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Improving Scientific Rigor of Renal Clinical Endpoints for Sickle Cell Anemia

Improving Scientific Rigor of Renal Clinical Endpoints for Sickle Cell Anemia

Status
Completed
Phases
Unknown
Study type
Observational
Source
ClinicalTrials.gov
Registry ID
NCT04380610
Enrollment
120
Registered
2020-05-08
Start date
2021-05-12
Completion date
2025-06-30
Last updated
2025-09-18

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Glomerular Disease, Renal Disease, Sickle Cell Disease

Brief summary

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

Interventions

DIAGNOSTIC_TESTiohexol GFR

We will perform mGFR in 400 SCA patients

Sponsors

National Heart, Lung, and Blood Institute (NHLBI)
CollaboratorNIH
University of Alabama at Birmingham
Lead SponsorOTHER

Study design

Observational model
COHORT
Time perspective
PROSPECTIVE

Eligibility

Sex/Gender
ALL
Age
5 Years to 50 Years
Healthy volunteers
Yes

Inclusion criteria

* SCA patients (HbSS and SB0 thalassemia) * Age: 5.0-50.0 at enrollment

Exclusion criteria

* 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

Design outcomes

Primary

MeasureTime frameDescription
To develop a novel SCA-specific eGFR equation and compare with existing eGFR equations validated for the general population5 yearsThe investigators will use biologically relevant variables to develop a new equation for eGFR in pediatric and adult sickle cell patients

Secondary

MeasureTime frameDescription
To determine the longitudinal concordance between pediatric and adult novel and standard eGFR equations with mGFR and variables that may alter longitudinal inferences.5 yearsTo compare the concordance between eGFR and mGFR from baseline to one year
To test the correlation of the novel mGFR method using VFI FAST and iohexol mGFR.5 yearsTo perform a 3 hour mGFR and compare this mGFR to the 6 hour mGFR (iohexol)

Countries

United States

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026