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.

An Open-Label, Dose Escalation and Double-Masked, Randomized, Controlled Study to Evaluate the Safety and Tolerability of Sepofarsen in Pediatric Subjects <8 Years of Age With Leber Congenital Amaurosis Type 10 (LCA10) Due to the c.2991 +1655A>G (p.Cys998X) Mutation.

• Status: Unknown
• Phase: Phase 2 / Phase 3
• Study type: Interventional
• Enrollment: 15 (estimated)
• Sponsor: ProQR Therapeutics · Industry
• Sex: All
• Age: 0 Years – 7 Years
• Healthy volunteers: Not accepted

Summary

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.

Detailed description

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.

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

Interventions

Type	Name	Description
DRUG	sepofarsen	RNA antisense oligonucleotide for intravitreal injection

Timeline

• Start date: 2021-03-23
• Primary completion: 2023-12-01
• Completion: 2023-12-01
• First posted: 2021-04-22
• Last updated: 2022-03-25

Locations

9 sites across 7 countries: Belgium, Brazil, Canada, Germany, Italy, Netherlands, United Kingdom