Clinical Trial: Pilot Study of Non-Myeloablative, HLA-Matched Allogeneic Stem Cell Transplantation for Pediatric Hematopoietic Malignancies

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

Official Title: Pilot Study of Non-Myeloablative, HLA-Matched Allogeneic Stem Cell Transplantation for Pediatric Hematopoietic Malignancies

Brief Summary:

Background:

  • Allogeneic blood and marrow stem cell transplantation (BMT) plays an important role in the curative treatment of a number of pediatric malignancies. Unfortunately, the success of conventional allogeneic BMT is limited in part by the multiple toxicities associated with myeloablative preparative regimens.
  • Non-myeloablative pre-transplant regimens are associated with less toxic side effects than standard BMT. Recently, a novel immunosuppressive, non-myeloablative pre-transplant chemotherapy regimen has been shown to facilitate complete donor engraftment in an adult trial at the NCI.

Objectives:

The primary objective of this protocol is to evaluate the efficacy and safety of this treatment approach in pediatric patients with hematopoietic malignancies

Eligibility:

Inclusion Criteria

Age: Patient must be greater than or equal to 5 years and less than 22 years of age.

Diagnosis:

  • Hodgkin s and Non-Hodgkin s Lymphoma: Refractory disease or relapse after salvage regimen.
  • Acute Myelogenous Leukemia: History of bone marrow relapse in remission (CR) #2 or greater.
  • Acute Lymphocytic Leukemia: History of bone marrow relapse in CR #2 or greater (CR#1 with Philadelphia chromosome positive or prior induction failure).
  • Acute Hybrid Leukemia including mixed lineage, biphenotypic and undifferentiated: History of bone marrow relap

    Detailed Summary:

    Background:

    • Allogeneic blood and marrow stem cell transplantation (BMT) plays an important role in the curative treatment of a number of pediatric malignancies. Unfortunately, the success of conventional allogeneic BMT is limited in part by the multiple toxicities associated with myeloablative preparative regimens.
    • Non-myeloablative pre-transplant regimens are associated with less toxic side effects than standard BMT. Recently, a novel immunosuppressive, non-myeloablative pre-transplant chemotherapy regimen has been shown to facilitate complete donor engraftment in an adult trial at the NCI.

    Objectives:

    The primary objective of this protocol is to evaluate the efficacy and safety of this treatment approach in pediatric patients with hematopoietic malignancies

    Eligibility:

    Inclusion Criteria

    Age: Patient must be greater than or equal to 5 years and less than 22 years of age.

    Diagnosis:

    • Hodgkin s and Non-Hodgkin s Lymphoma: Refractory disease or relapse after salvage regimen.
    • Acute Myelogenous Leukemia: History of bone marrow relapse in remission (CR) #2 or greater.
    • Acute Lymphocytic Leukemia: History of bone marrow relapse in CR #2 or greater (CR#1 with Philadelphia chromosome positive or prior induction failure).
    • Acute Hybrid Leukemia including mixed lineage, biphenotypic and undifferentiated: History of bone marrow relap
      Sponsor: National Cancer Institute (NCI)

      Current Primary Outcome:

      • To determine the efficacy and safety of this chemotherapy regimen in facilitating donor engraftment after allogeneic bone marrow transplantation (BMT).
      • Safety/Efficacy [ Time Frame: 5 years ]


      Original Primary Outcome:

      Current Secondary Outcome:

      • Toxicity of regimen [ Time Frame: 5 years ]
      • To determine the toxicity of this non-myelablative allogeneic BMT regimen.
      • fludarabine-based induction reducing T-cells [ Time Frame: 5 years ]
      • immune suppression [ Time Frame: 5 years ]
      • IL-7 levels [ Time Frame: 5 years ]
      • cytokine profiles [ Time Frame: 5 years ]
      • response rates and DFS [ Time Frame: 5 years ]
      • incidence and severity of GVHD [ Time Frame: 5 years ]
      • response rates, DFS rates, and incidence and severity ofGVHD following withdrawal of immunosuppression and donorlymphocyte infusions (DLI) for patients who developprogressive disease after day +28 post-transplant [ Time Frame: 5 years ]


      Original Secondary Outcome:

      Information By: National Institutes of Health Clinical Center (CC)

      Dates:
      Date Received: March 20, 2001
      Date Started: March 14, 2001
      Date Completion:
      Last Updated: May 12, 2017
      Last Verified: May 7, 2015