Trials / NCT03140969
Study to Evaluate QR-110 in Leber's Congenital Amaurosis (LCA) Due to the c.2991+1655A>G Mutation (p.Cys998X) in the CEP290 Gene
Completed Phase 1 / Phase 2 RNA / antisense
- Sponsor
- Laboratoires Thea
- Treatment
- QR-110
- Gene
- CEP290
- Conditions
- Leber's Congenital Amaurosis
- Ages
- 6 Years to no upper limit
- Enrollment
- 11 participants
- Started
- 2017-10-16
- Main results expected
- 2019-10-02
- Results posted
- Yes
- Record last updated
- 2024-10-15
What the study is testing
The purpose of this study is to evaluate the safety and tolerability of QR-110 administered via intravitreal injection in subjects with LCA due to the CEP290 p.Cys998X mutation.
Where it runs
Belgium (1)
- Ghent University Hospital and Ghent University, Ghent
United States (2)
- University of Iowa, Iowa City, Iowa
- Scheie Eye Institute, University of Pennsylvania, Philadelphia, Pennsylvania