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Leber Congenital Amaurosis 10 clinical trials

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NCT ID: NCT04855045 Recruiting - Eye Diseases Clinical Trials

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.

BRIGHTEN
Start date: March 23, 2021
Phase: Phase 2/Phase 3
Study type: Interventional

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.