NCT03127267

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

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)

• Status: Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 495 (estimated)
• Sponsor: AB Science · Industry
• Sex: All
• Age: 18 Years – 81 Years
• Healthy volunteers: Not accepted

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).

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).

Conditions

• Amyotrophic Lateral Sclerosis

Interventions

Type	Name	Description
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)

Timeline

• Start date: 2021-02-02
• Primary completion: 2027-12-01
• Completion: 2027-12-01
• First posted: 2017-04-25
• Last updated: 2025-09-12

Locations

56 sites across 17 countries: United States, Belgium, Denmark, France, Germany, Greece, Israel, Italy, Norway, Poland, Portugal, Russia, Serbia, Slovenia, Spain, Sweden, Ukraine

Regulatory

• FDA-regulated drug study