NCT05906953

Safety and Efficacy Trial of HG004 for Leber Congenital Amaurosis Related to Rpe65 Gene Mutations (STAR)

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
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 20 (estimated)
• Sponsor: HuidaGene Therapeutics Co., Ltd. · Industry
• Sex: All
• Age: 6 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 mutations in RPE65 gene.

Conditions

• Leber Congenital Amaurosis
• Inherited Retinal Diseases Caused by RPE65 Mutations

Interventions

Type	Name	Description
DRUG	HG004	Low dose Medium dose High dose

Timeline

• Start date: 2023-10-31
• Primary completion: 2025-12-01
• Completion: 2025-12-01
• First posted: 2023-06-18
• Last updated: 2024-09-19

Locations

3 sites across 2 countries: United States, China

Regulatory

• FDA-regulated drug study