SYH2053 Injection for adults with heterozygous familial hypercholesterolemia

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This multicenter, randomized, double-blind, placebo-controlled Phase III trial evaluates the efficacy, safety, and immunogenicity of SYH2053 Injection in adults with heterozygous familial hypercholesterolemia who are on stable statin therapy and a low-fat diet. Participants undergo a screening period followed by a treatment period, receiving either SYH2053 or placebo injections. The study assesses lipid outcomes alongside safety data to determine if SYH2053 can provide additional LDL-C lowering on top of background therapy. Key eligibility includes confirmed HeFH, LDL-C above threshold, and absence of major recent cardiovascular events or severe cardiovascular conditions. Lead sponsor is CSPC Zhongnuo Pharmaceutical, with the trial conducted at Peking University First Hospital in Beijing.

Why it matters

Who can join: the full rules

Inclusion Criteria:

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

Exclusion Criteria:

1. HoFH or suspected HoFH.
2. Use of medications that significantly affect LDL-C levels.
3. Hypersensitivity or suspected allergy to oligonucleotide drugs or excipients of the investigational product.
4. Major adverse cardiovascular events (MACE) within 180 days before signing the ICF; history of hemorrhagic stroke; or extreme-risk ASCVD at screening.
5. Uncontrolled (by medication/ablation) or severe arrhythmias within 180 days before signing the ICF.
6. NYHA Class III-IV heart failure or LVEF \<40% within 1 year before signing ICF or at screening.
7. Type 1 diabetes.
8. Uncontrolled severe illness or conditions that may interfere with study results/increase risk at screening, according to investigator's judgment.
9. History of malignancy or underlying malignancy within 5 years before signing ICF or at screening.
10. Major surgery within 180 days before signing ICF or planned during the study.
11. History of drug/alcohol abuse within 5 years before signing ICF.
12. Participation in another clinical trial within 90 days or 5 half-lives (whichever is longer) before signing ICF, or planned during the study.
13. 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.

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Where it's running

• Peking University First Hospital
  Beijing, Beijing Municipality, China Recruiting

Study contacts

• Study coordinator: Clinical Trials Information Group officer
• Email: ctr-contact@cspc.cn
• Phone: 0311-69085587

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.