MedTrace Logo

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

NCT06941389 · Status: RECRUITING · Type: OBSERVATIONAL · Enrollment: 480

Last updated 2025-06-19

No results posted yet for this study

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 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.

Conditions

  • Sickle Cell Disease (SCD)

Sponsors & Collaborators

  • Patient-Centered Outcomes Research Institute

    collaborator OTHER

  • University of Rochester

    lead OTHER

Eligibility

Min Age
3 Years
Max Age
20 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2024-06-01
Primary Completion
2030-10-01
Completion
2030-10-01

Countries

  • United States
  • Canada

Study Locations

More Related Trials

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT06941389 on ClinicalTrials.gov