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

AB Science394 enrolled

Overview

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).

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 Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

DOUBLE

Enrollment

394

Conditions

Amyotrophic Lateral Sclerosis (ALS)

Interventions

Masitinib (4.5)DRUG

4.5 mg/kg/day

RiluzoleDRUG

PlaceboDRUG

Masitinib (3.0)DRUG

3 mg/kg/day

Eligibility

Sex: ALLMin age: 18 YearsMax age: 75 Years

Medical Language ↔ Plain English

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

Locations (1)

Hospital Carlos III

Madrid, Spain

Outcomes

Primary Outcomes

Change in Amyotrophic Lateral Sclerosis functional rating scale (ALSFRS)-Revised

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)

Time frame: From baseline to week 48

Secondary Outcomes

Change of Forced Vital Capacity (FVC)

Forced vital capacity (FVC) measures the volume of air expelled from the lungs during a quick, forceful breath.

Time frame: From baseline to week 48

Progression Free Survival

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

Time frame: 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

Overall Survival

Overall survival is defined as time in months from the randomization date to the date of death due to any cause.

Time frame: Time from the randomization date until death, asssesd over a maximum of 60 months

Data from ClinicalTrials.gov

This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.