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Clinical trial

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)

NCT05906953 is registered in ClinicalTrials.gov with HuidaGene Therapeutics Co., Ltd. as lead sponsor.

NCT NCT05906953
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Registry record

As filed in ClinicalTrials.gov.

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 Congenital Amaurosis Type 2 (LCA2)
Lead sponsor
HuidaGene Therapeutics Co., Ltd.
Phase
Phase 1/2
Overall status
Recruiting
Start date
October 31, 2023
Completion date
December 31, 2025
Enrollment
20 (estimated)
Sites
3

Conditions

As the registry lists them; a condition with its own page is linked.

inherited retinal diseases caused by rpe65 mutations, leber congenital amaurosis