A Phase 3 interventional study of Zodasiran in Homozygous Familial Hypercholesterolemia, sponsored by Arrowhead Pharmaceuticals. Recruiting at 3 sites in 3 countries. Open to participants aged 12 Years to 17 Years. Per ClinicalTrials.gov, last updated 2026-10-26.
Sponsored by Arrowhead Pharmaceuticals · Phase 3, Interventional, and Treatment
This study will evaluate the efficacy and safety of zodasiran subcutaneous (sc) injection in participants 12 to \<18 years of age with genetically or clinically diagnosed homozygous familial hypercholesterolemia (HoFH) and low-density lipoprotein cholesterol (LDL-C) ≥116 milligrams per deciliter (mg/dL) on maximally tolerated lipid-lowering therapy.
68 studies on the registry are indexed under Homozygous Familial Hypercholesterolemia; 13 are open to participants now.
This study's planned enrollment of 10 is below the median of 21 across 51 interventional studies indexed under Homozygous Familial Hypercholesterolemia.
Browse Homozygous Familial Hypercholesterolemia studies →Arrowhead Pharmaceuticals is the lead sponsor of 50 studies on the registry; 9 are open to participants now.
Of its 24 completed or terminated interventional studies of FDA-regulated products, 12 (50%) have results posted.
Counted across the registry records on this site, refreshed daily.
The criteria were revised on the registry on Sep 30, 2026. See what changed
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
NOTE: Additional inclusion/exclusion criteria may apply per protocol
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
A blood test of average blood sugar over the past two to three months, given as a percentage or in mmol/mol. 6.5% (48 mmol/mol) or more is the usual threshold for diabetes, normally confirmed by a second test.
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
HbA1c
A blood test of average blood sugar over the past two to three months, given as a percentage or in mmol/mol. 6.5% (48 mmol/mol) or more is the usual threshold for diabetes, normally confirmed by a second test.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Bilirubin
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ULN (upper limit of normal)
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ALT and AST (liver enzymes)
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Corticosteroids (steroids)
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Drug: Zodasiran
By sc injection
Also known as: ARO-ANG3
Percent Change from Baseline to Month 12 in Fasting LDL-C
Time frame: Baseline, Month 12
Percent Change from Baseline to Month 12 in Fasting Apolipoprotein B (ApoB)
Time frame: Baseline, Month 12
Change from Baseline to Month 12 in Fasting ApoB
Time frame: Baseline, Month 12
Percent Change from Baseline to Month 12 in Fasting Non-High Density Lipoprotein Cholesterol (non-HDL-C)
Time frame: Baseline, Month 12
Change from Baseline to Month 12 in Fasting Non-HDL-C
Time frame: Baseline, Month 12
Change from Baseline to Month 12 in Fasting LDL-C
Time frame: Baseline, Month 12
Area Under the Plasma Concentration Versus the Time Curve (AUC) from Baseline to Month 12 for Fasting LDL-C
Time frame: Baseline, Month 12
Percent Change from Baseline to Month 12 in Fasting Triglycerides (TGs)
Time frame: Baseline, Month 12
Change from Baseline to Month 12 in Fasting TGs
Time frame: Baseline, Month 12
Percent Change from Baseline to Month 12 in Fasting Angiopoietin-like Protein 3 (ANGPTL3)
Time frame: Baseline, Month 12
Change from Baseline to Month 12 in Fasting ANGPTL3
Time frame: Baseline, Month 12
Percent Change from Baseline to Month 12 in Fasting Total Cholesterol
Time frame: Baseline, Month 12
Change from Baseline to Month 12 in Fasting Total Cholesterol
Time frame: Baseline, Month 12
Percent Change from Baseline to Month 12 in Fasting HDL-C
Time frame: Baseline, Month 12
Change from Baseline to Month 12 in Fasting HDL-C
Time frame: Baseline, Month 12
Number of Participants who Meet European Union (EU) LDL-C Apheresis Eligibility Criteria (per German Apheresis Working Group) at Month 12
Time frame: Month 12
Number of Participants who Meet United States (US) Apheresis Eligibility Criteria (per National Lipid Association) at Month 12
Time frame: Month 12
Number of Participants with Fasting LDL-C <100 mg/dL at Month 12
Time frame: Month 12
Percent Change from Baseline in Fasting LDL-C Over Time
Time frame: Baseline, up to Month 12
Change from Baseline in Fasting LDL-C Over Time
Time frame: Baseline, up to Month 12
Number of Participants with Treatment-Emergent Adverse Events (TEAEs)
Time frame: Up to 30.5 months
Plan to share: No
No publications or documents are linked to this record.
Start is estimated for Oct 2026; primary completion for Jul 2028, 21 months later.
Of 20 phase 3 interventional studies under Homozygous Familial Hypercholesterolemia, half reached primary completion within 22 months of starting (the median), counting those still running; a quarter within 16 and three quarters within 34. This study's planned 21 months is close to that.
Start moved once since we started tracking on Sep 25, 2026: Aug 2026 → Oct 2026, on Sep 30, 2026.
Earlier changes are on the record history on ClinicalTrials.gov ↗
How these figures are worked out, and which studies are left out of them.
From the registry record's own update history. This site started tracking changes on Sep 25, 2026; for anything earlier, see the record history on ClinicalTrials.gov ↗
Eligibility is decided by the study team. Share this record with your doctor or contact the team directly.
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Studies of the drug tested here: Zodasiran
Homozygous Familial Hypercholesterolemia →