Improving Scientific Rigor of Renal Clinical Endpoints for Sickle Cell Anemia

Sickle Cell Disease Clinical Trial

Official title:

Improving Scientific Rigor of Renal Clinical Endpoints for Sickle Cell Anemia

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT04380610
• Other study ID #: IRB-300005433
• Secondary ID: 1R01HL153386
• Status: Recruiting
• Start date: May 12, 2021
• Est. completion date: May 15, 2024

Study information

• Verified date: April 2024
• Source: University of Alabama at Birmingham
• Contact: Jeffrey Lebensburger, DO, MSPH
• Phone: 205 638-9285
• Email: jlebensburger[@]peds.uab.edu
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

The investigators will attempt to develop a more accurate equation to estimate eGFR in pediatric and adult sickle cell patients

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

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 400
• Est. completion date: May 15, 2024
• Est. primary completion date: May 15, 2024
• Accepts healthy volunteers: Accepts Healthy Volunteers
• Gender: All
• Age group: 5 Years to 50 Years

Eligibility

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

Related Conditions & MeSH terms

• Anemia, Sickle Cell
• Glomerular Disease
• Renal Disease
• Sickle Cell Disease

Intervention

Diagnostic Test:
• iohexol GFR
We will perform mGFR in 400 SCA patients

Locations

Country	Name	City	State
United States	University of Alabama at Birmingham	Birmingham	Alabama
United States	University of Illinois Chicage	Chicago	Illinois
United States	St Jude Childrens Research Hospital	Memphis	Tennessee
United States	University of Tennessee Health Science Center	Memphis	Tennessee

Sponsors (2)

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

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	5 years
Secondary	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	5 years
Secondary	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)	5 years