Heterozygous Familial Hypercholesterolemia (HeFH)Phase 3NCT07465263
A Study to Evaluate the SYH2053 Injection in Patients With Heterozygous Familial Hypercholesterolemia (HeFH)
A Phase 3 study testing SYH2053 Injection in people with Heterozygous Familial Hypercholesterolemia (HeFH), run by CSPC Zhongnuo Pharmaceutical (Shijiazhuang) Co., Ltd. at 1 site in China.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and olderHealthy volunteers can take part
- Conditions the study is for
- Heterozygous Familial Hypercholesterolemia (HeFH)
- What is being tested?
- SYH2053 Injection
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 135 participants
- Where
- 1 siteChina
- Study dates
- 31 Mar 2026to 31 May 2028
- Phase
- Phase 3
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- Healthy volunteers accepted
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
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- 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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Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site, 1 recruiting
- Recruiting
Peking University First Hospital
Beijing, Beijing Municipality, 100034
Jianping Li, PhD, 010-66119025, lijianping03455@pkufh.com
About this study
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
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 the 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.
- Being tested
- SYH2053 Injection, Placebo Injection
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SYH2053 InjectionDrug
Administered by subcutaneous injection.
Placebo InjectionDrug
Administered by subcutaneous injection. The placebo is identical to SYH2053 Injection in appearance.
| Study group | Role | What they receive |
|---|---|---|
| SYH2053 | Experimental | Not described Receives: Drug: SYH2053 Injection |
| Placebo | Placebo comparator | Not described Receives: Drug: Placebo Injection |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percentage Change from Baseline in Low-Density Lipoprotein Cholesterol (LDL-C) at Day 330
Fasting LDL-C is measured by a central laboratory.
Measured: Baseline and Day 330
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Mar 2026
- Study start31 Mar 2026
- Primary completion28 Feb 2028 (estimated)
- Study completion31 May 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Heterozygous Familial Hypercholesterolemia (HeFH)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- Parallel Assignment
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 135 participants (estimated)
Sponsor and investigators
- Lead sponsor
- CSPC Zhongnuo Pharmaceutical (Shijiazhuang) Co., Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07465263
- Sponsor's protocol ID
- SYH2053-006
- Status last verified
- Jul 2026
- First submitted
- 6 Mar 2026
- Last updated
- 8 Jul 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No