NCT07473843尚未招募3 期
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)
适应症
干预措施
试验速览
- 阶段
- 3 期
- 状态
- 尚未招募
- 入组人数
- 12
- 主要终点
- Percent Change from Baseline to Month 12 in Fasting LDL-C
研究概览
简要总结
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 12 Years 至 17 Years（Child）
- 性别
- All
- 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
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
- •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
研究组 & 干预措施
Zodasiran
Experimental
干预措施: Zodasiran (Drug)
结局指标
主要结局
Percent Change from Baseline to Month 12 in Fasting LDL-C
时间窗: Baseline, Month 12
次要结局
- Percent Change from Baseline to Month 12 in Fasting Total Cholesterol(Baseline, Month 12)
- Change from Baseline to Month 12 in Fasting HDL-C(Baseline, Month 12)
- Change from Baseline to Month 12 in Fasting Total Cholesterol(Baseline, Month 12)
- Percent Change from Baseline to Month 12 in Fasting HDL-C(Baseline, Month 12)
- Percent Change from Baseline to Month 12 in Fasting Apolipoprotein B (ApoB)(Baseline, Month 12)
- Change from Baseline to Month 12 in Fasting ApoB(Baseline, Month 12)
- Percent Change from Baseline to Month 12 in Fasting Non-High Density Lipoprotein Cholesterol (non-HDL-C)(Baseline, Month 12)
- Change from Baseline to Month 12 in Fasting Non-HDL-C(Baseline, Month 12)
- Change from Baseline to Month 12 in Fasting LDL-C(Baseline, Month 12)
- Area Under the Plasma Concentration Versus the Time Curve (AUC) from Baseline to Month 12 for Fasting LDL-C(Baseline, Month 12)
- Percent Change from Baseline to Month 12 in Fasting Triglycerides (TGs)(Baseline, Month 12)
- Change from Baseline to Month 12 in Fasting TGs(Baseline, Month 12)
- Percent Change from Baseline to Month 12 in Fasting Angiopoietin-like Protein 3 (ANGPTL3)(Baseline, Month 12)
- Change from Baseline to Month 12 in Fasting ANGPTL3(Baseline, Month 12)
- Number of Participants who Meet European Union (EU) LDL-C Apheresis Eligibility Criteria (per German Apheresis Working Group) at Month 12(Month 12)
- Number of Participants who Meet United States (US) Apheresis Eligibility Criteria (per National Lipid Association) at Month 12(Month 12)
- Number of Participants with Fasting LDL-C <100 mg/dL at Month 12(Month 12)
- Percent Change from Baseline in Fasting LDL-C Over Time(Baseline, up to Month 12)
- Change from Baseline in Fasting LDL-C Over Time(Baseline, up to Month 12)
- Number of Participants with Treatment-Emergent Adverse Events (TEAEs)(Up to 27.5 months)
研究者
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