Comparing the Effectiveness of Matched Related Donor Hematopoietic Stem Cell Transplantation to Disease Modifying Therapy in Pediatric Patients With Sickle Cell Disease

Recruiting now

Conditions studied: Sickle Cell Disease (SCD)

In brief

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.

Key facts

Study ID

NCT06941389

Run by

University of Rochester

People needed

480

Starts

2024-06-01

Expected to finish

2030-10-01

Last updated by the study team

2026-06-04

Who can join

Age: 3 and older, up to 20. Sex: any. Healthy volunteers: not accepted.

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.

Where it is running

• Hackensack University Hospital (MRD-HCT) — Hackensack, New Jersey, United States (enrolling)
• Atrium Health (MRD-HCT) — Charlotte, North Carolina, United States (enrolling)
• Roswell Park (MRD-HCT) — Buffalo, New York, United States (enrolling)
• Riley Children's Hospital (MRD-HCT) — Indianapolis, Indiana, United States (enrolling)
• Children's Hospital of Philadelphia (MRD-HCT) — Philadelphia, Pennsylvania, United States (enrolling)
• Nemours Children's Hospital, Delaware (MRD-HCT) — Wilmington, Delaware, United States (enrolling)
• St. Jude Children's Research Hospital (MRD-HCT) — Memphis, Tennessee, United States (enrolling)
• Boston Children's Hospital (MRD-HCT) — Boston, Massachusetts, United States (enrolling)
• Texas Children's Hospital (MRD-HCT) — Houston, Texas, United States (enrolling)
• Children's National Hospital (MRD-HCT) — Washington D.C., District of Columbia, United States (enrolling)
• Children's Healthcare of Atlanta (MRD-HCT) — Atlanta, Georgia, United States (enrolling)
• Alberta Children's Hospital (MRD-HCT) — Calgary, Alberta, Canada (enrolling)
• Children's Hospital at Montefiore (NT-DMT) — The Bronx, New York, United States (enrolling)
• HSC Winnipeg Children's Hospital/University of Manitoba (MRD-HCT) — Winnipeg, Manitoba, Canada (enrolling)
• Comer Children's Hospital (MRD-HCT) — Chicago, Illinois, United States (enrolling)
• Children's of Alabama (MRD-HCT) — Birmingham, Alabama, United States (enrolling)
• Texas Children's Hopsital (NT-DMT) — Houston, Texas, United States
• UT San Antonio (NT-DMT) — San Antonio, Texas, United States
• Alberta Children's Hospital (NT-DMT) — Calgary, Alberta, Canada
• British Columbia Children's Hosptial (MRD HCT) — Vancouver, British Columbia, Canada
• The Hospital for Sick Children (MRD-HCT) — Toronto, Ontario, Canada
• Universite de Montreal / Ste Justine (MRD HCT) — Montreal, Quebec, Canada
• Children's of Alabama (NT-DMT) — Birmingham, Alabama, United States
• Riley's Children Hospital (NT-DMT) — Indianapolis, Indiana, United States
• Boston Children's Hospital (NT-DMT) — Boston, Massachusetts, United States

Full record on ClinicalTrials.gov

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