Status:
COMPLETED
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
Lead Sponsor:
University of California, San Francisco
Collaborating Sponsors:
National Cancer Institute (NCI)
Conditions:
Congenital Amegakaryocytic Thrombocytopenia
Diamond-blackfan Anemia
Eligibility:
All Genders
Up to 17 years
Phase:
PHASE1
PHASE2
Brief Summary
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. Givin...
Detailed Description
OBJECTIVES: Primary * 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 c...
Eligibility Criteria
Inclusion
- 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:
- Closely matched related donor
- Unresponsive to immunosuppressive therapy within 3 months after follow-up AND alternative matched unrelated donor available
- Congenital marrow failure syndrome, including any of the following:
- Primary red blood cell aplasia (Diamond-Blackfan syndrome)
- Congenital neutropenia (Kostmann's syndrome)
- Amegakaryocytic thrombocytopenia
- Hemoglobinopathy including any of the following:
- β-thalassemia major
- Sickle cell anemia
- Severe immunodeficiency disease including any of the following:
- Chediak-Higashi disease
- Wiskott-Aldrich syndrome
- Combined immunodeficiency disease (Nezelof's)
- Hyperimmunoglobulin M syndrome
- Bare lymphocyte syndrome
- Other stem cell defects (e.g., osteopetrosis)
- Chronic myelogenous leukemia in first chronic phase
- Not eligible for other ongoing phase II/III studies
- Acute myeloid leukemia in first remission
- Not eligible for other ongoing phase II/III studies
- Inborn errors of metabolism
- No severe combined immunodeficiency disorder
- Available donor, meeting 1 of the following criteria:
- Related donor matched by high resolution DNA typing at both HLA Drβ1 alleles and ≤ 1 mismatch at the 4 HLA-A and -B alleles
- Unrelated donor, meeting one of the following criteria:
- Bone marrow matched by high resolution DNA typing at both HLA Drβ1 alleles and ≤ 1 mismatch by high resolution DNA typing at the 4 HLA-A and -B alleles
- Umbilical cord blood matched at 4/6 HLA-A, -B, and Drβ1 alleles by high resolution typing with ≥ 1 Drβ1 match and ≥ 3 X 10\^7 cells/kg body weight of recipient
- PATIENT CHARACTERISTICS:
- See Disease Characteristics
- No active bacterial, viral, or fungal infection
- Cardiac shortening fraction ≥ 27%
- Creatinine clearance ≥ 60 mL/min
- DLCO ≥ 60% of predicted (corrected for anemia/lung volume)
- PRIOR CONCURRENT THERAPY:
- See Disease Characteristics
Exclusion
Key Trial Info
Start Date :
August 1 2000
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
July 1 2004
Estimated Enrollment :
40 Patients enrolled
Trial Details
Trial ID
NCT00305708
Start Date
August 1 2000
End Date
July 1 2004
Last Update
November 12 2012
Active Locations (1)
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1
UCSF Comprehensive Cancer Center
San Francisco, California, United States, 94115