RECRUITING

Cooperative Assessment of Late Effects for SCD Curative Therapies

Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

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.

Official Title

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

Quick Facts

Study Start:2022-07-12
Study Completion:2026-02
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:RECRUITING

Study ID

NCT05153967

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:4 Years to 65 Years
Sexes Eligible for Study:ALL
Accepts Healthy Volunteers:No
Standard Ages:CHILD, ADULT, OLDER_ADULT
Inclusion CriteriaExclusion Criteria
  1. Age 18 years or older
  2. Willing and able to provide informed consent
  3. Able to understand and follow study procedures
  4. Stable medical condition
  1. Pregnancy or breastfeeding
  2. Severe psychiatric disorders
  3. Active substance abuse
  4. Unstable medical conditions
  5. Inability to comply with study requirements

Contacts and Locations

Study Contact

Leshana Saint Jean, PhD
CONTACT
6158751992
leshana.saint.jean@vumc.org
Kristin Wuichet, PhD
CONTACT
6159366098
kristin.wuichet@vumc.org

Principal Investigator

Michael R DeBaun, MD, MPH
PRINCIPAL_INVESTIGATOR
Vanderbilt University Medical Center

Study Locations (Sites)

Children's National Medical Center
Washington, District of Columbia, 20010
United States
Emory University School of Medicine
Atlanta, Georgia, 30322
United States
Johns Hopkins Hospital
Baltimore, Maryland, 21287
United States
National Institutes of Health Clinical Center
Bethesda, Maryland, 20814
United States
Vanderbilt University Medical Center
Nashville, Tennessee, 37232-9000
United States

Collaborators and Investigators

Sponsor: Vanderbilt University Medical Center

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

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

Study Start Date2022-07-12
Study Completion Date2026-02

Study Record Updates

Study Start Date2022-07-12
Study Completion Date2026-02

Terms related to this study

Keywords Provided by Researchers

  • Myeloablative Autologous Gene Editing
  • Myeloablative Autologous Gene Therapy
  • Myeloablative allo-HSCT
  • Nonmyeloablative allo-HSCT
  • Disease-Modifying Therapy

Additional Relevant MeSH Terms

  • Sickle Cell Disease
  • Pulmonary Disease
  • Renal Disease
  • Heart Disease