A Prospective, Multicenter, Randomised, Double-blind, Placebo-controlled, Parallel Groups, 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

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).

Disease:

Amyotrophic Lateral Sclerosis (ALS),  Familial ALS,  Sporadic ALS

Study Type:

Interventional Trial

Study Category:

Drug Trial

Study Status:

Enrolling

Phase:

Phase III

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

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

Clinicaltrials.gov ID (11 digit #):

NCT03127267

Neals Affiliated?

No

Coordinating Center Contact Information


Clinical Study Coordinator / .(JavaScript must be enabled to view this email address) / +33(0)147200014
/ .(JavaScript must be enabled to view this email address)

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

Participant Duration:

Estimated Enrollment:

495

Estimated Study Start Date:

10/01/2020

Estimated Study Completion Date:

12/31/2022

Posting Last Modified Date:

01/04/2021

Date Study Added to alsconsortium.org:

11/08/2017
  • Eligibility Criteria

    Gender:

    Female, Male

    Minimum Age:

    18

    Maximum Age:

    81

    Time since Symptom Onset:

    Time since Diagnosis:

    <24 months

    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

  • Site Contact Information

    Johns Hopkins
    Baltimore, Maryland 21205
    United States

    University of Ulm
    Ulm,
    Germany