RecruitingPHASE3INTERVENTIONAL

Efficacy and Safety of Masitinib Versus Placebo in the Treatment of ALS Patients

This research is looking into a new medication called Masitinib for people living with Amyotrophic Lateral Sclerosis (ALS), also known as Motor Neurone Disease. The main goal is to find out if Masitinib, when added to the standard treatment Riluzole, is safe and effective in slowing down the illness. We'll be comparing it to people who receive Riluzole plus a placebo (a dummy pill). Masitinib works by targeting certain cells in the body that are thought to be involved in the disease, potentially helping to reduce inflammation and protect nerve cells. This is a large study involving many clinics, where some people will receive Masitinib and others a placebo, but no one will know which they are getting until the study ends.

At a glance

Status

Recruiting

Phase

PHASE3

Sponsor

AB Science

Enrolment target

495

Start

02 Feb 2021

Estimated completion

01 Dec 2027

Amyotrophic Lateral Sclerosis

What is this study about?

This study is investigating a new oral medicine called Masitinib for people with Amyotrophic Lateral Sclerosis (ALS), which is also known as Motor Neurone Disease. ALS is a condition that affects nerve cells in the brain and spinal cord, leading to muscle weakness and wasting. There's currently no cure, but treatments like Riluzole can help manage symptoms and slow progression.

Masitinib is designed to work by targeting specific types of cells in the body, such as mast cells and microglia. These cells are known to play a role in inflammation and other processes within the nervous system. The idea behind this medication is that by acting on these cells, Masitinib might be able to reduce inflammation, protect nerve cells, and potentially slow down the progression of ALS. Researchers want to see if adding Masitinib to your current Riluzole treatment makes a difference compared to taking Riluzole with a placebo (an inactive dummy pill).

This is a 'Phase 3' study, which means it's a large, important trial where the medicine has already shown some promise in earlier stages. It's designed to be 'double-blind,' meaning neither you nor your doctor will know if you're receiving Masitinib or the placebo, which helps make the results as fair and reliable as possible. The study will also be 'randomised,' meaning a computer will decide which group you are in, ensuring the groups are as similar as possible.

Key takeaways

This study is testing a new drug, Masitinib, for Motor Neurone Disease (ALS).
It aims to see if Masitinib, combined with Riluzole, can slow disease progression.
Participants will receive either Masitinib or a placebo, alongside their Riluzole.
The study is 'double-blind', so you won't know which treatment you are on.
It involves regular clinic visits for health checks and assessments.

Who may be eligible?

To be considered for this study, you would generally need to have a confirmed diagnosis of ALS, whether it's the more common type or runs in your family. Your diagnosis should have been made within the last two years (24 months) before screening for the study. You also need to be taking Riluzole at a stable dose of 100mg per day for at least 12 weeks before your first study visit.

Researchers will also look at how quickly your ALS has progressed before you join the study. This is measured using a recognised scale for ALS symptoms. You'll need to have a certain score on this scale to be included.

There are also reasons why you might not be able to join. For example, if you have other significant health conditions like dementia, or other serious neurological or psychiatric illnesses that could affect your participation or the study results. You also wouldn't be able to join if your breathing function is too reduced, or if you are pregnant or breastfeeding.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Have I been diagnosed with ALS (Motor Neurone Disease)?
Was my ALS diagnosis made within the last two years (24 months)?
Am I currently taking Riluzole at a stable dose of 100mg daily for at least 12 weeks?
Do I have any other major health conditions, especially serious heart, lung, or mental health issues?
Am I pregnant or breastfeeding, or planning to become pregnant?

Answer every question to see your result.

What does participation involve?

If you decide to take part, you'll be given either Masitinib or a placebo (dummy pill) to take alongside your regular Riluzole. The study involves regular visits to the clinic where doctors and nurses will monitor your health, check for any changes in your ALS symptoms, and collect samples like blood. You'll also complete questionnaires about your health and how you are feeling. The study involves several assessments over time to track your condition. The total duration of your participation in the study, including all follow-up, will be explained by the study team, but typically, these types of trials can last for many months to a couple of years.

Potential risks and benefits

Participating in a clinical trial may offer potential benefits, such as access to a new treatment that isn't yet widely available, and close monitoring of your health by medical experts. However, there are also potential risks; Masitinib might not work for you, and it could cause side effects, some of which are not yet fully known. The placebo group will not receive the active study drug. You are free to withdraw from the study at any time, for any reason, without it affecting your medical care.

Locations (56)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

University of Alabama at Birmingham
Verified postcode
Birmingham, United States· Recruiting
University of Southern California
Verified postcode
Los Angeles, United States· Recruiting
University of Kentucky
Verified postcode
Lexington, United States· Recruiting
Johns Hopkins Medicine Brain Science Institute
Verified postcode
Baltimore, United States· Recruiting
Lahey Hospital and Medical Center
Verified postcode
Burlington, United States· Recruiting
University of Virginia Health System
Verified postcode
Charlottesville, United States· Recruiting
University Hospital Leuven (UZ Leuven)
Verified postcode
Leuven, Belgium· Recruiting
Bispebjerg Hospital
Verified postcode
Copenhagen, Denmark· Recruiting
CHU de Angers
Verified postcode
Angers, France· Recruiting
Groupe Hospitalier Pellegrin Tripode
Verified postcode
Bordeaux, France· Recruiting
Hôpital neurologique Pierre Wertheimer
Verified postcode
Bron, France· Recruiting
CHU Gabriel Montpied
Verified postcode
Clermont-Ferrand, France· Recruiting

Common questions

What is Masitinib?

Masitinib is an investigational medicine being tested for ALS. It works by targeting certain cells involved in inflammation and nerve health.

What is a placebo?

A placebo is a 'dummy pill' that looks exactly like the study medicine but contains no active drug. It helps researchers compare the effects of the active treatment.

Will I know if I'm taking the real medicine or the placebo?

No, this is a 'double-blind' study, meaning neither you nor your doctors will know if you are receiving Masitinib or the placebo until the study is over.

Will I still take my regular Riluzole?

Yes, all participants in this study will continue to take their stable dose of Riluzole throughout the trial.

What happens if I want to stop participating?

You are free to leave the study at any time, for any reason, and this decision will not affect your future medical care.

How to find out more

Clinical Study Coordinator

clinical@ab-science.com +33(0)147200014 Official trial record

Always speak to your GP or specialist before deciding to take part in a study.