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

NCT07353398 | Not Yet Recruiting Early Phase 1

• 1 other identifier: IIT-ATMP-2026001
• Study Type: interventional
• Target: 24
• Locations: 1 country
• Sites: 1

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.

Conditions

Heterozygous Familial Hypercholesterolemia

Keywords

ART002g1; LNP; HeFH

Outcome Measures

Primary Outcomes (1)

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

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

Secondary Outcomes (8)

• 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

Study Arms (3)

Experimental: ART002g1 Injection - Dose Group 1 ( Single IV Infusion)

EXPERIMENTAL

Participants will receive a single dose of ART002g1.

Drug: ART002g1 Injection

Experimental: ART002g1 Injection - Dose Group 2 (Single IV Infusion)

EXPERIMENTAL

Participants will receive a single dose of ART002g1.

Drug: ART002g1 Injection

Experimental: ART002g1 Injection - Extended Dose Group ( Single IV Infusion)

EXPERIMENTAL

Participants will receive a single Optimal Biological Dose (OBD) of ART002g1, which is based on the results of the ascending dose escalation.

Drug: ART002g1 Injection

Interventions

ART002g1 Injection DRUG

Intravenous (IV) infusion

Experimental: ART002g1 Injection - Dose Group 1 ( Single IV Infusion); Experimental: ART002g1 Injection - Dose Group 2 (Single IV Infusion); Experimental: ART002g1 Injection - Extended Dose Group ( Single IV Infusion)

Eligibility Criteria

• Age: 18 Years - 70 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may not qualify if:

• 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.

Sponsors & Collaborators

• Accuredit Therapeutics US Limited: collaborator
• Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine: lead

Study Sites (1)

Shanghai General Hospital

Shanghai, China

Location

Study Officials

• Xueying Ding, MD

  Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine

  PRINCIPAL INVESTIGATOR

• Rong Jiang, MD

  Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Xueying Ding, MD

CONTACT

+86-02136126102; dingxueying@126.com

Rong Jiang, MD

CONTACT

+86 13795493921; listening39@163.com

Study Design

• Study Type: interventional
• Phase: early phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Associate Chief Physician

Study Record Dates

• First Submitted: January 12, 2026
• First Posted: January 20, 2026
• Study Start: March 11, 2026
• Primary Completion (Estimated): July 1, 2027
• Study Completion (Estimated): July 1, 2027
• Last Updated: March 17, 2026

Record last verified: 2026-03

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)