Clinical trial Recruiting at 55 sites Phase 3 Interventional Ages 18 to 81

Masitinib added to riluzole versus placebo for people with ALS

Official title: 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)

Run by AB Science · NCT03127267 · Data updated 2026-11-12

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

Quick facts

PhasePhase 3
Study typeInterventional
Ages18 Years to 81 Years
SexAll
Enrollment495 (estimated)
SponsorAB Science
Industry-sponsored
Drugs / interventionsmasitinib
Locations56 sites (Birmingham, Alabama and 55 other locations)
StatusRecruiting
StartedFebruary 2, 2021
Main results expectedDecember 2027
Study endsDecember 2027
Registry record updatedSeptember 12, 2025
Trial IDNCT03127267 on ClinicalTrials.gov

Is this trial a fit for you?

Likely a 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.

Probably 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.

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

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.

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

Who can join: the full rules

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 it's running

  • University of Alabama at Birmingham
    Birmingham, Alabama, United States
    Recruiting
  • University of Southern California
    Los Angeles, California, United States
    Recruiting
  • University of Kentucky
    Lexington, Kentucky, United States
    Recruiting
  • Johns Hopkins Medicine Brain Science Institute
    Baltimore, Maryland, United States
    Recruiting
  • Lahey Hospital and Medical Center
    Burlington, Massachusetts, United States
    Recruiting
  • University of Virginia Health System
    Charlottesville, Virginia, United States
    Recruiting
Show 44 more locations
  • University Hospital Leuven (UZ Leuven)
    Leuven, Belgium
    Recruiting
  • Bispebjerg Hospital
    Copenhagen, Denmark
    Recruiting
  • CHU de Angers
    Angers, France
    Recruiting
  • Groupe Hospitalier Pellegrin Tripode
    Bordeaux, France
    Recruiting
  • Hôpital neurologique Pierre Wertheimer
    Bron, France
    Recruiting
  • CHU Gabriel Montpied
    Clermont-Ferrand, France
    Recruiting
  • CHU de Lille - Hopital Roger Salengro
    Lille, France
    Recruiting
  • CHU de Limoges - Hôpital Dupuytren
    Limoges, France
    Recruiting
  • CHU de Marseille - Hôpital de la Timone
    Marseille, France
    Recruiting
  • CHRU de Montpellier - Gui de Chauliac
    Montpellier, France
    Recruiting
  • CHU de Nancy - Hopital Central
    Nancy, France
    Recruiting
  • CHU Hôpital Pasteur Nice
    Nice, France
    Recruiting
  • CHRU de Tours - Hopital Bretonneau
    Tours, France
    Recruiting
  • Athens Naval Hospital
    Athens, Greece
    Recruiting
  • Eginition Hospital
    Athens, Greece
    Recruiting
  • University General Hospital of Larissa
    Larissa, Greece
    Recruiting
  • General University Hospital of Patras
    Rio, Greece
    Recruiting
  • Hadassah University Hospital
    Jerusalem, Israel
    Recruiting
  • Tel-Aviv Medical Center Hôpital Sourasky (ICHILOV)
    Tel Aviv, Israel
    Recruiting
  • Ospedale Civile Sant'Agostino - Estense
    Baggiovara, Modena, Italy
    Recruiting
  • ASST degli Spedali Civili di Brescia
    Brescia, Italy
    Recruiting
  • Centro Clinico NeMO Fondazione Serena Onlus
    Gussago, Italy
    Recruiting
  • Clinico Nemo Center (Centro Clinico NeMO Milano)
    Milan, Italy
    Recruiting
  • IRCCS Istituto Auxologico Italiano
    Milan, Italy
    Recruiting
  • Istituti Clinici Scientifici Maugeri IRCCS
    Milan, Italy
    Recruiting
  • San Raffaele Hospital (Ospedale San Raffaele)
    Milan, Italy
    Recruiting
  • University Hospital Maggiore della Carita
    Novara, Italy
    Recruiting
  • Azienda Ospedale-Università Padova
    Padova, Italy
    Recruiting
  • IRCCS Mondino Foundation
    Pavia, Italy
    Recruiting
  • University Hospital in Turin (Azienda Ospedaliero-Universitaria Città della Salute e della Scienza di Torino)
    Torino, Italy
    Recruiting
  • Oslo University Hospital HF Ullevål
    Oslo, Norway
    Recruiting
  • Centrum Medyczne Neuromed
    Bydgoszcz, Poland
    Recruiting
  • Hospital de Santa Maria
    Lisbon, Portugal
    Recruiting
  • Moscow city clinical Hospital after V.M. Buyanov
    Moscow, Russia
    Recruiting
  • Scientific Practical Medical Center "Innovation and Health"
    Novosibirsk, Russia
    Recruiting
  • Clinical Centre of Serbia
    Belgrade, Serbia
    Recruiting
  • Klinicni center Ljubljana
    Ljubljana, Slovenia
    Recruiting
  • Hospital General Universitario de Alicante
    Alicante, Spain
    Recruiting
  • Hospital Universitari de Bellvitge
    Barcelona, Spain
    Recruiting
  • Hospital Carlos III
    Madrid, Spain
    Recruiting
  • Hospital San Rafael
    Madrid, Spain
    Recruiting
  • Clinical Hospital Santiago de Compostela
    Santiago de Compostela, Spain
    Recruiting
  • Hospital Universitario y Politecnico La Fe
    Valencia, Spain
    Recruiting
  • Centralsjukhuset Karlstad (Central Hospital Karlstad)
    Karlstad, Sweden
    Recruiting

+6 more sites. See ClinicalTrials.gov for the full list.

Study contacts

  • Principal investigator: Albert Ludolph, MD, PhD — Department of Neurology, University of Ulm, Germany
  • Study coordinator: Clinical Study Coordinator
  • Email: clinical@ab-science.com
  • Phone: +33(0)147200014

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Other studies for the same condition or treatment. Recruiting studies are listed first.

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How to take part

  1. Review the eligibility criteria above with your treating physician.
  2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
  3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.
Last reviewed 2026-11-14 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.