Actively Recruiting
Comparing the Effectiveness of Matched Related Donor Hematopoietic Stem Cell Transplantation to Disease Modifying Therapy in Pediatric Patients With Sickle Cell Disease
Led by University of Rochester · Updated on 2025-06-19
480
Participants Needed
37
Research Sites
330 weeks
Total Duration
On this page
Sponsors
U
University of Rochester
Lead Sponsor
P
Patient-Centered Outcomes Research Institute
Collaborating Sponsor
AI-Summary
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.
CONDITIONS
Official Title
Comparing the Effectiveness of Matched Related Donor Hematopoietic Stem Cell Transplantation to Disease Modifying Therapy in Pediatric Patients With Sickle Cell Disease
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Pediatric patients aged between 3 and 20.9 years
- Children diagnosed with sickle cell anemia (HB SS or HBSB0 Thalassemia)
- Children who are candidates for matched related donor hematopoietic stem cell transplantation (MRD HCT) for the MRD HCT group
- Children receiving non-transplant disease-modifying therapies (NT-DMT) for SCD for the NT-DMT group
- Participants or their guardians must provide informed consent
- Participants must be willing to undergo assessments and follow-up visits over 3 years
You will not qualify if you...
- Children younger than 3 years or older than 20.9 years
- Children without sickle cell anemia or related conditions
- Children not eligible for MRD HCT or without a matched related donor in the MRD HCT group
- Children currently enrolled in other clinical trials that may interfere with this study
- Children unable to follow the study protocol or complete required follow-up
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 37 locations
1
Children's of Alabama (MRD-HCT)
Birmingham, Alabama, United States, 35294
Actively Recruiting
2
Children's of Alabama (NT-DMT)
Birmingham, Alabama, United States, 35294
Not Yet Recruiting
3
Nemours Children's Hospital, Delaware (MRD-HCT)
Wilmington, Delaware, United States, 19803
Actively Recruiting
4
Children's National Hospital (MRD-HCT)
Washington D.C., District of Columbia, United States, 20010
Actively Recruiting
5
Children's Healthcare of Atlanta (MRD-HCT)
Atlanta, Georgia, United States, 30329
Actively Recruiting
6
Comer Children's Hospital (MRD-HCT)
Chicago, Illinois, United States, 60637
Actively Recruiting
7
Riley Children's Hospital (MRD-HCT)
Indianapolis, Indiana, United States, 46202
Actively Recruiting
8
Riley's Children Hospital (NT-DMT)
Indianapolis, Indiana, United States, 46202
Not Yet Recruiting
9
Boston Children's Hospital (MRD-HCT)
Boston, Massachusetts, United States, 02115
Actively Recruiting
10
Boston Children's Hospital (NT-DMT)
Boston, Massachusetts, United States, 02115
Not Yet Recruiting
11
Wahington Univ in St Louis (NT-DMT)
St Louis, Missouri, United States, 63130
Not Yet Recruiting
12
Washington University in St Louis (MRD-HCT)
St Louis, Missouri, United States, 63130
Not Yet Recruiting
13
Hackensack University Hospital (MRD-HCT)
Hackensack, New Jersey, United States, 07601
Actively Recruiting
14
Roswell Park (MRD-HCT)
Buffalo, New York, United States, 14203
Actively Recruiting
15
Columbia Presbytarian (NT-DMT)
New York, New York, United States, 10032
Not Yet Recruiting
16
Cohen's Children Hospital (NT-DMT)
Queens, New York, United States, 11040
Not Yet Recruiting
17
University of Rochester (MRD-HCT)
Rochester, New York, United States, 14642
Not Yet Recruiting
18
University of Rochester (NT-DMT)
Rochester, New York, United States, 14642
Not Yet Recruiting
19
Children's Hospital at Montefiore (MRD HCT)
The Bronx, New York, United States, 10467
Not Yet Recruiting
20
Children's Hospital at Montefiore (NT-DMT)
The Bronx, New York, United States, 10467
Actively Recruiting
21
UNC Children's Hospital (MRD HCT)
Chapel Hill, North Carolina, United States, 27514
Not Yet Recruiting
22
UNC Children's Hospital (NT-DMT)
Chapel Hill, North Carolina, United States, 27599.
Not Yet Recruiting
23
Atrium Health (MRD-HCT)
Charlotte, North Carolina, United States, 28203
Actively Recruiting
24
Nationwide Children's Hospital (NT-DMT)
Columbus, Ohio, United States, 43205
Not Yet Recruiting
25
Oklahoma Children's Hospital (MRD-HCT)
Oklahoma City, Oklahoma, United States, 73104
Not Yet Recruiting
26
Children's Hospital of Philadelphia (MRD-HCT)
Philadelphia, Pennsylvania, United States, 19104
Actively Recruiting
27
St Jude Children Hospital (NT-DMT)
Memphis, Tennessee, United States, 38105
Not Yet Recruiting
28
St. Jude Children's Research Hospital (MRD-HCT)
Memphis, Tennessee, United States, 38105
Actively Recruiting
29
Texas Children's Hopsital (NT-DMT)
Houston, Texas, United States, 77030
Not Yet Recruiting
30
Texas Children's Hospital (MRD-HCT)
Houston, Texas, United States, 77030
Actively Recruiting
31
UT San Antonio (NT-DMT)
San Antonio, Texas, United States, 78229
Not Yet Recruiting
32
Alberta Children's Hospital (NT-DMT)
Calgary, Alberta, Canada, T3B 6A8
Not Yet Recruiting
33
Alberta Children's Hospital (MRD-HCT)
Calgary, Alberta, Canada, T3B6A8
Actively Recruiting
34
British Columbia Children's Hosptial (MRD HCT)
Vancouver, British Columbia, Canada
Not Yet Recruiting
35
HSC Winnipeg Children's Hospital/University of Manitoba (MRD-HCT)
Winnipeg, Manitoba, Canada, R3E 0V9
Actively Recruiting
36
The Hospital for Sick Children (MRD-HCT)
Toronto, Ontario, Canada, ON M5G 1E8
Not Yet Recruiting
37
Universite de Montreal / Ste Justine (MRD HCT)
Montreal, Quebec, Canada
Not Yet Recruiting
Research Team
J
John Horan, MD, MPH
CONTACT
P
Priya Kaushal, MPH
CONTACT
How is the study designed?
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
2
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