A Phase 1/2, Multicenter, Open-label, Dose Escalation and Expansion Clinical Study to Evaluate the Safety, Tolerability and Preliminary Efficacy of ASP2957 in Male Participants with Invasive Ventilator-dependent X-linked Myotubular Myopathy
This research study is looking into a new treatment called ASP2957 for boys and men who have a condition called X-linked myotubular myopathy. This is a genetic muscle disease that often causes severe muscle weakness, including breathing muscles, meaning many patients need help from a ventilator. The study is in its early stages (Phase 1/2), which means it's one of the first times this medicine is being given to people. The main goals are to find out if ASP2957 is safe to use, what kind of side effects it might have, and if there are any early signs that it could help improve the muscle weakness, particularly how much breathing support participants might need. Participants will be carefully monitored over 52 weeks to track their health and how their body reacts to the treatment.
At a glance
What is this study about?
This study is exploring a new medicine, ASP2957, for boys and men living with X-linked myotubular myopathy. This is a serious condition that causes severe muscle weakness throughout the body, including the muscles needed for breathing. Because of this, many people with this condition rely on machines called ventilators to help them breathe. The new medicine is being developed to try and address the underlying cause of this muscle weakness.
This is an early-stage study, known as "Phase 1/2." This means it's among the first times the medicine is being tested in people. The main focus at this stage is to understand how safe the medicine is. Researchers will be looking very closely for any side effects and how well people tolerate the treatment. They also want to see if there are any early signs that the medicine could improve muscle strength and potentially reduce the amount of time someone needs breathing support.
Participants will receive ASP2957, and doctors will carefully monitor their health over a period of 52 weeks. They will perform various tests, including blood tests, heart checks, and scans of muscles, to see how the body reacts to the treatment and whether there are any positive changes in the condition. The overall aim is to gather important information that will help decide if this new medicine has the potential to become a helpful treatment for X-linked myotubular myopathy.
Key takeaways
- This study is testing a new medicine (ASP2957) for X-linked myotubular myopathy.
- It's an early-stage trial primarily focused on safety and side effects.
- The study includes boys and men with the condition, of any age.
- Participants will be closely checked for 52 weeks to see how their body reacts.
- The aim is to find out if the medicine could help improve muscle weakness, especially breathing.
Who may be eligible?
This study is specifically for boys and men who have been diagnosed with X-linked myotubular myopathy. The study is open to those of any age, though specific details about age limits would be discussed with the study team.
Because this is an early-stage study, participants will need to meet certain health criteria to ensure their safety and that the study results are clear. Your doctor will be able to check if your specific health situation matches what the researchers are looking for. It's important to discuss your full medical history with the study team to see if this study is suitable for you.
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 or man?
- Have you been diagnosed with X-linked myotubular myopathy?
- Are you willing to attend regular clinic visits and undergo various medical checks?
- Are you able to commit to a 52-week study period?
What does participation involve?
If you decide to take part in this study, you will receive the new medicine, ASP2957. Since this is an early-stage trial, you can expect to have a number of visits to the study clinic. These visits will involve various tests and assessments to carefully monitor your health and how your body is responding to the treatment. These may include blood and urine tests, heart checks like ECGs and ECHOs, physical examinations, and scans of your muscles (MRIs) and potentially muscle biopsies (taking a small sample of muscle tissue).
Doctors will be looking closely for any side effects and changes in your condition, including tracking the amount of time you spend on a ventilator if you use one. The study is planned to last for 52 weeks (about a year), during which you'll have regular follow-up appointments. After this initial period, there might be further follow-up to continue monitoring your long-term health and the effects of the treatment. All these procedures are designed to ensure your safety and gather important information about the new medicine.
Potential risks and benefits
Locations (1)
- —UnverifiedFrance
Common questions
What is X-linked myotubular myopathy?
It's a serious genetic muscle condition that mainly affects boys and men, causing severe muscle weakness, especially in the muscles needed for breathing.
What does 'ventilator-dependent' mean?
It means someone needs a breathing machine (ventilator) to help them breathe because their own breathing muscles are too weak.
What is ASP2957?
It's a new medicine being tested in this study to see if it can help improve X-linked myotubular myopathy.
What does Phase 1/2 mean?
It means this is one of the first times the medicine is being given to people, focusing on safety and initial signs of effectiveness.
How long will the study last for each person?
Participants will be monitored for 52 weeks (about one year) after they start the treatment.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
Discussion
Community discussion
Powered by our forum at community.patient.info. Please be respectful — this is not medical advice.