Safety Evaluation of Gene Therapy Drug in the Treatment of Primary Hypertriglyceridemic Patients with Recurrent Pancreatitis

NCTID	NCT05860569 (View at clinicaltrials.gov)
Description	The study will evaluate safety and tolerance of intravenous delivery of GC304 gene therapy drug as a treatment of primary hypertriglyceridemic patients with previous onset of acute pancreatitis. (Show More)
Development Status	Active
Indication	Hypertriglyceridemia
Disease Ontology Term	DOID:1172
Compound Name	GC304
Compound Description	scAAV5.liver promotor.coLPL-S447X
Sponsor	GeneCradle Inc
Funder Type	Industry
Recruitment Status	Recruiting
Enrollment Count	7
Results Posted	Not Available

Target Gene/Variant	GPIHBP1 or LPL
Therapy Type	Gene transfer
Therapy Route	In-vivo
Mechanism of Action	Functional gene replacement, overexpression of protective allele/gene
Route of Administration	Intravenous
Drug Product Type	Viral vector
Target Tissue/Cell
Delivery System	Viral transduction
Vector Type	AAV5
Editor Type
Dose 1	3.0E12 vg/kg
Dose 2	1.0E13 vg/kg
Dose 3	3.0E13 vg/kg
Dose 4
Dose 5

No.of Trial Sites	1
Locations	China

Has US IND	False
FDA Designations
Recent Updates	Recruitment started October 2024