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Study of Zodasiran in Adolescent Participants With Homozygous Familial Hypercholesterolemia

RecruitingPhase 3

Phase 3 Single-Arm Open-Label Study to Evaluate the Efficacy and Safety of Zodasiran in Adolescent Participants (Age 12 to <18 Years) With Homozygous Familial Hypercholesterolemia (SPRUCE)

Who can join

Ages 12 to 17 · All sexes

Full eligibility criteria
Inclusion Criteria:

* Adolescents 12 to \<18 years of age who are nonpregnant, nonlactating, and do not plan to become pregnant during the study
* Body weight ≥35 kilograms (kg) at screening
* HoFH based on a supportive genetic test (from a source-verifiable medical record or based on screening genotype) or clinical diagnosis
* Screening LDL-C ≥116 mg/dL (3 mmol/L)
* Screening hemoglobin A1c (HbA1c) ≤9.5%
* Total bilirubin \<2×upper limit of normal (ULN), unless in previously confirmed cases of Gilbert's syndrome
* Alanine aminotransferase or aspartate aminotransferase \<3×ULN

Exclusion Criteria:

* Use of a hepatocyte-targeted siRNA within 365 days before Day 1 (except inclisiran, which is permitted; administration of inclisiran and study drug must be separated by at least 4 weeks)
* Use of an antisense oligonucleotide molecule within 3 months before Day 1
* Use of evinacumab within 3 months before Day 1
* Use of systemic corticosteroids (unless used as replacement therapy for pituitary/adrenal disease with a stable regimen)

NOTE: Additional inclusion/exclusion criteria may apply per protocol

About the study

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.

What is being tested

Sponsor: Arrowhead Pharmaceuticals · Participants: 10 · Started: 2026-10

Contact the study team

Official record on ClinicalTrials.gov — NCT07473843

Locations in the U.S.

MissouriResearch Site, St Louis

Conditions

From ClinicalTrials.gov, data retrieved Nov 15, 2026. Each study sets its own eligibility; the study team decides who can join.