Arimoclomol in Amyotropic Lateral Sclerosis

Zevra Therapeutics

Status and phase: Completed; Phase 3

Conditions: Amyotrophic Lateral Sclerosis

Treatments: Drug: Arimoclomol; Drug: Placebo

Study type: Interventional

Funder types: Industry

Identifiers

NCT03491462

ORARIALS-01

Details and patient eligibility

About

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

Full description

Screening up to 4 weeks Treatment up to 76 weeks

Enrollment: 245 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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245 participants in 2 patient groups, including a placebo group

Arimoclomol

Experimental group

Description:

248 mg arimoclomol base (equivalent to 400 mg arimoclomol citrate) 3 times daily

Treatment:

Drug: Arimoclomol

Placebo

Placebo Comparator group

Description:

248 mg matching placebo 3 times daily

Treatment:

Drug: Placebo