Research Studies

Study Title

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

Enrollment Status

Completed

Principal Investigators

Michael Benatar, MD, PhD (International Coordinating Investigator)

Study Type

Drug Trial

Goals

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

Enrolling

ALS patients

Eligibility

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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)

Contact

Primary Funding Source

Orphazyme

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.

https://pubmed.ncbi.nlm.nih.gov/29367439

Related Presentations & Lectures

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