A Phase 1/2, Open-label, Multi-national, Multiple-cohort, Dose-escalation Study to Evaluate the Safety, Tolerability, and Efficacy of HG004 Gene Therapy in Subjects with RPE65-associated Leber Congenital Amaurosis Type 2 (LCA2)
Status: Recruiting
Location: See all (3) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 1/Phase 2
SUMMARY
The purpose of the study is to determine whether HG004 as gene therapy is safe and effective for the treatment of Leber Congenital Amaurosis caused by mutations in RPE65 gene.
Eligibility
Participation Requirements
Sex: All
Minimum Age: 6
Maximum Age: 50
Healthy Volunteers: f
View:
• Male or females between 6 and 50 years of age at the time of signing the informed consent form.
• Willing to adhere to protocol as evidenced by written informed consent or parental permission and subject assent.
• Clinical confirmed diagnosis of Leber congenital amaurosis (LCA) and molecular diagnosis of LCA due to RPE65 mutations.
• Ability to perform tests of visual and retinal function.
• Visual acuity of ≤ 20/80 or visual field less than 20 degrees in the eye to be injected.
• Acceptable hematology, clinical chemistry, and urine laboratory parameters.
Locations
United States
California
Research Site
RECRUITING
Sacramento
Texas
Research Site
RECRUITING
Houston
Other Locations
China
Research Site
RECRUITING
Shanghai
Contact Information
Primary
Study Director
HG00402@huidagene.com
732-318-9873
Time Frame
Start Date: 2023-10-31
Estimated Completion Date: 2025-12
Participants
Target number of participants: 20
Treatments
Experimental: HG004
Related Therapeutic Areas
Sponsors
Leads: HuidaGene Therapeutics Co., Ltd.