Active not recruitingOBSERVATIONAL

An Observational Study in Adult Patients With Non-dystrophic Myotonic Disorders

This study is for adults in the UK, France, and Germany who have a rare muscle condition called non-dystrophic myotonic disorder and are taking, or about to start taking, a medication called Namuscla (mexiletine). It's an 'observational' study, which means researchers will simply collect information on how patients are doing with their usual medical care. They won't ask anyone to try new treatments or change their routine. The goal is to learn more about how safe Namuscla is over the long term and how well it helps control symptoms in real-world use. Patients will be followed for up to three years, and this information will help doctors understand more about treating this condition.

At a glance

Status

Active not recruiting

Sponsor

Lupin Ltd.

Enrolment target

Start

17 Dec 2020

Estimated completion

24 Jan 2026

Myotonic Dystrophy

What is this study about?

This study is about a muscle condition called non-dystrophic myotonic disorder. This condition causes muscles to stiffen up and can make everyday movements difficult. A medicine called Namuscla (which contains mexiletine) can help manage these symptoms.

Researchers want to understand how well Namuscla works and how safe it is for people with this condition over a longer period, in real-life situations. This isn't a typical clinical trial where people receive a new or experimental drug. Instead, this is an 'observational' study. This means doctors will simply collect information about how patients are doing on Namuscla as part of their regular care. They won't ask you to do anything different or take any new medications.

The information gathered from this study will be really important. It will help doctors learn more about the long-term effects of Namuscla, understand how it helps patients manage their condition, and ensure it's being used as safely and effectively as possible. This knowledge can improve care for people with non-dystrophic myotonic disorders in the future.

Key takeaways

This study collects information on Namuscla for non-dystrophic myotonic disorders.
It's an 'observational' study, which means no changes to your treatment.
Information gathered will help improve future patient care.
You'll be followed for up to three years by your doctor.
Eligibility depends on your medical history and current medications.
Participation is entirely voluntary and you can leave at any time.

Who may be eligible?

This study is looking for adult patients who have non-dystrophic myotonic disorders. You might be eligible if you are about to start taking Namuscla, or if you are already taking Namuscla or a similar medicine called mexiletine and switch to Namuscla.

There are certain health conditions that would prevent you from joining. For example, if you have severe heart problems, a history of certain heart rhythm issues, or if you're taking specific medications that might react badly with Namuscla, you wouldn't be able to participate. Also, if you know you're allergic to mexiletine or similar medicines, you shouldn't join. Being pregnant or breastfeeding would also mean you can't 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.

Are you an adult (18 years or older)?
Do you have a non-dystrophic myotonic disorder?
Are you about to start taking Namuscla, or are you already taking Namuscla/mexiletine?
Do you have any serious heart conditions or a history of certain heart rhythm problems?
Are you allergic to mexiletine or similar medicines?
Are you currently pregnant or breastfeeding?

Answer every question to see your result.

What does participation involve?

If you decide to join this study, your involvement will be quite straightforward because it's an observational study. This means you will continue with your usual medical appointments and take Namuscla as prescribed by your doctor. The researchers will simply collect information about your health and how you're responding to the medication during these routine visits. You won't be asked to change your treatment plan or take any extra medications.

They will record details about your symptoms, any side effects you experience, and how well the treatment is working. This information will be gathered during your regular check-ups with your doctor. You will be followed for up to three years, or until you stop taking Namuscla. There are no extra visits specifically for the study beyond your normal medical care.

Potential risks and benefits

The main benefit of taking part is contributing valuable information that will help doctors and researchers understand more about how Namuscla works over time for people with non-dystrophic myotonic disorders. This can improve future care for others. Since this is an observational study and you'll be taking a medicine already approved and prescribed by your doctor, there are no additional risks beyond those associated with your standard medical care and the prescribed medication. However, as with any treatment, Namuscla can have side effects. You have the right to withdraw from the study at any time without affecting your medical care.

Locations (6)

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

Hôpital Universitaire de La Pitié Salpêtrière
Verified postcode
Paris, France
CHRU Lille
Verified postcode
Lille, France
St. Josef-Hospital Klinikum der Ruhr Universitaet Bochum
Verified postcode
Bochum, Germany
Universitätsklinikum Ulm, Klinik für Neurologie
Verified postcode
Ulm, Germany
Institute of Neurology
Verified postcode
London, United Kingdom
Nottingham University Hospitals NHS Trust
Verified postcode
Nottingham, United Kingdom

Common questions

What is an 'observational study'?

It means researchers watch and collect information about your health and treatment during your normal doctor's visits, without asking you to do anything different.

Will I have to take a new medicine?

No, you will continue to take Namuscla (mexiletine) as prescribed by your doctor for your non-dystrophic myotonic disorder, exactly as you normally would.

How long will I be in the study?

You'll be followed for up to three years, or until you stop taking Namuscla, whichever comes first.

Will I have extra doctor's appointments?

No, your participation will be part of your routine medical care. There are no additional appointments just for the study.

Can I stop participating in the study?

Yes, you can decide to stop being part of the study at any time without it affecting your medical care.