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Gene Therapy Trial Report

Summary

A Gene Therapy Study for Homozygous Familial Hypercholesterolemia (HoFH)

NCTID NCT02651675 (View at clinicaltrials.gov)
Description
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 (ACTUAL)
Results Posted View Results

Therapy Information

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 (n=3)
Dose 2 7.5E12 GC/kg (n=6)
Dose 3
Dose 4
Dose 5

Study Record Dates

Current Stage Phase1, Phase2
Submit Date 2016-01-04
Completion Date 2020-11-27
Last Update 2023-07-13

Participation Criteria

Eligible Age >=18 Years
Standard Ages Adult, Older adult
Sexes Eligible for Study ALL

Locations

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

Regulatory Information

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

Resources/Links

News and Press Releases
Preclinical Publications