Principal Investigators: Michael Benatar, MD, PhD (University of Miami), Merit Cudkowicz, MD, MSc (MGH)
Site PI: Nazem Atassi, MD (MGH)
The Arimoclomol study is a seamless, adaptive design, phase II/III trial that aims to determine the safety and efficacy of Arimoclomol in patients with rapidly progressive SOD1 positive familial ALS. Patients from anywhere in the United States and Canada are eligible to participate and the study will cover the costs of travel to a study center. After screening procedures and confirmation of SOD1 mutation status, eligible participants travel to a study center (University of Miami or MGH) for Baseline study procedures and randomization. Study evaluations then occur monthly for 6 months and every other month for a further 6 months. The Month-2 visit again requires travel to a study center, but all other study procedures are performed via telephone and/or in participants’ homes.
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