Comparing the Effectiveness of Matched Related Donor Hematopoietic Stem Cell Transplantation to Disease Modifying Therapy in Pediatric Patients With Sickle Cell Disease
WeDecide
1 other identifier
observational
480
2 countries
37
Brief Summary
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P75+ for all trials
Started Jun 2024
Longer than P75 for all trials
37 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
Study Start
First participant enrolled
June 1, 2024
CompletedFirst Submitted
Initial submission to the registry
April 15, 2025
CompletedFirst Posted
Study publicly available on registry
April 23, 2025
CompletedPrimary Completion
Last participant's last visit for primary outcome
October 1, 2030
ExpectedStudy Completion
Last participant's last visit for all outcomes
October 1, 2030
June 19, 2025
June 1, 2025
6.3 years
April 15, 2025
June 17, 2025
Conditions
Keywords
Outcome Measures
Primary Outcomes (2)
Health-Related Quality of Life as Measured by the Pediatric Quality of Life Inventory (PedsQL) Generic Core Scale (Parent-Proxy Report)
Measures physical, emotional, social, and school functioning as reported by parents.
Baseline, 1-year, 2-year, 3-year follow-up
Cognitive Function as Measured by the NIH Toolbox Cognitive Battery
Assesses the following cognitive domains: attention, executive function, memory, language, processing speed, and working memory.
Baseline, 1-year, 2-year, 3-year follow-up
Secondary Outcomes (7)
Health-Related Quality of Life as Measured by the Pediatric Quality of Life Inventory (PedsQL) Generic Core Scale (Patient Reported)
Baseline, 1-year, 2-year, 3-year follow-up
Health Related Quality of Life Using the PedsQL Sickle Cell Disease Module (Patient and Parent-Proxy Reported)
Baseline, 1-year, 2-year, 3-year follow-up
Peds QL Family Impact Module (Parent Reported)
Baseline, 1-, 2- and 3-year follow-up
Health Quality of Life as Measured by the Patient Reported Outcomes Measurement Information System (PROMIS, Patient and Parent-Proxy Reports)
Baseline, 1-year, 2-year, 3-year follow-up
The Patient Reported Outcomes Measure System (PROMIS) Pain Behavior subscale (Patient and Parent-Proxy Reports)
Baseline, 1-year, 2-year and 3-year follow-up
- +2 more secondary outcomes
Study Arms (2)
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.
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.
Eligibility Criteria
The WeDecide study includes 480 pediatric patients with sickle cell disease (SCD) from two sub-studies: Project Sickle Cure (PSC), focusing on patients receiving transplant through the Sickle Cell Transplant Advocacy Research Alliance (STAR), and the Globin Research Network for Data and Discovery (GRNDaD), a registry of patients on non-transplant disease-modifying therapies (NT-DMT) run by the National Association of Sickle Cell Centers (NASCC). MRD HCT Group: 160 children receiving matched related donor hematopoietic stem cell transplantation (MRD HCT) will be assessed before and at multiple post-transplant time points to evaluate long-term effects. NT-DMT Group: 320 children receiving non-transplant disease-modifying therapies (NT-DMT) will be assessed at study start and annually for three years to track treatment effects.
You may qualify if:
- 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.
You may not qualify if:
- 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.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (37)
Children's of Alabama (MRD-HCT)
Birmingham, Alabama, 35294, United States
Children's of Alabama (NT-DMT)
Birmingham, Alabama, 35294, United States
Nemours Children's Hospital, Delaware (MRD-HCT)
Wilmington, Delaware, 19803, United States
Children's National Hospital (MRD-HCT)
Washington D.C., District of Columbia, 20010, United States
Children's Healthcare of Atlanta (MRD-HCT)
Atlanta, Georgia, 30329, United States
Comer Children's Hospital (MRD-HCT)
Chicago, Illinois, 60637, United States
Riley Children's Hospital (MRD-HCT)
Indianapolis, Indiana, 46202, United States
Riley's Children Hospital (NT-DMT)
Indianapolis, Indiana, 46202, United States
Boston Children's Hospital (MRD-HCT)
Boston, Massachusetts, 02115, United States
Boston Children's Hospital (NT-DMT)
Boston, Massachusetts, 02115, United States
Wahington Univ in St Louis (NT-DMT)
St Louis, Missouri, 63130, United States
Washington University in St Louis (MRD-HCT)
St Louis, Missouri, 63130, United States
Hackensack University Hospital (MRD-HCT)
Hackensack, New Jersey, 07601, United States
Roswell Park (MRD-HCT)
Buffalo, New York, 14203, United States
Columbia Presbytarian (NT-DMT)
New York, New York, 10032, United States
Cohen's Children Hospital (NT-DMT)
Queens, New York, 11040, United States
University of Rochester (MRD-HCT)
Rochester, New York, 14642, United States
University of Rochester (NT-DMT)
Rochester, New York, 14642, United States
Children's Hospital at Montefiore (MRD HCT)
The Bronx, New York, 10467, United States
Children's Hospital at Montefiore (NT-DMT)
The Bronx, New York, 10467, United States
UNC Children's Hospital (MRD HCT)
Chapel Hill, North Carolina, 27514, United States
UNC Children's Hospital (NT-DMT)
Chapel Hill, North Carolina, 27599., United States
Atrium Health (MRD-HCT)
Charlotte, North Carolina, 28203, United States
Nationwide Children's Hospital (NT-DMT)
Columbus, Ohio, 43205, United States
Oklahoma Children's Hospital (MRD-HCT)
Oklahoma City, Oklahoma, 73104, United States
Children's Hospital of Philadelphia (MRD-HCT)
Philadelphia, Pennsylvania, 19104, United States
St Jude Children Hospital (NT-DMT)
Memphis, Tennessee, 38105, United States
St. Jude Children's Research Hospital (MRD-HCT)
Memphis, Tennessee, 38105, United States
Texas Children's Hopsital (NT-DMT)
Houston, Texas, 77030, United States
Texas Children's Hospital (MRD-HCT)
Houston, Texas, 77030, United States
UT San Antonio (NT-DMT)
San Antonio, Texas, 78229, United States
Alberta Children's Hospital (NT-DMT)
Calgary, Alberta, T3B 6A8, Canada
Alberta Children's Hospital (MRD-HCT)
Calgary, Alberta, T3B6A8, Canada
British Columbia Children's Hosptial (MRD HCT)
Vancouver, British Columbia, Canada
HSC Winnipeg Children's Hospital/University of Manitoba (MRD-HCT)
Winnipeg, Manitoba, R3E 0V9, Canada
The Hospital for Sick Children (MRD-HCT)
Toronto, Ontario, ON M5G 1E8, Canada
Universite de Montreal / Ste Justine (MRD HCT)
Montreal, Quebec, Canada
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Central Study Contacts
Study Design
- Study Type
- observational
- Observational Model
- COHORT
- Time Perspective
- PROSPECTIVE
- Sponsor Type
- OTHER
- Responsible Party
- PRINCIPAL INVESTIGATOR
- PI Title
- Professor - Department of Pediatrics, Hematology and Oncology (SMD)
Study Record Dates
First Submitted
April 15, 2025
First Posted
April 23, 2025
Study Start
June 1, 2024
Primary Completion (Estimated)
October 1, 2030
Study Completion (Estimated)
October 1, 2030
Last Updated
June 19, 2025
Record last verified: 2025-06
Data Sharing
- IPD Sharing
- Will not share