Randomized, Double-Blind, Placebo-Controlled, Phase 2 Study of MRM-3379 in Male Participants with Fragile X Syndrome
This research study is looking at a new medicine called MRM-3379 for men diagnosed with Fragile X Syndrome. The main goal is to check how safe this medicine is and whether people can take it without too many problems. Researchers will carefully watch for any side effects. They also want to see if MRM-3379 might help improve thinking and learning skills, such as understanding words and reading. To do this, some participants will receive the actual medicine, while others will be given a placebo – a dummy pill that looks the same but contains no active medicine. This helps researchers compare the effects accurately. The study is particularly interested in how the medicine behaves in the body.
At a glance
What is this study about?
This study is trying to find out more about a new medicine called MRM-3379. It's specifically for men who have Fragile X Syndrome. Fragile X Syndrome is a genetic condition that can cause various learning difficulties and developmental challenges. The main purpose of this study is to see if MRM-3379 is safe for people to take and if it causes any unwanted side effects. Researchers will be carefully monitoring participants for any health changes throughout the study.
Beyond safety, the study also aims to explore if MRM-3379 could help improve certain thinking and learning abilities. This includes things like understanding words and recognising written words. To make sure the results are reliable, some men will receive the new medicine, while others will get a 'dummy' pill (called a placebo) that looks identical but has no active ingredients. Neither the participants nor the doctors will know who is getting which, to keep the study fair. This setup helps researchers work out if any changes are truly due to the medicine or just other factors.
Finally, the study will also look at how MRM-3379 moves through the body, how long it stays there, and how it is broken down. This information is important for understanding the best way to give the medicine if it proves to be helpful.
Key takeaways
- This study is testing a new medicine (MRM-3379) for men with Fragile X Syndrome.
- The main goals are to check the medicine's safety and see if it helps with thinking skills.
- Some participants will receive the medicine, others a dummy pill (placebo).
- Only men aged 18 and over can take part.
- Participation involves regular clinic visits, health checks, and specific tests.
- You can stop participating at any time.
Who may be eligible?
This study is specifically looking for men who have Fragile X Syndrome. You must be at least 18 years old to take part; there is no upper age limit for joining.
Before you can join, the study team will need to check some other details about your health and medical history to make sure the study is a good fit for you. This helps ensure your safety and that the study results are clear.
It's important to understand that even if you meet the main age and gender requirements, there might be other specific health checks that need to be done. A doctor from the study team will talk you through all of these points.
- Are you a man?
- Do you have a diagnosis of Fragile X Syndrome?
- Are you 18 years old or older?
- Are you able to attend regular clinic visits?
- Are you willing to take study medication (which might be a placebo)?
- Are you willing to undergo various health checks, including blood tests and thinking assessments?
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 will have several visits to the study clinic. During these visits, doctors and nurses will carry out various tests and check-ups to monitor your health. You will be given either the study medicine (MRM-3379) or a placebo (a dummy pill) to take regularly, depending on what the study team decides. You won't know which one you're receiving.
Assessments will include regular health check-ups, blood tests, and special tests to measure your thinking and learning abilities. These tests might involve looking at pictures, words, or answering questions. The study team will meticulously record any side effects you experience.
You'll be monitored throughout the study period, and there will be follow-up appointments after you stop taking the study medicine to ensure you are well. The total duration of your involvement in the study will be explained in detail by the research team.
Potential risks and benefits
Locations (1)
- —Spain
Common questions
What is Fragile X Syndrome?
Fragile X Syndrome is a genetic condition that can affect a person's learning abilities, behaviour, and overall development.
What is a 'placebo'?
A placebo is a 'dummy' pill or treatment that looks exactly like the real medicine but doesn't contain any active ingredients. It helps researchers compare the actual effects of the medicine.
Will I know if I'm getting the real medicine or the placebo?
No, both you and the study doctors won't know whether you're receiving the real medicine or the placebo. This is to ensure the study results are fair and unbiased.
What are 'side effects'?
Side effects are any unwanted or unexpected effects a medicine might have on your body. The study will carefully track all of these.
How long will I be in the study?
The total length of your involvement in the study will be clearly explained by the research team if you are eligible and interested in joining.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
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