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7.18.2019

Phase III Trial Evaluating Arimoclomol for ALS Patients Reaches Full Enrollment

Miller School Researcher Leads Multicenter International Trial

An international phase III clinical trial evaluating the drug Arimoclomol for the treatment of amyotrophic lateral sclerosis (ALS) has reached full enrollment, as reported by a University of Miami Miller School of Medicine researcher. The current phase III trial builds upon the results of a prior phase II trial that enrolled patients with a rapidly progressive, rare genetic form of ALS.

“Based on the mechanism of action of the drug and our understanding of the underlying biology of ALS, however, there is good reason to believe that all patients with ALS might benefit from Arimoclomol,” said Michael Benatar, M.D., Ph.D., professor of neurology, the Walter Bradley Chair in ALS Research, and executive director of the ALS Center at the University of Miami Miller School of Medicine.

Dr. Benatar, who designed and led the prior phase II trial, serves as the lead international coordinating investigator for the phase III trial, which includes patients at the University of Miami Health System. Altogether, 231 patients have been enrolled in the 76-week randomized, placebo-controlled trial being conducted at 30 ALS centers of excellence in North America and Europe. Local recruitment efforts at the University of Miami have been led by Volkan Granit, M.D., M.Sc., assistant professor of neurology, and Maria Elena Paredes. Follow-up of these enrolled patients is ongoing, with results from the trial expected in the first half of 2021. Patients completing the trial will be offered participation in an open-label extension trial.

ALS, also known as Lou Gehrig’s disease, is a rapidly progressive, fatal neurological disease that attacks the nerve cells responsible for controlling voluntary muscles. The Danish biopharmaceutical company Orphazyme A/S is sponsoring and running the phase III trial of Arimoclomol, a heat shock protein-based therapy that shows promise for the treatment of ALS.

The phase III trial completed enrollment sooner than anticipated. “It is thanks to the interest of ALS patients, families and caregivers, as well as the tireless efforts of physicians and research coordinators at all international participating centers, that we have been able to successfully complete enrollment,” said Dr. Benatar. “We look forward to topline results in hopes of delivering an important new treatment option for those living with ALS.”

The University of Miami and Dr. Benatar have financial interests related to the study.

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