Evaluation of masitinib in Amyotrophic Lateral Sclerosis (ALS)

Phase 1

Conditions: patients suffering from Amyotrophic Lateral Sclerosis (ALS)
MedDRA version: 14.1Level: PTClassification code 10002026Term: Amyotrophic lateral sclerosisSystem Organ Class: 10029205 - Nervous system disorders
Therapeutic area: Diseases [C] - Nervous System Diseases [C10]

Registration Number: EUCTR2010-024423-24-ES

Lead Sponsor: ABScience

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: ot Recruiting

Sex: All

Target Recruitment: 45

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 < 30 mg/dL on dipstick; in case of the proteinuria = 30 mg/dL, 24 hours proteinuria < 1.5g/24 hours
9. Man or woman of child bearing potential, (entering the study after a menstrual period and who have a negative
pregnancy test) who agree to use two methods (one for the patient and one for the partner) of medically
acceptable forms of contraception during the study and for three months after the last treatment intake
10. Patient able and willing to comply with study procedures as per protocol
11. Patient able to understand, and willing to sign, and date the written informed consent form at screening visit
prior to any protocol-specific procedures
12. patient able to understand and willing to folow the safety procedures mentioned on the patient card
13. Patient affiliated to a social security system
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 25
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 17

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