Home › Homozygous Familial Hypercholesterolemia › NCT07473843
Study of Zodasiran in Adolescent Participants With Homozygous Familial Hypercholesterolemia
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
- Zodasiran (drug)
Sponsor: Arrowhead Pharmaceuticals · Participants: 10 · Started: 2026-10
Contact the study team
- Medical Monitor · Phone: 626-304-3400
Official record on ClinicalTrials.gov — NCT07473843
Locations in the U.S.
| Missouri | Research Site, St Louis |
Conditions
- Homozygous Familial Hypercholesterolemia
- Hyperlipoproteinemia Type II
- Lipid Metabolism Disorders
- Inborn Errors Metabolism
- Genetic Disorders
- and Neonatal Diseases and Abnormalities Hereditary Congenital
- Hyperlipidemias
- Dyslipidemias
- Metabolic Disorders
From ClinicalTrials.gov, data retrieved Nov 15, 2026. Each study sets its own eligibility; the study team decides who can join.