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

Study Status: RECRUITING
Recruit Status: RECRUITING
Study Type: INTERVENTIONAL

Official Title: 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 Co

Brief 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.

Detailed Summary:
Sponsor: HuidaGene Therapeutics Co., Ltd.

Current Primary Outcome:

Original Primary Outcome:

Current Secondary Outcome:

Original Secondary Outcome:

Information By: HuidaGene Therapeutics Co., Ltd.

Dates:
Date Received: May 09, 2023
Date Started: October 31, 2023
Date Completion: October 31, 2023
Last Updated: November 09, 2023
Last Verified: November 01, 2023