A prospective, multicenter, randomised, double-blind, placebo-controlled, parallel groups, phase 2/3 study to compare the efficacy and safety of masitinib versus placebo in the treatment of patients suffering from Amyotrophic Lateral Sclerosis (ALS)
- Conditions
- ALSAmyotrophic Lateral Sclerosis10011305
- Registration Number
- NL-OMON44970
- Lead Sponsor
- AB Science
- Brief Summary
Trial is onging in other countries
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Completed
- Sex
- Not specified
- Target Recruitment
- 10
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. Female patient of childbearing potential (entering the study after a menstrual period and who have a negative pregnancy test), who agrees to use two highly effective methods (one for the patient and one for the partner) of medically acceptable forms of contraception during the study and for 3 months after the last treatment intake. Acceptable forms of contraception are explained in the protocol (v4.0 dd16-Mar-2015) on page 5.
10. Male patients must use medically acceptable methods of contraception if your female partner is pregnant, from the time of the first administration of the study drug until three months following administration of the last dose of study drug. Acceptable methods are explained in the protocol on page 5.
Male patients must use two highly effective methods (one for the patient and one for the partner) of medically acceptable forms of contraception during the study and for 3 months after the last treatment intake. Acceptable methods are explained in the protocol on page 5.
11. Female patient of childbearing potential must have a negative
pregnancy test at screening and baseline
12. Patient able and willing to comply with study procedures as per
protocol
13. Patient able to understand, and willing to sign, and date the written
informed consent form at screening visit prior to any protocol-specific
procedures
14. 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
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 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;12. Patient treated with any investigational agent within 3 months prior
to screening
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method <p>Change from baseline to week 48 in Amyotrophic Lateral Sclerosis functional<br /><br>rating scale (ALSFRS)-Revised </p><br>
- Secondary Outcome Measures
Name Time Method