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
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.
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.
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 →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.
Exclusion Criteria:
EDP167 200mg (N=10)
Drug: EDP167
EDP167 300mg (N=10)
Drug: EDP167
EDP167 sc injection
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
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.
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
To evaluate the immunogenicity of EDP167.
The incidence and titer of antibodies against EDP167.
Time frame: Up to month 6 of the extension phase
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Homozygous Familial Hypercholesterolemia→
Eddingpharm (Zhuhai) Co., Ltd.