A Phase 3, Multi-Center, Open Label Extension Study to Assess the Safety and Efficacy of Viltolarsen in Ambulant Boys with Duchenne Muscular Dystrophy (DMD)
This research is an ongoing study for boys with Duchenne Muscular Dystrophy (DMD) who are still able to walk. It's an extension of previous studies looking at a medicine called Viltolarsen. The main goal is to keep checking if Viltolarsen is safe to use over a longer period and if it continues to help boys with DMD. Researchers will closely monitor participants' health, including their heart, kidneys, and overall well-being. They will also assess how the boys' muscle strength and movement skills, like walking, running, and climbing stairs, change over time. This is a "Phase 3" study, which means it’s a big step towards understanding if Viltolarsen could become a widely available treatment.
At a glance
What is this study about?
Duchenne Muscular Dystrophy (DMD) is a genetic condition that causes muscles to weaken over time. This study is an important step in understanding a new medicine called Viltolarsen. It’s a continuation for boys who have already taken part in earlier studies with this medicine and are still able to walk. The main aim is to see how safe Viltolarsen is with long-term use and if it continues to help with muscle function in these boys.
Researchers will be carefully tracking many aspects of the boys' health. This includes regular check-ups like measuring their heart rate and blood pressure, taking blood and urine samples to check their general health, and doing heart scans (ECGs) and kidney scans (renal ultrasounds). They will also be looking out for any side effects from the medication.
Beyond safety, the study will also measure how well the boys are moving. This involves tests like how quickly they can stand up, run or walk a short distance, and climb stairs. They will also do a ‘Six-minute Walk Test’ to see how far they can walk in six minutes, and use special equipment to measure their muscle strength. These measurements help the researchers understand if Viltolarsen is making a difference to their muscle function and daily activities.
Key takeaways
- This study is for boys with Duchenne Muscular Dystrophy (DMD) who can still walk.
- It's an extension of previous studies looking at a medicine called Viltolarsen.
- The main goals are to check the long-term safety and effectiveness of Viltolarsen.
- Participants will have regular health checks, including physical exams, blood tests, heart scans, and kidney scans.
- Movement and muscle strength will be carefully monitored with specific tests.
- Participation involves regular clinic visits and receiving the study medication.
Who may be eligible?
This study is looking for participants who are boys diagnosed with Duchenne Muscular Dystrophy (DMD). An important requirement is that they must still be able to walk by themselves.
There are no specific age limits mentioned for this particular extension study, meaning it's open to boys of various ages who meet the other criteria. The study welcomes boys who have previously participated in earlier clinical trials for Viltolarsen.
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.
- Is the person in question a boy?
- Does he have a diagnosis of Duchenne Muscular Dystrophy (DMD)?
- Is he currently able to walk by himself?
- Has he previously participated in a Viltolarsen study?
What does participation involve?
If you or your child decides to take part in this study, it involves regular visits to the study clinic. During these visits, the medical team will carry out several assessments. These include checks of vital signs (like heart rate and blood pressure), physical examinations, and blood and urine tests. They will also do heart scans (ECGs) and kidney ultrasounds. You would also receive the study medication, Viltolarsen.
Additionally, there will be assessments of muscle strength and function, such as timed tests for standing up, walking, running, and climbing stairs, as well as a six-minute walk test. The medical team will also closely monitor for any side effects or health changes throughout the study. The total duration of participation will depend on the study plan and your child's individual progress, as this is an open-ended extension study.
Potential risks and benefits
Locations (6)
- —UnverifiedSpain
- —UnverifiedItaly
- —UnverifiedGreece
- —UnverifiedNorway
- —UnverifiedCzechia
- —UnverifiedNetherlands
Common questions
What is Duchenne Muscular Dystrophy (DMD)?
DMD is a genetic condition that causes muscles to become weak and waste away over time.
What is Viltolarsen?
Viltolarsen is a medicine being studied as a potential treatment for Duchenne Muscular Dystrophy.
What does 'ambulant' mean in this study?
It means the study is for boys who are still able to walk by themselves.
Why is this called an 'open-label extension study'?
This means participants and doctors know they are receiving Viltolarsen, and it's a way for participants who were in earlier studies to continue treatment.
Will my child definitely feel better on this medicine?
There's no guarantee that Viltolarsen will work for everyone, and it might not provide a direct benefit to your child. The study aims to gather more information.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
Discussion
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