An Open-label, Multicenter, Two Part, Ascending Dose Followed by a Controlled Trial to Assess the Safety and Efficacy of a Subretinal Administration of AAVB-039 in Participants with Stargardt Disease (STGD1) (CELESTE)
This research study, called CELESTE, is looking into a new treatment for Stargardt disease, an inherited eye condition that can cause vision loss. The treatment, called AAVB-039, is given directly into the eye. This is a very early-stage study, meaning it's the first time this specific treatment is being tested in humans. The main goal is to check how safe it is and if there are any side effects. Researchers will also be looking to see if the treatment helps improve vision or reduces the progression of the disease. This is important to understand if AAVB-039 could be a helpful new option for people with Stargardt disease in the future.
At a glance
What is this study about?
This study is about a new way to treat an eye condition called Stargardt disease (also known as STGD1). Stargardt disease is a genetic condition that causes vision to get worse over time, often starting in childhood or teenage years. It affects a part of your eye called the macula, which is important for sharp, central vision.
The new treatment being tested is called AAVB-039. It's given through a special injection under the retina, which is the light-sensitive layer at the back of your eye. This is a very important first step in testing a new medicine in people. Because it's a 'Phase 1 and Phase 2' study, it means researchers are focusing on two main things: first, checking if the treatment is safe and what dose works best; and second, seeing if it shows any signs of helping with the disease.
The main aim is to carefully watch for any side effects or problems caused by the treatment. They also want to see if it can help slow down the disease or even improve vision. They will do this by looking at how the vision changes, checking the health of the back of the eye, and testing how well people can see details.
Key takeaways
- This study is testing a brand-new treatment for Stargardt disease.
- It's the first time this treatment is being given to people.
- The main aims are to check safety and see if vision improves.
- Participants will be closely monitored with regular eye tests.
- Anyone 18 years or older with Stargardt disease can be considered.
Who may be eligible?
To join 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 the study. The researchers want to include a wide range of people to understand how the treatment works across different individuals.
Beyond age and gender, specific medical details about your Stargardt disease will be checked to make sure this study is right for you. Your eye doctor will be able to tell you if your particular情况 fits the study's 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 18 years old or older?
- Have you been diagnosed with Stargardt Disease?
- Are you able to attend regular hospital appointments?
- Are you generally in good health, apart from your eye condition?
What does participation involve?
If you decide to take part in this study, you would first go through a screening process to make sure you're a good fit. If eligible, you would receive the AAVB-039 treatment as a single injection into your eye. This is done by a specialist doctor.
After the treatment, you would have regular hospital visits for several months, and possibly longer, to carefully monitor your health and eye changes. These visits would involve various eye tests, such as checking your vision, taking pictures of the back of your eye, and other special scans. You might also have blood tests and general health checks.
The study is designed to follow you closely over time to understand how the treatment affects your eyes and overall health. The total duration of your participation could be quite long, potentially over a year, with a number of follow-up appointments planned.
Potential risks and benefits
Locations (4)
- —UnverifiedBelgium
- —UnverifiedItaly
- —UnverifiedNetherlands
- —UnverifiedNorway
Common questions
What is Stargardt disease?
Stargardt disease is an inherited eye condition that affects the central part of your vision, called the macula, making it harder to see details.
What is AAVB-039?
AAVB-039 is the name of the new treatment being tested in this study for Stargardt disease. It's given as an injection into the eye.
What does 'Phase 1 and 2' mean?
This means it's an early-stage study. Phase 1 mainly checks if the treatment is safe, and Phase 2 looks at whether it shows any signs of helping the condition.
Will I get better if I join this study?
Researchers hope the treatment might help, but there's no guarantee. The main goal is to find out if it's safe and if it has any positive effects.
How long will the study last if I join?
Participation in the study may involve a number of visits over several months or even longer, as researchers need to closely monitor the treatment's effects.
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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