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临床试验/NCT05217667
NCT05217667终止2 期

Phase 2 Study to Evaluate the Safety and Efficacy of ARO-ANG3 in Subjects With Homozygous Familial Hypercholesterolemia (HOFH)

Arrowhead Pharmaceuticals14 个研究点 分布在 4 个国家目标入组 18 人开始时间: 2022年4月22日最近更新:
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适应症
干预措施
相关药物

试验速览

阶段
2 期
状态
终止
入组人数
18
试验地点
14
主要终点
Percent Change from Baseline in Fasting Calculated Low-Density Lipoprotein-Cholesterol (LDL-C) and LDL-C by Preparative Ultracentrifugation (LDL-C [PUC]) up to Week 24
研究概览研究设计入排标准研究组 & 干预措施结局指标研究者研究点记录与标识符相似试验

研究概览

简要总结

Participants with documented homozygous familial hypercholesterolemia (HoFH) who have provided informed consent will receive 2 open-label doses of ARO-ANG3 and be evaluated for safety and efficacy parameters through 36 weeks. Participants who complete the first 36 week treatment period may opt to continue in an additional 24-month extension period during which they will receive up to 8 doses open-label doses of ARO-ANG3.

研究设计

研究类型
Interventional
分配方式
Randomized
干预模型
Parallel
主要目的
Treatment
盲法
None

入排标准

年龄范围
16 Years 至 —（Child, Adult, Older Adult）
性别
All
接受健康志愿者
否

入选标准

  • •Fasting LDL-C >100 mg/dL at Screening
  • •Weight of ≥ 40 kg and body mass index ≥ 18.5 and ≤ 40 kg/m2
  • •Diagnosis of HoFH based on a supportive genetic test or clinical diagnosis
  • •On stable maximally tolerated lipid lowering therapy
  • •Willing to abide by stable low-fat, low-cholesterol, heart-healthy diet for at least 4 weeks prior to Day 1
  • •Participants of childbearing potential (males & females) must agree to use highly-effective contraception during the study and for at least 24 weeks from the last dose of study medication.
  • •Women of childbearing potential must have a negative pregnancy test and cannot be breastfeeding
  • •Women of childbearing potential on hormonal contraceptives must be stable on the medications for > 2 menstrual cycles prior to Day 1
  • •Willing to provide written informed consent and to comply with study requirements

排除标准

  • •Current use or use within 365 days from Day 1 of any hepatocyte targeted small interfering RNA oligonucleotides (siRNA) or antisense oligonucleoside molecule
  • •Use of evinacumab (some exceptions apply)
  • •Fasting TG > 300 mg/dL at Screening
  • •Presence of any clinically significant uncontrolled endocrine disease known to influence serum lipids or lipoproteins
  • •Newly diagnosed (within 3 months prior to informed consent) or poorly controlled diabetes (Hemoglobin A1c > 9%)
  • •Use of systemic corticosteroids (some exceptions apply)
  • •Symptoms of myocardial ischemia or severe left ventricular dysfunction
  • •History of metastatic malignancy within 3 years of Day 1 (some exceptions apply)
  • •Planned cardiac procedure/surgery such as coronary artery bypass graft (CABG) surgery, percutaneous coronary intervention (PCI), carotid surgery or stenting, or carotid revascularization
  • •Note: additional inclusion/exclusion criteria may apply per protocol

研究组 & 干预措施

ARO-ANG3 Dose 1

Experimental

ARO-ANG3 Dose Level 1 subcutaneous (SC)

干预措施: ARO-ANG 3 Injection (Drug)

ARO-ANG3 Dose 2

Experimental

ARO-ANG3 Dose Level 2 SC

干预措施: ARO-ANG 3 Injection (Drug)

结局指标

主要结局

Percent Change from Baseline in Fasting Calculated Low-Density Lipoprotein-Cholesterol (LDL-C) and LDL-C by Preparative Ultracentrifugation (LDL-C [PUC]) up to Week 24

时间窗: Baseline, up to Week 24

次要结局

  • Percent Change from Baseline in Fasting LDL-C (PUC) Over Time(Baseline, up to Week 36 (initial treatment period), up to Month 24 (extension period))
  • Percent Change from Baseline in Fasting Non-High-Density Lipoprotein-Cholesterol (Non-HDL-C) Over Time(Baseline, up to Week 36 (initial treatment period), up to Month 24 (extension period))
  • Percent Change from Baseline in Fasting Very-Low-Density Lipoprotein-Cholesterol (VLDL-C) Over Time(Baseline, up to Week 36 (initial treatment period), up to Month 24 (extension period))
  • Absolute Change from Baseline in Fasting LDL-C (PUC) Over Time(Baseline, up to Week 36 (initial treatment period), up to Month 24 (extension period))
  • Percent Change from Baseline in Fasting Calculated LDL-C Over Time(Baseline, up to Week 36 (initial treatment period), up to Month 24 (extension period))
  • Absolute Change from Baseline in Fasting Non-HDL-C Over Time(Baseline, up to Week 36 (initial treatment period), up to Month 24 (extension period))
  • Absolute Change from Baseline in VLDL-C Over Time(Baseline, up to Week 36 (initial treatment period), up to Month 24 (extension period))
  • Number of Participants with Anti-Drug Antibodies (ADAs) to ARO-ANG3 Over Time(Baseline, up to Week 36 (initial treatment period), up to Month 24 (extension period))
  • Proportion of Participants meeting United States National Lipid Association Apheresis Eligibility Criteria of LDL-C ≥ 300 mg/dL at Week 24(Week 24)
  • Absolute Change from Baseline in Fasting Calculated LDL-C Over Time(Baseline, up to Week 36 (initial treatment period), up to Month 24 (extension period))
  • Percent Change from Baseline in Fasting Angiopoietin-like 3 (ANGPTL3) Over Time(Baseline, up to Week 36 (initial treatment period), up to Month 24 (extension period))
  • Absolute Change from Baseline in Fasting Total ApoB Over Time(Baseline, up to Week 36 (initial treatment period), up to Month 24 (extension period))
  • Absolute Change from Baseline in Fasting HDL-C Over Time(Baseline, up to Week 36 (initial treatment period), up to Month 24 (extension period))
  • Percent Change from Baseline in Fasting Total Cholesterol (TC) Over Time(Baseline, up to Week 36 (initial treatment period), up to Month 24 (extension period))
  • Absolute Change from Baseline in Fasting TG Over Time(Baseline, up to Week 36 (initial treatment period), up to Month 24 (extension period))
  • Absolute Change from Baseline in Fasting ANGPTL3 Over Time(Baseline, up to Week 36 (initial treatment period), up to Month 24 (extension period))
  • Percent Change from Baseline in Fasting High-Density Lipoprotein-Cholesterol (HDL-C) Over Time(Baseline, up to Week 36 (initial treatment period), up to Month 24 (extension period) 36)
  • Absolute Change from Baseline in Fasting TC Over Time(Baseline, up to Week 36 (initial treatment period), up to Month 24 (extension period))
  • Number of Participants with Treatment Emergent Adverse Events (TEAEs)(Baseline, up to Week 36 (initial treatment period), up to Month 24 (extension period))
  • Percent Change from Baseline in Fasting Triglycerides (TG) Over Time(Baseline, up to Week 36 (initial treatment period), up to Month 24 (extension period))
  • Percent Change from Baseline in Fasting Total Apolipoprotein B (ApoB) Over Time(Baseline, up to Week 36 (initial treatment period), up to Month 24 (extension period))
  • Proportion of Participants Meeting European Union (EU) Apheresis Eligibility Criteria per German Apheresis Working Group at Week 24(Week 24)

研究者

申办方类型
Industry
责任方
Sponsor

研究点 (14)

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