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

Research on Ultevursen for Retinitis Pigmentosa with USH2A Gene Mutations.

Recruiting
8 years and older
All
Phase 2
This study tests a new treatment called **ultevursen** for people with **Retinitis Pigmentosa (RP)**. RP is an eye disease that can cause vision loss. The study focuses on people with a specific gene change in **exon 13** of the **USH2A gene**. Ultevursen is given by an **intravitreal injection (IVT)**, which means it's injected into the eye. The study will compare ultevursen to a **sham procedure** (a fake treatment) to see if it is safe and works well. **Key Points to Consider:** - The study lasts about 18 months, with injections on Day 1, and again at months 6, 12, and 18. - Participants must be 8 years or older and be able to follow the study's rules and visits. - There are specific eye and genetic criteria to join the study, such as having RP due to exon 13 mutations. Participants will be randomly chosen to get either the treatment or the sham procedure. This study will help researchers understand if ultevursen is a safe and effective treatment for RP.
Study details
    Retinitis Pigmentosa (RP)
    Usher Syndrome Type 2
    Deaf Blind
    Retinal Disease
    Eye Diseases
    Hereditary
    Eye Disorders Congenital
    Vision Disorders

NCT06627179

Laboratoires Thea

7 March 2026

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