Clinical Trials and Rare Diseases

Abstract

In this issue of the Journal, Gallin and colleagues¹ report the results of a clinical trial of itraconazole for the prevention of severe fungal infection in children and adults with chronic granulomatous disease. This trial required 10 years to enroll just 39 patients. The study illustrates some of the problems and options that arise in the design of clinical trials of new therapies for rare diseases.When patients are scarce, one option is to design the study so that patients serve as their own controls. For example, in a two-group crossover study,² each patient receives the two study treatments sequentially . . .