Key result
This editorial discusses the challenges of designing clinical trials for rare diseases, using a 10-year, 39-patient trial of itraconazole for chronic granulomatous disease as an example.
Why the study?
Does itraconazole prevent severe fungal infection in children and adults with chronic granulomatous disease?
Does itraconazole prevent severe fungal infection in children and adults with chronic granulomatous disease?
This editorial highlights the difficulties of conducting clinical trials in rare diseases, using a 10-year, 39-patient trial of itraconazole as an example.
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 . . .
No takes yet. Share an insight, caveat, or question.
Stephen W. Lagakos (2003) conducted an editorial in chronic granulomatous disease (n=39). itraconazole was evaluated on prevention of severe fungal infection. This editorial discusses the challenges of designing clinical trials for rare diseases, using a 10-year, 39-patient trial of itraconazole for chronic granulomatous disease as an example.
Synapse has enriched 5 closely related papers on similar clinical questions. Consider them for comparative context: