Masitinib as an add-on therapy to riluzole in patients with amyotrophic lateral sclerosis: a randomized clinical trial

Publié le 2 janvier 2020 - Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration

Auteurs : Jesus Mora, Angela Genge, Adriano Chiò, Conrado Estol, Delia Chaverri, Maria Hernández, Saúl Marín, Javier Mascias, Gabriel Rodríguez, Monica Povedano, Andrés Paipa, Raúl Dominguez, Josep Gámez, Maria Salvado, Christian Lunetta, Carlos Ballario, Nilo Riva, Jessica Mandrioli, Alain Moussy, Jean-Pierre Kinet, Christian Auclair, Patrice Dubreuil, Vincent Arnold, Colin Mansfield, Olivier Hermine

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Objective: To assess masitinib in the treatment of ALS. Methods: Double-blind study, randomly assigning 394 patients (1:1:1) to receive riluzole (100 mg/d) plus placebo or masitinib at 4.5 or 3.0 mg/kg/d. Following a blinded transition from phase 2 to phase 2/3, a prospectively defined two-tiered design was implemented based on ALSFRS-R progression rate from disease-onset to baseline (ΔFS). This approach selects a more homogeneous primary efficacy population ("Normal Progressors", ΔFS