Efficacy and Safety of Masitinib Versus Placebo in the Treatment of ALS Patients
Amyotrophic Lateral Sclerosis
About this trial
This is an interventional treatment trial for Amyotrophic Lateral Sclerosis focused on measuring Amyotrophic Lateral Sclerosis, ALS, Tyrosine kinase inhibitor, Lou Gehrig's disease, Charcot's disease, Motor Neuron disease, MND
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
Sites / Locations
- University of Alabama at BirminghamRecruiting
- University of Southern CaliforniaRecruiting
- University of KentuckyRecruiting
- Johns Hopkins Medicine Brain Science InstituteRecruiting
- Lahey Hospital and Medical CenterRecruiting
- University of Virginia Health SystemRecruiting
- University Hospital Leuven (UZ Leuven)Recruiting
- Bispebjerg HospitalRecruiting
- CHU de AngersRecruiting
- Groupe Hospitalier Pellegrin TripodeRecruiting
- Hôpital neurologique Pierre WertheimerRecruiting
- CHU Gabriel MontpiedRecruiting
- CHU de Lille - Hopital Roger SalengroRecruiting
- CHU de Limoges - Hôpital DupuytrenRecruiting
- CHU de Marseille - Hôpital de la TimoneRecruiting
- CHRU de Montpellier - Gui de ChauliacRecruiting
- CHU de Nancy - Hopital CentralRecruiting
- CHU Hôpital Pasteur NiceRecruiting
- CHRU de Tours - Hopital BretonneauRecruiting
- Department of Neurology, University of Ulm
- Athens Naval HospitalRecruiting
- Eginition HospitalRecruiting
- University General Hospital of LarissaRecruiting
- General University Hospital of PatrasRecruiting
- Hadassah University HospitalRecruiting
- Tel-Aviv Medical Center Hôpital Sourasky (ICHILOV)Recruiting
- Ospedale Civile Sant'Agostino - EstenseRecruiting
- ASST degli Spedali Civili di BresciaRecruiting
- Centro Clinico NeMO Fondazione Serena OnlusRecruiting
- Clinico Nemo Center (Centro Clinico NeMO Milano)Recruiting
- IRCCS Istituto Auxologico ItalianoRecruiting
- Istituti Clinici Scientifici Maugeri IRCCSRecruiting
- San Raffaele Hospital (Ospedale San Raffaele)Recruiting
- University Hospital Maggiore della CaritaRecruiting
- Azienda Ospedale-Università PadovaRecruiting
- IRCCS Mondino FoundationRecruiting
- University Hospital in Turin (Azienda Ospedaliero-Universitaria Città della Salute e della Scienza di Torino)Recruiting
- Oslo University Hospital HF UllevålRecruiting
- Centrum Medyczne NeuromedRecruiting
- Hospital de Santa MariaRecruiting
- Moscow city clinical Hospital after V.M. BuyanovRecruiting
- Scientific Practical Medical Center "Innovation and Health"Recruiting
- Clinical Centre of SerbiaRecruiting
- Klinicni center LjubljanaRecruiting
- Hospital General Universitario de AlicanteRecruiting
- Hospital Universitari de BellvitgeRecruiting
- Hospital Carlos IIIRecruiting
- Hospital San RafaelRecruiting
- Clinical Hospital Santiago de CompostelaRecruiting
- Hospital Universitario y Politecnico La FeRecruiting
- Centralsjukhuset Karlstad (Central Hospital Karlstad)Recruiting
- Skåne University HospitalRecruiting
- Norrlands universitetssjukhusRecruiting
- The State Institution of Neurology, Psychiatry and Narcology of NAMS of UkraineRecruiting
- Medical Center of LLC Medical Center Dopomoga PlusRecruiting
- Communal Non-Profit Enterprise of Lviv Regional Council, Lviv Regional Clinical Hospital, Neurological DepartmentRecruiting
Arms of the Study
Arm 1
Arm 2
Arm 3
Experimental
Experimental
Placebo Comparator
Masitinib (4.5) & Riluzole
Masitinib (6.0) & Riluzole
Placebo & Riluzole
Participants receive masitinib (3.0 mg/kg/day), given orally twice daily, with a dose escalation to 4.5 mg/kg/day after 4 weeks of treatment. Each ascending dose titration is subjected to a safety control. Masitinib will be administered as an add-on to riluzole at 50 mg b.i.d
Participants receive masitinib (3.0 mg/kg/day), given orally twice daily, with a dose escalation to 4.5 mg/kg/day after 4 weeks of treatment, followed by dose escalation to 6.0 mg/kg/day after 4 weeks of treatment. Each ascending dose titration is subjected to a safety control. Masitinib will be administered as an add-on to riluzole at 50 mg b.i.d.
Participants receive a matched dose placebo, given orally twice daily, in combination with riluzole at 50 mg b.i.d.