Designs for clinical trials to test the efficacy of therapeutics in progressive multifocal leukoencephalopathy.
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.
Yiannoutsos
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Center for Biostatistics in AIDS Research, Harvard School of Public Health, Boston, Massachusetts 02115, USA. costas@hsph.harvard.edu
De Luca
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D000998:Antiviral Agents / Q000627:therapeutic use*; D006801:Humans; D007968:Leukoencephalopathy, Progressive Multifocal / Q000188:drug therapy*; D016032:Randomized Controlled Trials as Topic / Q000379:methods*
D000998:Antiviral Agents