Trials / Recruiting
RecruitingNCT06627179
Study to Evaluate Ultevursen in Subjects With Retinitis Pigmentosa (RP) Due to Mutations in Exon 13 of the USH2A Gene
A Two-Year Double-masked, Randomized, Sham-Controlled Study to Evaluate the Efficacy, Safety and Tolerability of Ultevursen in Subjects With Retinitis Pigmentosa (RP) Due to Mutations in Exon 13 of the USH2A Gene
- Status
- Recruiting
- Phase
- Phase 2
- Study type
- Interventional
- Enrollment
- 81 (estimated)
- Sponsor
- Laboratoires Thea · Industry
- Sex
- All
- Age
- 8 Years
- Healthy volunteers
- Not accepted
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.
Detailed description
A total of eighty-one (81) RP subjects will be enrolled in this study, randomized in a 2:1 ratio to either ultevursen or sham procedure, respectively. Subjects randomized to the active treatment group will receive therapy with ultevursen administered via intravitreal (IVT) injection to the treatment eye (TE) on Day 1 and at Months 6, 12, and 18. Subjects randomized to sham will undergo a sham procedure in the TE at the corresponding timepoints.
Conditions
- Retinitis Pigmentosa (RP)
- Usher Syndrome Type 2
- Deaf Blind
- Retinal Disease
- Eye Diseases, Hereditary
- Eye Disorders Congenital
- Vision Disorders
Interventions
| Type | Name | Description |
|---|---|---|
| DRUG | Intravitreal Injection of Ultevursen | Up to 4 doses over a 24-month period |
| OTHER | No intervention, will not receive any active study intervention | Sham-procedure (no experimental drug administered) |
Timeline
- Start date
- 2024-12-11
- Primary completion
- 2027-12-01
- Completion
- 2027-12-01
- First posted
- 2024-10-04
- Last updated
- 2026-03-30
Locations
27 sites across 10 countries: United States, Belgium, Brazil, Canada, Denmark, France, Germany, Italy, Netherlands, United Kingdom
Regulatory
- FDA-regulated drug study
Source: ClinicalTrials.gov record NCT06627179. Inclusion in this directory is not an endorsement.