Policy & Regulation
Orphazyme completes enrolment for phase 3 trial evaluating arimoclomol in ALS
19 July 2019 -

Orphazyme A/S (CPH:ORPHA.CO), a biopharmaceutical company developing treatments for patients living with life threatening or rare diseases, announced on Thursday the completion of enrolment in its phase 3 trial evaluating arimoclomol for the treatment of Amyotrophic Lateral Sclerosis (ALS) ahead of schedule.

Headline results from the full analysis remain on track for the first half of 2021.

Reportedly, this trial completed enrolment sooner than anticipated due to support from the global ALS community and the participation of hundreds of people living with ALS and their care partners. An interim analysis of the data is no longer necessary due to the proximity of the final data sets to the planned interim analysis.

This phase 3 trial of arimoclomol for ALS is a 76-week, randomised, placebo-controlled trial being conducted at 30 centres of excellence in North America and Europe. A total 213 participants have been randomised 2:1 to arimoclomol and placebo and up to an additional 18 individuals on stable treatment with edaravone may participate in the US. Patients completing the trial will be offered participation in an open-label extension trial.

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