A multicentre, randomised, double-blind, placebo-controlled and open label extension study to assess the efficacy, safety, and pharmacokinetic profile of two dose levels of ATL1102 administered by subcutaneous injection in nonambulatory participants with Duchenne Muscular Dystrophy
This research study is testing a new medicine called ATL1102 for boys living with Duchenne Muscular Dystrophy (DMD) who can no longer walk. The main goal is to find out if ATL1102 can help improve the use of their arms and hands, and how safe it is to use. Participants will be given either ATL1102 or a placebo (a dummy medicine) through injections under the skin. Initially, no one will know who is getting which. After a period, all participants will have the chance to receive ATL1102 in an 'open-label' part of the study. Researchers will carefully check muscle strength, how well arms and hands work, and monitor for any side effects.
At a glance
What is this study about?
This study is a medical investigation into a new potential treatment called ATL1102 for Duchenne Muscular Dystrophy (DMD). DMD is a serious condition that causes muscles to weaken over time. This particular study is focusing on boys with DMD who are no longer able to walk. The medicine, ATL1102, is designed to be given as an injection just under the skin.
The main purpose of this research is to see if ATL1102 can help improve how well the participants can use their arms and hands. This is measured using a special system called the Performance of Upper Limb (PUL 2.0) score. The researchers also want to thoroughly check how safe the medicine is by carefully looking for any side effects or changes in health.
This study is divided into different stages. In the first part, some participants will receive ATL1102, while others will receive a placebo – a substance that looks exactly like the medicine but contains no active drug. Neither the participants, their families, nor the study doctors will know who is getting which. This helps make sure the results are fair. After this first stage, everyone will have the chance to receive the ATL1102 medicine in an 'open-label' part of the study, where everyone knows what treatment they are getting. This type of study helps scientists understand if new medicines are both helpful and safe.
Key takeaways
- This study investigates a new medicine (ATL1102) for boys with Duchenne Muscular Dystrophy who can't walk.
- The main goals are to check if the medicine helps improve arm and hand function, and if it's safe.
- Participants will receive either ATL1102 or a placebo by injection in the first phase.
- Everyone will eventually have the chance to receive the active medicine.
- Regular clinic visits will involve assessments of muscle function, heart, breathing, and blood tests.
- Your safety will be closely monitored throughout the study.
Who may be eligible?
This study is specifically looking for boys who have Duchenne Muscular Dystrophy.
To be considered for this study, participants must no longer be able to walk. There are no specific age limits mentioned, meaning boys of varying ages who meet the other criteria might be able to take part.
It's important to remember that this is just a quick overview. If you or someone you know might be interested, a doctor involved in the study would need to check all the detailed requirements to see if it's a suitable fit. They will make sure it's safe and appropriate for everyone who joins.
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.
- Do you have a diagnosis of Duchenne Muscular Dystrophy?
- Are you a male?
- Are you no longer able to walk?
- Are you willing to attend regular clinic appointments for over 11 months?
- Are you comfortable with receiving injections under the skin?
What does participation involve?
If you decide to take part in this study, you would receive the study medicine (or placebo initially) as an injection under the skin. These injections would be given regularly over many weeks.
Throughout the study, you would have regular visits to the clinic. At these visits, doctors and nurses would perform various checks. This includes tests to see how well you can use your arms and hands, and to measure your muscle strength. They would also take blood and urine samples, check your heart with tests like an ECG (a heart tracing) and an echocardiogram (an ultrasound of the heart), check your breathing, and perform general physical examinations.
Your vital signs, such as blood pressure, heart rate, and temperature, would also be regularly monitored. The study will run for about 49 weeks (around 11-12 months) in total, with the first 25 weeks being the 'blinded' part and the remaining weeks being the 'open-label' part. Throughout, your safety will be closely watched.
Potential risks and benefits
Locations (1)
- —UnverifiedBulgaria
Common questions
What is Duchenne Muscular Dystrophy?
Duchenne Muscular Dystrophy (DMD) is a genetic condition that causes muscles to become weaker over time.
What is ATL1102?
ATL1102 is a new medicine being tested as a potential treatment for Duchenne Muscular Dystrophy.
What does a 'placebo' mean?
A placebo is a dummy medicine that looks just like the real medicine but contains no active drug. It helps researchers compare results fairly.
Will I know if I'm getting the real medicine?
Initially, no one (you, your family, or the study doctors) will know. Later in the study, everyone will receive the active medicine.
How is the medicine given?
The medicine is given by an injection just under the skin (subcutaneous injection).
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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