An Open-Label Extension Study to Evaluate the Long-Term Safety and Efficacy of Once Daily Mexiletine PR in Patients With Myotonic Dystrophy Type 1 and Type 2 Who Have Completed MEX-DM-302 Study.
This study is an extension of a previous trial, looking further into the long-term safety and how well a drug called mexiletine works for individuals with Myotonic Dystrophy Type 1 or Type 2. If you've already finished the initial MEX-DM-302 study and meet certain health requirements, you might be invited to join this extension. All participants in this part of the study will receive the active medication, not a dummy pill, for an additional 18 months. Doctors will carefully monitor your health and how the treatment affects your symptoms over time.
At a glance
What is this study about?
This study is about a medication called mexiletine, which is being tested for people living with Myotonic Dystrophy, specifically types 1 and 2. Myotonic Dystrophy is a condition that causes muscles to weaken and stop working properly over time.
This particular study is an 'extension' of a previous trial. This means that if you've already taken part in the earlier study (called MEX-DM-302) and found the treatment helpful, your doctors might invite you to continue receiving mexiletine for another 18 months. The main goal is to see if the medication continues to be safe and how well it works over a longer period.
It's important to understand that in this extension study, everyone will receive the active drug. There won't be a placebo (dummy pill) group. This allows researchers to gather more information about mexiletine's long-term effects, helping them understand if it can be a helpful treatment option for people with Myotonic Dystrophy.
Key takeaways
- This study is for people with Myotonic Dystrophy Type 1 or 2 who completed a previous trial.
- It aims to understand the long-term safety and effectiveness of mexiletine.
- All participants will receive the active drug for 18 months, not a placebo.
- The dose will be adjusted to suit you, taken once daily.
- Regular health checks, including heart monitoring, are a key part of participating.
- You can stop participating at any time.
Who may be eligible?
You might be able to join this study if you have Myotonic Dystrophy Type 1 or Type 2, and your diagnosis has been confirmed by genetic tests. A key requirement is that you must have already completed the previous MEX-DM-302 study.
To be considered, you'll need to be at least 16 years old. Your general health will be assessed, and doctors will check for any heart problems or other serious medical conditions that might make taking part unsafe. For example, if you have severe kidney problems or uncontrolled diabetes, you likely wouldn't be able to join.
There are also some requirements related to your physical abilities, such as having enough strength in your fingers to perform certain tests and not experiencing a lot of falls. If you are a woman who could become pregnant, you would need to use effective birth control throughout the study.
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.
- Do you have Myotonic Dystrophy Type 1 or Type 2, confirmed by genetic tests?
- Have you already completed the MEX-DM-302 study?
- Are you 16 years old or older?
- Do you have good general health, especially no serious heart problems?
- Are you able to follow study instructions and attend all appointments?
- If female, are you able to use effective birth control during the study?
What does participation involve?
If you join this study, you'll continue to take the mexiletine medication once a day, usually in the morning with food. The dose will start lower and gradually increase over the first couple of weeks to find the best amount for you, which could be up to a maximum of 500mg. Your doctors will adjust the dose if you have any side effects.
Throughout the study, which will last for an additional 18 months, you'll have regular check-ups. These will involve talking to your doctors about any side effects you might be experiencing. They will also do various tests to check your overall health, including blood tests, physical examinations, and checking your blood pressure and pulse. To specifically monitor your heart health, you'll have ECGs (heart rhythm tests), Holter monitors (small devices that record your heart rhythm over a day or two), and echocardiograms (ultrasound scans of your heart).
The study involves regular visits to the clinic for these assessments and to collect more medication. You'll need to be committed to attending all scheduled appointments until the study ends.
Potential risks and benefits
Locations (6)
- Laboratory for Muscle Diseases and NeuropathiesVerified postcodeLeuven, Belgium
- Aarhus University HospitalVerified postcodeAarhus, Denmark
- Ludug-Maximilians UniversityVerified postcodeMünchen, Germany
- Azienda Ospedaliera Universitaria Policlinico Tor VergataVerified postcodeRome, Italy
- University Hospital of MadridVerified postcodeMadrid, Spain
- University College HospitalVerified postcodeLondon, United Kingdom
Common questions
What is Myotonic Dystrophy?
Myotonic Dystrophy is a condition that causes muscles to become weak and unable to relax properly. It's a type of muscular dystrophy that can affect many parts of the body.
What is mexiletine and how does it work?
Mexiletine is a medication that helps with certain electrical signals in the body. In Myotonic Dystrophy, it's being studied to see if it can help with muscle stiffness and weakness.
Will I get a dummy pill in this study?
No, in this particular extension study, all participants will receive the active mexiletine medication, not a dummy pill (placebo).
How long will I take the medication?
If you join this extension study, you will take the medication for an additional 18 months after completing the previous study.
What health checks will be done?
You'll have regular checks including blood tests, physical exams, and tests to monitor your heart, such as ECGs and echocardiograms.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
Discussion
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