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:
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.
Related Presentations & Lectures
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