Toward more efficient clinical trials for amyotrophic lateral sclerosis

Abstract

More than 30 phase II or III clinical trials have been carried out in amyotrophic lateral sclerosis (ALS). Only riluzole, however, has been shown to extend survival and/or time to tracheostomy. Many early ALS trials lacked solid pharmacodynamic and pharmacokinetic data for the treatment being tested, challenging the interpretation of the efficacy and pathway relevance. Understanding of the genetics and pathophysiology of ALS has improved considerably in the past decade, but biomarkers of disease activity remain lacking. A more efficient approach to early phase clinical trials is needed to accelerate the identification of useful agents for ALS. Here we summarize our current thinking about phase II design options and the potential benefits of a clinical trial network for phase II trials in ALS.

Key words::

• Amyotrophic lateral sclerosis
• phase II
• network
• therapy development

Acknowledgements

We would like to thank the Muscular Dystrophy Association for organizing the workshop on phase II clinical trials in ALS.

Declaration of interest: The authors report no conflicts of interest. The authors alone are responsible for the content and writing of the paper.