A Pilot Study of Voxelotor for Sickle Cell Anemia Patients at Highest Risk for Progression of Chronic Kidney Disease
试验速览
- 阶段
- 1 期
- 状态
- 终止
- 入组人数
- 12
- 试验地点
- 1
- 主要终点
- Change in albuminuria in voxelotor-treated SCA patients compared to the observation patients by a one-sided test
研究概览
简要总结
This study is a single center, prospective exploratory pilot study of Sickle Cell Anemia (SCA) participants. The study will enroll patients with early stages of sickle cell nephropathy (Chronic Kidney Disease (CKD) stage 1 or 2) who are at the highest risk of CKD progression (presence of both hemoglobinuria and urine albumin concentration ≥ 30 mg/g creatinin
详细描述
This study is a single center, prospective exploratory pilot study of Sickle Cell Anemia (SCA) participants, age ≥18 years, with SCA. The study will enroll patients with early stages of sickle cell nephropathy (Chronic Kidney Disease (CKD) stage 1 or 2) who are at the highest risk of CKD progression (presence of both hemoglobinuria and urine albumin concentration ≥ 30 mg/g creatinin
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
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- 未提供
排除标准
- 未提供
研究组 & 干预措施
Voxelot
Voxelotor 1500mg once a day
干预措施: Voxelotor (Drug)
Standard of Care (SOC)
Observational while receiving SOC
干预措施: Voxelotor (Drug)
结局指标
主要结局
Change in albuminuria in voxelotor-treated SCA patients compared to the observation patients by a one-sided test
时间窗: 48 weeks
Albuminuria will be analyzed comparing the mean values from the Week 47 and 48 visits to the mean values from the baseline and screening visits
次要结局
- Change from the average of the Screening and Baseline values to the Week 47 and Week 48 values kidney function measure(48 weeks)
- Change from the average of the Screening and Baseline values to the Week 47 and Week 48 values oxidative injury biomarker(48 weeks)
- Change from the average of the Screening and Baseline values to the Week 47 and Week 48 values in albuminuria(48 weeks)
- Change from the average of the Screening and Baseline values to the Week 47 and Week 48 values in kidney function measure(48 weeks)
- Change from the average of the Screening and Baseline values to the Week 47 and Week 48 values in In kidney function measure(48 weeks)
- Change from the average of the Screening and Baseline values to the Week 47 and Week 48 values in Kidney function measure(48 weeks)
- Change from the average of the Screening and Baseline values to the Week 47 and Week 48 values markers of hemolysis(48 weeks)
- Change from the average of the Screening and Baseline values to the Week 47 and Week 48 values kidney injury biomarker(48 weeks)
研究者
Santosh L Saraf
Principal Investigator
University of Illinois at Chicago