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Trial record 2 of 2 for:    AB Science | Amyotrophic Lateral Sclerosis and Masitinib

Efficacy and Safety of Masitinib Versus Placebo in the Treatment of ALS Patients

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ClinicalTrials.gov Identifier: NCT03127267
Recruitment Status : Not yet recruiting
First Posted : April 25, 2017
Last Update Posted : April 6, 2020
Sponsor:
Information provided by (Responsible Party):
AB Science

Tracking Information
First Submitted Date  ICMJE April 13, 2017
First Posted Date  ICMJE April 25, 2017
Last Update Posted Date April 6, 2020
Estimated Study Start Date  ICMJE June 2020
Estimated Primary Completion Date June 2022   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: December 7, 2018)
ALSFRS-R [ Time Frame: 48 weeks ]
Change in Amyotrophic Lateral Sclerosis functional rating scale (ALSFRS)-Revised.
Original Primary Outcome Measures  ICMJE
 (submitted: April 20, 2017)
Absolute change from baseline to week 48 in Amyotrophic Lateral Sclerosis functional rating scale (ALSFRS)-Revised. [ Time Frame: Week 48 ]
Revised-ALS Functional Rating Score
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: December 7, 2018)
  • ALSAQ-40 [ Time Frame: 48 weeks ]
    Change in ALS quality of life patient questionnaire (ALSAQ-40)
  • PFS [ Time Frame: From day of randomization to disease progression or death, assessed for a maximum of 36 months ]
    Progression free survival (PFS) is defined as the time from randomization to progression (decline of more than 9 points in ALSFRS-R score from baseline) or death
  • FVC [ Time Frame: 48 weeks ]
    Change in Forced Vital Capacity (FVC)
  • HHD [ Time Frame: 48 weeks ]
    Change in evaluation of upper- and lower-limb muscle strength using hand-held dynamometry (HHD)
Original Secondary Outcome Measures  ICMJE
 (submitted: April 20, 2017)
Change in ALSAQ-40 from baseline to week 48 [ Time Frame: Week 48 ]
ALS quality of life patient questionnaire
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Efficacy and Safety of Masitinib Versus Placebo in the Treatment of ALS Patients
Official Title  ICMJE 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 Suffering From Amyotrophic Lateral Sclerosis (ALS)
Brief Summary 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).
Detailed Description 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 Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double (Participant, Investigator)
Primary Purpose: Treatment
Condition  ICMJE Amyotrophic Lateral Sclerosis
Intervention  ICMJE
  • Drug: Masitinib (6.0)
    Masitinib (titration to 6.0 mg/kg/day)
    Other Name: AB1010
  • Drug: Riluzole
    Riluzole 50 mg tablet, treatment per os
    Other Name: Rilutek
  • Drug: Placebo
    treatment per os
    Other Name: Placebo Oral Tablet
  • Drug: Masitinib (4.5)
    Masitinib (titration to 4.5 mg/kg/day)
    Other Name: AB1010
Study Arms  ICMJE
  • Experimental: Masitinib (4.5) & 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
    Interventions:
    • Drug: Riluzole
    • Drug: Masitinib (4.5)
  • Experimental: Masitinib (6.0) & 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, 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.
    Interventions:
    • Drug: Masitinib (6.0)
    • Drug: Riluzole
  • Placebo Comparator: Placebo & Riluzole
    Participants receive a matched dose placebo, given orally twice daily, in combination with riluzole at 50 mg b.i.d.
    Interventions:
    • Drug: Riluzole
    • Drug: Placebo
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Not yet recruiting
Estimated Enrollment  ICMJE
 (submitted: December 7, 2018)
495
Original Estimated Enrollment  ICMJE
 (submitted: April 20, 2017)
406
Estimated Study Completion Date  ICMJE September 2022
Estimated Primary Completion Date June 2022   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years to 81 Years   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Clinical Study Coordinator +33(0)147200014 clinical@ab-science.com
Listed Location Countries  ICMJE Germany
Removed Location Countries Canada
 
Administrative Information
NCT Number  ICMJE NCT03127267
Other Study ID Numbers  ICMJE AB19001
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: Undecided
Responsible Party AB Science
Study Sponsor  ICMJE AB Science
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: Albert Ludolph, MD, PhD Department of Neurology, University of Ulm, Germany
PRS Account AB Science
Verification Date April 2020

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP