Orphazyme finishes enrollment in phase 3 study assessing arizoclomol

The phase 3 study of arimoclomol for ALS is a 76-week, placebo-controlled, randomized study being conducted at 30 centers of excellence in Europe and North America.

The biopharmaceutical company Orphazyme A/S has reportedly announced that it has finished enrollment in its phase 3 study assessing arimoclomol for the treatment of Amyotrophic Lateral Sclerosis (ALS) before schedule. The full analysis remain on track for the first half of 2021.

Apparently, the trial has completed enrollment earlier than expected with the support of the global ALS community and contribution of hundreds of people living with ALS and their care partners. A provisional analysis of the data is no longer required due to the proximity of the final data sets to the planned interval analysis.

Michael Benatar, MD, Lead International Coordinating Investigator, Orphazyme, reportedly stated that the company is grateful to the diligent efforts of coordinators and investigators at participating centers throughout the world which enabled it to reach the important achievement, taking it one step nearer to definitively learning of benefits of arimoclomol for patients living with ALS.

Thomas Blaettler, MD, Chief Medical Officer, Orphazyme, reportedly stated that the conclusion of enrollment for pivotal ALS trial of Orphazyme is another great achievement for its clinical development program.

Calaneet Balas, President and CEO, ALS Association, Chairwoman, International Alliance of ALS/MND Associations, reportedly noted that the company is obliged to the hundreds of individuals tolerating ALS around the world for participating in the trial. Subjects urgently need new treatments for ALS and the company looks forward to the results.

Phase 3 study of arimoclomol for ALS is a 76-week, placebo-controlled, randomized study being conducted at 30 centers of excellence in Europe and North America. A sum of 213 subjects have been randomized 2:1 to arimoclomol and placebo and up to an extra 18 subjects on stable therapy with edaravone may join the U.S. Patients completing the study will be offered participation in an open-label extension trial.

Source credit: https://www.orphazyme.com/news-feed/2019/7/18/orphazyme-completes-enrollment-in-phase-3-trial-evaluating-arimoclomol-in-amyotrophic-lateral-sclerosis