The Predictive Capacity of Machine Learning Models for Progressive Kidney Disease in Individuals With Sickle Cell Anemia

NCT05214105 | Unknown

• 2 other identifiers: 2021-07461R01HL159376-01
• Study Type: observational
• Target: 400
• Locations: 1 country
• Sites: 3

Brief Summary

This is a multicenter prospective, longitudinal cohort study which will evaluate the predictive capacity of machine learning (ML) models for progression of CKD in eligible patients for a minimum of 12 months and potentially for up to 4 years.

Conditions

Sickle Cell Disease; Kidney Diseases, Chronic

Keywords

Machine Learning Models; Sickle Cell Disease; Chronic Kidney Disease; eGFR; Anemia, Sickle Cell; Albuminuria; Renal Insufficiency, Chronic; Renal Insufficiency; APOL1

Outcome Measures

Primary Outcomes (1)

• Develop two separate predictive models for progression of CKD (eGFR <90 mL/min/1·73 m2 and ≥25% drop in eGFR from baseline) and rapid eGFR decline (eGFR loss >3·0 mL/min/1·73 m2 per year) over the 12 months following the baseline clinic evaluation.

  At each visit following the first 12 months, rate of eGFR change will be calculated using data from current and earlier visits.

  12 months

Secondary Outcomes (2)

• Alternate definitions of CKD progression as eGFR decline <90 mL/min/1·73 m2 and ≥50% drop in eGFR from baseline, and rapid eGFR decline as eGFR loss >5·0 mL/min/1·73 m2 per year will be evaluated.

  12 months

• Evaluate the effect of APOL1 on the predictive capacity of ML models. Genomic DNA will be extracted from whole blood collected at baseline visits using standard techniques and genotyping will be performed as previously described.

  12 months

Study Arms (1)

Patients with sickle cell anemia

Prospective longitudinal study of patients with sickle cell anemia

Other: Biospecimen/DNA collection and analysis

Interventions

Biospecimen/DNA collection and analysis OTHER

Patients will be followed longitudinally with collection of CBC and chemistries as well as research biomarkers (urine, plasma, and genomic materials).

Patients with sickle cell anemia

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

Four hundred patients with SCD (HbSS or HbSB0 thalassemia) between the ages of 18 and 65 who meet the eligibility criteria and provide consent to participate in the study, will be enrolled in this prospective longitudinal trial.

You may qualify if:

• HbSS or HbSβ0 thalassemia, 18 - 65 years old;
• non-crisis, "steady state" with no acute pain episodes requiring medical contact in preceding 4 weeks;
• ability to understand the study requirements.

You may not qualify if:

• pregnant at enrollment;
• poorly controlled hypertension;
• long-standing diabetes with suspicion for diabetic nephropathy;
• connective tissue disease such as systemic lupus erythematosus (SLE);
• polycystic kidney disease or glomerular disease unrelated to SCD;
• stem cell transplantation;
• untreated human immunodeficiency virus (HIV), hepatitis B or C infection; h) history of cancer in last 5 years; i) End-stage renal disease (ESRD) on chronic dialysis; j) prior kidney transplantation.

Sponsors & Collaborators

• University of Tennessee: lead
• National Heart, Lung, and Blood Institute (NHLBI): collaborator
• University of Illinois at Chicago: collaborator
• University of Memphis: collaborator
• University of North Carolina, Charlotte: collaborator
• Wake Forest University: collaborator
• University of North Carolina, Chapel Hill: collaborator

Study Sites (3)

University of Illinois at Chicago

Chicago, Illinois, 60612, United States

RECRUITING

Wake Forest University

Winston-Salem, North Carolina, 27109, United States

NOT YET RECRUITING

The University of Tennessee Health Science Center

Memphis, Tennessee, 38104, United States

RECRUITING

Biospecimen

Retention: SAMPLES WITH DNA

Routine laboratory tests (CBC and chemistries), cystatin C, pregnancy tests (if female and of child-bearing capacity), urinalysis, spot urine albumin-creatinine ratio, and select plasma and urine biomarkers (ET-1, VEGF and soluble VCAM-1) and kidney function (urinary nephrin, KIM-1) and genomic DNA analyses for APOL1 G1/G2 alleles.

MeSH Terms

Conditions

Anemia, Sickle Cell; Renal Insufficiency, Chronic; Albuminuria; Renal Insufficiency

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; 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; Proteinuria; Urination Disorders; Urological Manifestations; Signs and Symptoms

Study Officials

• Kenneth I Ataga, MD

  The University of Tennessee Health Science Center

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Kenneth I Ataga, MD

CONTACT

901-448-2813; kataga@uthsc.edu

Santosh Saraf, MD

CONTACT

312-996-5680; ssaraf@uic.edu

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Principal Investigator

Study Record Dates

• First Submitted: December 17, 2021
• First Posted: January 28, 2022
• Study Start: July 5, 2022
• Primary Completion: January 31, 2026
• Study Completion: January 31, 2026
• Last Updated: December 14, 2023

Record last verified: 2023-12

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, CSR, ANALYTIC CODE
• Time Frame: From time of first patient enrollment to up to 7 years after completion of study.
• Access Criteria: Requests for data from academic investigators will be approved by the Executive Committee of the PREMIER Study. Following approval, de-identified data will be shared in a secure manner.

Locations

US (3)