Active not recruitingPHASE1, PHASE2INTERVENTIONAL

Dose-escalation Study to Evaluate the Safety and Tolerability of GS030 in Subjects With Retinitis Pigmentosa

This research is a Phase 1/2a study looking into a new treatment for non-syndromic Retinitis Pigmentosa, a genetic eye condition that causes progressive vision loss. The study treatment, called GS030, is a gene therapy delivered as a single injection into the eye. Patients will also use special stimulating glasses, GS030-MD, to work with the gene therapy. The main goal is to check how safe the treatment is and if people tolerate it well, meaning how their bodies react to it. This early-stage study will also help doctors understand potential effects on vision. It's a key step in developing new ways to help those living with RP.

At a glance

Status

Active not recruiting

Phase

PHASE1, PHASE2

Sponsor

GenSight Biologics

Enrolment target

Start

26 Sep 2018

Estimated completion

26 Oct 2027

Non-syndromic Retinitis Pigmentosa

What is this study about?

This study is designed to explore a new potential treatment for a condition called Retinitis Pigmentosa, often shortened to RP. RP is a group of inherited eye diseases that cause slow, progressive vision loss due to damage to the light-sensing cells at the back of the eye. This particular study is for 'non-syndromic' RP, meaning the vision loss isn't part of a wider health condition affecting other parts of the body.

The new treatment being investigated is a type of gene therapy called GS030. In simple terms, gene therapy aims to introduce new genetic material into cells to help them work properly or to produce a missing protein. For this study, the GS030 treatment is given as a single injection directly into the eye. After the injection, patients will also use a special medical device, which are glasses designed to stimulate the eye, to work alongside the gene therapy.

This is an early-stage study, known as Phase 1/2a. This means the primary focus is on understanding the safety of the treatment and how well people tolerate it at different doses. Researchers will carefully monitor participants for any side effects and see how their eyes respond to the treatment. While the main goal is safety, they will also be looking for any signs that the treatment might be helping with vision. This research is a crucial step in hopefully developing new and effective ways to help people living with RP.

Key takeaways

This study is testing a new gene therapy (GS030) for Retinitis Pigmentosa (RP).
It involves an eye injection and special stimulating glasses.
The main goal is to check for safety and how well people tolerate the treatment.
It is for adults aged 18-75 with non-syndromic RP and specific vision levels.
Participation includes regular clinic visits and eye tests.
You can withdraw from the study at any time.

Who may be eligible?

To be considered for this study, you would generally need to be an adult between 18 and 75 years old. You must have a diagnosis of non-syndromic Retinitis Pigmentosa confirmed by an eye specialist through specific tests.

Your vision in the eye being studied would need to be quite poor, generally no better than being able to tell light from dark in the early parts of the study. You also need to have certain healthy features in your eye, such as enough ganglion cells and retinal nerve fibre thickness, which will be checked with special scans. There are also specific measurements for your pupil distance and the strength of your glasses prescription.

You would not be able to join if you've already had gene therapy. Other reasons for not being suitable include recent eye surgery, certain other eye conditions like severe cataracts or glaucoma, a history of retinal detachment or inflammation, or if you have an active implanted medical device. Your eye doctor will carefully review all your medical history to ensure this study is right for you.

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 between 18 and 75 years old?
Do you have a diagnosis of non-syndromic Retinitis Pigmentosa?
Is your vision in the study eye quite poor (e.g., light perception or worse, depending on the stage)?
Have you NOT had any previous gene therapy?
Do you have good overall eye health apart from your RP?
Are you willing and able to attend regular follow-up appointments?

Answer every question to see your result.

What does participation involve?

If you participate in this study, you will receive a single injection of the study drug into one of your eyes. You will also be provided with special stimulating glasses that you will need to use regularly as instructed. The study involves a number of clinic visits over time, where researchers will monitor your eye health and general well-being. These visits will include various eye tests, scans, and possibly blood tests to check for safety and how your eye is responding. The total duration of your participation will involve regular follow-up appointments over an extended period, allowing the researchers to thoroughly assess the long-term effects of the treatment and device.

Potential risks and benefits

Participating in a clinical trial may offer potential benefits, such as access to a new treatment before it's widely available and the opportunity to contribute to medical knowledge that could help others. However, there are also potential risks, including side effects from the injection or the gene therapy itself, and the possibility that the treatment may not be effective for you. All medical procedures carry some risks, and you will be fully informed about these. You have the right to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (3)

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

UPMC Eye Center
Verified postcode
Pittsburgh, United States
Centre Hospitalier National d'Ophtalmologie (CHNP) des Quinze-Vingts
Verified postcode
Paris, France
Moorfields Eye Hospital NHS Foundation Trust, 162 City Road
Verified postcode
London, United Kingdom

Common questions

What is Retinitis Pigmentosa (RP)?

RP is an inherited eye condition that causes cells at the back of the eye to gradually break down, leading to progressive vision loss.

What is gene therapy?

Gene therapy is a new type of treatment that aims to correct faulty genes or add new genes to help cells work properly, potentially treating genetic diseases like RP.

Will this treatment restore my vision completely?

This is an early-stage study focused on safety. While researchers hope for vision improvement, it's too early to say what the long-term effects on vision might be, and complete restoration is not guaranteed.

What are the stimulating glasses for?

The special glasses are a key part of the treatment, designed to work alongside the gene therapy injection to help stimulate the eye and potentially improve its response to the treatment.

How long will I be in the study?

The study involves regular check-ups over an extended period. The exact duration will be explained by the study team, but it's designed to monitor safety and effects long-term.