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