RecruitingEarly Phase 1NCT06125847

NGGT006 Gene Therapy for Homozygous Familial Hypercholesterolemia

A Clinical Study for the Safety and Efficacy of Intravenous Infusion of NGGT006 in Treatment of Homozygous Familial Hypercholesterolemia With LDLR Mutations

Sponsor

First Affiliated Hospital Xi'an Jiaotong University

Enrollment

21 participants

Start Date

Oct 29, 2023

Study Type

INTERVENTIONAL

Conditions

Homozygous Familial Hypercholesterolemia

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).

Eligibility

Min Age: 12 YearsMax Age: 55 Years

Plain Language Summary

Simplified for easier understanding

This clinical trial is studying NGGT006 for people with homozygous familial hypercholesterolemia. The study is currently recruiting participants at 1 location. People eligible for this study include aged 12 Years to 55 Years.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

GENETICNGGT006

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).

Locations(1)

First Affiliated Hospital of Xian Jiaotong University

Xi'an, Shaanxi, China

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NCT06125847

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