Toward more efficient clinical trials for amyotrophic lateral sclerosis

Merit E. Cudkowicz, Jon Katz, Dan H. Moore, Gilmore O'Neill, Jonathan D. Glass, Hiroshi Mitsumoto, Stanley Appel, Bernard Ravina, Karl Kieburtz, Ira Shoulson, Petra Kaufmann, Jaffar Khan, Ericka Simpson, Jeremy Shefner, Bruce Levin, Valerie Cwik, David Schoenfeld, Swati Aggarwal, Michael P. McDermott, Robert G. Miller

Research output: Contribution to journalReview articlepeer-review

65 Scopus citations


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.

Original languageEnglish (US)
Pages (from-to)259-265
Number of pages7
JournalAmyotrophic Lateral Sclerosis
Issue number3
StatePublished - 2010


  • Amyotrophic lateral sclerosis
  • Network
  • Phase II
  • Therapy development

ASJC Scopus subject areas

  • Clinical Neurology
  • Neurology


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