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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
This is an early phase 1, open-label, single-center, dose-escalation pilot trial to evaluate the safety and efficacy of an intravenous infusion of NGGT006 in patients with refractory Hypercholesterolemia diagnosed by gene testing for familial hypercholesterolemia. NGGT006 uses adeno-associated virus (AAV) as a vector, carrying a liver specific promoter and codon optimized human LDLR gene, driving the expression of LDLR protein with normal function and promoting the clearance of low-density lipoprotein cholesterol (LDL-C).
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Development Status
Active
Indication
Refractory Hypercholesterolemia, Familial Hypercholesterolemia
Disease Ontology Term
DOID:13810
Compound Name
NGGT006
Sponsor
Suzhou Municipal Hospital
Funder Type
Other
Recruitment Status
Not yet recruiting
Enrollment Count
9
Results Posted
Not Available
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
Sexes Eligible for Study
ALL
Locations
No.of Trial Sites
Locations
Regulatory Information
Has US IND
False
FDA Designations
Recent Updates
Resources/Links
Preclinical Publications
(Abstract #1788) Rituximab Extends Duration of Efficacy of AAV-hLDLR in Reducing LDL-C in Cynomolgus Monkeys - ASGCT 2024
(Abstract #1009) NGGT006, an Advanced Gene Therapy Vector for Treating Familial Hypercholesterolemia - ASGCT 2024
Related NCTID
Early Phase 1: NCT06125847