Ignite Creation Date:
2025-12-26 @ 1:17 PM
Ignite Modification Date:
2026-02-21 @ 8:06 AM
Study NCT ID:
NCT02014506
Status:
UNKNOWN
Last Update Posted:
2017-03-28
First Post:
2013-12-04
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Haploidentical Allogeneic Hematopoietic Stem Cell Transplantation in Children and Adolescents
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, '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': ['PHASE1', 'PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 30}}, 'statusModule': {'overallStatus': 'UNKNOWN', 'lastKnownStatus': 'RECRUITING', 'startDateStruct': {'date': '2017-01', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2017-03', 'completionDateStruct': {'date': '2018-12', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2017-03-24', 'studyFirstSubmitDate': '2013-12-04', 'studyFirstSubmitQcDate': '2013-12-12', 'lastUpdatePostDateStruct': {'date': '2017-03-28', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2013-12-18', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2018-12', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'To evaluate tralsplant-related mortality after haploidentical hematopoietic stem cell transplantation using TCRαβ-depleted graft', 'timeFrame': '1 year posttransplant'}], 'secondaryOutcomes': [{'measure': 'To assess engraftment and graft failure', 'timeFrame': '28 days posttransplant'}, {'measure': 'To estimate the risk of acute GVHD', 'timeFrame': '100 days posttransplant'}, {'measure': 'To estimate the incidence of relapse', 'timeFrame': '100 days and 1 year post-transplant'}, {'measure': 'To estimate the incidence and severity of chronic GVHD', 'timeFrame': '1 year posttransplant'}, {'measure': 'To estimate the overall survival', 'timeFrame': '1 year posttransplant'}, {'measure': 'To estimate the incidence of bacterial, fungal and viral infection', 'timeFrame': '100 days and 1 year posttransplant'}, {'measure': 'To estimate the reactivation rate of CMV, EBV', 'timeFrame': '100 days and 1 year posttransplant'}, {'measure': 'To evaluate the immune reconstitution of T, B, and NK cells', 'timeFrame': 'days 7, 14, 21, 28, 60, 90, 180, 270, and 365 days post-transplant'}, {'measure': 'To evaluate the lineage-specific chimerism using flow cytomery of CD3+, CD19, CD56, TCR αβ, and TCRγδ at pre-transplant', 'timeFrame': 'days 7, 10, 14, 21, 28, 60, 90, 180, 270 and 365 post-transplant'}, {'measure': 'To assess event free survival', 'timeFrame': '1 year posttransplant'}]}, 'oversightModule': {'oversightHasDmc': True}, 'conditionsModule': {'keywords': ['Children and adolescents', 'Malignant disease', 'Non-malignant disease', 'TCRαβ depletion', 'Haploidentical hematopoietic stem cell transplantation'], 'conditions': ['Haploidentical Hematopoietic Stem Cell Transplantation', 'Malignant Disease', 'Non-malignant Disease']}, 'referencesModule': {'references': [{'pmid': '24141651', 'type': 'BACKGROUND', 'citation': 'Park JA, Koh KN, Choi ES, Jang S, Kwon SW, Park CJ, Seo JJ, Im HJ. Successful rescue of early graft failure in pediatric patients using T-cell-depleted haploidentical hematopoietic SCT. Bone Marrow Transplant. 2014 Feb;49(2):270-5. doi: 10.1038/bmt.2013.163. Epub 2013 Oct 21.'}, {'pmid': '23380343', 'type': 'BACKGROUND', 'citation': 'Im HJ, Koh KN, Choi ES, Jang S, Kwon SW, Park CJ, Chi HS, Seo JJ. Excellent outcome of haploidentical hematopoietic stem cell transplantation in children and adolescents with acquired severe aplastic anemia. Biol Blood Marrow Transplant. 2013 May;19(5):754-9. doi: 10.1016/j.bbmt.2013.01.023. Epub 2013 Feb 1.'}, {'pmid': '23993299', 'type': 'BACKGROUND', 'citation': 'Schumm M, Lang P, Bethge W, Faul C, Feuchtinger T, Pfeiffer M, Vogel W, Huppert V, Handgretinger R. Depletion of T-cell receptor alpha/beta and CD19 positive cells from apheresis products with the CliniMACS device. Cytotherapy. 2013 Oct;15(10):1253-8. doi: 10.1016/j.jcyt.2013.05.014.'}], 'seeAlsoLinks': [{'url': 'http://www.mayoclinic.com/health/stem-cell-transplant/MY00089/METHOD=print', 'label': 'Stem cell transplant'}, {'url': 'http://www.nlm.nih.gov/medlineplus/druginfo/meds/a692003.html', 'label': 'fludarabine'}, {'url': 'http://www.nlm.nih.gov/medlineplus/druginfo/meds/a682080.html', 'label': 'cyclophosphamide'}, {'url': 'http://www.nlm.nih.gov/medlineplus/leukemia.html', 'label': 'leukemia'}]}, 'descriptionModule': {'briefSummary': 'Purpose of study: This phase I/II trial is to evaluate the safety and feasibility of TCRαβ-depleted graft from haploidentical family donors in treating children and adolescents with malignant or non-malignant diseases.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT'], 'maximumAge': '21 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\nA. Disease inclusions\n\n1. Hematologic malignancy:\n\n * Acute lymphoblastic leukemia including induction failure, CR1 (Ph+, t(4:11), hypodiploid and other very high risk features), ≥ CR2, infant ALL with MLL or other unfavorable features\n * Acute myeloid leukemia excluding CR1 with t(8:21), inv(16), t(15:17), and Down syndrome\n * Myelodysplastic syndrome: RCC with -7 or RCC in need of transfusion\n * Chronic myeloid leukemia in AP\n * Juvenile myelomonocytic leukemia\n * Malignant lymphoma, NHL or HD, after failed autologous HSCT\n * Other\n2. Non-hematologic malignancy\n\n * Relapsed or refractory solid tumors including neuroblastoma, rhabdomyosarcoma and so on\n3. Non-malignant hematologic disease\n\n * Acquired severe and very severe aplastic anemia\n * Fanconi anemia\n * Paroxysmal nocturnal hemoglobinuria\n * Congenital dyserythropoietic anemia\n * Others\n4. Inherited or metabolic disease\n\n * Hemophagocytic lymphohistiocytosis\n * Malignant osteopetrosis\n * Storage diseases\n * Others B. Recipient inclusions\n\n1\\. Age \\< 21 years 2. No HLA-identical stem cell donor available 3. Lansky-Play performance score \\>60 4. No active infection at the time of transplantation\n\nExclusion Criteria:\n\n1. HIV-infection\n2. Presence of active and serious infection\n3. Cardiac ejection fraction \\<35% on echocardiography\n4. Severe pulmonary dysfunction (DLCO \\<30%)\n5. Liver function abnormalities with bilirubin \\>4mg/dL and elevation of transaminases \\> 400U/L\n6. Concurrent severe or uncontrolled medical disease\n7. Patients who are pregnant\n8. Patients unwilling or unable to comply with the protocol or unable to give informed consent'}, 'identificationModule': {'nctId': 'NCT02014506', 'briefTitle': 'Haploidentical Allogeneic Hematopoietic Stem Cell Transplantation in Children and Adolescents', 'organization': {'class': 'OTHER', 'fullName': 'Asan Medical Center'}, 'officialTitle': 'Haploidentical Allogeneic Hematopoietic Stem Cell Transplantation in Children and Adolescents', 'orgStudyIdInfo': {'id': 'AMCPHO-SCT1303'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'HAPLO', 'interventionNames': ['Drug: Fludarabine', 'Drug: Cyclophosphamide', 'Biological: anti-thymocyte globulin', 'Biological: filgrastim', 'Radiation: Total body irradiation', 'Procedure: TCRαβ-depleted hematopoietic cell transplantation', 'Device: CliniMACS']}], 'interventions': [{'name': 'Fludarabine', 'type': 'DRUG', 'description': '40mg/M2 once daily IV on days -7 to -2', 'armGroupLabels': ['HAPLO']}, {'name': 'Cyclophosphamide', 'type': 'DRUG', 'description': '50 mg/kg IV on day -3 and -2', 'armGroupLabels': ['HAPLO']}, {'name': 'anti-thymocyte globulin', 'type': 'BIOLOGICAL', 'armGroupLabels': ['HAPLO']}, {'name': 'filgrastim', 'type': 'BIOLOGICAL', 'description': '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-6 to -4 (eligible disease except aplastic anemia) 200 cGy per day on D-5 \\& -4 (severe aplastic anemia)', 'armGroupLabels': ['HAPLO']}, {'name': 'TCRαβ-depleted hematopoietic cell transplantation', 'type': 'PROCEDURE', 'armGroupLabels': ['HAPLO']}, {'name': 'CliniMACS', 'type': 'DEVICE', 'description': 'Immunogenetic depletion of TCRαβ cells', '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': 'Ho Joon Im, MD & PhD', 'role': 'PRINCIPAL_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'}}}}