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
- Are there results?
- Not yet: the study is under way
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
Study at a glance
From the registry record, last updated by the sponsor on 8 Jul 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT07465263
- Acronym / sponsor ID
- SYH2053-006
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 135 participants (estimated)
- Lead sponsor
- CSPC Zhongnuo Pharmaceutical (Shijiazhuang) Co., Ltd.Industry (pharma, biotech, devices)
- Study start
- 31 Mar 2026 (actual)
- Primary completion
- 28 Feb 2028 (estimated)
- Study completion
- 31 May 2028 (estimated)
- First posted
- 11 Mar 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
- Model description
- Parallel Assignment
| Arm | Type | Receives |
|---|---|---|
| SYH2053 | Experimental | Drug: SYH2053 Injection |
| Placebo | Placebo comparator | Drug: Placebo Injection |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- 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.
Key exclusion criteria (15 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| SYH2053 InjectionAdministered by subcutaneous injection. | Drug | SYH2053 |
| Placebo InjectionAdministered by subcutaneous injection. The placebo is identical to SYH2053 Injection in appearance. | Drug | Placebo |
Primary outcome
Percentage Change from Baseline in Low-Density Lipoprotein Cholesterol (LDL-C) at Day 330
Time frame: Baseline and Day 330
Fasting LDL-C is measured by a central laboratory.
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Jul 2026.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
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.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 15 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 8 Jul 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Clinical Trials Information Group officer (0311-69085587, ctr-contact@cspc.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Percentage Change from Baseline in Low-Density Lipoprotein Cholesterol (LDL-C) at Day 330 (time frame: Baseline and Day 330). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 8 Jul 2026; AzTrials last refreshed its copy on 26 Oct 2026.