2025-521792-31-01招募中3 期
Phase 3 Study to Evaluate the Efficacy and Safety of Zodasiran in Adolescent and Adult Subjects with Homozygous Familial Hypercholesterolemia (YOSEMITE)
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 入组人数
- 7
- 试验地点
- 7
- 主要终点
- Percent Change from Baseline to Month 12 in Fasting Low Density Lipoprotein Cholesterol (LDL-C) (Randomized period)
研究概览
简要总结
To demonstrate reduction of low-density lipoprotein cholesterol (LDL-C) with zodasiran compared with placebo in subjects with HoFH
入排标准
- 年龄范围
- 0 years 至 65+ years（18-64 Years, 0-17 Years, 65+ Years）
- 接受健康志愿者
- 是
入选标准
- •Age ≥12 years, non pregnant, non lactating, do not plan to become pregnant during the study
- •Body weight ≥35 kg at Screening as patients could theoretically be <35 kg as the study continues.
- •HoFH based on a supportive genetic test or a clinical diagnosis (total cholesterol >500 mg/dL[13 mmol/L] OR treated LDL-C concentration of ≥300 mg/dL [≥8 mmol/L] either accompanied by TGs <300 mg/dL [3.4 mmol/L] AND both parents with documented total cholesterol >250 mg/dL [6.5 mmol/L] OR cutaneous or tendinous xanthoma before 10 years of age)
- •LDL-C ≥70 mg/dL (1.8 mmol/L). For adolescents 12 to <18 years of age LDL-C ≥116 mg/dL (3 mmol/L)
- •Hemoglobin A1c (HbA1c) ≤9.5%
- •Total bilirubin <2xULN, unless in previously confirmed cases of Gilbert's syndrome
- •Alanine aminotransferase or aspartate aminotransferase <3×ULN
- •On standard of care, maximally tolerated lipid-lowering therapy
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 (exception: use of inclisiran is permitted; administration of inclisiran and study drug must be separated by at least 4 weeks)
- •Use of evinacumab within 3 months before Day
- •Evinacumab use is prohibited during the study. In regions where evinacumab is available, the physician must justify and document in source documents and eCRF(s) why a patient is not receiving evinacumab therapy as part of standard of care (eg, safety, tolerability, reimbursement/cost, patient choice, etc.)
- •Non-response to evinacumab, defined as LDL-C reduction <15% from baseline after 2 doses
- •Use of any other investigational agent or device within 30 days or 5 half-lives (whichever is longer) before Day 1
- •Use of systemic corticosteroids (unless used as replacement therapy for pituitary/adrenal disease with a stable regimen)
- •Estimated glomerular filtration rate <30 mL/min
结局指标
主要结局
Percent Change from Baseline to Month 12 in Fasting Low Density Lipoprotein Cholesterol (LDL-C) (Randomized period)
Percent Change from Baseline to Month 12 in Fasting Low Density Lipoprotein Cholesterol (LDL-C) (Randomized period)
次要结局
- 1. Percent Change from Baseline to Month 12 in Fasting Apolipoprotein B (ApoB) (Randomized Period)
- 2. Percent Change from Baseline to Month 12 in Fasting Non-High Density Lipoprotein Cholesterol (non-HDL-C) (Randomized Period)
- 3. Change from Baseline to Month 12 in Fasting LDL-C (Randomized Period)
- 4. Area Under the Plasma Concentration Versus the Time Curve (AUC) from Baseline to Month 12 for Fasting LDL-C (Randomized Period)
- 5. Percent Change from Baseline to Month 12 in Fasting Triglycerides (TGs) (Randomized Period)
- 6. Percent Change from Baseline to Month 12 in Fasting Angiopoietin-like Protein 3 (ANGPTL3) (Randomized Period)
- 7. Percent Change from Baseline to Month 12 in Fasting Total Cholesterol (Randomized Period)
- 8. Percent Change from Baseline to Month 12 in Fasting High-Density Lipoprotein Cholesterol (HDL-C) (Randomized Period)
- 9. Proportion of Participants who Meet European Union (EU) LDL-C Apheresis Eligibility Criteria (per German Apheresis Working Group) at Month 12 (Randomized Period)
- 10. Proportion of Participants who Meet United States (US) Apheresis Eligibility Criteria (per National Lipid Association) at Month 12 (Randomized Period)
- 11. Proportion of Participants with Fasting LDL-C <100 mg/dL (2.6 mmol/L) at Month 12 (Randomized Period)
- 12. Change from Baseline in Fasting LDL-C Over Time (Randomized Period)
- 13. Percent Change from Baseline in fasting LDL-C Over Time (Randomized Period)
- 14. Percent Change from Baseline to Month 12 in Fasting Lipoprotein(a) [Lp(a)] (Randomized Period)
研究者
Medical Monitor
Scientific
Arrowhead Pharmaceuticals Inc.
研究点 (7)
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