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Clinical Trial Report

Gene Therapy Trial Report

Summary

Safety and Efficacy Study of NGGT006 in Refractory Hypercholesterolemia Patients


NCTID NCT06293729 (View at clinicaltrials.gov)
Description
Indication Refractory Hypercholesterolemia, Familial Hypercholesterolemia
Compound Name NGGT006 (rAAV8-hLDLR)
Sponsor Suzhou Municipal Hospital
Funder Type Other
Status
Not yet recruiting
Enrollment Count 9

Therapy Information


Target Gene/Variant LDLR
Therapy Type Gene transfer
Therapy Route In-vivo
Mechanism of Action Overexpression of protective allele/gene
Route of Administration Intravenous
Drug Product Type Viral vector
Target Tissue/Cell
Delivery System Viral transduction
Vector Type AAV
Editor Type none
Dose 1 7.5E12 vg/kg
Dose 2 1.5E13 vg/kg
Dose 3 3E13 vg/kg
Dose 4
Dose 5

Study Record Dates


Current Stage Early phase1
Submit Date 2024-02-27
Completion Date 2029-03-01
Last Update 2024-04-17

Participation Criteria


Eligible Age 18 Years - 55 Years
Standard Ages Adult
Eligible Sex ALL

Locations


No.of Trial Sites
Locations

Regulatory Information


Has US IND False
Recent Updates

Resources/Links