A follow-on study for second-eye treatment for participants previously treated with gene therapy for X-Linked Retinitis Pigmentosa (XLRP)
This study is for males aged 5 and over who have a severe inherited eye condition called X-linked Retinitis Pigmentosa (XLRP). This condition can lead to blindness and is caused by a faulty gene. Participants in this study have already received a special gene therapy, called AAV5-hRKp.RPGR, in one eye as part of previous studies. This new study is looking into whether it's safe to give the same gene therapy treatment to their second eye. The treatment involves delivering a healthy version of the faulty gene to the eye, which researchers hope could help improve or preserve eyesight. The study will last about 5 and a half years, with regular check-ups to monitor safety and any changes in vision.
At a glance
What is this study about?
X-linked Retinitis Pigmentosa, or XLRP, is a serious inherited eye condition that can cause severe vision problems and eventually lead to blindness. It happens because of a change in a specific gene, a bit like a missing piece of an instruction manual in our body. Currently, there isn't an approved treatment for XLRP. This study uses a treatment called AAV5-hRKp.RPGR, which is a special way to deliver a healthy version of that faulty gene directly into the cells of the eye. The hope is that by doing this, it could help to improve or protect vision.
This study is a follow-on from previous trials where participants received this gene therapy in one eye. Now, researchers want to carefully check how safe it is to give the same treatment to the second eye. They also want to understand if the eye tolerates the treatment well. By understanding this, they can gather more information about whether this gene therapy could one day become a widely available treatment for people with XLRP.
It's important to remember that this is a research study. While there's a lot of hope surrounding gene therapies like this, it's still being investigated. The main goal here is to learn more about the safety and effects of treating the second eye. This kind of research is vital for finding new ways to help people living with serious conditions like XLRP.
Key takeaways
- This study is testing a gene therapy for X-linked Retinitis Pigmentosa (XLRP) in the second eye.
- It's only for men aged 5 and over who have already had this treatment in one eye in previous studies.
- The treatment involves a surgical procedure to deliver a healthy gene to the eye.
- The study lasts about 5 and a half years, with regular check-ups and monitoring.
- There's no guaranteed benefit to you, but your participation could help others with XLRP in the future.
- There are potential risks, including side effects from the treatment or surgery, which will be fully explained.
Who may be eligible?
This study is specifically for men who meet certain criteria. To be considered, you must be male and at least 5 years old. A key requirement is that you have previously taken part in other studies (MGT009 and MGT010) where you received the AAV5-hRKp.RPGR gene therapy in one of your eyes.
You also need to be willing to sign a consent form, which means you understand what the study involves and are happy to take part. It's also important that you are able to follow the study plan and attend all the long-term follow-up appointments.
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 male?
- Are you 5 years old or older?
- Have you previously received the AAV5-hRKp.RPGR gene therapy in one eye as part of studies MGT009 and MGT010?
- Are you willing to sign a consent form that confirms you understand the study?
- Are you able to attend all study appointments and follow the study instructions?
- Are you willing to undergo long-term follow-up appointments?
What does participation involve?
If you join this study, it will last for about 5 and a half years in total. It starts with a screening period that can last up to 6 months to make sure you're suitable for the study. If you are, you will likely receive the gene therapy treatment in your second eye on a specific day, delivered through a surgical procedure. The amount of treatment given might depend on how much you received in your first eye in the previous study.
After the treatment, there will be a 52-week assessment period where you'll have regular check-ups. These will involve various tests including blood tests, eye examinations, special imaging scans, and checks for any side effects. Following this, you'll enter a longer 4-year follow-up period to continue monitoring your health and vision over time. The study is organised into groups; some will receive the treatment first, while others will follow once safety has been confirmed.
Potential risks and benefits
Locations (1)
- -UnverifiedUnited Kingdom
Common questions
What is X-linked Retinitis Pigmentosa (XLRP)?
It's an inherited eye condition, passed down through genes, that can seriously affect vision and eventually lead to blindness.
What is gene therapy?
Gene therapy is a treatment that aims to correct faulty genes, in this case, by delivering a healthy version of a gene to cells in the eye.
Will I have to pay to be in this study?
No, the study drug and all study-related tests are paid for. You may also be reimbursed for travel costs.
How long will the study last?
The entire study will take about 5 years and 6 months, including several years of follow-up care.
Can I leave the study at any time?
Yes, you are free to withdraw from the study at any point without explanation.
How to find out more
Nicola Hall
Always speak to your GP or specialist before deciding to take part in a study.
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