title: Design of Major Randomized Trials: Part 3 of a 4-Part Series on Statistics for Clinical Trials.
abstract: This paper provides practical guidance on the fundamentals of design for major randomized controlled trials. Topics covered include the choice of patients, choice of treatment and control groups, choice of primary and secondary endpoints, methods of randomization, appropriate use of blinding, and determination of trial size. Insights are made with reference to contemporary major trials in cardiology.
authors.affiliation: Department of Medical Statistics, London School of Hygiene and Tropical Medicine, London, United Kingdom. Electronic address: stuart.pocock@lshtm.ac.uk.
authors.forename: Stuart J
authors.initials: SJ
authors.lastname: Pocock
authors.affiliation: Department of Medical Statistics, London School of Hygiene and Tropical Medicine, London, United Kingdom.
authors.forename: Tim C
authors.initials: TC
authors.lastname: Clayton
authors.affiliation: Columbia University Medical Center, New York-Presbyterian Hospital, and the Cardiovascular Research Foundation, New York, New York.
authors.forename: Gregg W
authors.initials: GW
authors.lastname: Stone
mesh_terms: D002318:Cardiovascular Diseases / Q000628:therapy*; D003627:Data Interpretation, Statistical; D006801:Humans; D018579:Patient Selection; D016032:Randomized Controlled Trials as Topic / Q000379:methods* / Q000706:statistics & numerical data*
keywords: avoidance of bias; randomized controlled trial; sample size; selection of patients; study design; treatments and endpoints