A Long-term Follow-up Study of Participants Who Received Delandistrogene Moxeparvovec (SRP-9001) in a Previous Clinical Study
This ongoing study is looking at the long-term health of individuals who have previously received a special treatment called delandistrogene moxeparvovec for Duchenne muscular dystrophy. The main goal is to carefully monitor how safe and effective this treatment has been for them over a longer period. Researchers want to collect information in a consistent way from everyone involved. It's important to know that participants in this study will not receive any new medication or treatment as part of it. Instead, the study focuses on observing their health and progress, providing valuable insights into the lasting effects of delandistrogene moxeparvovec. This helps doctors and scientists understand more about Duchenne muscular dystrophy and its potential treatments.
At a glance
What is this study about?
This study is designed to keep a close eye on people who have already received a treatment called delandistrogene moxeparvovec for Duchenne muscular dystrophy. You might have participated in an earlier study where you received this treatment. The main purpose now is to understand how you are doing over a longer period, focusing on both your safety and how well the treatment has worked for you in the long run.
Duchenne muscular dystrophy is a condition that causes muscles to become weaker over time. Treatments like delandistrogene moxeparvovec are designed to help with this. By following up with those who have received it, doctors hope to learn more about its lasting effects and how it impacts quality of life in the years following the initial treatment.
It's important to know that during this particular study, you will not be given any new medication or treatment. This study is all about observation and gathering information. By participating, you’re helping researchers collect vital information that can improve understanding and future treatments for Duchenne muscular dystrophy, benefitting others with the condition.
Key takeaways
- This study observes individuals who previously received delandistrogene moxeparvovec for Duchenne.
- No new medication or treatment will be given in this study.
- The main aim is to understand the long-term safety and effectiveness of the previous treatment.
- Participation helps researchers learn more about Duchenne muscular dystrophy and its treatments.
- It requires regular check-ups and monitoring of your health over time.
- You can stop participating at any time if you wish.
Who may be eligible?
This study is specifically for males who have Duchenne muscular dystrophy and have already received the treatment called delandistrogene moxeparvovec in a previous research study. You cannot join this study if you haven't had this treatment before.
To take part, if you are under 18, your parent or legal guardian must be able to understand and help you with all the study appointments and instructions. If you are 18 or older, you must be able to understand these things yourself and be happy to follow the study plan.
- Are you male?
- Do you have Duchenne muscular dystrophy?
- Have you previously received the treatment called delandistrogene moxeparvovec in another study?
- If under 18, can your parent/guardian understand and help with study requirements?
- If 18 or older, can you understand and agree to the study requirements?
This is a guide only — the research team will confirm whether you can take part.
What does participation involve?
If you decide to join this study, your involvement will focus on regular check-ups and assessments. You won't receive any new medication. Instead, the study team will monitor your health and how you’re doing over time. This might involve physical examinations, discussions about your health, and possibly some tests. The specific details of what each visit involves and how often you'll need to come in will be explained to you by the study team. The total duration of your participation will depend on the study plan, which aims to follow people for a long time to understand the lasting effects of the treatment you previously received.
Potential risks and benefits
Locations (38)
- Arkansas Children's HospitalLittle Rock, United States
- UCLA Medical CenterLos Angeles, United States
- Lucile Packard Children's Hospital Stanford (LPCH)Palo Alto, United States
- University of California, DavisSacramento, United States
- Rady Children's HospitalSan Diego, United States
- Children's Hospital of ColoradoAurora, United States
- University of Florida Clinical Research CenterGainesville, United States
- Ann & Robert H. Lurie Children's Hospital of ChicagoChicago, United States
- University of Iowa ICTS Clinical Research UnitIowa City, United States
- The Johns Hopkins Hospital, Rubenstein Child Health BldgBaltimore, United States
- Boston Children's HospitalBoston, United States
- Washington University School of MedicineSt Louis, United States
+26 more sites — see the official record for the full list.
Common questions
Will I receive new treatment in this study?
No, this study is only for observation. You will not receive any new medication or treatment as part of it.
Who can join this study?
This study is for males with Duchenne muscular dystrophy who have already received delandistrogene moxeparvovec in a previous study.
What is the main goal of this study?
The main goal is to monitor the long-term safety and how well delandistrogene moxeparvovec has worked for people who received it in an earlier study.
How long will I need to be in the study?
The study aims to follow participants for a long time. The exact duration will be discussed with you by the study team.
Will this study affect my usual medical care?
No, this study is designed to gather information and should not interfere with your regular medical care for Duchenne muscular dystrophy.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
Discussion
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