Assess efficacy of intra-arterial autologous myogenic stam cell therapy for m.3243A>G mutation carriers
This research is looking at a new treatment for a specific muscle condition caused by a genetic change called m.3243A>G mitochondrial myopathy. The treatment involves using some of the patient's own cells, called 'mesoangioblasts,' which are injected into the arm muscle. This is the very first time this treatment is being given to people, so the main goal is to carefully check if it's safe. Researchers will also be looking to see if it helps improve muscle strength and reduces muscle tiredness in the treated arm, compared to the untreated arm. This study helps us understand if this new cell therapy could be a good option for future treatments.
At a glance
What is this study about?
This study is a very early stage trial, meaning it's one of the first times this new treatment is being tested in people. It focuses on individuals who have a specific genetic muscle condition called m.3243A>G mitochondrial myopathy. This condition affects how the body's cells produce energy, leading to muscle weakness and tiredness. The new treatment involves using special cells from the patient themselves, called 'mesoangioblasts,' which are thought to have properties that could help repair or regenerate muscle.
The main purpose of this study is to find out if this new cell therapy is safe. Because it's a new treatment, the doctors will be watching participants very closely for any side effects or problems that might happen. They will also be looking to see if the treatment can make a difference to muscle strength and how tired the muscles feel in the arm where the cells are injected. They will compare the treated arm to the untreated arm to see if there's an improvement.
Researchers will use special equipment to measure muscle strength and fatigue both before the treatment and several weeks after. They will also carefully examine the muscle itself through small tissue samples to understand if the treatment changes the muscle in any way, such as its size or how well its energy factories (mitochondria) are working. This detailed investigation will help doctors understand more about how this new cell therapy works and whether it could be a helpful treatment option for this specific muscle condition in the future.
Key takeaways
- This study is for a specific genetic muscle condition (m.3243A>G mitochondrial myopathy).
- It uses your own cells as a potential new treatment.
- The main goals are to check for safety and potential improvements in muscle strength.
- This is an early-stage study, so the treatment is new to humans.
- Careful monitoring and assessments will be a big part of participation.
Who may be eligible?
To be part of this study, you need to be an adult, aged 18 years or older. There is no upper age limit, meaning older adults can also take part.
Both men and women are welcome to participate in this study. The researchers want to include a diverse group of people to understand how the treatment might affect different individuals.
It's important to remember that these are just general guidelines. Your doctor or the study team will be able to tell you for certain if you meet all the specific requirements to join this research.
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 18 years old or older?
- Do you have the m.3243A>G mutation that causes mitochondrial myopathy?
- Are you comfortable with the idea of receiving injections into your arm muscle?
- Are you willing to attend multiple clinic visits for monitoring and assessments?
What does participation involve?
If you join this study, you would receive the new cell therapy, which involves injections into one of your arm muscles. This is a very new treatment, so the team will monitor you very closely. You'll have several visits to the clinic involving checks like angiography (a special X-ray to look at blood vessels) and regular checks of your brain and nerve functions for at least 8 hours after the procedure to ensure safety. Your muscle strength and tiredness in both arms will be measured using a special machine called a Biodex dynamometer, both at the start and then again about 4 to 6 weeks after you've had three doses of the treatment. There will also be some muscle biopsies (small tissue samples) taken from your treated arm. The full duration of your participation in the study, including all treatments and follow-up assessments, will be explained in detail by the study team.
Potential risks and benefits
Locations (1)
- —UnverifiedNetherlands
Common questions
What is m.3243A>G mitochondrial myopathy?
It's a genetic condition where your body's cells struggle to make enough energy, leading to muscle weakness and tiredness.
What are 'autologous mesoangioblasts'?
These are special cells taken from your own body that are thought to help repair or grow new muscle tissue.
Is this treatment available to everyone?
No, this is a research study to test a new treatment, so it's only available to people who meet specific criteria.
Will I definitely get better if I join the study?
Not necessarily. This is an early study to check if the treatment is safe and if it starts to show any benefits.
What is a 'muscle biopsy'?
It's a procedure where a very small piece of muscle tissue is taken from your body for examination under a microscope.
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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