NCT06088992

Leber Congenital Amaurosis Inherited Blindness of Gene Therapy Trial(LIGHT)

An Investigator-Initiated Open-Label, Multiple-Dose Clinical Study to Evaluate the Safety,Tolerability, and Efficacy of Gene Therapy for 2Leber's Congenital Amaurosis with RPE65 Mutation (LCA2)

• Status: Active Not Recruiting
• Phase: EARLY_Phase 1
• Study type: Interventional
• Enrollment: 9 (estimated)
• Sponsor: Xinhua Hospital, Shanghai Jiao Tong University School of Medicine · Academic / Other
• Sex: All
• Age: 8 Years – 50 Years
• Healthy volunteers: Not accepted

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 mutationsin RPE65 gene.

Conditions

• Leber Congenital Amaurosis

Interventions

Type	Name	Description
GENETIC	HG004	Method of Administration: Once unilateralsubretinal injection; The duration of the study isabout 60 weeks for each subject including a 8-weekscreening period, enrollment/baseline visit,treatment visit, and 52 weeks follow-up period.

Timeline

• Start date: 2023-01-10
• Primary completion: 2024-10-30
• Completion: 2028-10-30
• First posted: 2023-10-18
• Last updated: 2024-09-19

Locations

1 site across 1 country: China