Amyotrophic Lateral Sclerosis (ALS)
Conditions
Keywords
Amyotrophic Lateral Sclerosis (ALS) - masitinib
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.
Interventions
Sponsors
AB Science
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
DOUBLE (Subject, Investigator)
Eligibility
Sex/Gender
ALL
Age
18 Years to 75 Years
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 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
| Measure | Time frame | Description |
|---|---|---|
| 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. |
Countries
Spain
Outcome results
None listed