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)
AB Science
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).
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 actio…
Eligibility
- Age range
- 18–81 years
- Sex
- All
- Healthy volunteers
- No
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 progressio…
Interventions
- DrugMasitinib (6.0)
Masitinib (titration to 6.0 mg/kg/day)
- DrugRiluzole
Riluzole 50 mg tablet, treatment per os
- DrugPlacebo
treatment per os
- DrugMasitinib (4.5)
Masitinib (titration to 4.5 mg/kg/day)
Locations (56)
- University of Alabama at BirminghamBirmingham, Alabama
- University of Southern CaliforniaLos Angeles, California
- University of KentuckyLexington, Kentucky
- Johns Hopkins Medicine Brain Science InstituteBaltimore, Maryland
- Lahey Hospital and Medical CenterBurlington, Massachusetts
- University of Virginia Health SystemCharlottesville, Virginia