Status:
COMPLETED
Donor Stem Cell Transplant in Treating Young Patients With Myelodysplastic Syndrome, Leukemia, Bone Marrow Failure Syndrome, or Severe Immunodeficiency Disease
Lead Sponsor:
University of California, San Francisco
Collaborating Sponsors:
National Cancer Institute (NCI)
Conditions:
Congenital Amegakaryocytic Thrombocytopenia
Leukemia
Eligibility:
All Genders
1-17 years
Phase:
PHASE1
Brief Summary
RATIONALE: Giving chemotherapy and total body irradiation before a donor bone marrow transplant or peripheral blood stem cell transplant helps stop the growth of cancer cells. It also helps stop the p...
Detailed Description
OBJECTIVES: * Determine the efficacy and toxicity of stem cell-enriched, T-cell-depleted, haplocompatible allogeneic hematopoietic stem cell transplantation in children with high-risk myelodysplastic...
Eligibility Criteria
Inclusion
- DISEASE CHARACTERISTICS:
- Diagnosis of one of the following:
- Acute lymphoblastic leukemia in ≥ 2nd remission or delayed remission induction
- High-risk myelodysplastic syndromes
- Refractory anemia with excess blasts (RAEB)
- RAEB in transformation
- Chronic myelogenous leukemia in second chronic phase
- No accelerated phase (\> 5% blasts in marrow)
- Juvenile myelomonocytic leukemia
- Acute nonlymphoblastic leukemia in \> 1st remission or induction failure and \< 30% blasts in marrow
- Severe aplastic anemia, defined as absolute neutrophil count \< 500/mm\^3 and platelet and/or red blood cell transfusion dependent
- Unresponsive to immunosuppressive therapy
- No Fanconi's anemia
- Congenital marrow aplasias unresponsive to cytokines and transfusion dependent
- Inherited immunodeficiency disease involving neutrophils or lymphocytes, including any of the following:
- Chediak-Higashi disease
- Wiskott-Aldrich syndrome
- Combined immunodeficiency disease (Nezelof's)
- Hyper IgM syndrome
- No relapsed disease
- Haplocompatible related donor, including parent, cousin, aunt, uncle, grandparent, half-sibling, or sibling (≥ 12 years of age), available
- 2 or 3 HLA antigen mismatch
- At least a 3 HLA antigen genotypic match
- No closely matched related or unrelated donor available in sufficient time to do the transplant
- PATIENT CHARACTERISTICS:
- No active hepatitis or cytomegalovirus infection
- Cardiac ejection fraction ≥ 30%
- Creatinine clearance ≥ 70 mL/min
- DLCO ≥ 70% of predicted
- No active infection
- No HIV positivity
- PRIOR CONCURRENT THERAPY:
- See Disease Characteristics
Exclusion
Key Trial Info
Start Date :
April 1 2005
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
December 1 2011
Estimated Enrollment :
21 Patients enrolled
Trial Details
Trial ID
NCT00295971
Start Date
April 1 2005
End Date
December 1 2011
Last Update
November 12 2012
Active Locations (2)
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1
UCSF Helen Diller Family Comprehensive Cancer Center
San Francisco, California, United States, 94115
2
Lineberger Comprehensive Cancer Center at University of North Carolina - Chapel Hill
Chapel Hill, North Carolina, United States, 27599-7295