NCT07473843RecruitingPhase 3
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)
Source: ClinicalTrials.gov · Last updated 26 Oct 2026
Brief summary (registry, in English)
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.
- Status (registry)
- Recruiting
- Phase (registry)
- Phase 3
- Study type
- Interventional
- Enrollment
- 10 (estimated)
- First submitted
- 11 Mar 2026
- Start date
- Oct 2026
- Primary completion date
- Jul 2028
- Lead sponsor
- Arrowhead Pharmaceuticals
- Diseases
- Familial hypercholesterolaemia
- NCT number
- NCT07473843
- EU CT number
- 2025-523662-24-00
What you commit to
- Study type: Interventional · Phase 3
- Planned participants: 10
- From Oct 2026 to the primary completion date, Jul 2028
- 6 sites in 6 countries
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- 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
Other illness
- 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
Who the study is looking for
- 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
Age in the registry: 12 years to 17 years · Sex: All · Does not accept healthy volunteers
Sites (6)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Region Midtjylland Coordinator of this site? Confirm its status | Viborg | Denmark | Recruiting |
| Amsterdam UMC Research B.V. Coordinator of this site? Confirm its status | Amsterdam | Netherlands | Not yet recruiting |
| Oslo Universitetssykehus HF Coordinator of this site? Confirm its status | Oslo | Norway | Not yet recruiting |
| Medical Network Sp z o o Coordinator of this site? Confirm its status | Warsaw | Poland | Recruiting |
| Karolinska University Hospital Coordinator of this site? Confirm its status | Solna | Sweden | Not yet recruiting |
| - Coordinator of this site? Confirm its status | St Louis | United States | Recruiting |
This page is information, not medical advice. Discuss any trial with your doctor.