Haploidentical Stem Cell Transplantation for Children With Acquired Severe Aplastic Anemia
1 other identifier
interventional
10
1 country
1
Brief Summary
Rationale: Fludarabine, cyclophosphamide, anti-thymocyte globulin and low-dose total body irradiation (LD-TBI) may induce the engraftment cross the immunologic barrier in the setting of HLA-haploidentical allogeneic hematopoietic cell transplantation. In addition, depletion of CD3 cells may contribute to prevent developing severe acute graft versus host disease (GVHD) in haploidentical transplantation. Purpose: Phase II trials to evaluate the efficacy 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
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for phase_2
Started Sep 2012
Typical duration for phase_2
1 active site
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
Study Start
First participant enrolled
September 1, 2012
CompletedFirst Submitted
Initial submission to the registry
December 30, 2012
CompletedFirst Posted
Study publicly available on registry
January 3, 2013
CompletedPrimary Completion
Last participant's last visit for primary outcome
December 1, 2014
CompletedStudy Completion
Last participant's last visit for all outcomes
December 1, 2015
CompletedJanuary 3, 2013
December 1, 2012
2.2 years
December 30, 2012
December 30, 2012
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
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
2 years posttransplant
Secondary Outcomes (4)
To assess engraftment and graft failure
28 days posttransplant
To estimate the risk of acute GVHD
100 days posttransplant
To assess treatment related mortality
100 days posttransplant
To estimate overall survival and failure free survival
1 year posttransplant
Study Arms (1)
HAPLO
EXPERIMENTALInterventions
Eligibility Criteria
You may qualify if:
- 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
You may not qualify if:
- 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (1)
Asan Medical Center
Seoul, 138-736, South Korea
Related Publications (5)
Burroughs LM, Woolfrey AE, Storer BE, Deeg HJ, Flowers ME, Martin PJ, Carpenter PA, Doney K, Appelbaum FR, Sanders JE, Storb R. Success of allogeneic marrow transplantation for children with severe aplastic anaemia. Br J Haematol. 2012 Jul;158(1):120-8. doi: 10.1111/j.1365-2141.2012.09130.x. Epub 2012 Apr 26.
PMID: 22533862BACKGROUNDTolar J, Deeg HJ, Arai S, Horwitz M, Antin JH, McCarty JM, Adams RH, Ewell M, Leifer ES, Gersten ID, Carter SL, Horowitz MM, Nakamura R, Pulsipher MA, Difronzo NL, Confer DL, Eapen M, Anderlini P. Fludarabine-based conditioning for marrow transplantation from unrelated donors in severe aplastic anemia: early results of a cyclophosphamide dose deescalation study show life-threatening adverse events at predefined cyclophosphamide dose levels. Biol Blood Marrow Transplant. 2012 Jul;18(7):1007-11. doi: 10.1016/j.bbmt.2012.04.014. Epub 2012 Apr 27.
PMID: 22546497BACKGROUNDXu LP, Liu KY, Liu DH, Han W, Chen H, Chen YH, Zhang XH, Wang Y, Wang FR, Wang JZ, Huang XJ. A novel protocol for haploidentical hematopoietic SCT without in vitro T-cell depletion in the treatment of severe acquired aplastic anemia. Bone Marrow Transplant. 2012 Dec;47(12):1507-12. doi: 10.1038/bmt.2012.79. Epub 2012 May 28.
PMID: 22635243BACKGROUNDKoh KN, Im HJ, Kim BE, Choi ES, Jang S, Kwon SW, Park CJ, Seo JJ. Haploidentical haematopoietic stem cell transplantation using CD3 or CD3/CD19 depletion and conditioning with fludarabine, cyclophosphamide and antithymocyte globulin for acquired severe aplastic anaemia. Br J Haematol. 2012 Apr;157(1):139-42. doi: 10.1111/j.1365-2141.2011.08924.x. Epub 2011 Nov 5. No abstract available.
PMID: 22055111RESULTIm HJ, Koh KN, Suh JK, Lee SW, Choi ES, Jang S, Kwon SW, Park CJ, Seo JJ. Refinement of treatment strategies in ex vivo T-cell-depleted haploidentical SCT for pediatric patients. Bone Marrow Transplant. 2015 Feb;50(2):225-31. doi: 10.1038/bmt.2014.232. Epub 2014 Oct 13.
PMID: 25310303DERIVED
Related Links
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Study Officials
- PRINCIPAL INVESTIGATOR
Ho Joon Im, MD, PhD
Asan Medical Center
Central Study Contacts
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- NA
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- OTHER
- Responsible Party
- PRINCIPAL INVESTIGATOR
- PI Title
- Professor
Study Record Dates
First Submitted
December 30, 2012
First Posted
January 3, 2013
Study Start
September 1, 2012
Primary Completion
December 1, 2014
Study Completion
December 1, 2015
Last Updated
January 3, 2013
Record last verified: 2012-12