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Brief Title: A Study of the Effect of Blood Stem Cell Transplant After Chemotherapy Alone in Patients With Fanconi Anemia
Official Title: A Phase II Trial of Hematopoietic Stem Cell Transplantation 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: NCT03600909
Brief Summary: The goal of this study is to see if the study therapy can decrease the chemotherapy-related side effects while maximizing the effectiveness of disease control. The physicians will also be studying the effect of removing T-cells from the donor"s stem cells before transplant. T-cells are a type of white blood cell that may help cause a serious side effect of transplant called Graft versus Host Disease (GVHD). The way it removes the T-cells from the donor stem cells is actually by selecting only the stem cells (called CD34 cells) by using a device called CliniMACS. This process is called CD34 selection. The CliniMACS® device is currently under the supervision of the FDA .
Detailed Description:
Minimum Age: 1 Month
Eligible Ages: CHILD, ADULT, OLDER_ADULT
Sex: ALL
Healthy Volunteers: No
Memorial Sloan Kettering Cancer Center, New York, New York, United States
Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio, United States
Fred Hutchinson Cancer Research Center, Seattle, Washington, United States
Name: Farid Boulad, MD
Affiliation: Memorial Sloan Kettering Cancer Center
Role: PRINCIPAL_INVESTIGATOR