A prospective, multicenter, randomised, double-blind, placebo-controlled, parallel groups, phase 3 Trial to compare the efficacy and safety of masitinib in combination with standard of care versus placebo in combination with standard of care in the treatment of patients suffering from Amyotrophic Lateral Sclerosis (ALS)
This study is looking into a new treatment called masitinib for people living with Amyotrophic Lateral Sclerosis (ALS), also known as Motor Neurone Disease. Researchers want to find out if masitinib, taken alongside your usual medications, is both safe and effective in slowing down the progression of the disease. Participants will be split into groups; some will receive masitinib and standard care, while others will get a dummy pill (placebo) and standard care. Neither the patients nor their doctors will know who is getting which treatment. The main goal is to see if masitinib helps improve or maintain a patient's ability to carry out daily activities over 48 weeks, and to check for any side effects.
At a glance
What is this study about?
This research study is about finding new ways to help people living with Amyotrophic Lateral Sclerosis (ALS), often called Motor Neurone Disease. ALS is a condition that affects the nerve cells in the brain and spinal cord, making it harder for people to move, speak, swallow, and breathe over time. Currently, there's no cure, so doctors are always looking for treatments that can slow down the disease or improve quality of life. This study is testing a new medicine called masitinib.
Masitinib would be taken alongside the standard treatments you might already be receiving. The study aims to compare how people who take masitinib do compared to those who take a dummy pill (called a placebo) – both groups will also receive their usual care. By doing this comparison, researchers can tell if any changes seen are due to masitinib or if they would have happened anyway. This type of study is called 'double-blind,' which means neither you nor your doctor will know if you're taking masitinib or the placebo. This helps make the study fair and accurate.
The main thing the researchers want to find out is whether masitinib can help to slow down the progression of ALS symptoms over 48 weeks. They will measure this by looking at how well people can carry out daily activities using a special score. They will also be very carefully monitoring for any side effects to make sure the drug is safe. This is a vital step in trying to find better treatments for ALS.
Key takeaways
- This study evaluates a new drug, masitinib, for Motor Neurone Disease (ALS).
- It aims to see if masitinib is safe and can slow down the disease's progression.
- Participants will receive either masitinib or a dummy pill (placebo) alongside their usual care.
- The study is 'double-blind,' meaning no one knows who gets which treatment.
- Researchers will closely monitor health and symptoms for 48 weeks.
- Participation is voluntary, and you can withdraw anytime.
Who may be eligible?
To be able to join this study, you would need to be an adult, aged 18 years or older, and have received a diagnosis of Amyotrophic Lateral Sclerosis (ALS).
Both men and women are welcome to take part in this research.
There might be other specific health requirements or conditions that would need to be checked by the study doctors to make sure the trial is safe and suitable for you.
- Are you 18 years old or older?
- Do you have a diagnosis of Amyotrophic Lateral Sclerosis (ALS)?
- Are you able to attend regular clinic appointments for about 48 weeks?
- Are you currently receiving or willing to receive standard ALS care?
This is a guide only — the research team will confirm whether you can take part.
What does participation involve?
If you decide to take part in this study, you would be randomly assigned to one of two groups: either you would receive the new drug, masitinib, along with your usual care, or you would receive a dummy pill (placebo) plus your usual care. You'll need to take the study medication regularly as instructed by the doctors.
Throughout the study, you'll have regular visits to the clinic. During these visits, doctors and nurses will carry out various tests and assessments. This will include checking your health, taking blood samples, monitoring your heart (with an ECG), checking your vital signs like blood pressure and weight, and assessing how your ALS symptoms are progressing. They will also ask about any side effects you might be experiencing. These visits will help the researchers understand how the treatment is working and make sure you're safe. The main part of the study monitoring will last for 48 weeks, but there might be some longer-term follow-up.
Potential risks and benefits
Locations (7)
- —Spain
- —Slovenia
- —Greece
- —Sweden
- —France
- —Latvia
- —Germany
Common questions
What is ALS?
ALS stands for Amyotrophic Lateral Sclerosis, also known as Motor Neurone Disease. It's a condition that affects nerve cells, leading to muscle weakness and problems with movement, speech, and breathing.
What is masitinib?
Masitinib is a drug being studied as a potential new treatment for ALS. This study is trying to find out if it's safe and effective in slowing the disease down.
What is a 'placebo'?
A placebo is a dummy pill that looks exactly like the study drug but doesn't contain any active medicine. It's used in studies to fairly compare the new drug's effects.
What does 'double-blind' mean?
'Double-blind' means that neither you nor your study doctor will know whether you are receiving masitinib or the placebo. This helps ensure the results are unbiased.
How long will the study last for me?
The main treatment and assessment period for the study is 48 weeks, which is about 11 months.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
Discussion
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