Clinical Trial: A Safety and Efficacy Study of HG004 in Subjects With Leber Congenital Amaurosis

Study Status: NOT_YET_RECRUITING
Recruit Status: NOT_YET_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: 2023-09
Date Completion: {'date': '2023-09', 'type': 'ESTIMATED'}
Last Updated: 2023-06-13
Last Verified: 2023-05