Cooperative Assessment of Late Effects for SCD Curative Therapies
Recruiting · NCT05153967 · Observational (researchers observe without assigning treatment) · Lead sponsor: Vanderbilt University Medical Center
View the official record on ClinicalTrials.gov →What this trial is about
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.
Who can take part
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
Where it is running
5 locations listed across 4 US states.
- Children's National Medical Center - Washington D.C., District of Columbia, United States
- Emory University School of Medicine - Atlanta, Georgia, United States
- Johns Hopkins Hospital - Baltimore, Maryland, United States
- National Institutes of Health Clinical Center - Bethesda, Maryland, United States
- Vanderbilt University Medical Center - Nashville, Tennessee, United States