Research Studies

Study Title

SOD1 Arimoclomol Trial (Phase II/III Randomized, Placebo-controlled Trial of Arimoclomol in SOD1 Positive Familial ALS)

Enrollment Status


Principal Investigators

Michael Benatar, MD, PhD

Study Type

Drug Trial


Demonstrate the safety, tolerability, and efficacy of arimoclomol in subjects with fast-progressing SOD1 ALS.


SOD1 ALS patients


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Study Involvement

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Primary Funding Source

FDA, ALS Association

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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Benatar M (presenter), Wuu J, Atassi N, Cudkowicz M, Schoenfeld D. Randomized, phase II trial of arimoclomol in rapidly progressive SOD1 ALS. Late-breaking session platform presentation at the 27th International Symposium on ALS/MND, Dublin, Ireland, Dec 7-9, 2016.

Gronka S, Walker M, Wuu J, Stanislaw C, Fanos J, Atassi N, Jaffa M, Andersen P, Cudkowicz M, Benatar M (presenter). Telemedicine to facilitate ALS research. Platform presentation at the 21st International Symposium on ALS/MND, Orlando, FL, Dec 11-13, 2010.

Gronka S, Wuu J, Usher S, Polak M, Benatar M (presenter). Tackling the challenges of ALS from a familial perspective. Platform presentation at the 20th International Symposium on ALS/MND, Berlin, Germany, Dec 4-10, 2009.

Benatar M (invited speaker). Evolving Paradigms in ALS Therapy Development. 147th Annual Meeting of the American Neurological Association. Chicago, IL, Oct 22-25, 2022.

Benatar M (invited speaker). Predictive, Preventive and Therapeutic Studies in Familial ALS. ALS Association Annual Leadership Forum. Orange County, CA, January 2008.