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

AB Science

Status and phase: Completed; Phase 3; Phase 2

Conditions: Amyotrophic Lateral Sclerosis (ALS)

Treatments: Drug: Riluzole; Drug: Masitinib (4.5); Drug: Masitinib (3.0); Drug: Placebo

Study type: Interventional

Funder types: Industry

Identifiers

NCT02588677

AB10015

Details and patient eligibility

About

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).

Full 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.

Enrollment: 394 patients

Sex: All

Ages: 18 to 75 years old

Volunteers: No Healthy Volunteers

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

Exclusion criteria

1. Patient who underwent tracheostomy and/or gastrostomy

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Double Blind

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394 participants in 3 patient groups, including a placebo group

Masitinib (3.0) & Riluzole

Experimental group

Description:

masitinib 3 mg/kg/day + riluzole

Treatment:

Drug: Masitinib (3.0)

Drug: Riluzole

Masitinib (4.5) & Riluzole

Experimental group

Description:

masitinib 4.5 mg/kg/day (2) + riluzole

Treatment:

Drug: Masitinib (4.5)

Drug: Riluzole

Placebo & Riluzole

Placebo Comparator group

Description:

Matched placebo

Treatment:

Drug: Placebo

Drug: Riluzole