Back to results

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

Primary Purpose

Amyotrophic Lateral Sclerosis (ALS)

Status

Completed

Phase

Phase 2

Locations

Spain

Study Type

Interventional

Intervention

Masitinib (4.5)

Riluzole

Placebo

Masitinib (3.0)

About this trial

This is an interventional treatment trial for Amyotrophic Lateral Sclerosis (ALS) focused on measuring Amyotrophic Lateral Sclerosis (ALS) - masitinib

Eligibility Criteria

18 Years - 75 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

Main inclusion criteria:

Familial or sporadic ALS
Patient diagnosed with probable of definite ALS
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

Sites / Locations

Hospital Carlos III

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm Type

Experimental

Placebo Comparator

Arm Label

Masitinib (3.0) & Riluzole

Masitinib (4.5) & Riluzole

Placebo & Riluzole

Arm Description

masitinib 3 mg/kg/day + riluzole

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

Matched placebo

Outcomes

Primary Outcome Measures

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)

Secondary Outcome Measures

Change of Forced Vital Capacity (FVC)

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

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

Overall Survival

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

Full Information

NCT ID

NCT02588677

First Posted

October 20, 2015

Last Updated

September 27, 2023

Sponsor

AB Science

1. Study Identification

Unique Protocol Identification Number

NCT02588677

Brief Title

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

Official Title

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

Study Type

Interventional

2. Study Status

Record Verification Date

September 2023

Overall Recruitment Status

Completed

Study Start Date

April 2013 (undefined)

Primary Completion Date

December 5, 2016 (Actual)

Study Completion Date

March 2018 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

AB Science

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Yes

Studies a U.S. FDA-regulated Device Product

Data Monitoring Committee

Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Amyotrophic Lateral Sclerosis (ALS)

Keywords

Amyotrophic Lateral Sclerosis (ALS) - masitinib

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 2, Phase 3

Interventional Study Model

Parallel Assignment

Masking

ParticipantInvestigator

Allocation

Randomized

Enrollment

394 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Masitinib (3.0) & Riluzole

Arm Type

Experimental

Arm Description

masitinib 3 mg/kg/day + riluzole

Arm Title

Masitinib (4.5) & Riluzole

Arm Type

Experimental

Arm Description

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

Arm Title

Placebo & Riluzole

Arm Type

Placebo Comparator

Arm Description

Matched placebo

Intervention Type

Drug

Intervention Name(s)

Masitinib (4.5)

Other Intervention Name(s)

AB1010

Intervention Description

4.5 mg/kg/day

Intervention Type

Drug

Intervention Name(s)

Riluzole

Other Intervention Name(s)

Rilutek

Intervention Type

Drug

Intervention Name(s)

Placebo

Other Intervention Name(s)

Placebo Oral Tablet

Intervention Type

Drug

Intervention Name(s)

Masitinib (3.0)

Other Intervention Name(s)

AB1010

Intervention Description

3 mg/kg/day

Primary Outcome Measure Information:

Title

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

Description

Time Frame

From baseline to week 48

Secondary Outcome Measure Information:

Title

Change of Forced Vital Capacity (FVC)

Description

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

Title

Progression Free Survival

Description

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

Title

Overall Survival

Description

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

10. Eligibility

Sex

All

Minimum Age & Unit of Time

18 Years

Maximum Age & Unit of Time

75 Years

Accepts Healthy Volunteers

Eligibility Criteria

Inclusion Criteria: Main inclusion criteria: Familial or sporadic ALS Patient diagnosed with probable of definite ALS 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

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Jesus S Mora, MD

Organizational Affiliation

Unidad de ELA, Hospital San Rafael, c/ Serrano, 199, 28016 Madrid, Spain

Official's Role

Principal Investigator

Facility Information:

Facility Name

Hospital Carlos III

City

Madrid

ZIP/Postal Code

28029

Country

Spain

12. IPD Sharing Statement

Citations:

PubMed Identifier

34457038

Citation

Mora JS, Bradley WG, Chaverri D, Hernandez-Barral M, Mascias J, Gamez J, Gargiulo-Monachelli GM, Moussy A, Mansfield CD, Hermine O, Ludolph AC. Long-term survival analysis of masitinib in amyotrophic lateral sclerosis. Ther Adv Neurol Disord. 2021 Jul 19;14:17562864211030365. doi: 10.1177/17562864211030365. eCollection 2021.

Results Reference

derived

Learn more about this trial

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

We'll reach out to this number within 24 hrs