A Phase 3, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Investigate the Efficacy of a Single Intravenous Dose of SGT-003 in Ambulant Males With Duchenne Muscular Dystrophy
This research is a study looking into a new treatment, SGT-003, for Duchenne muscular dystrophy. It's for boys with Duchenne who are still able to walk. The main goal is to check if a single dose of SGT-003, given directly into a vein, helps them get up from the floor more quickly after about a year and a half (540 days). The study will compare SGT-003 to a dummy treatment (placebo) to see if it makes a real difference. Researchers will also look at other things like how fast they walk, climb stairs, and how their muscles and breathing change, as well as checking for any side effects.
At a glance
What is this study about?
Duchenne muscular dystrophy is a serious condition that causes muscles to weaken over time. This study is testing an experimental treatment, SGT-003, for boys who have Duchenne muscular dystrophy and are still able to walk. The researchers want to find out if giving a single dose of SGT-003 can help improve how well these boys can move, particularly how quickly they can stand up from the floor.
This is a 'Phase 3' study, which means it's one of the final steps in testing a new medicine before it might be considered for wider use. To make sure the results are fair and accurate, some participants will receive the actual treatment (SGT-003), while others will receive a 'dummy' treatment called a placebo, which looks just like the real medicine but contains only salt water. Neither the participants, their families, nor the study doctors will know who is getting which treatment until the study is over. This is called 'double-blind' and helps prevent any bias in the results.
Over the course of about a year and a half, the study will carefully measure changes in movement, like walking speed and stair climbing ability. They will also look at how strong their breathing muscles are and check for any changes in the Duchenne muscular dystrophy proteins in the body. Importantly, the study will also keep a close eye on safety, checking for any side effects or unwanted reactions to the treatment.
Key takeaways
- Tests a new treatment (SGT-003) for Duchenne muscular dystrophy.
- For boys with Duchenne who can still walk.
- Compares SGT-003 to a dummy treatment (placebo).
- Focuses on how movement improves and safety over 18 months.
- A single dose of treatment is given by IV drip.
- Participation is voluntary, and you can withdraw at any time.
Who may be eligible?
This study is specifically looking for boys who have Duchenne muscular dystrophy.
To take part, boys must still be able to walk by themselves. There are no limits on age for this study, meaning boys of many different ages might be able to participate.
If you are interested, your doctor will be able to check if all the specific requirements are met to make sure this study is the right fit.
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 a boy with Duchenne muscular dystrophy?
- Are you still able to walk by yourself, without major help from others?
- Are you able to attend regular appointments for about 18 months?
- Are you willing for doctors to monitor your health closely during the study?
What does participation involve?
If you decide to take part, you would receive a single treatment through an IV (a drip in your arm). You would then have regular check-ups at the study centre over about 18 months (540 days) to see how you're doing. These visits will involve various tests to measure your movement, such as how quickly you can get up from the floor, walk, and climb stairs. You might also wear a special device that tracks your activity. Doctors will also check your breathing, take blood samples, and perform heart tests (like ECGs and ECHOs) to monitor your health and safety. The study team will carefully record any changes in your health or any side effects you experience throughout this period. The full study duration is about a year and a half.
Potential risks and benefits
Locations (6)
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Common questions
What is Duchenne muscular dystrophy?
Duchenne muscular dystrophy is a genetic condition that causes muscles to become weaker and waste away over time.
What does 'Phase 3' mean for a study?
Phase 3 means this is one of the final stages of testing a new medicine, typically involving many people, to confirm its effectiveness and safety.
What is a 'placebo'?
A placebo is a 'dummy' treatment that looks just like the study medicine but contains no active ingredients. It's used for comparison.
How is the treatment given?
The treatment, SGT-003, is given as a single dose directly into a vein through a drip (intravenously).
How long will the study last if I take part?
If you participate, the study will involve regular check-ups over about 18 months (540 days).
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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