Kidney Function in Sickle Cell Anemia

  • STATUS
    Recruiting
  • days left to enroll
    13
  • participants needed
    300
  • sponsor
    University of North Carolina, Chapel Hill
Updated on 30 January 2022
anemia
thalassemia

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"

Description

Sickle cell disease is a severe monogenic disorder which affects approximately 80,000 patients in the US. It is characterized by a vasculopathy with involvement of multiple organs and resulting in complications such as ischemic stroke, pulmonary hypertension, autosplenectomy, priapism, as well as chronic kidney disease (CKD). Despite the high prevalence of CKD and its known association with increased mortality, the natural history of CKD and the factors associated with changes in kidney function in patients with SCD remain incompletely defined. Furthermore, the available treatment options for albuminuria, an early manifestation of CKD, in patients with SCD are limited. In fact, no controlled studies have confirmed the long-term efficacy of angiotensin-converting enzyme (ACE) inhibitors, the current "standard of care." There is increasing evidence for a contribution of endothelial dysfunction to the pathophysiology of albuminuria in SCD. The association of biomarkers of endothelial function with albuminuria provides opportunities, not only to assess the effect of therapies which improve endothelial function, but also to evaluate the predictive value of these biomarkers for a decline in kidney function. The long-range goal is to develop a model to identify patients at particularly high risk for a decline in kidney function.

In this study, the investigators will evaluate rate of change in kidney function (decline in estimated glomerular filtration rates and increase in albuminuria) and identify biomarkers of endothelial function, metabolomic profiles and clinical characteristics for the worsening of kidney function and for a rapid decline in kidney function. At the conclusion of this proposed work, the investigators will have an improved understanding of the natural history of CKD in sickle cell anemia. With the limited available therapies for the treatment of albuminuria in SCD and the paucity of data on the long-term efficacy of available pharmacotherapies, identification of biomarkers for the progression of CKD will facilitate the development of treatments which may be more effective than the current "standard of care."

Details
Condition Sickle Cell Disease, Kidney Failure, Chronic
Clinical Study IdentifierNCT03277547
SponsorUniversity of North Carolina, Chapel Hill
Last Modified on30 January 2022

Eligibility

Yes No Not Sure

Inclusion Criteria

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

Exclusion Criteria

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
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