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Comparing stem cell transplantation to disease-modifying therapy for children with sickle cell disease

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

Observational University of Rochester · NCT06941389

This study is trying to see how well stem cell transplants work compared to other treatments for children with sickle cell disease over three years.

Quick facts

Study typeObservational
Enrollment480 (estimated)
Ages3 Years to 20 Years
SexAll
SponsorUniversity of Rochester Academic / other
Locations37 sites (Birmingham, Alabama and 36 other locations)
Trial IDNCT06941389 on ClinicalTrials.gov

What this trial studies

The WeDecide study is an observational study that compares the long-term effects of matched related donor hematopoietic stem cell transplantation (MRD HCT) and non-transplant disease-modifying therapies (NT-DMT) in pediatric patients with sickle cell disease (SCD). It aims to assess health-related quality of life, cognitive function, and the risks and benefits of both treatments over a three-year period. The study will involve 160 children receiving MRD HCT and 320 children receiving NT-DMT, with evaluations conducted at multiple time points to track health outcomes and treatment impacts. Additionally, it will consider factors such as disease severity and social determinants of health to provide a comprehensive understanding of treatment effects.

Who should consider this trial

Good fit: Ideal candidates for this study are pediatric patients aged 3 to 20.9 years diagnosed with sickle cell anemia who are either candidates for MRD HCT or currently receiving NT-DMT.

Not a fit: Patients who are younger than 3 years or older than 20.9 years, or those with significant comorbidities that interfere with outcomes, may not benefit from this study.

Why it matters

Potential benefit: If successful, this study could help identify the most effective treatment options for children with sickle cell disease, improving their quality of life and health outcomes.

How similar studies have performed: This study is significant as it is one of the first to comprehensively compare these two treatment approaches in pediatric patients with sickle cell disease, building on previous research but focusing on a novel comparative analysis.

Eligibility criteria

Show full inclusion / exclusion criteria 
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 this trial is running

Birmingham, Alabama and 36 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions Sickle Cell DiseaseSCDMRD HCTNT DMT
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.