Not Yet RecruitingEarly Phase 1

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

China24 participantsStarted 2026-03-11

Plain-language 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.

Who can participate

Age range18 Years – 70 Years

SexALL

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Exclusion criteria

✕. Diagnosis of compound heterozygous FH, double heterozygous FH, or homozygous FH (HoFH);
✕. Positive for hepatitis B surface antigen (HBsAg), or positive for hepatitis B core antibody (HBcAb) with peripheral blood HBV DNA titer ≥ 1 × 10² copies/L; positive for hepatitis C virus (HCV) antibody with positive peripheral blood HCV RNA; positive for human immunodeficiency virus (HIV) antibody;
✕. Any unstable systemic disease, including but not limited to: unstable angina; cerebrovascular accident or transient ischemic attack (within 6 months prior to screening); myocardial infarction (within 6 months prior to screening); history of heart failure (NYHA Class II-IV); severe arrhythmia requiring pharmacotherapy; liver, kidney, or metabolic diseases; or other unstable systemic diseases as determined by the investigator;
✕. History of percutaneous transluminal coronary angioplasty (PTCA), percutaneous coronary intervention (PCI), or coronary artery bypass grafting (CABG) within 6 months prior to the first dose; or documented severe coronary artery stenosis as confirmed by coronary CT or coronary angiography within 90 days prior to randomization.

What they're measuring

Incidence of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs)

Timeframe: As of Week 48 (W48) post-administration of ART002g1 for Injection

Trial details

NCT IDNCT07353398

SponsorShanghai General Hospital, Shanghai Jiao Tong University School of Medicine

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2027-07

Contact for this trial

Xueying Ding, MD

+86-02136126102 dingxueying@126.com

View on ClinicalTrials.gov More Heterozygous Familial Hypercholesterolemia trials