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

NCT07353398 · Status: NOT_YET_RECRUITING · Phase: EARLY_PHASE1 · Type: INTERVENTIONAL · Enrollment: 24

Last updated 2026-03-17

No results posted yet for this study

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

DRUG

ART002g1 Injection

Intravenous (IV) infusion

Sponsors & Collaborators

  • Accuredit Therapeutics US Limited

    collaborator INDUSTRY

  • Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine

    lead OTHER

Principal Investigators

  • Xueying Ding, MD · Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine

  • Rong Jiang, MD · Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SEQUENTIAL

Eligibility

Min Age
18 Years
Max Age
70 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2026-03-11
Primary Completion
2027-07-31
Completion
2027-07-31

Countries

  • China

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT07353398 on ClinicalTrials.gov