Clinical Trial

HSCT for Patients With Fanconi Anemia Using Risk-Adjusted Chemotherapy

Study acronym: RAFA
Recruiting Phase 2
View on ClinicalTrials.gov →
Summary
The purpose of this study is to determine whether the use of lower doses of busulfan and the elimination of cyclosporine will further reduce transplant-related side effects for patients with Fanconi Anemia (FA). Patients will undergo a transplant utilizing mis-matched related or matched unrelated donors following a preparative regimen of busulfan, fludarabine, anti-thymocyte globulin and cyclophosphamide.
Protocol Amendment History 14 amendments
This ClinicalTrials.gov record has been amended 14 times since 2014-05-16; most recent amendment 2025-11-10.
Trial Details
NCT Number NCT02143830
Lead Sponsor Children's Hospital Medical Center, Cincinnati
Collaborators: Memorial Sloan Kettering Cancer Center, Fred Hutchinson Cancer Center
Conditions Fanconi Anemia, Severe Marrow Failure, Myelodysplastic Syndrome (MDS), Acute Myelogenous Leukemia (AML)
Enrollment 70 participants
Start Date 2014-04
Primary Completion 2026-12 (estimated)
Study Completion 2028-12 (estimated)
Updated on ClinicalTrials.gov 2025-11-12