Designs for clinical trials to test the efficacy of therapeutics in progressive multifocal leukoencephalopathy.

Abstract

The design of a comparative treatment trial to evaluate the efficacy of therapeutics for progressive multifocal leukoencephalopathy (PML) is outlined. We propose a large simple randomized trial with patient survival as its primary endpoint, completed in a short period of time and involving a cohort with as few enrollment restrictions as possible. We use stratification as the counterweight to the lack of exclusion criteria and suggest that proper stratification will attenuate differences inherent in a heterogeneous subject cohort. Estimation of power, sample size, and study duration, implementation of interim analyses, toxicity management, and validation of secondary clinical measures are also addressed.

• Authors: C T Yiannoutsos, A De Luca
• Journal: Journal of neurovirology (J Neurovirol) Vol. 7 Issue 4 Pg. 369-74 (Aug 2001) ISSN: 1355-0284 [Print] United States
• PMID: 11517419 (Publication Type: Journal Article, Research Support, U.S. Gov't, P.H.S., Review)

Chemical References

• Antiviral Agents

Topics

• Antiviral Agents (therapeutic use)
• Humans
• Leukoencephalopathy, Progressive Multifocal (drug therapy)
• Randomized Controlled Trials as Topic (methods)