Cooperative Assessment of Late Effects for SCD Curative Therapies

Part of paid clinical trials in Washington D.C., District of Columbia.

Sponsor
Vanderbilt University Medical Center
Study ID
NCT05153967
Status
Recruiting
Apply to this trial

Conditions

Eligibility Criteria

Sex
ALL
Age
4 Years - 65 Years
Healthy Volunteers
Not accepted

Study Details

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.

Key Dates

Start date
Jul 12, 2022
Status verified
Jan 2026
Primary completion
Jun 30, 2026
Completion
Feb 28, 2027

Study Design

Enrollment
750 participants (estimated)

Arms

  • Arm: Pediatric Myeloablative allo-HSCT
    Participants ages 4 to 17 years old with SCD who underwent or are scheduled to undergo myeloablative allo-HSCT.
  • Arm: Pediatric Standard Disease-Modifying Therapy
    Participants ages 4 to 17 years old with SCD who receive standard therapy.
  • Arm: 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.
  • Arm: Adult Standard Disease-Modifying Therapy
    Participants ages 18 to 65 years old with SCD who receive standard therapy.

Primary Outcome Measure

Measurement of longitudinal change in FEV1 [ Time Frame: Through study completion, an average of four years ]

Central Contacts

Locations (5)

FacilityCityStateZIPSite coordinators
Children's National Medical CenterWashington D.C.District of Columbia20010
Allistair Abraham, MD
[email protected]
202-476-6690
Emory University School of MedicineAtlantaGeorgia30322
Vivien Sheehan, MD, PhD
[email protected]
404-727-7100
Johns Hopkins HospitalBaltimoreMaryland21287
Richard Jones, MD
[email protected]
667-312-2400
National Institutes of Health Clinical CenterBethesdaMaryland20814
Courtney Fitzhugh, MD
[email protected]
301-402-6496
Vanderbilt University Medical CenterNashvilleTennessee37232-9000
Michael R. DeBaun, MD, MPH
[email protected]
615-875-3040

Find similar trials in Washington D.C., DC

By condition
Sickle cell disease
By research site
Children's National Medical Center· Washington D.C., DCEmory University School of Medicine· Atlanta, GAJohns Hopkins Hospital· Baltimore, MDNational Institutes of Health Clinical Center· Bethesda, MDVanderbilt University Medical Center· Nashville, TN

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