NCT03127267 · AB Science
Efficacy and Safety of Masitinib Versus Placebo in the Treatment of ALS Patients
What this study is about
The objective is to compare the effectiveness and safety of masitinib in combination with riluzole versus matched placebo in combination with riluzole for the treatment of Amyotrophic Lateral Sclerosis (ALS).
View original scientific description
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).
Interventions
DRUG
Masitinib (6.0)
Masitinib (titration to 6.0 mg/kg/day)
DRUG
Riluzole
Riluzole 50 mg tablet, treatment per os
DRUG
Placebo
treatment per os
DRUG
Masitinib (4.5)
Masitinib (titration to 4.5 mg/kg/day)
Primary outcome measures
ALSFRS-R
Time frame: 48 weeks
Change in Amyotrophic Lateral Sclerosis functional rating scale (ALSFRS)-Revised.
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
Inclusion criteria
- 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
Where
- Birmingham, Alabama
- Los Angeles, California
- Lexington, Kentucky
- Baltimore, Maryland
- Burlington, Massachusetts
- Charlottesville, Virginia
Related conditions & keywords
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced Sep 12, 2025 · Source of record for eligibility and locations