A Double-Masked, Randomized, Controlled, Multiple-Dose Study to Evaluate the Efficacy, Safety, Tolerability and Systemic Exposure of QR-110 in Subjects with Leber's Congenital Amaurosis (LCA) due to c.2991+1655A>G Mutation (p.Cys998X) in the CEP290 Gene
Phase of Trial: Phase II/III
Latest Information Update: 06 Feb 2019
At a glance
- Drugs QR 110 (Primary)
- Indications Leber congenital amaurosis
- Focus Registrational; Therapeutic Use
- Acronyms ILLUMINATE
- Sponsors ProQR Therapeutics
- 29 Jan 2019 According to the ProQR Therapeutics, updates from the study announced in ProQR Vision 2023 strategy and will be presented at an R&D day event.
- 29 Jan 2019 According to a ProQR Therapeutics media release, the company plans to complete pivotal program around year-end 2020 for submission of a New Drug Application (NDA) in the U.S. and a Marketing Authorization Application (MAA) in Europe in 2021.
- 07 Jan 2019 According to a ProQR Therapeutics media release, the company has reached agreement with the U.S. Food and Drug Administration (FDA) on the design of this trial. The company anticipates top line results for base-case of 30 trial participants around year-end 2020.