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Masitinib added to riluzole versus placebo for people with ALS

Phase 3 Study to Compare the Efficacy and Safety of Masitinib in Combination With Riluzole Versus Placebo in Combination With Riluzole in the Treatment of Patients Suffering From Amyotrophic Lateral Sclerosis (ALS)

Phase 3 Interventional AB Science · NCT03127267

This trial tests whether adding masitinib to standard riluzole treatment slows disease progression in people with ALS.

Quick facts

PhasePhase 3
Study typeInterventional
Enrollment495 (estimated)
Ages18 Years to 81 Years
SexAll
SponsorAB Science Industry-sponsored
Drugs / interventionsmasitinib
Locations56 sites (Birmingham, Alabama and 55 other locations)
Trial IDNCT03127267 on ClinicalTrials.gov

What this trial studies

Masitinib is an oral selective tyrosine kinase inhibitor that targets mast cells and microglia to reduce neuroinflammation, with supportive neuroprotective findings in preclinical work. This multicenter, double-blind, randomized, placebo-controlled phase 3 study compares two ascending dose regimens of oral masitinib plus standard riluzole against matching placebo plus riluzole. Eligible participants must have probable or definite ALS within 24 months of diagnosis, be on a stable riluzole dose, and show recent functional decline on the ALSFRS-R prior to randomization. The trial tracks functional outcomes and safety measures to determine whether masitinib provides additional clinical benefit over riluzole alone.

Who should consider this trial

Good fit: Ideal candidates are people with laboratory-supported probable, probable, or definite ALS within 24 months of diagnosis who are on a stable riluzole dose and have shown recent ALSFRS-R decline.

Not a fit: Patients with longer-standing or more advanced disease, those not taking riluzole, or those who do not meet the required ALSFRS-R progression criteria are unlikely to benefit or be eligible for this protocol.

Why it matters

Potential benefit: If successful, adding masitinib to riluzole could slow functional decline and help patients maintain independence longer than riluzole alone.

How similar studies have performed: Masitinib demonstrated neuroprotective effects in preclinical models and earlier-phase clinical studies suggested potential benefit, but larger phase 3 confirmation is needed.

Eligibility criteria

Show full inclusion / exclusion criteria 
Main inclusion criteria include:

* Patients diagnosed with laboratory supported probable, clinically probable or definite ALS according to the World Federation of Neurology Revised El Escorial criteria
* Patient with a familial or sporadic ALS
* ALS disease duration from diagnosis no longer than 24 months at the screening visit
* Patient treated with a stable dose of riluzole (100 mg/day) for at least 12 weeks days prior to the baseline visit
* Patient with an ALSFRS-R score progression between onset of the disease and screening of \> 0.3 per month, confirmed with an ALSFRS-R score progression of ≥ 1 point during a 12-week run-in period between screening and randomization.
* Patient with a score, at screening, of at least 26 overall, including a score of at least 3 on item #3 and at least 2 on each of the 12 ALSFRS-R individual component items and with a score, at randomization, of at least 2 on each of the 12 ALSFRS-R individual component items

Main exclusion criteria include:

* Patient with dementia or significant neurological, psychiatric, systemic or organic disease, uncontrolled or that may interfere with the conduct of the trial or its results
* Patient with a FVC \< 60% predicted normal value for gender, height, and age at screening and baseline
* Pregnant, or nursing female patient

Where this trial is running

Birmingham, Alabama and 55 other locations

+6 more sites — see ClinicalTrials.gov for the full list.

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions Amyotrophic Lateral SclerosisALSTyrosine kinase inhibitorLou Gehrig's diseaseCharcot's diseaseMotor Neuron diseaseMND
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.