Spots Global Cancer Trial Database for HSCT for Patients With Fanconi Anemia Using Risk-Adjusted Chemotherapy

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Trial Identification

Brief Title: HSCT for Patients With Fanconi Anemia Using Risk-Adjusted Chemotherapy

Official Title: A Phase II Trial of HSCT for the Treatment of Patients With Fanconi Anemia Lacking a Genotypically Identical Donor, Using a Risk-Adjusted Chemotherapy Only Cytoreduction With Busulfan, Cyclophosphamide and Fludarabine

Study ID: NCT02143830

Conditions

Fanconi Anemia

Severe Marrow Failure

Myelodysplastic Syndrome (MDS)

Acute Myelogenous Leukemia (AML)

Interventions

Peripheral blood stem cell

Study Description

Brief 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.

Detailed Description: 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.