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NGGT006 Gene Therapy for Homozygous Familial Hypercholesterolemia

NCT06125847 · Status: RECRUITING · Phase: EARLY_PHASE1 · Type: INTERVENTIONAL · Enrollment: 12

Last updated 2024-12-09

No results posted yet for this study

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).

Conditions

  • Homozygous Familial Hypercholesterolemia

Interventions

GENETIC

NGGT006

Single intravenous infusion of NGGT006 at dose 1 (7.5e12vg/kg), dose 2 (1.5e13vg/kg) , dose 3 (3e13vg/kg) and dose 4 (4e13vg/kg).

Sponsors & Collaborators

  • First Affiliated Hospital Xi'an Jiaotong University

    lead OTHER

Principal Investigators

  • Zuyi Yuan, M.D. · First Affiliated Hospital of Xian Jiaotong University

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SEQUENTIAL

Eligibility

Min Age
12 Years
Max Age
55 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2023-10-29
Primary Completion
2024-11-30
Completion
2028-11-30

Countries

  • China

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT06125847 on ClinicalTrials.gov