Clinical Trial

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

Not Yet Recruiting Early Phase 1
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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.
Protocol Amendment History 3 amendments
This ClinicalTrials.gov record has been amended 3 times since 2026-01-19; most recent amendment 2026-03-15.
Trial Details
NCT Number NCT07353398
Lead Sponsor Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine
Collaborators: Accuredit Therapeutics US Limited
Conditions Heterozygous Familial Hypercholesterolemia
Enrollment 24 participants
Start Date 2026-03-11
Primary Completion 2027-07 (estimated)
Study Completion 2027-07 (estimated)
Updated on ClinicalTrials.gov 2026-03-17