Evaluation of masitinib in Amyotrophic Lateral Sclerosis (ALS)

• Register: EUCTR
• Last refreshed on: 30 April 2018
• Main ID: EUCTR2010-024423-24-ES
• Date of registration: 29/11/2012
• Prospective Registration: Yes
• Primary sponsor: ABScience
• Public title: Evaluation of masitinib in Amyotrophic Lateral Sclerosis (ALS)
• Scientific title: A prospective, multicenter, randomized, double-blind, placebocontrolled, parallel group, phase 2 study to compare the efficacy and safety of masitinib versus placebo in the treatment of patients suffering from Amyotrophic Lateral Sclerosis (ALS) - not applicable
• Date of first enrolment: 29/01/2013
• Target sample size: 45
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2010-024423-24
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: yes Randomised: yes Open: no Single blind: no Double blind: yes Parallel group: yes Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: no Placebo: yes Other: no Number of treatment arms in the trial: 3
• Phase: Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): yes Therapeutic confirmatory - (Phase III): no Therapeutic use (Phase IV): no

Countries of recruitment

France, Greece, Hungary, Ireland, Italy, Netherlands, Portugal, Slovakia, Spain

Contacts

• Name: Alain Moussy
• Affiliation: ABScience

• Name: Alain Moussy
• Affiliation: ABScience

Key inclusion & exclusion 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 < 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


Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Therapeutic area: Diseases [C] - Nervous System Diseases [C10]

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

Intervention(s)

Product Name: masitinib
Product Code: AB1010
Pharmaceutical Form: Film-coated tablet
INN or Proposed INN: Masitinib mesylate
CAS Number: 790-299-79-5
Current Sponsor code: AB1010
Other descriptive name: na
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 100-
Pharmaceutical form of the placebo: Film-coated tablet
Route of administration of the placebo: Oral use

Product Name: masitinib
Product Code: AB1010
Pharmaceutical Form: Film-coated tablet
INN or Proposed INN: Masitinib mesylate
CAS Number: 790299-79-5
Current Sponsor code: AB1010
Other descriptive name: MASITINIB
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 200-
Pharmaceutical form of the placebo: Film-coated tablet
Route of administration of the placebo: Oral use

Primary Outcome(s)

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).

Primary end point(s): Primary endpoints
Amyotrophic Lateral Sclerosis functional rating scale (ALSFRS)-Revised at week 48

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)

Timepoint(s) of evaluation of this end point: week 48

Secondary Outcome(s)

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

Secondary ID(s)

AB10015

Source(s) of Monetary Support

ABScience