A multicenter, randomized, double-blind, placebo-controlled, parallel group trial to evaluate the efficacy and safety of arimoclomol in amyotropic lateral sclerosis (ALS)
Screening up to 4 weeks Treatment up to 76 weeks
Inclusion Criteria:
- Subject meets revised El Escorial criteria for clinically possible, clinically
probable / clinically probable ALS laboratory-supported, clinically definite ALS or
clinically definite familial ALS laboratory-supported
- 18 months or less since first appearance of weakness (e.g. limb weakness, dysarthria,
dysphagia, shortness of breath)
- ALS Functional Rating Scale-Revised (ALSFRS-R) equal to or above 35 and erect (seated)
Slow Vital Capacity (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 and 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