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)
HuidaGene Therapeutics Co., Ltd.
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
- Age range
- 6–50 years
- Sex
- All
- Healthy volunteers
- No
Inclusion Criteria: * 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. Exclusion Crit…
Interventions
- DrugHG004
Low dose Medium dose High dose
Locations (3)
- Research SiteSacramento, California
- Research SiteHouston, Texas
- Research SiteShanghai, Shanghai Municipality