RAFA
HSCT for Patients With Fanconi Anemia Using Risk-Adjusted Chemotherapy (RAFA)
What will happen during the trial?
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.
The trial proposed is a three arm phase II treatment protocol designed to investigate the safety and efficacy of risk-adjusted chemotherapy-based cytoreductive regimen plus a CD34+ selected T-cell depleted peripheral blood stem cell (PBSC) stem cell transplant for the treatment of patients with Fanconi anemia and severe hematologic disease. Candidates for this trial will include patients with Fanconi anemia presenting with severe marrow failure (transfusion dependent) or myelodysplastic syndrome, or acute myelogenous leukemia for whom an allogeneic stem cell transplant is indicated.
More Information
- Trial Status
- Accepting patients
- Trial Phase
- Phase 2
- Enrollment
- 70 patients (estimated)
- Sponsors
- Children's Hospital Medical Center, Cincinnati
- Collaborators
- Fred Hutchinson / University of Washington Cancer Consortium, Memorial Sloan Kettering Cancer Center
- Tags
- Allogeneic Stem Cell Transplant, Chemotherapy
- Trial Type
- Treatment
- Last Update
- SparkCures ID
- 1617
- NCT Identifier
- NCT02143830
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