NCT07353398

Early-phase Study of ART002g1 Injection in HeFH: Safety, Tolerability and Preliminary Efficacy

Early-phase Clinical Study on Safety, Tolerability and Preliminary Efficacy of ART002g1 Injection in the Treatment of Heterozygous Familial Hypercholesterolemia

• Status: Not Yet Recruiting
• Phase: EARLY_Phase 1
• Study type: Interventional
• Enrollment: 24 (estimated)
• Sponsor: Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine · Academic / Other
• Sex: All
• Age: 18 Years – 70 Years
• Healthy volunteers: Not accepted

Summary

This study is an open-label, single ascending dose (SAD) study designed to evaluate the safety and tolerability of ART002g1 in patients with heterozygous familial hypercholesterolemia (HeFH) who require further reduction in low-density lipoprotein cholesterol (LDL-C). ART002g1 uses base editing technology, which is designed to interfere with the expression of the PCSK9 gene in the liver, thereby reducing the circulating levels of PCSK9 and LDL-C. The primary objectives of this study are to determine the safety and pharmacodynamic (PD) profiles of ART002g1 in this patient population.

Conditions

• Heterozygous Familial Hypercholesterolemia

Interventions

Type	Name	Description
DRUG	ART002g1 Injection	Intravenous (IV) infusion

Timeline

• Start date: 2026-03-11
• Primary completion: 2027-07-01
• Completion: 2027-07-01
• First posted: 2026-01-20
• Last updated: 2026-03-17

Locations

1 site across 1 country: China