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Sponsors and Collaborators: |
UCSF Helen Diller Family Comprehensive Cancer Center National Cancer Institute (NCI) |
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Information provided by: | National Cancer Institute (NCI) |
ClinicalTrials.gov Identifier: | NCT00305708 |
RATIONALE: Drugs used in chemotherapy, such as busulfan and fludarabine, work in different ways to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. Giving more than one drug (combination chemotherapy) may kill more cancer cells. A donor peripheral blood, bone marrow , or umbilical cord blood transplant may be able to replace blood-forming cells that were destroyed by chemotherapy. Sometimes the transplanted cells from a donor can make an immune response against the body's normal cells. Giving antithymocyte globulin before the transplant may stop this from happening.
PURPOSE: This phase I/II trial is studying the side effects of busulfan, antithymocyte globulin, and fludarabine when given together with a donor stem cell transplant in treating young patients with blood disorders, bone marrow disorders, chronic myelogenous leukemia in first chronic phase, or acute myeloid leukemia in first remission.
Condition | Intervention | Phase |
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Cancer-Related Problem/Condition Congenital Amegakaryocytic Thrombocytopenia Diamond-Blackfan Anemia Fanconi Anemia Leukemia Severe Congenital Neutropenia |
Biological: anti-thymocyte globulin Drug: busulfan Drug: fludarabine phosphate Procedure: allogeneic bone marrow transplantation Procedure: peripheral blood stem cell transplantation Procedure: umbilical cord blood transplantation Radiation: radiation therapy |
Phase I Phase II |
Study Type: | Interventional |
Study Design: | Treatment, Open Label |
Official Title: | Bone Marrow Stem Cell Transplantation for Children With Stem Cell Defects, Marrow Failure Syndromes, or Myeloid Leukemia in 1Remission |
Estimated Enrollment: | 40 |
Study Start Date: | August 2000 |
OBJECTIVES:
Primary
Secondary
OUTLINE: Patients receive one of the following cytoreductive regimens:
Regimen 2 (patients with an HLA closely matched related [not genotypic] or unrelated donor): Patients receive busulfan and fludarabine as in regimen
1, and ATG IV over 10 hours on days -4 to -1.
Regimen 3 (patients with Fanconi's anemia or severe aplastic anemia with genotypic matched sibling donor): Patients receive fludarabine as in regimen
1 and ATG as in regimen 2.
All patients undergo allogeneic bone marrow, umbilical cord blood, or peripheral blood stem cell transplantation on day 0.
After the completion of study treatment, patients are followed periodically for 20 years.
PROJECTED ACCRUAL: A total of 40 patients will be accrued for this study.
Ages Eligible for Study: | up to 17 Years |
Genders Eligible for Study: | Both |
Accepts Healthy Volunteers: | No |
DISEASE CHARACTERISTICS:
Diagnosis of one of the following hematopoietic disorders:
Severe aplastic anemia with marrow aplasia (i.e., absolute neutrophil count < 500/mm^3, platelet and/or red blood cell transfusion dependent), meeting 1 of the following criteria:
Congenital marrow failure syndrome, including any of the following:
Hemoglobinopathy including any of the following:
Severe immunodeficiency disease including any of the following:
Chronic myelogenous leukemia in first chronic phase
Acute myeloid leukemia in first remission
Available donor, meeting 1 of the following criteria:
Unrelated donor, meeting one of the following criteria:
PATIENT CHARACTERISTICS:
PRIOR CONCURRENT THERAPY:
United States, California | |
UCSF Comprehensive Cancer Center | |
San Francisco, California, United States, 94115 |
Study Chair: | Morton J. Cowan, MD | UCSF Helen Diller Family Comprehensive Cancer Center |
Study ID Numbers: | CDR0000462443, UCSF-01152, UCSF-H411-17802-06 |
Study First Received: | March 21, 2006 |
Last Updated: | February 6, 2009 |
ClinicalTrials.gov Identifier: | NCT00305708 History of Changes |
Health Authority: | United States: Federal Government |
thrombocytopenia childhood acute myeloid leukemia in remission childhood chronic myelogenous leukemia Diamond-Blackfan anemia |
congenital amegakaryocytic thrombocytopenia Fanconi anemia severe congenital neutropenia chronic phase chronic myelogenous leukemia |
Antimetabolites Diamond-Blackfan Anemia Immunologic Factors Aplastic Anemia Leukocyte Disorders Leukemia, Myeloid, Chronic-Phase Leukemia, Myeloid, Acute Granulocytopenia Fanconi's Anemia Red-Cell Aplasia, Pure Leukemia Severe Congenital Neutropenia Aase Syndrome Acute Myelocytic Leukemia Thrombocytopenia |
Anemia, Aplastic Anemia, Diamond-Blackfan Alkylating Agents Metabolic Disorder Metabolic Diseases Hematologic Diseases Fanconi Anemia Blood Platelet Disorders Anemia Myeloproliferative Disorders Agranulocytosis Congenital Amegakaryocytic Thrombocytopenia Fludarabine monophosphate Leukemia, Myeloid Immunosuppressive Agents |
Antimetabolites Antimetabolites, Antineoplastic Molecular Mechanisms of Pharmacological Action Immunologic Factors Antineoplastic Agents Physiological Effects of Drugs DNA Repair-Deficiency Disorders Leukocyte Disorders Leukemia, Myeloid, Acute Red-Cell Aplasia, Pure Leukemia Thrombocytopenia Therapeutic Uses Anemia, Aplastic Anemia, Diamond-Blackfan |
Alkylating Agents Metabolic Diseases Neoplasms by Histologic Type Hematologic Diseases Fanconi Anemia Blood Platelet Disorders Anemia Myeloproliferative Disorders Agranulocytosis Fludarabine monophosphate Leukemia, Myeloid Immunosuppressive Agents Pharmacologic Actions Antilymphocyte Serum Neutropenia |