Trials / NCT05176717
Study to Evaluate the Efficacy Safety and Tolerability of QR-421a in Subjects With RP Due to Mutations in Exon 13 of the USH2A Gene With Early to Moderate Vision Loss (Celeste)
Terminated Phase 2 / Phase 3 RNA / antisense
Why it stopped: Business decision
- Sponsor
- Laboratoires Thea
- Treatment
- QR-421a; Sham-procedure
- Gene
- USH2A
- Conditions
- Retinitis Pigmentosa, Usher Syndrome Type 2, Deaf Blind, Retinal Disease, Eye Diseases, Eye Diseases, Hereditary, Eye Disorders Congenital, Vision Disorders
- Ages
- 12 Years to no upper limit
- Enrollment
- 5 participants
- Started
- 2021-12-15
- Main results expected
- 2022-08-02
- Results posted
- Yes
- Record last updated
- 2024-08-07
What the study is testing
The purpose of this study is to evaluate the efficacy safety and tolerability of QR-421a administered via intravitreal injection (IVT) in subjects with Retinitis Pigmentosa (RP) due to mutations in exon 13 of the USH2A gene with early to moderate vision loss.
Where it runs
United Kingdom (1)
- Moorfields Eye Hospital, London
United States (2)
- Retina Foundation of the Southwest, Dallas, Texas
- University of Wisconsin - Madison, Madison, Wisconsin