Not yet recruitingPHASE2INTERVENTIONAL

Repurposing Empagliflozin for DMD-associated Cardiomyopathy in Children 6-18 Years of Age

This study is investigating a medicine called empagliflozin for children and teenagers aged 6 to 18 who have heart problems due to Duchenne muscular dystrophy (DMD). While this medicine helps adults with heart failure and young people with type 2 diabetes, its effects on children with heart failure, especially the best dose, are not yet known. The study aims to work out the ideal dose, check how safe it is, see if it's easy to take, and understand if it can help improve heart health over 3 to 6 months. Participants will attend 5 study visits over 6 months, plus a final check-up, and take the medicine daily.

At a glance

Status

Not yet recruiting

Phase

PHASE2

Sponsor

Sebastiano Lava

Enrolment target

Start

01 Oct 2025

Estimated completion

31 Mar 2027

DMD-associated Dilated Cardiomyopathy

What is this study about?

This study is about Duchenne muscular dystrophy (DMD), a condition that unfortunately often leads to serious heart problems. These heart issues are a major concern for young people with DMD. Doctors are always looking for better ways to treat these heart conditions, as current treatments for children with heart failure, unfortunately, aren't always as effective as everyone would like.

Recently, a type of medicine, including one called empagliflozin, has shown great promise in helping adults with heart failure. It's been found to reduce time spent in hospital and prevent serious heart problems. This medicine is already used safely in teenagers with type 2 diabetes. So, researchers want to find out if empagliflozin can also help children and teenagers aged 6 to 18 who have heart problems related to DMD.

Because this medicine hasn't been widely used for this specific group before, the main goals of this study are to figure out the best dose to give, make sure it's safe, and see if it can improve heart health in the medium term (over a few months). The study will also check if the tablets are easy for young people to swallow.

Key takeaways

New study for children and teens (6-18) with DMD-related heart problems.
Investigating empagliflozin, a medicine known to help adults with heart failure.
Aims to find the best dose and check safety and effectiveness.
Requires 6 hospital visits over about 7-8 months, including one full day.
All participants will receive the active study medicine.
Your regular medical care will not be affected if you choose not to join.

Who may be eligible?

To join this study, young people must be between 6 and 18 years old and have heart problems linked to Duchenne muscular dystrophy. They should already be taking medicines for their heart condition and be considered by their hospital doctor and the study team as potentially benefiting from this new treatment.

They need to be on a stable heart medicine plan, meaning no new heart drugs started in the last two weeks, and no major changes to their existing heart drug doses. It's also important that they can have a heart MRI scan without needing to be put to sleep, and that they, or their parents/carers, can understand and agree to take part in the study.

There are some reasons why someone might not be able to join. For example, if they have certain types of diabetes, kidney problems, very low body weight (under 15kg), or if they smoke or use other nicotine products. They also can't join if they've had recent major surgery or blood transfusions, or if they have certain serious infections or uncontrolled heart rhythm issues.

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 6 and 18 years old?
Do you have heart problems caused by Duchenne muscular dystrophy?
Are you currently taking medicine for your heart condition?
Can you have a heart MRI scan without needing to be put to sleep?
Do you not have type 1 or type 2 diabetes, or serious kidney problems?
Are you able to take medicines by mouth?

Answer every question to see your result.

What does participation involve?

If you join this study, you will need to come to the hospital for 5 study visits over 6 months, plus one final check-up visit between 2 and 12 weeks after the 6 months are up. The first visit will be a full day (about 8 hours) in the hospital. The other visits will be shorter outpatient appointments, lasting about 2 hours each. Throughout the 6 months, you will take the study medicine, empagliflozin, once every day.

During the study, the team will check how the medicine moves through the body (this helps find the best dose) and will regularly monitor your safety. They will also ask about how easy it is to swallow the tablets. You won't be compared to another group of people not taking the medicine in this particular study, as the main focus is on finding the right dose and checking safety.

Potential risks and benefits

Taking part in a study like this might offer potential benefits, such as receiving a new medicine that could help improve heart health related to DMD, though this is not guaranteed. You will also receive very close medical supervision. As with any medicine, there could be potential risks or side effects, and the study team will monitor you carefully for these. The study aims to understand these risks better in young people. It's important to remember that joining is completely voluntary, and you have the right to withdraw from the study at any time without affecting your usual medical care.

Locations (1)

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

Great Ormond Street Hospital NHS Foundation Trust
Verified postcode
London, United Kingdom

Common questions

What is Duchenne muscular dystrophy (DMD)?

Duchenne muscular dystrophy is a genetic condition that causes muscles to weaken over time. It can also affect the heart.

What is empagliflozin?

Empagliflozin is a medicine that helps the body get rid of sugar through the urine. It has been found to help adults with heart failure and young people with type 2 diabetes.

What is 'heart failure'?

Heart failure means the heart isn't pumping blood as well as it should. It can lead to symptoms like tiredness and shortness of breath.

Will I get the actual medicine or a dummy pill?

Everyone in this study will receive the active medicine, empagliflozin. There is no 'comparison group' getting a dummy pill in this particular study.

How long will I take the medicine for?

You will take the study medicine once daily for 6 months.

How to find out more

Official trial record

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