Clinical trials design and conduct

References

• Chan AW, Tetzlaff JM, Altman DG, et al. SPIRIT 2013 statement: defining standard protocol items for clinical trials. Ann Intern Med. 2013;158(3):200–207.
   Google Scholar
• Schulz KF, Altman DG, Moher D, for The CONSORT Group. CONSORT 2010 statement: updated guidelines for reporting parallel group randomized trials. PLoS Med. 2010;7(3):1–7.
   Google Scholar
• Friedman LM, Furberg CD, DeMets DL. Fundamentals of clinical trials. 3rd ed. New York (NY): Springer-Verlag; 1998. p. 3–5.
   Google Scholar
• Pocock SJ. Clinical trials. A practical approach. New York (NY): Wiley; 1983.
   Google Scholar
• Hully SB, Cummings SR, Browner WS, et al. Designing clinical research. 4th ed. Philadelphia (PA): Lippincott, Williams & Wilkins; 2013.
   Google Scholar
• Itani KMF, Reda DJ, editors. Clinical trials design in operative and non operative invasive procedures. Cham (Switzerland): Springer International Publishing AG; 2017.
   Google Scholar
• Woolf SH. The meaning of translational research and why it matters. J Amer Med Assoc. 2008;299(2):211–213.
   Google Scholar
• Thabane L, Ma J, Chu R, et al. A tutorial on pilot studies: the what, why, and how. BMC Med Res Methodol. 2010;10:1.
   Google Scholar
• Hynes DM, Stroupe KT, Greer JW, et al. Potential cost savings of erythropoietin administration in end-stage renal disease. Am J Med. 2002;112(3):169–175.
   Google Scholar
• Henderson WG, Moritz T, Goldman S, et al. Use of cumulative meta-analysis in the design, monitoring, and final analysis of a clinical trial: a case study. Control Clin Trials. 1995;16(5):331–341.
   Google Scholar
• Friedman LM, Furberg CD, DeMets DL. Fundamentals of clinical trials. 3rd ed. New York (NY): Springer-Verlag; 1998. p. 98.
   Google Scholar
• The Cardiac Arrhythmia Suppression Trial (CAST) Investigators. Preliminary report: effect of Encainide and Flecainide on mortality in a randomized trial of arrhythmia suppression after myocardial infarction. New Engl J Med. 1989;321(6):406–412.
   Google Scholar
• US Department of Health and Human Services, Food and Drug Administration, Center for Drug Evaluation and Research and Center for Biologics Evaluation and Research. Nov. 2016. http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM202140.pdf
   Google Scholar
• Tunis SR, Stryer DB, Clancy CM. Practical clinical trials: increasing the value of clinical research for decision making in clinical and health policy. J Amer Med Assoc. 2003;290(12):1624–1632.
   Google Scholar
• Chalkidou K, Tunis S, Whicher D, et al. The role for pragmatic randomized controlled trials (pRCTs) in comparative effectiveness research. Clin Trials. 2012;9(4):436–446.
   Google Scholar
• Materson BJ, Reda DJ, Cushman WC, et al. Single-drug therapy for hypertension in men. A comparison of six antihypertensive agents with placebo. New Engl J Med. 1993;328:914–921.
   Google Scholar
• Donta ST, Clauw DJ, Engel CC Jr, et al. Cognitive behavioral therapy and aerobic exercise for gulf war veterans’ illnesses. A randomized controlled trial. J Amer Med Assoc. 2003;289(11):1396–1404.
   Google Scholar
• Larson VD, Williams DW, Henderson WG, et al. Efficacy of 3 commonly used hearing aid circuits: a crossover trial. J Amer Med Assoc. 2000;284(14):1806–1813.
   Google Scholar
• Wyrick DL, Rulison KL, Fearnow-Kenney M, et al. Moving beyond the treatment package approach to developing behavioral interventions: addressing questions that arose during an application of the multiphase optimization strategy (MOST). Transl Behav Med. 2014;4:252–259.
   Google Scholar
• Baer L, Ivanova A. When should the sequential parallel comparison design be used in clinical trials? Clin Invest. 2013;3(9):823–833.
   Google Scholar
• Henderson WG, Demakis J, Fihn SD, et al. Cooperative studies in health services research in the Department of Veterans Affairs. Control Clin Trials. 1998;19(2):134–148.
   Google Scholar
• Treweek S, Pitethly M, Cook J, et al. Strategies to improve recruitment to randomized trials. Cochran Database Syst Rev. 2018.
   Google Scholar
• Huang GD, Bull J, McKee KJ, et al. Clinical trials recruitment planning: a proposed framework from the clinical trials transformation initiative. Contemp Clin Trials. 2018;66:74–79.
   Google Scholar
• Walters SJ, Henriques-Cadby B, Bortolami O, et al. Recruitment and retention of participants in randomized controlled trials: a review of trials funded and published by the United Kingdom Technical Assessment Programme. BMJ Open. 2017;7:3.
   Google Scholar
• Bower P, Brueton V, Gamble C, et al. Interventions to improve recruitment and retention in clinical trials: a survey and workshop to assess current practice and future priorities. Trials. 2014;15:399.
   Google Scholar
• Efron B. Forcing a sequential experiment to be balanced. Biometrika. 1971;58:403–417.
   Google Scholar
• Pocock SJ, Simon R. Sequential treatment assignment with balancing for prognostic factors in the controlled clinical trial. Biometrics. 1975;31:103–115.
   Google Scholar
• Ivers NM, Halperin IJ, Barnsley J, et al. Allocation techniques for balance at baseline in cluster randomized trials: a methodological review. Trials. 2012;13:120.
   Google Scholar
• Raub GM, Butcher I. Balance in cluster randomized trials. Stat Med. 2001;20(3):351–365.
   Google Scholar
• Moulton LH. Covariate-based constrained randomization of group-randomized trials. Clin Trials. 2004;1(3):297–305.
   Google Scholar
• Borrelli B, Sepinwall D, Ernst D, et al. A new tool to assess treatment fidelity and evaluation of treatment fidelity across ten years of health behavior research. J Consult Clin Psychol. 2005;73(5):852–860.
   Google Scholar
• Handley MA, Schillinger D, Shiboski S. Quasi-experimental designs in practice-based research settings: design and implementation considerations. J Amer Board Fam Med. 2011;24(5):589–596.
   Google Scholar
• Mdege ND, Man MS, Taylor CA, et al. Systematic Review of stepped wedge cluster randomized trials shows that design is particularly used to evaluate interventions during routine implementation. J Clin Epidemiol. 2011;64:936–948.
   Google Scholar
• Ezekowitz MD, Bridgers SL, James KE, et al. Warfarin in the prevention of stroke associated with Nonrheumatic atrial fibrillation. New Engl J Med. 1992;327(20):1406–1412.
   Google Scholar
• Steering Committee of the Physicians’ Health Study Research Group. Final report on the aspirin component of the ongoing physicians’ health study. New Engl J Med. 1989;321(3):129–135.
   Google Scholar
• Dunn OJ. Multiple comparisons among means. J Am Stat Assoc. 1961;56:52–64.
   Google Scholar
• Hochberg Y. A sharper bonferroni procedure for multiple tests of significance. Biometrika. 1988;75:800–802.
   Google Scholar
• Holm S. A simple sequentially rejective multiple test procedure. Scan J Stat. 1979;6:65–70.
   Google Scholar
• Henderson WG, Fisher SG, Cohen N, et al. Use of principal components analysis to develop a composite score as a primary outcome variable in a clinical trial. Control Clin Trials. 1990;11:199–214.
   Google Scholar
• Cohen NL, Waltzman SB, Fisher SG, et al. A prospective, randomized study of cochlear implants. New Engl J Med. 1993;328(4):233–237.
   Google Scholar
• Cohen J. Statistical power analysis for the behavioral sciences. 2nd ed. Hillsdale (NJ): Lawrence Erlbaum Associates; 1988.
   Google Scholar
• Laupacis A, Sackett DL, Roberts RS. An assessment of clinically useful measures of the consequences of treatment. N Engl J Med. 1988;318:1728–1733.
   Google Scholar
• Henderson WG, Fisher SG, Weber L, et al. Conditional power for arbitrary survival curves to decide whether to extend a clinical trial. Control Clin Trials. 1991;12:304–313.
   Google Scholar
• Hammermeister KE, Sethi GK, Henderson WG, et al. A comparison of outcomes in men 11 years after heart-valve replacement with a mechanical valve or bioprothesis. New Engl J Med. 1993;328(18):1289–1296.
   Google Scholar
• Hammermeister K, Sethi GK, Henderson WG, et al. Outcomes 15 years after valve replacement with a mechanical versus a bioprosthetic valve: final report of the veterans affairs randomized trial. J Am Coll Cardiol. 2000;36(4):1152–1158.
   Google Scholar
• Weinberger M, Oddone EZ, Henderson WG, et al. Does increased access to primary care reduce hospital readmissions? New Engl J Med. 1996;334(22):1441–1447.
   Google Scholar
• Harris PA, Taylor R, Thielke R, et al. Research electronic data capture (REDCap)—a metadata-driven methodology and workflow process for providing translational research informatics support. J Biomed Inform. 2009;42:377–381.
   Google Scholar
• Friedman LM, Furberg CD, DeMets DL. Fundamentals of clinical trials. 3rd ed. New York (NY): Springer-Verlag; 1998. 163 p.
   Google Scholar
• http://www.hhs.gov/ohrp/policy/advevntguid.html, accessed 4/17/14.
   Google Scholar
• Friedman LM, Furberg CD, DeMets DL. Fundamentals of clinical trials. 3rd ed. New York: Springer-Verlag; 1998. 97 p.
   Google Scholar
• Murray DM, Varnell SP, Blitstein JL. Design and analysis of group-randomized trials: a review of recent methodological developments. Am J Public Health. 2004;94(3):423–432.
   Google Scholar
• Informed Consent Checklist. 1998. HHS.gov. https://www.hhs.gov/ohrp/regulations-and-policy/guidance/checklists/index.html.
   Google Scholar
• Flynn KE, Hahn CL, Kramer JM, et al. Using central IRBs for multicenter clinical trials in the united states. PLoS One. 2013;8:1.
   Google Scholar
• Moseley JB, O'Malley K, Petersen NJ, et al. A controlled trial of arthroscopic surgery for osteoarthritis of the Knee. New Engl J Med. 2002;347:81–88.
   Google Scholar
• Taljaard M, Chaudhry SH, Brehaut JC, et al. Survey of consent practices in cluster randomized trials: improvements are needed in ethical conduct and reporting. Clin Trials. 2014;11(1):60–69.
   Google Scholar
• Friedman LM, Furberg CD, DeMets DL. Fundamentals of clinical trials. 3rd ed. Chapter 15. New York (NY): Springer-Verlag; 1998.
   Google Scholar
• O'Brien PC, Fleming TR. A multiple testing procedure for clinical trials. Biometrics. 1979;35(3):549–556.
   Google Scholar
• http://www.nhlbi.nih.gov/funding/policies/dsmpolicy.htm.
   Google Scholar
• The Department of Veterans Affairs Laryngeal Cancer Study Group. Induction chemotherapy plus radiation compared with surgery plus radiation in patients with advanced laryngeal cancer. New Engl J Med. 1991;324:1685–1690.
   Google Scholar
• Hillman RE, Walsh MJ, Wolf GT, et al. Functional outcomes following treatment for advanced laryngeal cancer. Ann Oto Rhinol Laryn. 1998 Supplement 172;107(5, Part 2):1–27.
   Google Scholar