U01 Cooperative Assessment of Late Effects for Sickle Cell Disease Curative Therapies

Status: Recruiting
Location: See all (5) locations...
Study Type: Observational
SUMMARY

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.

Eligibility
Participation Requirements
Sex: All
Minimum Age: 4
Maximum Age: 65
Healthy Volunteers: f
View:

• 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

Locations
United States
Washington, D.c.
Children's National Medical Center
NOT_YET_RECRUITING
Washington
Georgia
Emory University School of Medicine
NOT_YET_RECRUITING
Atlanta
Maryland
Johns Hopkins Hospital
NOT_YET_RECRUITING
Baltimore
National Institutes of Health Clinical Center
NOT_YET_RECRUITING
Bethesda
Tennessee
Vanderbilt University Medical Center
RECRUITING
Nashville
Contact Information
Primary
Leshana Saint Jean, PhD
leshana.saint.jean@vumc.org
6158751992
Backup
Kristin Wuichet, PhD
kristin.wuichet@vumc.org
6159366098
Time Frame
Start Date: 2022-07-12
Estimated Completion Date: 2026-02
Participants
Target number of participants: 750
Treatments
Pediatric Myeloablative allo-HSCT
Participants ages 4 to 17 years old with SCD who underwent or are scheduled to undergo myeloablative allo-HSCT.
Pediatric Standard Disease-Modifying Therapy
Participants ages 4 to 17 years old with SCD who receive standard therapy.
Adult Non-Myeloablative allo-HSCT
Participants ages 18 to 65 years old with SCD who underwent or are scheduled to undergo non-myeloablative allo-HSCT.
Adult Standard Disease-Modifying Therapy
Participants ages 18 to 65 years old with SCD who receive standard therapy.
Sponsors
Collaborators: Emory University, Children's National Research Institute, Children's Healthcare of Atlanta, Johns Hopkins University, University of Illinois at Chicago
Leads: Vanderbilt University Medical Center

This content was sourced from clinicaltrials.gov