Clinical Trial in Patients With Barth Syndrome- 4TAZPower
This study, called 4TAZPower, is for boys and men aged 5 to 55 with Barth Syndrome. We're testing a medicine called elamipretide to see if it makes muscles stronger and if it's safe. Participants will get either the study medicine or a dummy medicine (placebo) for 72 weeks, given as a shot under the skin each day. The study is 'double-blind,' meaning neither you nor your doctor will know if you're getting the actual medicine or the placebo. Elamipretide is already approved in the United States to help improve muscle strength in people with Barth Syndrome, and this study aims to confirm those benefits.
At a glance
What is this study about?
Barth Syndrome is a serious condition that mainly affects boys and men. It can cause heart problems, weak muscles, and tiredness. This study is testing a medicine called elamipretide, which is designed to help the cells in your body work better, particularly those in your muscles. The hope is that this medicine will help improve muscle strength for people with Barth Syndrome.
This study will last for 72 weeks, which is about a year and a half. During this time, participants will receive a daily injection just under the skin. Some participants will receive elamipretide, while others will receive a placebo – a dummy medicine that looks just like the real thing but has no active ingredients. This is a common and important way to test new medicines, as it helps researchers see if the real medicine is truly making a difference. Neither you nor your study doctor will know whether you are receiving the real medicine or the placebo.
The medicine, elamipretide, is already available in the United States under the name FORZINITY™. It was approved there because earlier studies showed it helped improve knee muscle strength. This new study aims to gather more information and confirm these benefits in a larger group of people, making sure it's a good and safe option for more individuals with Barth Syndrome.
Key takeaways
- The study is for males aged 5 to 55 with Barth Syndrome.
- It tests a daily injection of medicine called elamipretide or a placebo.
- The main goal is to see if elamipretide improves muscle strength and is safe.
- The study lasts about 72 weeks (1.5 years).
- Elamipretide is already approved in the US for Barth Syndrome.
Who may be eligible?
To join this study, participants need to be male, between 5 and 55 years old, and have a confirmed diagnosis of Barth Syndrome. It's important that your heart is working reasonably well, and if you're taking heart medication, it should have been stable for at least three months.
There are also some reasons why you might not be able to join. For example, if you've had an organ transplant (unless it was more than a year ago for your heart and you're stable), if you've been in hospital for heart failure recently, or if you're on a waiting list for a heart transplant. Also, you need to be able to do some simple physical tests for the study.
- Are you male and between 5 and 55 years old?
- Do you have a genetically confirmed diagnosis of Barth Syndrome?
- Is your heart function (ejection fraction) at 50% or better?
- If you take heart medicine, has the dose been stable for at least 3 months?
- Are you able to do some simple physical tests?
- Have you not been in hospital for heart failure in the last 6 months?
This is a guide only — the research team will confirm whether you can take part.
What does participation involve?
If you decide to take part, you'll be in the study for about 72 weeks (around 1 year and 5 months). During this time, you'll need to visit the study clinic regularly. At these visits, doctors will check your general health, measure your muscle strength (especially in your knees), and do tests like echocardiograms (heart scans) and blood tests.
Each day, you'll give yourself an injection under the skin, or someone who can help you will do it. This will be either the study medicine (elamipretide) or a dummy medicine (placebo). You won't know which one you're getting. The study team will teach you or your helper exactly how to give these injections safely. They'll also monitor you closely for any side effects or changes in your health throughout the study.
Potential risks and benefits
Locations (2)
- Trial Not Offered in the U.SNeedham, United States
- Bristol Royal Hospital for Children Upper Maudlin Street Paul O'Gorman BuildingBristol, United Kingdom
Common questions
What is Barth Syndrome?
Barth Syndrome is a rare genetic condition that mainly affects boys. It can cause heart problems, weak muscles, and make people feel very tired.
What is elamipretide?
Elamipretide is a medicine being tested in this study. It's designed to help the tiny powerhouses within your cells work better, aiming to improve muscle strength.
What is a 'placebo'?
A placebo is a dummy medicine that looks exactly like the real medicine but contains no active ingredients. It helps researchers see if the real medicine is truly working.
How long will the study last?
The study will last for 72 weeks, which is about a year and a half.
Can I stop being in the study if I change my mind?
Yes, you can leave the study at any time, for any reason, without it affecting your regular medical care.
How to find out more
Rekha Sathyanarayana
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
Discussion
Community discussion
Powered by our forum at community.patient.info. Please be respectful — this is not medical advice.