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

NCT07465263 | Not Yet Recruiting Phase 3

• 1 other identifier: SYH2053-006
• Study Type: interventional
• Target: 135
• Locations: 0 countries
• Sites: N/A

Brief Summary

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.

Conditions

Heterozygous Familial Hypercholesterolemia (HeFH)

Outcome Measures

Primary Outcomes (1)

• Percentage Change from Baseline in Low-Density Lipoprotein Cholesterol (LDL-C) at Day 330

  Fasting LDL-C is measured by a central laboratory.

  Baseline and Day 330

Study Arms (2)

SYH2053

EXPERIMENTAL

Drug: SYH2053 Injection

Placebo

PLACEBO COMPARATOR

Drug: Placebo Injection

Interventions

SYH2053 Injection DRUG

Administered by subcutaneous injection.

SYH2053

Placebo Injection DRUG

Administered by subcutaneous injection. The placebo is identical to SYH2053 Injection in appearance.

Placebo

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64), Older Adult (65+)

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:
• ）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.

Sponsors & Collaborators

• CSPC Zhongnuo Pharmaceutical (Shijiazhuang) Co., Ltd.: lead

Central Study Contacts

Clinical Trials Information Group officer

CONTACT

0311-69085587; ctr-contact@cspc.cn

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Parallel Assignment

Study Record Dates

• First Submitted: March 6, 2026
• First Posted: March 11, 2026
• Study Start: March 31, 2026
• Primary Completion (Estimated): February 28, 2028
• Study Completion (Estimated): May 31, 2028
• Last Updated: March 19, 2026

Record last verified: 2026-03