Clinical trial NCT04380610: Improving Scientific Rigor of Renal Clinical Endpoints for Sickle Cell Anemia
Official title: Improving Scientific Rigor of Renal Clinical Endpoints for Sickle Cell Anemia
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Sickle Cell Disease
Renal Disease
Glomerular Disease
Lead sponsor: University of Alabama at Birmingham
Sponsor class: OTHER
Collaborators: name: National Heart, Lung, and Blood Institute (NHLBI)
First posted: 2020-05-08
Enrollment: 400
Sex: ALL
Minimum age (years): 5
Maximum age (years): 50
Standard ages: CHILD
ADULT
Accepts healthy volunteers: yes
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: PROBABILITY_SAMPLE
Study population: Patients with sickle cell anemia

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

Eligibility criteria: 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

Primary outcomes:
measure: To develop a novel SCA-specific eGFR equation and compare with existing eGFR equations validated for the general population
timeFrame: 5 years
Secondary outcomes:
measure: To determine the longitudinal concordance between pediatric and adult novel and standard eGFR equations with mGFR and variables that may alter longitudinal inferences.
timeFrame: 5 years
measure: To test the correlation of the novel mGFR method using VFI FAST and iohexol mGFR.
timeFrame: 5 years
Other outcomes:
Locations:
city: Birmingham
country: United States
facility: University of Alabama at Birmingham
state: Alabama
city: Chicago
country: United States
facility: University of Illinois Chicage
state: Illinois
city: Memphis
country: United States
facility: St Jude Childrens Research Hospital
state: Tennessee
city: Memphis
country: United States
facility: University of Tennessee Health Science Center
state: Tennessee
Study officials:
affiliation: University of Alabama at Birmingham
name: Jeffrey Lebensburger, DO, MSPH
role: PRINCIPAL_INVESTIGATOR
References: