A Phase 3, Randomised, Placebo-Controlled Trial of Arimoclomol in Amyotropic Lateral Sclerosis

ORARIALS-01

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

2018-07-31

2020-12-18

2020-12-18

Completed

Early phase I

245

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

{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE3'], 'designInfo': {'allocation': 'RANDOMIZED', 'interventionModel': 'PARALLEL', 'interventionModelDescription': 'Placebo-controlled, parallel group', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'QUADRUPLE', 'maskingDescription': 'Double-blind', 'whoMasked': ['PARTICIPANT', 'CARE_PROVIDER', 'INVESTIGATOR', 'OUTCOMES_ASSESSOR']}}, 'enrollmentInfo': {'count': 245, 'type': 'ACTUAL'}}

2023-08-24