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

    Current Primary Outcome: Percent of Graft Failure [ Time Frame: Day 30 ]

    Graft failure = ANC <5 x 10^8/L by day 30.


    Original Primary Outcome:

    Current Secondary Outcome:

    • 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 ]


    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