A Phase 1/2 Open-label, Multi-centre, Dose-exploration Trial to Evaluate the Safety and Preliminary Efficacy of VG801 via Subretinal Injection in Treatment of Patients with Biallelic ABCA4 Mutation-Associated Retinal Dystrophy.
This research study is looking into a new treatment called VG801 for a genetic eye disease that can lead to significant vision problems. The condition is caused by specific changes in the ABCA4 gene. This study is in its early stages (Phase 1/2), meaning it's one of the first times this treatment is being given to people. The main goals are to check if VG801 is safe and to see if it shows any signs of improving vision or slowing down the disease. Researchers will carefully monitor participants for any side effects and will perform various eye tests to track changes in their vision over time. This trial is open to adults of all genders.
At a glance
What is this study about?
This study is about a new treatment, called VG801, for a specific inherited eye condition that can cause your eyesight to get worse over time. This condition happens when there are changes, or 'mutations', in both copies of a gene called ABCA4. These changes stop the gene from working properly, leading to problems with the light-sensing cells at the back of your eye, called the retina.
The researchers want to see if VG801 can help. They will inject VG801 directly into your eye, underneath the retina. The aim is for VG801 to help the retina work better and potentially slow down or even improve vision loss. Because this is an early-stage study, they will be very carefully checking for safety to make sure the treatment doesn't cause any unexpected problems.
Alongside checking for safety, the study will also look for any early signs that the treatment is working. This involves lots of different eye tests that measure things like how well you see, how your retina looks, and how your eyes react to light. This information will help doctors understand if VG801 could be a helpful treatment for people with this condition in the future.
Key takeaways
- This study is testing a new gene therapy (VG801) for a specific genetic eye disease.
- It's an early-stage study focused on safety, but also looking for signs of vision improvement.
- The treatment is given as an injection into one eye.
- Participants will have many eye tests and health checks.
- The study is for adults aged 18 and over.
- You can withdraw from the study at any time.
Who may be eligible?
To be considered for this study, you must be an adult, aged 18 years or older. The study is open to both men and women.
The most important requirement is that you must have been diagnosed with the specific eye condition caused by changes in both copies of the ABCA4 gene. There will be other detailed medical checks to make sure your overall health and eye health are suitable for you to take part safely in the study. These checks are important to protect your well-being and to ensure the study results are as clear as possible.
Before you can join, the study doctors will review your medical history and perform a full eye exam to confirm you meet all the necessary criteria. They will explain exactly what these criteria are and answer any questions you have.
- Are you 18 years old or older?
- Have you been diagnosed with an eye condition caused by changes in both your ABCA4 genes?
- Are you able to attend regular clinic visits for eye tests and health checks?
- Are you generally in good health other than your eye condition?
This is a guide only — the research team will confirm whether you can take part.
What does participation involve?
If you decide to take part in this study, you will have the new treatment, VG801, injected into one of your eyes. This is a one-off procedure. After the injection, you will have a number of visits to the clinic so the doctors can closely monitor your health and your eyes. These visits will involve various tests.
These tests will include detailed eye examinations, such as looking at the inside of your eye with special cameras (like OCT and fundus photography), checking your eye pressure, and assessing your vision in different ways (like reading eye charts and checking your side vision). You will also have blood, tear, and saliva samples taken to check for any sign of the treatment leaving your body and to monitor your general health.
Throughout the study, you will have regular check-ups to make sure you are doing well and to see if the treatment is having any effect on your vision. The study duration will involve ongoing follow-up over a period of time, which the study team will explain in full.
Potential risks and benefits
Locations (1)
- —Germany
Common questions
What is VG801?
VG801 is a new experimental treatment being tested for an inherited eye condition. It's a type of gene therapy designed to help improve the function of your vision.
What is 'Phase 1/2' mean for a study?
Phase 1/2 means this is one of the earliest times this treatment is being given to people. The main goals are to check if it's safe and to see if it shows any early signs of working.
Will I get the actual treatment, or a dummy treatment?
This is an 'open-label' study, which means everyone who takes part and meets the requirements will receive the VG801 treatment. There is no dummy treatment being used in this trial.
How is the treatment given?
The VG801 treatment is given as a single injection directly into one of your eyes, underneath the retina.
Will this study cure my eye condition?
It's too early to say if VG801 will be a cure. This study aims to see if it's safe and if it can improve vision or slow down the progression of the condition. Many more studies would be needed before we know if it can be a cure.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
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