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

Starting soon · Early Phase 1

Conditions studied: Heterozygous Familial Hypercholesterolemia

In brief

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.

Key facts

Study ID
NCT07353398
Run by
Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine
People needed
24
Starts
2026-03-11
Expected to finish
2027-07-01
Last updated by the study team
2026-03-17

Who can join

Age: 18 and older, up to 70. Sex: any. Healthy volunteers: not accepted.

Where it is running

Full record on ClinicalTrials.gov

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