ATSN-201 Gene Therapy in RS1-Associated X-linked Retinoschisis
This research study is investigating a new gene therapy called ATSN-201 for an inherited eye condition known as X-linked Retinoschisis (XLRS). XLRS affects the retina, the light-sensitive layer at the back of your eye, leading to vision problems. This study wants to find out if ATSN-201 is both safe to use and whether it can improve vision or slow down the progression of XLRS in people aged 6 and older. The treatment involves a one-time injection into the eye. Some people in the study will receive the treatment, while others won't at first, but may have the option later. Your participation could help scientists understand if this new therapy could be a future treatment option for XLRS.
At a glance
What is this study about?
This study is looking into a new treatment called ATSN-201 for a condition called X-linked Retinoschisis, or XLRS. XLRS is a rare, inherited eye condition that mainly affects boys and men. It happens because of a problem with a specific gene (RS1), which causes layers of the retina – the light-sensitive tissue at the back of your eye – to split. This splitting can lead to blurry vision and sometimes more severe vision loss.
ATSN-201 is a type of 'gene therapy'. This means it aims to correct the underlying genetic problem causing XLRS. The idea is to deliver a healthy version of the RS1 gene into the eye, which hopefully will help the retina work better and prevent further vision problems. The study will carefully check if this treatment is safe and if it helps improve or stabilise vision in people with XLRS.
The study is divided into different parts. In one part, people will be randomly chosen to either receive the ATSN-201 treatment or to be in a group that doesn't get the treatment initially. Those who receive the treatment will have a single injection into one or both eyes, depending on their specific eye condition. Everyone involved will have regular check-ups over a year, and then follow-up for another four years to see how they are doing in the long term. This careful approach helps the researchers understand the full effects of the treatment.
Key takeaways
- This study investigates a new gene therapy (ATSN-201) for X-linked Retinoschisis (XLRS).
- It aims to check the safety and effectiveness of the treatment in people aged 6 and over.
- The treatment involves a one-time injection into the eye.
- Participation includes regular check-ups over a period of about five years.
- Some participants will receive the treatment, while others may get it later.
- This research could help develop future treatments for XLRS.
Who may be eligible?
To join this study, participants generally need to be male and have been diagnosed with X-linked Retinoschisis (XLRS) caused by a problem with the RS1 gene. They need to be at least 6 years old. Your vision in the eye being studied will also need to be within a certain range – not too good, but not severely poor either. Doctors will check for specific signs of the condition in your eye, like splitting in certain areas of the retina.
There are also reasons why someone might not be able to join. For example, if you have other serious eye conditions that could make the treatment risky, or if you've had eye surgery recently. Also, if you've previously had other types of gene or cell therapy for your eyes, you wouldn't be able to take part in this particular study. The study team will review all your medical information carefully to see if it's safe and appropriate for you to participate.
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 and diagnosed with X-linked Retinoschisis (XLRS)?
- Is your XLRS caused by a problem with the RS1 gene?
- Are you 6 years old or older?
- Is your vision in the affected eye within a specific range (not too good or too bad)?
- Do you have signs of retinal splitting in your eye as seen by a doctor?
- Have you *not* had recent eye surgery or other gene therapies for your eyes?
What does participation involve?
If you decide to take part, the study involves several steps. First, doctors will check if you're suitable to join. If you are, you'll be assigned to either receive the ATSN-201 treatment or to a group that doesn't get the treatment straight away. If you receive the treatment, it will be given as a single injection into one or both of your eyes.
After this, you'll have regular hospital visits and check-ups for about one year to see how you're responding and to monitor your eye health. These checks might include eye exams, vision tests, and scans of your retina. Following this initial year, there will be further check-ups over the next four years to see the long-term effects of the treatment. If you were in the group that didn't receive the treatment initially, you might have the option to receive it after the first year, if you're still eligible. The total study duration, including follow-up, could be around five years.
Potential risks and benefits
Locations (21)
- Associated Retina ConsultantsVerified postcodePhoenix, United States· Recruiting
- Shiley Eye InstituteVerified postcodeLa Jolla, United States· Not yet recruiting
- Children's Hospital of Los AngelesVerified postcodeLos Angeles, United States· Recruiting
- Stanford UniversityVerified postcodePalo Alto, United States· Not yet recruiting
- UC San FranciscoVerified postcodeSan Francisco, United States· Recruiting
- Bascom Palmer Eye InstituteVerified postcodeMiami, United States· Recruiting
- Massachusetts Eye and EarVerified postcodeBoston, United States· Recruiting
- Boston Children's HospitalVerified postcodeBoston, United States· Not yet recruiting
- University of MichiganVerified postcodeAnn Arbor, United States· Recruiting
- Mayo ClinicVerified postcodeRochester, United States· Recruiting
- Columbia UniversityVerified postcodeNew York, United States· Recruiting
- Flaum Eye Institute, University of RochesterVerified postcodeRochester, United States· Not yet recruiting
Common questions
What is X-linked Retinoschisis (XLRS)?
XLRS is a rare, inherited eye condition that causes layers of the retina (the light-sensitive part at the back of your eye) to split, leading to vision problems.
What is gene therapy?
Gene therapy is a new type of treatment that aims to fix the underlying genetic cause of a disease, in this case, by delivering a healthy gene into the eye.
Will I definitely get the treatment if I join?
Not everyone will get the active treatment straight away. Some participants will be in a group that receives the treatment, while others will be in a comparison group. However, those in the comparison group may have the option to receive the treatment later.
How is the treatment given?
The treatment, ATSN-201, is given as a single injection into the eye, specifically under the retina.
How long will I be involved in the study?
You will have regular checks for one year after the treatment, followed by additional checks over the next four years, making it a commitment of around five years.
How to find out more
Atsena Therapeutics Clinical Trials
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
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