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Phase 3 Single-Arm Open-Label Study to Evaluate the Efficacy and Safety of Zodasiran in Adolescent Subjects (age 12 to <18 years) with Homozygous Familial Hypercholesterolemia (SPRUCE)

Status
Not yet recruiting
Phases
Phase 3
Study type
Interventional
Source
EU CTIS
Registry ID
CTIS2025-523662-24-00
Enrollment
5
Registered
2026-07-20
Start date
Unknown
Completion date
Unknown
Last updated
2026-07-21

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Homozygous Familial Hypercholesterolemia (HoFH)

Brief summary

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

Detailed description

1. Percent Change from Baseline to Month 12 in Fasting Apolipoprotein B (ApoB), 2. Percent Change from Baseline to Month 12 in in Fasting Non-High Density Lipoprotein Cholesterol (non-HDL-C), 3. Change from Baseline to Month 12 in Fasting LDL-C, 4. Area Under the Plasma Concentration Versus the Time Curve (AUC) from Baseline to Month 12 for Fasting LDL-C, 5. Percent Change from Baseline to Month 12 in Fasting Triglycerides (TGs), 6. Percent Change from Baseline to Month 12 in Fasting Angiopoietin-like Protein 3 (ANGPTL3), 7. Percent Change from Baseline to Month 12 in Fasting Total Cholesterol, 8. Percent Change from Baseline to Month 12 in in Fasting HDL-C, 9. Number of Participants who Meet European Union (EU) LDL-C Apheresis Eligibility Criteria (per German Apheresis Working Group) at Month 12, 10. Number of Participants who Meet United States (US) Apheresis Eligibility Criteria (per National Lipid Association) at Month 12, 11. Number of Participants with Fasting LDL-C <100 mg/dL at Month 12, 12. Change from Baseline in Fasting LDL-C Over Time, 13. Percent Change from Baseline in Fasting LDL-C Over Time, 14. Number of Participants with Treatment-Emergent Adverse Events (TEAEs)

Interventions

Sponsors

Arrowhead Pharmaceuticals Inc.
Lead SponsorINDUSTRY

Eligibility

Sex/Gender
All
Age
0 Years to 64 Years

Design outcomes

Primary

MeasureTime frame
Percent Change from Baseline to Month 12 in Fasting LDL-C

Secondary

MeasureTime frame
1. Percent Change from Baseline to Month 12 in Fasting Apolipoprotein B (ApoB), 2. Percent Change from Baseline to Month 12 in in Fasting Non-High Density Lipoprotein Cholesterol (non-HDL-C), 3. Change from Baseline to Month 12 in Fasting LDL-C, 4. Area Under the Plasma Concentration Versus the Time Curve (AUC) from Baseline to Month 12 for Fasting LDL-C, 5. Percent Change from Baseline to Month 12 in Fasting Triglycerides (TGs), 6. Percent Change from Baseline to Month 12 in Fasting Angiopoietin-like Protein 3 (ANGPTL3), 7. Percent Change from Baseline to Month 12 in Fasting Total Cholesterol, 8. Percent Change from Baseline to Month 12 in in Fasting HDL-C, 9. Number of Participants who Meet European Union (EU) LDL-C Apheresis Eligibility Criteria (per German Apheresis Working Group) at Month 12, 10. Number of Participants who Meet United States (US) Apheresis Eligibility Criteria (per National Lipid Association) at Month 12, 11. Number of Participants with Fasting LDL-C <100 mg/dL at

Outcome results

None listed

Source: EU CTIS · Data processed: Jul 22, 2026