A Gene Therapy Study for Homozygous Familial Hypercholesterolemia (HoFH)

NCTID	NCT02651675 (View at clinicaltrials.gov)
Description	This first-in-human study is intended to evaluate the safety and preliminary effectiveness of AAV (Adeno-associated virus)-based liver-directed gene therapy in the treatment of adults with Homozygous Familial Hypercholesterolemia (HoFH). (Show More)
Development Status	Inactive
Indication	Homozygous Familial Hypercholesterolemia (HoFH)
Disease Ontology Term	DOID:13810
Compound Name	RGX-501
Compound Description	AAV8.TBG.hLDLR
Sponsor	REGENXBIO Inc.
Funder Type	Industry
Recruitment Status	Terminated
Enrollment Count	9
Results Posted	View Results

Target Gene/Variant	LDLR
Therapy Type	Gene transfer
Therapy Route	In-vivo
Mechanism of Action	Functional gene replacement
Route of Administration	Intravenous
Drug Product Type	Viral vector
Target Tissue/Cell	Liver
Delivery System	Viral transduction
Vector Type	AAV8
Editor Type
Dose 1	2.5E12 GC/kg
Dose 2	7.5E12 GC/kg
Dose 3
Dose 4
Dose 5

No.of Trial Sites	9
Locations	Canada,Netherlands,United States,Italy

Has US IND	True
FDA Designations	Orphan Drug Designation
Recent Updates	Terminated by Sponsor for business reasons