A Study to Evaluate the SYH2053 Injection in Patients With Heterozygous Familial Hypercholesterolemia (HeFH)

Recruiting now · Phase 3 · Has a placebo group

Conditions studied: Heterozygous Familial Hypercholesterolemia (HeFH)

In brief

Heterozygous Familial Hypercholesterolemia (HeFH) is an autosomal dominant disorder characterized by markedly elevated low-density lipoprotein cholesterol (LDL-C) and increased risk of atherosclerotic cardiovascular disease (ASCVD). This trial aims to evaluate the SYH2053 Injection in patients with HeFH.

Key facts

Study ID

NCT07465263

Run by

CSPC Zhongnuo Pharmaceutical (Shijiazhuang) Co., Ltd.

People needed

135

Starts

2026-03-31

Expected to finish

2028-05-31

Last updated by the study team

2026-07-08

Who can join

Age: 18 and older. Sex: any. Healthy volunteers: accepted.

You may qualify if…

• Male or female participants ≥18 years of age.
• HeFH.
• Stable moderate-to-high intensity statin therapy (± cholesterol absorption inhibitors) .
• Maintained a low-fat diet for ≥4 weeks before signing the ICF.
• Fasting LDL-C at screening ≥ 2.6 mmol/L or ≥ 1.4 mmol/L without ASCVD or with ASCVD.
• Fasting TG ≤5.6 mmol/L at screening.

You may not qualify if…

• HoFH or suspected HoFH.
• Use of medications that significantly affect LDL-C levels.
• Hypersensitivity or suspected allergy to oligonucleotide drugs or excipients of the investigational product.
• Major adverse cardiovascular events (MACE) within 180 days before signing the ICF; history of hemorrhagic stroke; or extreme-risk ASCVD at screening.
• Uncontrolled (by medication/ablation) or severe arrhythmias within 180 days before signing the ICF.
• NYHA Class III-IV heart failure or LVEF <40% within 1 year before signing ICF or at screening.
• Type 1 diabetes.
• Uncontrolled severe illness or conditions that may interfere with study results/increase risk at screening, according to investigator's judgment.
• History of malignancy or underlying malignancy within 5 years before signing ICF or at screening.
• Major surgery within 180 days before signing ICF or planned during the study.
• History of drug/alcohol abuse within 5 years before signing ICF.
• Participation in another clinical trial within 90 days or 5 half-lives (whichever is longer) before signing ICF, or planned during the study.
• Any of the following at screening:
• 1）SBP ≥160 mmHg or DBP ≥100 mmHg. 2）ALT/AST >3× ULN, or total bilirubin >1.5× ULN. 3）CK >2.5× ULN. 4）QTcF interval: >450 ms for male, >470 ms for female. 5）eGFR <30 mL/min/1.73 m². 6）HBsAg positive with HBV DNA positive; or HCV/syphilis/HIV antibody positive. 7）TSH<LLN, or TSH>ULN. 8）HbA1c >8.5%. 14.Pregnancy/lactation or planned parenthood, and/or without effective contraception for female and male participants of childbearing potential from the study to 3 months after the end of treatment.

Where it is running

• Peking University First Hospital — Beijing, Beijing Municipality, China (enrolling)

Full record on ClinicalTrials.gov

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