Amyotrophic Lateral Sclerosis (ALS)Phase 3NCT07174492
Efficacy and Safety of Masitinib in Combination With SoC Versus Placebo in the Treatment of ALS Patients
A Phase 3 study testing Masitinib 4.5 mg/kg/day and Riluzole (100 mg) in people with Amyotrophic Lateral Sclerosis (ALS), run by AB Science at 1 site in Greece.
Trial 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). Read more about this study
- Who is it for?
- Ages 18 to 80 years
- Conditions the study is for
- Amyotrophic Lateral Sclerosis (ALS)
- What is being tested?
- Masitinib 4.5 mg/kg/day and Riluzole (100 mg)
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 412 participants
- Where
- 1 siteGreece
- Study dates
- 1 Jan 2026to 31 Dec 2029
- Phase
- Phase 3
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- The patient must have been diagnosed with clinically probable or definite ALS based on the World Federation of Neurology Revised El Escorial criteria.
- Patients aged 18 to 80 years old and who have either familial or sporadic ALS.
- Patients must have been treated with a stable dose of Riluzole (100 mg/day) for at least 30 days before the entering the trial.
- If they are taking it, patients must have been on a stable dose for at least 30 days before study entry;
- Patients should have experienced the first symptom of ALS no longer than 36 months before the screening visit
- Disease Progression Rate: The ALSFRS-R progression rate (measured between onset and screening) should be between 0.3 and 1.1 point/month
Show all 9Show fewer
- ALSFRS-R Total Score: At screening and baseline, patients need to meet the following criteria:
- Item #3: At least 3 points. Item #12: At least 2 points. Other Items: At least 1 point on each of the remaining 10 items
- Contraception: Female patients of childbearing potential and male patients must use a highly effective method of contraception during the trial and for a specified period afterwards
Who cannot join
- Patients should not have any significant neurological, psychiatric, systemic or organic disease that could interfere with the trial
- Hypersensitivity to masitinib excipients or riluzole
- Lung function: patients with an FVC (forced vital capacity) of less than 70% of the predicted normal value for their gender, height, and age are excluded
- Patients with a weight less than 41 kg and a BMI (body mass index) less than 18 or greater than 35 kg/m² are not eligible.
- Pregnant or nursing female patients at screening and baseline are excluded
- Patients with pre-existing conditions that may be aggravated with the use of masitinib (severe skin toxicities or reactions, liver disorders, severe renal impairment, viral infections, cancer, severe cardiovascular disease, high risk of heart attack and/or stroke).
Show all 9Show fewer
- Patient treated concomitantly with medications that may interact with masitinib
- Participants with a significant pulmonary disorder not attributed to ALS or who require treatments that might complicate the evaluation of the effect of ALS on respiratory function
- Previous treatment with masitinib
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
About this study
Official title: A Prospective, Multicenter, Randomised, Double-blind, Placebo-controlled, Parallel Groups, Phase 3 Trial to Compare the Efficacy and Safety of Masitinib in Combination With Standard of Care Versus Placebo in Combination With Standard of Care 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).
Read the 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 doses of masitinib and matching placebo), comparative study of oral masitinib in the treatment of patients with amyotrophic lateral sclerosis (ALS).
- Conditions
- Amyotrophic Lateral Sclerosis (ALS)
- Being tested
- Masitinib 4.5 mg/kg/day, Placebo, Riluzole (100 mg)
- Medical subject headings
- Amyotrophic Lateral Sclerosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Masitinib 4.5 mg/kg/dayDrug
Masitinib (titration to 4.5 mg/kg/day)
PlaceboDrug
treatment per os
Riluzole (100 mg)Drug
Riluzole 50 mg tablet, treatment per os
| Study group | Role | What they receive |
|---|---|---|
| masitinib + riluzole | Experimental | 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 Receives: Drug: Masitinib 4.5 mg/kg/day; Drug: Riluzole (100 mg) |
| placebo + riluzole | Placebo comparator | Participants receive a matched dose placebo, given orally twice daily, in combination with riluzole at 50 mg b.i.d. Receives: Drug: Placebo; Drug: Riluzole (100 mg) |
What is being measured
The primary outcome is the main question the study is designed to answer.
ALSFRS-R
The primary endpoint is ALSFRS-R after 48-week treatment of masitinib versus matching placebo in patients diagnosed with ALS treated with standard of care.The ALSFRS-R is a validated tool for tracking disability progression in ALS patients. It improves on the original ALSFRS by adding assessments of breathing difficulties and ventilatory support needs, addressing the earlier scale's underweighting of respiratory function. It includes 12 items rated from 0 (can't do) to 4 (normal), with a total score range of 0-48. The ALSFRS-R has strong internal consistency and correlates well with quality of life, highlighting the link between functional ability and well-being in ALS.
Measured: 48 weeks
Secondary outcomes (2)
Progression free survival (PFS)
It is defined as the time from randomization to progression (decline of more than 9 points in ALSFRS-R score from baseline) or death in the trial period.
Measured: 48 weeks
Quality of Life assessment:
Change from baseline in ALSAQ- 40
Measured: 48 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Sep 2025
- Study start1 Jan 2026 (estimated)
- Primary completion31 Dec 2028 (estimated)
- Study completion31 Dec 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Amyotrophic Lateral Sclerosis (ALS)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- A multicenter, randomised, double-blind, placebo-controlled, parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 412 participants (estimated)
Sponsor and investigators
- Lead sponsor
- AB Science
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07174492
- Sponsor's protocol ID
- AB23005
- Other IDs
- 2024-511244-12-01
- Status last verified
- Oct 2025
- First submitted
- 11 Sep 2025
- Last updated
- 3 Oct 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 3 Oct 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Prospective, Multicenter, Randomised, Double-blind, Placebo-controlled, Parallel Groups, Phase 3 Trial to Compare the Efficacy and Safety of Masitinib in Combination With Standard of Care Versus Placebo in Combination With Standard of Care in the Treatment of Patients Suffering From Amyotrophic Lateral Sclerosis (ALS)
- ClinicalTrials.gov ID
- NCT07174492
- Acronym / sponsor ID
- AB23005
- Conditions
- Amyotrophic Lateral Sclerosis (ALS)
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 412 participants (estimated)
- Lead sponsor
- AB ScienceIndustry (pharma, biotech, devices)
- Study start
- 1 Jan 2026 (estimated)
- Primary completion
- 31 Dec 2028 (estimated)
- Study completion
- 31 Dec 2029 (estimated)
- First posted
- 16 Sep 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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).
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 doses of masitinib and matching placebo), comparative study of oral masitinib in the treatment of patients with amyotrophic lateral sclerosis (ALS).
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
- Model description
- A multicenter, randomised, double-blind, placebo-controlled, parallel groups
| Arm | Type | Receives |
|---|---|---|
| masitinib + riluzole | Experimental | Drug: Masitinib 4.5 mg/kg/day, Drug: Riluzole (100 mg)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 |
| placebo + riluzole | Placebo comparator | Drug: Placebo, Drug: Riluzole (100 mg)Participants receive a matched dose placebo, given orally twice daily, in combination with riluzole at 50 mg b.i.d. |
Who may qualify
- Ages
- 18 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (9 listed)
- The patient must have been diagnosed with clinically probable or definite ALS based on the World Federation of Neurology Revised El Escorial criteria.
- Patients aged 18 to 80 years old and who have either familial or sporadic ALS.
- Patients must have been treated with a stable dose of Riluzole (100 mg/day) for at least 30 days before the entering the trial.
- If they are taking it, patients must have been on a stable dose for at least 30 days before study entry;
- Patients should have experienced the first symptom of ALS no longer than 36 months before the screening visit
Key exclusion criteria (9 listed)
- Patients should not have any significant neurological, psychiatric, systemic or organic disease that could interfere with the trial
- Hypersensitivity to masitinib excipients or riluzole
- Lung function: patients with an FVC (forced vital capacity) of less than 70% of the predicted normal value for their gender, height, and age are excluded
- Patients with a weight less than 41 kg and a BMI (body mass index) less than 18 or greater than 35 kg/m² are not eligible.
- Pregnant or nursing female patients at screening and baseline are excluded
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Masitinib 4.5 mg/kg/dayMasitinib (titration to 4.5 mg/kg/day) | Drug | masitinib + riluzole |
| Placebotreatment per os | Drug | placebo + riluzole |
| Riluzole (100 mg)Riluzole 50 mg tablet, treatment per os | Drug | masitinib + riluzole, placebo + riluzole |
Primary outcome
ALSFRS-R
Time frame: 48 weeks
The primary endpoint is ALSFRS-R after 48-week treatment of masitinib versus matching placebo in patients diagnosed with ALS treated with standard of care.The ALSFRS-R is a validated tool for tracking disability progression in ALS patients. It improves on the original ALSFRS by adding assessments of breathing difficulties and ventilatory support needs, addressing the earlier scale's underweighting of respiratory function. It includes 12 items rated from 0 (can't do) to 4 (normal), with a total score range of 0-48. The ALSFRS-R has strong internal consistency and correlates well with quality of life, highlighting the link between functional ability and well-being in ALS.
2 secondary outcomes
Progression free survival (PFS)
Time frame: 48 weeks
Quality of Life assessment:
Time frame: 48 weeks
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Oct 2025.
Related trials for Amyotrophic Lateral Sclerosis (ALS)
RecruitingPhase 1/2
Study is to Assess the Safety and Tolerability of VTx-002 in Participants With ALS
Amyotrophic Lateral Sclerosis
Not yet recruiting
A Study on the Addition of Alsum30 for the Treatment of Amyotrophic Lateral Sclerosis
Amyotrophic Lateral Sclerosis (ALS), Bacillus Coagulans
Active, not recruiting
Functional Outcomes and Control Using Synchron BCI - Australia
Neurologic Disorder, Neurologic Diseases
Not yet recruitingPhase 1
Safety, Tolerability, and Pharmacodynamic Exploration of Orelabrutinib in Patients With Amyotrophic Lateral Sclerosis (ALS)
Amyotrophic Lateral Sclerosis (ALS)
Recruiting
Clinical Outcome Assessment for AT & BCI
Spinal Cord Injury, ALS (Amyotrophic Lateral Sclerosis)
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Masitinib 4.5 mg/kg/day and Riluzole (100 mg) in people with Amyotrophic Lateral Sclerosis (ALS), run by AB Science at 1 site in Greece.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 9 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: opening soon, as of the sponsor's last update on 3 Oct 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Greece. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Clinical Study Coordinator (+33(0)147200014, clinical@ab-science.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
ALSFRS-R (time frame: 48 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 3 Oct 2025.