Repurposing empagliflozin for Duchenne muscular dystrophy-associated cardiomyopathy in children 6-18 years of age
This study is exploring a new way to treat heart problems in children and young people, aged 6 to 18, who have Duchenne muscular dystrophy (DMD). Heart issues are a big concern for people with DMD, and current treatments aren't always enough. The research is testing a medicine called empagliflozin, which has helped adults with heart failure. We want to find out the best dose for children, check how safe it is, and see if it can improve their heart health. This could lead to a new treatment option for a serious complication of Duchenne muscular dystrophy.
At a glance
What is this study about?
Heart problems are a very common and serious concern for children and young people with Duchenne muscular dystrophy (DMD). Sadly, they can be a major cause of health issues and even death. Doctors are always looking for better ways to protect and treat the heart in children with DMD, especially because current treatments don't always work as well as everyone hopes.
This study is looking at a medicine called empagliflozin. This medicine has already shown great promise in helping adults with heart failure by reducing serious problems like hospital stays or even death. Researchers believe it might also be helpful for children and teenagers with DMD who have heart problems. Instead of developing a brand new medicine, this study is 'repurposing' an existing one, meaning we're exploring if it can be used for a different condition.
The main goal of this study is to find the best and safest dose of empagliflozin for children and young people aged 6 to 18 with DMD-related heart problems. We also want to see if the medicine is easy to take and if it shows early signs of helping the heart. The information gathered will be really important for doctors treating children with DMD and for planning more in-depth studies in the future.
Key takeaways
- Exploring a new heart treatment for children (6-18) with Duchenne muscular dystrophy.
- Testing an existing medicine, empagliflozin, which helps adult heart failure.
- Aims to find the best dose and check safety and early signs of benefit.
- Study involves 6 months of medicine and 6 hospital visits.
- Potential side effects include low blood sugar or infections, carefully monitored.
- Results will help doctors and guide future research.
Who may be eligible?
This study is looking for children and young people between 6 and 18 years old who have Duchenne muscular dystrophy and are also being treated for heart problems related to it. You would need to be already taking other heart medicines, and your doctor would think that this new medicine might be helpful for you.
To join, you or your parents would need to be able to understand the study and agree to take part. You would also need to be able to have an MRI scan of your heart without needing to be put to sleep.
Some reasons why someone might not be able to join include having diabetes (type 1 or type 2), or other conditions that cause very low blood sugar. Also, children weighing less than 15kg (about 2 stone, 5 pounds) cannot take part, as the current medicine forms might be too strong for them.
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 Duchenne muscular dystrophy with heart problems?
- Are you currently taking other heart medicines?
- Can you have an MRI scan without sedation?
- Do you (or your parents) understand and agree to take part?
- Do you currently have diabetes (Type 1 or 2)? (If yes, you can't join.)
What does participation involve?
If you join this study, you will take the empagliflozin medicine every day for 6 months. You will have a total of 6 visits. The first visit will be a full day (about 8 hours) at the special clinic at Great Ormond Street Hospital. During this visit, we'll check how easy it is to swallow the medicine and do tests like a physical check-up, blood tests, heart scans (echocardiogram and MRI).
The other 4 visits will be spread out over the 6 months (after 1 week, 6 weeks, 3 months, and 6 months) and will be like regular heart check-ups, but with some extra tests including blood tests. There will also be a final visit 2 to 12 weeks after you finish taking the medicine to see how you are doing.
Potential risks and benefits
Locations (1)
- Great Ormond Street HospitalApproximateLondon, England
Common questions
What is Duchenne muscular dystrophy (DMD)?
DMD is a genetic condition that causes muscles to become weaker over time, including the heart muscle.
What is empagliflozin?
It is a medicine that has been shown to help adults with heart failure, and we want to see if it can help children with DMD-related heart problems too.
Who is funding this study?
The study is being funded by Duchenne UK, a charity dedicated to fighting Duchenne muscular dystrophy.
Will I have to stay in the hospital?
The first visit involves a one-day stay of about 8 hours at the hospital clinic, but follow-up visits are shorter, like regular clinic appointments.
How long will the study last for me?
You would take the medicine for 6 months, with regular visits during that time, and then a final check-up 2-12 weeks after you stop the medicine.
How to find out more
GOSH R&D CTIMPs Team
Always speak to your GP or specialist before deciding to take part in a study.
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