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Not yet recruiting NCT07353398

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

Early Phase I Interventional Heterozygous Familial Hypercholesterolemia

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: ART002g1 Injection.
Who it may be relevant to
Registry conditions: Heterozygous Familial Hypercholesterolemia. Basic parameters: 18 years — 70 years · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
China
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

Official title

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

Overview

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.

Interventions

  • Drug ART002g1 Injection
    Intravenous (IV) infusion

Primary outcome measures

  • Incidence of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs) [Time frame: As of Week 48 (W48) post-administration of ART002g1 for Injection]
Secondary outcome measures (8)
  • Pharmacokinetic (PK) Assessments: PK Parameters of ART002g1: Tmax [Time frame: As of Week 2 (W2) post-administration of ART002g1 for Injection]
  • Pharmacodynamic (PD) Assessments: Serum PCSK9 protein [Time frame: As of Week 48 (W48) post-administration of ART002g1 for Injection]
  • Pharmacokinetic (PK) Assessments: PK Parameters of ART002g1: Cmax [Time frame: As of Week 2 (W2) post-administration of ART002g1 for Injection]
  • Pharmacokinetic (PK) Assessments: PK Parameters of ART002g1: AUC [Time frame: As of Week 2 (W2) post-administration of ART002g1 for Injection]
  • Pharmacokinetic (PK) Assessments: PK Parameters of ART002g1: t½ [Time frame: As of Week 2 (W2) post-administration of ART002g1 for Injection]
  • Pharmacokinetic (PK) Assessments: PK Parameters of ART002g1: CL [Time frame: As of Week 2 (W2) post-administration of ART002g1 for Injection]
  • Pharmacokinetic (PK) Assessments: PK Parameters of ART002g1:Vss [Time frame: As of Week 2 (W2) post-administration of ART002g1 for Injection]
  • Pharmacodynamic (PD) Assessments: Serum LDL-C [Time frame: As of Week 48 (W48) post-administration of ART002g1 for Injection]

Eligibility criteria

Subjects must meet all the following criteria to be eligible for enrollment:

  • Male or female, aged 18 to 70 years (inclusive) at the time of signing the Informed Consent Form (ICF);
  • Body weight between 45 and 90 kg (inclusive) at screening;
  • Definite diagnosis of heterozygous familial hypercholesterolemia (HeFH), meeting either of the following two criteria (1) or (2):

（1) HeFH diagnosed to be caused by mutations in the LDLR, APOB, or PCSK9 gene;

(2) Meeting 2 out of the 3 following criteria for adults per the Dutch Lipid Clinical Network (DLCN) criteria:

  • Serum LDL-C ≥ 4.7 mmol/L without prior lipid-lowering treatment;
  • Cutaneous or tendinous xanthomas, or arcus cornealis (in subjects < 45 years old);
  • Presence of FH or early-onset atherosclerotic cardiovascular disease (ASCVD) in first-degree relatives.

Subjects must not be enrolled if they meet any one or more of the following 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.

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation
Non-randomized
Model
Sequential
Masking
Open label
Primary purpose
Treatment

Study locations

China · 1 center
  • Shanghai General Hospital — Shanghai

Identifiers

NCT: NCT07353398 · IIT-ATMP-2026001

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗