Orphazyme Arimoclomol (ORARIALS-01) study

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Active but not recruiting anymore

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

This phase 3 clinical trial aims to evaluate the effect of arimoclomol on the disease progression in patients with ALS. Arimoclomol stimulates a stress response in cells, so misfolded proteins can be brought back into their original state. Since ALS is characterized by an accumulation of misfolded proteins in motor neuron cells, one hopes that this treatment strategy can slow down the disease. 

A dose of 1200 mg per day will be compared with placebo at a ratio of 2:1. In order to be eligible to participate the disease may not yet have progressed  too far: patients with the disease up to 18 months, must have a score of ALSFRS-R of ≥ 35 and a lung function (SVC) of ≥ 70%. The study takes place in over 20 centers worldwide. There will be about 300 patients being asked to participate.

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