Efficacy and Safety of Masitinib in the Treatment of Progressive Multiple Sclerosis

Study Overview
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Brief Summary
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The purpose of this study is to compare the safety and efficacy of masitinib 6 mg/kg/day versus placebo in the treatment of patients with primary progressive multiple sclerosis or relapse-free secondary progressive multiple sclerosis.

Detailed Description
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Masitinib is a selective tyrosine kinase inhibitor that is thought to exert a neuroprotective effect through its activity on mast cells and other non-neuronal cells of the central nervous system, with subsequent modulation of inflammatory and neurodegenerative processes. The objective of this study is to compare the efficacy and safety of masitinib at 4.5 mg/kg/day versus matched placebo, or masitinib at 4.5 mg/kg/day with a dose escalation to 6 mg/kg/day after 3 months of treatment versus matched placebo, in the treatment of patients with primary progressive multiple sclerosis or relapse-free secondary progressive multiple sclerosis. Approximately 600 patients will be randomized into four treatment groups with a 2:2:1:1 design. The primary outcome measure is the Expanded Disability Status Scale (EDSS) after 96 weeks of treatment in the overall study population with subgroup analysis performed in stratum (primary progressive multiple sclerosis / secondary progressive multiple sclerosis).

Official Title
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A 96 Week, Prospective, Multicentre, Randomized, Double-blind, Placebo-controlled, 2 Parallel-groups, Phase 3 Study to Compare Efficacy and Safety of Masitinib 4.5 mg/kg/Day Versus Placebo in the Treatment of Patients With Primary Progressive or Relapse-free Secondary Progressive Multiple Sclerosis

Conditions
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Multiple Sclerosis, Secondary Progressive, Multiple Sclerosis, Primary Progressive, Multiple Sclerosis, Relapse Free

Intervention / Treatment
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* Drug: Masitinib
* Drug: Placebo


Participation Criteria
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Eligibility Criteria
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Main inclusion criteria: - Patient suffering from either primary progressive or secondary progressive multiple sclerosis without relapse within 2 years before inclusion according to the revised McDonald's criteria. Main exclusion criteria: - Patient suffering from a disease other than MS that would better explain the patient's neurological clinical signs and symptoms and/or MRI lesions

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 75 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Experimental Arm A<br>Participants receive masitinib (4.5 mg/kg/day), given orally twice daily. | Drug: Masitinib<br> <br> * Other names: AB1010;|
| Experimental: Experimental Arm B<br>Participants receive masitinib (4.5 mg/kg/day), given orally twice daily, with a dose escalation to 6 mg/kg/day after 3 months of treatment. | Drug: Masitinib<br> <br> * Other names: AB1010;|
| Placebo Comparator: Placebo Comparator A<br>Participants receive placebo given orally twice daily. | Drug: Placebo<br> <br> * Other names: Placebo Oral Tablet;|
| Placebo Comparator: Placebo Comparator B<br>Participants receive placebo, given orally twice daily, with a matched dose escalation after 3 months of treatment. | Drug: Placebo<br> <br> * Other names: Placebo Oral Tablet;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| EDSS | Expanded Disability Status Scale (EDSS) after 96 weeks of treatment | 96 weeks | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| MSQOL-54 | Multiple Sclerosis Quality of Life 54 items (MSQOL-54) | 96 weeks | 
| MSFC | Multiple Sclerosis Functional Composite (MSFC) | 96 weeks | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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secondary progressive, multiple sclerosis, primary progressive, relapse-free