Evaluation of masitinib in Amyotrophic Lateral Sclerosis (ALS)

Phase 1

Conditions: patients suffering from Amyotrophic Lateral Sclerosis (ALS)
MedDRA version: 19.0Level: 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-NL

Lead Sponsor: ABScience

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: ot Recruiting

Sex: All

Target Recruitment: 300

Inclusion Criteria

INCLUSION CRITERIA:
1.Female or male patient aged between 18 and 75 years of age, with a weight > 50 kg and BMI between 18 and 35 kg/m².
2.Familial or sporadic ALS
3.Patient diagnosed with laboratory supported, clinically probable or definite ALS according to the World Federation of Neurology Revised El Escorial criteria (Brooks, 1994)
4.Disease duration from symptoms onset no longer than 36 months at the screening visit
5.Patient treated with a stable dose of riluzole (100 mg/day) for at least 30 days prior to screening
6.Patient with a FVC (Forced Vital Capacity) equal to or more than 60% predicted normal value for gender, height, and age at the screening visit
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 = 3 ULN
•Bilirubin = 1.5 ULN
•Albuminemia > 1 x LLN
•Creatinine clearance > 60 mL/min (Cockcroft and Gault formula)
•Proteinuria < 30 mg/dL (1+) on dipstick; in case of the proteinuria = 1+ on the dipstick, 24 hours proteinuria must be < 1.5g/24 hours
9.Male patient or female patient of child bearing potential, who must agree to use two methods (one for the patient and one for the partner) of medically acceptable forms of contraception (with failure rate < 1%) during the study and for three months after the last treatment intake
10.Female patient of childbearing potential must have a negative pregnancy test at screening and baseline
11.Patient able and willing to comply with study procedures as per protocol
12.Patient able to understand, and willing to sign, and date the written informed consent form at screening visit prior to any protocol-specific procedures
13.Patient able to understand, and willing to follow the safety procedures mentioned on the patient card in case of signs or symptoms of severe neutropenia or severe cutaneous toxicity, during the first two months of treatment

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 280
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 20

Exclusion Criteria

EXCLUSION CRITERIA
1.Patient with history of hematologic, hepatic, respiratory disorder that is clinically significant for his/her 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 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 with a known diagnosis of human immunodeficiency virus (HIV) infection

8.Patient with known active hepatitis B, hepatitis C or tuberculosis

9.Patient with any severe and/or uncontrolled medical condition
10.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 (atrio-ventricular 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
11.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 WASH OUT
12.Patient treated with any investigational agent within 3 months prior to screening