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Main
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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
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Register:
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ClinicalTrials.gov |
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Last refreshed on:
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19 February 2015 |
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Main ID: |
NCT01759732 |
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Date of registration:
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30/12/2012 |
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Prospective Registration:
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No |
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Primary sponsor: |
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Public title:
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Haploidentical Stem Cell Transplantation for Children With Acquired Severe Aplastic Anemia
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Scientific title:
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Haploidentical Stem Cell Transplantation With Fixed Dose of T Cells After in Vitro T Cell Depletion Using CD3 Monoclonal Antibody for Children With Acquired Severe Aplastic Anemia |
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Date of first enrolment:
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September 2012 |
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Target sample size:
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10 |
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Recruitment status: |
Recruiting |
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URL:
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http://clinicaltrials.gov/show/NCT01759732 |
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Study type:
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Interventional |
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Study design:
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Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
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Phase:
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Phase 2
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Countries of recruitment
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Korea, Republic of
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Contacts
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Name:
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Ho Joon Im, MD, PhD |
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Address:
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Telephone:
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82-2-3010-3371 |
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Email:
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hojim@amc.seoul.kr |
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Affiliation:
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Name:
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Ho Joon Im, MD, PhD |
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Address:
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Telephone:
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82-2-3010-3371 |
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Email:
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hojim@amc.seoul.kr |
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Affiliation:
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Name:
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Ho Joon Im, MD, PhD |
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Address:
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Telephone:
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Email:
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Affiliation:
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Asan Medical Center |
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Key inclusion & exclusion criteria
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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
Age minimum:
N/A
Age maximum:
21 Years
Gender:
Both
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Health Condition(s) or Problem(s) studied
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Acquired Aplastic Anemia
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Intervention(s)
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Drug: Cyclophosphamide
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Radiation: Total body irradiation
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Procedure: CD3-depleted hematopoietic cell transplantation
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Biological: anti-thymocyte globulin
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Biological: filgrastim
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Drug: Fludarabine
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Primary Outcome(s)
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To assess engraftment rate and survival of haploidentical stem cell transplantation with fixed dose of T cells after in vitro T cell depletion using CD3 monoclonal antibody for children with acquired severe aplastic anemia
[Time Frame: 2 years posttransplant]
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Secondary Outcome(s)
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To estimate the risk of acute GVHD
[Time Frame: 100 days posttransplant]
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To assess engraftment and graft failure
[Time Frame: 28 days posttransplant]
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To estimate overall survival and failure free survival
[Time Frame: 1 year posttransplant]
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To assess treatment related mortality
[Time Frame: 100 days posttransplant]
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Secondary ID(s)
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AMCPHO-SCT1202
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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Results
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Results available:
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Date Posted:
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Date Completed:
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URL:
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