HoFH Adult Patient Recruitment | SYH2070 Injection (Double-Stranded Small Interfering RNA Drug) Phas
Dear patients with homozygous familial hypercholesterolemia (HoFH),
Hello everyone!
On May 11, 2026, a [document/report/etc.] was released.A multicenter, open-label phase II clinical trial evaluating the efficacy and safety of SYH2070 injection in Chinese participants with homozygous familial hypercholesterolemia.Public NoticeThis clinical trial was applied for by CSPC Zhongnuo Pharmaceutical (Shijiazhuang) Co., Ltd. and CSPC Zhongqi Pharmaceutical Technology (Shijiazhuang) Co., Ltd.The aim is to evaluate the efficacy of SYH2070 injection in...HoFHThe effectiveness of LDL-C was reduced among participants.
Image source: [Source Name]Drug Clinical Trial Registration and Information Disclosure Platform
To help everyone understand the details of this clinical trial, we have compiled the key information as follows:
Registration Number:CTR20261813
Experiment title:A multicenter, open-label phase II clinical trial evaluating the efficacy and safety of SYH2070 injection in Chinese participants with homozygous familial hypercholesterolemia.
Research drug:SYH2070 Injection
Drug Introduction:SYH2070 injection (double-stranded small interfering RNA (siRNA) drug), a Class 1 new chemical drug independently developed by CSPC Pharmaceutical Group, was approved by the National Medical Products Administration of the People's Republic of China on September 30, 2025, and can be used for clinical trials in China.
SYH2070 is a liver-targeted siRNA drug delivered via conjugation to acetylgalactosamine (GalNAc). It targets angiopoietin-like protein 3 (ANGPTL3) via subcutaneous administration, effectively reducing ANGPTL3 levels and lowering LDL-C independently of the LDLR receptor pathway.
Drug administration route:Subcutaneous injection, administered once every 12 weeks, for a total of 4 administrations.
Advances in drug researchThe Phase I clinical trial is being conducted on healthy participants with dyslipidemia. There are five dose groups in total. Enrollment for the highest dose group has been completed and follow-up is underway.
Existing research results show that: 1) Safety: The overall safety of the five dose groups is good, with no grade 3 or higher adverse events and no SAEs; 2) Efficacy: A certain ability of SYH2070 to reduce LDL-C has been observed in healthy participants with dyslipidemia.
Target number of participants:Plan to recruit18A HoFH patient.
1) When signing the ICFAge ≥ 18 years oldGender not limited;
2) Genetic or clinical diagnosis confirms homozygous familial hypercholesterolemia:
a.Genetic diagnostic criteria:Genetic testing at the LDL receptor, ApoB, PCSK9, or LDL RAP1 loci identified two allele mutations: homozygous, compound heterozygous, and double heterozygous.or
b.Clinical diagnostic criteria:It meets the following criteria: ①+②+③ or ①+②+④:
① Serum LDL-C level: Untreated serum LDL-C ≥ 10 mmol/L, or treated serum LDL-C ≥ 8 mmol/L;
② The patient developed xanthomas on multiple sites of the skin, such as the buttocks, elbows, or tendons, before the age of 10;
③ Both parents had untreated LDL-C levels consistent with HeFH (LDL-C ≥ 4.7 mmol/L), or one parent had untreated LDL-C ≥ 10.0 mmol/L;
④ Family members have a history of early-onset ASCVD.
3) Before LDL-C testing during the screening periodShe has received stable and tolerable lipid-lowering therapy.a) Statins with or without other lipid-lowering drugs (e.g., cholesterol absorption inhibitors) maintained at a stable dose for ≥4 weeks and the dose is planned to remain unchanged during the study period; and/or b) PCSK9 monoclonal antibody for ≥8 weeks; and/or c) Lometab for ≥12 weeks.
4) Screening period inspectionFasting LDL-C≥2.6 mmol/L；
5) During screeningfasting TG≤5.6 mmol/L；
6) During screeningbody weight≥40 kg；
7) During screeningBMI< 40 kg/m2；
Participants must understand and cooperate in completing this trial, voluntarily participate in this trial, and sign the ICF.
1) Genetic diagnosisHeterozygous familial hypercholesterolemia;
2) Uncontrolled screeningOther diseases that affect blood lipids or lipoproteins(e.g., nephrotic syndrome, severe liver disease, glycogen storage disease, systemic lupus erythematosus, Cushing's syndrome, etc.) Researchers believe that these conditions may interfere with the accurate assessment of the validity of the study results.
3) Those who have received or are currently receiving LDL-C testing within 5 months prior to the screening period or within 5 half-lives (whichever is longer)Monoclonal antibodies targeting ANGPTL3。
4) Have you used any [products/services] within the 12 months prior to LDL-C testing during the screening period?ASO or siRNA drugs；
5) Use within the specified time limit before LDL-C testing during the screening period.Any of the following treatments:1) Mipomisen (within 5 months); 2) Bepadulic acid (within 4 weeks); 3) Lipid purification or plasma exchange (within 8 weeks); 4) Liver transplantation or CRISPR-based gene editing therapy (at any time);
6) Those who have received LDL-C testing within 5 months or 5 half-lives (whichever is longer) prior to the screening period must have received [treatment/treatment].In addition to lipid-lowering drugsDrug use that significantly affects LDL-C levels (e.g., oral or intravenous glucocorticoids, tacrolimus, cyclosporine, sirolimus, estrogens, vitamin A derivatives, antidepressants, etc.).
7) Currently in use during filteringMedications for the treatment of thyroid diseasesFurthermore, the patient had been using thyroid disease medication stably for less than 12 weeks prior to the LDL-C screening period.
8) Allergy or suspected allergy to oligonucleotide drugs or excipients used in investigational drugs;
9) Occurred within the preceding 180 days.Serious adverse cardiovascular events(e.g., myocardial infarction, stroke or cerebrovascular event, unstable angina, worsening heart failure or hospitalization).
10) Having uncontrolled (after drug or ablation treatment) or severe [condition] within 180 days prior to randomization.arrhythmia(e.g., atrial fibrillation with rapid ventricular rate, recurrent/persistent supraventricular or ventricular tachycardia);
11) Having NYHA Level III-IV status within the year prior to randomization or at the time of screeningHeart failureOr left ventricular ejection fraction <30%;
12) Type 1 diabetes was present at the time of screening, or type 2 diabetes was being used.hypoglycemic drugsAt that time, patients had been using hypoglycemic drugs stably for less than 12 weeks before LDL-C testing during the screening period;
13) Randomly selected within the previous 5 years or at the time of screening.History of malignant tumors(Except for cured basal cell carcinoma of the skin, etc.) or potential malignant tumors;
14) Had or planned to have undergone randomization within 180 days prior to randomization during the study period.Major surgery(Including CABG, etc.);
15) Within the year prior to the random selectionHistory of drug abuse or alcohol abuse;
16) Those who have received or plan to receive LDL-C testing within 90 days prior to the screening period or within 5 half-lives (whichever is longer) during the study period.Other clinical research treatments(Drugs or devices);
17) The screening period checks meet any of the following criteria:
a. SBP ≥ 160 mmHg, or DBP ≥ 100 mmHg (untreated or after drug stabilization treatment);
b. ALT or AST > 3.0 × ULN, or total bilirubin > 1.5 × ULN;
c.CK>2.5×ULN;
d. QTcF interval: males > 450 ms, females > 470 ms;
e.eGFR<30mL/min/1.73m2(CKD-EPI formula);
f. Positive for HBsAg and HBV DNA; or positive for HCV antibody and HCV RNA; or positive for either HIV antibody or anti-Treponema pallidum antibody.
g.TSH＜LLN，或TSH>1.5ULN；
h.HbA1c>9%；
18) Female participants of fertility were in the process of being screened.Pregnancy or lactationOr, the screening results showed a positive pregnancy test before randomization; or female or male participants of childbearing age had plans to conceive (including sperm or egg donation) and/or were unable to use effective contraception during the study period and 6 months after the end of treatment.
19) Other circumstances that the researchers deem unsuitable for participation in this trial (including poor compliance, etc.).
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Information source: Drug Clinical Trial Registration and Information Disclosure Platform, CSPC Pharmaceutical Group
