Study to Evaluate Ultevursen in Subjects With Retinitis Pigmentosa (RP) Due to Mutations i
The purpose of this Phase 2b study is to evaluate the safety and tolerability of ultevursen administered via intravitreal injection (IVT) in subjects with Retinitis Pigmentosa (RP) due to mutations in exon 13 of the USH2A gene. This is a multicenter Double-masked, Randomized, Sham-controlled study which will enroll 81
| Condition(s) | Retinitis Pigmentosa (RP), Usher Syndrome Type 2, Deaf Blind, Retinal Disease, Eye Diseases, Hereditary, Eye Disorders Congenital, Vision Disorders |
|---|---|
| Status | Recruiting |
| Phase | Phase 2 |
| Study type | Interventional |
| Summary | The purpose of this Phase 2b study is to evaluate the safety and tolerability of ultevursen administered via intravitreal injection (IVT) in subjects with Retinitis Pigmentosa (RP) due to mutations in exon 13 of the USH2A gene. This is a multicenter Double-masked, Randomized, Sham-controlled study which will enroll 81 subjects. |
| Who can participate | Inclusion Criteria: 1. An adult (≥18 years) willing and able to provide informed consent for participation prior to performing any study related procedures 2. OR A minor (8 to \<18 years) able to provide age-appropriate assent for study participation with a parent or legal guardian willing and able to provide written permission for the subject's participation prior to performing any study related procedures. An adult willing to comply with the protocol, follow study instructions, attend study visits as required and willing and able to complete all study assessments, in the opinion of the Investigator. OR A minor able to complete all study assessments and comply with the protocol and has a parent or caregiver willing and able to follow study instructions and attend study visits with the sub |
| Ages | 8 Years |
| Sex | All |
| Lead sponsor | Laboratoires Thea |
| Locations | San Francisco, California, United States; Miami, Florida, United States; Atlanta, Georgia, United States; Boston, Massachusetts, United States; Ann Arbor, Michigan, United States; Durham, North Carolina, United States (+21 more sites) |
| Start date | 2024-12-11 |
| NCT ID | NCT06627179 |
| Official listing | https://clinicaltrials.gov/study/NCT06627179 |