ALS amyotrophic lateral sclerosis
Conditions
Interventions
None listed
Sponsors
AB Science
Eligibility
Age
18 Years to 99 Years
Inclusion criteria
Inclusion criteria: 1.Patient, male or female, diagnosed with laboratory supported probable, clinically probable or definite ALS according to the World Federation of Neurology Revised El Escorial criteria 2.Patient with a familial or sporadic ALS 3.Patient aged between 18 and 75 years old inclusive at screening 4.Patient treated with a stable dose of Riluzole (100 mg/day) for at least 12 weeks prior to the baseline visit 5.Patient with an ALS disease duration from diagnosis no longer than 24 months at screening 6. Patient with an ALSFRS-R total score progression between onset of the disease and screening of > 0.3 and
Exclusion criteria
Exclusion criteria: 1. Patient with dementia or significant neurological, psychiatric, systemic or organic disease, uncontrolled or that may interfere with the conduct of the trial or its results 2. Patient with hypersensitivity to masitinib excipients 3. Patient with an FVC 30 kg/m² at screening and at baseline 5. Pregnant, or nursing female patient 6. Patient with history (or family history) of severe skin toxicities or reactions 7. Patients treated by drugs known to be at high risk for Stevens-Johnson Syndrome or for Drug Reaction with Eosinophilia and Systemic Symptoms (DRESS) syndrome 8. Patient with history of severe bone marrow disorders such as agranulocytosis or aplasia, or with abnormal laboratory results from local laboratory assessments at screening and baseline : - Neutropenia with ANC 2 ULN at baseline, or - Total bilirubin level > 1.5 ULN at baseline, or - Both hepatic transaminase levels and total bilirubin level outside of the normal ranges at screening and baseline, or - Albuminemia 30 mg/dL (1+) on dipstick; in case of the proteinuria * 1+ on the dipstick, 24 hours proteinuria must be > 1.5g/24 hours 11. Patient with active severe infection such as herpes, tuberculosis, viral hepatitis, human immunodeficiency virus infection 12. Patient with autoimmune conditions such as systemic lupus erythematosus 13. Patient with a diagnosis of cancer or evidence of continued disease within five years before screening 14. Patient with severe cardiac conditions: - Patient with recent history of severe cardiovascular conditions including acute myocardial infarction, unstable angina pectoris, coronary revascularization procedure, congestive heart failure of NYHA Class III or IV, stroke, including a transient ischemic attack - Patient with cardiac conduction abnormalities at study entry including a QTc Fredericia interval >450 milliseconds for males and >470 milliseconds for females, a second- or third-degree atrioventricular block not successfully treated with a pacemaker - Patient presenting with edema of cardiac origin and left ventricular ejection fraction * 50% 15. Patient with risk factors for sudden unexpected death of cardiovascular origin 16. Patient who has been exposed to an investigational treatment within 3 months prior to screening 17. Patient who has been exposed to Edaravone within at least 30 days prior to screening 18. Patient treated concomitantly with drugs known to interact with cytochrome P450 (CYP450) isoenzymes (2C9, 2D6 and 3A4)
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Absolute change from baseline of ALSFRS-R total score at week 48. | — |
Secondary
| Measure | Time frame |
|---|---|
| Progression free survival (PFS) defined as the time from randomization to progression (decline of more than 9 points in ALSFRS-R score from baseline) or death * Amyotrophic Lateral Sclerosis Assessment Questionnaire 40 (ALSAQ-40) change * Forced Vital Capacity (FVC) change * Upper- and lower-limb muscle strength using hand-held dynamometry (HHD) * Clinician-rated Clinical Global Impression (CGI) * Combined Assessment of Function and Survival (CAFS) * Overall Survival (OS) * Event free survival (EFS) defined as the time from randomization to the first occurrence of either death or tracheostomy | — |
Countries
Netherlands
Outcome results
None listed