Clinical Trial: Hematopoietic Stem Cell Transplantation in High Risk Patients With Fanconi Anemia
Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional
Official Title: Hematopoietic Stem Cell Transplantation in High Risk Patients With Fanconi Anemia MT2002-02
Brief Summary:
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 patients with high risk Fanconi's anemia.
PURPOSE: This clinical trial is studying how well combination chemotherapy works in treating high risk patients who are undergoing a donor stem cell transplant for Fanconi's anemia.
Detailed 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 intravenously (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. Sponsor: Masonic Cancer Center, University of Minnesota
- Incidence of Acute and Chronic Graft-Versus-Host Disease [ Time Frame: Day 42 and 1 Year ]
- Incidence of Relapse [ Time Frame: 1 Year ]
- Incidence of Major Infections [ Time Frame: Day 1 through End of Treatment ]
- Transplant-Related Toxicity [ Time Frame: Day 100 ]
- Overall Survival [ Time Frame: 1 Year ]cumulative proportion surviving
- Incidence of Chronic Graft-Versus-Host Disease [ Time Frame: Day 42 and 1 Year ]
Current Primary Outcome: Percent of Graft Failure [ Time Frame: Day 30 ]
Original Primary Outcome:
Current Secondary Outcome:
Original Secondary Outcome:
Information By: Masonic Cancer Center, University of Minnesota
Dates:
Date Received: November 22, 2005
Date Started: March 26, 2002
Date Completion: January 2019
Last Updated: February 17, 2017
Last Verified: February 2017