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RecruitingNCT07489209Updated May 20, 2026

A Dose-exploration Study of EDP167 in HoFH

A Phase 2 interventional study of EDP167 in Homozygous Familial Hypercholesterolemia (HoFH), sponsored by Eddingpharm (Zhuhai) Co., Ltd.. Recruiting at 1 site in China. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2026-05-20.

Sponsored by Eddingpharm (Zhuhai) Co., Ltd. · Phase 2, Interventional, and Treatment

From the registry’s dates

  • Started Feb 2026; still recruiting 8 months later.
Phase
Phase 2
Study type
Interventional
Enrollment
20
Allocation
Randomized
Ages
18 Years and older
Sex
All
01

Study summary

EDP167 is a double-stranded small interfering RNA (siRNA) drug targeting angiopoietin-like 3 protein (ANGPTL3), which may bring benefits for patients with dyslipidemia conditions. This is a dose exploration study in Homozygous Familial Hypercholesterolaemia (HoFH) patients to evaluate the efficacy and safety and pharmacokinetics (PK)/pharmacodynamics (PD) profiles of multiple EDP167 injections.

Read the detailed description

Angiopoietin-like 3 protein (ANGPTL3) is a key regulator of lipid metabolism. Clinical studies have shown that inhibition of ANGPTL3 could exert lipid-lowering effects in patients with dyslipidemia. EDP167 is a novel N-Acetylgalactosamine (GalNAc)-conjugated siRNA therapeutic that selectively silences hepatic ANGPTL3 mRNA expression, offering a promising strategy for lipid lowering. This trial includes two phase, main study phase (including screening period and 24 weeks treatment period) and extension phase (including 6 months treatment period). In main study phase, patients with HoFH will be randomized into two dose cohorts (200mg and 300mg, 10 subjects each), receiving EDP167 injections on Day 1 and at Week 12. At Week 24 of the main study phase, subjects will be evaluated and enter the extension phase, receiving EDP167 injections at Month 0 and at Month 3. The follow-up will last till Month 6 in extension phase to evaluate the efficacy, safety, PK profile and PD effects (ANGPTL3, low-density lipoprotein cholesterol [LDL-C], triglyceride [TG], and other lipid parameters) in HoFH patients after multiple EDP167 injections.

02

Conditions studied

  • Homozygous Familial Hypercholesterolemia (HoFH)

Keywords

  • Dyslipidemia
  • ANGPTL3
  • siRNA
  • HoFH
03

In context

Homozygous Familial Hypercholesterolemia

70 studies on the registry are indexed under Homozygous Familial Hypercholesterolemia; 12 are open to participants now.

This study's planned enrollment of 20 is close to the median of 20 across 51 interventional studies indexed under Homozygous Familial Hypercholesterolemia.

Browse Homozygous Familial Hypercholesterolemia studies →

Lead sponsor

Eddingpharm (Zhuhai) Co., Ltd. is the lead sponsor of 4 studies on the registry; 3 are open to participants now.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
18 Years and older
Sexes eligible
All
Accepts healthy volunteers
No

Inclusion criteria

  1. Age ≥18 years old, male or female, and weight ≥40 kg.
  2. Genetic diagnosis or clinical diagnosis of HoFH.
  3. Fasting serum LDL-C ≥2.6 mmol/L.
  4. Follow a daily low-fat diet during the study.
  5. Receiving stable and tolerable lipid-lowering treatment or other drugs for chronic diseases treatments for certain periods before the study, and maintain the stable treatments throughout the study.
  6. Women of childbearing potential (WOCBP) must have a negative serum pregnancy test at screening and a negative pregnancy test prior to receiving EDP167 at baseline.
  7. Agree to use contraceptive measures that comply with regulations and the protocol requirements during the study, and until 6 months after the last dose.
  8. Understand the study procedures, voluntarily participate, and sign the informed consent form.

Exclusion criteria

Exclusion Criteria:

  1. Allergic to the drug in this study, its components or similar drugs.
  2. Used any antisense oligonucleotide (ASO) or small interfering nucleic acid (siRNA) drugs within 12 months prior to randomization.
  3. Received treatment targeting ANGPTL3 or Apolipoprotein C3 (ApoC3), or have participated in other clinical trials within 6 months or 5 half-life (whichever longer) prior to screening.
  4. Received health supplements or other medications that have been used for lipid-lowering purposes within 4 weeks prior to screening.
  5. Received Lipoprotein apheresis within 8 weeks prior to screening.
  6. A weight change of >10% within 4 weeks prior to randomization, or planning to undergo weight-loss surgery or weight intervention treatment during the study period.
  7. Starting a new diet plan or having significant differences from the previous diet within 4 weeks prior to randomization.
  8. Presence of diseases that would affect lipid or lipoprotein levels, such as nephrotic syndrome, severe liver diseases, Cushing's syndrome, hypothyroidism or hyperthyroidism, etc., which are poorly controlled, and in the opinion of the Investigator will interfere with the accurate assessment of the study results.
  9. Had New York Heart Association (NYHA) grade III-IV heart failure within 12 months prior to randomization, or acute coronary syndrome or stroke within 6 months prior to randomization.
  10. Performed coronary intervention within 6 months prior to randomization, or plan to perform coronary intervention during the study.
  11. Have a history of major surgery within 3 months prior to screening, or plan to undergo major surgery during the study.
  12. History of malignancy, unless considered cured by adequate treatment with no evidence of recurrence for ≥3 years prior to the first dose of EDP167; excluding adequately treated basal cell or squamous cell carcinoma of the skin, carcinoma in situ of the cervix, carcinoma in situ of the breast, or incidental histological findings of prostate cancer (TNM stage T1a or T1b).
  13. Clinical evidence of active infections or other major or poorly controlled serious diseases, any other conditions that in the opinion of the Investigator may interfere with the study results or put the subjects at excessive risk.
  14. Have a history of current existence of alcohol or drug abuse per evaluation of the investigator.
  15. Uncontrolled hypertension at screening (blood pressure >160/100 mmHg).
  16. Subjects with any of the following laboratory abnormalities at screening: a) fasting serum TG≥5.6 mmol/L; b) Glycosylated hemoglobin A1C (HbA1c)>8.5%; c) alanine aminotransferase (ALT) or aspartate aminotransferase (AST) or gamma-glutamyl transpeptidase (GGT)>1.5×ULN (Upper Limit Of Normal), total bilirubin (TBIL)>2×ULN; d) prothrombin time (PT) or activated partial thromboplastin time (APTT) or International Normalized Ratio (INR) clinically significant abnormal; e) Hepatitis B surface antigen (HBsAg) or antibody to hepatitis C virus (HCVAb) or human immunodeficiency virus (HIV) positive; f) estimated glomerular filtration rate (eGFR)\<30 mL/min/1.73m2.
  17. Donated or lost blood ≥400 mL within 3 months prior to screening.
  18. Women who are pregnant, breastfeeding or planning for pregnancy.
  19. Other conditions that the Investigator would consider the subject is not suitable to participate in this study.
05

Study design

Phase
Phase 2
Primary purpose
Treatment
Allocation
Randomized
Intervention model
Parallel assignment
Masking
None (open label)
Enrollment
20 participants (estimated)

Study arms

  • Experimental
    EDP167-200mg

    EDP167 200mg (N=10)

    Drug: EDP167

  • Experimental
    EDP167-300mg

    EDP167 300mg (N=10)

    Drug: EDP167

Interventions

  • DrugEDP167

    EDP167 sc injection

06

What researchers measure

Primary outcomes

  1. To evaluate the change from baseline in serum low-density lipoprotein cholesterol (LDL-C) level at week 24 of the main study phase.

    Time frame: Up to week 24 of the main study phase

Secondary outcomes

  1. To evaluate the change from baseline in serum LDL-C level.

    Time frame: Up to month 6 of the extension phase

  2. To evaluate the change from baseline in serum angiopioetin-like protein 3 (ANGPTL3) level.

    Time frame: Up to month 6 of the extension phase

  3. To evaluate the change from baseline in serum triglyceride (TG) level.

    Time frame: Up to month 6 in the extension phase

  4. To evaluate the change from baseline in serum total cholesterol (TC) level.

    Time frame: Up to month 6 in the extension phase

  5. To evaluate the change from baseline in serum non-high-density lipoprotein cholesterol (non-HDL-C) level.

    Time frame: Up to month 6 in the extension phase

  6. To evaluate the change from baseline in serum high-density lipoprotein cholesterol (HDL-C) level.

    Time frame: Up to month 6 in the extension phase

  7. To evaluate the change from baseline in serum lipoprotein (a) [Lp (a)] level.

    Time frame: Up to month 6 in the extension phase

  8. To evaluate the change from baseline in serum Apolipoprotein B (ApoB) level.

    Time frame: Up to month 6 in the extension phase

  9. The proportion of subjects with serum LDL-C level <2.6mmol/L at week 24.

    Time frame: Up to week 24 of the main study phase

  10. The proportion of subjects with serum LDL-C level decreased by ≥50% compared with the baseline at week 24.

    Time frame: Up to week 24 of the main study phase

  11. The serum concentration of EDP167 over time.

    Time frame: Up to month 6 of the extension phase

  12. To evaluate the safety and tolerability of EDP167 in subjects with HoFH.

    Number of participants with adverse events and serious adverse events, with clinically significant changes in vital signs, in electrocardiogram readings, in physical examination, and in laboratory tests.

    Time frame: Up to month 6 of the extension phase

  13. To evaluate the immunogenicity of EDP167.

    The incidence and titer of antibodies against EDP167.

    Time frame: Up to month 6 of the extension phase

07

Study locations

1 of 1 sites recruiting
  • Fuwai Hospital
    Beijing, China
    • Ethics Committee of Fuwai Hospital · Contact · +86 010 88396281
    Recruiting
08

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on May 20, 2026, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
09

Registry details

Key details

Study ID
NCT07489209
Lead sponsor
Eddingpharm (Zhuhai) Co., Ltd.
Responsible party
Sponsor
First posted
Mar 24, 2026
Start date
Feb 6, 2026
Primary completion
Oct 4, 2026 (estimated)
Completion
Mar 31, 2027 (estimated)
Last update
May 20, 2026

Study contacts

Wei Song
Contact
Sophie.Song@eddingpharm.com
86-13817957624
Kefei Dou, MD
principal investigator · Chinese Academy of Medical Sciences, Fuwai Hospital

Oversight

Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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