ProQR Therapeutics is recruiting patients for the clinical trial of Sepofarsen in Children (<8 Years of Age) with LCA10
This 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.
The study consists of two parts: an open-label dose escalation part, followed by a double-masked randomized part. In the open label part; subjects will be assigned to one of 3 planned dose groups using a staggered dose escalation design. After at least 1 patient is dosed in each group; the Data Monitoring Committee (DMC) will review at least 4 weeks of safety data post dosing; and may recommend initiation of the next dose group. The DMC may recommend initiation of the double-masked randomized part of the study after completion of the last dose group in the dose escalation part of the study.
In the double-masked, randomized, controlled part of the study; subjects will be randomized to one of 2 planned dose groups . Subjects will receive a unilateral IVT injection of sepofarsen on Day 1. Thereafter a 6-monthly dosing schedule is planned. After each dosing subjects will be assessed for safety and tolerability at follow up visits.
March 23, 2021 is the study start date. The indicative completion of the clinical trial will be expected in December 2023.
Among primary outcome measures are the Incidence and severity of ocular adverse events (AEs) and Incidence and severity of ocular adverse events (AEs).
The study will take place at the Universitair Ziekenhuis Gent (UZ), Ghent, Belgium; Justus-Liebig Universität - Department of Ophthalmology, Gießen, Germany; University of Tübingen - Institute for Ophthalmic Research, Tübingen, Germany; Amsterdam University Medica Center - Locatie AMC, Amsterdam, Netherlands; Moorfields Eye Hospital - NHS Foundation Trust, London, United Kingdom.
The page dedicated to this clinical trial that provides more details on inclusion and exclusion criteria can be found here: https://ichgcp.net/clinical-trials-registry/NCT04855045.
