Condition: Sickle Cell Disease · Pulmonary Disease · Renal Disease · Sponsor: Vanderbilt University Medical Center
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 ther…
This description comes directly from the study's public registry record.
Leshana Saint Jean, PhD · 6158751992 · leshana.saint.jean@vumc.org
Kristin Wuichet, PhD · 6159366098 · kristin.wuichet@vumc.org
Always discuss trial participation with your own doctor first.
| Children's National Medical Center | Washington D.C., District of Columbia, United States | Not Yet Recruiting |
| Emory University School of Medicine | Atlanta, Georgia, United States | Not Yet Recruiting |
| Johns Hopkins Hospital | Baltimore, Maryland, United States | Not Yet Recruiting |
| National Institutes of Health Clinical Center | Bethesda, Maryland, United States | Not Yet Recruiting |
| Vanderbilt University Medical Center | Nashville, Tennessee, United States | Recruiting |
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Source record: clinicaltrials.gov/study/NCT05153967