NCT03905694已完成3 期
ILLUMINATE-B: An Open-Label Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Lumasiran in Infants and Young Children With Primary Hyperoxaluria Type 1
Alnylam Pharmaceuticals1 个研究点 分布在 1 个国家目标入组 18 人开始时间: 2019年4月22日最近更新:
适应症
干预措施
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 发起方
- 入组人数
- 18
- 试验地点
- 1
- 主要终点
- Percentage Change in Spot Urinary Oxalate:Creatinine Ratio From Baseline to Month 6
研究概览
简要总结
The purpose of this study is to evaluate the efficacy, safety, pharmacokinetics (PK), and pharmacodynamics (PD) of lumasiran in infants and young children with confirmed primary hyperoxaluria type 1 (PH1).
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
- 0 Years 至 5 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Has genetic confirmation of primary hyperoxaluria type 1 (PH1)
- •Meets urinary oxalate excretion requirements
- •If taking Vitamin B6 (pyridoxine), must have been on stable regimen for at least 90 days
排除标准
- •If <12 months old at screening, has an abnormally high serum creatinine
- •If ≥12 months old at screening, has an estimated glomerular filtration rate (GFR) of ≤45 mL/min/1.73m^2
- •Clinical evidence of systemic oxalosis
- •History of kidney or liver transplant
研究组 & 干预措施
Lumasiran
Experimental
Lumasiran will be administered by subcutaneous (SC) injection.
干预措施: Lumasiran (Drug)
结局指标
主要结局
Percentage Change in Spot Urinary Oxalate:Creatinine Ratio From Baseline to Month 6
时间窗: Baseline to Month 6
Percent change in spot urinary oxalate:creatinine ratio was estimated by an average percent change from baseline across Months 3 through 6. A negative change from Baseline indicates a favorable outcome.
次要结局
- Percentage Change in Spot Urinary Oxalate: Creatinine Ratio in the Extension Period (Month 6 to End of Study [Month 60])(From Month 6 to Month 60)
- Percentage of Time That Spot Urinary Oxalate: Creatinine Ratio is at or Below the Near-normalization Threshold (≤1.5 × Upper Limit of Normal (ULN) for Age)(Up to 60 months)
- Absolute Change in Spot Urinary Oxalate: Creatinine Ratio From Baseline(From Baseline to Month 6 and Month 60)
- Percentage of Participants With Spot Urinary Oxalate: Creatinine Ratio Levels ≤ the ULN for Age(Up to 60 months)
- Percentage of Participants With Spot Urinary Oxalate: Creatinine Ratio Levels ≤ 1.5xULN for Age(Up to 60 months)
- Percentage Change in Plasma Oxalate From Baseline to End of Study (Month 60)(From Baseline to Month 6 and Month 60)
- Absolute Change in Plasma Oxalate From Baseline to End of Study (Month 60)(From Baseline to Month 6 and Month 60)
- Maximum Observed Plasma Concentration (Cmax) of Lumasiran(2 hours, 4 hours, 8 hours, 12 hours, and 24 hours post-dose on Day 1, Month 6, Month 12, Month 18 and Month 24)
- Time to Maximum Observed Plasma Concentration (Tmax) of Lumasiran(2 hours, 4 hours, 8 hours, 12 hours, and 24 hours post-dose on Day 1, Month 6, Month 12, Month 18 and Month 24)
- Elimination Half-life (t1/2beta) of Lumasiran(2 hours, 4 hours, 8 hours, 12 hours, and 24 hours post-dose on Day 1, Month 6, Month 12, Month 18 and Month 24)
- Area Under the Concentration-time Curve From 0 to 24 Hours (AUC0-24) of Lumasiran(24 hours post-dose on Day 1, Month 6, Month 12, Month 18 and Month 24)
- Area Under the Concentration-time Curve From 0 to Last Quantifiable Concentration (AUC0-last) of Lumasiran(2 hours, 4 hours, 8 hours, 12 hours, and 24 hours post-dose on Day 1, Month 6, Month 12, Month 18 and Month 24)
- Area Under the Concentration-time Curve From 0 to Infinity (AUC0-infinity) of Lumasiran(2 hours, 4 hours, 8 hours, 12 hours, and 24 hours post-dose on Day 1, Month 6, Month 12, Month 18 and Month 24)
- Apparent Clearance (CL/F) of Lumasiran(2 hours, 4 hours, 8 hours, 12 hours, and 24 hours post-dose on Day 1, Month 6, Month 12, Month 18 and Month 24)
- Apparent Volume of Distribution (V/F) of Lumasiran(2 hours, 4 hours, 8 hours, 12 hours, and 24 hours post-dose on Day 1, Month 6, Month 12, Month 18 and Month 24)
- Change From Baseline in Estimated Glomerular Filtration Rate (eGFR)(From Baseline to Month 6 and Month 60)
- Number of Participants With Adverse Events (AEs)(Up to 60 months)
研究者
研究点 (1)
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