Busulfan, Antithymocyte Globulin, and Fludarabine Followed By 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
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT00305708|
Recruitment Status : Completed
First Posted : March 22, 2006
Last Update Posted : November 12, 2012
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 or disease||Intervention/treatment||Phase|
|Congenital Amegakaryocytic Thrombocytopenia Diamond-blackfan Anemia Fanconi Anemia Leukemia Severe Congenital Neutropenia Thrombocytopenia||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 1 Phase 2|
- Determine the efficacy, in terms of graft rejection at 4 weeks, of a conditioning regimen comprising busulfan, anti-thymocyte globulin, and fludarabine followed by donor stem cell transplantation (SCT) in children with stem cell defects, marrow failure syndromes, chronic myelogenous leukemia in first chronic phase, or acute myeloid leukemia in first remission.
- Determine the pharmacokinetics of busulfan in children undergoing donor SCT.
- Determine the toxicity of this regimen in these patients.
- Determine engraftment at 3, 6, 9, and 12 months and mixed chimerism in patients treated with this regimen.
- Determine overall and disease-free survival of patients treated with this regimen.
OUTLINE: Patients receive one of the following cytoreductive regimens:
- Regimen 1 (patients with an HLA genotypic matched sibling donor): Patients receive busulfan IV over 2 hours every 6 hours on days -9 to -6, fludarabine IV on days -5 to -2, and anti-thymocyte globulin (ATG) IV over 10 hours on days -3 to -1.
- 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.
- Regimen 4 (patients with Fanconi's anemia who have a closely matched related [not genotypic] or unrelated donor): Patients undergo thoracoabdominal irradiation on day -6 and 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.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||40 participants|
|Masking:||None (Open Label)|
|Official Title:||Bone Marrow Stem Cell Transplantation for Children With Stem Cell Defects, Marrow Failure Syndromes, or Myeloid Leukemia in 1Remission|
|Study Start Date :||August 2000|
|Actual Primary Completion Date :||July 2004|
|Actual Study Completion Date :||July 2004|
- Graft rejection measured by ANC < 500 with no evidence of donor cells in blood or marrow from transplantation to week 4 post transplantation
- Toxicity grades 3 or 4 assessed from conditioning through 1 year post transplantation
- Engraftment at 1, 3, 6, 9, and 12 months post transplantation
- Mixed chimerism at 1, 3, 6, 9, and 12 months post transplantation
- Survival measured from the day of first dose of conditioning
- Disease-free survival measured from the day of first dose of conditioning
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00305708
|United States, California|
|UCSF Comprehensive Cancer Center|
|San Francisco, California, United States, 94115|
|Study Chair:||Morton J. Cowan, MD||University of California, San Francisco|