Arimoclomol in Amyotropic Lateral Sclerosis

• Register: ClinicalTrials.gov
• Last refreshed on: 4 September 2023
• Main ID: NCT03491462
• Date of registration: 27/03/2018
• Prospective Registration: Yes
• Primary sponsor: ZevraDenmark
• Public title: Arimoclomol in Amyotropic Lateral Sclerosis
• Scientific title: A Phase 3, Randomised, Placebo-Controlled Trial of Arimoclomol in Amyotropic Lateral Sclerosis
• Date of first enrolment: July 31, 2018
• Target sample size: 245
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/ct2/show/NCT03491462
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
• Phase: Phase 3

Countries of recruitment

Belgium, Canada, Denmark, France, Germany, Italy, Netherlands, Poland, Spain, Sweden, Switzerland, United Kingdom, United States

Contacts

• Name: Michael Benatar, MD PhD
• Affiliation: University of Miami

Key inclusion & exclusion criteria

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



Age minimum: 18 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Amyotrophic Lateral Sclerosis

Intervention(s)

Drug: Placebo

Drug: Arimoclomol

Primary Outcome(s)

Combined Assessment of Function and Survival (CAFS) [Time Frame: Over 76 Weeks]

Secondary Outcome(s)

Change From Baseline to Week 76 (or End-of-trial) in the Revised ALS Functional Rating Scale (ALSFRS-R) [Time Frame: Week 76 (or end of trial)]

Time to Permanent Assisted Ventilation (PAV) / Tracheostomy / Death [Time Frame: Over 76 weeks]

Change From Baseline to Week 76 (or End-of-trial) in Percent (%) Predicted Slow Vital Capacity (SVC) [Time Frame: Week 76 (or end of trial)]

Secondary ID(s)

ORARIALS-01

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 24/08/2023
• URL: https://clinicaltrials.gov/ct2/show/results/NCT03491462