Brief Title
Human Leukocyte Antigen (HLA)-Haploidentical Hematopoietic Stem Cell Transplantation for Patients With Aplastic Anemia
Official Title
HLA-haploidentical Allogeneic Hematopoietic Cell Transplantation Using CD3±CD19 Depletion for Patients With Aplastic Anemia After Conditioning of Fludarabine, Cyclophosphamide and Antithymocyte Globulin
Brief Summary
Rationale: Chemotherapy with fludarabine, cyclophosphamide and anti-thymocyte globulin may
induce the engraftment cross the immunologic barrier in the setting of HLA-haploidentical
allogeneic hematopoietic cell transplantation. In addition, depletion CD3±CD19 cells may
contribute to prevent developing severe acute graft versus host disease (GVHD) in
haploidentical transplantation.
Purpose: This phase I/II trial is to evaluate the safety and efficacy of fludarabine,
cyclophosphamide and antithymocyte globulin with CD3±CD19 depleted graft from haploidentical
donors in treating patients with aplastic anemia.
Study Phase
Phase 1/Phase 2
Study Type
Interventional
Primary Outcome
To assess the engraftment rate and survival of CD3±CD19 depleted haploidentical peripheral blood stem cell transplantation after conditioning with fludarabine, cyclophosphamide and anti-thymocyte globulin.
Secondary Outcome
To assess engraftment and graft failure
Condition
Aplastic Anemia
Intervention
anti-thymocyte globulin
Study Arms / Comparison Groups
HAPLO
Description:
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Recruitment Status
Biological
Estimated Enrollment
12
Start Date
July 2009
Completion Date
July 2012
Primary Completion Date
July 2012
Eligibility Criteria
Inclusion Criteria:
- Diagnosis of life-threatening marrow failure (severe aplastic anemia) of nonmalignant
etiology meeting 2 of the following criteria:
- Granulocyte count < 500/mm3,
- Corrected reticulocyte count < 1%,
- Platelet count < 20,000/mm3
- No HLA-identical family member or closely matched (8 of 8 HLA-locus match) unrelated
marrow donor available
- HLA-haploidentical related donor available
Exclusion Criteria:
- Paroxysmal nocturnal hemoglobinuria or Fanconi anemia
- Clonal cytogenetic abnormalities or myelodysplastic syndromes
- Active fungal infections
- HIV positive
- Severe disease other than aplastic anemia that would severely limit the probability of
survival during the graft procedure
- Pregnant or nursing
Gender
All
Ages
N/A - 21 Years
Accepts Healthy Volunteers
No
Contacts
Ho Joon Im, MD & PhD, ,
Location Countries
Korea, Republic of
Location Countries
Korea, Republic of
Administrative Informations
NCT ID
NCT01105273
Organization ID
AMCPHO-SCT0802
Responsible Party
Principal Investigator
Study Sponsor
Asan Medical Center
Study Sponsor
Ho Joon Im, MD & PhD, Principal Investigator, Asan Medical Center
Verification Date
December 2012