Tests SYH2053 injection in adults with familial high cholesterol
Tests SYH2053 injection against placebo in adults with inherited high cholesterol.
Plain-language title generated from the registry record
Registry title: A Study to Evaluate the SYH2053 Injection in Patients With Heterozygous Familial Hypercholesterolemia (HeFH)
Key facts
- Where
- Beijing, ChinaSee the site
- Phase
- Phase 3
Where in China
- Peking University First HospitalBeijing, ChinaTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- CSPC Zhongnuo Pharmaceutical (Shijiazhuang) Co., Ltd.
- Trial code
- NCT07465263
- Record updated
- 8 July 2026
- Original description
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.Read full original descriptionShow less
A Study to Evaluate the SYH2053 Injection in Patients With Heterozygous Familial Hypercholesterolemia (HeFH): A Multicenter, Randomized, Double-Blind, Placebo-Controlled Phase Ⅲ Clinical Trial
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.
Inclusion: 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.
Exclusion: 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; Major surgery within 180 days before signing ICF or planned during the study; 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; History of drug/alcohol abuse within 5 years before signing ICF; NYHA Class III-IV heart failure or LVEF <40% within 1 year before signing ICF or at screening; 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; -; HoFH or suspected HoFH; Participation in another clinical trial within 90 days or 5 half-lives (whichever is longer) before signing ICF, or planned during the study.