Comparing the Effectiveness of Matched Related Donor Hematopoietic Stem Cell Transplantation to Disease Modifying Therapy in Pediatric Patients With Sickle Cell Disease
NCT ID: NCT06941389
Last Updated: 2025-06-19
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
480 participants
OBSERVATIONAL
2024-06-01
2030-10-01
Brief Summary
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Detailed Description
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The study includes two groups: 160 children receiving MRD HCT and 320 children receiving NT-DMT. Participants, aged 3-20.9 years, are being followed for three years. The MRD HCT group will be assessed before the transplant and then at several points post-transplant. The NT-DMT group will be assessed at the start of the study and then annually for three years.
The research also considers factors like disease severity, treatment history, and social determinants of health (such as family finances and caregiver health literacy) to better understand how these elements might influence treatment outcomes. The study tracks the use of disease-modifying therapies, as well as hospital visits and other care events, throughout the three years. It will also monitor survival rates and other important health outcomes.
This study is significant because it is the first large-scale research comparing these two treatment options for SCD in children. The results will provide essential insights into how these treatments impact long-term health and help guide clinical decisions and treatment recommendations. The goal is to help families and healthcare providers make informed decisions about the best treatment options for SCD.
The study uses advanced methods to ensure fair comparisons between the two groups by accounting for differences in their characteristics. It will also adjust for any factors that could influence the results, helping to identify meaningful differences in health outcomes between the two treatments. Ultimately, the WeDecide study aims to improve our understanding of sickle cell disease treatment and provide a foundation for future research into new therapies.
Conditions
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Study Design
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COHORT
PROSPECTIVE
Study Groups
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NT-DMT Cohort
This group includes 320 participants from U.S. and Canadian sites receiving non-transplant disease-modifying therapies, monitored annually over three years to assess health-related quality of life, cognitive function, disease progression, and healthcare utilization.
No interventions assigned to this group
MRD HCT Cohort
This group includes 160 participants from U.S. and Canadian sites receiving non-transplant disease-modifying therapies, monitored annually over three years to assess health-related quality of life, cognitive function, disease progression, and healthcare utilization.
No interventions assigned to this group
Eligibility Criteria
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Inclusion Criteria
* Children diagnosed with sickle cell Anemia (HB SS or HBSB0 Thalassemia)
* For the MRD HCT group, children who are candidates for matched related donor hematopoietic stem cell transplantation (MRD HCT).
* For the NT-DMT group, children who are receiving non-transplant disease-modifying therapies (NT-DMT) for SCD.
* Participants (or their guardians) must provide informed consent to be part of the study.
* Participants must be willing to undergo the necessary assessments and follow-up visits over the 3-year study period.
Exclusion Criteria
* Children who do not have sickle cell anemia or related conditions.
* For the MRD HCT group, children who are not eligible for the transplant or do not have a matched related donor.
* Children who are currently enrolled in other clinical trials that might interfere with the WeDecide study.
* Children who are unable to adhere to the study protocol or follow-up requirements.
3 Years
20 Years
ALL
No
Sponsors
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Patient-Centered Outcomes Research Institute
OTHER
University of Rochester
OTHER
Responsible Party
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John Horan
Professor - Department of Pediatrics, Hematology and Oncology (SMD)
Locations
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Children's of Alabama (MRD-HCT)
Birmingham, Alabama, United States
Children's of Alabama (NT-DMT)
Birmingham, Alabama, United States
Nemours Children's Hospital, Delaware (MRD-HCT)
Wilmington, Delaware, United States
Children's National Hospital (MRD-HCT)
Washington D.C., District of Columbia, United States
Children's Healthcare of Atlanta (MRD-HCT)
Atlanta, Georgia, United States
Comer Children's Hospital (MRD-HCT)
Chicago, Illinois, United States
Riley Children's Hospital (MRD-HCT)
Indianapolis, Indiana, United States
Riley's Children Hospital (NT-DMT)
Indianapolis, Indiana, United States
Boston Children's Hospital (MRD-HCT)
Boston, Massachusetts, United States
Boston Children's Hospital (NT-DMT)
Boston, Massachusetts, United States
Wahington Univ in St Louis (NT-DMT)
St Louis, Missouri, United States
Washington University in St Louis (MRD-HCT)
St Louis, Missouri, United States
Hackensack University Hospital (MRD-HCT)
Hackensack, New Jersey, United States
Roswell Park (MRD-HCT)
Buffalo, New York, United States
Columbia Presbytarian (NT-DMT)
New York, New York, United States
Cohen's Children Hospital (NT-DMT)
Queens, New York, United States
University of Rochester (MRD-HCT)
Rochester, New York, United States
University of Rochester (NT-DMT)
Rochester, New York, United States
Children's Hospital at Montefiore (MRD HCT)
The Bronx, New York, United States
Children's Hospital at Montefiore (NT-DMT)
The Bronx, New York, United States
UNC Children's Hospital (MRD HCT)
Chapel Hill, North Carolina, United States
UNC Children's Hospital (NT-DMT)
Chapel Hill, North Carolina, United States
Atrium Health (MRD-HCT)
Charlotte, North Carolina, United States
Nationwide Children's Hospital (NT-DMT)
Columbus, Ohio, United States
Oklahoma Children's Hospital (MRD-HCT)
Oklahoma City, Oklahoma, United States
Children's Hospital of Philadelphia (MRD-HCT)
Philadelphia, Pennsylvania, United States
St Jude Children Hospital (NT-DMT)
Memphis, Tennessee, United States
St. Jude Children's Research Hospital (MRD-HCT)
Memphis, Tennessee, United States
Texas Children's Hopsital (NT-DMT)
Houston, Texas, United States
Texas Children's Hospital (MRD-HCT)
Houston, Texas, United States
UT San Antonio (NT-DMT)
San Antonio, Texas, United States
Alberta Children's Hospital (NT-DMT)
Calgary, Alberta, Canada
Alberta Children's Hospital (MRD-HCT)
Calgary, Alberta, Canada
British Columbia Children's Hosptial (MRD HCT)
Vancouver, British Columbia, Canada
HSC Winnipeg Children's Hospital/University of Manitoba (MRD-HCT)
Winnipeg, Manitoba, Canada
The Hospital for Sick Children (MRD-HCT)
Toronto, Ontario, Canada
Universite de Montreal / Ste Justine (MRD HCT)
Montreal, Quebec, Canada
Countries
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Central Contacts
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Facility Contacts
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Other Identifiers
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BPS-2023C1-31041
Identifier Type: OTHER_GRANT
Identifier Source: secondary_id
BPS-2023C1-31041
Identifier Type: -
Identifier Source: org_study_id
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