Arimoclomol in Amyotropic Lateral Sclerosis

• Status: Completed
• Trial end date: 2020-12-18
• Target enrollment: 0
• Participant gender: All

Summary

A multicenter, randomized, double-blind, placebo-controlled, parallel group trial to evaluate the efficacy and safety of arimoclomol in amyotropic lateral sclerosis (ALS)

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Orphazyme

Criteria

Inclusion Criteria:

• Subject meets revised El Escorial criteria for clinically possible, clinically probable / Clinically probable ALS laboratory-supported or clinically definite ALS, or familial ALS

• 18 months or less since first appearance of weakness (e.g. limb weakness, dysarthria, dysphagia, shortness of breath).

• ALSFRS-R equal to or above 35 and erect (seated) SVC% predicted equal to or above 70% at screening

Exclusion Criteria:

• Tracheostomy or use of non-invasive ventilation for more than 2 hours during waking hours at the time of screening or baseline

• pregnant or breast-feeding

• current or anticipated use of diaphragmatic pacing

• Any other relevant medically significant condition which could present risk to the subject or interfere with the assessment of safety or has an increased risk of causing death during the trial