RecruitingRecruiting
Efficacy and Safety of Masitinib Versus Placebo in the Treatment of ALS Patients
NCT03127267 · AB Science
In plain English
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Official title
Phase 3 Study to Compare the Efficacy and Safety of Masitinib in Combination With Riluzole Versus Placebo in Combination With Riluzole in the Treatment of Patients Suffering From Amyotrophic Lateral Sclerosis (ALS)
About this study
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).
Eligibility criteria
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
Study design
Enrollment target: 495 participants
Allocation: randomized
Masking: double
Age groups: adult, older_adult
Timeline
Starts: 2021-02-02
Estimated completion: 2027-12
Last updated: 2025-09-12
Interventions
Drug: Masitinib (6.0)Drug: RiluzoleDrug: PlaceboDrug: Masitinib (4.5)
Primary outcomes
- • ALSFRS-R (48 weeks)
Sponsor
AB Science · industry
Contacts & investigators
ContactClinical Study Coordinator · contact · clinical@ab-science.com · +33(0)147200014
InvestigatorAlbert Ludolph, MD, PhD · principal_investigator, Department of Neurology, University of Ulm, Germany
All locations (56)
University of Alabama at BirminghamRecruiting
Birmingham, Alabama, United States
University of Southern CaliforniaRecruiting
Los Angeles, California, United States
University of KentuckyRecruiting
Lexington, Kentucky, United States
Johns Hopkins Medicine Brain Science InstituteRecruiting
Baltimore, Maryland, United States
Lahey Hospital and Medical CenterRecruiting
Burlington, Massachusetts, United States
University of Virginia Health SystemRecruiting
Charlottesville, Virginia, United States
University Hospital Leuven (UZ Leuven)Recruiting
Leuven, Belgium
Bispebjerg HospitalRecruiting
Copenhagen, Denmark
CHU de AngersRecruiting
Angers, France
Groupe Hospitalier Pellegrin TripodeRecruiting
Bordeaux, France
Hôpital neurologique Pierre WertheimerRecruiting
Bron, France
CHU Gabriel MontpiedRecruiting
Clermont-Ferrand, France
CHU de Lille - Hopital Roger SalengroRecruiting
Lille, France
CHU de Limoges - Hôpital DupuytrenRecruiting
Limoges, France
CHU de Marseille - Hôpital de la TimoneRecruiting
Marseille, France
CHRU de Montpellier - Gui de ChauliacRecruiting
Montpellier, France
CHU de Nancy - Hopital CentralRecruiting
Nancy, France
CHU Hôpital Pasteur NiceRecruiting
Nice, France
CHRU de Tours - Hopital BretonneauRecruiting
Tours, France
Department of Neurology, University of UlmNot Yet Recruiting
Ulm, Germany
Athens Naval HospitalRecruiting
Athens, Greece
Eginition HospitalRecruiting
Athens, Greece
University General Hospital of LarissaRecruiting
Larissa, Greece
General University Hospital of PatrasRecruiting
Rio, Greece
Hadassah University HospitalRecruiting
Jerusalem, Israel
Tel-Aviv Medical Center Hôpital Sourasky (ICHILOV)Recruiting
Tel Aviv, Israel
Ospedale Civile Sant'Agostino - EstenseRecruiting
Baggiovara, Modena, Italy
ASST degli Spedali Civili di BresciaRecruiting
Brescia, Italy
Centro Clinico NeMO Fondazione Serena OnlusRecruiting
Gussago, Italy
Clinico Nemo Center (Centro Clinico NeMO Milano)Recruiting
Milan, Italy
IRCCS Istituto Auxologico ItalianoRecruiting
Milan, Italy
Istituti Clinici Scientifici Maugeri IRCCSRecruiting
Milan, Italy
San Raffaele Hospital (Ospedale San Raffaele)Recruiting
Milan, Italy
University Hospital Maggiore della CaritaRecruiting
Novara, Italy
Azienda Ospedale-Università PadovaRecruiting
Padova, Italy
IRCCS Mondino FoundationRecruiting
Pavia, Italy
University Hospital in Turin (Azienda Ospedaliero-Universitaria Città della Salute e della Scienza di Torino)Recruiting
Torino, Italy
Oslo University Hospital HF UllevålRecruiting
Oslo, Norway
Centrum Medyczne NeuromedRecruiting
Bydgoszcz, Poland
Hospital de Santa MariaRecruiting
Lisbon, Portugal
Moscow city clinical Hospital after V.M. BuyanovRecruiting
Moscow, Russia
Scientific Practical Medical Center "Innovation and Health"Recruiting
Novosibirsk, Russia
Clinical Centre of SerbiaRecruiting
Belgrade, Serbia
Klinicni center LjubljanaRecruiting
Ljubljana, Slovenia
Hospital General Universitario de AlicanteRecruiting
Alicante, Spain
Hospital Universitari de BellvitgeRecruiting
Barcelona, Spain
Hospital Carlos IIIRecruiting
Madrid, Spain
Hospital San RafaelRecruiting
Madrid, Spain
Clinical Hospital Santiago de CompostelaRecruiting
Santiago de Compostela, Spain
Hospital Universitario y Politecnico La FeRecruiting
Valencia, Spain
Centralsjukhuset Karlstad (Central Hospital Karlstad)Recruiting
Karlstad, Sweden
Skåne University HospitalRecruiting
Malmö, Sweden
Norrlands universitetssjukhusRecruiting
Umeå, Sweden
The State Institution of Neurology, Psychiatry and Narcology of NAMS of UkraineRecruiting
Kharkiv, Ukraine
Medical Center of LLC Medical Center Dopomoga PlusRecruiting
Kyiv, Ukraine
Communal Non-Profit Enterprise of Lviv Regional Council, Lviv Regional Clinical Hospital, Neurological DepartmentRecruiting
Lviv, Ukraine