Sickle Cell DiseaseNCT04380610
Improving Scientific Rigor of Renal Clinical Endpoints for Sickle Cell Anemia
An observational study of Sickle Cell Disease and Renal Disease, run by University of Alabama at Birmingham at 4 sites in United States.
Trial summary
The investigators will attempt to develop a more accurate equation to estimate eGFR in pediatric and adult sickle cell patients Read more about this study
- Who is it for?
- Ages 5 to 50 yearsHealthy volunteers can take part
- Conditions the study is for
- Sickle Cell Disease, Renal Disease, Glomerular Disease
- What is being tested?
- iohexol GFR
- How many people
- 120 participants
- Where
- 4 sitesUnited States
- Study dates
- 12 May 2021to 30 Jun 2025
- Phase
- Observational
- 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 5 to 50 years
- All sexes
- Healthy volunteers accepted
- Child, Adult
Study population: Patients with sickle cell anemia
Who may be able to join
- SCA patients (HbSS and SB0 thalassemia)
- Age: 5.0-50.0 at enrollment
Who cannot join
- Recent SCA complication associated with hospitalization (within 30 days) or ED visit (within 14 days)
- Current AKI defined as >0.3mg/g increase in SCr from prior visit
- Known history of anaphylaxis with contrast agent or known pregnancy
Trial locations and contacts
4 sites in 1 country.
United States4 sites
University of Alabama at Birmingham
Birmingham, Alabama, 35223
University of Illinois Chicage
Chicago, Illinois, 60612
St Jude Childrens Research Hospital
Memphis, Tennessee, 38106
University of Tennessee Health Science Center
Memphis, Tennessee, 38163
About this study
The investigators will attempt to develop a more accurate equation to estimate eGFR in pediatric and adult sickle cell patients
Read the detailed description
200 pediatric and 200 adult SCA participants will undergo mGFR using iohexol at baseline and at one year. On the same day, participants will undergo clinical and laboratory variables important for developing an eGFR equation. From baseline data, the investigators will perform a training and validation cohort of biologically relevant predictor variables to develop a novel equation. Then the investigators will compare the novel SCA eGFR equation to the established eGFR equation. From one year data, the investigators will determine the longitudinal concordance between pediatric and adult novel and standard eGFR equations with mGFR and variables that may alter longitudinal inferences.
In a subset of 30 adult participants, the investigators will also evaluate the correlation between mGFR using iohexol and a novel measure of mGFR that is more time efficient
- Conditions
- Sickle Cell Disease, Renal Disease, Glomerular Disease
- Being tested
- iohexol GFR
- Medical subject headings
- Anemia, Sickle Cell, Kidney Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
iohexol GFRDiagnostic test
We will perform mGFR in 400 SCA patients
| Study group | Role | What they receive |
|---|---|---|
| Pediatric SCA | We will develop a novel eGFR equation in 200 pediatric participants Receives: Diagnostic Test: iohexol GFR | |
| Adult SCA | We will develop a novel eGFR equation in 200 adult participants Receives: Diagnostic Test: iohexol GFR |
What is being measured
The primary outcome is the main question the study is designed to answer.
To develop a novel SCA-specific eGFR equation and compare with existing eGFR equations validated for the general population
The investigators will use biologically relevant variables to develop a new equation for eGFR in pediatric and adult sickle cell patients
Measured: 5 years
Secondary outcomes (2)
To determine the longitudinal concordance between pediatric and adult novel and standard eGFR equations with mGFR and variables that may alter longitudinal inferences.
To compare the concordance between eGFR and mGFR from baseline to one year
Measured: 5 years
To test the correlation of the novel mGFR method using VFI FAST and iohexol mGFR.
To perform a 3 hour mGFR and compare this mGFR to the 6 hour mGFR (iohexol)
Measured: 5 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 May 2020
- Study start12 May 2021
- Primary completion30 Jun 2025
- Study completion30 Jun 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Sickle Cell DiseaseStudies of the same condition, open and under way.
- More trials run by University of Alabama at BirminghamThe 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
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 120 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Alabama at Birmingham
- Sponsor type
- University, hospital or other
- Collaborators
- National Heart, Lung, and Blood Institute (NHLBI)
- Responsible party
- Principal investigator, Jeffrey D. Lebensburger, DO, University of Alabama at Birmingham
- Investigators
- Jeffrey Lebensburger, DO, MSPH, Principal investigator, University of Alabama at Birmingham
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04380610
- Sponsor's protocol ID
- IRB-300005433
- Other IDs
- 1R01HL153386
- Status last verified
- Sep 2025
- First submitted
- 5 May 2020
- Last updated
- 18 Sep 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: Data collected in this study will be made available to other researchers, after the goals have been met and primary aims have been published. Disclosure of sensitive health and behavior data present a potential risk to human subjects, and we will take all steps to ensure that this does not happen when data are shared. In addition, we will strongly recommend that all recipient groups ensure that every member of the staff sign a Confidentiality Agreement. We will develop a data and resource sharing agreement with the legal advice from the Compliance Office of the UAB. Before data is shared with researchers, we will require that researchers enter into a data- and resource-sharing agreement. This agreement will ensure that: 1) data are used only for research purposes; 2) data do not identify any individual participant; 3) data are secure, using appropriate computer technology; and 4) there is a commitment to destroy or return data after analyses are completed.
Study at a glance
From the registry record, last updated by the sponsor on 18 Sep 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Improving Scientific Rigor of Renal Clinical Endpoints for Sickle Cell Anemia
- ClinicalTrials.gov ID
- NCT04380610
- Acronym / sponsor ID
- IRB-300005433
- Conditions
- Sickle Cell Disease, Renal Disease, Glomerular Disease
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 120 participants (actual)
- Lead sponsor
- University of Alabama at BirminghamUniversity, hospital or other
- Collaborators
- National Heart, Lung, and Blood Institute (NHLBI)
- Study start
- 12 May 2021 (actual)
- Primary completion
- 30 Jun 2025 (actual)
- Study completion
- 30 Jun 2025 (actual)
- First posted
- 8 May 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.
The investigators will attempt to develop a more accurate equation to estimate eGFR in pediatric and adult sickle cell patients
Detailed description
200 pediatric and 200 adult SCA participants will undergo mGFR using iohexol at baseline and at one year. On the same day, participants will undergo clinical and laboratory variables important for developing an eGFR equation. From baseline data, the investigators will perform a training and validation cohort of biologically relevant predictor variables to develop a novel equation. Then the investigators will compare the novel SCA eGFR equation to the established eGFR equation. From one year data, the investigators will determine the longitudinal concordance between pediatric and adult novel and standard eGFR equations with mGFR and variables that may alter longitudinal inferences.
In a subset of 30 adult participants, the investigators will also evaluate the correlation between mGFR using iohexol and a novel measure of mGFR that is more time efficient
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Pediatric SCA | – | Diagnostic Test: iohexol GFRWe will develop a novel eGFR equation in 200 pediatric participants |
| Adult SCA | – | Diagnostic Test: iohexol GFRWe will develop a novel eGFR equation in 200 adult participants |
Who may qualify
- Ages
- 5 to 50 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child, Adult
- Study population
- Patients with sickle cell anemia
Key inclusion criteria (2 listed)
- SCA patients (HbSS and SB0 thalassemia)
- Age: 5.0-50.0 at enrollment
Key exclusion criteria (3 listed)
- Recent SCA complication associated with hospitalization (within 30 days) or ED visit (within 14 days)
- Current AKI defined as >0.3mg/g increase in SCr from prior visit
- Known history of anaphylaxis with contrast agent or known pregnancy
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 |
|---|---|---|
| iohexol GFRWe will perform mGFR in 400 SCA patients | Diagnostic test | Adult SCA, Pediatric SCA |
Primary outcome
To develop a novel SCA-specific eGFR equation and compare with existing eGFR equations validated for the general population
Time frame: 5 years
The investigators will use biologically relevant variables to develop a new equation for eGFR in pediatric and adult sickle cell patients
2 secondary outcomes
To determine the longitudinal concordance between pediatric and adult novel and standard eGFR equations with mGFR and variables that may alter longitudinal inferences.
Time frame: 5 years
To test the correlation of the novel mGFR method using VFI FAST and iohexol mGFR.
Time frame: 5 years
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Sep 2025.
- Individual participant dataSharing planned: Yes. Data collected in this study will be made available to other researchers, after the goals have been met and primary aims have been published. Disclosure of sensitive health and behavior data present a potential risk to human subjects, and we will take all steps to ensure that this does not happen when data are shared. In addition, we will strongly recommend that all recipient groups ensure that every member of the staff sign a Confidentiality Agreement. We will develop a data and resource sharing agreement with the legal advice from the Compliance Office of the UAB. Before data is shared with researchers, we will require that researchers enter into a data- and resource-sharing agreement. This agreement will ensure that: 1) data are used only for research purposes; 2) data do not identify any individual participant; 3) data are secure, using appropriate computer technology; and 4) there is a commitment to destroy or return data after analyses are completed.
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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.
Jeffrey Lebensburger, DO, MSPH principal investigator
University of Alabama at Birmingham
4 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Sickle Cell Disease and Renal Disease, run by University of Alabama at Birmingham at 4 sites 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 2 inclusion and 3 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: finished, as of the sponsor's last update on 18 Sep 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites 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.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
To develop a novel SCA-specific eGFR equation and compare with existing eGFR equations validated for the general population (time frame: 5 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 18 Sep 2025.