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
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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Study Involvement
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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, Hansen T, Rom D, Geist MA, Blaettler T, Camu W, Kuzma-Kozakiewicz M, van den Berg LH, Morales RJ, Chio A, Andersen PM, Pradat PF, Lange D, Van Damme P, Mora G, Grudniak M, Elliott M, Petri S, Olney N, Ladha S, Goyal NA, Meyer T, Hanna MG, Quinn C, Genge A, Zinman L, Jabari D, Shoesmith C, Ludolph AC, Neuwirth C, Nations S, Shefner JM, Turner MR, Wuu J, Bennett R, Dang H, Sundgreen C; ORARIALS-01 trial team. Safety and efficacy of arimoclomol in patients with early amyotrophic lateral sclerosis (ORARIALS-01): a randomised, double-blind, placebo-controlled, multicentre, phase 3 trial. Lancet Neurol. 2024 Jul;23(7):687-699. Epub 2024 May 20.
https://pubmed.ncbi.nlm.nih.gov/38782015
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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