Ignite Creation Date:
2025-12-24 @ 9:42 PM
Ignite Modification Date:
2026-02-19 @ 4:25 AM
Study NCT ID:
NCT01759732
Status:
UNKNOWN
Last Update Posted:
2013-01-03
First Post:
2012-12-30
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Haploidentical Stem Cell Transplantation for Children With Acquired Severe Aplastic Anemia
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D000741', 'term': 'Anemia, Aplastic'}], 'ancestors': [{'id': 'D000740', 'term': 'Anemia'}, {'id': 'D006402', 'term': 'Hematologic Diseases'}, {'id': 'D006425', 'term': 'Hemic and Lymphatic Diseases'}, {'id': 'D000080983', 'term': 'Bone Marrow Failure Disorders'}, {'id': 'D001855', 'term': 'Bone Marrow Diseases'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'C024352', 'term': 'fludarabine'}, {'id': 'D003520', 'term': 'Cyclophosphamide'}, {'id': 'D000961', 'term': 'Antilymphocyte Serum'}, {'id': 'D000069585', 'term': 'Filgrastim'}, {'id': 'D014916', 'term': 'Whole-Body Irradiation'}], 'ancestors': [{'id': 'D010752', 'term': 'Phosphoramide Mustards'}, {'id': 'D009588', 'term': 'Nitrogen Mustard Compounds'}, {'id': 'D009150', 'term': 'Mustard Compounds'}, {'id': 'D006846', 'term': 'Hydrocarbons, Halogenated'}, {'id': 'D006838', 'term': 'Hydrocarbons'}, {'id': 'D009930', 'term': 'Organic Chemicals'}, {'id': 'D063088', 'term': 'Phosphoramides'}, {'id': 'D009943', 'term': 'Organophosphorus Compounds'}, {'id': 'D007106', 'term': 'Immune Sera'}, {'id': 'D000906', 'term': 'Antibodies'}, {'id': 'D007136', 'term': 'Immunoglobulins'}, {'id': 'D007162', 'term': 'Immunoproteins'}, {'id': 'D001798', 'term': 'Blood Proteins'}, {'id': 'D011506', 'term': 'Proteins'}, {'id': 'D000602', 'term': 'Amino Acids, Peptides, and Proteins'}, {'id': 'D012712', 'term': 'Serum Globulins'}, {'id': 'D005916', 'term': 'Globulins'}, {'id': 'D001688', 'term': 'Biological Products'}, {'id': 'D045424', 'term': 'Complex Mixtures'}, {'id': 'D016179', 'term': 'Granulocyte Colony-Stimulating Factor'}, {'id': 'D003115', 'term': 'Colony-Stimulating Factors'}, {'id': 'D006023', 'term': 'Glycoproteins'}, {'id': 'D006001', 'term': 'Glycoconjugates'}, {'id': 'D002241', 'term': 'Carbohydrates'}, {'id': 'D016298', 'term': 'Hematopoietic Cell Growth Factors'}, {'id': 'D016207', 'term': 'Cytokines'}, {'id': 'D036341', 'term': 'Intercellular Signaling Peptides and Proteins'}, {'id': 'D010455', 'term': 'Peptides'}, {'id': 'D001685', 'term': 'Biological Factors'}, {'id': 'D011878', 'term': 'Radiotherapy'}, {'id': 'D013812', 'term': 'Therapeutics'}, {'id': 'D008919', 'term': 'Investigative Techniques'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 10}}, 'statusModule': {'overallStatus': 'UNKNOWN', 'lastKnownStatus': 'RECRUITING', 'startDateStruct': {'date': '2012-09'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2012-12', 'completionDateStruct': {'date': '2015-12', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2012-12-30', 'studyFirstSubmitDate': '2012-12-30', 'studyFirstSubmitQcDate': '2012-12-30', 'lastUpdatePostDateStruct': {'date': '2013-01-03', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2013-01-03', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2014-12', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': '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', 'timeFrame': '2 years posttransplant'}], 'secondaryOutcomes': [{'measure': 'To assess engraftment and graft failure', 'timeFrame': '28 days posttransplant', 'description': 'Number of patients who failed to engraft by 28 days'}, {'measure': 'To estimate the risk of acute GVHD', 'timeFrame': '100 days posttransplant', 'description': 'Number of patients with acute GVHD.'}, {'measure': 'To assess treatment related mortality', 'timeFrame': '100 days posttransplant', 'description': 'Number of death after transplantation'}, {'measure': 'To estimate overall survival and failure free survival', 'timeFrame': '1 year posttransplant'}]}, 'conditionsModule': {'keywords': ['Aplastic anemia', 'T cell dose', 'CD3 depletion', 'Haploidentical hematopoietic stem cell transplantation'], 'conditions': ['Acquired Aplastic Anemia']}, 'referencesModule': {'references': [{'pmid': '22533862', 'type': 'BACKGROUND', 'citation': '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': '22546497', 'type': 'BACKGROUND', 'citation': 'Tolar 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': '22635243', 'type': 'BACKGROUND', 'citation': 'Xu 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': '22055111', 'type': 'RESULT', 'citation': 'Koh 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': '25310303', 'type': 'DERIVED', 'citation': 'Im 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.'}], 'seeAlsoLinks': [{'url': 'http://www.nlm.nih.gov/medlineplus/anemia.html', 'label': 'Anemia'}, {'url': 'http://druginfo.nlm.nih.gov/drugportal/ProxyServlet?mergeData=true&objectHandle=DBMaint&...', 'label': 'Fludarabine'}, {'url': 'http://druginfo.nlm.nih.gov/drugportal/ProxyServlet', 'label': 'Cyclophosphamide'}]}, 'descriptionModule': {'briefSummary': '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.\n\nPurpose: 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'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT'], 'maximumAge': '21 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Diagnosis of life-threatening marrow failure (severe aplastic anemia) of nonmalignant etiology meeting 2 of the following criteria:\n\n * Granulocyte count \\< 500/mm3,\n * Corrected reticulocyte count \\< 1%,\n * Platelet count \\< 20,000/mm3\n* No HLA-identical family member or closely matched (8 of 8 HLA-locus match) unrelated marrow donor available\n* HLA-haploidentical related donor available\n\nExclusion Criteria:\n\n* Paroxysmal nocturnal hemoglobinuria or Fanconi anemia\n* Clonal cytogenetic abnormalities or myelodysplastic syndromes\n* Active fungal infections\n* HIV positive\n* Severe disease other than aplastic anemia that would severely limit the probability of survival during the graft procedure\n* Pregnant or nursing'}, 'identificationModule': {'nctId': 'NCT01759732', 'briefTitle': 'Haploidentical Stem Cell Transplantation for Children With Acquired Severe Aplastic Anemia', 'organization': {'class': 'OTHER', 'fullName': 'Asan Medical Center'}, 'officialTitle': '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', 'orgStudyIdInfo': {'id': 'AMCPHO-SCT1202'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'HAPLO', 'interventionNames': ['Drug: Fludarabine', 'Drug: Cyclophosphamide', 'Biological: anti-thymocyte globulin', 'Biological: filgrastim', 'Radiation: Total body irradiation', 'Procedure: CD3-depleted hematopoietic cell transplantation']}], 'interventions': [{'name': 'Fludarabine', 'type': 'DRUG', 'description': '40mg/M2 once daily IV on days -7 to -4', 'armGroupLabels': ['HAPLO']}, {'name': 'Cyclophosphamide', 'type': 'DRUG', 'description': '60 mg/kg IV on day-3 and -2', 'armGroupLabels': ['HAPLO']}, {'name': 'anti-thymocyte globulin', 'type': 'BIOLOGICAL', 'armGroupLabels': ['HAPLO']}, {'name': 'filgrastim', 'type': 'BIOLOGICAL', 'otherNames': ['Beginning on day 4 and continuing until blood counts recover'], 'armGroupLabels': ['HAPLO']}, {'name': 'Total body irradiation', 'type': 'RADIATION', 'description': '200 cGy per day on D-5 \\& -4', 'armGroupLabels': ['HAPLO']}, {'name': 'CD3-depleted hematopoietic cell transplantation', 'type': 'PROCEDURE', 'description': 'Immunogenetic depletion on CliniMACS', 'armGroupLabels': ['HAPLO']}]}, 'contactsLocationsModule': {'locations': [{'zip': '138-736', 'city': 'Seoul', 'status': 'RECRUITING', 'country': 'South Korea', 'contacts': [{'name': 'Ho Joon Im, MD, PhD', 'role': 'CONTACT', 'email': 'hojim@amc.seoul.kr', 'phone': '82-2-3010-3371'}, {'name': 'Kyung Nam Koh, MD, PhD', 'role': 'SUB_INVESTIGATOR'}], 'facility': 'Asan Medical Center', 'geoPoint': {'lat': 37.566, 'lon': 126.9784}}], 'centralContacts': [{'name': 'Ho Joon Im, MD, PhD', 'role': 'CONTACT', 'email': 'hojim@amc.seoul.kr', 'phone': '82-2-3010-3371'}], 'overallOfficials': [{'name': 'Ho Joon Im, MD, PhD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Asan Medical Center'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Asan Medical Center', 'class': 'OTHER'}, 'responsibleParty': {'type': 'PRINCIPAL_INVESTIGATOR', 'investigatorTitle': 'Professor', 'investigatorFullName': 'Ho Joon Im', 'investigatorAffiliation': 'Asan Medical Center'}}}}