Research Studies

Study Title

Orphazyme Arimoclomol Trial (A Phase III, Randomized, Placebo-Controlled Trial of Arimoclomol in ALS)

Enrollment Status


Principal Investigators

Michael Benatar, MD, PhD (International Coordinating Investigator)

Study Type

Drug Trial


  • Determine if arimoclomol is effective in extending survival and improving functional status
  • Investigate individuals’ response to the study drug following treatment for up to 76 weeks, and its safety and tolerability in patients with ALS


ALS patients


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Individuals affected with ALS, with symptoms of weakness appearing less than 18 months prior to enrollment and preserved respiratory function

Study Involvement

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Multiple study visits (onsite and remote) over a period of 18-months (double blind), followed by an open-label extension phase that may last up to 4 years.
Study procedures include:

  • Medical history and medication review
  • Drug dosing with diary tracking
  • Breathing testing
  • Neuropsychological testing
  • Questionnaires/Surveys
  • Physical exam
  • Neurological exam
  • Electrocardiogram
  • Blood, urine, and CSF collection (optional)


Primary Funding Source


Related Publications

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Benatar M, Wuu J, Andersen PM, Atassi N, David W, Cudkowicz M, Schoenfeld D. Randomized, double-blind, placebo-controlled trial of arimoclomol in rapidly progressive SOD1 ALS. Neurology. 2018 Feb 13;90(7):e565-e574. Epub 2018 Jan 24.

Related Presentations & Lectures

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