A Dose-exploration Study of EDP167 in HoFH

NCT07489209 | Recruiting Phase 2

• 1 other identifier: EDP167D201
• Study Type: interventional
• Target: 20
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

Homozygous Familial Hypercholesterolemia (HoFH)

Keywords

Dyslipidemia; ANGPTL3; siRNA; HoFH

Outcome Measures

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.

  Up to week 24 of the main study phase

Secondary Outcomes (13)

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

  Up to month 6 of the extension phase

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

  Up to month 6 of the extension phase

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

  Up to month 6 in the extension phase

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

  Up to month 6 in the extension phase

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

  Up to month 6 in the extension phase

Study Arms (2)

EDP167-200mg

EXPERIMENTAL

EDP167 200mg (N=10)

Drug: EDP167

EDP167-300mg

EXPERIMENTAL

EDP167 300mg (N=10)

Drug: EDP167

Interventions

EDP167 DRUG

EDP167 sc injection

EDP167-200mg; EDP167-300mg

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.
• Fasting serum LDL-C ≥2.6 mmol/L.
• Follow a daily low-fat diet during the study.
• 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.
• 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.
• Agree to use contraceptive measures that comply with regulations and the protocol requirements during the study, and until 6 months after the last dose.
• Understand the study procedures, voluntarily participate, and sign the informed consent form.

You may not qualify if:

• Allergic to the drug in this study, its components or similar drugs.
• Used any antisense oligonucleotide (ASO) or small interfering nucleic acid (siRNA) drugs within 12 months prior to randomization.
• 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.
• Received health supplements or other medications that have been used for lipid-lowering purposes within 4 weeks prior to screening.
• Received Lipoprotein apheresis within 8 weeks prior to screening.
• 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.
• Starting a new diet plan or having significant differences from the previous diet within 4 weeks prior to randomization.
• 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.
• 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.
• Performed coronary intervention within 6 months prior to randomization, or plan to perform coronary intervention during the study.
• Have a history of major surgery within 3 months prior to screening, or plan to undergo major surgery during the study.
• 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).
• 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.
• Have a history of current existence of alcohol or drug abuse per evaluation of the investigator.
• Uncontrolled hypertension at screening (blood pressure \>160/100 mmHg).

Sponsors & Collaborators

• Eddingpharm (Zhuhai) Co., Ltd.: lead

Study Sites (1)

Fuwai Hospital

Beijing, China

RECRUITING

MeSH Terms

Conditions

Homozygous Familial Hypercholesterolemia; Dyslipidemias

Condition Hierarchy (Ancestors)

Hyperlipoproteinemia Type II; Lipid Metabolism, Inborn Errors; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Hyperlipoproteinemias; Hyperlipidemias; Lipid Metabolism Disorders; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Officials

• Kefei Dou, MD

  Chinese Academy of Medical Sciences, Fuwai Hospital

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Wei Song

CONTACT

86-13817957624; Sophie.Song@eddingpharm.com

Study Design

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

Study Record Dates

• First Submitted: March 19, 2026
• First Posted: March 24, 2026
• Study Start: February 6, 2026
• Primary Completion (Estimated): October 4, 2026
• Study Completion (Estimated): March 31, 2027
• Last Updated: May 20, 2026

Record last verified: 2026-05

Locations

CN (1)