A Phase 1/2 interventional study of anti-thymocyte globulin and busulfan in Congenital Amegakaryocytic Thrombocytopenia, Diamond-blackfan Anemia and Fanconi Anemia, sponsored by University of California, San Francisco. Completed at 1 site in United States. Open to participants aged Up to 17 Years. Per ClinicalTrials.gov, last updated 2012-11-12.
Sponsored by University of California, San Francisco · Phase 1/2, Interventional, and Treatment
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.
OBJECTIVES:
Primary
Secondary
OUTLINE: Patients receive one of the following cytoreductive regimens:
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.
5,441 studies on the registry are indexed under Leukemia; 636 are open to participants now.
This study's planned enrollment of 40 is close to the median of 38 across 4,247 interventional studies indexed under Leukemia.
Browse Leukemia studies →University of California, San Francisco is the lead sponsor of 2,132 studies on the registry; 375 are open to participants now.
Of its 262 completed or terminated interventional studies of FDA-regulated products, 196 (75%) have results posted.
Counted across the registry records on this site, refreshed daily.
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:
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
This study is completed, as verified in Nov 2012. You cannot join it, but the record below documents what was studied.
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University of California, San Francisco