NCT03913143

A Study to Evaluate Efficacy, Safety, Tolerability and Exposure After a Repeat-dose of Sepofarsen (QR-110) in LCA10 (ILLUMINATE)

Double-masked, Randomized, Controlled, Multiple-dose Study to Evaluate Efficacy, Safety, Tolerability and Syst. Exposure of QR-110 in Leber's Congenital Amaurosis (LCA) Due to c.2991+1655A>G Mutation (p.Cys998X) in the CEP290 Gene

• Status: Active Not Recruiting
• Phase: Phase 2 / Phase 3
• Study type: Interventional
• Enrollment: 36 (actual)
• Sponsor: ProQR Therapeutics · Industry
• Sex: All
• Age: 8 Years
• Healthy volunteers: Not accepted

Summary

The purpose of this double-masked, randomized, controlled, multiple-dose study is to evaluate the efficacy, safety, tolerability and systemic exposure of sepofarsen (QR-110) administered via intravitreal injection in subjects with Leber's Congenital Amaurosis (LCA) due to the CEP290 p.Cys998X mutation after 24 months of treatment

Detailed description

The purpose of this double-masked, randomized, controlled, multiple-dose study is to evaluate the efficacy, safety, tolerability and systemic exposure of sepofarsen (QR-110) administered via intravitreal injection in subjects with Leber's Congenital Amaurosis (LCA) due to the CEP290 p.Cys998X mutation after 24 months of treatment. At study start subjects will be randomized to one of 3 treatment groups with either active study drug or sham treatment. Sepofarsen (QR-110) will be administered via intravitreal (IVT) injection into the subject's treatment eye (the subject's worse eye). Subjects in the sham-procedure group will undergo a procedure that will closely mimic the active injection. After each dosing subjects will be assessed for safety and tolerability at follow up visits. After the first eye has been treated for at least 12 months, treatment of the contralateral eye and cross-over of subjects assigned to sham procedure may be initiated in eligible eyes (in a masked manner) based on assessment of benefit/risk (including review of data from all clinical trials), and with concurrence of the Medical Monitor.

Conditions

• Leber Congenital Amaurosis 10
• Blindness
• Leber Congenital Amaurosis
• Vision Disorders
• Sensation Disorders
• Neurologic Manifestations
• Eye Diseases
• Eye Diseases, Hereditary
• Eye Disorders Congenital
• Retinal Disease

Interventions

Type	Name	Description
DRUG	sepofarsen	RNA antisense oligonucleotide for intravitreal injection
OTHER	Sham	Sham-Procedure (no experimental drug administered)

Timeline

• Start date: 2019-04-04
• Primary completion: 2022-01-31
• Completion: 2023-03-01
• First posted: 2019-04-12
• Last updated: 2022-03-17

Locations

14 sites across 9 countries: United States, Belgium, Brazil, Canada, France, Germany, Italy, Netherlands, United Kingdom

Regulatory

• FDA-regulated drug study