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

NCTID	NCT05906953 (View at clinicaltrials.gov)
Description	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. (Show More)
Development Status	Active
Indication	Leber Congenital Amaurosis, Inherited Retinal Diseases Caused by RPE65 Mutations
Disease Ontology Term	DOID:0110016; DOID:0110353
Compound Name	HG004
Sponsor	HuidaGene Therapeutics Co., Ltd.
Funder Type	Industry
Recruitment Status	Recruiting
Enrollment Count	20
Results Posted	Not Available

Target Gene/Variant	RPE65
Therapy Type	Gene transfer
Therapy Route	In-vivo
Mechanism of Action	Functional gene replacement
Route of Administration	Subretinal
Drug Product Type	Viral vector
Target Tissue/Cell
Delivery System	Viral transduction
Vector Type	AAV9
Editor Type	none
Dose 1	Undisclosed low dose
Dose 2	Undisclosed medium dose
Dose 3	Undisclosed high dose
Dose 4
Dose 5

No.of Trial Sites	3
Locations	United States,China

Has US IND	True
FDA Designations	Orphan Drug Designation, Rare Pediatric Disease Designation
Recent Updates	First patient dosed November 2023