Tests zodasiran injections in teenagers with a rare inherited high cholesterol disease
Tests zodasiran injections in teens with inherited homozygous familial hypercholesterolemia.
Plain-language title generated from the registry record
Registry title: Study of Zodasiran in Adolescent Participants With Homozygous Familial Hypercholesterolemia
Key facts
- Where
- St Louis, United StatesSee the site
- Phase
- Phase 3
Could this be for you?
Answer for yourself. This is not a decision: only the study team can say whether you can take part.
- Are you between 12 and 17 years old?
Source: NCT07473843, who can take part
Where in United States
- St LouisSt Louis, United StatesTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- Arrowhead Pharmaceuticals
- Trial code
- NCT07473843EU 2025-523662-24-00
- Record updated
- 26 October 2026
- Original description
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.Read full original descriptionShow less
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)
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.
Inclusion: 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: 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.