Actively Recruiting
A Multicenter, Dose-exploration Study to Evaluate the Safety and Efficacy of EDP167 in Adults With Homozygous Familial Hypercholesterolemia
Led by Eddingpharm (Zhuhai) Co., Ltd. · Updated on 2026-05-20
20
Participants Needed
1
Research Sites
25 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
This research aims to evaluate the effects of EDP167, a small interfering RNA drug targeting the angiopoietin-like 3 protein (ANGPTL3), in adults with Homozygous Familial Hypercholesterolemia (HoFH), a genetic condition causing very high cholesterol levels. By inhibiting ANGPTL3, EDP167 may help manage lipid levels in these patients. The study is a phase II, dose-exploration trial designed to assess the drug's safety, effectiveness, and how it behaves in the body (pharmacokinetics and pharmacodynamics). Participants will be randomly assigned to receive either 200 mg or 300 mg doses of EDP167 through subcutaneous injections. The main study phase includes a screening period and 24 weeks of treatment, with injections given on Day 1 and Week 12. After this, those who complete the main phase may enter a 6-month extension phase receiving injections at Month 0 and Month 3, followed by a 6-month follow-up to further monitor effects and safety. Throughout the study, participants will have their cholesterol levels and other lipid markers regularly measured, including LDL-C, triglycerides, total cholesterol, and ANGPTL3 levels. Safety and immune response to the drug will also be assessed. Participants are monitored to observe changes from baseline up to 6 months after treatment during the extension phase. The total participation duration includes initial screening, treatment periods, and follow-up visits to evaluate both short- and longer-term study outcomes.
CONDITIONS
Brief Title
A Dose-exploration Study of EDP167 in HoFH
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Age 18 years or older, male or female, weighing at least 40 kg
- Genetic or clinical diagnosis of Homozygous Familial Hypercholesterolemia (HoFH)
- Fasting serum LDL cholesterol level of 2.6 mmol/L or higher
- Following a daily low-fat diet during the study
- Receiving stable and tolerable lipid-lowering or other chronic disease treatments before and during the study
- Women of childbearing potential must have negative pregnancy tests before and during treatment
- Agree to use approved contraceptive methods during the study and for 6 months after the last dose
- Understand the study and voluntarily consent to participate
You will not qualify if you...
- Allergy to the study drug or similar drugs
- Use of antisense oligonucleotide or siRNA drugs within 12 months before randomization
- Treatment targeting ANGPTL3 or ApoC3, or participation in other clinical trials within 6 months or 5 half-lives prior to screening
- Use of lipid-lowering supplements or medications within 4 weeks before screening
- Lipoprotein apheresis within 8 weeks before screening
- Weight change over 10% in the 4 weeks before randomization or planned weight-loss surgery during the study
- Starting a new diet or significant diet changes within 4 weeks before randomization
- Poorly controlled diseases affecting lipid levels, such as nephrotic syndrome, severe liver disease, Cushing's syndrome, or thyroid disorders
- Recent severe heart conditions or interventions within specified timeframes before randomization
- Major surgery within 3 months before screening or planned during the study
- History of cancer except certain treated skin and cervical cancers or low-stage prostate cancer without recurrence for 3 years
- Active infections or serious uncontrolled diseases
- History of alcohol or drug abuse
- Uncontrolled high blood pressure (above 160/100 mmHg)
- Specific abnormal lab values including high triglycerides, elevated liver enzymes, abnormal coagulation tests, positive viral markers, or low kidney function
- Blood donation or loss of 400 mL or more within 3 months before screening
- Pregnant, breastfeeding, or planning pregnancy
- Other conditions deemed unsuitable by the investigator
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - 24 weeks
Participants receive EDP167 injections at Day 1 and Week 12 during the 24-week main study phase to evaluate safety and efficacy.
2 injection visits (in-person)
Duration - 6 months
Participants continuing into the extension phase receive EDP167 injections at Month 0 and Month 3 to further evaluate treatment effects.
2 injection visits (in-person)
Duration - 3 months
Participants are monitored for efficacy, safety, pharmacokinetics, and pharmacodynamics for 3 months after the last extension phase injection.
Multiple visits during follow-up
Trial Site Locations
Total: 1 location
1
Fuwai Hospital
Beijing, China
Actively Recruiting
Research Team
W
Wei Song
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
2
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