NCT06293729

Safety and Efficacy Study of NGGT006 in Refractory Hypercholesterolemia Patients

A Clinical Study for the Safety and Efficacy of Intravenous Infusion of NGGT006 in Treatment of Refractory Hypercholesterolemia

• Status: Not Yet Recruiting
• Phase: EARLY_Phase 1
• Study type: Interventional
• Enrollment: 9 (estimated)
• Sponsor: Suzhou Municipal Hospital · Academic / Other
• Sex: All
• Age: 18 Years – 55 Years
• Healthy volunteers: Not accepted

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 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).

Detailed description

Familial Hypercholesterolemia caused by common genetic mutations can be divided into heterozygous hypercholesterolemia (HeFH, Heterozygous Familial Hypercholesterolemia) and homozygous hypercholesterolemia (HoFH, Homozygous Familial Hypercholesterolemia). Refractory hypercholesterolemia was defined as an LDL-C level of 70 mg/dL or greater, or 100 mg/dL or greater, for patients with or without clinical ASCVD, respectively. A large proportion of patients with familial hypercholesterolemia belong to refractory hypercholesterolemia. This is an early phase 1, open-label, single-center, dose-escalation pilot trial to evaluate the safety and efficacy of a single intravenous infusion of NGGT006 in patients with refractory Hypercholesterolemia diagnosed by gene testing for familial hypercholesterolemia. 3-9 subjects will be enrolled and divided into 3 groups according to the principle of dose escalation, respectively administered intravenous infusion of NGGT006 at low dose (7.5e12vg/kg), medium dose (1.5e13vg/kg) and high dose (3e13vg/kg). All subjects will undergo 52 weeks of treatment observation and further 260 weeks of long-term follow-up.

Conditions

• Refractory Hypercholesterolemia
• Familial Hypercholesterolemia

Interventions

Type	Name	Description
DRUG	NGGT006	Single intravenous infusion of NGGT006 at low dose (7.5e12vg/kg), medium dose (1.5e13vg/kg) and high dose (3e13vg/kg).

Timeline

• Start date: 2024-06-01
• Primary completion: 2025-03-31
• Completion: 2029-03-01
• First posted: 2024-03-05
• Last updated: 2024-04-17