Efficacy and Safety of Masitinib Versus Placebo in the Treatment of ALS Patients

September 8, 2025 updated by: AB Science

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)

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).

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Recruiting

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Detailed Description

Masitinib is a selective, oral tyrosine kinase inhibitor with neuroprotective capability demonstrated via numerous preclinical studies. Two of masitinib's main cellular targets are the mast cell and microglia cell. It is well-established that mast cells play a prominent role in neuroinflammatory processes. Microglia, resident immune cells of the central nervous system (CNS), also constitute an important source of neuroinflammatory mediators and may have fundamental roles in numerous neurodegenerative disorders. The development of masitinib in ALS is therefore based on the pharmacological action of masitinib in microglia cells and mast cells, thereby slowing microglial-related disease progression, reducing neuro-inflammation, and modulating the neuronal microenvironment in both central and peripheral nervous systems. This is a multicenter, double-blind, randomized, placebo-controlled, parallel-group (two ascending dose titrations of masitinib and matching placebo), comparative study of oral masitinib in the treatment of patients with amyotrophic lateral sclerosis (ALS).

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Estimated)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
495

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact

The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.

Study Locations

  • Belgium
      • Leuven, Belgium
        • Recruiting
        • University Hospital Leuven (UZ Leuven)
  • Denmark
      • Copenhagen, Denmark
        • Recruiting
        • Bispebjerg Hospital
  • France
      • Angers, France
        • Recruiting
        • CHU de Angers
      • Bordeaux, France
        • Recruiting
        • Groupe Hospitalier Pellegrin Tripode
      • Bron, France
        • Recruiting
        • Hôpital Neurologique Pierre Wertheimer
      • Clermont-Ferrand, France
        • Recruiting
        • CHU Gabriel Montpied
      • Lille, France
        • Recruiting
        • CHU de Lille - Hôpital Roger Salengro
      • Limoges, France
        • Recruiting
        • Chu de Limoges - Hopital Dupuytren
      • Marseille, France
        • Recruiting
        • CHU de Marseille - Hôpital de la Timone
      • Montpellier, France
        • Recruiting
        • CHRU de Montpellier - Gui de Chauliac
      • Nancy, France
        • Recruiting
        • CHU de Nancy - Hôpital Central
      • Nice, France
        • Recruiting
        • CHU Hôpital Pasteur Nice
      • Tours, France
        • Recruiting
        • CHRU de Tours - Hôpital Bretonneau
  • Germany
      • Ulm, Germany, 89081
        • Not yet recruiting
        • Department of Neurology, University of Ulm
  • Greece
      • Athens, Greece
        • Recruiting
        • Athens Naval Hospital
      • Athens, Greece
        • Recruiting
        • Eginition Hospital
      • Larissa, Greece
        • Recruiting
        • University General Hospital of Larissa
      • Rio, Greece
        • Recruiting
        • General University Hospital of Patras
  • Israel
      • Jerusalem, Israel
        • Recruiting
        • Hadassah University Hospital
      • Tel Aviv, Israel
        • Recruiting
        • Tel-Aviv Medical Center Hôpital Sourasky (ICHILOV)
  • Italy
      • Brescia, Italy
        • Recruiting
        • Asst Degli Spedali Civili Di Brescia
      • Gussago, Italy
        • Recruiting
        • Centro Clinico NeMO Fondazione Serena Onlus
      • Milan, Italy
        • Recruiting
        • IRCCS Istituto Auxologico Italiano
      • Milan, Italy
        • Recruiting
        • Clinico Nemo Center (Centro Clinico NeMO Milano)
      • Milan, Italy
        • Recruiting
        • Istituti Clinici Scientifici Maugeri IRCCS
      • Milan, Italy
        • Recruiting
        • San Raffaele Hospital (Ospedale San Raffaele)
      • Novara, Italy
        • Recruiting
        • University Hospital Maggiore della Carita
      • Padua, Italy
        • Recruiting
        • Azienda Ospedale-Universita Padova
      • Pavia, Italy
        • Recruiting
        • IRCCS Mondino Foundation
      • Torino, Italy
        • Recruiting
        • University Hospital in Turin (Azienda Ospedaliero-Universitaria Città della Salute e della Scienza di Torino)
    • Modena
      • Baggiovara, Modena, Italy
        • Recruiting
        • Ospedale Civile Sant'Agostino - Estense
  • Norway
      • Oslo, Norway
        • Recruiting
        • Oslo University Hospital HF Ullevål
  • Poland
      • Bydgoszcz, Poland
        • Recruiting
        • Centrum Medyczne Neuromed
  • Portugal
      • Lisbon, Portugal
        • Recruiting
        • Hospital de Santa Maria
  • Russia
      • Moscow, Russia
        • Recruiting
        • Moscow city clinical Hospital after V.M. Buyanov
      • Novosibirsk, Russia
        • Recruiting
        • Scientific Practical Medical Center "Innovation and Health"
  • Serbia
      • Belgrade, Serbia
        • Recruiting
        • Clinical Centre of Serbia
  • Slovenia
      • Ljubljana, Slovenia
        • Recruiting
        • Klinicni center Ljubljana
  • Spain
      • Alicante, Spain
        • Recruiting
        • Hospital General Universitario de Alicante
      • Barcelona, Spain
        • Recruiting
        • Hospital Universitari de Bellvitge
      • Madrid, Spain
        • Recruiting
        • Hospital Carlos III
      • Madrid, Spain
        • Recruiting
        • Hospital San Rafael
      • Santiago de Compostela, Spain
        • Recruiting
        • Clinical Hospital Santiago de Compostela
      • Valencia, Spain
        • Recruiting
        • Hospital Universitario y Politécnico La Fe
  • Sweden
      • Karlstad, Sweden
        • Recruiting
        • Centralsjukhuset Karlstad (Central Hospital Karlstad)
      • Malmo, Sweden
        • Recruiting
        • Skåne University Hospital
      • Umeå, Sweden
        • Recruiting
        • Norrlands Universitetssjukhus
  • Ukraine
      • Kharkiv, Ukraine
        • Recruiting
        • The State Institution of Neurology, Psychiatry and Narcology of NAMS of Ukraine
      • Kyiv, Ukraine
        • Recruiting
        • Medical Center of LLC Medical Center Dopomoga Plus
      • Lviv, Ukraine
        • Recruiting
        • Communal Non-Profit Enterprise of Lviv Regional Council, Lviv Regional Clinical Hospital, Neurological Department
  • United States
    • Alabama
      • Birmingham, Alabama, United States, 35294
        • Recruiting
        • University of Alabama at Birmingham
    • California
      • Los Angeles, California, United States, 90007
        • Recruiting
        • University of Southern California
    • Kentucky
      • Lexington, Kentucky, United States, 40506
        • Recruiting
        • University Of Kentucky
    • Maryland
      • Baltimore, Maryland, United States, 21205
        • Recruiting
        • Johns Hopkins Medicine Brain Science Institute
    • Massachusetts
      • Burlington, Massachusetts, United States, 01805
        • Recruiting
        • Lahey Hospital and Medical Center
    • Virginia
      • Charlottesville, Virginia, United States, 22903
        • Recruiting
        • University of Virginia Health System

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

18 years to 81 years (Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Double

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
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
Drug: Riluzole
Riluzole 50 mg tablet, treatment per os
Other Names:
  • Rilutek
Drug: Masitinib (4.5)
Masitinib (titration to 4.5 mg/kg/day)
Other Names:
  • AB1010
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.
Drug: Riluzole
Riluzole 50 mg tablet, treatment per os
Other Names:
  • Rilutek
Drug: Masitinib (6.0)
Masitinib (titration to 6.0 mg/kg/day)
Other Names:
  • AB1010
Placebo Comparator: Placebo & Riluzole
Participants receive a matched dose placebo, given orally twice daily, in combination with riluzole at 50 mg b.i.d.
Drug: Riluzole
Riluzole 50 mg tablet, treatment per os
Other Names:
  • Rilutek
Drug: Placebo
treatment per os
Other Names:
  • Placebo Oral Tablet

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
ALSFRS-R
Time Frame: 48 weeks
Change in Amyotrophic Lateral Sclerosis functional rating scale (ALSFRS)-Revised.
48 weeks

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
ALSAQ-40
Time Frame: 48 weeks
Change in ALS quality of life patient questionnaire (ALSAQ-40)
48 weeks
PFS
Time Frame: From day of randomization to disease progression or death, assessed for a maximum of 36 months
Progression free survival (PFS) is defined as the time from randomization to progression (decline of more than 9 points in ALSFRS-R score from baseline) or death
From day of randomization to disease progression or death, assessed for a maximum of 36 months
FVC
Time Frame: 48 weeks
Change in Forced Vital Capacity (FVC)
48 weeks
HHD
Time Frame: 48 weeks
Change in evaluation of upper- and lower-limb muscle strength using hand-held dynamometry (HHD)
48 weeks
Change in the Combined Assessment of Function and Survival (CAFS) score from baseline to week 48
Time Frame: 48 weeks
CAFS ranks patients' clinical outcomes based on survival time and change in the ALS Functional Rating Scale-Revised (ALSFRS-R) score. Each patient's outcome is compared to every other patient's outcome, assigned a score, and the summed scores are ranked. The mean rank score for each treatment group can then be calculated. A higher mean CAFS score indicates a better group outcome.
48 weeks

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
AB Science

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Principal Investigator: Albert Ludolph, MD, PhD, Department of Neurology, University of Ulm, Germany

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
February 2, 2021

Primary Completion (Estimated)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
December 1, 2027

Study Completion (Estimated)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
December 1, 2027

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
April 13, 2017

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
April 20, 2017

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
April 25, 2017

Study Record Updates

Last Update Posted (Estimated)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
September 12, 2025

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
September 8, 2025

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
September 1, 2025

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • AB19001

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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