Completed

Orphazyme Arimoclomol trial (“A Phase 3, Randomised, Placebo-Controlled Trial of Arimoclomol in Amyotrophic Lateral Sclerosis”)

This phase 3 study aims to investigate the effect of arimoclomol on disease progression in patients with ALS. Arimoclomol stimulates a stress response in cells, allowing poorly folded proteins to return to their original state. Since ALS is characterised by an accumulation of poorly folded proteins in motor nerve cells, it is hoped that this treatment strategy could slow down the disease.

A dose of 1200 mg per day will be compared with placebo, with a ratio of 2:1. To participate, the disease must not yet be too advanced: patients must be ill for up to 18 months, have an ALSFRS-R score of ≥35 and lung function (SVC) of ≥70%. The study is taking place in more than 20 centres worldwide. About 300 patients will be asked to participate.

Detailed information and the list of inclusion and exclusion criteria can be found at this link.