Amyotrophic Lateral Sclerosis (ALS)Phase 2/3NCT02588677
Masitinib in Combination With Riluzole for the Treatment of Patients Suffering From Amyotrophic Lateral Sclerosis (ALS)
A Phase 2/3 study testing Masitinib (4.5), Riluzole and Masitinib (3.0) in people with Amyotrophic Lateral Sclerosis (ALS), run by AB Science at 1 site in Spain.
Trial 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). Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Amyotrophic Lateral Sclerosis (ALS)
- What is being tested?
- Masitinib (4.5), Riluzole and Masitinib (3.0)
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 394 participants
- Where
- 1 siteSpain
- Study dates
- Apr 2013to Mar 2018
- Phase
- Phase 2/3
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 1. Patient who underwent tracheostomy and/or gastrostomy
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Multicenter, Randomised, Double-blind, Placebo-controlled, Parallel Group, Phase 2/3 Study to Compare the Efficacy and Safety of Masitinib
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).
Read the 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.
- Conditions
- Amyotrophic Lateral Sclerosis (ALS)
- Being tested
- Masitinib (4.5), Riluzole, Placebo, Masitinib (3.0)
- Keywords
- Amyotrophic Lateral Sclerosis (ALS) - masitinib
- Medical subject headings
- Amyotrophic Lateral Sclerosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Masitinib (4.5)Drug
Also called AB1010
4.5 mg/kg/day
RiluzoleDrug
Also called Rilutek
PlaceboDrug
Also called Placebo Oral Tablet
Masitinib (3.0)Drug
Also called AB1010
3 mg/kg/day
| Study group | Role | What they receive |
|---|---|---|
| Masitinib (3.0) & Riluzole | Experimental | masitinib 3 mg/kg/day + riluzole Receives: Drug: Riluzole; Drug: Masitinib (3.0) |
| Masitinib (4.5) & Riluzole | Experimental | masitinib 4.5 mg/kg/day (2) + riluzole Receives: Drug: Masitinib (4.5); Drug: Riluzole |
| Placebo & Riluzole | Placebo comparator | Matched placebo Receives: Drug: Riluzole; Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
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)
Measured: From baseline to week 48
Secondary outcomes (3)
Change of Forced Vital Capacity (FVC)
Forced vital capacity (FVC) measures the volume of air expelled from the lungs during a quick, forceful breath.
Measured: 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
Measured: 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.
Measured: Time from the randomization date until death, asssesd over a maximum of 60 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Oct 2015 (estimated)
- Study startApr 2013
- Primary completion5 Dec 2016
- Study completionMar 2018
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Amyotrophic Lateral Sclerosis (ALS)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2/3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 394 participants (actual)
Sponsor and investigators
- Lead sponsor
- AB Science
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Jesus S Mora, MD, Principal investigator, Unidad de ELA, Hospital San Rafael, c/ Serrano, 199, 28016 Madrid, Spain
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02588677
- Sponsor's protocol ID
- AB10015
- Status last verified
- Sep 2023
- First submitted
- 20 Oct 2015
- Last updated
- 29 Sep 2023
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Publications
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.
PubMed 34457038
Study at a glance
From the registry record, last updated by the sponsor on 29 Sep 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Multicenter, Randomised, Double-blind, Placebo-controlled, Parallel Group, Phase 2/3 Study to Compare the Efficacy and Safety of Masitinib
- ClinicalTrials.gov ID
- NCT02588677
- Acronym / sponsor ID
- AB10015
- Conditions
- Amyotrophic Lateral Sclerosis (ALS)
- Keywords
- Amyotrophic Lateral Sclerosis (ALS) - masitinib
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 394 participants (actual)
- Lead sponsor
- AB ScienceIndustry (pharma, biotech, devices)
- Study start
- Apr 2013
- Primary completion
- 5 Dec 2016 (actual)
- Study completion
- Mar 2018 (actual)
- First posted
- 28 Oct 2015 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Masitinib (3.0) & Riluzole | Experimental | Drug: Riluzole, Drug: Masitinib (3.0)masitinib 3 mg/kg/day + riluzole |
| Masitinib (4.5) & Riluzole | Experimental | Drug: Masitinib (4.5), Drug: Riluzolemasitinib 4.5 mg/kg/day (2) + riluzole |
| Placebo & Riluzole | Placebo comparator | Drug: Riluzole, Drug: PlaceboMatched placebo |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- 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
Key exclusion criteria (1 listed)
- 1. Patient who underwent tracheostomy and/or gastrostomy
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Masitinib (4.5)Also: AB10104.5 mg/kg/day | Drug | Masitinib (4.5) & Riluzole |
| RiluzoleAlso: Rilutek | Drug | Masitinib (3.0) & Riluzole, Masitinib (4.5) & Riluzole, Placebo & Riluzole |
| PlaceboAlso: Placebo Oral Tablet | Drug | Placebo & Riluzole |
| Masitinib (3.0)Also: AB10103 mg/kg/day | Drug | Masitinib (3.0) & Riluzole |
Primary outcome
Change in Amyotrophic Lateral Sclerosis functional rating scale (ALSFRS)-Revised
Time frame: 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)
3 secondary outcomes
Change of Forced Vital Capacity (FVC)
Time frame: From baseline to week 48
Progression Free Survival
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
Time frame: Time from the randomization date until death, asssesd over a maximum of 60 months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 Sep 2023.
- Derived from this study: PubMed 34457038Mora 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Jesus S Mora, MD principal investigator
Unidad de ELA, Hospital San Rafael, c/ Serrano, 199, 28016 Madrid, Spain
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing Masitinib (4.5), Riluzole and Masitinib (3.0) in people with Amyotrophic Lateral Sclerosis (ALS), run by AB Science at 1 site in Spain.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 29 Sep 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Spain. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in Amyotrophic Lateral Sclerosis functional rating scale (ALSFRS)-Revised (time frame: From baseline to week 48). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02588677, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 29 Sep 2023.