Phase II Clinical Trial to Evaluate the Efficacy and Safety of SYH2070 Injection in Participants With Homozygous Familial Hypercholesterolemia

NCT07591298 | Not Yet Recruiting Phase 2

• 1 other identifier: SYH2070-003
• Study Type: interventional
• Target: 18
• Locations: 0 countries
• Sites: N/A

Brief 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.

Conditions

Diagnosed With HoFH

Outcome Measures

Primary Outcomes (1)

• The percentage change in serum LDL-C level from baseline

  week 24

Study Arms (2)

SYH2070 injection dose1

EXPERIMENTAL

SYH2070 injection dose2

Drug: SYH2070 injection dose1

SYH2070 injection dose2

EXPERIMENTAL

SYH2070 injection

Drug: SYH2070 injection dose2

Interventions

SYH2070 injection dose1 DRUG

The patient will receive a treatment period of 48weeks

SYH2070 injection dose1

SYH2070 injection dose2 DRUG

The patient will receive a treatment period of 48weeks

SYH2070 injection dose2

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.);
• 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;

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 2
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: This trial is a multicenter, open-label, phase II clinical trial.

Study Record Dates

• First Submitted: May 9, 2026
• First Posted: May 15, 2026
• Study Start (Estimated): May 24, 2026
• Primary Completion (Estimated): September 16, 2027
• Study Completion (Estimated): September 30, 2027
• Last Updated: May 15, 2026

Record last verified: 2026-05