patients suffering from Amyotrophic Lateral Sclerosis (ALS) MedDRA version: 21.1 Level: PT Classification code 10002026 Term: Amyotrophic lateral sclerosis System Organ Class: 10029205 - Nervous system disorders
Conditions
Sponsors
ABScience
Eligibility
Sex/Gender
All
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 [52] 2. Patient with a familial or sporadic ALS 3. Patient aged between 18 and 80 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 =65 years) yes F.1.3.1 Number of subjects for this age range 45
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 or its excipients and riluzole or its excipients 3. Patient with an FVC 35 kg/m² at screening or 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 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. patients with current or history of severe cardiovascular disease: - Myocardial infarction, - Unstable angina pectoris - Coronary revascularization procedure - Congestive heart failure of NYHA Class III or IV - Stroke, including a transient ischemic attack, - Second degree or third-degree atrioventricular block not successfully treated with a pacemaker, - Bi-fascicular block, - QTc Fridericia interval > 450 milliseconds for males and > 470 milliseconds for females, - Drug induced heart failure or ischemic heart disease. - Radiotherapy induced cardiomyopathy. - Family history of unexpected death of cardiovascular origin. 15. Patients, with two or more of the risk factors listed below assessed by a cardiologist as Very High Risk (calculated SCORE =10%.) or High Risk calculated SCORE =5% and <10%) according to the Systematic Coronary Risk Estimation (SCORE): - Hypertension (uncontrolled) - Diabete - Kidney disease, - Current tabagism (= 10 Pack-year: equivalent to 1 pack of 20 cigarettes for 10 years with the formula N (number of packs of 20 cigarettes smoked daily) x T (number years smoking)) Patients who stopped smoking 6 months prior to the evaluation, are not concerned. - Hypercholesterolemia - COPD This assessment is done according to the Systematic Coronary Risk Estimatio
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: The primary objective is to demonstrate statistically significant improvement from baseline in ALSFRS-R after 48-week treatment of two doses of masitinib versus matching placebo in patients diagnosed with ALS treated with Riluzole.;Secondary Objective: Secondary objectives are to assess the efficacy and safety of two doses of masitinib versus matching placebo in the treatment of patients diagnosed with ALS treated with Riluzole ;Primary end point(s): Absolute Change from baseline to week 48 in Amyotrophic Lateral Sclerosis functional rating scale (ALSFRS)-Revised total score ;Timepoint(s) of evaluation of this end point: week 48 | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): - 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 Safety Analysis: - Adverse events - Vital signs, physical examination, ECGs - Clinical laboratory tests (haematology, biochemistry, urinalysis and urinary cytology) ;Timepoint(s) of evaluation of this end point: at W48 or event occurence (death) depending on secondary endpoint | — |
Countries
Argentina, Austria, Belgium, Canada, Denmark, France, Germany, Greece, Ireland, Israel, Italy, Netherlands, Norway, Poland, Portugal, Russian Federation, Slovenia, Spain, Sweden, Ukraine, United Kingdom, United States
Contacts
Public ContactAlain Moussy
ABScience
Outcome results
None listed