Masitinib in Combination With Riluzole for the Treatment of Patients Suffering From Amyotrophic Lateral Sclerosis (ALS)

Phase 2

Completed

• Conditions: Amyotrophic Lateral Sclerosis (ALS)
• Interventions: Drug: Riluzole; Drug: Masitinib (4.5); Drug: Masitinib (3.0); Drug: Placebo

• Registration Number: NCT02588677
• Lead Sponsor: AB Science

Brief Summary

The objective is to compare the efficacy and safety of masitinib in combination with riluzole in the treatment of patients suffering from Amyotrophic Lateral Sclerosis (ALS).

Detailed Description

Masitinib is novel tyrosine kinase inhibitor that targets microglia and mast cells through inhibiting a limited number of kinases. Masitinib blocks microglia proliferation and activation, and mast cell-mediated degranulation, the release of cytotoxic substances that might further damage the motor nerves.

There are two distinct populations of ALS patients: population of "Normal progressors" and population of "Faster progressors". Targeted population for primary analysis is population of "Normal progressors".

"Normal progressors" are ALS patients whose progression of ALSFRS-R score before randomization is less than 1.1 point per month.

Study Timeline

• Study Start: 2013-04
• Study First Submitted: 2015-10-20
• Study First Submitted QC: 2015-10-27
• Study First Posted: 2015-10-28
• Primary Completion: 2016-12-05
• Study Completion: 2018-03
• Disposition First Submitted: 2018-12-12
• Disposition First Submitted QC: 2018-12-12
• Disposition First Posted: 2018-12-14
• Status Verified: 2023-09
• Last Update Submitted: 2023-09-27
• Last Update Posted: 2023-09-29

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 394

Inclusion Criteria

Main inclusion criteria:

1. Familial or sporadic ALS
2. Patient diagnosed with probable of definite ALS
3. Patient treated with a stable dose of riluzole (100 mg/day) for at least 30 days prior to screening

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Exclusion Criteria

1. Patient who underwent tracheostomy and/or gastrostomy

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Masitinib (3.0) & Riluzole	Riluzole	masitinib 3 mg/kg/day + riluzole
Masitinib (3.0) & Riluzole	Masitinib (3.0)	masitinib 3 mg/kg/day + riluzole
Placebo & Riluzole	Placebo	Matched placebo
Masitinib (4.5) & Riluzole	Masitinib (4.5)	masitinib 4.5 mg/kg/day (2) + riluzole
Placebo & Riluzole	Riluzole	Matched placebo
Masitinib (4.5) & Riluzole	Riluzole	masitinib 4.5 mg/kg/day (2) + riluzole

Primary Outcome Measures

Name	Time	Method
Change in Amyotrophic Lateral Sclerosis functional rating scale (ALSFRS)-Revised	From baseline to week 48	The amyotrophic lateral sclerosis functional rating scale (ALSFRS), which is a Validated instrument that assesses the functional status and the disease progression in patients with amyotrophic lateral sclerosis (ALS)

Secondary Outcome Measures

Name	Time	Method
Change of Forced Vital Capacity (FVC)	From baseline to week 48	Forced vital capacity (FVC) measures the volume of air expelled from the lungs during a quick, forceful breath.
Progression Free Survival	Time from the randomization date until the earliest date for a decline of more than 9-points in ALSFRS-R score, asssesd over a maximum of 60 months	Progression Free Survival is defined as the time from the randomization date until the earliest date for a decline of more than 9-points in ALSFRS-R score
Overall Survival	Time from the randomization date until death, asssesd over a maximum of 60 months	Overall survival is defined as time in months from the randomization date to the date of death due to any cause.

Trial Locations

Locations (1)

Hospital Carlos III; 🇪🇸 Madrid, Spain