Abstract
The purpose of any clinical trial is to evaluate treatment(s) in an at-risk population. Clinical trial design principles are the same regardless of the setting. Three very readable books that cover fundamentals and specifics of clinical trial design are those by Friedman, Furberg, and DeMets (1); Meinert (2); and Pocock (3). Clinical trial design is much more than inclusion/exclusion criteria, sample size, randomization to treatment arm, and counting endpoints. Defining and monitoring data collection, monitoring and maintaining compliance, and monitoring and reporting potential adverse events are often the most demanding design aspects.
Access this chapter
Tax calculation will be finalised at checkout
Purchases are for personal use only
Preview
Unable to display preview. Download preview PDF.
References
Friedman L, Furberg C, DeMets D. Fundamentals of Clinical Trials, 3rd ed. Springer Verlag, 1998.
Meinert CL. Clinical Trials: Design, Conduct and Analysis. Oxford University Press, 1986.
Pocock SJ. Clinical Trials: A Practical Approach. John Wiley & Sons, 1984.
Cairns JA, Hallstrom A, Held P. Should all trials have a data safety and monitoring committee? Am Heart J 2001; 141:156–163.
Packer M, Wittes J, Stump D. Terms of reference for Data and Safety Monitoring Committees. Am Heart J 2001; 141:542–547.
Pocock S, Furberg CD. Procedures of data and safety monitoring committees. Am Heart J 2001; 141: 289–294.
Califf RM, Ellenberg SS. Statistical approaches and policies for the operations of Data and Safety Monitoring Committees. Am Heart J. 2001; 141:301–305.
Fisher L, Klibaner M. Regulatory issues for Data and Safety Monitoring Committees. Am Heart J 2001; 141:536–541
Weaver WD, Greenberg S. Making changes in clinical trials. Am Heart J. 2001; 141:295–300.
Whitehead J. The Design and Analysis of Sequential Clinical Trials. Chichester: Ellis Horwood, 1983.
O’Brien PC, Fleming TR. A multiple testing procedure for clinical trials. Biometrics 1979; 35:549–556.
Pocock SJ. Group sequential methods in the design and analysis of sequential clinical trials. Biometrika 1977; 64:191–199.
Lin DY. A General Theory on Stochastic Curtailment for Censored Survival Data. Journal of the American Statistical Association 1999; 94:510–521.
Fisher LD. Self-designing clinical trials. Stat Med 1998; 17:1551–1562.
Gould AL, Pecore VJ. Group sequential methods for clinical trials allowing early acceptance of H0 and incorporating costs. Biometrika 1982; 69:75–80.
Begg CB. Ruminations on the intent-to-treat principle. Control Clin Trials 2000; 21:241–243.
Gibaldi M, Sullivan S. Intention-to-treat analysis in randomized trials: who gets counted? J Clin Pharmacol 1997; 37:667–672.
Prentice RL. Surrogate endpoints in clinical trials: definition and operational criteria. Stat Med 1989; 8:431–440.
Hughes MD, DeGruttola V, Welles SL. Evaluating surrogate markers. J Acquir Immune Defic Syndr Hum Retrovirol 1995; 10(Suppl 2):S1–S8.
Hallstrom AP, Greene HL, Wilkoff BL, et al. Relationship between rehospitalization and future death in patients treated for potentially lethal arrhythmia. J Cardiovasc Electrophysiol 2001; 12:990–995.
Koepsell TD, Wagner EH, Cheadle AC, et al. Selected methodological issues in evaluating community-based health promotion and disease prevention programs. Annu Rev Public Health 1992; 13:31–57.
Koepsell TD, Martin DC, Diehr PH, et al. Data analysis and sample size issues in evaluations of community-based health promotion and disease prevention programs: a mixed-model analysis of variance approach. J Clin Epidemiol 1991; 44:701–713.
Hallstrom AP. Should time from cardiac arrest until call to emergency medical services (EMS) be collected in EMS research? Crit Care Med 2002; 30:S127–S130.
Weaver WD, Cerqueira M, Hallstrom AP, et al. Prehospital-initiated vs hospital-initiated thrombolytic therapy. The Myocardial Infarction Triage and Intervention Trial. JAMA 1993; 270:1211–1216.
Hallstrom A, Cobb L, Johnson E, et al. Cardiopulmonary resuscitation by chest compression alone or with mouth-to-mouth ventilation. N Engl J Med 2000; 342:1546–1553.
Kudenchuk PJ, Cobb LA, Copass MK, et al. Amiodarone for resuscitation after out-of-hospital cardiac arrest due to ventricular fibrillation. N Engl J Med 1999; 341:871–878.
PAD Trial Investigators. The Public Access Defibrillation (PAD) Trial study design and rationale. Resuscitation 2003; 56:135–147.
Enkin MW. One and two sided tests of significance. One sided tests should be used more often. BMJ 1994; 309:874.
Dunnett CW, Gent M. An alternative to the use of two-sided tests in clinical trials. Stat Med 1996; 15: 1729–1738.
Bland JM, Altman DG. One and two sided tests of significance. BMJ 1994; 309:248.
Knottnerus JA, Bouter LM. The ethics of sample size: two-sided testing and one-sided thinking. J Clin Epidemiol 2001; 54:109,110.
Fisher LD. The use of one-sided tests in drug trials: an FDA advisory committee member’s perspective. J Biopharm Stat 1991; 1:151–156.
Moye LA, Tita AT. Defending the rationale for the two-tailed test in clinical research. Circulation 2002; 105:3062–3065.
Author information
Authors and Affiliations
Editor information
Editors and Affiliations
Rights and permissions
Copyright information
© 2005 Humana Press Inc., Totowa, NJ
About this chapter
Cite this chapter
Hallstrom, A. (2005). Design of Clinical Trials Relating to Medical Emergencies. In: Ornato, J.P., Peberdy, M.A. (eds) Cardiopulmonary Resuscitation. Contemporary Cardiology. Humana Press. https://doi.org/10.1385/1-59259-814-5:695
Download citation
DOI: https://doi.org/10.1385/1-59259-814-5:695
Publisher Name: Humana Press
Print ISBN: 978-1-58829-283-4
Online ISBN: 978-1-59259-814-4
eBook Packages: MedicineMedicine (R0)