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Quick Info
Trial Type
Randomized, parallel assignment
Treatment Type
Drug - oral
Start Date
Contact Information
Belgium, Other
University Hospital Leuven (UZ Leuven), Leuven, Belgium
Denmark, Other
Bispebjerg Hospital, Copenhagen, Denmark
France, Other
CHU de Angers, Angers, France
Groupe Hospitalier Pellegrin Tripode, Bordeaux, France
Hôpital neurologique Pierre Wertheimer, Bron, France
CHU Gabriel Montpied, Clermont Ferrand, France
CHU de Lille - Hopital Roger Salengro, Lille, France
CHU de Limoges - Hôpital Dupuytren, Limoges, France
CHU de Marseille - Hôpital de la Timone, Marseille, France
CHRU de Montpellier - Gui de Chauliac, Montpellier, France
CHU de Nancy - Hopital Central, Nancy, France
CHU Hôpital Pasteur Nice, Nice, France
CHRU de Tours - Hopital Bretonneau, Tours, France
Germany, Other
Department of Neurology, University of Ulm, Ulm, 89081, Germany
Greece, Other
Athens Naval Hospital, Athens, Greece
Eginition Hospital, Athens, Greece
University General Hospital of Larissa, Larissa, Greece
General University Hospital of Patras, Río, Greece
Israel, Other
Hadassah University Hospital, Jerusalem, Israel
Tel-Aviv Medical Center Hôpital Sourasky (ICHILOV), Tel Aviv, Israel
Italy, Other
Ospedale Civile Sant'Agostino - Estense, Baggiovara, Italy
ASST degli Spedali Civili di Brescia, Brescia, Italy
Centro Clinico NeMO Fondazione Serena Onlus, Gussago, Italy
Clinico Nemo Center (Centro Clinico NeMO Milano), Milano, Italy
IRCCS Istituto Auxologico Italiano, Milano, Italy
Istituti Clinici Scientifici Maugeri IRCCS, Milano, Italy
San Raffaele Hospital (Ospedale San Raffaele), Milano, Italy
University Hospital Maggiore della Carita, Novara, Italy
Azienda Ospedale-Università Padova, Padova, Italy
IRCCS Mondino Foundation, Pavia, Italy
University Hospital in Turin (Azienda Ospedaliero-Universitaria Città della Salute e della Scienza d, Torino, Italy
University Hospital in Turin (Azienda Ospedaliero-Universitaria Città della Salute e della Scienza d, Torino, Italy
Norway, Other
Oslo University Hospital HF Ullevål, Oslo, Norway
Other, Other
Clinical Centre of Serbia, Belgrad, Other
Poland, Other
Centrum Medyczne Neuromed, Bydgoszcz, Poland
Portugal, Other
Hospital de Santa Maria, Lisboa, Portugal
Russian Federation, Other
Moscow city clinical Hospital after V.M. Buyanov, Moscow, Russian Federation
Scientific Practical Medical Center "Innovation and Health", Novosibirsk, Russian Federation
Slovenia, Other
Klinicni center Ljubljana, Ljubljana, Slovenia
Spain, Other
Hospital General Universitario de Alicante, Alicante, Spain
Hospital Universitari de Bellvitge, Barcelona, Spain
Hospital Carlos III, Madrid, Spain
Hospital San Rafael, Madrid, Spain
Clinical Hospital Santiago de Compostela, Santiago De Compostela, Spain
Hospital Universitario y Politecnico La Fe, Valencia, Spain
Sweden, Other
Centralsjukhuset Karlstad (Central Hospital Karlstad), Karlstad, Sweden
Skåne University Hospital, Malmö, Sweden
Norrlands universitetssjukhus, Umeå, Sweden
Ukraine, Other
The State Institution of Neurology, Psychiatry and Narcology of NAMS of Ukraine, Kharkiv, Ukraine
Medical Center of LLC Medical Center Dopomoga Plus, Kyiv, Ukraine
Communal Non-Profit Enterprise of Lviv Regional Council, Lviv Regional Clinical Hospital, Neurologic, Lviv, Ukraine
Communal Non-Profit Enterprise of Lviv Regional Council, Lviv Regional Clinical Hospital, Neurologic, Lviv, Ukraine
United States, Alabama
University of Alabama at Birmingham, Birmingham, AL, 35294, United States
United States, California
University of Southern California, Los Angeles, CA, 90007, United States
United States, Kentucky
University of Kentucky, Lexington, KY, 40506, United States
United States, Maryland
Johns Hopkins Medicine Brain Science Institute, Baltimore, MD, 21205, United States
United States, Massachusetts
Lahey Hospital and Medical Center, Burlington, MA, 01805, United States
United States, Virginia
University of Virginia Health System, Charlottesville, VA, 22903, United States
Enrollment Criteria
Breathing Ability
Percent lung function (FVC) or (SVC)
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?
Diaphragm Pacer (DPS)
Can PALS use a DPS in the trial?
Edaravone Usage
Can a PALS use edaravone (Radicut/Radicava) while enrolled in the trial?
Open Label
Update Notes
Sites updated
Sites added, star/completion date updated
Enrolling, new site added
Primary Contact and Location Changed
Updated trial description
No significant updates
No significant updates
No significant updates
Location added
Contact info updated
Trial added

Other Information

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).
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
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).
  • AB Science
Trial Protocol as Published on
NCT03127267 (First Published: 4/13/2017)