ATA-100 (Formerly GNT0006) Gene Therapy Trial in Patients With LGMDR9
This research study is looking at a new treatment called ATA-100, which is a type of gene therapy. It's for adults with a rare muscle condition called LGMDR9. We’re in the very first stage of testing (Phase 1) to make sure ATA-100 is safe and that people can tolerate it well. The treatment aims to deliver a healthy FKRP gene into the body. We'll start with a small number of participants, up to six in total. These participants will receive a single dose of the treatment, and medical teams will monitor them very carefully for five years to see how they respond and if there are any side effects. The study is a step towards hopefully finding new ways to help people with LGMDR9.
At a glance
What is this study about?
This study is about a new treatment called ATA-100 for a rare muscle condition known as LGMDR9. Our muscles need specific instructions (genes) to work properly. In LGMDR9, there's a problem with one of these instructions, the FKRP gene, which leads to muscle weakness, including weakness in the breathing muscles.
ATA-100 is a 'gene therapy'. Think of it as a special delivery system designed to carry a healthy copy of the FKRP gene into the muscle cells. The goal is to help these cells get the correct instructions so they can start working better. This is an early-stage study, meaning it's one of the first times this treatment is being tested in people. We'll be closely watching its safety and how the body handles it.
By carefully studying a small group of people, we can learn a lot about how this new therapy might work and if it could potentially help people with LGMDR9 in the future. The information we gather will be very important for developing better treatments for this condition.
Key takeaways
- It's a Phase 1 study testing a new gene therapy (ATA-100) for LGMDR9.
- The main goal is to check the treatment's safety and how the body tolerates it.
- Up to six adults with LGMDR9 will receive a single treatment dose.
- Participants will be monitored closely for five years after treatment.
- The study helps us understand if this new therapy could be a future option for LGMDR9.
Who may be eligible?
To join this study, you need to be an adult, aged 16 or older, and able to walk. Both men and women can take part. You must also have a clear diagnosis of LGMDR9, confirmed by genetic tests showing changes in your FKRP gene, and have some weakness in your diaphragm, which is a muscle important for breathing.
There are some reasons why you might not be able to join. For example, if your body already has certain natural protections (called antibodies) against the type of 'carrier' used to deliver the new gene (AAV9), you wouldn't be able to participate. Also, if you have a heart condition called cardiomyopathy, you wouldn't be eligible for this study. The study doctors will perform checks to see if you meet all the requirements.
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 16 years old or older?
- Can you walk without help?
- Do you have a confirmed diagnosis of LGMDR9?
- Do you have some weakness in your breathing muscle (diaphragm)?
- Have you been told you don't have certain antibodies against AAV9?
- Have you been told you don't have cardiomyopathy (a heart condition)?
What does participation involve?
If you decide to take part in this study, you would primarily be helping us understand if the new treatment, ATA-100, is safe and how your body reacts to it over time. After an initial health check to ensure you meet all the study requirements, you would receive a single dose of the study treatment. This will be given either as a drip. The study aims to enrol a total of six people, with three receiving one dose, and three others receiving a slightly higher dose.
Following the treatment, you will have regular medical appointments and tests for up to five years. These might include blood tests, muscle strength checks, and assessments of your breathing and general well-being. These check-ups are very important so the doctors can carefully monitor your health and see how you are responding to the treatment. The total duration of your participation, including all follow-up visits, would be for a maximum of five years.
Potential risks and benefits
Locations (3)
- Rigshospitalet, University of Copenhagen Blegdamsvej 9Verified postcodeCopenhagen, Denmark
- Institute of Myology Pitié-Salpêtrière Hospital 47 Bd de l'HôpitalVerified postcodeParis, France
- Royal Victoria Infirmary Queen Victoria Road Level 6 Leazes WingVerified postcodeNewcastle upon Tyne, United Kingdom
Common questions
What is LGMDR9?
LGMDR9 is a rare condition that causes muscles to weaken, including muscles needed for movement and breathing.
What is gene therapy?
Gene therapy aims to fix faulty genes by delivering healthy copies to cells, helping them work correctly.
Is ATA-100 a cure for LGMDR9?
This is an early-stage study, so we don't know yet if it's a cure. We are testing its safety and effects.
How long will I be in the study?
You will be followed by the study team for up to five years after receiving the treatment.
Can I leave the study if I change my mind?
Yes, you can choose to leave the study at any time, and it won't affect your regular medical care.
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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