Cooperative Assessment of Late Effects for SCD Curative Therapies
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:
🦠 Sickle Cell Disease 🦠 Pulmonary Disease 🦠 Renal Disease 🦠 Heart Disease
πŸ—“οΈ Study Start (Actual) 12 July 2022
πŸ—“οΈ Primary Completion (Estimated) December 2025
βœ… Study Completion (Estimated) February 2026
πŸ‘₯ Enrollment (Estimated) 750
πŸ”¬ Study Type OBSERVATIONAL
πŸ“Š Phase N/A
Locations:
πŸ“ Washington, District of Columbia, United States
πŸ“ Atlanta, Georgia, United States
πŸ“ Baltimore, Maryland, United States
πŸ“ Bethesda, Maryland, United States
πŸ“ Nashville, Tennessee, United States

πŸ“‹ Eligibility Criteria

Description

  • 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
  • β€’History of non-compliance
Ages Eligible for Study: 4 Years to 65 Years (CHILD, ADULT, OLDER_ADULT)
Sexes Eligible for Study: ALL
Accepts Healthy Volunteers: No

πŸ—“οΈ Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

  • First Submitted 25 October 2021
  • First Submitted that Met QC Criteria 29 November 2021
  • First Posted 10 December 2021

Study Record Updates

  • Last Update Submitted that Met QC Criteria 7 September 2023
  • Last Update Posted 8 September 2023
  • Last Verified September 2023