Can we eliminate placebo in ALS clinical trials?

Research output: Contribution to journalComment/debatepeer-review

9 Scopus citations


Amyotrophic lateral sclerosis (ALS) is a fatal disease with limited treatment options. Controlled studies are a necessary part of Phase II and Phase III assessments of proposed therapies. Due to the relatively small number of patients with ALS, several study designs have been proposed to improve the efficiency of Phase II studies. Some of these advocate the use of historical controls in place of placebo controls. However, the characteristics of historical controls may not mirror those of patients in the treatment group. Novel study designs can be used to decrease the number of patients required for Phase II studies. The use of placebo controls rather than historical controls in these novel study designs likely leads to better predictions of treatments that will be successful in Phase III studies. There is general agreement on the necessity of placebo controls in Phase III studies.

Original languageEnglish (US)
Pages (from-to)861-865
Number of pages5
JournalMuscle and Nerve
Issue number6
StatePublished - Jun 1 2009

All Science Journal Classification (ASJC) codes

  • Physiology
  • Clinical Neurology
  • Cellular and Molecular Neuroscience
  • Physiology (medical)


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