Phase 3 Study Comparing Masitinib and Riluzole Combination to Placebo with Riluzole for Treating Amyotrophic Lateral Sclerosis ALS

What this Trial Is About

Researchers are evaluating the effectiveness and safety of masitinib combined with riluzole compared to a placebo combined with riluzole for treating Amyotrophic Lateral Sclerosis ALS. This phase 3 study focuses on patients diagnosed with probable or definite ALS, aiming to understand how masitinib, a drug that targets cells involved in neuroinflammation, might slow disease progression and affect the nervous systems environment. Participants receive oral masitinib at 3.0 mgkgday twice daily, with dose increases to 4.5 mgkgday after 4 weeks, and for some, an additional increase to 6.0 mgkgday after another 4 weeks. Each dose increase includes safety monitoring. Masitinib is given alongside riluzole, a standard ALS treatment at 50 mg twice daily. The control group receives a matching placebo with riluzole. The study is randomized, double-blind, and includes two different masitinib dose escalation schedules. During the 48-week study period, participants undergo regular assessments including functional rating scales ALSFRS-R, quality of life questionnaires ALSAQ-40, survival and progression monitoring, lung function tests FVC, and muscle strength measurements HHD. Researchers measure changes in combined function and survival scores. Safety is closely monitored throughout, with follow-up extending up to 36 months for disease progression or death. Total participation includes baseline screening, treatment, and long-term observation.

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