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

NCT07174492 | Not Yet Recruiting Phase 3 DMC FDA Drug

• 2 other identifiers: AB230052024-511244-12-01
• Study Type: interventional
• Target: 412
• Locations: 1 country
• Sites: 1

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 (ALS)

Outcome Measures

Primary Outcomes (1)

• 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.

  48 weeks

Secondary Outcomes (2)

• Progression free survival (PFS)

  48 weeks

• Quality of Life assessment:

  48 weeks

Study Arms (2)

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

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.

Drug: Placebo; Drug: Riluzole (100 mg)

Interventions

Masitinib 4.5 mg/kg/day DRUG

Masitinib (titration to 4.5 mg/kg/day)

masitinib + riluzole

Placebo DRUG

treatment per os

placebo + riluzole

Riluzole (100 mg) DRUG

Riluzole 50 mg tablet, treatment per os

masitinib + riluzole; placebo + riluzole

Eligibility Criteria

• Age: 18 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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
• 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

You may not qualify if:

• 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).
• 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

Sponsors & Collaborators

• AB Science: lead

Study Sites (1)

Aiginition Hospital

Athens, Greece

Location

MeSH Terms

Conditions

Amyotrophic Lateral Sclerosis

Interventions

masitinib; Riluzole

Condition Hierarchy (Ancestors)

Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases; Motor Neuron Disease; Neurodegenerative Diseases; TDP-43 Proteinopathies; Neuromuscular Diseases; Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases

Intervention Hierarchy (Ancestors)

Thiazoles; Sulfur Compounds; Organic Chemicals; Benzothiazoles; Azoles; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Heterocyclic Compounds, 2-Ring; Heterocyclic Compounds, Fused-Ring

Central Study Contacts

Clinical Study Coordinator

CONTACT

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

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: A multicenter, randomised, double-blind, placebo-controlled, parallel groups

Study Record Dates

• First Submitted: September 11, 2025
• First Posted: September 16, 2025
• Study Start: January 1, 2026
• Primary Completion (Estimated): December 31, 2028
• Study Completion (Estimated): December 31, 2029
• Last Updated: October 3, 2025

Record last verified: 2025-10

Locations

GR (1)