A 96-Week, Prospective, Multicenter, Randomised, Double-Blind, Placebo-Controlled, Phase 3 Study to Compare Efficacy and Safety of Masitinib Dose Titration to 4.5 mg/kg/day versus Placebo in the Treatment of Patients with Primary Progressive or Secondary Progressive Multiple Sclerosis Without Relapse
This Phase 3 clinical trial is researching a new medicine called masitinib for adults with primary progressive or secondary progressive multiple sclerosis (MS) who are not having sudden flare-ups (relapses). Progressive MS causes symptoms to build up slowly over time, and there are currently very few treatments available to stop this gradual decline. The study aims to find out if taking masitinib daily can slow down changes in walking, hand movement, and daily independence compared with an inactive dummy pill (placebo). Over the course of 96 weeks (just under two years), doctors will track participants' walking speed, memory, brain scans (MRIs), and overall well-being. Half the participants will receive the active study medicine and half will receive the placebo. The results will help determine if masitinib is a safe and effective long-term treatment option for progressive MS.
At a glance
What is this study about?
Multiple sclerosis (MS) is a long-term condition where the body's immune system mistakenly attacks the nerves in the brain and spinal cord. In progressive forms of MS—known as primary progressive or secondary progressive MS—symptoms tend to slowly worsen over time rather than coming and going in sudden flare-ups or relapses. For many people, this gradual change can make everyday tasks like walking, working, or using their hands more difficult. Doctors are urgently searching for treatments that can slow or halt this steady progression.
This trial is looking at an experimental drug called masitinib. Masitinib works by targeting specific immune cells that are thought to cause nerve swelling and damage in the brain. Researchers want to see whether calming these cells helps protect nerve fibres, helping people keep their mobility and independence for longer. Because progressive MS is hard to treat, finding a medicine that protects against slow nerve damage would be an important breakthrough.
To make sure the results are fair and reliable, this is a 'double-blind, placebo-controlled' study. This means participants will take either masitinib or an inactive dummy tablet (a placebo) that looks identical. Neither you nor your study doctor will know which one you are taking until the trial ends. Comparing the two groups allows researchers to clearly see whether any improvements or side effects are truly caused by the study medicine.
Key takeaways
- Tests whether the study drug masitinib slows down physical decline in progressive MS.
- Open to adults aged 18 and older with primary or secondary progressive MS without relapses.
- Lasts for 96 weeks (nearly two years) with regular clinic check-ups and brain MRI scans.
- Half of the participants will receive masitinib and half will receive a dummy pill (placebo).
- Participation is completely voluntary, and you can withdraw at any time.
Who may be eligible?
This study is open to men and women aged 18 and older who have been diagnosed with either primary progressive MS (PPMS) or secondary progressive MS (SPMS). It is specifically designed for people whose MS is progressing steadily without active relapses or sudden flare-ups.
Because this trial looks closely at changes in mobility, doctors will check your current level of disability using a standard medical scale. The trial team will ensure that you are well enough to take part and that joining the study is safe for your overall health.
People who have relapsing-remitting MS, are having frequent sudden flare-ups, or have other serious medical conditions that might make taking part unsafe may not be able to join. The study team will review your full medical history and current medications to confirm if the trial is right for you.
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.
- I am aged 18 or older.
- I have been diagnosed with primary progressive MS or secondary progressive MS.
- My MS symptoms are gradually progressing rather than coming in sudden relapses.
- I am able and willing to attend hospital appointments over a two-year period.
- I am comfortable taking daily tablets at home and having regular brain MRI scans.
What does participation involve?
If you decide to take part, your participation will last for about 96 weeks (nearly two years). You will take tablets by mouth every day at home. The dose starts at a lower level and is increased after four weeks if you tolerate it well. You will have a 50% chance of receiving masitinib and a 50% chance of receiving dummy tablets (placebo).
Throughout the study, you will visit the hospital clinic regularly for routine health checks. These visits will include blood and urine tests, physical check-ups, and discussions about your mood, energy levels, and quality of life.
During visits, the team will run practical tests to check your nerve function. These include a timed 25-foot walking test, a simple pegboard test to check hand and finger movement, and quick puzzles to test memory and thinking. You will also have brain MRI scans at the beginning, at week 48, and at the end of the trial to look for any changes in brain tissue.
Potential risks and benefits
Locations (4)
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Common questions
What is a placebo and why is it used?
A placebo is a dummy pill that looks just like the real medicine but contains no active ingredients. It helps researchers prove whether the real medicine genuinely works better than taking nothing at all.
Can I choose to receive masitinib instead of the placebo?
No. A computer randomly decides who gets masitinib and who gets the placebo, like flipping a coin. Neither you nor your doctor can choose or know which one you are taking.
How long will I be in the study?
The trial lasts for 96 weeks, which is just under two years, with regular visits to the clinic throughout this time.
Will taking part cost me anything?
No, the study medicine and all trial-related tests are provided free of charge. Many trials also help cover travel expenses to and from the hospital.
What happens if I want to leave the trial early?
You are free to stop taking part at any time. Leaving the trial will never affect the standard of care or treatment you receive from your regular doctors.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
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