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).
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 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.
Parallel
Masitinib 4.5 mg/kg/day
DrugMasitinib (titration to 4.5 mg/kg/day)
Placebo
Drugtreatment per os
Riluzole (100 mg)
DrugRiluzole 50 mg tablet, treatment per os
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.
Progression free survival (PFS)
It is defined as the time from randomization to progression (decline of more than 9 points in ALSFRS-R score from baseline) or death in the trial period.
Quality of Life assessment:
Change from baseline in ALSAQ- 40
This trial is not recruiting at the moment. You can still explore other options:
