Safety and Tolerability Subretinal OPGx-001 for LCA5-Associated Inherited Retinal Degeneration (LCA5-IRD)

NCTID	NCT05616793 (View at clinicaltrials.gov)
Description	The goal of this clinical trial is to evaluate the safety and preliminary efficacy of subretinal gene therapy with OPGx-001 in patients with inherited retinal degeneration due to biallelic mutations in the LCA5 gene. Funding Source- FDA Office of Orphan Products Development (OOPD). (Show More)
Development Status	Active
Indication	Leber Congenital Amaurosis-Type 5
Disease Ontology Term	DOID:0110215
Compound Name	OPGx-LCA5
Compound Description	AAV8.hLCA5
Sponsor	Opus Genetics, Inc
Funder Type	Industry
Recruitment Status	Recruiting (Click here for study contact information)
Enrollment Count	15 (ESTIMATED)
Results Posted	Not Available

Target Gene/Variant	LCA5
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	AAV8
Editor Type	none
Dose 1	1E10 vg/eye (up to 300ul)
Dose 2	3E10 vg/eye
Dose 3	1E11 vg/eye
Dose 4
Dose 5

No.of Trial Sites	1
Locations	United States

Has US IND	True
FDA Designations	Orphan Drug Designation, Rare Pediatric Disease Designation, Regenerative Medicine Advanced Therapy
Recent Updates	First participant enrolled in run-in period for planned adaptive Phase 3 trial