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Cooperative Assessment of Late Effects for SCD Curative Therapies
U01 Cooperative Assessment of Late Effects for Sickle Cell Disease Curative Therapies
Who can join
Ages 4 to 65 · All sexes
Full eligibility criteria
Inclusion Criteria * Confirmed laboratory diagnosis of SCD * Ability to give informed consent * Ability to provide pre- and post-curative therapy data * Treated with either one HSCT or with standard disease-modifying therapy Exclusion Criteria •History of non-compliance
About the study
Sickle Cell Disease is one of the most common genetic diseases in the United States, occurring in approximately 1 in 400 births. Approximately 100,000 individuals are diagnosed with SCD in the United States. Mortality for children with SCD has decreased substantially over the past 4 decades, with \>99% of those born in high resource settings, including the United States, France, and England, now surviving to 18 years of age. However, the life expectancy of adults with SCD is severely shortened. Dysfunction of the heart, lung, and kidney is directly associated with decreased life expectancy. With the variety of curative therapies that are now available for SCD, long-term health outcomes studies are time-sensitive. As of now, efforts to determine long-term health outcomes following curative therapies for SCD have been limited. Though curative therapies initially should provide a cure for symptoms of SCD, there is the risk of late health outcomes to consider. Defining health outcomes following curative therapy is essential to improve personalized decision-making when considering curative versus disease-modifying therapeutic options. The primary goal of this study is to determine whether curative therapies for individuals with SCD will result in improved or worsening heart, lung, and kidney damage when compared to individuals with SCD receiving standard therapy. The investigators will also explore whether certain genes are associated with a good or bad outcome after curative therapy for SCD.
Sponsor: Vanderbilt University Medical Center · Participants: 750 · Started: Jul 12, 2022
Contact the study team
- Leshana Saint Jean, PhD · Phone: 6158751992
- Kristin Wuichet, PhD · Phone: 6159366098
Official record on ClinicalTrials.gov — NCT05153967
Locations in the U.S.
| District of Columbia | Children's National Medical Center, Washington D.C. (Not yet recruiting) |
| Georgia | Emory University School of Medicine, Atlanta (Not yet recruiting) |
| Maryland | Johns Hopkins Hospital, Baltimore (Not yet recruiting) National Institutes of Health Clinical Center, Bethesda (Not yet recruiting) |
| Tennessee | Vanderbilt University Medical Center, Nashville |
Conditions
From ClinicalTrials.gov, data retrieved Sep 30, 2026. Each study sets its own eligibility; the study team decides who can join.