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Cooperative Assessment of Late Effects for SCD Curative Therapies

NCT05153967 · Status: RECRUITING · Type: OBSERVATIONAL · Enrollment: 750

Last updated 2026-01-30

No results posted yet for this study

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.

Conditions

Sponsors & Collaborators

  • Johns Hopkins University

    collaborator OTHER

  • Children's National Research Institute

    collaborator OTHER

  • Emory University

    collaborator OTHER

  • University of Illinois at Chicago

    collaborator OTHER

  • Children's Healthcare of Atlanta

    collaborator OTHER

  • Vanderbilt University Medical Center

    lead OTHER

Principal Investigators

  • Michael R DeBaun, MD, MPH · Vanderbilt University Medical Center

Eligibility

Min Age
4 Years
Max Age
65 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2022-07-12
Primary Completion
2026-06-30
Completion
2027-02-28

Countries

  • United States

Study Locations

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Diseases
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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05153967 on ClinicalTrials.gov