Active not recruitingPHASE2, PHASE3INTERVENTIONAL

A Clinical Trial Evaluating the Safety and Efficacy of a Single Subretinal Injection of AGTC-501 in Participants With XLRP

This research is looking at a new treatment called AGTC-501, which is a type of gene therapy, for young men aged 12 to 50 who have an inherited eye condition called X-linked retinitis pigmentosa (XLRP). This condition causes vision loss. The study wants to find out if AGTC-501 is safe and effective in improving or stabilising eyesight. Participants will be split into three groups: two groups will receive different doses of the AGTC-501 treatment, given as a single injection into the eye, and one group will not receive the treatment initially but will be closely monitored. Later, the untreated group may have the chance to receive the treatment too. The study hopes to understand how well this new therapy works and how safe it is for people with XLRP.

At a glance

Status

Active not recruiting

Phase

PHASE2, PHASE3

Sponsor

Beacon Therapeutics

Enrolment target

Start

14 Mar 2024

Estimated completion

31 Oct 2031

X-Linked Retinitis Pigmentosa

What is this study about?

This study is investigating a new potential treatment for an eye condition called X-linked retinitis pigmentosa, or XLRP. This is a genetic condition that mainly affects males and leads to progressive vision loss over time. The experimental treatment, called AGTC-501, is a type of gene therapy. This means it aims to deliver a healthy copy of a gene that is faulty in people with XLRP, hoping to correct the problem and potentially improve or save their vision.

The researchers want to carefully test this new treatment. They will be looking at its safety – making sure it doesn't cause harmful side effects – and its effectiveness – seeing if it actually helps improve or prevent further vision loss. The treatment involves a single injection directly into the eye. By comparing different doses of the treatment to a group that doesn't receive it straight away, the scientists can gather important information about how well it works and what the best approach might be.

Finding new treatments for conditions like XLRP is really important because currently, there are limited options. This study could be a step towards a new way to help people living with this challenging eye condition, potentially making a real difference to their quality of life and preserving their sight.

Key takeaways

Targets inherited vision loss (XLRP) in males.
Uses a new gene therapy (AGTC-501) as a single eye injection.
Compares two treatment doses to an observational group.
Aims to check both safety and effectiveness.
Participation involves regular eye checks and clinic visits.

Who may be eligible?

To join this study, participants must be male and between 12 and 50 years old. You need to have a confirmed diagnosis of X-linked retinitis pigmentosa (XLRP) caused by specific changes in a gene called RPGR. Your vision must be within a certain range – not too good, but also not extremely poor – in the eye being treated.

It's important that you also have some measurable vision in your central sight, where you see details, and certain parts of your eye should still be working, as seen in eye scans. You must be able to complete all the eye tests required by the study.

You cannot join if you have other eye conditions that might affect how the treatment works or if you have certain other serious health issues that could interfere with the study's findings.

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 between 12 and 50 years old?
Do you have a diagnosis of X-linked retinitis pigmentosa (XLRP) caused by an RPGR gene change?
Is your vision, in the eye to be treated, not too good (worse than about 20/32) but also not too poor (better than about 20/200)?
Can you comfortably complete detailed eye tests?
Do you not have other eye conditions that might interfere with the study?

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, you'll first have to go through a screening process to make sure you're a good fit. If you're eligible, you'll be randomly placed into one of three groups. Two groups will receive different doses of the study drug, AGTC-501, given as a single injection into one eye. The third group will be observed without receiving the treatment initially, but they may have the chance to get the treatment after a certain period.

Throughout the study, you'll have regular hospital visits. These visits will involve various eye tests to check your vision, take pictures of your eye, and assess how your eye is functioning. This is to monitor the safety of the treatment and see if it's having any effect on your vision. You'll need to follow instructions carefully and attend all scheduled appointments. The total duration of your participation in the study, including follow-up, will be explained by the study team.

Potential risks and benefits

Taking part in any medical study has potential benefits and risks. You might benefit from the new treatment if it helps your vision, especially since current treatments for XLRP are limited. However, there's also the chance that the treatment might not work for you, or it could cause side effects. Like any eye procedure, the injection carries risks such as infection or damage to the eye. You will be told about all known risks before you decide to take part. Remember, joining is entirely voluntary, and you have the right to withdraw from the study at any time, for any reason, without it affecting your medical care.

Locations (25)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

Retina Macula Institute of Arizona
Verified postcode
Scottsdale, United States
Children's Hospital Los Angeles
Verified postcode
Los Angeles, United States
University of Florida Health Jacksonville, Department of Ophthalmology
Verified postcode
Jacksonville, United States
Bascom Palmer Eye Institute- University of Miami
Verified postcode
Miami, United States
Midwest Eye Institute (Retina Partners Midwest)
Verified postcode
Carmel, United States
Wilmer Eye Institute at Johns Hopkins
Verified postcode
Baltimore, United States
Ophthalmic Consultants of Boston
Verified postcode
Boston, United States
Mayo Clinic
Verified postcode
Rochester, United States
Duke Eye Center
Verified postcode
Durham, United States
Cincinnati Eye Institute
Verified postcode
Cincinnati, United States
Cole Eye Institute - Cleveland Clinic
Verified postcode
Cleveland, United States
Casey Eye Institute, OHSU
Verified postcode
Portland, United States

Common questions

What is X-linked retinitis pigmentosa (XLRP)?

XLRP is an inherited eye disease that mostly affects males, causing vision to gradually get worse over time.

What is AGTC-501?

AGTC-501 is an investigational gene therapy designed to replace a faulty gene in the eye that causes XLRP, in the hope of improving or stabilising vision.

How is the treatment given?

The treatment is given as a single injection directly into one eye.

Will I definitely get the treatment if I join?

You will be randomly assigned to one of three groups; two groups receive the treatment, and one group is observed first. The observed group may receive treatment later.

How long will the study last for?

The total length of your participation, including follow-up, will be explained by the study team, but it will involve multiple visits over time.