Sickle Cell DiseasePhase 1/2NCT04335721
A Voxelotor for Sickle Cell Anemia Patients at Highest Risk for Progression of Chronic Kidney Disease
A Phase 1/2 study testing Voxelotor in people with Sickle Cell Disease and Sickle Cell Nephropathy, run by University of Illinois Chicago at 1 site in United States.
Trial 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… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Sickle Cell Disease, Sickle Cell Nephropathy
- What is being tested?
- Voxelotor
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 12 participants
- Where
- 1 siteUnited States
- Study dates
- 16 Mar 2021to 7 Oct 2024
- Phase
- Phase 1/2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Documentation of SCA genotype (HbSS or HbSβ0-thalassemia) may be based on history of laboratory testing or must be confirmed by laboratory testing during screening
- Participants have had urine dipstick defined hemoglobinuria (positive for blood (+1 or higher) and ≤ 2 red blood cells per high power field) on 2 prior outpatient visits
- Participants with albuminuria (urine albumin ≥ 30 mg/g creatinine) and an eGFR ≥ 60 mL/min/1.73m2 calculated using the CKD-EPI equation on 2 prior outpatient visits
- Age ≥18 years
- Hemoglobin (Hb) ≥ 5.5 and ≤ 10.0 g/dL during screening
- For participants taking hydroxyurea (HU), the dose of HU (mg/kg) must be stable for at least 90 days prior to signing the ICF and with no anticipated need for dose adjustments or initiation during the study, in the opinion of the Investigator
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- Endari stable dose for one month.
- For participants taking an angiotensin converting enzyme (ACE)-inhibitor or angiotensin receptor blocker, the dose must be stable for at least 90 days prior to signing the ICF and with no anticipated need for dose adjustments or initiation during the study, in the opinion of the Investigator
- Participants, who if female and of child bearing potential, are using highly effective methods of contraception from study start to 30 days after the last dose of study drug, and who if male are willing to use barrier methods of contraception, from study start to 30 days after the last dose of study drug
- Participant has provided documented informed consent (the informed consent form [ICF] must be reviewed and signed by each participant
Who cannot join
- Female who is breast feeding or pregnant
- Patients who are receiving regularly scheduled blood (RBC) transfusion therapy (also termed chronic, prophylactic, or preventive transfusion) or have received a RBC transfusion for any reason within 30 days of signing the ICF or at any time during the screening period
- Hospitalized for sickle cell crisis or other vaso-occlusive event within 14 days prior to signing the ICF (i.e., a vaso-occlusive event cannot be within 14 days prior to ICF)
- Hepatic dysfunction characterized by alanine aminotransferase (ALT) >4 × ULN
- Participants with clinically significant bacterial, fungal, parasitic or viral infection which require therapy:
- Participants with acute bacterial infection requiring antibiotic use should delay screening/enrollment until the course of antibiotic therapy has been completed
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- Participants with known active hepatitis A, B, or C or who are known to be human immunodeficiency virus (HIV) positive
- Severe renal dysfunction (estimated glomerular filtration rate at the Screening visit; calculated by the central laboratory) < 60mL/min/1.732, on chronic dialysis, or have received a kidney transplantation
- History of malignancy within the past 2 years prior to treatment Day 1 requiring chemotherapy and/or radiation (with the exception of local therapy for non-melanoma skin malignancy)
- History of unstable or deteriorating cardiac or pulmonary disease within 6 months prior to consent including but not limited to the following:
- Unstable angina pectoris or myocardial infarction or elective coronary intervention
- Congestive heart failure requiring hospitalization
- Uncontrolled clinically significant arrhythmias
- Any condition affecting drug absorption, such as major surgery involving the stomach or small intestine (prior cholecystectomy is acceptable)
- Participated in another clinical trial of an investigational agent (or medical device) within 30 days or 5 half-lives of date of informed consent, whichever is longer, or is currently participating in another trial of an investigational agent (or medical device)
- Inadequate venous access as determined by the investigator/site staff
- Medical, psychological, or behavioral conditions, which, in the opinion of the Investigator, may preclude safe participation, confound study interpretation, interfere with compliance, or preclude informed consent
- Receipt of erythropoietin or other hematopoietic growth factors within 28 days of signing ICF or anticipated need for such agents during the study
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Pilot Study of Voxelotor for Sickle Cell Anemia Patients at Highest Risk for Progression of Chronic Kidney Disease
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
Read the 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
- Conditions
- Sickle Cell Disease, Sickle Cell Nephropathy
- Being tested
- Voxelotor
- Medical subject headings
- Anemia, Sickle Cell
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
VoxelotorDrug
Voxelotor 1500mg once a day
| Study group | Role | What they receive |
|---|---|---|
| Voxelot | Experimental | Voxelotor 1500mg once a day Receives: Drug: Voxelotor |
| Standard of Care (SOC) | Other | Observational while receiving SOC Receives: Drug: Voxelotor |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: 48 weeks
Secondary outcomes (24)
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
Measured: 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
Measured: 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
Measured: 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
Measured: 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
Measured: 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
Measured: 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
Measured: 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
Measured: 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
Measured: 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
Measured: 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
Measured: 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
Measured: 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)
Measured: 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)
Measured: 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)
Measured: 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
Measured: 48 weeks
Change from the average of the Screening and Baseline values to the Week 47 and Week 48 values markers of hemolysis
Reticulocyte%
Measured: 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
Measured: 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
Measured: 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
Measured: 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
Measured: 48weeks
Change from the average of the Screening and Baseline values to the Week 47 and Week 48 values kidney injury biomarker
Urine NGAL
Measured: 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)
Measured: 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
Measured: 48 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Apr 2020
- Study start16 Mar 2021
- Primary completion7 Oct 2024
- Study completion7 Oct 2024
Why it stopped: Sponsor voluntarily suspended distribution and removed product from the market
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Sickle Cell DiseaseStudies of the same condition, open and under way.
- More trials run by University of Illinois ChicagoThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- 6 participants will take voxelotor 1500mg once a day 6 participants will be observed while receiving standard of care (SOC)
- Masking
- None (open label)
- Enrolment
- 12 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Illinois Chicago
- Sponsor type
- University, hospital or other
- Collaborators
- Global Blood Therapeutics
- Responsible party
- Principal investigator, Santosh L Saraf, University of Illinois Chicago
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04335721
- Sponsor's protocol ID
- 2020-0047
- Status last verified
- Mar 2025
- First submitted
- 2 Apr 2020
- Last updated
- 3 Apr 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Publications
Obadina M, Wilson S, Derebail VK, Little J. Emerging Therapies and Advances in Sickle Cell Disease with a Focus on Renal Manifestations. Kidney360. 2023 Jul 1;4(7):997-1005. doi: 10.34067/KID.0000000000000162. Epub 2023 May 31.
PubMed 37254256
Study at a glance
From the registry record, last updated by the sponsor on 3 Apr 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Pilot Study of Voxelotor for Sickle Cell Anemia Patients at Highest Risk for Progression of Chronic Kidney Disease
- ClinicalTrials.gov ID
- NCT04335721
- Acronym / sponsor ID
- 2020-0047
- Conditions
- Sickle Cell Disease, Sickle Cell Nephropathy
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Sponsor voluntarily suspended distribution and removed product from the market
- Enrolment
- 12 participants (actual)
- Lead sponsor
- University of Illinois ChicagoUniversity, hospital or other
- Collaborators
- Global Blood Therapeutics
- Study start
- 16 Mar 2021 (actual)
- Primary completion
- 7 Oct 2024 (actual)
- Study completion
- 7 Oct 2024 (actual)
- First posted
- 6 Apr 2020 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
- Model description
- 6 participants will take voxelotor 1500mg once a day 6 participants will be observed while receiving standard of care (SOC)
| Arm | Type | Receives |
|---|---|---|
| Voxelot | Experimental | Drug: VoxelotorVoxelotor 1500mg once a day |
| Standard of Care (SOC) | Other | Drug: VoxelotorObservational while receiving SOC |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (10 listed)
- Documentation of SCA genotype (HbSS or HbSβ0-thalassemia) may be based on history of laboratory testing or must be confirmed by laboratory testing during screening
- Participants have had urine dipstick defined hemoglobinuria (positive for blood (+1 or higher) and ≤ 2 red blood cells per high power field) on 2 prior outpatient visits
- Participants with albuminuria (urine albumin ≥ 30 mg/g creatinine) and an eGFR ≥ 60 mL/min/1.73m2 calculated using the CKD-EPI equation on 2 prior outpatient visits
- Age ≥18 years
- Hemoglobin (Hb) ≥ 5.5 and ≤ 10.0 g/dL during screening
Key exclusion criteria (18 listed)
- Female who is breast feeding or pregnant
- Patients who are receiving regularly scheduled blood (RBC) transfusion therapy (also termed chronic, prophylactic, or preventive transfusion) or have received a RBC transfusion for any reason within 30 days of signing the ICF or at any time during the screening period
- Hospitalized for sickle cell crisis or other vaso-occlusive event within 14 days prior to signing the ICF (i.e., a vaso-occlusive event cannot be within 14 days prior to ICF)
- Hepatic dysfunction characterized by alanine aminotransferase (ALT) >4 × ULN
- Participants with clinically significant bacterial, fungal, parasitic or viral infection which require therapy:
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| VoxelotorVoxelotor 1500mg once a day | Drug | Standard of Care (SOC), Voxelot |
Primary outcome
Change in albuminuria in voxelotor-treated SCA patients compared to the observation patients by a one-sided test
Time frame: 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
24 secondary outcomes
Change from the average of the Screening and Baseline values to the Week 47 and Week 48 values in albuminuria
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
Time frame: 48weeks
Change from the average of the Screening and Baseline values to the Week 47 and Week 48 values kidney injury biomarker
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
Time frame: 48weeks
Change from the average of the Screening and Baseline values to the Week 47 and Week 48 values oxidative injury biomarker
Time frame: 48 weeks
Results and safety
The study was stopped early (reason given: Sponsor voluntarily suspended distribution and removed product from the market). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Apr 2025.
- Derived from this study: PubMed 37254256Obadina M, Wilson S, Derebail VK, Little J. Emerging Therapies and Advances in Sickle Cell Disease with a Focus on Renal Manifestations. Kidney360. 2023 Jul 1;4(7):997-1005. doi: 10.34067/KID.0000000000000162. Epub 2023 May 31.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Voxelotor in people with Sickle Cell Disease and Sickle Cell Nephropathy, run by University of Illinois Chicago at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 10 inclusion and 18 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 3 Apr 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was stopped early (reason given: Sponsor voluntarily suspended distribution and removed product from the market). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in albuminuria in voxelotor-treated SCA patients compared to the observation patients by a one-sided test (time frame: 48 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04335721, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 3 Apr 2025.