NCT04152200已完成3 期
ILLUMINATE-C: A Single Arm Study to Evaluate Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Lumasiran in Patients With Advanced Primary Hyperoxaluria Type 1 (PH1)
Alnylam Pharmaceuticals1 个研究点 分布在 1 个国家目标入组 21 人开始时间: 2020年1月21日最近更新:
适应症
干预措施
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 发起方
- 入组人数
- 21
- 试验地点
- 1
- 主要终点
- Cohort A: Percent Change in Plasma Oxalate 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 patients with Advanced 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
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Has documented diagnosis of primary hyperoxaluria type 1 (PH1)
- •Estimated glomerular filtration rate (eGFR) ≤45 mL/min/1.73 m^2 for patients ≥12 months of age (<12 months of age, must have serum creatinine considered elevated for age)
- •Meets plasma oxalate level requirements
- •If taking Vitamin B6 (pyridoxine), must have been on stable regimen for at least 90 days
- •If on dialysis, may be on hemodialysis therapy only and must have been on a stable regimen for at least 4 weeks
排除标准
- •Hemodialysis/peritoneal dialysis combination therapy or peritoneal dialysis alone
- •Diagnosis of conditions other than PH1 contributing to renal insufficiency
- •History of liver transplant
- •History of kidney transplant and currently receiving immunosuppressants
研究组 & 干预措施
Lumasiran
Experimental
All patients will receive open-label lumasiran.
干预措施: Lumasiran (Drug)
结局指标
主要结局
Cohort A: Percent Change in Plasma Oxalate From Baseline to Month 6
时间窗: Baseline to Month 6
Percent change in plasma oxalate (umol/L) was estimated by an average percent change from baseline across Months 3 through 6. A negative change from Baseline indicates a favorable outcome. For Cohort A, the baseline was defined as the mean of all plasma oxalate level values collected prior to the first dose of lumasiran.
Cohort B: Percent Change in Pre-dialysis Plasma Oxalate From Baseline to Month 6
时间窗: Baseline to Month 6
Percent change in plasma oxalate (umol/L) was estimated by an average percent change from baseline across Months 3 through 6. A negative change from Baseline indicates a favorable outcome. For Cohort B, the baseline is defined as the mean of the last four pre-dialysis plasma oxalate samples collected prior to the first dose of lumasiran. In Cohort B, only pre-dialysis samples are utilized.
次要结局
- Change in Quality of Life As Assessed by Pediatric Quality of Life Inventory (PedsQL) Total Score From Baseline to Month 6 in Patients >=2 to 18 Years of Age at Time of Informed Consent(Baseline to Month 6)
- Cohort A: Percent Change in 24-hour Urinary Oxalate Excretion Corrected for Body Surface Area (BSA) From Baseline to Month 6(Baseline to Month 6)
- Apparent Clearance (CL/F) of Lumasiran(Day 1; Month 6)
- Maximum Plasma Concentration (Cmax) of Lumasiran(Day 1; Month 6)
- Percent Change in Plasma Oxalate Area Under the Curve From 0-24 Hours [AUC(0-24)] Between Dialysis Sessions From Baseline to Month 60(Baseline to Month 60)
- Percent Change in 24-hour Urinary Oxalate Excretion Corrected for Body Surface Area (BSA) From Baseline to Month 60(Baseline to Month 60)
- Absolute Change in Plasma Oxalate From Baseline to Month 6(Baseline to Month 6)
- Cohort A: Absolute Change in 24-hour Urinary Oxalate Excretion Corrected for Body Surface Area (BSA) From Baseline to Month 6(Baseline to Month 6)
- Cohort A: Percent Change in Spot Urinary Oxalate:Creatinine Ratio From Baseline to Month 6(Baseline to Month 6)
- Absolute Change in Plasma Oxalate From Baseline to Month 60(Baseline to Month 60)
- Change in Quality of Life As Assessed by Pediatric Quality of Life Inventory (PedsQL) Total Score From Baseline to Month 60 in Patients >=2 to 18 Years of Age at Time of Informed Consent(Baseline to Month 60)
- Cohort B: Percent Change in Plasma Oxalate Area Under the Curve From 0-24 Hours [AUC(0-24)] Between Dialysis Sessions From Baseline to Month 6(Baseline to Month 6)
- Cohort A: Absolute Change in Spot Urinary Oxalate:Creatinine Ratio From Baseline to Month 6(Baseline to Month 6)
- Change in Quality of Life As Assessed by Kidney Disease and Quality of Life (KDQOL) Scores From Baseline to Month 6 in Patients ≥18 Years of Age at Time of Informed Consent(Baseline to Month 6)
- Time to Maximum Plasma Concentration (Tmax) of Lumasiran(Day 1; Month 6)
- Elimination Half-life (t½β) of Lumasiran(Day 1; Month 6)
- Area Under the Concentration-time Curve From 0 to 24 Hours [AUC(0-24)] for Lumasiran(Day 1; Month 6)
- Apparent Volume of Distribution (V/F) of Lumasiran(Day 1; Month 6)
- Percent Change in Spot Urinary Oxalate:Creatinine Ratio From Baseline to Month 60(Baseline to Month 60)
- Change in Frequency of Dialysis From Baseline to End of Study(Baseline to Month 60)
- Absolute Change in Spot Urinary Oxalate:Creatinine Ratio From Baseline to Month 60(Baseline to Month 60)
- Change in Nephrocalcinosis From Baseline to End of Study(Baseline to Month 60)
- Change in Mode of Dialysis From Baseline to End of Study(Baseline to Month 60)
- Change in Frequency of Renal Stone Events From Baseline to End of Study(Baseline to Month 60)
- Change in Measures of Systemic Oxalosis From Baseline to End of Study(Baseline to Month 60)
- Absolute Change in 24-hour Urinary Oxalate Excretion Corrected for Body Surface Area (BSA) From Baseline to Month 60(Baseline to Month 60)
- Change in Renal Function Assessed by Estimated Glomerular Filtration Rate (eGFR) From Baseline to End of Study(Baseline to Month 60)
- Change in Quality of Life As Assessed by Kidney Disease and Quality of Life (KDQOL) Scores From Baseline to Month 60 in Patients ≥18 Years of Age at Time of Informed Consent(Baseline to Month 60)
- Percent Change in Plasma Oxalate From Baseline to End of Study(Baseline to Month 60)
研究者
研究点 (1)
Loading locations...
相似试验
进行中（未招募）
3 期
Efficacy, Safety, PK, PD, and ADA of Eculizumab in Chinese Adults With NMOSDNMOSDNeuromyelitis Optica Spectrum DisordersNCT06724809Alexion Pharmaceuticals, Inc.21
已完成
1 期
Single Dose Study of MK-2060 to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics in Older Japanese Participants on Dialysis (MK-2060-012)End-Stage Renal Disease (ESRD)End-Stage Kidney Disease (ESKD)Kidney Failure, ChronicNCT05769595Merck Sharp & Dohme LLC17
暂停
早期 1 期
Universal CAR-T Cells (REVO-UWD-00B) for Refractory and Relapsed Multiple MyelomaMultiple MyleomaRefractory and Relapsed Multiple MyelomaNCT06663046Wondercel Biotech (ShenZhen)30
已完成
早期 1 期
Research Development（RD）13-02 Chimeric Antigen Receptor(CAR) -T Cell Injection for Patients With r/r Cluster Of Differentiation 7（CD7）+ T-Acute Lymphoblastic Leukemia(ALL)/T-Lymphoblastic Lymphoma(LBL) /Acute Myelogenous Leukemia(AML)NeoplasmsHematologic NeoplasmsHematologic DiseasesNCT05907603Kai Lin Xu，MD8
进行中（未招募）
1 期
Study to Evaluate Pharmacokinetics, Safety & Tolerability of 101-PGC-005 in Healthy, Adult, Human SubjectsGlucocorticoids ToxicityCOVID-19Infectious DiseaseVirus DiseasesNCT05619770101 Therapeutics6