Ongoing, recruitingTherapeutic confirmatory (Phase III)Interventional

The efficacy and safety of Metoprolol as add-on treatment to standard of care in preventing cardiomyopathy in patients with Duchenne Muscular Dystrophy aged 8-17 years. A randomized, double-blind, placebo controlled study

This medical study is trying to find out if a common heart medicine, Metoprolol, can protect the hearts of young people aged 8-17 who have Duchenne Muscular Dystrophy. Duchenne can sometimes weaken the heart muscle, a condition called cardiomyopathy. Researchers want to see if adding Metoprolol to their usual care can slow down or stop this heart weakening. Participants will either receive Metoprolol or a placebo (a dummy pill) without knowing which one they are getting. The main goal is to measure how well the heart pumps blood and if Metoprolol helps keep it strong over time, hopefully preventing serious heart problems.

At a glance

Status

Ongoing, recruiting

Phase

Therapeutic confirmatory (Phase III)

Sponsor

Medical University Of Gdansk

Enrolment target

144

Start

16 Dec 2024

Duchenne muscular dystrophy Cardiomyopathy Tachycardia

What is this study about?

This study is about a condition called Duchenne Muscular Dystrophy (DMD), which is a serious muscle-wasting illness. One of the ways DMD can affect people is by weakening the heart muscle over time. This heart problem is called cardiomyopathy, and it can lead to other serious health issues.

The researchers in this study want to see if an existing medicine called Metoprolol can help protect the hearts of young people with DMD. Metoprolol is a type of medicine known as a beta-blocker, often used to treat heart conditions. The idea is to give Metoprolol alongside the usual care these young people receive, to see if it can prevent or slow down the development of cardiomyopathy.

To make sure the results are fair and accurate, this study is 'randomised' (meaning participants are put into groups by chance), 'double-blind' (meaning neither the participants nor the doctors know who is getting the real medicine and who is getting a dummy pill), and 'placebo-controlled' (meaning one group gets the real medicine and another gets a dummy pill). This allows researchers to clearly see if Metoprolol is really making a difference compared to just receiving standard care.

Key takeaways

A study for young people with Duchenne Muscular Dystrophy (DMD).
Investigating if Metoprolol can prevent heart problems (cardiomyopathy).
Compares Metoprolol to a dummy pill (placebo).
Participants aged 8-17 years old.
Regular heart checks (echocardiograms) will be part of the study.
Aims to find new ways to improve heart health for people with DMD.

Who may be eligible?

This study is looking for young people with Duchenne Muscular Dystrophy between 8 and 17 years old. Both boys and girls can take part.

There will be other specific health requirements that your doctor will check to make sure the study is a good fit for you. For example, they might need to check your heart health and other medical conditions to ensure it's safe for you to take Metoprolol.

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 between 8 and 17 years old?
Do you have a diagnosis of Duchenne Muscular Dystrophy?
Are you able to take a tablet regularly?
Are you comfortable with regular hospital or clinic visits?
Are you able to have heart scans (echocardiograms)?

Answer every question to see your result.

What does participation involve?

If you decide to take part, you would be given either Metoprolol or a placebo (a dummy pill that looks the same but has no medicine in it) to take regularly. You, your family, and your doctors wouldn't know which one you're receiving.

Throughout the study, you would have regular visits to your doctor or clinic. These visits would include check-ups and special heart scans, such as an echocardiogram (a type of ultrasound for your heart). These scans help the doctors measure how well your heart is working. Your doctors will also monitor your overall health. Your participation in the study would continue for a period, with checks at certain points, like an 'interim analysis' and a 'final analysis', to see how the treatment is working. The total duration of the study and how many visits are needed would be explained in detail by the study team.

Potential risks and benefits

Taking part in this study could potentially offer a benefit if Metoprolol proves to help protect your heart, but there's no guarantee. You might also gain a better understanding of your heart health through the regular check-ups. However, like all medicines, Metoprolol can have side effects, and some people might experience them. The study team will explain all known side effects. There is also a small risk involved with any medical procedure, like the echocardiograms. You are always free to withdraw from the study at any time, for any reason, without it affecting your medical care.

Locations (1)

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

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Unverified
Poland

Common questions

What is Duchenne Muscular Dystrophy?

It's a serious condition that causes muscles to become weaker over time, including the heart muscle.

What is cardiomyopathy?

This is when the heart muscle becomes weak and struggles to pump blood effectively around the body.

What is Metoprolol?

It's a common medicine often used to treat heart conditions, like high blood pressure or an irregular heartbeat.

What is a 'placebo'?

A placebo is a dummy pill that looks like the real medicine but contains no active ingredients. It helps researchers compare the effects of the actual drug.

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

No, in this 'double-blind' study, neither you, your family, nor your doctors will know if you're receiving Metoprolol or the placebo until the study is over.