NCT06941389 Comparing the Effectiveness of Matched Related Donor Hematopoietic Stem Cell Transplantation to Disease Modifying Therapy in Pediatric Patients With Sickle Cell Disease
| NCT ID | NCT06941389 |
| Status | Recruiting |
| Phase | — |
| Sponsor | University of Rochester |
| Condition | Sickle Cell Disease (SCD) |
| Study Type | OBSERVATIONAL |
| Enrollment | 480 participants |
| Start Date | 2024-06-01 |
| Primary Completion | 2030-10-01 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
This is an observational study. You will not receive an experimental treatment; researchers will collect data based on your existing condition or standard treatment.
This trial targets 480 participants in total. It began in 2024-06-01 with a primary completion date of 2030-10-01.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
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 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.
Eligibility 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.
Contact & Investigator
Frequently Asked Questions
Who can join the NCT06941389 clinical trial?
This trial is open to participants of all sexes, aged 3 Years or older, up to 20 Years, studying Sickle Cell Disease (SCD). Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
Is NCT06941389 currently recruiting?
Yes, NCT06941389 is actively recruiting participants. Contact the research team at John_Horan@URMC.Rochester.edu for enrollment information.
Where is the NCT06941389 trial being conducted?
This trial is being conducted at Birmingham, United States, Birmingham, United States, Wilmington, United States, Washington D.C., United States and 11 additional locations.
Who is sponsoring the NCT06941389 clinical trial?
NCT06941389 is sponsored by University of Rochester. The trial plans to enroll 480 participants.
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