Kidney Function in Sickle Cell Anemia

NCT03277547 | Active Not Recruiting

• 2 other identifiers: 17-09361R01FD006030-01
• Study Type: observational
• Target: 300
• Locations: 1 country
• Sites: 3

Brief Summary

This is a prospective clinical cohort study that involves a baseline study visit followed by up to 3 annual follow-up study visits for a total follow-up of 36-48 months to evaluate the age- and sex-adjusted rate of change in kidney function, and to identify biomarkers of endothelial function, metabolomic profiles and clinical characteristics for the worsening of kidney function and for a rapid decline in kidney function. "Funding Source - FDA OOPD"

Conditions

Sickle Cell Disease; Kidney Failure, Chronic

Outcome Measures

Primary Outcomes (4)

• Age- and sex-adjusted rate of change, over 36 - 48 months, in estimated glomerular filtration rate in patients with sickle cell anemia

  Estimated glomerular filtration rate will be ascertained using the CKD EPI equation

  36-48 months

• Age- and sex-adjusted rate of change, over 36 - 48 months, in albuminuria in patients with sickle cell anemia

  Evaluate the rate of change in albuminuria by spot urine measurements of albumin-creatinine ratio during designated study visits

  36-48 months

• Cross-sectional association of biomarkers of endothelial function with kidney function (estimated glomerular filtration rate and albuminuria) in patients with sickle cell anemia

  Plasma levels of ET-1, VEGF and soluble VCAM-1 from samples obtained at designated study visits will serve as measures of endothelial function

  36-48 months

• Cross-sectional association of urine and plasma metabolomics profiles with kidney function (estimated glomerular filtration rates and albuminuria) in patients with sickle cell anemia

  Untargeted metabolic profiling of plasma and urine will be performed using high-resonance nuclear magnetic resonance spectrometry. Plasma and urine analytes which are significantly associated with estimated glomerular filtration rate and albumin-creatinine ratio will be ascertained.

  36-48 months

Eligibility Criteria

• Age: 18 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Patients with HbSS or HbSB0 thalassemia between the ages of 18 and 65 years who meet the eligibility criteria and provide consent to participate in the study

You may qualify if:

• age of 18 to 65 years;
• confirmed diagnosis of sickle cell anemia (HbSS and SB0 thalassemia);
• non-crisis, "steady state" with no severe pain episodes requiring medical contact during the preceding 4 weeks;
• ability to understand the requirements of the study and be willing to give informed consent.

You may not qualify if:

• bone marrow transplantation;
• history of long-standing diabetes mellitus with suspicion for diabetic nephropathy as determined by a nephrologist;
• known diagnosis of hepatitis B or C infection (patients will not be screened specifically for this during the study);
• known HIV positive (patients will not be screened specifically for this);
• history of cancer, except non-melanoma skin cancer;
• pregnant or breastfeeding;
• connective tissue disease such as SLE;
• known glomerular disease unrelated to SCD;
• patients with ESRD on chronic dialysis.

Sponsors & Collaborators

• Kenneth Ataga MD: lead
• Ohio State University: collaborator
• University of North Carolina: collaborator

Study Sites (3)

University of North Carolina-Chapel Hill

Chapel Hill, North Carolina, 27599, United States

Location

Ohio State Adult Sickle Cell Program

Columbus, Ohio, 43210, United States

Location

UTHSC Center for Sickle Cell Disease

Memphis, Tennessee, 38163, United States

Location

Related Publications (4)

• Ataga KI, Brittain JE, Moore D, Jones SK, Hulkower B, Strayhorn D, Adam S, Redding-Lallinger R, Nachman P, Orringer EP. Urinary albumin excretion is associated with pulmonary hypertension in sickle cell disease: potential role of soluble fms-like tyrosine kinase-1. Eur J Haematol. 2010 Sep;85(3):257-63. doi: 10.1111/j.1600-0609.2010.01471.x. Epub 2010 Jun 3.

  PMID: 20491879 BACKGROUND

• Ataga KI, Derebail VK, Archer DR. The glomerulopathy of sickle cell disease. Am J Hematol. 2014 Sep;89(9):907-14. doi: 10.1002/ajh.23762. Epub 2014 Jun 19.

  PMID: 24840607 BACKGROUND

• Ataga KI, Brittain JE, Jones SK, May R, Delaney J, Strayhorn D, Desai P, Redding-Lallinger R, Key NS, Orringer EP. Association of soluble fms-like tyrosine kinase-1 with pulmonary hypertension and haemolysis in sickle cell disease. Br J Haematol. 2011 Feb;152(4):485-91. doi: 10.1111/j.1365-2141.2010.08410.x. Epub 2011 Jan 11.

  PMID: 21223248 BACKGROUND

• Zhou LY, Derebail VK, Desai PC, Elsherif L, Patillo KL, McCune P, Wichlan D, Landes K, Ogu UO, Nelson M, Loehr LR, Cronin RM, Tang Y, Cai J, Ataga KI. Persistent albuminuria and chronic kidney disease in adults with sickle cell anaemia: Results from a multicenter natural history study. Br J Haematol. 2024 Sep;205(3):1159-1169. doi: 10.1111/bjh.19636. Epub 2024 Jul 8.

  PMID: 38978309 DERIVED

Biospecimen

Retention: SAMPLES WITHOUT DNA

Plasma samples will be collected to measure biomarkers of endothelial function. Plasma and urine samples will be collected for measurement of metabolic profiles.

MeSH Terms

Conditions

Anemia, Sickle Cell; Kidney Failure, Chronic

Condition Hierarchy (Ancestors)

Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Renal Insufficiency, Chronic; Renal Insufficiency; Kidney Diseases; Urologic Diseases; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Male Urogenital Diseases; Chronic Disease; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Study Officials

• Kenneth Ataga, MD

  UTHSC Center for Sickle Cell Disease

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Professor

Study Record Dates

• First Submitted: September 1, 2017
• First Posted: September 11, 2017
• Study Start: November 17, 2017
• Primary Completion: June 1, 2025
• Study Completion: December 1, 2025
• Last Updated: January 27, 2025

Record last verified: 2025-01

Data Sharing

• IPD Sharing: Will not share

Locations

US (3)