Recruiting
Phase 3

Masitinib

Sponsor:

AB Science

Code:

NCT03127267

Conditions

Amyotrophic Lateral Sclerosis

Eligibility Criteria

Sex: All

Age: 18 - 70+

Healthy Volunteers: Not accepted

Interventions

Masitinib (6.0)

Riluzole

Placebo

Masitinib (4.5)

Study Details

Brief summary:

The objective is to compare the efficacy and safety of masitinib in combination with riluzole versus matched placebo in combination with riluzole for the treatment of Amyotrophic Lateral Sclerosis (ALS).

Conditions

Amyotrophic Lateral Sclerosis

Study ID

NCT03127267

Start date

Feb 2, 2021

Status verified date

Sep, 2025

Completion date

Dec, 2027

Anticipated

Primary completion date

Dec, 2027

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 18 - 70+

Healthy Volunteers: Not accepted

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

Study Design

Enrollment

495 participants

Anticipated

Allocation

Randomized

Intervention Model

Parallel Assignment

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: Masitinib (4.5) & Riluzole

Participants receive masitinib (3.0 mg/kg/day), given orally twice daily, with a dose escalation to 4.5 mg/kg/day after 4 weeks of treatment. Each ascending dose titration is subjected to a safety control. Masitinib will be administered as an add-on to riluzole at 50 mg b.i.d

experimental: Masitinib (6.0) & Riluzole

Participants receive masitinib (3.0 mg/kg/day), given orally twice daily, with a dose escalation to 4.5 mg/kg/day after 4 weeks of treatment, followed by dose escalation to 6.0 mg/kg/day after 4 weeks of treatment. Each ascending dose titration is subjected to a safety control. Masitinib will be administered as an add-on to riluzole at 50 mg b.i.d.

placebo comparator: Placebo & Riluzole

Participants receive a matched dose placebo, given orally twice daily, in combination with riluzole at 50 mg b.i.d.

Interventions

Masitinib (6.0)

Masitinib (titration to 6.0 mg/kg/day)

Riluzole

Riluzole 50 mg tablet, treatment per os

Placebo

treatment per os

Masitinib (4.5)

Masitinib (titration to 4.5 mg/kg/day)

Primary outcome measure

  • ALSFRS-R [ Time Frame: 48 weeks ]

Central Contacts and Locations

Central contacts

Clinical Study Coordinator

+33(0)147200014clinical@ab-science.com

Locations

University of Alabama at Birmingham

Recruiting

Birmingham, Alabama, United States, 35294

University of Southern California

Recruiting

Los Angeles, California, United States, 90007

University of Kentucky

Recruiting

Lexington, Kentucky, United States, 40506

Johns Hopkins Medicine Brain Science Institute

Recruiting

Baltimore, Maryland, United States, 21205

Lahey Hospital and Medical Center

Recruiting

Burlington, Massachusetts, United States, 01805

University of Virginia Health System

Recruiting

Charlottesville, Virginia, United States, 22903

More Information

Sponsor

AB Science

Last update posted

Sep 12, 2025

Last verified

Sep, 2025

Keywords

  • Amyotrophic Lateral Sclerosis
  • ALS
  • Tyrosine kinase inhibitor
  • Lou Gehrig's disease
  • Charcot's disease
  • Motor Neuron disease
  • MND

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-10. This information was provided to ClinicalTrials.gov by AB Science on 2025-09-12.