Recommendations for Planning Pilot Studies in Clinical and Translational Research
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A rigorous definition of a pilot study is presented, recommendations for the design, analysis and sample size justification of pilot studies in clinical and translational research are offered, and the important role that well‐designed pilot studies play in the advancement of science and scientific careers are emphasized.Abstract:
Advances in clinical and translation science are facilitated by building on prior knowledge gained through experimentation and observation. In the context of drug development, preclinical studies are followed by a progression of phase I through phase IV clinical trials. At each step, the study design and statistical strategies are framed around research questions that are prerequisites for the next phase. In other types of biomedical research, pilot studies are used for gathering preliminary support for the next research step. However, the phrase "pilot study" is liberally applied to projects with little or no funding, characteristic of studies with poorly developed research proposals, and usually conducted with no detailed thought of the subsequent study. In this article, we present a rigorous definition of a pilot study, offer recommendations for the design, analysis and sample size justification of pilot studies in clinical and translational research, and emphasize the important role that well-designed pilot studies play in the advancement of science and scientific careers.read more
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Annie M. Young,Andrea Marshall,Jenny Thirlwall,Oliver Chapman,Anand Lokare,Catherine Hill,Danielle Hale,Janet A. Dunn,Gary H. Lyman,Charles Hutchinson,Peter MacCallum,Ajay K. Kakkar,FD Richard Hobbs,Stavros Petrou,Jeremy Dale,Christopher J. Poole,Anthony Maraveyas,Mark Levine +17 more
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Defining Feasibility and Pilot Studies in Preparation for Randomised Controlled Trials: Development of a Conceptual Framework.
Sandra Eldridge,Gillian Lancaster,Michael J. Campbell,Lehana Thabane,Sally Hopewell,Claire L. Coleman,Christine Bond +6 more
TL;DR: A framework for defining pilot and feasibility studies focusing on studies conducted in preparation for a randomised controlled trial is described, suggesting that to facilitate their identification, these studies should be clearly identified using the terms ‘feasibility’ or ‘pilot’ as appropriate.
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An audit of sample sizes for pilot and feasibility trials being undertaken in the United Kingdom registered in the United Kingdom Clinical Research Network database
TL;DR: The target sample sizes set for pilot and feasibility randomised controlled trials, currently running within the United Kingdom, are described to describe the observed sample sizes in feasibility and pilot randomisedcontrolled trials on the UKCRN Database.
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Adipose-derived mesenchymal stem cell therapy in the treatment of knee osteoarthritis: a randomized controlled trial.
Julien Freitag,D. Bates,James Wickham,Kiran Shah,Leesa Huguenin,Abi Tenen,Kade L. Paterson,Richard L. Boyd +7 more
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Verapamil and beta cell function in adults with recent-onset type 1 diabetes.
Fernando Ovalle,Tiffany Grimes,Guanlan Xu,Anish Patel,Truman Grayson,Lance A. Thielen,Peng Li,Anath Shalev +7 more
TL;DR: A randomized double-blind placebo-controlled phase 2 clinical trial ( NCT02372253 ) was conducted to assess the efficacy and safety of oral verapamil added for 12 months to a standard insulin regimen in adult subjects with recent-onset Type 1 diabetes as mentioned in this paper.
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Design and analysis of pilot studies: recommendations for good practice
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Journal ArticleDOI
Sample size of 12 per group rule of thumb for a pilot study
TL;DR: When designing a clinical trial an appropriate justification for the sample size should be provided in the protocol, but there are a number of settings when undertaking a pilot trial when there is no prior information to base a sample size on.
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