patients suffering from Amyotrophic Lateral Sclerosis (ALS) MedDRA version: 14.1 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: INCLUSION CRITERIA: 1. Female or male patient aged between 18 and below 80 and whose weight = 50 kg 2. Familial or sporadic ALS 3. Patient with a verifiable diagnosis of probable or definite ALS 4. Disease duration no longer than 36 months 5. Patient treated with a steady regimen of riluzole (100 mg/day) for a minimum of 1 month before study entry 6. Patient with a FVC (Forced Vital Capacity) = 60% 7. Patient with life expectancy = 6 months 8. Patient with adequate organ function at screening and baseline: · Absolute Neutrophils Count (ANC) = 2 x 109/L · Hemoglobin = 10 g/dL · Platelets (PTL) = 100 x 109/L · AST/ALT = 2.5 ULN · Bilirubin = 1.5 ULN · Albuminemia = 1 x LLN · Urea = 1.5 x ULN · Creatinine clearance = 50 mL/min (Cockcroft and Gault formula) · Proteinuria =65 years) yes F.1.3.1 Number of subjects for this age range 17
Exclusion criteria
Exclusion criteria: EXCLUSION CRITERIA 1. Patient with history of cardiac, hematologic, hepatic, respiratory that is clinically significant for their participation in the study 2. Patient who underwent tracheotomy and /or gastrostomy 3. Patient with a diagnosis of cancer or evidence of continued disease within five years before starting study treatment 4. Patient with significant sensory abnormalities, dementia, other neurologic diseases, uncompensated medical illness, substance abuse and psychiatric illness 5. Patient who have participated in a clinical trial within 3 months prior to screening 6. Pregnant, or nursing female patient 7. Patient having cardiac disorders defined by at least one of the following conditions: · Patient with recent cardiac history (within 6 months) of: - Acute coronary syndrome - Acute heart failure (class III or IV of the NYHA classification) - Significant ventricular arrhythmia (persistent ventricular tachycardia, ventricular fibrillation, resuscitated sudden death) · Patient with cardiac failure class III or IV of the NYHA classification · Patient with severe conduction disorders which are not prevented by permanent pacing (atrioventricular block 2 and 3, sino-atrial block) · Syncope without known aetiology within 3 months · Uncontrolled severe hypertension, according to the judgment of the investigator, or symptomatic hypertension 8. Patient with history of poor compliance or history of drug/alcohol abuse, or excessive alcohol beverage consumption that would interfere with the ability to comply with the study protocol, or current or past psychiatric disease that might interfere with the ability to comply with the study protocol or give informed consent PREVIOUS TREATMENTS 9. Patient treated with any investigational agent within 3 months of screening
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: The objective is to compare the efficacy and safety of masitinib versus placebo in the treatment of patients suffering from Amyotrophic Lateral Sclerosis (ALS).;Secondary Objective: Primary endpoint : Amyotrophic Lateral Sclerosis functional rating scale (ALSFRS)-Revised at week 48 Secondary Endpoints: - Amyotrophic Lateral Sclerosis functional rating scale (ALSFRS)-Revised at week 4, 8, 12 and week 36 - Number of failure defined by a 6 point drop in the ALSFRS-R or death - Survival rate at week 12, 24, 36 and week 48 - Overall survival rate - Cystatin C level at week 12, 24, 36, 48 - Forced vital capacity (FVC) score at week 4, 8, 12, 24, 36 and week 48 - Time to first tracheotomy - ALS SQOLquality of life scale at week 4, 8, 12, 24, 36 and week 48 - Safety: occurrence of Adverse Events (AE), changes on clinical examination including vital signs (blood pressure, pulse rate) and weight, ECG and laboratory exams (biochemistry, hematology and urinalysis);Primary end point(s): Primary endpoints Amyotrophic Lateral Sclerosis functional rating scale (ALSFRS)-Revised at week 48;Timepoint(s) of evaluation of this end point: week 48 | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Secondary endpoints - Amyotrophic Lateral Sclerosis functional rating scale (ALSFRS)-Revised at week 4, 8, 12, 24 and week 36 - Number of failure deined as 6 drop point in ALSFRS-R or death - Survival rate at week 12, 24, 36 and week 48 - Overall survival rate - Cystatin C level from baseline to week 48 - Forced vital capacity (FVC) score at week 4, 8, 12, 24, 36 and week 48 - Time to first tracheotomy - ALS SQOL quality of life scale at week 4, 8, 12, 24, 36 and week 48 - Safety: occurrence of Adverse Events (AE), changes on clinical examination including vital signs (blood pressure, pulse rate) and weight, ECG and laboratory exams (biochemistry, hematology and urinalysis);Timepoint(s) of evaluation of this end point: week 4, 8, 12, 24, 36 | — |
Countries
France, Greece, Hungary, Ireland, Italy, Netherlands, Portugal, Slovakia, Spain
Contacts
Public ContactAlain Moussy
ABScience
Outcome results
None listed