RecruitingPHASE2INTERVENTIONAL

Study to Evaluate Ultevursen in Subjects With Retinitis Pigmentosa (RP) Due to Mutations in Exon 13 of the USH2A Gene

This study, called a Phase 2b trial, is investigating a new medication named Ultevursen for people who have an eye condition called Retinitis Pigmentosa (RP). Specifically, it's for RP that happens because of changes in a particular part of the USH2A gene (exon 13). The main goal is to check if Ultevursen is safe and how well people can cope with it. The treatment is given as an injection directly into the eye. This research will involve 81 participants and is designed as a 'double-masked, randomized, sham-controlled study.' This means some people will receive the actual treatment, and others will have a 'sham' procedure (which looks and feels like the injection but doesn't contain the medicine), and neither the participants nor their doctors will know who is getting what until the study ends. This helps ensure fair and accurate results.

At a glance

Status

Recruiting

Phase

PHASE2

Sponsor

Laboratoires Thea

Enrolment target

Start

11 Dec 2024

Estimated completion

01 Dec 2027

Retinitis Pigmentosa (RP)Usher Syndrome Type 2 Deaf Blind Retinal Disease Eye Diseases, Hereditary Eye Disorders Congenital Vision Disorders

What is this study about?

This study is exploring a new medicine called Ultevursen for a specific eye condition called Retinitis Pigmentosa (RP). RP is a group of eye diseases that cause vision loss over time. The particular focus of this study is on RP that occurs because of certain genetic changes in a gene known as USH2A, especially when the changes are in a section called 'exon 13'.

The main purpose of this research is to see if Ultevursen is safe to use and whether people can tolerate it well. As part of the study, the medication will be given as an injection directly into the eye. This is a 'Phase 2b' study, which means it's an earlier stage of testing a new medicine in people, usually after it's been shown to be safe in a small number of people and before bigger studies start. The hope is that this treatment might help slow down or manage the vision loss associated with this type of RP.

Eighty-one people with this specific type of RP will take part. To make sure the study results are reliable, participants will be randomly assigned to one of two groups: either they will receive the Ultevursen treatment, or they will have a 'sham' procedure. A sham procedure looks and feels like the actual treatment but doesn't involve the active medicine. This helps researchers compare the effects of the treatment fairly. Neither the participants nor the doctors will know who is in which group until the study is over, which is called 'double-masked'.

Key takeaways

This study is testing a new medicine, Ultevursen, for a specific type of Retinitis Pigmentosa (RP).
It's exploring the safety and tolerability of the treatment, given as an eye injection.
The study involves 81 participants, including children from 8 years old and adults.
Participants will be randomly assigned to receive either the treatment or a 'sham' procedure.
The study will last approximately 18 months, with regular eye examinations.
Your participation is voluntary, and you can withdraw at any time.

Who may be eligible?

To join this study, you need to meet certain requirements. You can be an adult aged 18 or older, or a child between 8 and 18 years old. If you're a child, your parent or guardian must give their permission, and you'll need to agree to take part as well. Everyone involved must be able to follow the study instructions and attend all necessary appointments.

You must have been diagnosed with Retinitis Pigmentosa (RP) in both eyes. This could be part of Usher syndrome type 2 (where you have RP and hearing loss from birth) or just RP by itself (without the hearing loss). A key part of qualifying is having a genetic test that shows changes in the USH2A gene, specifically with at least one of those changes being in the 'exon 13' section of the gene.

Your vision will also be checked. This includes having a certain level of vision in both eyes (for example, being able to read at least 55 letters on a standard eye chart, which is about 20/80 vision or better). Your peripheral (side) vision and other eye measurements will also need to be within specific ranges as determined by the study doctors.

Quick self-check

Are you 8 years old or older?
Do you have a diagnosis of Retinitis Pigmentosa (RP) in both eyes?
Have you had a genetic test confirming changes in the USH2A gene, specifically in exon 13?
Is your vision (including central and side vision) within specific ranges set by the study?
Are you willing and able to attend all study visits and follow instructions for about 18 months?

This is a guide only — the research team will confirm whether you can take part.

What does participation involve?

If you decide to take part in this study, you will be one of 81 people. You will be randomly assigned to one of two groups: either you'll receive the Ultevursen treatment, or you'll have a 'sham' procedure. This assignment will be decided by chance, like flipping a coin.

If you are in the treatment group, you will receive an injection of Ultevursen into one of your eyes (the 'treatment eye') at the start of the study, and then again at 6 months, 12 months, and 18 months. If you are in the sham group, you will have a 'sham' procedure at the same time points. This sham procedure is designed to feel similar to the actual injection but without any medicine. Neither you nor your study doctor will know which group you are in until the study is finished.

Throughout the study, you will have regular visits to the clinic for various eye tests and check-ups to monitor your vision, eye health, and overall well-being. The total duration of your participation in the study will be approximately 18 months, with follow-up appointments after the last treatment.

Potential risks and benefits

Taking part in a clinical study can offer potential benefits, such as receiving a new and innovative treatment that might help your condition, even though it's still being tested. You will also receive careful medical monitoring from specialists throughout the study. However, there are also potential risks, as the long-term effects and full safety profile of Ultevursen are still being evaluated. Injections into the eye, even sham ones, carry a small risk of discomfort, bruising, infection, or other eye complications. You will be fully informed about all known risks before you decide to join. It is very important to remember that participating in this study is completely voluntary, and you have the right to withdraw at any time, for any reason, without it affecting your usual medical care.

Locations (27)

The University of California, San Francisco
San Francisco, United States· Recruiting
Bascom Palmer Eye Institute/University of Miami
Miami, United States· Recruiting
Emory University
Atlanta, United States· Recruiting
Massachusetts Eye and Ear
Boston, United States· Recruiting
University of Michigan- Kellogg Eye Center
Ann Arbor, United States· Recruiting
Duke Eye Center
Durham, United States· Recruiting
Casey Eye Institute, Oregon Health & Science University
Portland, United States· Recruiting
University of Pennsylvania, Scheie Eye Institute
Philadelphia, United States· Recruiting
Retina Foundation of the Southwest
Dallas, United States· Recruiting
Baylor College of Medicine
Houston, United States· Recruiting
University of Wisconsin- Madison
Madison, United States· Recruiting
Ghent University Hospital
Ghent, Belgium· Recruiting

+15 more sites — see the official record for the full list.

Common questions

What is Retinitis Pigmentosa (RP)?

RP is a group of inherited eye diseases that cause damage to the retina (the light-sensitive tissue at the back of the eye) over time, leading to worsening vision.

What is Ultevursen?

Ultevursen is an investigational new medicine being tested in this study to see if it can help people with a specific type of RP.

What does 'intravitreal injection' mean?

It means the medicine is given directly into the gel-like substance in the middle of your eye using a very fine needle.

What is a 'sham' procedure?

A sham procedure is like a 'dummy' treatment. It looks and feels similar to the real injection but doesn't contain any active medicine, helping researchers compare results fairly.

How long will the study last?

If you join, your participation in the study will involve treatments and check-ups over about 18 months.

How to find out more

Sepul Bio Advocacy Director

contact@sepulbio.com +31 617060791 Official trial record

Always speak to your GP or specialist before deciding to take part in a study.