Diagnosed With HoFHPhase 2NCT07591298
Phase II Clinical Trial to Evaluate the Efficacy and Safety of SYH2070 Injection in Participants With Homozygous Familial Hypercholesterolemia
A Phase 2 study testing SYH2070 injection dose1 and SYH2070 injection dose2 in people with Diagnosed With HoFH, run by CSPC Zhongnuo Pharmaceutical (Shijiazhuang) Co., Ltd. at 1 site in China.
Trial summary
This trial is a multicenter, open-label, phase II clinical study, aiming to evaluate the efficacy and safety of SYH2070 injection in participants with HoFH. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Diagnosed With HoFH
- What is being tested?
- SYH2070 injection dose1 and SYH2070 injection dose2
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 18 participants
- Where
- 1 siteChina
- Study dates
- 24 May 2026to 30 Sep 2027
- Phase
- Phase 2
- 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
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Age ≥18 years old, male or female, and weight ≥40 kg.
- Genetic diagnosis or clinical diagnosis of HoFH.
- Receiving stable and tolerable lipid-lowering treatment or other drugs for chronic disease treatment for certain periods before the study, and maintaining the stable -treatments throughout the study.
- Fasting serum LDL-C ≥2.6 mmol/L.
- Fasting TG during screening is ≤5.6 mmol/L
- BMI< 40 kg/m ² during screening
- Understand the study procedures, voluntarily participate, and sign the informed consent form.
Who cannot join
- The genetic diagnosis was for heterozygous familial hypercholesterolemia;
- During the screening process, the participant has uncontrolled other diseases that affect blood lipids or lipoproteins (such as nephrotic syndrome, severe liver diseases, glycogen storage disease, systemic lupus erythematosus, Cushing's syndrome, etc.) that the researchers believed would interfere with the accurate assessment of the study validity;
- The participant has received or is receiving monoclonal antibodies targeting ANGPTL3 within 5 months or 5 half-lives (whichever is longer) before the LDL-C test during the screening period;
- Within 12 months before the LDL-C test during the screening period, use of any ASO or siRNA type drugs;
- Those who used any of the following treatments within the specified time limit before the LDL-C test: 1) mipomersen (within 5 months); 2) bepatide acid (within 4 weeks); 3) lipid purification or plasma exchange (within 8 weeks); 4) liver transplantation or CRISPR-based gene editing treatment (at any time);
- During the screening period, within 5 months or 5 half-lives (whichever is longer) prior to the LDL-C test, the subject had received significant medications other than lipid-lowering drugs that affected LDL-C levels (such as oral or intravenous glucocorticoids, tacrolimus, cyclosporine, sirolimus, estrogens, vitamin A derivatives, antidepressants, etc.);
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- At the time of screening, the subject was using medications for thyroid disorders and the use of thyroid disorder medications was stable for less than 12 weeks before the LDL-C test;
- History of allergic or suspected allergic reactions to oligonucleotide drugs or excipients of investigational drugs;
- History of having a serious adverse cardiovascular event within 180 days before randomization (such as myocardial infarction, stroke or cerebrovascular event, unstable angina pectoris, deterioration of heart failure or hospitalization);
- History of having uncontrolled (after drug or ablation therapy) or severe arrhythmias (such as atrial fibrillation with rapid ventricular rate, recurrent or persistent supraventricular or ventricular tachycardia) within 180 days before randomization;
- History of having NYHA class Ⅲ-Ⅳ grade heart failure or left ventricular ejection fraction <30% within 1 year before randomization or at the time of screening;
- History of having type 1 diabetes at the time of screening, or had type 2 diabetes and was using hypoglycemic drugs, and the use of hypoglycemic drugs was stable for less than 12 weeks before the LDL-C test.
- History of malignancy (except for cured skin basal cell cancer, etc.) or potential malignancy within the previous 5 years before randomization or at the time of screening;
- Major surgery (including CABG, etc.) was performed within 180 days before randomization or was planned to be performed during the study period;
- History of drug abuse or alcohol abuse within 1 year before randomization;
- Within 90 days before LDL-C testing during the screening period or within 5 half-lives (whichever is longer), the participant has received or plans to receive other clinical research treatments (drugs or devices) during the study period;
- During the screening period, the participant met any of the following criteria:
- SBP ≥ 160 mmHg, or DBP ≥ 100 mmHg (untreated or after drug stabilization treatment); ALT or AST > 3.0 × ULN, or total bilirubin > 1.5 × ULN; CK > 2.5 × ULN; QTcF interval: male > 450 ms, female > 470 ms; eGFR < 30 mL/min/1.73 m2 (CKD-EPI formula, see Appendix 13.6); HBsAg positive and HBV DNA positive; or HCV antibody positive and HCV RNA positive; or positive for HIV antibody, anti-Neisseria meningitidis antibody; TSH < LLN, or TSH > 1.5 ULN; HbA1c > 9%;
- Female participants with reproductive capacity who were in pregnancy or lactation at the time of screening, or had a positive pregnancy test result before randomization; or male and female participants with reproductive capacity who had a fertility plan (including sperm donation, egg donation) and/or were unable to take effective contraceptive measures during the study period until after the end of treatment;
- Other situations considered by the investigator as not suitable for participating in this trial (including poor compliance, etc.).
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
The Second Xiangya Hospital
Changsha, Hunan, 410011
Daoquan Peng, PhD, 13974822567, pengdq@hotmail.com
About this study
Official title: A Multicenter, Open-label Phase II Clinical Trial Evaluating the Efficacy and Safety of SYH2070 Injection in Chinese Participants With Homozygous Familial Hypercholesterolemia
This trial is a multicenter, open-label, phase II clinical study, aiming to evaluate the efficacy and safety of SYH2070 injection in participants with HoFH.
Read the detailed description
This trial is a multicenter, open-label, phase II clinical trial aimed at evaluating the efficacy and safety of SYH2070 injection in participants with HoFH. Approximately 18 patients with HoFH who are receiving stable lipid-lowering treatment are planned to be enrolled. Avoid duplicating information that will be entered elsewhere, such as Eligibility Criteria or Outcome Measures.
- Conditions
- Diagnosed With HoFH
- Being tested
- SYH2070 injection dose1, SYH2070 injection dose2
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SYH2070 injection dose1Drug
The patient will receive a treatment period of 48weeks
SYH2070 injection dose2Drug
The patient will receive a treatment period of 48weeks
| Study group | Role | What they receive |
|---|---|---|
| SYH2070 injection dose1 | Experimental | SYH2070 injection dose2 Receives: Drug: SYH2070 injection dose1 |
| SYH2070 injection dose2 | Experimental | SYH2070 injection Receives: Drug: SYH2070 injection dose2 |
What is being measured
The primary outcome is the main question the study is designed to answer.
The percentage change in serum LDL-C level from baseline
Measured: week 24
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 May 2026
- Study start24 May 2026
- Primary completion16 Sep 2027 (estimated)
- Study completion30 Sep 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Diagnosed With HoFHStudies of the same condition, open and under way.
- More trials run by CSPC Zhongnuo Pharmaceutical (Shijiazhuang) Co., Ltd.The sponsor's other registered studies.
- 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 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- This trial is a multicenter, open-label, phase II clinical trial.
- Masking
- None (open label)
- Enrolment
- 18 participants (estimated)
Sponsor and investigators
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07591298
- Sponsor's protocol ID
- SYH2070-003
- Status last verified
- May 2026
- First submitted
- 9 May 2026
- Last updated
- 31 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 31 Jul 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multicenter, Open-label Phase II Clinical Trial Evaluating the Efficacy and Safety of SYH2070 Injection in Chinese Participants With Homozygous Familial Hypercholesterolemia
- ClinicalTrials.gov ID
- NCT07591298
- Acronym / sponsor ID
- SYH2070-003
- Conditions
- Diagnosed With HoFH
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 18 participants (estimated)
- Lead sponsor
- CSPC Zhongnuo Pharmaceutical (Shijiazhuang) Co., Ltd.Industry (pharma, biotech, devices)
- Study start
- 24 May 2026 (actual)
- Primary completion
- 16 Sep 2027 (estimated)
- Study completion
- 30 Sep 2027 (estimated)
- First posted
- 15 May 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.
This trial is a multicenter, open-label, phase II clinical study, aiming to evaluate the efficacy and safety of SYH2070 injection in participants with HoFH.
Detailed description
This trial is a multicenter, open-label, phase II clinical trial aimed at evaluating the efficacy and safety of SYH2070 injection in participants with HoFH. Approximately 18 patients with HoFH who are receiving stable lipid-lowering treatment are planned to be enrolled. Avoid duplicating information that will be entered elsewhere, such as Eligibility Criteria or Outcome Measures.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
- Model description
- This trial is a multicenter, open-label, phase II clinical trial.
| Arm | Type | Receives |
|---|---|---|
| SYH2070 injection dose1 | Experimental | Drug: SYH2070 injection dose1SYH2070 injection dose2 |
| SYH2070 injection dose2 | Experimental | Drug: SYH2070 injection dose2SYH2070 injection |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- Age ≥18 years old, male or female, and weight ≥40 kg.
- Genetic diagnosis or clinical diagnosis of HoFH.
- Receiving stable and tolerable lipid-lowering treatment or other drugs for chronic disease treatment for certain periods before the study, and maintaining the stable -treatments throughout the study.
- Fasting serum LDL-C ≥2.6 mmol/L.
- Fasting TG during screening is ≤5.6 mmol/L
Key exclusion criteria (20 listed)
- The genetic diagnosis was for heterozygous familial hypercholesterolemia;
- During the screening process, the participant has uncontrolled other diseases that affect blood lipids or lipoproteins (such as nephrotic syndrome, severe liver diseases, glycogen storage disease, systemic lupus erythematosus, Cushing's syndrome, etc.) that the researchers believed would interfere with the accurate assessment of the study validity;
- The participant has received or is receiving monoclonal antibodies targeting ANGPTL3 within 5 months or 5 half-lives (whichever is longer) before the LDL-C test during the screening period;
- Within 12 months before the LDL-C test during the screening period, use of any ASO or siRNA type drugs;
- Those who used any of the following treatments within the specified time limit before the LDL-C test: 1) mipomersen (within 5 months); 2) bepatide acid (within 4 weeks); 3) lipid purification or plasma exchange (within 8 weeks); 4) liver transplantation or CRISPR-based gene editing treatment (at any time);
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 |
|---|---|---|
| SYH2070 injection dose1The patient will receive a treatment period of 48weeks | Drug | SYH2070 injection dose1 |
| SYH2070 injection dose2The patient will receive a treatment period of 48weeks | Drug | SYH2070 injection dose2 |
Primary outcome
The percentage change in serum LDL-C level from baseline
Time frame: week 24
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 31 Jul 2026.
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 2 study testing SYH2070 injection dose1 and SYH2070 injection dose2 in people with Diagnosed With HoFH, 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 7 inclusion and 20 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 31 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. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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?
The percentage change in serum LDL-C level from baseline (time frame: week 24). 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 31 Jul 2026.