Improving Scientific Rigor of Renal Clinical Endpoints for Sickle Cell Anemia

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

Study Overview

• Status: Recruiting
• Conditions: Sickle Cell Disease; Glomerular Disease; Renal Disease
• Intervention / Treatment: Diagnostic test: iohexol GFR

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 Type: Observational
• Enrollment (Anticipated): 400

Contacts and Locations

• Study Contact: Name: Jeffrey Lebensburger, DO, MSPH; Phone Number: 205 638-9285; Email: jlebensburger@peds.uab.edu

Study Locations

• United States
  • Alabama
    • Birmingham, Alabama, United States, 35223
      • Recruiting
      • University of Alabama at Birmingham
      • Contact: Jeffrey D Lebensburger, DO; Phone Number: 205-638-9285; Email: jlebensburger@peds.uab.edu
      • Principal Investigator: Julie Kanter Washko, MD
      • Principal Investigator: Jeffrey Lebensburger, DO, MSPH
  • Illinois
    • Chicago, Illinois, United States, 60612
      • Recruiting
      • University of Illinois Chicage
      • Contact: Santosh Saraf, MD, BS
  • Tennessee
    • Memphis, Tennessee, United States, 38163
      • Recruiting
      • University of Tennessee Health Science Center
      • Contact: Kenneth Ataga, MBBS
    • Memphis, Tennessee, United States, 38106
      • Recruiting
      • St Jude Childrens Research Hospital
      • Contact: Jane Hankins, MD, MS

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 5 years to 50 years (Child, Adult)
• Accepts Healthy Volunteers: Yes

• Sampling Method: Probability Sample

Study Population

Patients with sickle cell anemia

Description

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

Study Plan

How is the study designed?

Number of groups / cohorts

2

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Pediatric SCA; We will develop a novel eGFR equation in 200 pediatric participants	Diagnostic test: iohexol GFR; We will perform mGFR in 400 SCA patients
Adult SCA; We will develop a novel eGFR equation in 200 adult participants	Diagnostic test: iohexol GFR; We will perform mGFR in 400 SCA patients

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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 Outcome Measures

Outcome Measure	Measure Description	Time Frame
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
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

Collaborators and Investigators

• Sponsor: University of Alabama at Birmingham
• Collaborators: National Heart, Lung, and Blood Institute (NHLBI)
• Investigators: Principal Investigator: Jeffrey Lebensburger, DO, MSPH, University of Alabama at Birmingham

Study record dates

Study Major Dates

• Study Start (Actual): May 12, 2021
• Primary Completion (Anticipated): May 15, 2024
• Study Completion (Anticipated): May 15, 2024

Study Registration Dates

• First Submitted: May 5, 2020
• First Submitted That Met QC Criteria: May 5, 2020
• First Posted (Actual): May 8, 2020

Study Record Updates

• Last Update Posted (Actual): April 26, 2023
• Last Update Submitted That Met QC Criteria: April 24, 2023
• Last Verified: April 1, 2023

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Hematologic Diseases; Genetic Diseases, Inborn; Anemia; Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Hemoglobinopathies; Anemia, Sickle Cell
• Other Study ID Numbers: 1R01HL153386 (U.S. NIH Grant/Contract)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: 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.

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No