Clinical trial NCT02239016: Screening Patients With Sickle Cell Disease for Kidney Damage
Official title: Screening Patients With Sickle Cell Disease for Kidney Damage
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Sickle Cell Disease
Keywords: Sickle cell disease
Hgb SS
Hgb SC
Sβ-Thalassemia
Lead sponsor: Children's Hospital Medical Center, Cincinnati
Sponsor class: OTHER
First posted: 2014-09-12
Enrollment: 320
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Sickle cell clinics at various universities and hospitals.

Brief summary: This study aims to study the temporal course of sickle nephropathy and assess novel biomarkers that can predict patients prone to nephropathy.

Detailed description: Sickle cell disease causes kidney damage that gets worse with increasing age, leading to chronic kidney disease and kidney failure in nearly one third of patients with sickle cell disease. Some patients develop kidney damage at a young age and others show mild kidney damage at older ages. We do not know the natural progression of kidney damage in sickle cell disease patients, nor do we know who is more prone to develop severe kidney damage. Therefore, currently, there are no preventative measures or treatments for sickle cell related kidney disease. The purpose of this research study is to collect data that will help in assessing the progression of kidney damage in sickle cell disease, develop novel urine and blood tests that can predict kidney damage early, and developing treatment ideas for intervention and prevention of kidney damage that eventually leads to kidney failure.

Eligibility criteria: Inclusion Criteria:

* Sickle cell disease (i.e. Hgb SS, Hgb SC, Sβ-Thalassemia)
* Individuals at baseline/steady state (absence of fever or acute sickle event, defined as vaso-occlusive pain crises, acute chest syndrome, splenic sequestration, stroke, priapism) for three weeks.
* Adult Subjects \> 18 years of age: ability to consent to donate blood and/or urine for research purposes only.
* Newborn to \< 18 years of age: ability of parent/legal guardian to consent for peripheral blood and/or urine samples to be obtained for research purposes only.

Exclusion Criteria:

* Hematologic malignancy
* Patients that either do not have the ability to undergo the informed consent process or whose parent/legal guardian does not have the ability to undergo the informed consent process

Primary outcomes:
measure: Rate of progression of microalbuminuria.
timeFrame: Baseline through 36 months
Secondary outcomes:
measure: Evaluation of novel urinary biomarkers.
timeFrame: baseline, year 1, year 2 and year 3
Other outcomes:
Locations:
city: Atlanta
country: United States
facility: Emory University (Children's Healthcare of Atlanta Pediatric Hospital)
state: Georgia
city: Louisville
country: United States
facility: Univeristy of Louisville (Kosair Children's Hospital)
state: Kentucky
city: Bethesda
country: United States
facility: National Institutes of Health Clinical Center
state: Maryland
city: Akron
country: United States
facility: Akron Childen's Hospital
state: Ohio
Study officials:
affiliation: Children's Hospital Medical Center, Cincinnati
name: Punam Malik, MD
role: PRINCIPAL_INVESTIGATOR
References:
citation: Sundaram N, Bennett M, Wilhelm J, Kim MO, Atweh G, Devarajan P, Malik P. Biomarkers for early detection of sickle nephropathy. Am J Hematol. 2011 Jul;86(7):559-66. doi: 10.1002/ajh.22045. Epub 2011 May 31.
citation: Niss O, Lane A, Asnani MR, Yee ME, Raj A, Creary S, Fitzhugh C, Bodas P, Saraf SL, Sarnaik S, Devarajan P, Malik P. Progression of albuminuria in patients with sickle cell anemia: a multicenter, longitudinal study. Blood Adv. 2020 Apr 14;4(7):1501-1511. doi: 10.1182/bloodadvances.2019001378.