Homozygous Familial Hypercholesterolemia (HoFH)Phase 2NCT07489209
A Dose-exploration Study of EDP167 in HoFH
A Phase 2 study testing EDP167 in people with Homozygous Familial Hypercholesterolemia (HoFH), run by Eddingpharm (Zhuhai) Co., Ltd. at 1 site in China.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Homozygous Familial Hypercholesterolemia (HoFH)
- What is being tested?
- EDP167
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- 1 siteChina
- Study dates
- 6 Feb 2026to 31 Mar 2027
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
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- 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).
- 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.
- Donated or lost blood ≥400 mL within 3 months prior to screening.
- Women who are pregnant, breastfeeding or planning for pregnancy.
- Other conditions that the Investigator would consider the subject is not suitable to participate in this study.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site, 1 recruiting
- Recruiting
Fuwai Hospital
Beijing
Ethics Committee of Fuwai Hospital, +86 010 88396281
About this study
Official title: A Multicenter, Dose-exploration, Open-label Phase II Study to Evaluate the Efficacy and Safety of EDP167 in Adult Patients With Homozygous Familial Hypercholesterolaemia
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.
- Being tested
- EDP167
- Keywords
- Dyslipidemia, ANGPTL3, siRNA, HoFH
- Medical subject headings
- Homozygous Familial Hypercholesterolemia, Dyslipidemias
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
EDP167Drug
EDP167 sc injection
| Study group | Role | What they receive |
|---|---|---|
| EDP167-200mg | Experimental | EDP167 200mg (N=10) Receives: Drug: EDP167 |
| EDP167-300mg | Experimental | EDP167 300mg (N=10) Receives: Drug: EDP167 |
What is being measured
The primary outcome is the main question the study is designed to answer.
To evaluate the change from baseline in serum low-density lipoprotein cholesterol (LDL-C) level at week 24 of the main study phase.
Measured: Up to week 24 of the main study phase
Secondary outcomes (13)
To evaluate the change from baseline in serum LDL-C level.
Measured: Up to month 6 of the extension phase
To evaluate the change from baseline in serum angiopioetin-like protein 3 (ANGPTL3) level.
Measured: Up to month 6 of the extension phase
To evaluate the change from baseline in serum triglyceride (TG) level.
Measured: Up to month 6 in the extension phase
To evaluate the change from baseline in serum total cholesterol (TC) level.
Measured: 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.
Measured: Up to month 6 in the extension phase
To evaluate the change from baseline in serum high-density lipoprotein cholesterol (HDL-C) level.
Measured: Up to month 6 in the extension phase
To evaluate the change from baseline in serum lipoprotein (a) [Lp (a)] level.
Measured: Up to month 6 in the extension phase
To evaluate the change from baseline in serum Apolipoprotein B (ApoB) level.
Measured: Up to month 6 in the extension phase
The proportion of subjects with serum LDL-C level <2.6mmol/L at week 24.
Measured: Up to week 24 of the main study phase
The proportion of subjects with serum LDL-C level decreased by ≥50% compared with the baseline at week 24.
Measured: Up to week 24 of the main study phase
The serum concentration of EDP167 over time.
Measured: Up to month 6 of the extension phase
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.
Measured: Up to month 6 of the extension phase
To evaluate the immunogenicity of EDP167.
The incidence and titer of antibodies against EDP167.
Measured: Up to month 6 of the extension phase
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Mar 2026
- Study start6 Feb 2026
- Primary completion4 Oct 2026 (estimated)
- Study completion31 Mar 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Homozygous Familial Hypercholesterolemia (HoFH)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 20 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Eddingpharm (Zhuhai) Co., Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Kefei Dou, MD, Principal investigator, Chinese Academy of Medical Sciences, Fuwai Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07489209
- Sponsor's protocol ID
- EDP167D201
- Status last verified
- May 2026
- First submitted
- 19 Mar 2026
- Last updated
- 20 May 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 20 May 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multicenter, Dose-exploration, Open-label Phase II Study to Evaluate the Efficacy and Safety of EDP167 in Adult Patients With Homozygous Familial Hypercholesterolaemia
- ClinicalTrials.gov ID
- NCT07489209
- Acronym / sponsor ID
- EDP167D201
- Keywords
- Dyslipidemia, ANGPTL3, siRNA, HoFH
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 20 participants (estimated)
- Lead sponsor
- Eddingpharm (Zhuhai) Co., Ltd.Industry (pharma, biotech, devices)
- Study start
- 6 Feb 2026 (actual)
- Primary completion
- 4 Oct 2026 (estimated)
- Study completion
- 31 Mar 2027 (estimated)
- First posted
- 24 Mar 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| EDP167-200mg | Experimental | Drug: EDP167EDP167 200mg (N=10) |
| EDP167-300mg | Experimental | Drug: EDP167EDP167 300mg (N=10) |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- 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.
Key exclusion criteria (19 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| EDP167EDP167 sc injection | Drug | EDP167-200mg, EDP167-300mg |
Primary outcome
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
13 secondary outcomes
To evaluate the change from baseline in serum LDL-C level.
Time frame: Up to month 6 of the extension phase
To evaluate the change from baseline in serum angiopioetin-like protein 3 (ANGPTL3) level.
Time frame: Up to month 6 of the extension phase
To evaluate the change from baseline in serum triglyceride (TG) level.
Time frame: Up to month 6 in the extension phase
To evaluate the change from baseline in serum total cholesterol (TC) level.
Time frame: 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.
Time frame: Up to month 6 in the extension phase
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
To evaluate the change from baseline in serum lipoprotein (a) [Lp (a)] level.
Time frame: Up to month 6 in the extension phase
To evaluate the change from baseline in serum Apolipoprotein B (ApoB) level.
Time frame: Up to month 6 in the extension phase
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
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
The serum concentration of EDP167 over time.
Time frame: Up to month 6 of the extension phase
To evaluate the safety and tolerability of EDP167 in subjects with HoFH.
Time frame: Up to month 6 of the extension phase
To evaluate the immunogenicity of EDP167.
Time frame: Up to month 6 of the extension phase
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 May 2026.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Kefei Dou, MD principal investigator
Chinese Academy of Medical Sciences, Fuwai Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing EDP167 in people with Homozygous Familial Hypercholesterolemia (HoFH), run by Eddingpharm (Zhuhai) Co., Ltd. at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 19 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 20 May 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Wei Song (86-13817957624, Sophie.Song@eddingpharm.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 20 May 2026.