Open-label extension trial of Arimoclomol in Patients with ALS
Research type
Research Study
Full title
Open-label, Non-randomised Extension Trial to Assess the Long-Term Safety and Efficacy of 1200 mg/day Arimoclomol 400 mg Three Times a Day (t.i.d.) in Subjects with Amyotrophic Lateral Sclerosis (ALS) who have Completed the ORARIALS-01 Trial
IRAS ID
260607
Contact name
Michael Hanna
Contact email
Eudract number
2019-000374-39
Clinicaltrials.gov Identifier
Duration of Study in the UK
2 years, 1 months, 1 days
Research summary
ALS is a progressive, fatal neurodegenerative disorder. The loss of motor neurons causes the muscles under their control to weaken and waste away, leading to paralysis and death, usually due to respiratory failure, within 3-5 years of symptom onset.
Arimoclomol amplifies the heat shock response under conditions of cellular stress. The heat shock response promotes natural folding of nascent proteins and refolding of damaged or mutated proteins via enhanced heat shock protein expression, a mechanism of action that is thought to be highly relevant to an essential and early pathophysiological event that leads to neurodegeneration in ALS
Subjects diagnosed with ALS must have completed the double-blinded ORARIALS-01 trial. They will either have met the surrogate survival endpoint (tracheostomy or PAV) or they will have completed the 76 weeks randomised treatment period. The end of treatment visit of the double-blinded ORARIALS-01 trial corresponds to the Visit 1 of the OLE trial. All subjects will receive open label arimoclomol treatment giving them the opportunity to experience improvement in their condition.Study-related tests and procedures include: neurological and physical examination,
completion of questionnaires, blood, urine analyses & ECG.REC name
London - Brighton & Sussex Research Ethics Committee
REC reference
19/LO/0974
Date of REC Opinion
15 Jul 2019
REC opinion
Favourable Opinion