NCT03913130

Extension Study to Study PQ-110-001 (NCT03140969)

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

• Status: Terminated
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 9 (actual)
• Sponsor: Laboratoires Thea · Industry
• Sex: All
• Age: 6 Years
• Healthy volunteers: Not accepted

Summary

Subjects completing participation in study PQ-110-001 (EudraCT 2017-000813-22 / NCT03140969) will be given the opportunity to enroll into the extension study for continued dosing if available data support current and/or future benefits for the subject. Study PQ-110-002 will provide long-term safety, tolerability, pharmacokinetic (PK), and efficacy data of QR-110.

Detailed description

Subjects completing participation in study PQ-110-001 (EudraCT 2017-000813-22 / NCT03140969) will be given the opportunity to enroll into the extension study for continued dosing if available data support current and/or future benefits for the subject. Subjects will be given the opportunity to enroll into this extension study for continued dosing if available data support current and/or future benefits for the subject. The Investigator, in consultation and agreement with the Medical Monitor, will decide on enrollment of each individual subject, as well as on dosing of the first treated eye and treatment initiation of the contralateral eye. Continued subject treatment in this study is desirable, but cannot be guaranteed, since it will depend on the risks and benefit of further treatment on a case-by-case basis, as discussed and agreed upon with the Medical Monitor. The contralateral eye and the first treated eye will be injected 3 months apart. The injection interval of 3 months between both eyes will limit burden for the subjects, with a 3 month-visit frequency during the course of the study. This between-eye interval could be adapted if safety data are supportive, and for logistic reasons, and in agreement with the Medical Monitor. The same safety monitoring protocol and efficacy assessments will apply to both eyes. QR-110 will be administered via intravitreal (IVT) injection.

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	QR-110	First treated eye: maintenance dose every 6 months, intravitreal administration Contralateral eye: loading dose followed by maintenance dose, every 6 months, intravitreal administration

Timeline

• Start date: 2019-05-13
• Primary completion: 2022-10-03
• Completion: 2022-10-03
• First posted: 2019-04-12
• Last updated: 2024-10-15
• Results posted: 2023-01-04

Locations

3 sites across 2 countries: United States, Belgium

Regulatory

• FDA-regulated drug study