Comparing the Effectiveness of Matched Related Donor Hematopoietic Stem Cell Transplantation to Disease Modifying Therapy in Pediatric Patients With Sickle Cell Disease
Recruiting · NCT06941389 · Observational (researchers observe without assigning treatment) · Lead sponsor: University of Rochester
View the official record on ClinicalTrials.gov →What this trial is about
The WeDecide study is a large observational study comparing the long-term effects of matched related donor hematopoietic stem cell transplantation (MRD HCT) and non-transplant disease-modifying therapies (NT-DMT) for pediatric patients with sickle cell disease (SCD). The study aims to assess health-related quality of life (HRQoL), cognitive function, risks, and benefits of both treatments, including survival rates, chronic complications, and organ damage prevention. With 160 children in the MRD HCT group and 320 in the NT-DMT group, aged 3-20.9 years, the study will follow participants for three years, examining factors like disease severity, treatment history, and social determinants of health. By providing a comprehensive comparison, the study seeks to inform clinical decisions and improve understanding of SCD treatment outcomes, ultimately supporting families and healthcare providers in choosing the best treatment options.
Who can take part
Inclusion criteria
- Pediatric patients aged between 3 and 20.9 years.
- 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 younger than 3 years or older than 20.9 years.
- 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.
Where it is running
37 locations listed across 16 US states.
- 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