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

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

Status

Not yet recruiting

Phases

Early Phase 1

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT07353398

Enrollment

Registered

2026-01-20

Start date

2026-03-11

Completion date

2027-07-01

Last updated

2026-03-17

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Heterozygous Familial Hypercholesterolemia

Keywords

ART002g1, LNP, HeFH

Brief 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.

Interventions

DRUGART002g1 Injection

Intravenous (IV) infusion

Study design

Allocation

NON_RANDOMIZED

Intervention model

SEQUENTIAL

Primary purpose

TREATMENT

Masking

NONE

Eligibility

Sex/Gender

ALL

Age

18 Years to 70 Years

Healthy volunteers

Inclusion criteria

Subjects must meet all the following criteria to be eligible for enrollment: 1. Male or female, aged 18 to 70 years (inclusive) at the time of signing the Informed Consent Form (ICF); 2. Body weight between 45 and 90 kg (inclusive) at screening; 3. 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: 1. Serum LDL-C ≥ 4.7 mmol/L without prior lipid-lowering treatment; 2. Cutaneous or tendinous xanthomas, or arcus cornealis (in subjects \< 45 years old); 3. 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

1. Diagnosis of compound heterozygous FH, double heterozygous FH, or homozygous FH (HoFH); 2. 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; 3. 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; 4. 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.

Design outcomes

Primary

Measure	Time frame
Incidence of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs)	As of Week 48 (W48) post-administration of ART002g1 for Injection

Secondary

Measure	Time frame
Pharmacokinetic (PK) Assessments: PK Parameters of ART002g1: Tmax	As of Week 2 (W2) post-administration of ART002g1 for Injection
Pharmacodynamic (PD) Assessments: Serum PCSK9 protein	As of Week 48 (W48) post-administration of ART002g1 for Injection
Pharmacokinetic (PK) Assessments: PK Parameters of ART002g1: Cmax	As of Week 2 (W2) post-administration of ART002g1 for Injection
Pharmacokinetic (PK) Assessments: PK Parameters of ART002g1: AUC	As of Week 2 (W2) post-administration of ART002g1 for Injection
Pharmacokinetic (PK) Assessments: PK Parameters of ART002g1: t½	As of Week 2 (W2) post-administration of ART002g1 for Injection
Pharmacokinetic (PK) Assessments: PK Parameters of ART002g1: CL	As of Week 2 (W2) post-administration of ART002g1 for Injection
Pharmacokinetic (PK) Assessments: PK Parameters of ART002g1:Vss	As of Week 2 (W2) post-administration of ART002g1 for Injection
Pharmacodynamic (PD) Assessments: Serum LDL-C	As of Week 48 (W48) post-administration of ART002g1 for Injection

Countries

China

Contacts

CONTACTXueying Ding, MD

dingxueying@126.com+86-02136126102

CONTACTRong Jiang, MD

listening39@163.com+86 13795493921

PRINCIPAL_INVESTIGATORXueying Ding, MD

Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine

PRINCIPAL_INVESTIGATORRong Jiang, MD