Trials / NCT04855045
An Open-label, Dose Escalation and Double-masked, Randomized, Controlled Trial Evaluating Safety and Tolerability of Sepofarsen in Children (<8 Years of Age) With LCA10 Caused by Mutations in the CEP290 Gene.
Status unknown Phase 2 / Phase 3 RNA / antisense
- Sponsor
- ProQR Therapeutics
- Treatment
- sepofarsen
- Gene
- CEP290
- Conditions
- Leber Congenital Amaurosis 10, Blindness, Leber Congenital Amaurosis, Vision Disorders, Sensation Disorders, Neurologic Manifestations, Eye Diseases, Eye Disorders Congenital, Retinal Disease, Retinal Degeneration, Retinal Dystrophies
- Ages
- 0 Years to 7 Years
- Enrollment
- 15 participants
- Started
- 2021-03-23
- Main results expected
- 2023-12
- Results posted
- Not yet
- Record last updated
- 2022-03-25
What the study is testing
PQ-110-005 (BRIGHTEN) is an open-label, dose escalation and double-masked, randomized, controlled study evaluating safety and tolerability of sepofarsen administered via intravitreal (IVT) injection in pediatric subjects (\<8 years of age) with LCA10 due to the c.2991+1655A>G mutation over 24 months of treatment.
Where it runs
Belgium (1)
- Universitair Ziekenhuis Gent (UZ), Ghent (recruiting now)
Brazil (2)
- INRET Clinica e Centro de Pesquisa / Santa Casa BH, Belo Horizonte (recruiting now)
- Federal University of Sao Paulo - Hospital Sao Paulo, São Paulo (recruiting now)
Canada (1)
- University of Alberta, Edmonton, Alberta (not yet recruiting)
Germany (2)
- Justus-Liebig Universität - Department of Ophthalmology, Giessen (recruiting now)
- University of Tübingen - Institute for Ophthalmic Research, Tübingen (not yet recruiting)
Italy (1)
- Eye Clinic University of Campania Liugi Vanvitelli, Naples (not yet recruiting)
Netherlands (1)
- Amsterdam University Medica Center - Locatie AMC, Amsterdam (recruiting now)
United Kingdom (1)
- Moorfields Eye Hospital - NHS Foundation Trust, London (not yet recruiting)