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Study identifier: NCT06125847 Synced from ClinicalTrials.gov · July 28, 2026
● Recruiting

NGGT006 Gene Therapy for Homozygous Familial Hypercholesterolemia

Condition: Homozygous Familial Hypercholesterolemia  ·  Sponsor: First Affiliated Hospital Xi'an Jiaotong University

PhaseEARLY_Phase 1
Planned participants12
Who can joinAll sexes, 12 Years to 55 Years
Healthy volunteersNo

About this study

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).

This description comes directly from the study's public registry record.

Talk to the study team

Tao Zheng, M.D.  ·  086-15229218127  ·  zhengtao900305@163.com

Ge Gao, M.M.  ·  086-18229068097  ·  gaoge1030@163.com

Always discuss trial participation with your own doctor first.

Locations (1)

First Affiliated Hospital of Xian Jiaotong UniversityXi'an, Shaanxi, ChinaRecruiting

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Source record: clinicaltrials.gov/study/NCT06125847