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Quick Info
Status
Currently Recruiting
Phase
3
Trial Type
Randomized, parallel assignment
Treatment Type
Drug - oral
Randomization
Unkown
Enrollment
495
Start Date
10/1/2020
Sponsor
Contact Information
Locations
Germany, Other
Department of Neurology ,University of Ulm, Ulm, 89081, Germany
United States, Maryland
Johns Hopkins Medicine Brain Science Institute, Baltimore, MD, 21205, United States
Enrollment Criteria
Breathing Ability
Percent lung function (FVC) or (SVC)
>60
Months Since Onset
Number of months since first symptoms of ALS.
<24 months
Non-Invasive Ventilation (NIV)
Can PALS use a BiPAP in the trial?
N/A
Diaphragm Pacer (DPS)
Can PALS use a DPS in the trial?
N/A
Edaravone Usage
Can a PALS use edaravone (Radicut/Radicava) while enrolled in the trial?
Unknown
Open Label
No
Update Notes
Enrolling, new site added
11/12/2020
Primary Contact and Location Changed
4/3/2020
Updated trial description
12/2/2019
No significant updates
7/1/2019
No significant updates
5/1/2019
No significant updates
5/1/2019
Location added
2/5/2019
Contact info updated
12/7/2018
Trial added
4/26/2017

Other Information

Purpose
The objective is to compare the efficacy and safety of masitinib in combination with riluzole versus matched placebo in combination with riluzole for the treatment of Amyotrophic Lateral Sclerosis (ALS).
Eligibility
Main inclusion criteria include:
- Patients diagnosed with laboratory supported probable, clinically probable or definite ALS according to the World Federation of Neurology Revised El Escorial criteria - Patient with a familial or sporadic ALS - ALS disease duration from diagnosis no longer than 24 months at the screening visit - Patient treated with a stable dose of riluzole (100 mg/day) for at least 12 weeks days prior to the baseline visit - Patient with an ALSFRS-R score progression between onset of the disease and screening of > 0.3 per month, confirmed with an ALSFRS-R score progression of ≥ 1 point during a 12-week run-in period between screening and randomization.
- Patient with a score, at screening, of at least 26 overall, including a score of at least 3 on item #3 and at least 2 on each of the 12 ALSFRS-R individual component items and with a score, at randomization, of at least 2 on each of the 12 ALSFRS-R individual component items Main exclusion criteria include:
- 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 - Patient with a FVC < 60% predicted normal value for gender, height, and age at screening and baseline - Pregnant, or nursing female patient
Details
Masitinib is a selective, oral tyrosine kinase inhibitor with neuroprotective capability demonstrated via numerous preclinical studies. Two of masitinib's main cellular targets are the mast cell and microglia cell. It is well-established that mast cells play a prominent role in neuroinflammatory processes. Microglia, resident immune cells of the central nervous system (CNS), also constitute an important source of neuroinflammatory mediators and may have fundamental roles in numerous neurodegenerative disorders. The development of masitinib in ALS is therefore based on the pharmacological action of masitinib in microglia cells and mast cells, thereby slowing microglial-related disease progression, reducing neuro-inflammation, and modulating the neuronal microenvironment in both central and peripheral nervous systems. This is a multicenter, double-blind, randomized, placebo-controlled, parallel-group (two ascending dose titrations of masitinib and matching placebo), comparative study of oral masitinib in the treatment of patients with amyotrophic lateral sclerosis (ALS).
Collaborator(s)
  • AB Science
Trial Protocol as Published on Clinicaltrials.gov
NCT03127267 (First Published: 4/13/2017)