A Voxelotor for Sickle Cell Anemia Patients at Highest Risk for Progression of Chronic Kidney Disease

Phase 1

Terminated

• Conditions: Sickle Cell Disease; Sickle Cell Nephropathy
• Interventions: Drug: Voxelotor

• Registration Number: NCT04335721
• Lead Sponsor: University of Illinois at Chicago

Brief Summary

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

Detailed Description

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 Timeline

• Study First Submitted: 2020-04-02
• Study First Submitted QC: 2020-04-03
• Study First Posted: 2020-04-06
• Study Start: 2021-03-16
• Primary Completion: 2024-10-07
• Study Completion: 2024-10-07
• Status Verified: 2025-03
• Last Update Submitted: 2025-03-31
• Last Update Posted: 2025-04-03

Recruitment & Eligibility

• Status: TERMINATED
• Sex: All
• Target Recruitment: 12

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Voxelot	Voxelotor	Voxelotor 1500mg once a day
Standard of Care (SOC)	Voxelotor	Observational while receiving SOC

Primary Outcome Measures

Name	Time	Method
Change in albuminuria in voxelotor-treated SCA patients compared to the observation patients by a one-sided test	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

Secondary Outcome Measures

Name	Time	Method
Change from the average of the Screening and Baseline values to the Week 47 and Week 48 values kidney function measure	48 weeks	Urine dipstick-defined hemoglobinuria)
Change from the average of the Screening and Baseline values to the Week 47 and Week 48 values oxidative injury biomarker	48 weeks	Serum 8-OHd
Change from the average of the Screening and Baseline values to the Week 47 and Week 48 values in albuminuria	48 weeks	Proportion of subjects achieving a 25% decline in albuminuria in the voxelotor-treated group compared to the observation group
Change from the average of the Screening and Baseline values to the Week 47 and Week 48 values in kidney function measure	48 weeks	24 hour serum creatinine
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	24 hour urine eGFR
Change from the average of the Screening and Baseline values to the Week 47 and Week 48 values in Kidney function measure	48 weeks	CKD stage
Change from the average of the Screening and Baseline values to the Week 47 and Week 48 values markers of hemolysis	48 weeks	Hemoglobin concentration
Change from the average of the Screening and Baseline values to the Week 47 and Week 48 values kidney injury biomarker	48 weeks	Urine NGAL

Trial Locations

Locations (1)

University of Illinois; 🇺🇸 Chicago, Illinois, United States