Multicenter, Randomised, Double-blind, Placebo-controlled, Parallel Group, Phase 2/3 Study to Compare the Efficacy and Safety of Masitinib

Study Purpose:

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).

Study Status:

Not recruiting


Amyotrophic Lateral Sclerosis (ALS)

Study Type:


Type of Intervention:


Intervention Name:

Masitinib (4.5), Riluzole, Placebo, Masitinib (3.0)



Phase 2/Phase 3

Study Chair(s)/Principal Investigator(s):

Jesus S Mora, MD, Unidad de ELA, Hospital San Rafael, c/ Serrano, 199, 28016 Madrid, Spain ID:


Neals Affiliated?


Coordinating Center Contact Information

Madrid, United States

Full Study Summary:

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 Sponsor:

AB Science

Participant Duration:

up to 48 weeks

Estimated Enrollment:


Estimated Study Start Date:

03 / 31 / 2014

Estimated Study Completion Date:

03 / 01 / 2018

Posting Last Modified Date:

12 / 17 / 2018

Date Study Added to

10 / 28 / 2015

This trial is currently not recruiting in the United States.

Minimum Age:

18 Years

Maximum Age:


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

Hospital Carlos III

Madrid 28029