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Combination Chemotherapy in Treating Patients Who Are Undergoing a Donor Stem Cell Transplant for Fanconi's Anemia - NCT00258427-55455(Clinical Trial 133313)



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City:  Minneapolis
State:  
MN
Zip Code: 55455
Conditions: Fanconi Anemia
Purpose: RATIONALE: A bone marrow or umbilical cord blood transplant may be able to replace blood-forming cells that were destroyed by chemotherapy. Giving combination chemotherapy before a donor stem cell transplant may make the transplant more likely to work. This may be an effective treatment for Fanconi's anemia. PURPOSE: This clinical trial is studying how well combination chemotherapy works in treating patients who are undergoing a donor stem cell transplant for Fanconi's anemia.
Study summary: OBJECTIVES: Primary - Determine whether the incidence of neutrophil engraftment is acceptable in high-risk patients with Fanconi's anemia treated with busulfan, cyclophosphamide, fludarabine, and antithymocyte globulin followed by allogeneic hematopoietic stem cell transplantation. Secondary - Determine the tolerability of mycophenolate mofetil in these patients. - Determine the incidence of acute and chronic graft-vs-host disease in patients treated with this regimen. - Determine the incidence of major infections in patients with a history of major infections treated with this regimen. - Determine the incidence of relapse in patients with refractory anemia with excess blasts, refractory anemia with excess blasts in transformation, or acute myeloid leukemia treated with this regimen - Determine the probability of 1-year survival of patients treated with this regimen. OUTLINE: Patients are stratified according to donor/recipient HLA type (identical vs other). - Cytoreductive combination chemotherapy: Patients receive busulfan IV over 2 hours twice daily on days -7 and -6 and cyclophosphamide IV over 2 hours and fludarabine IV over 30 minutes once daily on days -5 to -2. - Graft failure prophylaxis: Patients receive methylprednisolone IV twice daily on days -5 to 30 and anti-thymocyte globulin IV over 4-6 hours twice daily on days -5 to -1. - Graft-vs-host disease prophylaxis: Patients receive cyclosporine IV over 2 hours twice daily on days -3 to 100 (if patient has a matched sibling donor) or days -3 to 180 (if patient has another donor type). Patients also receive mycophenolate mofetil orally or IV twice daily on days -3 to 45. - Allogeneic hematopoietic stem cell transplantation (HSCT): Patients undergo allogeneic HSCT (using bone marrow or umbilical cord blood) on day 0. Patients receive filgrastim (G-CSF) subcutaneously beginning on day 1 and continuing until blood counts recover. After completion of study treatment, patients are followed periodically for 3 years. PROJECTED ACCRUAL: A total of 25 patients will be accrued for this study.
Criteria: Inclusion Criteria: - Patients must be <45 years of age with a diagnosis of high-risk Fanconi anemia with high risk disease being defined as: 1) presence of advanced myelodysplastic syndrome (MDS) (i.e., RAEB or RAEBt or acute leukemia), 2) history of malignancy, currently in remission; 3) history at any time of systemic fungal or gramnegative infection, or 4) severe renal disease with a creatinine clearance <40 mL/min. - Patients must have an HLA-A, B, DRB1 identical or 1 antigen mismatched related or unrelated BM donor or have an HLA-A, B, DRB1 identical, 1 antigen or 2 antigen mismatched related or unrelated umbilical cord blood (UCB) donor. Patients and donors will be typed for HLA-A and B using serological level typing and for DRB1 using high resolution molecular typing. - Adequate major organ function including: - Cardiac: ejection fraction >45% - Hepatic: no clinical evidence of hepatic failure (e.g. coagulopathy, ascites, no cirrhosis) - Karnofsky performance status >70% or Lansky >50% - Women of child bearing potential must be using adequate birth control and have a negative pregnancy test. Exclusion Criteria: - Patients any age with aplastic anemia or early MDS, creatinine clearance >40 mL/min, and an HLA genotypic identical donor. - Aplastic anemia is defined as having at least one of the following (with or without cytogenetic abnormalities): - platelet count <20 x 10^9/L - absolute neutrophil count (ANC) <5 x 10^8/L - Hgb <8 g/dL - Active central nervous system (CNS) leukemia at time of hematopoietic stem cell transplant (HSCT). - Active uncontrolled infection within one week of HSCT. - Pregnant or lactating female. Donor Inclusion Criteria: - Donor must be in good health based on review of systems and results of physical examination. - Donor must have a normal hemoglobin, white count, platelet count and PTT, and a negative DEB test. - HIV-NAT negative, HTLV-1, HTLV-2 negative, Hepatitis B and C negative. - Female donors of childbearing potential must have a negative pregnancy test. - Unrelated donors must agree to PBSC donation Donor Exclusion Criteria: - Donor is a lactating female.
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Data Source: ClinicalTrials.gov
Date Processed: July 12, 2010
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