A Phase 1/2, First-in-Human, Open-label, Assessor-Masked, Randomized, Controlled, Dose Escalation/Expansion Study to Evaluate the Safety, Tolerability and Preliminary Efficacy of a Subretinal Injection of SB-007 in Subjects with Stargardt Disease (STGD1) Caused by Bi-Allelic Autosomal Recessive Mutations in the ATP Binding Cassette Subfamily A Member 4 (ABCA4) Gene (ASTRA).
This research study is looking at a new treatment called SB-007 for Stargardt disease, a genetic eye condition that leads to vision loss. It's the very first time this treatment is being tried in humans, which means it will be carefully overseen. The main goal is to find out if SB-007 is safe and well-tolerated when injected into the eye. Researchers will also be looking to see if it helps to slow down or stop the progression of the disease. They will compare people who receive SB-007 with those who don't. Anyone aged 18 and over with Stargardt disease caused by specific genetic changes can potentially take part.
At a glance
What is this study about?
This research study is about a new way to treat a condition called Stargardt disease. Stargardt disease is an inherited eye condition that damages the macula, which is the central part of your retina that helps you see fine details. This damage can lead to blurry vision, blind spots, and difficulty seeing in low light. This particular type of Stargardt disease is caused by changes (mutations) in a specific gene called ABCA4.
The new treatment being tested is called SB-007. It's given as an injection directly into the back part of the eye (under the retina). This study is a very important step because it's the first time doctors are trying this treatment in people. When a treatment is first given to people, doctors closely monitor their safety and how their bodies react.
Over the course of about two years, the study will focus on making sure the treatment is safe and doesn't cause too many side effects. Doctors will also carefully track whether the treatment has any positive effects on the eye, such as slowing down the growth of damaged areas in the retina or improving vision. They will compare these changes in people receiving SB-007 to those who don't.
Key takeaways
- This study is testing a new eye injection (SB-007) for Stargardt disease.
- It's the first time this treatment is being tried in people.
- The main goals are to check the treatment's safety and see if it helps vision or slows disease progression.
- You need to be aged 18+ and have Stargardt disease caused by specific genetic changes.
- Participation involves eye injections and frequent eye check-ups over about two years.
- There might be potential benefits, but also unknown risks, as it's a new treatment.
Who may be eligible?
To be considered for this study, you must be 18 years old or older. The study is open to both men and women.
Crucially, you would need to have been diagnosed with Stargardt disease. Specifically, your Stargardt disease must be caused by certain genetic changes in a gene called ABCA4. The study team will be able to confirm this for you.
There will be other detailed criteria that the study doctors will check to make sure the study is a good fit for you and that taking part would be safe. They will discuss all of these with you during the screening process.
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 a diagnosis of Stargardt disease?
- Has your doctor told you your Stargardt disease is caused by changes in the ABCA4 gene?
- Are you able to attend regular appointments for about two years?
What does participation involve?
If you decide to take part, you would receive the SB-007 treatment as an injection into your eye. Because this is a new treatment in humans, you would have many appointments, especially early on, to carefully check your eyes and overall health. These visits will involve various eye tests to measure your vision and look at the health of your retina. For example, doctors might take special pictures of the back of your eye or test how well you can see details.
Your participation would involve regular check-ups for at least 96 weeks (about two years). These visits are crucial for the doctors to monitor your safety very closely and also see if the treatment is having any effect on your Stargardt disease. You will also have blood tests and general health checks. The study team will explain the exact schedule of visits and what each appointment will involve.
Potential risks and benefits
Locations (2)
- —UnverifiedGermany
- —UnverifiedBelgium
Common questions
What is Stargardt disease?
Stargardt disease is an inherited eye condition that causes damage to the macula, the part of your eye responsible for sharp, central vision.
What is SB-007?
SB-007 is a new experimental treatment being tested for Stargardt disease. It's given as an injection into the eye.
What does a 'Phase 1/2' study mean?
This means it's one of the first studies in humans. Phase 1 focuses on safety, and Phase 2 starts looking at how well the treatment works while still checking for safety.
How long will the study last if I take part?
The study will actively monitor participants for at least 96 weeks, which is about two years.
Will I definitely get the new treatment?
This is a 'controlled' study, meaning some participants will receive SB-007, and others may not, to allow for comparison. The study team will explain how this works.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
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