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NCT Number: NCT07473843

Study of Zodasiran in Adolescent Participants With Homozygous Familial Hypercholesterolemia

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.

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Key information

Conditions

Homozygous Familial Hypercholesterolemia Congenital, Hereditary, and Neonatal Diseases and Abnormalities Dyslipidemias Genetic Diseases, Inborn Hyperlipidemias Hyperlipoproteinemia Type II Hyperlipoproteinemias Lipid Metabolism Disorders Lipid Metabolism, Inborn Errors Metabolic Diseases Metabolism, Inborn Errors Nutritional and Metabolic Diseases

Age range

12 year–17 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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
  • Non-response to evinacumab, defined as LDL-C reduction <15% from baseline after 2 doses
  • 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

Treatment and study plan

Zodasiran

Drug

By sc injection

Other names: ARO-ANG3

Primary outcomes

  1. Percent Change from Baseline to Month 12 in Fasting LDL-C

    Time frame: Baseline, Month 12

Secondary outcomes

  1. Percent Change from Baseline to Month 12 in Fasting Apolipoprotein B (ApoB)

    Time frame: Baseline, Month 12

  2. Change from Baseline to Month 12 in Fasting ApoB

    Time frame: Baseline, Month 12

  3. Percent Change from Baseline to Month 12 in Fasting Non-High Density Lipoprotein Cholesterol (non-HDL-C)

    Time frame: Baseline, Month 12

  4. Change from Baseline to Month 12 in Fasting Non-HDL-C

    Time frame: Baseline, Month 12

  5. Change from Baseline to Month 12 in Fasting LDL-C

    Time frame: Baseline, Month 12

  6. Area Under the Plasma Concentration Versus the Time Curve (AUC) from Baseline to Month 12 for Fasting LDL-C

    Time frame: Baseline, Month 12

  7. Percent Change from Baseline to Month 12 in Fasting Triglycerides (TGs)

    Time frame: Baseline, Month 12

  8. Change from Baseline to Month 12 in Fasting TGs

    Time frame: Baseline, Month 12

  9. Percent Change from Baseline to Month 12 in Fasting Angiopoietin-like Protein 3 (ANGPTL3)

    Time frame: Baseline, Month 12

  10. Change from Baseline to Month 12 in Fasting ANGPTL3

    Time frame: Baseline, Month 12

  11. Percent Change from Baseline to Month 12 in Fasting Total Cholesterol

    Time frame: Baseline, Month 12

  12. Change from Baseline to Month 12 in Fasting Total Cholesterol

    Time frame: Baseline, Month 12

  13. Percent Change from Baseline to Month 12 in Fasting HDL-C

    Time frame: Baseline, Month 12

  14. Change from Baseline to Month 12 in Fasting HDL-C

    Time frame: Baseline, Month 12

  15. Number of Participants who Meet European Union (EU) LDL-C Apheresis Eligibility Criteria (per German Apheresis Working Group) at Month 12

    Time frame: Month 12

  16. Number of Participants who Meet United States (US) Apheresis Eligibility Criteria (per National Lipid Association) at Month 12

    Time frame: Month 12

  17. Number of Participants with Fasting LDL-C <100 mg/dL at Month 12

    Time frame: Month 12

  18. Percent Change from Baseline in Fasting LDL-C Over Time

    Time frame: Baseline, up to Month 12

  19. Change from Baseline in Fasting LDL-C Over Time

    Time frame: Baseline, up to Month 12

  20. Number of Participants with Treatment-Emergent Adverse Events (TEAEs)

    Time frame: Up to 27.5 months

Study contacts

Contact information is provided by the study sponsor or research team.

Medical Monitor

CONTACT

[email protected]

626-304-3400

Sponsors and collaborators

Lead sponsor

Arrowhead Pharmaceuticals

Industry

Registry information

Official study 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)

Acronym: SPRUCE

Important dates

Study start
2026
Primary completion
2028
Study completion
2028
First posted
Mar 16, 2026
Registry last updated
May 29, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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