Homozygous Familial HypercholesterolemiaPhase 3SPRUCENCT07473843
Study of Zodasiran in Adolescent Participants With Homozygous Familial Hypercholesterolemia
A Phase 3 study testing Zodasiran in people with Homozygous Familial Hypercholesterolemia, run by Arrowhead Pharmaceuticals at 2 sites in 2 countries.
Trial summary
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… Read more about this study
- Who is it for?
- Ages 12 to 17 years
- Conditions the study is for
- Homozygous Familial Hypercholesterolemia
- What is being tested?
- Zodasiran
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 2 sitesin 2 countries
- Study dates
- Oct 2026to Aug 2028
- Phase
- Phase 3
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 12 to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
2 sites in 2 countries. Contact the study team or a site near you to ask about taking part.
2 sites in 2 countries
United States1 site, 1 recruiting
- Recruiting
Research Site
St Louis, Missouri, 63110
About this study
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)
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.
- Conditions
- Homozygous Familial Hypercholesterolemia
- Being tested
- Zodasiran
- Keywords
- Hyperlipoproteinemia Type II, Lipid Metabolism, Inborn Errors, Metabolism, Inborn Errors, Genetic Diseases, Inborn, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Hyperlipoproteinemias, Hyperlipidemias, Dyslipidemias, Lipid Metabolism Disorders, Metabolic Diseases, Nutritional and Metabolic Diseases, Homozygous Familial Hypercholesterolemia, Zodasiran
- Medical subject headings
- Homozygous Familial Hypercholesterolemia, Hyperlipoproteinemia Type II, Lipid Metabolism, Inborn Errors, Metabolism, Inborn Errors, Genetic Diseases, Inborn, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Hyperlipoproteinemias, Hyperlipidemias
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ZodasiranDrug
Also called ARO-ANG3
By sc injection
| Study group | Role | What they receive |
|---|---|---|
| Zodasiran | Experimental | Not described Receives: Drug: Zodasiran |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percent Change from Baseline to Month 12 in Fasting LDL-C
Measured: Baseline, Month 12
Secondary outcomes (20)
Percent Change from Baseline to Month 12 in Fasting Apolipoprotein B (ApoB)
Measured: Baseline, Month 12
Change from Baseline to Month 12 in Fasting ApoB
Measured: Baseline, Month 12
Percent Change from Baseline to Month 12 in Fasting Non-High Density Lipoprotein Cholesterol (non-HDL-C)
Measured: Baseline, Month 12
Change from Baseline to Month 12 in Fasting Non-HDL-C
Measured: Baseline, Month 12
Change from Baseline to Month 12 in Fasting LDL-C
Measured: Baseline, Month 12
Area Under the Plasma Concentration Versus the Time Curve (AUC) from Baseline to Month 12 for Fasting LDL-C
Measured: Baseline, Month 12
Percent Change from Baseline to Month 12 in Fasting Triglycerides (TGs)
Measured: Baseline, Month 12
Change from Baseline to Month 12 in Fasting TGs
Measured: Baseline, Month 12
Percent Change from Baseline to Month 12 in Fasting Angiopoietin-like Protein 3 (ANGPTL3)
Measured: Baseline, Month 12
Change from Baseline to Month 12 in Fasting ANGPTL3
Measured: Baseline, Month 12
Percent Change from Baseline to Month 12 in Fasting Total Cholesterol
Measured: Baseline, Month 12
Change from Baseline to Month 12 in Fasting Total Cholesterol
Measured: Baseline, Month 12
Percent Change from Baseline to Month 12 in Fasting HDL-C
Measured: Baseline, Month 12
Change from Baseline to Month 12 in Fasting HDL-C
Measured: Baseline, Month 12
Number of Participants who Meet European Union (EU) LDL-C Apheresis Eligibility Criteria (per German Apheresis Working Group) at Month 12
Measured: Month 12
Number of Participants who Meet United States (US) Apheresis Eligibility Criteria (per National Lipid Association) at Month 12
Measured: Month 12
Number of Participants with Fasting LDL-C <100 mg/dL at Month 12
Measured: Month 12
Percent Change from Baseline in Fasting LDL-C Over Time
Measured: Baseline, up to Month 12
Change from Baseline in Fasting LDL-C Over Time
Measured: Baseline, up to Month 12
Number of Participants with Treatment-Emergent Adverse Events (TEAEs)
Measured: Up to 30.5 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Mar 2026
- Study startOct 2026 (estimated)
- Primary completionJul 2028 (estimated)
- Study completionAug 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Homozygous Familial HypercholesterolemiaStudies of the same condition, open and under way.
- More trials run by Arrowhead PharmaceuticalsThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 10 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Arrowhead Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07473843
- Sponsor's protocol ID
- AROANG3-3003
- Other IDs
- 2025-523662-24-00, 2025
- Acronym
- SPRUCE
- Status last verified
- Oct 2026
- First submitted
- 11 Mar 2026
- Last updated
- 6 Oct 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No