MIAMI, FL – The Principal Investigator at the University of Miami announced that enrollment has closed for the FDA-sponsored phase II/III randomized, double-blind, placebo-controlled trial of the experimental drug Arimoclomol, in SOD1+ familial ALS. This administrative decision to close enrollment at the end of the phase II component was made in consultation with the trial’s steering committee. The decision was based on the relatively slow recruitment rate and the resulting need to raise additional funds to support the continued development of Arimoclomol in ALS. Trial data have not been analyzed, and results are not available. All investigators remain blinded to treatment allocation.
Patients who have consented by June 30, 2014 to participate in the trial will have the opportunity to enroll if they are eligible. All new enrollees as well as patients already enrolled in the trial will remain on study drug through the originally planned 12 months of follow-up. Once follow-up has been completed for all enrolled patients, investigators will proceed to analyze and publish the phase II trial results. Orphazyme, the company that owns and develops Arimoclomol, has expressed a keen interest to support a phase III clinical trial if the phase II data are promising.
Investigators believe that the decision to close enrollment and to analyze and publish the results of the phase II component of this trial represents the most efficient and expeditious way forward in developing Arimoclomol as a potential therapeutic agent for patients with ALS.