Zodasiran for teens with homozygous familial hypercholesterolemia
Official title: 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)
Run by Arrowhead Pharmaceuticals · NCT07473843 · Data updated 2026-11-12
This study tests whether subcutaneous Zodasiran can help adolescents with HoFH who have very high LDL-C despite lipid-lowering therapy.
Quick facts
| Phase | Phase 3 |
|---|---|
| Study type | Interventional |
| Ages | 12 Years to 17 Years |
| Sex | All |
| Enrollment | 10 (estimated) |
| Sponsor | Arrowhead Pharmaceuticals Industry-sponsored |
| Drugs / interventions | evinacumab |
| Locations | 3 sites (St Louis, Missouri and 2 other locations) |
| Status | Recruiting |
| Started | October 2026 |
| Main results expected | July 2028 |
| Study ends | August 2028 |
| Registry record updated | October 26, 2026 |
| Trial ID | NCT07473843 on ClinicalTrials.gov |
Is this trial a fit for you?
✓Likely a good fit
Adolescents aged 12 to under 18 with HoFH and LDL-C ≥116 mg/dL on maximum lipid-lowering therapy, weight ≥35 kg, and appropriate genetic or clinical confirmation.
✕Probably not a fit
Patients with recent use of excluded therapies or with laboratory or medical exclusions may not benefit from the study intervention.
A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.
What this trial studies
This Phase 3 interventional trial evaluates the efficacy and safety of Zodasiran given by injection in adolescents aged 12 to under 18 with genetically or clinically diagnosed homozygous familial hypercholesterolemia and elevated LDL-C on maximal tolerated therapy. Participants must meet age, weight, genetic/clinical diagnosis, and lipid criteria, and have acceptable liver and metabolic safety labs. Exclusion criteria include recent exposure to certain RNA-targeted therapies and specific concurrent medications. The study is led by Arrowhead Pharmaceuticals with sites in St. Louis, Viborg, and Warsaw. Outcomes will inform whether Zodasiran can reduce LDL-C and maintain safety in this high-risk pediatric population.
Why it matters
Potential benefit: If successful, participants may experience lower LDL-C levels and improved cardiovascular risk profile.
How similar studies have gone: Previous RNA-targeted lipid therapies have shown potential in similar patient groups, but pediatric data for Zodasiran are limited and this study tests a novel approach.
Who can join: the full rules
Show full inclusion / exclusion 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
Where it's running
-
Research Site
St Louis, Missouri, United States Recruiting -
Research Site
Viborg, Denmark Recruiting -
Research Site
Warsaw, Poland Recruiting
Study contacts
- Study coordinator: Medical Monitor
- Email: SPRUCE@arrowheadpharma.com
- Phone: 626-304-3400
Similar studies
Other studies for the same condition or treatment. Recruiting studies are listed first.
-
Gene therapy for treating a rare cholesterol disorder
This study is testing a new gene therapy to see if it can help people with a rare cholesterol disorder called homozygous familial hypercholesterolemia lower their cholesterol levels.
-
Registry for patients with homozygous familial hypercholesterolemia
This study is creating a registry to collect health information from people with homozygous familial hypercholesterolemia to better understand this rare condition and improve future treatments.
-
A repository for monitoring health in patients with homozygous familial hypercholesterolemia
This study is trying to gather health information from people with homozygous familial hypercholesterolemia to better understand their condition and find ways to improve their treatment.
-
CTX310 for refractory high triglycerides or LDL cholesterol
This trial will test whether a single IV dose of CTX310 is safe and tolerated in adults whose high triglycerides or LDL cholesterol have not responded to available treatments.
-
EDP167 dose testing for homozygous familial hypercholesterolemia
This trial tests whether two injections of an siRNA drug called EDP167 can lower ANGPTL3 and improve lipid levels in adults with homozygous familial hypercholesterolemia (HoFH).
-
ORIGIN-FH: Newborn screening to detect familial high cholesterol
This project will test a genetic cheek swab of parents plus blood screening of newborns to find familial high cholesterol in babies born to parents with familial hypercholesterolemia.
How to take part
- Review the eligibility criteria above with your treating physician.
- Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
- Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.