Clinical Trial

NGGT006 Gene Therapy for Homozygous Familial Hypercholesterolemia

Recruiting Early Phase 1
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Summary
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 homozygous familial hypercholesterolemia (HoFH) patients with LDLR mutations. NGGT006 is an adeno-associated viral (AAV) vector carrying 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).
Protocol Amendment History 3 amendments
This ClinicalTrials.gov record has been amended 3 times since 2023-11-05; most recent amendment 2024-12-04.
Status change: Not Yet Recruiting → Recruiting 2024-03-05
Trial Details
NCT Number NCT06125847
Lead Sponsor First Affiliated Hospital Xi'an Jiaotong University
Conditions Homozygous Familial Hypercholesterolemia
Enrollment 12 participants
Start Date 2023-10-29
Primary Completion 2024-11-30 (estimated)
Study Completion 2028-11 (estimated)
Updated on ClinicalTrials.gov 2024-12-09