scholarly journals Progression from external pilot to definitive randomised controlled trial: a methodological review of progression criteria reporting

BMJ Open ◽  
2021 ◽  
Vol 11 (6) ◽  
pp. e048178
Author(s):  
Katie Mellor ◽  
Saskia Eddy ◽  
Nicholas Peckham ◽  
Christine M Bond ◽  
Michael J Campbell ◽  
...  
Keyword(s):  
Randomised Controlled Trial ◽  
Controlled Trial ◽  
A Priori ◽  
Pilot Trial ◽  
Public Library ◽  
Feasibility Studies ◽  
Methodological Review ◽  
Pilot Trials ◽  
Trial Protocols ◽  
Randomised Controlled

ObjectivesPrespecified progression criteria can inform the decision to progress from an external randomised pilot trial to a definitive randomised controlled trial. We assessed the characteristics of progression criteria reported in external randomised pilot trial protocols and results publications, including whether progression criteria were specified a priori and mentioned in prepublication peer reviewer reports.Study designMethodological review.MethodsWe searched four journals through PubMed: British Medical Journal Open, Pilot and Feasibility Studies, Trials and Public Library of Science One. Eligible publications reported external randomised pilot trial protocols or results, were published between January 2018 and December 2019 and reported progression criteria. We double data extracted 25% of the included publications. Here we report the progression criteria characteristics.ResultsWe included 160 publications (123 protocols and 37 completed trials). Recruitment and retention were the most frequent indicators contributing to progression criteria. Progression criteria were mostly reported as distinct thresholds (eg, achieving a specific target; 133/160, 83%). Less than a third of the planned and completed pilot trials that included qualitative research reported how these findings would contribute towards progression criteria (34/108, 31%). The publications seldom stated who established the progression criteria (12/160, 7.5%) or provided rationale or justification for progression criteria (44/160, 28%). Most completed pilot trials reported the intention to proceed to a definitive trial (30/37, 81%), but less than half strictly met all of their progression criteria (17/37, 46%). Prepublication peer reviewer reports were available for 153/160 publications (96%). Peer reviewer reports for 86/153 (56%) publications mentioned progression criteria, with peer reviewers of 35 publications commenting that progression criteria appeared not to be specified.ConclusionsMany external randomised pilot trial publications did not adequately report or propose prespecified progression criteria to inform whether to proceed to a future definitive randomised controlled trial.

scholarly journals Randomised controlled trial to investigate the effectiveness of local oestrogen treatment in postmenopausal women undergoing pelvic organ prolapse surgery (LOTUS): a pilot study to assess feasibility of a large multicentre trial

BMJ Open ◽  
2020 ◽  
Vol 10 (9) ◽  
pp. e025141
Author(s):  
Tina Sara Verghese ◽  
Lee Middleton ◽  
Versha Cheed ◽  
Lisa Leighton ◽  
Jane Daniels ◽  
...  
Keyword(s):  
Pelvic Organ Prolapse ◽  
Randomised Controlled Trial ◽  
Postmenopausal Women ◽  
Controlled Trial ◽  
Pilot Trial ◽  
Pelvic Organ ◽  
Oestrogen Treatment ◽  
Pop Surgery ◽  
Randomised Controlled ◽  
Oestrogen Supplementation

ObjectiveTo evaluate the feasibility of a multicentre randomised controlled trial (RCT) comparing oestrogen treatment with no oestrogen supplementation in women undergoing pelvic organ prolapse (POP) surgery.Design and settingA randomised, parallel, open, external pilot trial involving six UK urogynaecology centres (July 2015–August 2016).ParticipantsPostmenopausal women with POP opting for surgery, unless involving mesh or for recurrent POP in same compartment.InterventionWomen were randomised (1:1) to preoperative and postoperative oestrogen or no treatment. Oestrogen treatment (oestradiol hemihydrate 10 μg vaginal pessaries) commenced 6 weeks prior to surgery (once daily for 2 weeks, twice weekly for 4 weeks) and twice weekly for 26 weeks from 6 weeks postsurgery.Outcome measuresThe main outcomes were assessment of eligibility and recruitment rates along with compliance and data completion. To obtain estimates for important aspects of the protocol to allow development of a definitive trial.Results325 women seeking POP surgery were screened over 13 months and 157 (48%) were eligible. Of these, 100 (64%) were randomised, 50 to oestrogen and 50 to no oestrogen treatment, with 89 (44/45 respectively) ultimately having surgery. Of these, 89% (79/89) returned complete questionnaires at 6 months and 78% (32/41) reported good compliance with oestrogen. No serious adverse events were attributable to oestrogen use.ConclusionsA large multicentre RCT of oestrogen versus no treatment is feasible, as it is possible to randomise and follow up participants with high fidelity. Four predefined feasibility criteria were met. Compliance with treatment regimens is not a barrier. A larger trial is required to definitively address the role of perioperative oestrogen supplementation.Trial registration numberISRCTN46661996.

scholarly journals The effect of alcohol strength on alcohol consumption: findings from a randomised controlled cross-over pilot trial.

2021 ◽  
Author(s):  
Parvati Rose Perman-Howe ◽  
Emma L Davies ◽  
David R Foxcroft
Keyword(s):  
Randomised Controlled Trial ◽  
Alcohol Consumption ◽  
Sample Size ◽  
Study Protocol ◽  
Controlled Trial ◽  
Pilot Trial ◽  
Recruitment Rate ◽  
Participant Recruitment ◽  
Licensed Premises ◽  
Randomised Controlled

Abstract Background Reducing the alcohol content of drinks has the potential to reduce alcohol consumption. Aims: (1) test the feasibility of a randomised controlled trial (RCT) to assess the effect of alcohol strength on alcohol consumption within licensed premises in the United Kingdom (UK), (2) provide data to estimate key parameters for a RCT. Methods Double-blind randomised controlled cross-over pilot trial based within four licensed premises in the UK. Participants (n=36) purchased and consumed ad libitum a 3.5% lager and a 4.8% lager during two separate study sessions. Descriptive statistics reported the efficacy and efficiency of the study processes, and the rates of licensed premises recruitment, and participant recruitment and attrition. Mean and the 95% confidence interval (CI) compared alcohol consumption between conditions. The mean, standard deviation (SD) and CI of UK units of alcohol consumed were used to calculate a sample size for a RCT. Responses to participant questionnaires and duration of participation in study sessions between conditions were analysed.Results Components of the study protocol were effective and efficient. The venue recruitment rate was less than anticipated. The participant recruitment rate was greater than anticipated. The rate of attrition was 23% and varied by less than 1% according to the arm of the trial. There was a reduction of alcohol consumed under the intervention conditions. Estimated mean difference, and 95% CI (UK units): -3.76 (-5.01 to -2.52).The sample size required for a RCT is 53. Participants did not find one lager more pleasant in taste: (on a scale of one to 10) -0.95 (-2.11 to 0.21). Participants found the reduced-strength lager less enjoyable: (on a scale of one to 10) -1.44 (-2.64 to -0.24) and they perceived themselves to be less intoxicated after consuming it: (on a scale of one to 10) -1.00 (-1.61 to -0.40).Conclusion A RCT is feasible with minor alterations to the study protocol and scoping work to establish different brands of alcohol that are more alike and more enjoyable than the products used in the pilot trial. Trial registration Registered in the American Economic Association (AEA) Randomised Controlled Trial (RCT) Registry as of 16 June 2017: https://www.socialscienceregistry.org/trials/2266. Unique identifying number: AEARCTR-0002266.

scholarly journals Ross for Valve replacement In AduLts (REVIVAL) pilot trial: rationale and design of a randomised controlled trial

BMJ Open ◽  
2021 ◽  
Vol 11 (9) ◽  
pp. e046198
Author(s):  
Richard Whitlock ◽  
Emilie Belley-Cote ◽  
Filip Rega ◽  
Michael W.A. Chu ◽  
Graham R McClure ◽  
...  
Keyword(s):  
Randomised Controlled Trial ◽  
Valve Replacement ◽  
Controlled Trial ◽  
Pilot Trial ◽  
Rank Test ◽  
Event Analysis ◽  
Ross Procedure ◽  
Elderly Adults ◽  
Randomised Controlled ◽  
The Impact

IntroductionIn non-elderly adults, aortic valve replacement (AVR) with conventional prostheses yield poor long-term outcomes. Recent publications suggest a benefit of the Ross procedure over conventional AVR and highlight the need for high-quality randomised controlled trial (RCTs) on the optimal AVR. We have initiated a pilot trial assess two feasibility criteria and one assumption: (1) evaluate the capacity to enrol six patients per centre per year in at least five international centre, (2) validate greater than 90% compliance with allocation and (3) to validate the proportion of mechanical (≥65%) vs biological (≤35%) valves in the conventional arm.Methods and analysisRoss for Valve replacement In AduLts (REVIVAL) is a multinational, expertise-based RCT in adults aged 18–60 years undergoing AVR, comparing the Ross procedure versus one of the alternative approaches (mechanical vs stented or stentless bioprosthesis). The feasibility objectives will be assessed after randomising 60 patients; we will then make a decision regarding whether to expand the trial with the current protocol. We will ultimately examine the impact of the Ross procedure as compared with conventional AVR in non-elderly adults on survival free of valve-related life-threatening complications (major bleeding, systemic thromboembolism, valve thrombosis and valve reoperation) over the duration of follow-up. The objectives of the pilot trial will be analysed using descriptive statistics. In the full trial, the intention-to-treat principle will guide all primary analyses. A time-to-event analysis will be performed and Kaplan-Meier survival curves with comparison between groups using a log rank test will be presented.Ethics and disseminationREVIVAL will answer whether non-elderly adults benefit from the Ross procedure over conventional valve replacement. The final results at major meetings, journals, regional seminars, hospital rounds and via the Reducing Global Perioperative Risk Multimedia Resource Centre.Trial registration numberClinicalTrials.gov Identifier: NCT03798782Protocol versionJanuary 29, 2019 (Final Version 1.0)

scholarly journals Comparing an optimised physiotherapy treatment package with usual physiotherapy care for people with Tennis Elbow – protocol for the OPTimisE pilot and feasibility randomised controlled trial.

2021 ◽  
Author(s):  
Marcus Bateman ◽  
Benjamin Saunders ◽  
Chris Littlewood ◽  
Daniel Davis ◽  
Jacqueline Beckhelling ◽  
...  
Keyword(s):  
Randomised Controlled Trial ◽  
Tennis Elbow ◽  
Controlled Trial ◽  
Pilot Trial ◽  
Physiotherapy Treatment ◽  
Consent Rate ◽  
Efficient Treatment ◽  
Feasibility Randomised Controlled Trial ◽  
Treatment Package ◽  
Randomised Controlled

Abstract BackgroundPhysiotherapy is recommended for people with Tennis Elbow, but whilst a wide array of treatments is available, the optimal approach remains uncertain. We have therefore recently developed an optimised physiotherapy treatment package for Tennis Elbow based on a synthesis of the evidence, patient input, and clinical consensus. It consists of detailed advice and education, a structured progressive exercise programme and provision of a counter-force elbow brace. Here we report the protocol for our multi-centre pilot and feasibility randomised controlled trial (RCT) designed to a) examine the feasibility of our optimised physiotherapy treatment package, and b) to pilot trial processes for a future fully-powered RCT to test clinical and cost-effectiveness compared with usual physiotherapy treatment.MethodsA multi-centre pilot and feasibility RCT will be conducted across three sites in England, recruiting up to 50 patients (or for a maximum of 12 months). Participants with Tennis Elbow, identified from physiotherapy clinic waiting lists and general practice surgeries, will be randomly allocated to receive the optimised physiotherapy treatment package or usual physiotherapy care. Analysis will focus on feasibility measures including; consent rate, intervention fidelity, follow-up rate, and outcome completion rate. A nested qualitative study will explore the acceptability of the study processes and patient and physiotherapist experiences of the new optimised intervention.DiscussionThis study will determine the feasibility of a new optimised physiotherapy treatment package for people with Tennis Elbow and pilot the processes for a future fully-powered RCT. In the longer term, this treatment package may improve pain and quality of life outcomes for people with Tennis Elbow and help to guide a more clinically and economically efficient treatment pathway design.Trial RegistrationRegistered with the ISRCTN database 19/7/2021. https://www.isrctn.com/ISRCTN64444585

scholarly journals Sugammadex, neostigmine and postoperative pulmonary complications: an international randomised feasibility and pilot trial

2021 ◽  
Vol 7 (1) ◽  
Author(s):  
Kate Leslie ◽  
Matthew T. V. Chan ◽  
Jai N. Darvall ◽  
Anurika P. De Silva ◽  
Sabine Braat ◽  
...  
Keyword(s):  
Randomised Controlled Trial ◽  
Neuromuscular Blockade ◽  
Controlled Trial ◽  
Pilot Trial ◽  
Pulmonary Complications ◽  
Trial Registration ◽  
Postoperative Pulmonary Complications ◽  
Link Type ◽  
Randomised Controlled ◽  
Intrathoracic Surgery

Abstract Background Sugammadex reduces residual neuromuscular blockade after anaesthesia, potentially preventing postoperative pulmonary complications. However, definitive evidence is lacking. We therefore conducted a feasibility and pilot trial for a large randomised controlled trial of sugammadex, neostigmine, and postoperative pulmonary complications. Methods Patients aged ≥40 years having elective or expedited abdominal or intrathoracic surgery were recruited in Australia and Hong Kong. Perioperative care was at the discretion of clinicians, except for the use of rocuronium and/or vecuronium for neuromuscular blockade and the randomised intervention (sugammadex or neostigmine) for reversal. Feasibility measurements included recruitment, crossover, acceptability, completeness, and workload. Trial coordinator feedback was systematically sought. Patient-reported quality of life was measured using the EQ-5D-5L score. The primary pilot outcome was the incidence of new pulmonary complications up to hospital discharge (or postoperative day 7 if still in hospital). Results Among 150 eligible patients, 120 consented to participate (recruitment rate 80%, 95% confidence interval [CI] 73 to 86%). The randomised intervention was administered without crossover to 115 of 117 patients who received reversal (98%, 95% CI 94 to 100%). The protocol was acceptable or highly acceptable to the anaesthetist in 108 of 116 cases (93%, 95% CI 87 to 97%; missing = 4). Four patients of the 120 patients were lost to follow-up at 3 months (3.3%, 95% CI 0.9 to 8.3%). Case report forms were complete at 3 months for all remaining patients. The median time to complete trial processes was 3.5 h (range 2.5–4.5 h). Trial coordinators reported no barriers to trial processes. Patients were aged 64 (standard deviation 11) years, 70 (58%) were male and 50 (42%) were female, and planned surgeries were thoracic (23 [19%]), upper abdominal (41 [34%]), and lower abdominal (56 [47%]). The primary outcome was observed in 5 (8.5%) of the 59 sugammadex patients and 5 (8.2%) of the 61 neostigmine patients (odds ratio 1.02, 95% CI 0.28 to 3.67). Conclusions A large international randomised controlled trial of sugammadex, neostigmine and postoperative pulmonary complications in adult patients having abdominal and intrathoracic surgery, including collection of cost-effectiveness evidence for Health Technology Appraisal, is feasible. Trial registration Prospectively registered at the Australian and New Zealand Clinical Trials Registry (ACTRN12620001313921) on December 7, 2020. www.anzctr.org.au/Trial/Registration/TrialReview.aspx?id=380645&isReview=true.

scholarly journals Singing for people with aphasia (SPA): a protocol for a pilot randomised controlled trial of a group singing intervention to improve well-being

BMJ Open ◽  
2018 ◽  
Vol 8 (9) ◽  
pp. e025167 ◽  
Author(s):  
Mark Tarrant ◽  
Mary Carter ◽  
Sarah Gerard Dean ◽  
Rod S Taylor ◽  
Fiona C Warren ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Randomised Controlled Trial ◽  
Public Involvement ◽  
Controlled Trial ◽  
Pilot Trial ◽  
Well Being ◽  
Retention Rates ◽  
Short Version ◽  
Randomised Controlled

IntroductionThe singing for people with aphasia (SPA) intervention aims to improve quality of life and well-being for people with poststroke aphasia. A definitive randomised controlled trial (RCT) is required to assess the clinical and cost effectiveness of SPA. The purpose of this pilot study is to assess the feasibility of such a definitive trial and inform its design.Methods and analysisA two-group, assessor-blinded, randomised controlled external pilot trial with parallel mixed methods process evaluation and economic evaluation. Forty-eight participants discharged from clinical speech and language therapy will be individually randomised 1:1 to SPA (10 group sessions plus a resource booklet) or control (resource booklet only). Outcome assessment at baseline, 3 and 6 months postrandomisation include: ICEpop CAPability measure for adults, Stroke and Aphasia Quality of Life, EQ-5D-5L, modified Reintegration into Normal Living Index, Communication Outcome After Stroke, Very Short Version of the Minnesota Aphasia Test, Service Receipt Inventory and Care Related Quality of Life. Feasibility, acceptability and process outcomes include recruitment and retention rates, with measurement burden and trial experiences being explored in qualitative interviews (15 participants, 2 music facilitators and 2 music champions). Analyses include: descriptive statistics, with 95% CIs where appropriate; qualitative themes; intervention fidelity from videos and session checklists; rehearsal of health economic analysis.Ethics and disseminationNHS National Research Ethics Service and the Health Research Authority confirmed approval in April 2017; recruitment commenced in June 2017. Outputs will include: pilot data to inform whether to proceed to a definitive RCT and support a funding application; finalised intervention manual for multicentre replication of SPA; presentations at conferences, public involvement events; internationally recognised peer reviewed journal publications, open access sources and media releases.Trial registration numberNCT03076736.

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