2025-523662-24-00招募中3 期
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)
适应症
干预措施
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 入组人数
- 9
- 试验地点
- 5
- 主要终点
- Percent Change from Baseline to Month 12 in Fasting LDL-C
研究概览
简要总结
To demonstrate reduction of LDL-C with zodasiran in adolescent participants with HoFH
入排标准
- 年龄范围
- 0 years 至 64 years（18-64 Years, 0-17 Years）
- 接受健康志愿者
- 否
入选标准
- •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
- •NOTE: Additional inclusion/exclusion criteria may apply per protocol
排除标准
- •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
研究组 & 干预措施
ARO-ANG3
Test
干预措施: ARO-ANG3 (Drug)
结局指标
主要结局
Percent Change from Baseline to Month 12 in Fasting LDL-C
Percent Change from Baseline to Month 12 in Fasting LDL-C
次要结局
- 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)
研究者
Medical Monitor
Scientific
Arrowhead Pharmaceuticals Inc.
研究点 (5)
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