NCT06941389 · University of Rochester
Comparing the Effectiveness of Matched Related Donor Hematopoietic Stem Cell Transplantation to Disease Modifying Therapy in Pediatric Patients With Sickle Cell Disease
(WeDecide)
What this study 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).
View original scientific description
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 participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
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
- Birmingham, Alabama
- Wilmington, Delaware
- Washington D.C., District of Columbia
- Atlanta, Georgia
- Chicago, Illinois
- Indianapolis, Indiana
- Boston, Massachusetts
- St Louis, Missouri
- Hackensack, New Jersey
- Buffalo, New York
- New York, New York
- Queens, New York
And 10 more locations — see the full list below.
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced Jun 4, 2026 · Source of record for eligibility and locations