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)

Study 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).

Study Status:

Recruiting

Disease:

Amyotrophic Lateral Sclerosis

Study Type:

Interventional

Type of Intervention:

Drug

Intervention Name:

Masitinib (6.0), Riluzole, Placebo, Masitinib (4.5)

Placebo:

Yes

Phase:

Phase 3

Study Chair(s)/Principal Investigator(s):

Albert Ludolph, MD, PhD, Department of Neurology, University of Ulm, Germany

Clinicaltrials.gov ID:

NCT03127267

Neals Affiliated?

No

Coordinating Center Contact Information

Clinical Study Coordinator / email hidden; JavaScript is required / +33(0)147200014

Full Study Summary:

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).

Study Sponsor:

AB Science

Estimated Enrollment:

495

Estimated Study Start Date:

02 / 02 / 2021

Estimated Study Completion Date:

12 / 01 / 2023

Posting Last Modified Date:

03 / 18 / 2022

Date Study Added to neals.org:

04 / 25 / 2017

Minimum Age:

18 Years

Maximum Age:

81 Years

Can participants use Riluzole?

Yes

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

University of Alabama at Birmingham, Department of Neurology, Division of Neuromuscular Disease | Recruiting

Birmingham, Alabama 35294
United States

University of Southern California | Recruiting

Los Angeles, California 90007
United States

University of California, Irvine - ALS & Neuromuscular Center | Recruiting

Orange, California 92868
United States

Northwestern University Feinberg School of Medicine | Recruiting

Chicago, Illinois 60611
United States

University of Chicago Neurology | Recruiting

Chicago, Illinois 60637
United States

University of Kentucky | Recruiting

Lexington, Kentucky 40506
United States

Johns Hopkins Medicine Brain Science Institute | Recruiting

Baltimore, Maryland 21205
United States

Lahey Hospital and Medical Center | Recruiting

Burlington, Massachusetts 01805
United States

Mount Sinai Downtown Union Square | Recruiting

New York, New York 10003
United States

Temple University | Recruiting

Philadelphia, Pennsylvania 19122
United States

Bispebjerg Hospital | Recruiting

Copenhagen
Denmark

CHU de Marseille - Hôpital de la Timone | Recruiting

Marseille
France

CHRU de Tours - Hopital Bretonneau | Recruiting

Tours
France

Hôpital Pasteur 2 Centre de Reference SLA et autres maladies du Neurone Moteur Pole Neurosciences Cliniques | Recruiting

Nice
France

CHU de Nancy - Hopital Central | Recruiting

Nancy
France

CHU de Angers | Recruiting

Angers
France

CHRU de Montpellier - Gui de Chauliac | Recruiting

Montpellier
France

CHU de Limoges - Hôpital Dupuytren | Recruiting

Limoges
France

CHU de Lille - Hopital Roger Salengro | Recruiting

Lille
France

CHU Gabriel Montpied | Recruiting

Clermont Ferrand
France

Hôpital neurologique Pierre Wertheimer | Recruiting

Bron
France

Groupe Hospitalier Pellegrin Tripode | Recruiting

Bordeaux
France

Department of Neurology, University of Ulm | Not yet recruiting

Ulm 89081
Germany

Hadassah University Hospital | Recruiting

Jerusalem
Israel

Tel-Aviv Medical Center Hôpital Sourasky (ICHILOV) | Recruiting

Tel Aviv
Israel

Azienda Ospedaliero-Universitaria di Modena | Recruiting

Modena
Italy

Istituto Auxologico Ospedale San Luca | Recruiting

Milano
Italy

Istituti Clinici Scientifici Maugeri IRCCS | Recruiting

Milano
Italy

Centro Clinico NeMO Fondazione Serena Onlus | Recruiting

Gussago
Italy

ASST degli Spedali Civili di Brescia | Recruiting

Brescia
Italy

Oslo University Hospital HF Ullevål | Recruiting

Oslo
Norway

Centrum Medyczne Neuromed | Recruiting

Bydgoszcz
Poland

Moscow city clinical Hospital after V.M. Buyanov | Recruiting

Moscow
Russian Federation

Scientific Practical Medical Center "Innovation and Health" | Recruiting

Novosibirsk
Russian Federation

Klinicni center Ljubljana | Recruiting

Ljubljana
Slovenia

Hospital Universitari de Bellvitge | Recruiting

Barcelona
Spain

Hospital Carlos III | Recruiting

Madrid
Spain

Hospital San Rafael | Recruiting

Madrid
Spain

Clinical Hospital Santiago de Compostela | Recruiting

Santiago De Compostela
Spain

Hospital Universitario y Politecnico La Fe | Recruiting

Valencia
Spain

Norrlands universitetssjukhus | Recruiting

Umeå
Sweden

The State Institution of Neurology, Psychiatry and Narcology of NAMS of Ukraine | Recruiting

Kharkiv
Ukraine

Medical Center of LLC Medical Center Dopomoga Plus | Recruiting

Kyiv
Ukraine

Communal Non-Profit Enterprise of Lviv Regional Council, Lviv Regional Clinical Hospital, Neurological Department | Recruiting

Lviv
Ukraine