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
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
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
12
Voxelotor 1500mg once a day
University of Illinois
Chicago, Illinois, United States
Change in albuminuria in voxelotor-treated SCA patients compared to the observation patients by a one-sided test
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
Time frame: 48 weeks
Change from the average of the Screening and Baseline values to the Week 47 and Week 48 values in albuminuria
Proportion of subjects achieving a 25% decline in albuminuria in the voxelotor-treated group compared to the observation group
Time frame: 48 weeks
Change from the average of the Screening and Baseline values to the Week 47 and Week 48 values in kidney function measure
24 hour urine protein
Time frame: 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
24 hour urine eGFR
Time frame: 48 weeks
Change from the average of the Screening and Baseline values to the Week 47 and Week 48 values in kidney function measure
24 hour urine albumin concentration
Time frame: 48 weeks
Change from the average of the Screening and Baseline values to the Week 47 and Week 48 values in kidney function measure
24 hour serum creatinine
Time frame: 48 weeks
Change from the average of the Screening and Baseline values to the Week 47 and Week 48 values in Kidney function measure
24 hour serum cystatin C
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Time frame: 48 weeks
Change from the average of the Screening and Baseline values to the Week 47 and Week 48 values in Kidney function measure
24 hour serum BUN
Time frame: 48 weeks
Change from the average of the Screening and Baseline values to the Week 47 and Week 48 values in Kidney function measure
CKD stage
Time frame: 48 weeks
Change from the average of the Screening and Baseline values to the Week 47 and Week 48 values kidney function measure
24 hour urine retinol binding protein
Time frame: 48 weeks
Change from the average of the Screening and Baseline values to the Week 47 and Week 48 values kidney function measure
24 hour urine β2 microglobulin
Time frame: 48 weeks
Change from the average of the Screening and Baseline values to the Week 47 and Week 48 values kidney function measure
Plasma cell-free hemoglobin
Time frame: 48 weeks
Change from the average of the Screening and Baseline values to the Week 47 and Week 48 values kidney function measure
Urine hemoglobin
Time frame: 48 weeks
Change from the average of the Screening and Baseline values to the Week 47 and Week 48 values kidney function measure
Urine dipstick-defined hemoglobinuria)
Time frame: 48 weeks
Change from the average of the Screening and Baseline values to the Week 47 and Week 48 values markers of hemolysis
Lactate dehydrogenase (LDH)
Time frame: 48 weeks
Change from the average of the Screening and Baseline values to the Week 47 and Week 48 values markers of hemolysis
Aspartate aminotransferase (AST)
Time frame: 48 weeks
Change from the average of the Screening and Baseline values to the Week 47 and Week 48 values markers of hemolysis
Indirect bilirubin
Time frame: 48 weeks
Change from the average of the Screening and Baseline values to the Week 47 and Week 48 values markers of hemolysis
Reticulocyte%
Time frame: 48 weeks
Change from the average of the Screening and Baseline values to the Week 47 and Week 48 values markers of hemolysis
Hemoglobin concentration
Time frame: 48 weeks
Change from the average of the Screening and Baseline values to the Week 47 and Week 48 values kidney injury biomarker
Urine nephrin
Time frame: 48 weeks
Change from the average of the Screening and Baseline values to the Week 47 and Week 48 values kidney injury biomarker
Urine podocalyxin
Time frame: 48 weeks
Change from the average of the Screening and Baseline values to the Week 47 and Week 48 values kidney injury biomarker
Urine KIM-1
Time frame: 48weeks
Change from the average of the Screening and Baseline values to the Week 47 and Week 48 values kidney injury biomarker
Urine NGAL
Time frame: 48 weeks
Change from the average of the Screening and Baseline values to the Week 47 and Week 48 values oxidative injury biomarker
Serum Methylenedioxyamphetamine (MDA)
Time frame: 48weeks
Change from the average of the Screening and Baseline values to the Week 47 and Week 48 values oxidative injury biomarker
Serum 8-OHd
Time frame: 48 weeks