NCT03140969

Study to Evaluate QR-110 in Leber's Congenital Amaurosis (LCA) Due to the c.2991+1655A>G Mutation (p.Cys998X) in the CEP290 Gene

An Open-label, Multiple Dose, Dose Escalation Study to Evaluate the Safety and Tolerability of QR-110 in Subjects With Leber's Congenital Amaurosis (LCA) Due to c.2991+1655A>G Mutation (p.Cys998X) in the CEP290 Gene

• Status: Completed
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 11 (actual)
• Sponsor: Laboratoires Thea · Industry
• Sex: All
• Age: 6 Years
• Healthy volunteers: Not accepted

Summary

The purpose of this study is to evaluate the safety and tolerability of QR-110 administered via intravitreal injection in subjects with LCA due to the CEP290 p.Cys998X mutation.

Detailed description

The purpose of this study is to evaluate the safety and tolerability of QR-110 administered via intravitreal injection in subjects with LCA due to the CEP290 p.Cys998X mutation. Subjects will receive QR-110 in one eye every 3 months, for a maximum of 4 doses. Up to 3 dose levels of QR-110 will be evaluated.

Conditions

• Leber's Congenital Amaurosis

Interventions

Type	Name	Description
DRUG	QR-110	RNA antisense oligonucleotide for intravitreal injection

Timeline

• Start date: 2017-10-16
• Primary completion: 2019-10-02
• Completion: 2019-10-02
• First posted: 2017-05-04
• Last updated: 2024-10-15
• Results posted: 2022-12-27

Locations

3 sites across 2 countries: United States, Belgium

Regulatory

• FDA-regulated drug study