Trials / NCT03913130
Extension Study to Study PQ-110-001 (NCT03140969)
Terminated Phase 1 / Phase 2 RNA / antisense
Why it stopped: Terminated \[Study prematurely terminated due to sponsor decision for reasons unrelated to safety\]
- Sponsor
- Laboratoires Thea
- Treatment
- QR-110
- Gene
- CEP290
- Conditions
- Leber Congenital Amaurosis 10, Blindness, Leber Congenital Amaurosis, Vision Disorders, Sensation Disorders, Neurologic Manifestations, Eye Diseases, Eye Diseases, Hereditary, Eye Disorders Congenital, Retinal Disease
- Ages
- 6 Years to no upper limit
- Enrollment
- 9 participants
- Started
- 2019-05-13
- Main results expected
- 2022-10-03
- Results posted
- Yes
- Record last updated
- 2024-10-15
What the study is testing
Subjects completing participation in study PQ-110-001 (EudraCT 2017-000813-22 / NCT03140969) will be given the opportunity to enroll into the extension study for continued dosing if available data support current and/or future benefits for the subject. Study PQ-110-002 will provide long-term safety, tolerability, pharmacokinetic (PK), and efficacy data of QR-110.
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