A Study to Evaluate the SYH2053 Injection in Patients With Heterozygous Familial Hypercholesterolemia (HeFH)
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: SYH2053 Injection, Placebo Injection.
- Who it may be relevant to
- Registry conditions: Heterozygous Familial Hypercholesterolemia (HeFH). Basic parameters: from 18 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- China
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
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Official title
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
Overview
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.
Detailed description
This is a multicenter, randomized, double-blind, placebo-controlled Phase III clinical trial conducted in patients with heterozygous familial hypercholesterolemia.
It aims to evaluate the efficacy, safety, and immunogenicity of SYH2053 Injection when administered on the basis of background lipid-lowering therapy and a low-fat diet. The study consists of a screening period and a treatment period.
Interventions
- Drug SYH2053 Injection
Administered by subcutaneous injection. - Drug Placebo Injection
Administered by subcutaneous injection. The placebo is identical to SYH2053 Injection in appearance.
Primary outcome measures
- Percentage Change from Baseline in Low-Density Lipoprotein Cholesterol (LDL-C) at Day 330 [Time frame: Baseline and Day 330]
Eligibility criteria
Inclusion criteria
- 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 criteria
- 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.
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Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: Yes
Study design
- Allocation
- Randomized
- Model
- Parallel assignment
- Masking
- Quadruple blind
- Primary purpose
- Treatment
Study locations
China · 1 center
- Peking University First Hospital — Beijing
Identifiers
NCT: NCT07465263 · SYH2053-006