BridgeMD
A prospective, multicenter, randomised, double-blind, placebo-controlled, parallel groups, phase 3 Trial to compare the efficacy and safety of masitinib in combination with standard of care versus placebo in combination with standard of care in the treatment of patients suffering from Amyotrophic Lateral Sclerosis (ALS)
EU CTIS ID: 2024-511244-12-01
What this study is testing
The objective of the trial is to evaluate the efficacy and safety of masitinib 4.5 mg/kg/day as add-on standard of care therapy in patients diagnosed with ALS
- Therapeutic confirmatory (Phase III)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- 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] at screening
- 7. Patient with a ALSFRS-R progression rate of > 0.3 and <1.1 point/month at screening visit i) as measured between onset of the disease and screening
- 8. Patient with an ALSFRS-R total score at screening and baseline following rules below: - at least 3 on item #3 and - at least 2 on item #12 and - at least 1 on each of the other 10 items (i.e. item #1, #2, #4, #5a or #5b, #6, #7, #8, #9, #10, and #11)
- 9. Contraception at screening and baseline: - Female patient of childbearing potential (entering the trial 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 trial and for 8 months 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 trial and for 5 months 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 trial and for 5 months after the last treatment intake Contraceptive use by men and women should be consistent with local regulations regarding the methods of contraception for those participating in clinical trials. Highly effective and effective methods of contraception are detailed in appendix 16.1.
- Patients who are not a candidate for QALSODY® Tofersen treatment
- 10. At screening visit patient is 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 trial 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 trial participation.
You likely can't join if
- 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 at screening visit
- 18. Any medical condition that, in the opinion of the Investigator, might interfere with the patient’s participation in the trial, poses any added risk for the patient, or confounds the assessment of the patient at screening visit
- 19. Patient under psychiatric care, patient protected by law under guardianship or curatorship, patient in emergency situations, prisoners and patient without National health insurance at screening visit
- 2. Patient with hypersensitivity to masitinib excipients or riluzole at screening visit
- 3. Patient with an FVC < 70%, predicted normal value for gender, height, and age, at screening and baseline
- 4. Patient with a weight < 41 kg and a BMI < 18 or > 35 kg/m² at screening and baseline visit
See the full eligibility criteria
Who can join
- 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] at screening
- 7. Patient with a ALSFRS-R progression rate of > 0.3 and <1.1 point/month at screening visit i) as measured between onset of the disease and screening
- 8. Patient with an ALSFRS-R total score at screening and baseline following rules below: - at least 3 on item #3 and - at least 2 on item #12 and - at least 1 on each of the other 10 items (i.e. item #1, #2, #4, #5a or #5b, #6, #7, #8, #9, #10, and #11)
- 9. Contraception at screening and baseline: - Female patient of childbearing potential (entering the trial 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 trial and for 8 months 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 trial and for 5 months 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 trial and for 5 months after the last treatment intake Contraceptive use by men and women should be consistent with local regulations regarding the methods of contraception for those participating in clinical trials. Highly effective and effective methods of contraception are detailed in appendix 16.1.
- Patients who are not a candidate for QALSODY® Tofersen treatment
- 10. At screening visit patient is 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 trial 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 trial participation.
- 11. At screening visit patient is able and willing to comply with trial protocol and to come on-site as per protocol visits schedule
- 12. At screening visit patient is 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
- 2. Patient with a familial or sporadic ALS at screening
- 3. Patient aged between 18 and 80 years old inclusive at screening
- 4. Patient treated with a stable dose of Riluzole (100 mg/day) for at least 30 days prior to screening visit and baseline visit
- 5. Patient receiving or not Edaravone. If receiving Edaravone (oral suspension only), patients must have been taking it at stable dose for at least 30 days prior to screening visit
- 6. Patient with onset of first symptom of ALS no longer than 36 months at screening
What rules you out
- 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 at screening visit
- 18. Any medical condition that, in the opinion of the Investigator, might interfere with the patient’s participation in the trial, poses any added risk for the patient, or confounds the assessment of the patient at screening visit
- 19. Patient under psychiatric care, patient protected by law under guardianship or curatorship, patient in emergency situations, prisoners and patient without National health insurance at screening visit
- 2. Patient with hypersensitivity to masitinib excipients or riluzole at screening visit
- 3. Patient with an FVC < 70%, predicted normal value for gender, height, and age, at screening and baseline
- 4. Patient with a weight < 41 kg and a BMI < 18 or > 35 kg/m² at screening and baseline visit
- 5. Pregnant, or nursing female patient at screening and baseline
- 6. Patient with history (or family history) of severe skin toxicities or reactions at screening
- 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 at screening and baseline
- 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 < 1.5x109/L, or - Anemia with Hgb < LLN or red blood cell count below the LLN, or - Thrombocytopenia with platelets counts < 150 x 109/L
- 9. Patient with history of hepatic disorders, with a known liver disease or recent alcohol abuse, or with abnormal laboratory results from local laboratory assessments, at screening and baseline, defined as: - Hepatic transaminase levels > 2 ULN, or - Total bilirubin level > 1.5 ULN, or - Both hepatic transaminase levels and total bilirubin level outside of the normal ranges, or - Albuminemia < 1 x LLN - Patients with concomitant medication known to be associated with severe hepatotoxicity
- 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) or - 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
- Patients who have received a live vaccine within 30 days before the first administration of the investigational medicinal product
- Patient with interstitial lung disease or pulmonary fibrosis.
- 11. Patient with active tuberculosis infection, viral hepatitis, human immunodeficiency virus infection at screening
- 12. Patient with a diagnosis of cancer or evidence of continued disease within five years before screening
- 13. Patients with current or history of severe cardiovascular disease, assessed at screening - Myocardial infarction, - Unstable angina pectoris - Coronary revascularization procedure - Congestive heart failure of NYHA Class III or IV - Stroke, including a transient ischemic attack, - Second degree or third-degree atrioventricular block not successfully treated with a pacemaker, - Bi-fascicular block, - QTc Fridericia interval > 450 milliseconds for males and > 470 milliseconds for females, - Drug induced heart failure or ischemic heart disease. - Radiotherapy induced cardiomyopathy. - Family history of unexpected death of cardiovascular origin. - Oedema of cardiac origin and left ventricular ejection fraction ≤ 50%
- 14. Patients, with two or more of the risk factors listed below assessed, at screening, as Very High Risk (calculated SCORE ≥10%.) or High Risk calculated SCORE ≥5% and <10%) according to the Systematic Coronary Risk Estimation (SCORE): - Hypertension (uncontrolled) - Diabete - Chronic kidney disease - Current tabagism (≥ 10 Pack-year: equivalent to 1 pack of 20 cigarettes for 10 years with the formula N (number of packs of 20 cigarettes smoked daily) x T (number years smoking)) Patients who stopped smoking 6 months prior to the evaluation, are not concerned. - Hypercholesterolemia - COPD This assessment is done according to the Systematic Coronary Risk Estimation (SCORE) using the country specific free full version of HeartScore°, the interactive tool for predicting and managing the risk of heart attack and stroke in Europe, available at https://www.heartscore.org/en_GB/access If the country specific version is not available, EU one should be used.
- 15. Patient treated concomitantly with substrates, inhibitors or inducers of BCRP at screening and baseline
- 16. Participants with a significant pulmonary disorder not attributed to ALS or who require treatments that might complicate the evaluation of the effect of ALS on respiratory function at screening visit
- 17. Previous treatments with masitinib
The study team makes the final eligibility decision.
Where it's taking place
- Argentina
- United States
- Serbia
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 65+ years, 18-64 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Argentina; United States; Serbia. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.