including ‘burn’, ‘scald’ ‘thermal injury’ and ‘RCT’. Two authors independently screened papers, extracted outcomes verbatim and recorded the timing of outcome measurement. Duplicate outcomes (exact wording ± different spelling), similar outcomes (albumin in blood, serum albumin) and identical outcomes measured at different times were removed. Variation in outcome reporting was determined by assessing the number of unique outcomes reported across all included trials. Outcomes were classified into domains. Bias was reduced using five researchers and a patient working independently and together.Results147 trials were included, of which 127 (86.4%) were RCTs, 13 (8.8%) pilot studies and 7 (4.8%) RCT protocols. 1494 verbatim clinical outcomes were reported; 955 were unique. 76.8% of outcomes were measured within 6 months of injury. Commonly reported outcomes were defined differently. Numbers of unique outcomes per trial varied from one to 37 (median 9; IQR 5,13). No single outcome was reported across all studies demonstrating inconsistency of reporting. Outcomes were classified into 54 domains. Numbers of outcomes per domain ranged from 1 to 166 (median 11; IQR 3,24).ConclusionsThis review has demonstrated heterogeneity in outcome reporting in burn care research which will hinder amalgamation of study data. We recommend the development of a Core Outcome Set.PROSPERO registration numberCRD42017060908.
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Mellish, Louise C., Colin Dunkley, Colin D. Ferrie und Deb K. Pal. „Antiepileptic drug treatment of rolandic epilepsy and Panayiotopoulos syndrome: clinical practice survey and clinical trial feasibility“. Archives of Disease in Childhood 100, Nr. 1 (08.09.2014): 62–67. http://dx.doi.org/10.1136/archdischild-2013-304211.
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BackgroundThe evidence base for management of childhood epilepsy is poor, especially for the most common specific syndromes such as rolandic epilepsy (RE) and Panayiotopoulos syndrome (PS). Considerable international variation in management and controversy about non-treatment indicate the need for high quality randomised controlled trials (RCT). The aim of this study is, therefore, to describe current UK practice and explore the feasibility of different RCT designs for RE and PS.MethodsWe conducted an online survey of 590 UK paediatricians who treat epilepsy. Thirty-two questions covered annual caseload, investigation and management practice, factors influencing treatment, antiepileptic drug preferences and hypothetical trial design preferences.Results132 responded (22%): 81% were paediatricians and 95% at consultant seniority. We estimated, annually, 751 new RE cases and 233 PS cases. Electroencephalography (EEG) is requested at least half the time in approximately 70% of cases; MRI brain at least half the time in 40%–65% cases and neuropsychological evaluation in 7%–8%. Clinicians reported non-treatment in 40%: main reasons were low frequency of seizures and parent/child preferences. Carbamazepine is the preferred older, and levetiracetam the preferred newer, RCT arm. Approximately one-half considered active and placebo designs acceptable, choosing seizures as primary and cognitive/behavioural measures as secondary outcomes.ConclusionsManagement among respondents is broadly in line with national guidance, although with possible overuse of brain imaging and underuse of EEG and neuropsychological assessments. A large proportion of patients in the UK remains untreated, and clinicians seem amenable to a range of RCT designs, with carbamazepine and levetiracetam the preferred active drugs.
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Kragsnaes, Maja Skov, Shaun Theodor Sødergren, Jens Kjeldsen, Hans Christian Horn, Heidi Lausten Munk, Jens Kristian Pedersen, Camilla Schufri Klinkby et al. „Experiences and perceptions of patients with psoriatic arthritis participating in a trial of faecal microbiota transplantation: a nested qualitative study“. BMJ Open 11, Nr. 3 (März 2021): e039471. http://dx.doi.org/10.1136/bmjopen-2020-039471.
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ObjectivesPatients’ first-hand experiences of faecal microbiota transplantation (FMT) performed in a rheumatological care setting have yet to be elucidated. The objectives were to explore participants’ perceptions of being part of an FMT trial thereby identifying potential trial participation effects and enlightening the patient perspective on the outlook for future FMT trials in rheumatic diseases.DesignIn a qualitative study nested within a double-blind, randomised, placebo-controlled trial (RCT) testing FMT as a potential new antirheumatic treatment, semistructured telephone interviews were conducted following the trial participants’ final 26-week visit. Qualitative researchers, who did not take part in the main trial, performed the interviews and the primary analysis. The experiences explored related to the conduct of the RCT and changes in the participants’ everyday life. The analysis was carried out using a thematic approach.SettingA Danish rheumatology university outpatient clinic with nationwide inclusion.ParticipantsThe study included 10 patients with psoriatic arthritis (PsA) who were unaware of their treatment allocation (FMT/sham transplantation) and completed the final 26-week trial visit.ResultsParticipation in the RCT influenced the patients’ understanding of PsA and induced positive changes in their everyday life. Renewed hopes for the future in addition to a feeling of enhanced care contributed to significant trial participation effects. FMT was deemed a tolerable and safe treatment.ConclusionsDiscrepancies between the clinical and the research setting should be considered when discussing the clinical relevance of the results of the RCT. Overall, patients with PsA who have participated in an RCT testing FMT find the treatment acceptable and safe encouraging more research into the field of microbiota-targeted interventions in rheumatic diseases.Trial registration numberNCT03058900; Pre-results.
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Ladwa, Rahul, Elizabeth P. Pinkham, Laisa Teleni, Brigid Hanley, Gemma Lock, Jodie Nixon, Oluwaseyifunmi Andi Agbejule et al. „Telehealth cancer-related fatigue clinic model for cancer survivors: a pilot randomised controlled trial protocol (the T-CRF trial)“. BMJ Open 12, Nr. 5 (Mai 2022): e059952. http://dx.doi.org/10.1136/bmjopen-2021-059952.
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IntroductionCancer-related fatigue (CRF) is one of the most common and debilitating adverse effects of cancer and its treatment reported by cancer survivors. Physical activity, psychological interventions and management of concurrent symptoms have been shown to be effective in alleviating CRF. This pilot randomised controlled trial (RCT) will determine the feasibility of a telehealth CRF clinic intervention (T-CRF) to implement evidence-based strategies and assess the impact of the intervention on CRF and other clinical factors in comparison to usual care.Methods and analysisA parallel-arm (intervention vs usual care) pilot RCT will be conducted at the Princess Alexandra Hospital in Queensland, Australia. Sixty cancer survivors aged 18 years and over, who report moderate or severe fatigue on the Brief Fatigue Inventory and meet other study criteria will be recruited. Participants will be randomised (1:1) to receive the T-CRF intervention or usual care (ie, specialist-led care, with a fatigue information booklet). The intervention is a 24-week programme of three telehealth nurse-led consultations and a personalised CRF management plan. The primary objective of this pilot RCT is to determine intervention feasibility, with a secondary objective to determine preliminary clinical efficacy. Feasibility outcomes include the identification of recruitment methods; recruitment rate and uptake; attrition; adherence; fidelity; apathy; and intervention functionality, acceptability and satisfaction. Clinical and resource use outcomes include cancer survivor fatigue, symptom burden, level of physical activity, productivity loss, hospital resource utilisation and carer’s fatigue and productivity loss. Descriptive statistics will be used to report on feasibility and process-related elements additional to clinical and resource outcomes.Ethics and disseminationThis trial is prospectively registered (ACTRN12620001334998). The study protocol has been approved by the Metro South Health and Hospital Services Human Research Ethics Committee (MSHHS HREC/2020/QMS/63495). Findings will be disseminated through peer-reviewed publications, national and international conferences and seminars or workshops.Trial registration numberAustralian New Zealand Clinical Trials Registry ID: ACTRN12620001334998; Pre-results. Trial Version: Version 1.1. Last updated 10 December 2020.
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Ene, Crina Georgiana, Fergus Gracey und Catherine Ford. „Trainee tips for conducting a clinical trial during the Clinical Psychology Doctorate“. Clinical Psychology Forum 1, Nr. 377 (28.06.2024): 25–28. http://dx.doi.org/10.53841/bpscpf.2024.1.377.25.
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The research component of doctoral training in clinical psychology can evoke a range of emotions, from excitement to apprehension, often influenced by prior experience and the nature of the research undertaken. This article shares reflections on conducting a feasibility randomised controlled trial (RCT) as part of a clinical psychology thesis, offering practical advice to ease the process for trainee clinical psychologists considering a clinical trial for their doctoral research. Key insights include the importance of selecting experienced supervisors, starting early, deciding between a feasibility or full-scale trial, determining recruitment sources, staying organised, building positive relationships with researchers and participants, and choosing a topic of genuine interest. Conducting a clinical trial during clinical psychology training, despite its challenges, can provide invaluable experience and skills, fostering a deeper understanding of clinical research methodologies and their practical applications.
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Abbott, Penelope, Deborah Askew, Chelsea Watego, Wendy CY Hu, Letitia Campbell, Claudette Tyson, Robyn Walsh et al. „Randomised clinical trial research within Aboriginal and Torres Strait Islander primary health services: a qualitative study“. BMJ Open 11, Nr. 12 (Dezember 2021): e050839. http://dx.doi.org/10.1136/bmjopen-2021-050839.
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ObjectiveTo better understand how to undertake valuable, ethical and sustainable randomised controlled clinical trial (RCT) research within Aboriginal and Torres Strait Islander primary health services.DesignIn a qualitative approach, we utilised data collected between 2013 and 2020 during the planning and implementation of two RCTs. The data comprised agreed records of research meetings, and semistructured interviews with clinical trial stakeholders. The stakeholders were parents/carers of child participants, and site-based research officers, healthcare providers and community advisory groups. Our thematic analysis was informed by constructivist grounded theory.SettingThe RCTs investigated the management of otitis media in Aboriginal and Torres Strait Islander children, with the first RCT commencing recruitment in 2014 and the second in 2017. They took place in Aboriginal Medical Services (AMSs), large primary health services for Aboriginal and Torres Strait Islander people, based in urban and regional communities across two Australian states and one territory.ResultsWe analysed data from 56 meetings and 67 interviews, generating themes on making research valuable and undertaking ethical and sustainable RCTs. Aboriginal and Torres Strait Islander leadership, and support of AMSs in their service delivery function were critical. The broad benefits of the trials were considered important to sustainability, including workforce development, enhanced ear healthcare and multidirectional research capacity building. Participants emphasised the long-term responsibility of research teams to deliver benefits to AMSs and communities regardless of RCT outcomes, and to focus on relationships, reciprocity and creating positive experiences of research.ConclusionWe identify principles and strategies to assist in undertaking ethical and sustainable RCTs within Aboriginal and Torres Strait Islander primary health services. Maintaining relationships with AMSs and focusing on mutual workforce development and capacity building creates opportunities for long-term benefits so that health research and RCTs work for Aboriginal and Torres Strait Islander peoples, services, communities and researchers.Trial registration numberACTRN12613001068752 (Pre-results); ACTRN12617001652369 (Pre-results).
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Ong, Sean W. X., Todd C. Lee, Robert A. Fowler, Robert Mahar, Ruxandra L. Pinto, Asgar Rishu, Lina Petrella et al. „Evaluating the impact of a SIMPlified LaYered consent process on recruitment of potential participants to theStaphylococcus aureusNetwork Adaptive Platform trial: study protocol for a multicentre pragmatic nested randomised clinical trial (SIMPLY-SNAP trial)“. BMJ Open 14, Nr. 1 (Januar 2024): e083239. http://dx.doi.org/10.1136/bmjopen-2023-083239.
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IntroductionInformed consent forms (ICFs) for randomised clinical trials (RCTs) can be onerous and lengthy. The process has the potential to overwhelm patients with information, leading them to miss elements of the study that are critical for an informed decision. Specifically, overly long and complicated ICFs have the potential to increase barriers to trial participation for patients with mild cognitive impairment, those who do not speak English as a first language or among those with lower medical literacy. In turn, this can influence trial recruitment, completion and external validity.Methods and analysisSIMPLY-SNAP is a pragmatic, multicentre, open-label, two-arm parallel-group superiority RCT, nested within a larger trial, theStaphylococcus aureusNetwork Adaptive Platform (SNAP) trial. We will randomise potentially eligible participants of the SNAP trial 1:1 to a full-length ICF or a SIMPlified LaYered (SIMPLY) consent process where basic information is summarised with embedded hyperlinks to supplemental information and videos. The primary outcome is recruitment into the SNAP trial. Secondary outcomes include patient understanding of the clinical trial, patient and research staff satisfaction with the consent process, and time taken for consent. As an exploratory outcome, we will also compare measures of diversity (eg, gender, ethnicity), according to the consent process randomised to. The planned sample size will be 346 participants.Ethics and disseminationThe study has been approved by the ethics review board (Sunnybrook Health Sciences Research Ethics Board) at sites in Ontario. We will disseminate study results via the SNAP trial group and other collaborating clinical trial networks.Trial registration numberClinicalTrials.gov Registry (NCT06168474;www.clinicaltrials.gov).
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Novick, D., A. Gonzalez-Pinto, J. M. Haro, J. Bertsch, C. Reed, E. Perrin und M. Tohen. „Translation of Randomised Controlled Trial Findings into Clinical Practice: Comparison of Olanzapine and Valproate in the Emblem Study“. European Psychiatry 24, S1 (Januar 2009): 1. http://dx.doi.org/10.1016/s0924-9338(09)70547-4.
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Aims:To contrast the outcomes of olanzapine- and valproate-treated patients in an observational study of acute mania with the results of a RCT assessing the same treatments (Tohen et al., 2002).Methods:EMBLEM (European Mania in Bipolar Evaluation of Medication) was a 2-year, prospective, observational study of health outcomes associated with treatment of mania. Severity of mania and depression was assessed at baseline and 6 weeks using the YMRS and 5-item version of the HAMD, respectively. The RCT was a 3-week, randomised, double-blind comparison of olanzapine (n=125) and divalproate-treated (n=123) patients hospitalised for acute manic or mixed episodes. The YMRS and HAMD were used to quantify manic and depressive symptoms, respectively.Results:621 EMBLEM patients were analysed (n=107 valproate, n=514 olanzapine). Both observed groups improved from baseline to 6 weeks in mean YMRS and HAMD-5 total scores, with significantly greater mean improvements in the olanzapine compared with the valproate group using linear regression to adjust for baseline differences. The RCT reported significantly greater mean YMRS improvement (but not HAMD) in the olanzapine-treated group. EMBLEM patients treated with olanzapine experienced significantly greater weight gain than patients treated with valproate, similar to RCT results. There was a significantly greater incidence of treatment-emergent gastrointestinal adverse events in EMBLEM patients treated with valproate.Conclusions:The EMBLEM results support those of the RCT, which suggest that olanzapine monotherapy may be more effective than valproate monotherapy in the treatment of acute mania. Contrasting observational and RCT results present methodological challenges but can provide important complementary information.
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Little, John D., Lorraine Davison und Robert D. Little. „Clinically, what ought we to believe?“ Australasian Psychiatry 27, Nr. 1 (31.10.2018): 72–74. http://dx.doi.org/10.1177/1039856218810160.
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Objective: To question the status of the randomised controlled trial (RCT) in the hierarchy of evidence. Conclusions: The RCT provides important and clinically relevant information, particularly in psychopharmacology. However, and as with other methodologies, RCTs too are flawed and automatic abdication to their conclusions, especially in complex social interventions, is unwise. A clinical example with conflicting and polarising views, each with their evidence base, is described alongside a suggested clinical strategy for resolving differences of opinion.
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Asai, Nobuhiro, Yuichi Shibata, Jun Hirai, Wataru Ohashi, Daisuke Sakanashi, Hideo Kato, Mao Hagihara, Hiroyuki Suematsu und Hiroshige Mikamo. „A Gap of Patients with Infective Endocarditis between Clinical Trials and the Real World“. Journal of Clinical Medicine 12, Nr. 4 (16.02.2023): 1566. http://dx.doi.org/10.3390/jcm12041566.
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Introduction: A randomized control trial (RCT) is considered to be the highest level in the Evidence-Based Medicine (EBM) pyramid. While EBM is essential to make a practical tool such as a prognostic guideline, it has been unclear how many patients in the real world can be eligible for a randomized control trial (RCT). Patients and method: This study was performed to clarify if there is a difference in patients’ profiles and clinical outcomes between the patients eligible and not eligible for any RCT. We reviewed all IE patients at our institute between 2007 and 2019. The patients were divided into two groups: those eligible for RCTs (RCT appropriate group) and those who were not (RCT inappropriate group). Exclusion criteria for clinical trials were set based on previous clinical trials. Results: A total of 66 patients were enrolled in the study. The median age was 70 years (range 18 to 87 years), and 46 (70%) were male. Seventeen (26%) of the patients were eligible for RCTs. Comparing the two groups, patients in the RCT appropriate group were younger and had fewer comorbidities. The disease severity was milder in the RCT appropriate groups than in the RCT inappropriate groups. Patients in the RCT appropriate group showed significantly longer overall survival times than those in the RCT inappropriate group (Log-Rank test, p < 0.001). Conclusions: We found a significant gap in patients’ characteristics and clinical outcomes between the groups. Physicians should be aware that RCT can never reflect the real-world population.
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Russell, Ailsa, Kate Cooper, Stephen Barton, Ian Ensum, Daisy Gaunt, Jeremy Horwood, Barry Ingham et al. „Protocol for a feasibility study and randomised pilot trial of a low-intensity psychological intervention for depression in adults with autism: the Autism Depression Trial (ADEPT)“. BMJ Open 7, Nr. 12 (Dezember 2017): e019545. http://dx.doi.org/10.1136/bmjopen-2017-019545.
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IntroductionHigh rates of co-occurring depression are reported in autism spectrum disorder (ASD), a neurodevelopmental condition characterised by social communication impairments and repetitive behaviours. Cognitive-behavioural interventions adapted for ASD have been effective for anxiety problems. There have been evaluation studies of group cognitive-behavioural therapy for co-occurring depression, but no randomised trials investigating low-intensity psychological interventions as recommended in clinical guidelines for mild-moderate depression.Methods and analysisA feasibility study comprising a randomised controlled trial (RCT) and nested qualitative evaluation is under way as preparation for a definitive RCT. Participants (n=70) will be randomised to Guided Self-Help: a low-intensity psychological intervention based on behavioural activation adapted for ASD or treatment as usual. Outcomes including depression symptoms, anxiety, social function and service use will be measured at 10, 16 and 24 weeks postrandomisation and will be blind to group allocation for measures that are not self-administered. The analysis will aim to establish the rates of recruitment and retention for a larger-scale RCT as well as the most appropriate measure of depression to serve as primary outcome. The qualitative study will purposively sample up to 24 participants from each treatment group to consider the acceptability and feasibility of the intervention and the trial design.Ethics and disseminationEthical approval has been received from WALES REC 3 (IRAS project ID: 191558) and the Health Research Authority with R&D approval from Avon and Wiltshire Mental Health Partnership and Northumberland, Tyne and Wear Foundation NHS Trusts. To our knowledge, this is the first study of a low-intensity intervention for depression in adults with autism. The results will inform the design of a definitive RCT. Dissemination will include peer-reviewed journal publications reporting the quantitative and qualitative research findings of the study and presentations at national and international conferences.Trial registration numberISRCTN54650760; Pre-results.
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Adams, Scott C., Julia McMillan, Kirsten Salline, Jessica Lavery, Chaya S. Moskowitz, Konstantina Matsoukas, Maggie M. Z. Chen, Daniel Santa Mina, Jessica M. Scott und Lee W. Jones. „Comparing the reporting and conduct quality of exercise and pharmacological randomised controlled trials: a systematic review“. BMJ Open 11, Nr. 8 (August 2021): e048218. http://dx.doi.org/10.1136/bmjopen-2020-048218.
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ObjectiveEvaluate the quality of exercise randomised controlled trial (RCT) reporting and conduct in clinical populations (ie, adults with or at risk of chronic conditions) and compare with matched pharmacological RCTs.DesignSystematic review.Data sourcesEmbase (Elsevier), PubMed (NLM) and CINAHL (EBSCO).Study selectionRCTs of exercise in clinical populations with matching pharmacological RCTs published in leading clinical, medical and specialist journals with impact factors ≥15.Review methodsOverall RCT quality was evaluated by two independent reviewers using three research reporting guidelines (ie, Consolidated Standards of Reporting Trials (CONSORT; pharmacological RCTs)/CONSORT for non-pharmacological treatments; exercise RCTs), CONSORT-Harms, Template for Intervention Description and Replication) and two risk of bias assessment (research conduct) tools (ie, Cochrane Risk of Bias, Jadad Scale). We compared research reporting and conduct quality within exercise RCTs with matched pharmacological RCTs, and examined factors associated with quality in exercise and pharmacological RCTs, separately.FindingsForty-eight exercise RCTs (11 658 patients; median sample n=138) and 48 matched pharmacological RCTs were evaluated (18 501 patients; median sample n=160). RCTs were conducted primarily in cardiovascular medicine (43%) or oncology (31%). Overall quality score (composite of all research reporting and conduct quality scores; primary endpoint) for exercise RCTs was 58% (median score 46 of 80; IQR: 39–51) compared with 77% (53 of 68; IQR: 47–58) in the matched pharmacological RCTs (p≤0.001). Individual quality scores for trial reporting and conduct were lower in exercise RCTs compared with matched pharmacological RCTs (p≤0.03). Factors associated with higher overall quality scores for exercise RCTs were journal impact factor (≥25), sample size (≥152) and publication year (≥2013).Conclusions and relevanceResearch reporting and conduct quality within exercise RCTs is inferior to matched pharmacological RCTs. Suboptimal RCT reporting and conduct impact the fidelity, interpretation, and reproducibility of exercise trials and, ultimately, implementation of exercise in clinical populations.PROSPERO registration numberCRD42018095033.
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Bajwa, Mandeep S., Richard Jackson, Jagtar Dhanda, Catrin Tudur Smith, Richard J. Shaw und Andrew G. Schache. „Determining the Effectiveness of Fibrin Sealants in Reducing Complications in Patients Undergoing Lateral Neck Dissection (DEFeND): A Randomised External Pilot Trial“. Cancers 15, Nr. 20 (20.10.2023): 5073. http://dx.doi.org/10.3390/cancers15205073.
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Objectives: High-quality randomised controlled trials (RCT) to support the use of Fibrin Sealants (FS) in neck dissection (ND) are lacking. The DEFeND trial assessed critical pilot/feasibility questions and signals from clinical outcomes to inform a future definitive trial. Patients and Methods: The study design piloted was a blinded surgical RCT. All participants underwent unilateral ND for head and neck cancer. Interventional arm: ND with application of FS. Control arm: ND alone. Feasibility outcomes included recruitment, effectiveness of blinding, protocol adherence and evaluating administrative processes. Clinical outcomes included surgical complications (primary outcome), drainage volume, time to drain removal, length of hospital stay, pain and the Neck Dissection Impairment Index. Results: Recruitment completed ahead of time. Fifty-three patients were recruited, and 48 were randomised at a rate of 5.3 patients/month. Blinding of patients, research nurses and outcome assessors was effective. Two protocol deviations occurred. Two patients were lost to follow-up. The mean (SD) Comprehensive Complication Index in the interventional arm was 6.5 (12.8), and it was 9.9 (14.2) in the control arm. The median (IQR) time to drain removal (days) was shorter in the interventional arm (2.67 (2.42, 3.58) vs. 3.40 (2.50, 4.27)). However, this did not translate to a clinically significant reduction in median (IQR) length of hospital stay in days (intervention: 3.48 (2.64, 4.54), control: 3.74 (3.11, 4.62)). Conclusion: The proposed trial design was effective, and a definitive surgical trial is feasible. Whilst there was a tendency for FS to improve clinical outcomes, the effect size did not reach clinical or statistical significance. (ISRCTN99181100).
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Gryaznov, Dmitry, Belinda von Niederhäusern, Benjamin Speich, Benjamin Kasenda, Elena Ojeda-Ruiz, Anette Blümle, Stefan Schandelmaier et al. „Reporting quality of clinical trial protocols: a repeated cross-sectional study about the Adherence to SPIrit Recommendations in Switzerland, CAnada and GErmany (ASPIRE-SCAGE)“. BMJ Open 12, Nr. 5 (Mai 2022): e053417. http://dx.doi.org/10.1136/bmjopen-2021-053417.
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ObjectivesComprehensive protocols are key for the planning and conduct of randomised clinical trials (RCTs). Evidence of low reporting quality of RCT protocols led to the publication of the Standard Protocol Items: Recommendations for Interventional Trials (SPIRIT) checklist in 2013. We aimed to examine the quality of reporting of RCT protocols from three countries before and after the publication of the SPIRIT checklist.DesignRepeated cross sectional study.SettingSwiss, German and Canadian research ethics committees (RECs).ParticipantsRCT protocols approved by RECs in 2012 (n=257) and 2016 (n=292).Primary and secondary outcome measuresThe primary outcomes were the proportion of reported SPIRIT items per protocol and the proportion of trial protocols reporting individual SPIRIT items. We compared these outcomes in protocols approved in 2012 and 2016, and built regression models to explore factors associated with adherence to SPIRIT. For each protocol, we also extracted information on general trial characteristics and assessed whether individual SPIRIT items were reportedResultsThe median proportion of reported SPIRIT items among RCT protocols showed a non-significant increase from 72% (IQR, 63%–79%) in 2012 to 77% (IQR, 68%–82%) in 2016. However, in a preplanned subgroup analysis, we detected a significant improvement in investigator-sponsored protocols: the median proportion increased from 64% (IQR, 55%–72%) in 2012 to 76% (IQR, 64%–83%) in 2016, while for industry-sponsored protocols median adherence was 77% (IQR 72%–80%) for both years. The following trial characteristics were independently associated with lower adherence to SPIRIT: single-centre trial, no support from a clinical trials unit or contract research organisation, and investigator-sponsorship.ConclusionsIn 2012, industry-sponsored RCT protocols were reported more comprehensively than investigator-sponsored protocols. After publication of the SPIRIT checklist, investigator-sponsored protocols improved to the level of industry-sponsored protocols, which did not improve.
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Mitchell-Jones, Nicola, Jessica Alice Farren, Aurelio Tobias, Tom Bourne und Cecilia Bottomley. „Ambulatory versus inpatient management of severe nausea and vomiting of pregnancy: a randomised control trial with patient preference arm“. BMJ Open 7, Nr. 12 (Dezember 2017): e017566. http://dx.doi.org/10.1136/bmjopen-2017-017566.
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ObjectiveTo determine whether ambulatory (outpatient (OP)) treatment of severe nausea and vomiting of pregnancy (NVP) is as effective as inpatient (IP) care.DesignNon-blinded randomised control trial (RCT) with patient preference arm.SettingTwo multicentre teaching hospitals in London.ParticipantsWomen less than 20 weeks’ pregnant with severe NVP and associated ketonuria (>1+).MethodsWomen who agreed to the RCT were randomised via web-based application to either ambulatory or IP treatment. Women who declined randomisation underwent the treatment of their choice in the patient preference trial (PPT) arm. Treatment protocols, data collection and follow-up were the same for all participants.Main outcome measuresPrimary outcome was reduction in Pregnancy Unique Quantification of Emesis (PUQE) score 48 hours after starting treatment. Secondary outcome measures were duration of treatment, improvement in symptom scores and ketonuria at 48 hours, reattendances within 7 days of discharge and comparison of symptoms at 7 days postdischarge.Results152/174 eligible women agreed to participate with 77/152 (51%) recruited to the RCT and 75/152 (49%) to the PPT.Patients were initially compared in four groups (randomised IP, randomised OP, non-randomised IP and non-randomised OP). Comprehensive cohort analysis of participants in the randomised group (RCT) and non-randomised group (PPT) did not demonstrate any differences in patient demographics or baseline clinical characteristics. Pooled analysis of IP versus OP groups showed no difference in reduction in PUQE score at 48 hours (p=0.86). There was no difference in change in eating score (p=0.69), drinking score (p=0.77), well-being rating (p=0.64) or reduction in ketonuria (p=0.47) at 48 hours, with no difference in duration of index treatment episode (p=0.83) or reattendances within 7 days (p=0.52).ConclusionsAmbulatory management is an effective direct alternative to IP management of severe NVP. The trial also demonstrated that many women requiring treatment for severe NVP have strong preferences regarding treatment setting, which may need to be considered by care providers, especially given the psychological impact of severe NVP.Trial registration numberhttp://www.isrctn.com/ISRCTN24659467(March 2014).
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Heyard, Rachel, Leonhard Held, Sebastian Schneeweiss und Shirley V. Wang. „Design differences and variation in results between randomised trials and non-randomised emulations: meta-analysis of RCT-DUPLICATE data“. BMJ Medicine 3, Nr. 1 (Februar 2024): e000709. http://dx.doi.org/10.1136/bmjmed-2023-000709.
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ObjectiveTo explore how design emulation and population differences relate to variation in results between randomised controlled trials (RCT) and non-randomised real world evidence (RWE) studies, based on the RCT-DUPLICATE initiative (Randomised, Controlled Trials Duplicated Using Prospective Longitudinal Insurance Claims: Applying Techniques of Epidemiology).DesignMeta-analysis of RCT-DUPLICATE data.Data sourcesTrials included in RCT-DUPLICATE, a demonstration project that emulated 32 randomised controlled trials using three real world data sources: Optum Clinformatics Data Mart, 2004-19; IBM MarketScan, 2003-17; and subsets of Medicare parts A, B, and D, 2009-17.Eligibility criteria for selecting studiesTrials where the primary analysis resulted in a hazard ratio; 29 RCT-RWE study pairs from RCT-DUPLICATE.ResultsDifferences and variation in effect sizes between the results from randomised controlled trials and real world evidence studies were investigated. Most of the heterogeneity in effect estimates between the RCT-RWE study pairs in this sample could be explained by three emulation differences in the meta-regression model: treatment started in hospital (which does not appear in health insurance claims data), discontinuation of some baseline treatments at randomisation (which would have been an unusual care decision in clinical practice), and delayed onset of drug effects (which would be under-reported in real world clinical practice because of the relatively short persistence of the treatment). Adding the three emulation differences to the meta-regression reduced heterogeneity from 1.9 to almost 1 (absence of heterogeneity).ConclusionsThis analysis suggests that a substantial proportion of the observed variation between results from randomised controlled trials and real world evidence studies can be attributed to differences in design emulation.
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Audisio, Katia, Hillary Lia, Newell Bryce Robinson, Mohamed Rahouma, Giovanni Soletti, Gianmarco Cancelli, Roberto Perezgrovas Olaria et al. „Impact of the COVID-19 Pandemic on Non-COVID-19 Clinical Trials“. Journal of Cardiovascular Development and Disease 9, Nr. 1 (10.01.2022): 19. http://dx.doi.org/10.3390/jcdd9010019.
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Randomized controlled trials (RCT) were impacted by the COVID-19 pandemic, but no systematic analysis has evaluated the overall impact of COVID-19 on non-COVID-19-related RCTs. The ClinicalTrials.gov database was queried in February 2020. Eligible studies included all randomized trials with a start date after 1 January 2010 and were active during the period from 1 January 2015 to 31 December 2020. The effect of the pandemic period on non-COVID-19 trials was determined by piece-wise regression models using 11 March 2020 as the start of the pandemic and by time series analysis (models fitted using 2015–2018 data and forecasted for 2019–2020). The study endpoints were early trial stoppage, normal trial completion, and trial activation. There were 161,377 non-COVID-19 trials analyzed. The number of active trials increased annually through 2019 but decreased in 2020. According to the piece-wise regression models, trial completion was not affected by the pandemic (p = 0.56) whereas trial stoppage increased (p = 0.001). There was a pronounced decrease in trial activation early during the pandemic (p < 0.001) which then recovered. The findings from the time series models were consistent comparing forecasted and observed results (trial completion p = 0.22; trial stoppage p < 0.01; trial activation, p = 0.01). During the pandemic, there was an increase in non-COVID-19 RCTs stoppage without changes in RCT completion. There was a sharp decline in new RCTs at the beginning of the pandemic, which later recovered.
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SINGH, JASVINDER A., STEPHEN MURPHY und MOHIT BHANDARI. „Assessment of the Methodologic Quality of Medical and Surgical Clinical Trials in Patients with Arthroplasty“. Journal of Rheumatology 36, Nr. 12 (02.11.2009): 2642–54. http://dx.doi.org/10.3899/jrheum.090333.
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Objective.To assess the methodological quality of randomized controlled trials (RCT) of medical and surgical therapy in patients with arthroplasty.Methods.We conducted a Medline database search for all arthroplasty RCT from 1997 and 2006. The quality of the methods of all eligible RCT was assessed by a trained abstractor. We used a checklist of trial quality characteristics, and the overall trial quality was assessed by 3 scales: Jadad (range 0–5), Delphi list (range 0–9), and numeric rating scale (NRS; range 1–10), based on User’s Guides to the Medical Literature.Results.A total of 196 articles were included in the analysis; most included hip (n = 81) or knee (n = 80) or both hip/knee arthroplasty (n = 19); 66 (34%) assessed pharmacological treatments, 117 (60%) nonpharmacological treatments, and 13 (7%) both. Mean (SEM) overall quality scores of arthroplasty RCT were low: Jadad score 2.36 (1.4), Delphi list 5.33 (1.6), and NRS score 4.30 (2.6). Multivariable analyses revealed that nonpharmacological intervention RCT had lower odds (odds ratio 0.28–0.39; p = 0.008–0.033) and those with no funding had lower odds (OR 0.28–0.50; p = 0.014–0.119) of being in the highest quartiles of the 3 overall quality scores. In contrast, multicenter RCT had 1.8–4.7 times higher odds of being in highest tertiles of quality scores (p = 0.017–0.185).Conclusion.Methodological deficiencies in reporting of hip/knee arthroplasty RCT offer an opportunity for improvement. Type of intervention, number of trial centers, and presence of funding were independently associated with overall trial quality. In future, multicenter RCT (rather than single-center) and modeling protocols of single-center RCT similar in rigor to multicenter RCT may improve the quality of arthroplasty RCT.
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Tarrant, Mark, Mary Carter, Sarah Gerard Dean, Rod Taylor, Fiona C. Warren, Anne Spencer, Jane Adamson et al. „Singing for people with aphasia (SPA): results of a pilot feasibility randomised controlled trial of a group singing intervention investigating acceptability and feasibility“. BMJ Open 11, Nr. 1 (Januar 2021): e040544. http://dx.doi.org/10.1136/bmjopen-2020-040544.
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ObjectivesPilot feasibility randomised controlled trial (RCT) for the singing groups for people with aphasia (SPA) intervention to assess: (1) the acceptability and feasibility of participant recruitment, randomisation and allocation concealment; (2) retention rates; (3) variance of continuous outcome measures; (4) outcome measure completion and participant burden; (5) fidelity of intervention delivery; (6) SPA intervention costs; (7) acceptability and feasibility of trial and intervention to participants and others involved.DesignA two-group, assessor-blinded, randomised controlled external pilot trial with parallel mixed methods process evaluation and economic evaluation.SettingThree community-based cohorts in the South-West of England.ParticipantsEligible participants with post-stroke aphasia were randomised 1:1 to SPA or control.InterventionThe manualised SPA intervention was delivered over 10 weekly singing group sessions, led by a music facilitator and assisted by an individual with post-stroke aphasia. The intervention was developed using the Information-Motivation-Behavioural skills model of behaviour change and targeted psychosocial outcomes. Control and intervention participants all received an aphasia information resource pack.Outcome measuresCollected at baseline, 3 and 6 months post-randomisation, candidate primary outcomes were measured (well-being, quality of life and social participation) as well as additional clinical outcomes. Feasibility, acceptability and process outcomes included recruitment and retention rates, and measurement burden; and trial experiences were explored in qualitative interviews.ResultsOf 87 individuals screened, 42 participants were recruited and 41 randomised (SPA=20, control=21); 36 participants (SPA=17, control=19) completed 3-month follow-up, 34 (SPA=18, control=16) completed 6-month follow-up. Recruitment and retention (83%) were acceptable for a definitive RCT, and participants did not find the study requirements burdensome. High fidelity of the intervention delivery was shown by high attendance rates and facilitator adherence to the manual, and participants found SPA acceptable. Sample size estimates for a definitive RCT and primary/secondary outcomes were identified.ConclusionsThe SPA pilot RCT fulfilled its objectives, and demonstrated that a definitive RCT of the intervention would be both feasible and acceptable.Trial registration numberNCT03076736.
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Magro, Elsa, Jean-Christophe Gentric, André Lima Batista, Marc Kotowski, Chiraz Chaalala, David Roberge, Alain Weill et al. „The Treatment of Brain AVMs Study (TOBAS): an all-inclusive framework to integrate clinical care and research“. Journal of Neurosurgery 128, Nr. 6 (Juni 2018): 1823–29. http://dx.doi.org/10.3171/2017.2.jns162751.
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OBJECTIVEThe management of brain arteriovenous malformations (bAVMs) remains controversial. The Treatment of Brain AVMs Study (TOBAS) was designed to manage patients with bAVMs within a clinical research framework. The objective of this study was to study trial feasibility, recruitment rates, patient allocation to the various management groups, and compliance with treatment allocation.METHODSTOBAS combines two randomized care trials (RCTs) and a registry. Designed to be all-inclusive, the study offers randomized allocation of interventional versus conservative management to patients eligible for both options (first RCT), a second RCT testing the role of preembolization as an adjunct to surgery or radiotherapy, and a registry of patients managed using clinical judgment alone. The primary outcome of the first RCT is death from any cause or disabling stroke (modified Rankin Scale score > 2) at 10 years. A pilot phase was initiated at one center to test study feasibility, record the number and characteristics of patients enrolled in the RCTs, and estimate the frequency of crossovers.RESULTSAll patients discussed at the multidisciplinary bAVM committee between June 2014 and June 2016 (n = 107) were recruited into the study; 46 in the randomized trials (23 in the first RCT with 21 unruptured bAVMs, 40 in the second RCT with 17 unruptured bAVMs, and 17 in both RCTs), and 61 patients in the registry. Three patients crossed over from surgery to observation (first RCT).CONCLUSIONSClinical research was successfully integrated with normal practice using TOBAS. Recruitment rates in a single center are encouraging. Whether the trial will provide meaningful results depends on the recruitment of a sufficient number of participating centers.Clinical trial registration no.: NCT02098252 (clinicaltrials.gov)
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Coon, Eric R., Corrie E. McDaniel, Natalia Paciorkowski, Meg Grimshaw, Elizabeth Frakes, Lilliam Ambroggio, Katherine A. Auger et al. „Prioritization of Randomized Clinical Trial Questions for Children Hospitalized With Common Conditions“. JAMA Network Open 7, Nr. 5 (15.05.2024): e2411259. http://dx.doi.org/10.1001/jamanetworkopen.2024.11259.
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ImportanceThere is a lack of randomized clinical trial (RCT) data to guide many routine decisions in the care of children hospitalized for common conditions. A first step in addressing the shortage of RCTs for this population is to identify the most pressing RCT questions for children hospitalized with common conditions.ObjectiveTo identify the most important and feasible RCT questions for children hospitalized with common conditions.Design, Setting, and ParticipantsFor this consensus statement, a 3-stage modified Delphi process was used in a virtual conference series spanning January 1 to September 29, 2022. Forty-six individuals from 30 different institutions participated in the process. Stage 1 involved construction of RCT questions for the 10 most common pediatric conditions leading to hospitalization. Participants used condition-specific guidelines and reviews from a structured literature search to inform their development of RCT questions. During stage 2, RCT questions were refined and scored according to importance. Stage 3 incorporated public comment and feasibility with the prioritization of RCT questions.Main Outcomes and MeasuresThe main outcome was RCT questions framed in a PICO (population, intervention, control, and outcome) format and ranked according to importance and feasibility; score choices ranged from 1 to 9, with higher scores indicating greater importance and feasibility.ResultsForty-six individuals (38 who shared demographic data; 24 women [63%]) from 30 different institutions participated in our modified Delphi process. Participants included children’s hospital (n = 14) and community hospital (n = 13) pediatricians, parents of hospitalized children (n = 4), other clinicians (n = 2), biostatisticians (n = 2), and other researchers (n = 11). The process yielded 62 unique RCT questions, most of which are pragmatic, comparing interventions in widespread use for which definitive effectiveness data are lacking. Overall scores for importance and feasibility of the RCT questions ranged from 1 to 9, with a median of 5 (IQR, 4-7). Six of the top 10 selected questions focused on determining optimal antibiotic regimens for 3 common infections (pneumonia, urinary tract infection, and cellulitis).Conclusions and RelevanceThis consensus statementhas identified the most important and feasible RCT questions for children hospitalized with common conditions. This list of RCT questions can guide investigators and funders in conducting impactful trials to improve care and outcomes for hospitalized children.
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McCrann, Saoirse, Ian Flitcroft, Niall C. Strang, Kathryn J. Saunders, Nicola S. Logan, Samantha Szeyee Lee, David A. Mackey, John S. Butler und James Loughman. „Myopia Outcome Study of Atropine in Children (MOSAIC): an investigator-led, double-masked, placebo-controlled, randomised clinical trial protocol“. HRB Open Research 2 (25.09.2019): 15. http://dx.doi.org/10.12688/hrbopenres.12914.2.
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Background: The Myopia Outcome Study of Atropine in Children (MOSAIC) aims to explore the efficacy, safety, acceptability and mechanisms of action of 0.01% unpreserved atropine for myopia control in a European population. Methods: MOSAIC is an investigator-led, double-masked, placebo-controlled, randomised clinical trial (RCT) investigating the efficacy, safety and mechanisms of action of 0.01% atropine for managing progression of myopia. During Phase 1 of the trial, 250 children aged 6-16 years with progressive myopia instil eye drops once nightly in both eyes from randomisation to month 24. From month 24 to 36 participants are re-randomised in Phase 2 of the trial, into continued 0.01% atropine, and washout, at 1:1 ratio for those participants initially randomised to the intervention arm (n=167), during which any potential rebound effects on cessation of treatment will be monitored. All participants initially assigned to the placebo (n=83) crossover to the intervention arm of the study for Phase 2, and from month 24 to 36, instil 0.01% atropine eye drops in both eyes once nightly. Further treatment and monitoring beyond 36 months is planned (Phase 3) and will be designed dependent on the outcomes of Phase 1. Results: The primary outcome measure is cycloplegic spherical equivalent refractive error progression at 24 months. Secondary outcome measures include axial length change as well as the rebound, safety and acceptability profile of 0.01% atropine. Additional analyses will include the mechanisms of action of 0.01% atropine for myopia control. Conclusions: The generalisability of results from previous clinical trials investigating atropine for myopia control is limited by the predominantly Asian ethnicity of previous study populations. MOSAIC is the first RCT to explore the efficacy, safety and mechanisms of action of unpreserved 0.01% atropine in a predominantly White population.
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Apte, Sameer S., Husein Moloo, Ahwon Jeong, Michelle Liu, Lisa Vandemeer, Kathryn Suh, Kednapa Thavorn, Dean A. Fergusson, Mark Clemons und Rebecca C. Auer. „Prospective randomised controlled trial using the REthinking Clinical Trials (REaCT) platform and National Surgical Quality Improvement Program (NSQIP) to compare no preparation versus preoperative oral antibiotics alone for surgical site infection rates in elective colon surgery: a protocol“. BMJ Open 10, Nr. 7 (Juli 2020): e036866. http://dx.doi.org/10.1136/bmjopen-2020-036866.
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IntroductionDespite 40 randomised controlled trials (RCTs) investigating preoperative oral antibiotics (OA) and mechanical bowel preparation (MBP) to reduce surgical site infection (SSI) rate following colon surgery, there has never been an RCT published comparing OA alone versus no preparation. Of the four possible regimens (OA alone, MBP alone, OA plus MBP and no preparation), randomised evidence is conflicting for studied groups. Furthermore, guidelines vary, with recommendations for OA alone, OA plus MBP or no preparation. The National Surgical Quality Improvement Program (NSQIP) has automated data collection for surgical patients. Similarly, the ‘REthinking Clinical Trials’ (REaCT) platform increases RCT enrolment by simplifying pragmatic trial design. In this novel RCT protocol, we combine REaCT and NSQIP to compare OA alone versus no preparation for SSI rate reduction in elective colon surgery. To our knowledge, this is the first published RCT protocol that leverages NSQIP for data collection. In our feasibility study, 67 of 74 eligible patients (90%) were enrolled and 63 of 67 (94%) were adherent to protocol. The ‘REaCT–NSQIP’ trial design has great potential to efficiently generate level I evidence for other perioperative interventions.Methods and analysisSSI rates following elective colorectal surgery after preoperative OA or no preparation will be compared. We predict 45% relative rate reduction of SSI, improvement in length of stay, reduced costs and increased quality of life, with similar antibiotic-related complications. Consent, using the ‘integrated consent model’, and randomisation on a mobile device are completed by the surgeon in a single clinical encounter. Data collection for the primary end point is automatic through NSQIP. Analysis of cost per weighted case, cost utility and quality-adjusted life years will be done.Ethics and disseminationThis study is approved by The Ontario Cancer Research Ethics Board. Results will be disseminated in surgical conferences and peer-reviewed journals.Trial registration numberNCT03663504; Pre-results, recruitment phase.
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Badian, Reza A., Brendan McCormack und Vibeke Sundling. „Person-Centered Research: A novel approach to Randomized Controlled Trials“. European Journal for Person Centered Healthcare 6, Nr. 2 (01.06.2018): 209. http://dx.doi.org/10.5750/ejpch.v6i2.1435.
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Introduction: Integrating person-centered values with randomized controlled trials methodology is a novel idea. Person-centeredness is gaining steadily more prominence and attention in healthcare and health-related policy and research. Randomized controlled trials are considered as the gold standard in evidence-based medicine for evaluating the effects of treatment or determining the causal effect. A wide array of study designs is available, but there is a lack of designs with both strong person-centered principles and a strong position with respect to the level of evidence. In this paper we intend to introduce a novel design to fill such a gap.Aims and objectives: The aim of this paper is to introduce a novel study design where essential values of person-centered care (PCC) are integrated with randomized controlled trial (RCT) methodology into a novel study design termed a person-centered randomized controlled trial (PC-RCT).Methods: In this paper we discuss the importance and role of evidence in clinical research, levels of evidence, as well as the significance of study design in evidence-based medicine. Moreover, we discuss randomized controlled trials that are considered the gold standard to achieve high quality evidence. In this paper we will explain what the concept of person-centered care is and discuss the values associated with person-centeredness.The theoretical and methodological considerations that are relevant in applying this concept will be discussed before presenting how we intend to incorporate person-centered values into a randomized controlled trial in a novel study design that is both person-centered and randomized controlled (PC-RCT). Different aspects of this proposed novel study design will be discussed, including the theory and methods underlying this new proposed design, its novelty, different stages and practical steps involved in this proposed design. Challenges, drawbacks and possible solutions for addressing challenges of this novel design will be explored, focusing on the construct, dynamics, advantages, disadvantages and novelty of PC-RCT design.Conclusion: This paper presents how person-centered values and traditional randomised controlled trial principal values are integrated into one study design where the strengths of both concepts are merged into one. The proposed novel study design has stronger person-centered characteristics and is solid in its RCT features. This design ensures that participants have much more active participation in decision-making and gain more choice in their treatment. The proposed novel study design in this paper has clearly an important role to play in satisfying the need for a study design that can address both the need for rendering higher levels of evidence as well as simultaneously securing greater integration of person-centered values in the same study design.
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PAI, N., J. Popov, E. Hartung, L. Hill, K. Grzywacz, D. Godin, L. Thabane, C. Lee, M. Surette und P. Moayyedi. „P375 Feasibility of the first paediatric randomised controlled pilot trial of faecal microbiota transplant for ulcerative colitis“. Journal of Crohn's and Colitis 14, Supplement_1 (Januar 2020): S353—S354. http://dx.doi.org/10.1093/ecco-jcc/jjz203.504.
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Abstract Background The first multicentre paediatric randomised-controlled trial (RCT) of faecal microbiota transplant (FMT) in ulcerative colitis (UC) was recently completed in Ontario and Quebec, Canada. The use of FMT in paediatric UC treatment has been limited to small, open-label case series. Unique clinical and patient factors necessitated a paediatric-specific pilot RCT. Our aim was to assess key feasibility measures of conducting an FMT trial in paediatric patients to inform design, and feasibility of larger, future multicentre studies. Methods Patients were randomised (1:1) to receive rectal enemas 2×/week for 6 weeks, followed by a 6-month follow-up period. Enemas contained normal saline (placebo), or RBX2660 (Rebiotix; Roseville, USA), a faecal microbiota preparation of stool from healthy adult donors (active). Patients were eligible for the open-label extension following completion or withdrawal from the placebo arm. Study feasibility was assessed through patient eligibility, recruitment, completion of sample collections, adverse events, and hospitalisations. Results Over 36 months across two tertiary IBD clinics, 48 paediatric patients were screened for participation. 75.0% (36) met eligibility criteria for enrolment and 69.4% (25) participated in the randomised trial. 53.8% (7) of patients randomised to the placebo arm entered the open-label arm. We stratified completion of sample collections by intervention (Weeks 0–6), and follow-up phases of the trial (Weeks 6–30). 75.3% of required bloodwork was obtained during both phases; patient-provided stool samples declined from 76.0% (intervention) to 45% (follow-up). 83.0% (10) reported adverse events in the active arm, and 38.4% (5) in placebo; these were all minor and self-limited (muscle pain, cough, fatigue, etc.). No patients experienced adverse events related to enemas. Five patients experienced serious adverse events: IBD flare (2 active, 1 placebo), and reactivation of C. difficile infection (2 active) (Table 1). Conclusion In this first paediatric RCT of FMT for UC, we established important feasibility data regarding the design of FMT trials in children. Patient acceptance of FMT was high, and well-tolerated with minor, self-limited adverse events, and few serious adverse events. Return-rates of stool samples were low during follow-up; strategies to improve uptake are needed, particularly given the value of microbiome data for analyses. Overall, our study demonstrates strong interest and tolerance among paediatric patients for FMT-based trials. Future studies will build upon our protocol to enhance clinical data on the effects of microbiota-based therapeutics in children.
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De Neubourg, Diane, Lara Janssens, Iris Verhaegen, Elke Smits, Ben W. Mol und Ella Roelant. „Live birth after additional tubal flushing with oil-based contrast versus no additional flushing: a randomised, multicentre, parallel-group pragmatic trial in infertile women with at least one patent tube at hysterosalpingo-foam sonography (HYFOIL study)“. BMJ Open 11, Nr. 11 (November 2021): e054845. http://dx.doi.org/10.1136/bmjopen-2021-054845.
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IntroductionTubal patency testing is an essential part in the fertility workup of many subfertile women. Hysterosalpingography (HSG) has long been the test of choice in many clinics. There is evidence from a large randomised multicentre trial and from a recent meta-analysis that women who had HSG using oil soluble contrast medium (OSCM) had higher rates of ongoing pregnancy compared with women who underwent this procedure using water contrast. However, the field is moving away from HSG and nowadays hysterosalpingo-foam sonography (Hyfosy) using ultrasound guidance is considered as the first line office tubal patency test. Therefore, a large multicentre randomised clinical trial (RCT) will be initiated to evaluate if flushing the fallopian tubes with OSCM after a normal Hyfosy showing at least one patent fallopian tube will increase the live birth rate as compared with no flushing.Methods and analysisWe plan a multicentre two arm, 1:1 randomised, open-label pragmatic comparative trial in 12 Belgian centres. After informed consent, we will randomise infertile women between 18 and 40 years of age, undergoing Hyfosy as part of the fertility workup to Hyfosy with additional tubal flushing with OSCM versus Hyfosy without additional flushing. Infertility is defined as lack of conception despite 12 months of unprotected intercourse, or three cycles of donor insemination without pregnancy or three ovulatory ovulation induction cycles without pregnancy, all three in combination with at least one patent tube on Hyfosy. Primary endpoint will be live birth with conception within 6 months after randomisation.Ethics and disseminationApproval on 11 May 2021 by the Ethics Committee from ZNA Hospital Antwerp who was the central Ethics Committee for the Clinical Trial Regulation Pilot (Pilot 412) in the 12 centres. The findings from this RCT will be disseminated in peer-reviewed publications and presentations at scientific international meetings.Trial registration numbersEudraCT number: 2020-002135-30 and NCT04379973.
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Hill, L., J. Popov, E. Hartung, M. Moshkovich, M. Figueiredo und N. Pai. „P517 A randomised, placebo-controlled pilot trial of faecal microbiota transplantation for paediatric Crohn’s disease“. Journal of Crohn's and Colitis 14, Supplement_1 (Januar 2020): S447—S448. http://dx.doi.org/10.1093/ecco-jcc/jjz203.645.
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Abstract Background The role of faecal microbiota transplant (FMT) in Crohn’s disease (CD) remains unclear. Small, open-label case series have shown high rates of clinical remission but protocols have varied across studies, and no randomised controlled trials (RCT) have been performed. We present a protocol for the first pilot RCT of FMT in paediatric CD patients, using a novel colonoscopic + oral capsular intervention that uses fresh-frozen and prepared, lyophilised donor stool. We will measure improvements in clinical disease activity, inflammatory biomarkers, endoscopic markers of mucosal inflammation, as well as, assess key aspects of trial feasibility. Methods Patients 3–17 years with active CD, on stable medication doses for 4 weeks are eligible. Patients will have an initial colonoscopy during which they will receive an infusion into the terminal ileum of normal saline (placebo) or prepared healthy donor stool (RBX2660; Rebiotix, USA) (active). This will be followed by 6-weeks of bi-weekly oral capsular therapy, containing methylcellulose (placebo) or lyophilised healthy adult donor stool (RBX7455; Rebiotix, USA). Randomisation is 2:1 to active and placebo arms (n = 45). Patients will be followed over 24 weeks. (Figure 1) Results Study feasibility will be assessed by: rate of recruitment, completion of sample collections, and frequency and type of adverse events. Clinical outcomes will be measured serially using: Paediatric Crohn’s Disease Activity Index (PCDAI), faecal calprotectin, blood inflammatory markers, and Simple Endoscopic Score for Crohn’s Disease (SES-CD) at baseline and end-of-study. Stool 16s rRNA, metagenomics, and urine metabolomics profiles will be performed on collected stool and urine samples. Patients who complete the placebo arm will be eligible to enter an open-label extension study. Results will be measured through ITT and PP analyses. Proportions and percentages will be reported on feasibility outcomes; odds ratios, mean differences and 95% confidence intervals will be reported on clinical outcomes as preliminary estimates of efficacy. Conclusion This is the first pilot RCT of FMT in the treatment of CD. Recruitment commenced April 2019 and 13 patients have met screening criteria, with 5 enrolled thus far. This study is novel for its focus on paediatric CD patients, and its use of a combined oral + colonoscopic FMT delivery method. The results of this trial will offer preliminary estimates of efficacy for FMT-based therapies in CD, and may support expansion to a future larger multicenter paediatric RCT using our validated study protocol.
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Griffin, Damian, Peter Wall, Alba Realpe, Ann Adams, Nick Parsons, Rachel Hobson, Juul Achten et al. „UK FASHIoN: feasibility study of a randomised controlled trial of arthroscopic surgery for hip impingement compared with best conservative care“. Health Technology Assessment 20, Nr. 32 (April 2016): 1–172. http://dx.doi.org/10.3310/hta20320.
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BackgroundFemoroacetabular impingement (FAI) is a syndrome of hip or groin pain associated with shape abnormalities of the hip joint. Treatments include arthroscopic surgery and conservative care. This study explored the feasibility of a randomised controlled trial to compare these treatments.ObjectivesThe objectives of this study were to estimate the number of patients available for a full randomised controlled trial (RCT); to explore clinician and patient willingness to participate in such a RCT; to develop consensus on eligibility criteria, surgical and best conservative care protocols; to examine possible outcome measures and estimate the sample size for a full RCT; and to develop trial procedures and estimate recruitment and follow-up rates.MethodsPre-pilot work: we surveyed all UK NHS hospital trusts (n = 197) to identify all FAI surgeons and to estimate how much arthroscopic FAI surgery they performed. We interviewed a purposive sample of 18 patients, 36 physiotherapists, 18 surgeons and two sports physicians to explore attitudes towards a RCT and used consensus-building methods among them to develop treatment protocols and patient information. Pilot RCT: we performed a pilot RCT in 10 hospital trusts. Patients were randomised to receive either hip arthroscopy or best conservative care and then followed up at 3, 6 and 12 months using patient-reported questionnaires for hip pain and function, activity level, quality of life, and a resource-use questionnaire. Qualitative recruitment intervention: we performed semistructured interviews with all researchers and clinicians involved in the pilot RCT in eight hospital trusts and recorded and analysed diagnostic and recruitment consultations with eligible patients.ResultsWe identified 120 surgeons who reported treating at least 1908 patients with FAI by hip arthroscopy in the NHS in the financial year 2011/12. There were 34 hospital trusts that performed ≥ 20 arthroscopic FAI operations in the year. We found that clinicians were positive about a RCT: only half reported equipoise, but most said that they would be prepared to randomise patients. Patients strongly supported a RCT, but expressed concerns about its design; these were used to develop patient information for the pilot RCT. We developed a surgical protocol and showed that this could be used in a RCT. We developed a physiotherapy-led exercise-based package of best conservative care called ‘personalised hip therapy’ and showed that this was practicable. In the pilot RCT, we recruited 42 out of 60 eligible patients (70%) across nine sites. The mean duration and recruitment rate across all sites were 4.5 months and one patient per site per month, respectively. The lead site recruited for the longest period (9.3 months) and accrued the largest number of patients (2.1 patients per month). We recorded and analysed 84 diagnostic and recruitment consultations in 60 patients and used these to develop a model for an optimal recruitment consultation. We identified the International Hip Outcome Tool at 12 months as an appropriate outcome measure and estimated the sample size for a full trial as 344 participants: a number that could be recruited in 25 centres over 18 months.ConclusionWe have demonstrated that it is feasible to perform a RCT to establish the clinical effectiveness of hip arthroscopy compared with best conservative care for FAI. We have designed a full trial and developed and tested procedures for it, including an innovative approach to recruitment. We propose that a full trial be implemented.FundingThe National Institute for Health Research Health Technology Assessment programme.
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Smeets, Annemie, Feryal Ghafelzadeh Ahwaz, Stijn Bogaerts, An De Groef, Pieter Berger, Jean-François Kaux, Christophe Daniel et al. „Pilot study to investigate the feasibility of conducting a randomised controlled trial that compares Immediate versus Optional Delayed surgical repair for treatment of acute Anterior cruciate ligament injury: IODA pilot trial“. BMJ Open 12, Nr. 3 (März 2022): e055349. http://dx.doi.org/10.1136/bmjopen-2021-055349.
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IntroductionStandard care for anterior cruciate ligament (ACL) injuries includes surgical reconstruction of the ACL. However, two randomised controlled trials (RCTs) concluded that conservative treatment does not result in inferior clinical outcomes compared with immediate ACL reconstruction. More research is needed to in the first place verify these results, and second to assess whether patient-specific parameters determine whether a patient would benefit from one treatment option over the other. However, before running a full RCT, it seems necessary to perform a pilot study that assesses the feasibility of recruiting patients with ACL for such a RCT. This is because recruitment may be challenging as many patients have strong treatment beliefs. Therefore, this pilot study will assess whether a large RCT is feasible with regard to participant recruitment, adherence to the allocated treatment arm and protocol feasibility. These pilot findings will help deciding about progressing to a future full RCT.Methods and analysisThis is a pragmatic, multicentre, randomised controlled pilot trial with two parallel groups. Patients with an acute ACL injury will be recruited from two Belgian hospitals. Patients will be randomised to either conservative treatment or surgical treatment. Patients will be followed-up at 3, 6 and 12 months postrandomisation. Recruitment feasibility will be evaluated by calculating the recruitment rate 4 months after the two sites have been initiated. Clear criteria for progression to a full trial are defined. Adherence to the protocol will be assessed by calculating the proportion of patients who complete the assessments. Furthermore the proportion of patients who cross-over between treatment arms during the follow-up period will be assessed.Ethics and disseminationThe study was approved by the ethical committees: Ethische Commissie Onderzoek UZ/KU Leuven (S62004) and Comité d’Ethique Hospitalo-Facultaire Universitaire de Liège (2020212). Results will be made available to caregivers, researchers and funder.Trial registration numberThis trial is registered on ClinicalTrials.gov (NCT04408690) on 29 May 2020.
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Palmer, Shea, Fiona Cramp, Emma Clark, Rachel Lewis, Sara Brookes, William Hollingworth, Nicky Welton et al. „The feasibility of a randomised controlled trial of physiotherapy for adults with joint hypermobility syndrome“. Health Technology Assessment 20, Nr. 47 (Juni 2016): 1–264. http://dx.doi.org/10.3310/hta20470.
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BackgroundJoint hypermobility syndrome (JHS) is a heritable disorder associated with laxity and pain in multiple joints. Physiotherapy is the mainstay of treatment, but there is little research investigating its clinical effectiveness.ObjectivesTo develop a comprehensive physiotherapy intervention for adults with JHS; to pilot the intervention; and to conduct a pilot randomised controlled trial (RCT) to determine the feasibility of conducting a future definitive RCT.DesignPatients’ and health professionals’ perspectives on physiotherapy for JHS were explored in focus groups (stage 1). A working group of patient research partners, clinicians and researchers used this information to develop the physiotherapy intervention. This was piloted and refined on the basis of patients’ and physiotherapists’ feedback (stage 2). A parallel two-arm pilot RCT compared ‘advice’ with ‘advice and physiotherapy’ (stage 3). Random allocation was via an automated randomisation service, devised specifically for the study. Owing to the nature of the interventions, it was not possible to blind clinicians or patients to treatment allocation.SettingStage 1 – focus groups were conducted in four UK locations. Stages 2 and 3 – piloting of the intervention and the pilot RCT were conducted in two UK secondary care NHS trusts.ParticipantsStage 1 – patient focus group participants (n = 25, three men) were aged > 18 years, had a JHS diagnosis and had received physiotherapy within the preceding 12 months. The health professional focus group participants (n = 16, three men; 14 physiotherapists, two podiatrists) had experience of managing JHS. Stage 2 – patient participants (n = 8) were aged > 18 years, had a JHS diagnosis and no other musculoskeletal conditions causing pain. Stage 3 – patient participants for the pilot RCT (n = 29) were as for stage 2 but the lower age limit was 16 years.InterventionFor the pilot RCT (stage 3) the advice intervention was a one-off session, supplemented by advice booklets. All participants could ask questions specific to their circumstances and receive tailored advice. Participants were randomly allocated to ‘advice’ (no further advice or physiotherapy) or ‘advice and physiotherapy’ (an additional six 30-minute sessions over 4 months). The physiotherapy intervention was supported by a patient handbook and was delivered on a one-to-one patient–therapist basis. It aimed to increase patients’ physical activity through developing knowledge, understanding and skills to better manage their condition.Main outcome measuresData from patient and health professional focus groups formed the main outcome from stage 1. Patient and physiotherapist interview data also formed a major component of stages 2 and 3. The primary outcome in stage 3 related to the feasibility of a future definitive RCT [number of referrals, recruitment and retention rates, and an estimate of the value of information (VOI) of a future RCT]. Secondary outcomes included clinical measures (physical function, pain, global status, self-reported joint count, quality of life, exercise self-efficacy and adverse events) and resource use (to estimate cost-effectiveness). Outcomes were recorded at baseline, 4 months and 7 months.ResultsStage 1 – JHS is complex and unpredictable. Physiotherapists should take a long-term holistic approach rather than treating acutely painful joints in isolation. Stage 2 – a user-informed physiotherapy intervention was developed and evaluated positively. Stage 3 – recruitment to the pilot RCT was challenging, primarily because of a perceived lack of equipoise between advice and physiotherapy. The qualitative evaluation provided very clear guidance to inform a future RCT, including enhancement of the advice intervention. Some patients reported that the advice intervention was useful and the physiotherapy intervention was again evaluated very positively. The rate of return of questionnaires was low in the advice group but reasonable in the physiotherapy group. The physiotherapy intervention showed evidence of promise in terms of primary and secondary clinical outcomes. The advice arm experienced more adverse events. The VOI analysis indicated the potential for high value from a future RCT. Such a trial should form the basis of future research efforts.ConclusionA future definitive RCT of physiotherapy for JHS seems feasible, although the advice intervention should be made more robust to address perceived equipoise and subsequent attrition.Trial registrationCurrent Controlled Trials ISRCTN29874209.FundingThis project was funded by the National Institute for Health Research (NIHR) Health Technology Assessment programme and will be published in full inHealth Technology Assessment; Vol. 20, No. 47. See the NIHR Journals Library website for further project information.
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Nixon, Andrew C., Theodoros M. Bampouras, Helen J. Gooch, Hannah M. L. Young, Kenneth W. Finlayson, Neil Pendleton, Sandip Mitra, Mark E. Brady und Ajay P. Dhaygude. „Home-based exercise for people living with frailty and chronic kidney disease: A mixed-methods pilot randomised controlled trial“. PLOS ONE 16, Nr. 7 (01.07.2021): e0251652. http://dx.doi.org/10.1371/journal.pone.0251652.
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Background Frailty is associated with adverse health outcomes in people with chronic kidney disease (CKD). Evidence supporting targeted interventions is needed. This pilot randomised controlled trial (RCT) aimed to inform the design of a definitive RCT evaluating the effectiveness of a home-based exercise intervention for pre-frail and frail older adults with CKD. Methods Participants were recruited from nephrology outpatient clinics to this two-arm parallel group mixed-methods pilot RCT. Inclusion criteria were: ≥65 years old; CKD G3b-5; and Clinical Frailty Scale score ≥4. Participants categorised as pre-frail or frail using the Frailty Phenotype were randomised to a 12-week progressive multi-component home-based exercise programme or usual care. Primary outcome measures included eligibility, recruitment, adherence, outcome measure completion and participant attrition rate. Semi-structured interviews were conducted with participants to explore trial and intervention acceptability. Results Six hundred and sixty-five patients had an eligibility assessment with 217 (33%; 95% CI 29, 36) eligible. Thirty-five (16%; 95% CI 12, 22) participants were recruited. Six were categorised as robust and withdrawn prior to randomisation. Fifteen participants were randomised to exercise and 14 to usual care. Eleven (73%; 95% CI 45, 91) participants completed ≥2 exercise sessions/week. Retained participants completed all outcome measures (n = 21; 100%; 95% CI 81, 100). Eight (28%; 95% CI 13, 47) participants were withdrawn. Fifteen participated in interviews. Decision to participate/withdraw was influenced by perceived risk of exercise worsening symptoms. Participant perceived benefits included improved fitness, balance, strength, well-being, energy levels and confidence. Conclusions This pilot RCT demonstrates that progression to definitive RCT is possible provided recruitment and retention challenges are addressed. It has also provided preliminary evidence that home-based exercise may be beneficial for people living with frailty and CKD. Trial registration ISRCTN87708989; https://clinicaltrials.gov/.
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John, Joseph, und Naveen Kumar I. „Blinding Induced Risk of Bias in Randomized Controlled Trials of Physiotherapy Interventions — A Retrospective Study“. JOURNAL OF CLINICAL AND BIOMEDICAL SCIENCES 13, Nr. 4 (28.12.2023): 108–14. http://dx.doi.org/10.58739/jcbs/v13i4.23.2.
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Introduction: In randomized clinical trials, the methodological idea of preventing bias by withholding knowledge of the allocation status is known as blinding. Blinding refers back to the concealment of group allocation from one or extra individuals concerned in scientific studies, most commonly a randomized controlled trial (RCT). Even though randomization minimizes variations among treatment groups on the outset of the trial, it does nothing to prevent differential treatment of the groups later within the trial or the differential assessment of outcomes, either of which may also bring about biased estimates of treatment outcomes. The most beneficial strategy to limit the chance of differential remedy or assessments of results is to blind as many individuals as viable in a trial. Objective: To study the extent of blinding induced bias in RCT of physiotherapy interventions and to evaluate the extent of interpretative consideration it the trials are not blinded in the sample of RCT included. Methodology : We conducted a retrospective analysis to estimate the blinding bias in the randomized controlled trials published in physiotherapy interventions from 2016 to 2022. Results & Conclusion : We included 50 RCTs for blinding assessment. About 88% of included articles were not having participants blinding 90% has not done therapist blinding and nearly 50% of studies were conducted without assessors blinding. Based on the results of this study blinding of important participants were infrequently reported in the included studies. Keywords: Blinding, Risk of bias, Physiotherapy, Randomized controlled trials
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Metcalfe, Andrew, Elke Gemperle Mannion, Helen Parsons, Jaclyn Brown, Nicholas Parsons, Josephine Fox, Rebecca Kearney et al. „Protocol for a randomised controlled trial of Subacromial spacer for Tears Affecting Rotator cuff Tendons: a Randomised, Efficient, Adaptive Clinical Trial in Surgery (START:REACTS)“. BMJ Open 10, Nr. 5 (Mai 2020): e036829. http://dx.doi.org/10.1136/bmjopen-2020-036829.
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IntroductionShoulder pain due to irreparable rotator cuff tears can cause substantial disability, but treatment options are limited. A balloon spacer is a relatively simple addition to a standard arthroscopic debridement procedure, but it is costly and there is no current randomised trial evidence to support its use. This trial will evaluate the clinical and cost-effectiveness of a subacromial balloon spacer for individuals undergoing arthroscopic debridement for irreparable rotator cuff tears.New surgical procedures can provide substantial benefit to patients. Good quality randomised controlled trials (RCTs) are needed, but trials in surgery are typically long and expensive, exposing patients to risk and the healthcare system to substantial costs. One way to improve the efficiency of trials is with an adaptive sample size. Such methods are well established in drug trials but have rarely, if ever, been used in surgical trials.Methods and analysisSubacromial spacer for Tears Affecting Rotator cuff Tendons: a Randomised, Efficient, Adaptive Clinical Trial in Surgery (START:REACTS) is a participant and assessor blinded, adaptive, multicentre RCT comparing arthroscopic debridement with the InSpace balloon (Stryker, USA) to arthroscopic debridement alone for people with a symptomatic irreparable rotator cuff tear. It uses a group sequential adaptive design where interim analyses are performed using all of the 3, 6 and 12-month data that are available at each time point. A maximum of 221 participants will be randomised (1:1 ratio), this will provide 90% power (at the 5% level) for a 6 point difference in the primary outcome; the Oxford Shoulder Score at 12 months. A substudy will use deltoid-active MRI scans in 56 participants to assess the function of the balloon. Analysis will be on an intention-to-treat basis and reported according to principles established in the Consolidated Standards of Reporting Trials statement.Ethics and disseminationNRES number 18/WM/0025. The results will be disseminated via peer-reviewed publications, presentations at conferences, lay summaries and social media.Trial registration numberISRCTN17825590
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Ulbrich-Zürni, Susanne, Michael Teut, Stephanie Roll und Robert Mathie. „The N-of-1 Clinical Trial: A Timely Research Opportunity in Homeopathy“. Homeopathy 107, Nr. 01 (23.01.2018): 010–18. http://dx.doi.org/10.1055/s-0037-1621731.
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Background The randomised controlled trial (RCT) is considered the ‘gold standard’ for establishing treatment efficacy or effectiveness of an intervention, but its data do not infer response in an individual patient. Individualised clinical care, a fundamental principle in complementary and alternative medicine (CAM), including homeopathy, seems well disposed in principle to being researched by single-patient (N-of-1) study design. Guidelines for reporting N-of-1 trials have recently been developed. Objective To overview the current status in the literature of the N-of-1 method and its application in medicine, including CAM. To consider whether the N-of-1 trial design offers an opportunity for novel research in homeopathy. N-of-1 Trial Design The N-of-1 trial applies the principles of the conventional crossover, blinded, RCT design. The treatment under study and the comparator are repeated in a randomised order, and with suitable washout time, over a defined period. N-of-1 design is constrained for use in chronic stable conditions, and for interventions that have quick onset and cessation of effect, with modest or negligible carryover. Outcome data can be aggregated and interpreted for the individual subject; they can also be pooled with data from several similar N-of-1 trials, enabling more generalisable conclusions. The N-of-1 Trial in CAM The typical individualisation of patient care can be accommodated in N-of-1 study design if the patient and the specific therapeutic intervention are selected within the constraints of the method. Application of the N-of-1 method in CAM has been advocated but has been mainly limited, in practice, to a small number of studies in herbal and traditional Chinese medicine. The N-of-1 Trial in Homeopathy Individualised homeopathy can be accommodated for investigation within the same methodological constraints; less in-depth homeopathic approaches to prescribing are also amendable to investigation using the N-of-1 method. No such studies have been published. We identify three main targets in its ready applicability to homeopathy: (1) to optimise clinical care in an individual patient; (2) to investigate whether the outcomes of treatment using homeopathy differ from those of placebo; (3) to aggregate data from a series of N-of-1 trials to enable broader conclusions about a group of patients or intervention. Conclusion The N-of-1 trial design offers important new investigative possibilities in homeopathy and should be explored as a means to optimise individualised health care or investigate effectiveness of the homeopathic intervention compared with placebo in individual subjects.
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Cheung, Teris, Yuen Shan Ho, Kwan Hin Fong, Yuen Ting Joyce Lam, Man Ho Li, Andy Choi-Yeung Tse, Cheng-Ta Li, Calvin Pak-Wing Cheng und Roland Beisteiner. „Evaluating the Safety and Efficacy of Transcranial Pulse Stimulation on Autism Spectrum Disorder: A Double-Blinded, Randomized, Sham-Controlled Trial Protocol“. International Journal of Environmental Research and Public Health 19, Nr. 23 (24.11.2022): 15614. http://dx.doi.org/10.3390/ijerph192315614.
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Autistic spectrum disorder (ASD) is a common developmental disorder in children. The latest non-intrusive brain stimulation (NIBS) technology—transcranial pulse stimulation (TPS)—has been proven effective in older adults with mild neurocognitive disorders and adults with major depressive disorder. Nonetheless, there is so far no robust randomized controlled trial (RCT) conducted on adolescents with ASD nationwide. This study proposes a two-armed (verum TPS group vs. sham TPS group), double-blinded, randomized, sham-controlled trial. Both groups will be measured at four timepoints, namely, baseline (T1), 2 weeks immediately after post-TPS intervention (T2), and at the 1-month (T3) and 3-month (T4) follow-ups. Thirty-four subjects, aged between 12 and 17, diagnosed with ASD will be recruited in this study. All subjects will be computerized randomised into the verum TPS group or the sham TPS group on a 1:1 ratio. All subjects will undertake functional MRI (fMRI) before and after the 2-weeks TPS interventions, which will be completed in 2 weeks’ time. This will be the first RCT evaluating the efficacy of TPS adolescents with ASD in Hong Kong. Clinical Trial Registration: ClinicalTrials.gov, identifier: NCT05408793.
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Hillner, Bruce E. „Trends in Clinical Trials Reports in Common Cancers Between 1989 and 2000“. Journal of Clinical Oncology 21, Nr. 9 (01.05.2003): 1850–58. http://dx.doi.org/10.1200/jco.2003.08.147.
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Purpose: Cancer clinical trials can be dichotomized into pilot (phase I and phase II) and randomized controlled clinical trials (RCTs). The best data source for evidence-based medicine is from RCTs. However, many patients prefer to enroll onto pilot trials, and many investigators prefer to conduct or refer their patients to pilot trials. This exploratory study sought to describe the epidemiologic patterns of clinical trial reports in common cancers. Methods: A structured review was conducted of MEDLINE citations of all English clinical trials reports published between 1989 and 2000 in breast, lung, colorectal, prostate, and female genital cancers, plus leukemias and lymphomas. Each report was classified by design (RCT, pilot, or other) and country. The abstracts of RCTs were reviewed for sample size. Reports addressing screening or prevention were excluded. Results: A total of 12,035 reports, of which 3,560 were from RCTs, were found. The annual growth in RCT reports in breast, colorectal, and prostate cancer was significant (range, 4.8% to 8.5% per year) but was insignificant in leukemias, lymphomas, and female genital and lung cancers (range, 0.1% to 4.3% per year). Within each cancer, the average sample size per report did not change during the 12 years. Nonrandomized trial reports increased on average 15.1% per year (range, 10.1% to 23.2%). The United States accounted for 30% of all RCT reports and 45% of pilot trial reports. Conclusion: The faster growth in nonrandomized compared with RCT reports may reflect rapid advances in cancer biology or different structural, commercial, and financial incentives, especially in the United States compared with Europe. Unless additional studies show evidence of an increase in their quality, the modest growth in RCT reports may limit future evidence-based cancer care.
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Williams, Mark A., Peter J. Heine, Esther M. Williamson, Francine Toye, Melina Dritsaki, Stavros Petrou, Richard Crossman et al. „Active Treatment for Idiopathic Adolescent Scoliosis (ACTIvATeS): a feasibility study“. Health Technology Assessment 19, Nr. 55 (Juli 2015): 1–242. http://dx.doi.org/10.3310/hta19550.
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BackgroundThe feasibility of conducting a definitive randomised controlled trial (RCT) evaluating the clinical effectiveness and cost-effectiveness of scoliosis-specific exercises (SSEs) for adolescent idiopathic scoliosis (AIS) is uncertain.ObjectivesThe aim of this study was to assess the feasibility of conducting a large, multicentre trial of SSE treatment for patients with AIS, in comparison with standard care, and to refine elements of the study design. The objectives were to (1) update a systematic review of controlled trials evaluating the efficacy of SSE in AIS; (2) survey UK orthopaedic surgeons and physiotherapists to determine current practice, patient populations and equipoise; (3) randomise 50 adolescents to a feasibility trial of either usual care or SSE interventions across a range of sites; (4) develop, document and assess acceptability and adherence of interventions; (5) assess and describe training requirements of physiotherapists; and (6) gain user input in all relevant stages of treatment and protocol design.DesignMulticomponent feasibility study including UK clinician survey, systematic literature review and a randomised feasibility trial.SettingThe randomised feasibility study involved four secondary care NHS trusts providing specialist care for patients with AIS.ParticipantsThe randomised feasibility study recruited people aged 10–16 years with mild AIS (Cobb angle of < 50°).InterventionsThe randomised study allocated participants to standard practice of advice and education or a physiotherapy SSE programme supported by a home exercise plan. Our choice of intervention was informed by a systematic review of exercise interventions for AIS.Main outcome measuresThe main outcome was feasibility of recruitment to the randomised study. Other elements were to inform choice of outcomes for a definitive trial and included curve severity, quality of life, requirement for surgery/brace, adverse events, psychological symptoms, costs and health utilities.ResultsA UK survey of orthopaedic consultants and physiotherapists indicated a wide variation in current provision of exercise therapy through physiotherapy services. It also found that clinicians from at least 15 centres would be willing to have their patients involved in a full study. A systematic review update found five new studies that were generally of low quality but showed some promise of effectiveness of SSE. The randomised study recruited 58 patients from four NHS trusts over 11 months and exceeded the pre-specified target recruitment rate of 1.4 participants per centre per month, with acceptable 6-month follow-up (currently 73%). Adherence to treatment was variable (56% of participants completed treatment offered). The qualitative study found the exercise programme to be highly acceptable. We learnt important lessons from patient and public involvement during the study in terms of study and intervention presentation, as well as practical elements such as scheduling of intervention sessions.ConclusionsA definitive RCT evaluating clinical effectiveness and cost-effectiveness of SSE for idiopathic scoliosis is warranted and feasible. Such a RCT is a priority for future work in the area. There is a sufficiently large patient base, combined with willingness to be randomised within specialist UK centres. Interventions developed during the feasibility study were acceptable to patients, families and physiotherapists and can be given within the affordability envelope of current levels of physiotherapy commissioning.Trial registrationCurrent Controlled Trials ISRCTN90480705.FundingThis project was funded by the NIHR Health Technology Assessment programme and will be published in full inHealth Technology Assessment; Vol. 19, No. 55. See the NIHR Journals Library website for further project information.
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Kessing, Lars Vedel, Natacha Blauenfeldt Kyster, Pernille Bondo-Kozuch, Ellen Margrethe Christensen, Birgitte Vejstrup, Birte Smidt, Anne-Marie Bangsgaard Jørgensen et al. „Effect of specialised versus generalised outpatient treatment for bipolar disorder: the CAG Bipolar trial - study protocol for a randomised controlled trial“. BMJ Open 11, Nr. 10 (Oktober 2021): e048821. http://dx.doi.org/10.1136/bmjopen-2021-048821.
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IntroductionDespite current available treatment patients with bipolar disorder often experience relapses and decreased overall functioning. Furthermore, patients with bipolar disorder are often not treated medically or psychologically according to guidelines and recommendations. A Clinical Academic Group is a new treatment initiative bringing together clinical services, research, education and training to offer care and treatment that is based on reliable evidence backed up by research. The present Clinical Academic Group for bipolar disorder (the CAG Bipolar) randomised controlled trial (RCT) aims for the first time to investigate whether specialised outpatient treatment in CAG Bipolar versus generalised community-based treatment improves patient outcomes and clinician’s satisfaction with care in patients with bipolar disorder.Methods and analysisThe CAG Bipolar trial is a pragmatic randomised controlled parallel-group trial undertaken in the Capital Region of Denmark covering a catchment area of 1.85 million people. Patients with bipolar disorder are invited to participate as part of their outpatient treatment in the Mental Health Services. The included patients will be randomised to (1) specialised outpatient treatment in the CAG Bipolar (intervention group) or (2) generalised community-based outpatient treatment (control group). The trial started 13 January 2020 and has currently included more than 600 patients. The outcomes are (1) psychiatric hospitalisations and cumulated number and duration of psychiatric hospitalisations (primary), and (2) self-rated depressive symptoms, self-rated manic symptoms, quality of life, perceived stress, satisfaction with care, use of medication and the clinicians’ satisfaction with their care (secondary). A total of 1000 patients with bipolar disorder will be included.Ethics and disseminationThe CAG Bipolar RCT is funded by the Capital Region of Denmark and ethical approval has been obtained from the Regional Ethical Committee in The Capital Region of Denmark (H-19067248). Results will be published in peer-reviewed academic journals, presented at scientific meetings and disseminated to patient organisations and media outlets.Trial registration numberNCT04229875.