RecruitingPHASE3INTERVENTIONAL

The Efficacy and Safety of Once Daily Mexiletine PR in Patients With Myotonic Dystrophy Type 1 and Type 2

This research study is looking into a new, once-a-day version of a medicine called mexiletine for people who have Myotonic Dystrophy, both Type 1 and Type 2. The main goal is to find out if this medicine is both effective at helping with the muscle problems caused by the condition and if it's safe to use. Half the participants will receive the active medicine, while the other half will receive a dummy pill (placebo). Neither you nor your doctor will know which you are taking. The study will last for 26 weeks, with several visits to the clinic to check your progress and overall health. We hope this study will lead to better treatments for Myotonic Dystrophy.

At a glance

Status

Recruiting

Phase

PHASE3

Sponsor

Lupin Ltd.

Enrolment target

176

Start

13 Feb 2025

Estimated completion

22 Apr 2026

Myotonic Dystrophy

What is this study about?

Myotonic Dystrophy is a condition that affects your muscles, making it hard for them to relax after you've used them, like when you grip something tightly. This study is testing a new version of a medicine called mexiletine. This new version is designed to be taken once a day, which could make it easier for people to manage.

The study aims to see if this new mexiletine helps to improve the muscle stiffness and difficulty relaxing experienced by people with Myotonic Dystrophy Type 1 and Type 2. We also want to make sure it's safe and doesn't cause too many side effects. By comparing it to a dummy pill (placebo), researchers can get a clear picture of how well the actual medicine works.

This kind of study is called a 'Phase 3' trial, which means it's a larger study that follows earlier successful research. The results from studies like this are very important because they help doctors understand if new treatments are good enough to be made available to everyone who needs them.

Key takeaways

This study is testing a new, once-a-day mexiletine for Myotonic Dystrophy.
It aims to see if the medicine improves muscle stiffness and is safe.
The study lasts for 26 weeks, with regular clinic visits.
You might receive the active medicine or a dummy pill (placebo).
Eligibility is based on genetic diagnosis, age (16+), and specific health criteria.
You can stop participating at any time.

Who may be eligible?

To be considered for this study, you need to have a confirmed diagnosis of Myotonic Dystrophy (either Type 1 or Type 2) that has been checked with a genetic test. You must be at least 16 years old. If you're a woman who could have children, you'll need to use very effective contraception throughout the study and for a short time afterwards. Your overall health is important, and doctors will check that you don't have certain serious heart, kidney, or other medical conditions that could make taking part unsafe.

You also need to be able to understand what the study involves and be willing to stay in it for the full 26 weeks. Because the study looks at muscle strength and relaxation, you'll need to be able to grip things well enough for measurements, and have some 'handgrip myotonia' (slow muscle relaxation) that doctors can measure. You should also be able to walk 10 metres on your own, even if you use a walking aid.

There are also some reasons why you wouldn't be able to join. For example, if you are pregnant or breastfeeding, have certain uncontrolled medical conditions like diabetes, severe kidney problems, or other serious illnesses that could interfere with the study or muscle function. If you have severe arthritis or other hand problems not related to Myotonic Dystrophy, you might also not be able to take part.

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 a genetically confirmed diagnosis of Myotonic Dystrophy Type 1 or Type 2?
Are you 16 years of age or older?
Can you walk 10 metres, even with an aid like a cane?
Have you been told you have 'myotonia' (slow muscle relaxation) in your hand grip?
Are you a woman of childbearing potential willing to use reliable contraception?
Do you have any serious heart or kidney problems, or certain other severe medical conditions?

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, it will involve a few steps over 26 weeks. First, there's a 'screening' period of about 4 weeks where doctors will check if the study is right for you. This will include health checks, blood tests, and assessments of your muscle strength.

Once you’re in the study, you’ll either receive the new mexiletine medicine or a dummy pill (placebo) in the form of granules (small grains) to mix into a drink, once a day. You won't know which one you're taking, and neither will your study doctor – this is to make sure the results are as fair as possible.

You'll have several visits to the clinic during the 26-week treatment period to see how you're getting on. These visits will be at the start (called 'baseline'), then at Weeks 1, 2, 14, and finally at Week 26. At these visits, doctors will check your general health, measure your muscle function, and ask about any side effects you might be experiencing. The overall duration from your initial check to your final visit will be about 30 weeks.

Potential risks and benefits

Taking part in this study could offer some potential benefits. You would receive regular medical check-ups and monitoring by specialists. If you receive the active medicine, it might help improve your muscle symptoms. However, there's no guarantee the medicine will help you personally, and you might receive a dummy pill (placebo). Like all medicines, mexiletine can have side effects, which the study staff will carefully explain to you. These might include upset stomach, dizziness, or other issues. You are free to withdraw from the study at any time and for any reason, without it affecting your usual medical care.

Locations (7)

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

Laboratory for Muscle Diseases and Neuropathies
Verified postcode
Leuven, Belgium· Recruiting
Aarhus University Hospital
Verified postcode
Aarhus, Denmark· Recruiting
Ludug-Maximilians University
Verified postcode
München, Germany· Recruiting
Azienda Ospedaliera Universitaria Policlinico Tor Vergata
Verified postcode
Rome, Italy· Recruiting
University Hospital of Madrid
Verified postcode
Madrid, Spain· Recruiting
Saint George's University Hospitals NHS Foundation Trust
Verified postcode
London, United Kingdom· Recruiting
University College Hospital
Verified postcode
London, United Kingdom· Recruiting

Common questions

What is Myotonic Dystrophy?

Myotonic Dystrophy is a condition that affects your muscles, making them slow to relax after you've used them, for example, after a strong grip. It can also cause other symptoms in different parts of your body.

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 drug. It's used in studies to see if the medicine works better than no treatment at all.

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

No, this is a 'blinded' study. Neither you nor your study doctor will know whether you are receiving mexiletine or the placebo. This helps ensure the study results are fair and accurate.

How long will I need to be in the study?

The study involves a 4-week screening period, followed by a 26-week treatment period. So, in total, you would be involved for about 30 weeks.

What happens after the study ends?

Once the study treatment period is over, your participation in the trial ends. Your study doctor will discuss your further care options with you.

How to find out more

Nikki Adetoro

nikkiadetoro@lupin.com 1 443-447-4534 Official trial record

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