Evaluation of masitinib in Amyotrophic Lateral Sclerosis (ALS)
- Conditions
- patients suffering from Amyotrophic Lateral Sclerosis (ALS)MedDRA version: 21.1Level: PTClassification code 10002026Term: Amyotrophic lateral sclerosisSystem Organ Class: 10029205 - Nervous system disordersTherapeutic area: Diseases [C] - Nervous System Diseases [C10]
- Registration Number
- EUCTR2019-001862-13-IE
- Lead Sponsor
- AB Science
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 495
1.Patient, male or female, diagnosed with laboratory supported probable, clinically probable or definite ALS according to the World Federation of Neurology Revised El Escorial criteria [52]
2.Patient with a familial or sporadic ALS
3.Patient aged between 18 and 75 years old inclusive at screening
4.Patient treated with a stable dose of Riluzole (100 mg/day) for at least 12 weeks prior to baseline visit
5.Patient with an ALS disease duration from diagnosis no longer than 24 months at screening
6.Patient with an ALSFRS-R total score progression between onset of the disease and screening of > 0.3 and <1.1 point/month
7.Patient with an ALSFRS-R total score decrease of = 1 point between screening and baseline
8.Patient with an ALSFRS-R total score of at least 26 at screening following rules below:
-at least 3 on item #3 and
-at least 2 on each of the other 11 items (i.e. item #1, #2, #4, #5a or #5b, #6, #7, #8, #9, #10, #11 and #12)
9.Patient with an ALSFRS-R total score of at least 25 at randomization following rules below:
-at least 3 on item #3 and
-at least 2 on each of the other 11 items (i.e. item #1, #2, #4, #5a or #5b, #6, #7, #8, #9, #10, #11 and #12)
10.Contraception:
-Female patient of childbearing potential (entering the study after a menstrual period and who has a negative pregnancy test), who agrees to use a highly effective method of contraception and an effective method of contraception by her male partner during the study and for 3 months and a half after the last treatment intake
-Male patient with a female partner of childbearing potential who agrees to use a highly effective method of contraception and an effective method of contraception by his female partner during the study and for 3 months and a half after the last treatment intake OR who agrees to use an effective method of contraception and a highly effective method of contraception by his female partner during the study and for 3 months and a half after the last treatment intake
Highly effective and effective methods of contraception are detailed in appendix 15.1
11.Patient able to understand, and willing to sign, and date the written informed consent form prior to any protocol-specific procedures. If patients are duly capable of study consent but are unable to sign by themselves due to aggravation of disease condition, written informed consent can be obtained from a legally authorized representative who can sign on behalf of the patients after confirming the patients' agreement to study participation.
12.Patient able and willing to comply with study protocol and to come on-site as per protocol visits schedule
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
Are the trial subjects under 18? no
Number of subjects for this age range: 0
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 450
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 45
1.Patient with dementia or significant neurological, psychiatric, systemic or organic disease, uncontrolled or that may interfere with the conduct of the trial or its results
2.Patient with hypersensitivity to masitinib excipients
3.Patient with an FVC < 60% predicted normal value for gender, height, and age at screening
4.Patient with a weight < 41 kg and a BMI < 21 or > 30 kg/m² at screening and at baseline
5.Pregnant, or nursing female patient
6.Patient with history (or family history) of severe skin toxicities or reactions
7.Patients treated by drugs known to be at high risk for Stevens-Johnson Syndrome or for Drug Reaction with Eosinophilia and Systemic Symptoms (DRESS) syndrome
8.Patient with history of severe bone marrow disorders such as agranulocytosis or aplasia, or with abnormal laboratory results from local laboratory assessments at screening and baseline:
-Neutropenia with ANC < 1.5 x 109/L
-Anemia with Hgb < LLN and red blood cell count below the LLN
-Thrombocytopenia with platelets counts < 150 x 109/L
9.Patient with history of hepatic disorders, with a known liver disease or alcohol abuse, or with abnormal laboratory results from local laboratory assessments defined as:
-hepatic transaminase levels > 2 ULN at baseline, or
-total bilirubin level > 1.5 ULN at baseline, or
-both hepatic transaminase levels and total bilirubin level outside of the normal ranges at screening and baseline, or
-albuminemia < 1 x LLN at screening and baseline
10.Patient with pre-existing severe renal impairment, or with abnormal laboratory results from local laboratory assessments at screening and baseline:
-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
11.Patient with active severe infection such as herpes, tuberculosis, viral hepatitis, human immunodeficiency virus infection
12.Patient with autoimmune conditions such as systemic lupus erythematosus
13.Patient with a diagnosis of cancer or evidence of continued disease within five years before screening
14.Patient with severe cardiac conditions:
-Patient with recent history of severe cardiovascular conditions including acute myocardial infarction, unstable angina pectoris, coronary revascularization procedure, congestive heart failure of NYHA Class III or IV, stroke, including a transient ischemic attack
-Patient with cardiac conduction abnormalities at study entry including a QTc Fredericia interval >450 milliseconds for males and >470 milliseconds for females, a second- or third-degree atrioventricular block not successfully treated with a pacemaker
-Patient presenting with edema of cardiac origin and left ventricular ejection fraction = 50%
15.Patient with risk factors for sudden unexpected death of cardiovascular origin
16.Patient who has been exposed to an investigational treatment within 3 months prior to screening
17.Patient who has been exposed to Edaravone within at least 30 days prior to screening
18.Patient treated concomitantly with drugs known to interact with cytochrome P450 (CYP450) isoenzymes (2C9, 2D6 and 3A4)
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method