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

A regiszterben nem jelölt feltételek

• 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.