Status:

RECRUITING

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

Lead Sponsor:

University of Rochester

Collaborating Sponsors:

Patient-Centered Outcomes Research Institute

Conditions:

Sickle Cell Disease (SCD)

Eligibility:

All Genders

3-20 years

Brief Summary

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 therap...

Detailed Description

The WeDecide study is a large observational study comparing the long-term effects of two treatment options for pediatric patients with sickle cell disease (SCD): matched related donor hematopoietic st...

Eligibility Criteria

Inclusion

  • 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

  • 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.

Key Trial Info

Start Date :

June 1 2024

Trial Type :

OBSERVATIONAL

Allocation :

ESTIMATED

End Date :

October 1 2030

Estimated Enrollment :

480 Patients enrolled

Trial Details

Trial ID

NCT06941389

Start Date

June 1 2024

End Date

October 1 2030

Last Update

June 19 2025

Active Locations (37)

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Page 1 of 10 (37 locations)

1

Children's of Alabama (MRD-HCT)

Birmingham, Alabama, United States, 35294

2

Children's of Alabama (NT-DMT)

Birmingham, Alabama, United States, 35294

3

Nemours Children's Hospital, Delaware (MRD-HCT)

Wilmington, Delaware, United States, 19803

4

Children's National Hospital (MRD-HCT)

Washington D.C., District of Columbia, United States, 20010

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