Comparing the Effectiveness of Matched Related Donor Hematopoietic Stem Cell Transplantation to Disease Modifying Therapy in Pediatric Patients With Sickle Cell Disease (NCT06941389)
Comparing the Effectiveness of Matched Related Donor Hematopoietic Stem Cell Transplantation to Disease Modifying Therapy in Pediatric Patients With Sickle Cell Disease.
🩺 Plain-English Summary (8th-Grade Level)
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 join: Review the eligibility criteria below.
Location: Children's of Alabama (MRD-HCT) — Birmingham, Alabama
Age range: 3 Years to 20 Years
📋 "Do I Qualify?" — 1-Minute Self Screener
Instant ChecklistFull eligibility criteria are available on the official NIH protocol. View the criteria ↗
📍 All Participating Trial Locations (37 Sites)
Showing the first 25 of 37 sites.
- Children's of Alabama (MRD-HCT) — Birmingham, Alabama
- Children's of Alabama (NT-DMT) — Birmingham, Alabama
- Nemours Children's Hospital, Delaware (MRD-HCT) — Wilmington, Delaware
- Children's National Hospital (MRD-HCT) — Washington D.C., District of Columbia
- Children's Healthcare of Atlanta (MRD-HCT) — Atlanta, Georgia
- Comer Children's Hospital (MRD-HCT) — Chicago, Illinois
- Riley Children's Hospital (MRD-HCT) — Indianapolis, Indiana
- Riley's Children Hospital (NT-DMT) — Indianapolis, Indiana
- Boston Children's Hospital (MRD-HCT) — Boston, Massachusetts
- Boston Children's Hospital (NT-DMT) — Boston, Massachusetts
- Wahington Univ in St Louis (NT-DMT) — St Louis, Missouri
- Washington University in St Louis (MRD-HCT) — St Louis, Missouri
- Hackensack University Hospital (MRD-HCT) — Hackensack, New Jersey
- Roswell Park (MRD-HCT) — Buffalo, New York
- Columbia Presbytarian (NT-DMT) — New York, New York
- Cohen's Children Hospital (NT-DMT) — Queens, New York
- University of Rochester (MRD-HCT) — Rochester, New York
- University of Rochester (NT-DMT) — Rochester, New York
- Children's Hospital at Montefiore (MRD HCT) — The Bronx, New York
- Children's Hospital at Montefiore (NT-DMT) — The Bronx, New York
- UNC Children's Hospital (MRD HCT) — Chapel Hill, North Carolina
- UNC Children's Hospital (NT-DMT) — Chapel Hill, North Carolina
- Atrium Health (MRD-HCT) — Charlotte, North Carolina
- Nationwide Children's Hospital (NT-DMT) — Columbus, Ohio
- Oklahoma Children's Hospital (MRD-HCT) — Oklahoma City, Oklahoma
📞 Contact the Study Coordinator
Phone: (585) 274-2345
Email: John_Horan@URMC.Rochester.edu
Coordinator details come directly from the registered NIH protocol. Confirm the study is still recruiting before traveling.
💰 Cost, Insurance & Patient Rights
- 100% Free Items: The investigational treatment and protocol-required procedures are supplied at zero cost by the sponsor.
- Routine Patient Care: Under federal law (ACA § 2709) and many state statutes, routine care costs are covered during participation.
- Voluntary Participation: You may withdraw at any time without affecting your standard medical care.
More studies in Sickle Cell Disease (SCD)
- Study of Two Doses of Crizanlizumab Versus Placebo in Adolescent and Adult Sickle Cell Disease Patients — Atlanta, Georgia
- Megakaryocyte Heterogeneity in Sickle Cell Disease — Pointe-à-Pitre,
- Optimizing Hydroxyurea Dosage With Pharmakokinetic in Patients Suffering of Moderate to Severe Sickle Cell Anemia — Montreal, Quebec
- Clinical and Biomarker Effects of Depot Medroxyprogesterone Acetate in Females With Sickle Cell Disease — Atlanta, Georgia
Explore by condition: All Sickle Cell Disease (SCD) clinical trials
💡 How to Participate
- Check the checklist above to see if you may qualify.
- Contact the study coordinator using the phone/email above.
- Ask about the visit schedule, what is covered at no cost, and any travel support.
- Confirm with your own doctor before making a decision.