Ignite Creation Date:
2025-12-24 @ 6:34 PM
Ignite Modification Date:
2026-03-17 @ 12:00 AM
Study NCT ID:
NCT02120157
Status:
COMPLETED
Last Update Posted:
2021-11-26
First Post:
2014-04-16
Is NOT Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
Myeloablative Haploidentical BMT With Post-transplant Cyclophosphamide for Pediatric Patients With Hematologic Malignancies
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D054198', 'term': 'Precursor Cell Lymphoblastic Leukemia-Lymphoma'}, {'id': 'D015470', 'term': 'Leukemia, Myeloid, Acute'}, {'id': 'D054429', 'term': 'Leukemia, Myelomonocytic, Juvenile'}], 'ancestors': [{'id': 'D007945', 'term': 'Leukemia, Lymphoid'}, {'id': 'D007938', 'term': 'Leukemia'}, {'id': 'D009370', 'term': 'Neoplasms by Histologic Type'}, {'id': 'D009369', 'term': 'Neoplasms'}, {'id': 'D006402', 'term': 'Hematologic Diseases'}, {'id': 'D006425', 'term': 'Hemic and Lymphatic Diseases'}, {'id': 'D008232', 'term': 'Lymphoproliferative Disorders'}, {'id': 'D008206', 'term': 'Lymphatic Diseases'}, {'id': 'D007160', 'term': 'Immunoproliferative Disorders'}, {'id': 'D007154', 'term': 'Immune System Diseases'}, {'id': 'D007951', 'term': 'Leukemia, Myeloid'}, {'id': 'D054437', 'term': 'Myelodysplastic-Myeloproliferative Diseases'}, {'id': 'D001855', 'term': 'Bone Marrow Diseases'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'D003520', 'term': 'Cyclophosphamide'}, {'id': 'D002066', 'term': 'Busulfan'}, {'id': 'D016559', 'term': 'Tacrolimus'}, {'id': 'D009173', 'term': 'Mycophenolic Acid'}], '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': 'D002072', 'term': 'Butylene Glycols'}, {'id': 'D006018', 'term': 'Glycols'}, {'id': 'D000438', 'term': 'Alcohols'}, {'id': 'D008698', 'term': 'Mesylates'}, {'id': 'D000476', 'term': 'Alkanesulfonates'}, {'id': 'D017738', 'term': 'Alkanesulfonic Acids'}, {'id': 'D000473', 'term': 'Alkanes'}, {'id': 'D006839', 'term': 'Hydrocarbons, Acyclic'}, {'id': 'D013451', 'term': 'Sulfonic Acids'}, {'id': 'D013456', 'term': 'Sulfur Acids'}, {'id': 'D013457', 'term': 'Sulfur Compounds'}, {'id': 'D018942', 'term': 'Macrolides'}, {'id': 'D007783', 'term': 'Lactones'}, {'id': 'D002208', 'term': 'Caproates'}, {'id': 'D000144', 'term': 'Acids, Acyclic'}, {'id': 'D002264', 'term': 'Carboxylic Acids'}, {'id': 'D005227', 'term': 'Fatty Acids'}, {'id': 'D008055', 'term': 'Lipids'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'hsymons2@jhmi.edu', 'phone': '410-502-9961', 'title': 'Heather Symons, MD', 'organization': 'Johns Hopkins University'}, 'certainAgreement': {'piSponsorEmployee': True}}, 'adverseEventsModule': {'timeFrame': 'Adverse event data was collected up to 180 days after transplantation. Mortality was observed for up to 2 year post-intervention.', 'description': 'Adverse events reported at each visit during time at transplant center and up to 180 days post-transplantation.', 'eventGroups': [{'id': 'EG000', 'title': 'Haploidentical BMT With PTCy for Acute Leukemias and MDS', 'description': 'Patients with acute myelogenous leukemia (AML) and myelodysplastic syndrome (MDS):\n\nDays -6 through -3: Busulfan q 5-6h IV q24h x 4 days Days -2 and -1: Cyclophosphamide 50mg/kg/day IV x 2 days+ Mesna 40 mg/kg/day IV\n\nFor patients with acute lymphocytic leukemia (ALL) and lymphoblastic lymphoma:\n\nDays -5 through -4: Cyclophosphamide 50mg/kg/day IV q24h x 2 days+Mesna 40 mg/kg/day IV Days -3 through -1: total body irradiation (TBI) 200 Centigray (cGy) twice a day for 3 days All patients Day 0: Infuse unmanipulated bone marrow Day +3 and +4: Cyclophosphamide 50 mg/kg/day IV + Mesna 40 mg/kg IBW/day IV Day +5: Begin tacrolimus 0.015mg/kg IBW/dose IV over 4 hours q 12h and mycophenolate mofetil (MMF)15mg/kg po/IV tid with maximum daily dose 3 gm/day Day +30: Assess chimerism and disease status in bone marrow Day +35: Discontinue MMF Day +60: Assess chimerism and disease status in bone marrow Day 180: Discontinue tacrolimus Cyclophosphamide: Chemotherapy administration TBI: Radiation Therapy Busulfan: Chemotherapy Administered Unmanipulated Bone Marrow: Bone Marrow Transplant Tacrolimus: Immunosuppressive Drug Administered Mycophenolate mofetil: Immunosuppressive Drug Administered', 'otherNumAtRisk': 32, 'deathsNumAtRisk': 32, 'otherNumAffected': 13, 'seriousNumAtRisk': 32, 'deathsNumAffected': 8, 'seriousNumAffected': 14}], 'otherEvents': [{'term': 'Anemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 20, 'numAffected': 4}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'NCI CTCAE v4.0'}, {'term': 'Anorexia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 4, 'numAffected': 4}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'NCI CTCAE v4.0'}, {'term': 'Diarrhea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 10, 'numAffected': 8}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'NCI CTCAE v4.0'}, {'term': 'Fever', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 6, 'numAffected': 5}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'NCI CTCAE v4.0'}, {'term': 'Hypertension', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 4, 'numAffected': 3}], 'organSystem': 'Vascular disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'NCI CTCAE v4.0'}, {'term': 'Hypokalemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 4, 'numAffected': 4}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'NCI CTCAE v4.0'}, {'term': 'Hypomagnesemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 5, 'numAffected': 5}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'NCI CTCAE v4.0'}, {'term': 'Oral Mucositis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 13, 'numAffected': 11}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'NCI CTCAE v4.0'}, {'term': 'Nausea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 5, 'numAffected': 4}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'NCI CTCAE v4.0'}, {'term': 'Neutrophil Count Decreased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 8, 'numAffected': 2}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'NCI CTCAE v4.0'}, {'term': 'Platelet Count Decreased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 27, 'numAffected': 3}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'NCI CTCAE v4.0'}, {'term': 'Vomiting', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 11, 'numAffected': 8}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'NCI CTCAE v4.0'}, {'term': 'White Blood cells decreased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 19, 'numAffected': 3}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'NCI CTCAE v4.0'}], 'seriousEvents': [{'term': 'Fever', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 3, 'numAffected': 3}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'NCI CTCAE v4.0'}, {'term': 'Veno-occlusive Disease/ Hepatic Sinusoidal Obstruction Syndrome', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 4, 'numAffected': 4}], 'organSystem': 'Hepatobiliary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'NCI CTCAE v4.0'}, {'term': 'Graft Failure', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 3, 'numAffected': 3}], 'organSystem': 'Surgical and medical procedures', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'NCI CTCAE v4.0'}, {'term': 'Acute GVHD', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Surgical and medical procedures', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'NCI CTCAE v4.0'}, {'term': 'Broviac Cather Infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'NCI CTCAE v4.0'}, {'term': 'Intracranial Hemorrhage', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'NCI CTCAE v4.0'}, {'term': 'Cystitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Renal and urinary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'NCI CTCAE v4.0'}], 'frequencyThreshold': '5'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Cumulative Incidence of Non-relapse Mortality', 'denoms': [{'units': 'Participants', 'counts': [{'value': '32', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Haploidentical BMT With PTCy for Acute Leukemias and MDS', 'description': 'Patients with acute myelogenous leukemia (AML) and myelodysplastic syndrome (MDS):\n\nDays -6 through -3: Busulfan q 5-6h IV q24h x 4 days Days -2 and -1: Cyclophosphamide 50mg/kg/day IV x 2 days+ Mesna 40 mg/kg/day IV\n\nFor patients with acute lymphocytic leukemia (ALL) and lymphoblastic lymphoma:\n\nDays -5 through -4: Cyclophosphamide 50mg/kg/day IV q24h x 2 days+Mesna 40 mg/kg/day IV Days -3 through -1: total body irradiation (TBI) 200 Centigray (cGy) twice a day for 3 days All patients Day 0: Infuse unmanipulated bone marrow Day +3 and +4: Cyclophosphamide 50 mg/kg/day IV + Mesna 40 mg/kg IBW/day IV Day +5: Begin tacrolimus 0.015mg/kg IBW/dose IV over 4 hours q 12h and mycophenolate mofetil (MMF)15mg/kg po/IV tid with maximum daily dose 3 gm/day Day +30: Assess chimerism and disease status in bone marrow Day +35: Discontinue MMF Day +60: Assess chimerism and disease status in bone marrow Day 180: Discontinue tacrolimus Cyclophosphamide: Chemotherapy administration TBI: Radiation Therapy Busulfan: Chemotherapy Administered Unmanipulated Bone Marrow: Bone Marrow Transplant Tacrolimus: Immunosuppressive Drug Administered Mycophenolate mofetil: Immunosuppressive Drug Administered'}], 'classes': [{'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'Day 180', 'description': 'Cumulative incidence (measured as a percentage) of non-relapse mortality at 180 days following myeloablative, Human Leukocyte Antigen (HLA)-mismatched bone marrow transplant (BMT) for patients with high risk hematologic malignancies.', 'unitOfMeasure': 'percent', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Number of Participants With Donor Cell Engraftment', 'denoms': [{'units': 'Participants', 'counts': [{'value': '32', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Haploidentical BMT With PTCy for Acute Leukemias and MDS', 'description': 'Patients with AML and MDS:\n\nDays -6 through -3: Busulfan q 5-6h IV q24h x 4 days Days -2 and -1: Cyclophosphamide 50mg/kg/day IV x 2 days+ Mesna 40 mg/kg/day IV\n\nFor patients with ALL and lymphoblastic lymphoma:\n\nDays -5 through -4: Cyclophosphamide 50mg/kg/day IV q24h x 2 days+Mesna 40 mg/kg/day IV Days -3 through -1: TBI 200 Centigray (cGy) twice a day for 3 days All patients Day 0: Infuse unmanipulated bone marrow Day +3 and +4: Cyclophosphamide 50 mg/kg/day IV + Mesna 40 mg/kg IBW/day IV Day +5: Begin tacrolimus 0.015mg/kg IBW/dose IV over 4 hours q 12h and mycophenolate mofetil (MMF)15mg/kg po/IV tid with maximum daily dose 3 gm/day Day +30: Assess chimerism and disease status in bone marrow Day +35: Discontinue MMF Day +60: Assess chimerism and disease status in bone marrow Day 180: Discontinue tacrolimus Cyclophosphamide: Chemotherapy administration TBI: Radiation Therapy Busulfan: Chemotherapy Administered Unmanipulated Bone Marrow: Bone Marrow Transplant Tacrolimus: Immunosuppressive Drug Administered Mycophenolate mofetil: Immunosuppressive Drug Administered'}], 'classes': [{'categories': [{'measurements': [{'value': '27', 'groupId': 'OG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'Day 60', 'description': 'Number of Participants with Donor Cell Engraftment at Day 60 following myeloablative, HLA-mismatched BMT.', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Cumulative Incidence of Acute Graft Versus Host Disease (GVHD) Grades 2-4 and Grades 3-4', 'denoms': [{'units': 'Participants', 'counts': [{'value': '32', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Haploidentical BMT With PTCy for Acute Leukemias and MDS', 'description': 'Patients with AML and MDS:\n\nDays -6 through -3: Busulfan q 5-6h IV q24h x 4 days Days -2 and -1: Cyclophosphamide 50mg/kg/day IV x 2 days+ Mesna 40 mg/kg/day IV\n\nFor patients with ALL and lymphoblastic lymphoma:\n\nDays -5 through -4: Cyclophosphamide 50mg/kg/day IV q24h x 2 days+Mesna 40 mg/kg/day IV Days -3 through -1: TBI 200 Centigray (cGy) twice a day for 3 days All patients Day 0: Infuse unmanipulated bone marrow Day +3 and +4: Cyclophosphamide 50 mg/kg/day IV + Mesna 40 mg/kg IBW/day IV Day +5: Begin tacrolimus 0.015mg/kg IBW/dose IV over 4 hours q 12h and mycophenolate mofetil (MMF)15mg/kg po/IV tid with maximum daily dose 3 gm/day Day +30: Assess chimerism and disease status in bone marrow Day +35: Discontinue MMF Day +60: Assess chimerism and disease status in bone marrow Day 180: Discontinue tacrolimus Cyclophosphamide: Chemotherapy administration TBI: Radiation Therapy Busulfan: Chemotherapy Administered Unmanipulated Bone Marrow: Bone Marrow Transplant Tacrolimus: Immunosuppressive Drug Administered Mycophenolate mofetil: Immunosuppressive Drug Administered'}], 'classes': [{'title': 'Grades 2-4', 'categories': [{'measurements': [{'value': '10', 'groupId': 'OG000'}]}]}, {'title': 'Grades 3-4', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}], 'paramType': 'NUMBER', 'timeFrame': '100 days', 'description': 'Cumulative incidence (measured as a percentage) of acute GVHD grades 2-4 (overall) and grades 3-4 (severe).', 'unitOfMeasure': 'percent', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Cumulative Incidence of Chronic GVHD', 'denoms': [{'units': 'Participants', 'counts': [{'value': '32', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Haploidentical BMT With PTCy for Acute Leukemias and MDS', 'description': 'Patients with AML and MDS:\n\nDays -6 through -3: Busulfan q 5-6h IV q24h x 4 days Days -2 and -1: Cyclophosphamide 50mg/kg/day IV x 2 days+ Mesna 40 mg/kg/day IV\n\nFor patients with ALL and lymphoblastic lymphoma:\n\nDays -5 through -4: Cyclophosphamide 50mg/kg/day IV q24h x 2 days+Mesna 40 mg/kg/day IV Days -3 through -1: TBI 200 Centigray (cGy) twice a day for 3 days All patients Day 0: Infuse unmanipulated bone marrow Day +3 and +4: Cyclophosphamide 50 mg/kg/day IV + Mesna 40 mg/kg IBW/day IV Day +5: Begin tacrolimus 0.015mg/kg IBW/dose IV over 4 hours q 12h and mycophenolate mofetil (MMF)15mg/kg po/IV tid with maximum daily dose 3 gm/day Day +30: Assess chimerism and disease status in bone marrow Day +35: Discontinue MMF Day +60: Assess chimerism and disease status in bone marrow Day 180: Discontinue tacrolimus Cyclophosphamide: Chemotherapy administration TBI: Radiation Therapy Busulfan: Chemotherapy Administered Unmanipulated Bone Marrow: Bone Marrow Transplant Tacrolimus: Immunosuppressive Drug Administered Mycophenolate mofetil: Immunosuppressive Drug Administered'}], 'classes': [{'categories': [{'measurements': [{'value': '11', 'groupId': 'OG000'}]}]}], 'paramType': 'NUMBER', 'timeFrame': '2 years', 'description': 'Cumulative incidence (measured as a percentage) of chronic graft versus host disease (GVHD).', 'unitOfMeasure': 'percent', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Primary and Secondary Graft Failure', 'denoms': [{'units': 'Participants', 'counts': [{'value': '32', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Haploidentical BMT With PTCy for Acute Leukemias and MDS', 'description': 'Patients with AML and MDS:\n\nDays -6 through -3: Busulfan q 5-6h IV q24h x 4 days Days -2 and -1: Cyclophosphamide 50mg/kg/day IV x 2 days+ Mesna 40 mg/kg/day IV\n\nFor patients with ALL and lymphoblastic lymphoma:\n\nDays -5 through -4: Cyclophosphamide 50mg/kg/day IV q24h x 2 days+Mesna 40 mg/kg/day IV Days -3 through -1: TBI 200 Centigray (cGy) twice a day for 3 days All patients Day 0: Infuse unmanipulated bone marrow Day +3 and +4: Cyclophosphamide 50 mg/kg/day IV + Mesna 40 mg/kg IBW/day IV Day +5: Begin tacrolimus 0.015mg/kg IBW/dose IV over 4 hours q 12h and mycophenolate mofetil (MMF)15mg/kg po/IV tid with maximum daily dose 3 gm/day Day +30: Assess chimerism and disease status in bone marrow Day +35: Discontinue MMF Day +60: Assess chimerism and disease status in bone marrow Day 180: Discontinue tacrolimus Cyclophosphamide: Chemotherapy administration TBI: Radiation Therapy Busulfan: Chemotherapy Administered Unmanipulated Bone Marrow: Bone Marrow Transplant Tacrolimus: Immunosuppressive Drug Administered Mycophenolate mofetil: Immunosuppressive Drug Administered'}], 'classes': [{'title': 'Primary', 'categories': [{'measurements': [{'value': '16', 'groupId': 'OG000'}]}]}, {'title': 'Secondary', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}], 'paramType': 'NUMBER', 'timeFrame': '2 years', 'description': 'Incidence (measured as a percentage) of primary and secondary graft failure.', 'unitOfMeasure': 'percentage of graft failure', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Steroid and Non-steroid Immunosuppressants', 'denoms': [{'units': 'Participants', 'counts': [{'value': '32', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Haploidentical BMT With PTCy for Acute Leukemias and MDS', 'description': 'Patients with AML and MDS:\n\nDays -6 through -3: Busulfan q 5-6h IV q24h x 4 days\n\nDays -2 and -1: Cyclophosphamide 50mg/kg/day IV x 2 days+ Mesna 40 mg/kg/day IV\n\nFor patients with ALL and lymphoblastic lymphoma:\n\nDays -5 through -4: Cyclophosphamide 50mg/kg/day IV q24h x 2 days+Mesna 40 mg/kg/day IV\n\nDays -3 through -1: TBI 200 Centigray (cGy) twice a day for 3 days\n\nAll patients Day 0: Infuse unmanipulated bone marrow\n\nDay +3 and +4: Cyclophosphamide 50 mg/kg/day IV + Mesna 40 mg/kg IBW/day IV\n\nDay +5: Begin tacrolimus 0.015mg/kg IBW/dose IV over 4 hours q 12h and mycophenolate mofetil (MMF)15mg/kg po/IV tid with maximum daily dose 3 gm/day\n\nDay +30: Assess chimerism and disease status in bone marrow\n\nDay +35: Discontinue MMF\n\nDay +60: Assess chimerism and disease status in bone marrow\n\nDay 180: Discontinue tacrolimus\n\nCyclophosphamide: Chemotherapy administration\n\nTBI: Radiation Therapy\n\nBusulfan: Chemotherapy Administered\n\nUnmanipulated Bone Marrow: Bone Marrow Transplant\n\nTacrolimus: Immunosuppressive Drug Administered\n\nMycophenolate mofetil: Immunosuppressive Drug Administered'}], 'classes': [{'title': 'Steroid immunosuppressants', 'categories': [{'measurements': [{'value': '4', 'groupId': 'OG000'}]}]}, {'title': 'Non-steroid immunosuppressants', 'categories': [{'measurements': [{'value': '2', 'groupId': 'OG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'Two Years', 'description': 'Number of participants who used steroid and non-steroid immunosuppressants to treat GVHD.', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Steroid and Non-steroid Immunosuppressants Use Duration', 'denoms': [{'units': 'Participants', 'counts': [{'value': '2', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Haploidentical BMT With PTCy for Acute Leukemias and MDS', 'description': 'Patients with AML and MDS:\n\nDays -6 through -3: Busulfan q 5-6h IV q24h x 4 days\n\nDays -2 and -1: Cyclophosphamide 50mg/kg/day IV x 2 days+ Mesna 40 mg/kg/day IV\n\nFor patients with ALL and lymphoblastic lymphoma:\n\nDays -5 through -4: Cyclophosphamide 50mg/kg/day IV q24h x 2 days+Mesna 40 mg/kg/day IV\n\nDays -3 through -1: TBI 200 Centigray (cGy) twice a day for 3 days\n\nAll patients Day 0: Infuse unmanipulated bone marrow\n\nDay +3 and +4: Cyclophosphamide 50 mg/kg/day IV + Mesna 40 mg/kg IBW/day IV\n\nDay +5: Begin tacrolimus 0.015mg/kg IBW/dose IV over 4 hours q 12h and mycophenolate mofetil (MMF)15mg/kg po/IV tid with maximum daily dose 3 gm/day\n\nDay +30: Assess chimerism and disease status in bone marrow\n\nDay +35: Discontinue MMF\n\nDay +60: Assess chimerism and disease status in bone marrow\n\nDay 180: Discontinue tacrolimus\n\nCyclophosphamide: Chemotherapy administration\n\nTBI: Radiation Therapy\n\nBusulfan: Chemotherapy Administered\n\nUnmanipulated Bone Marrow: Bone Marrow Transplant\n\nTacrolimus: Immunosuppressive Drug Administered\n\nMycophenolate mofetil: Immunosuppressive Drug Administered'}], 'classes': [{'title': 'Steroid immunosuppressants', 'denoms': [{'units': 'Participants', 'counts': [{'value': '1', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '18', 'groupId': 'OG000'}]}]}, {'title': 'Non-steroid immunosuppressants', 'denoms': [{'units': 'Participants', 'counts': [{'value': '1', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '19', 'groupId': 'OG000'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'Two Years', 'description': 'Duration of use of steroid and non-steroid immunosuppressants (in months) to treat GVHD.', 'unitOfMeasure': 'months', 'reportingStatus': 'POSTED', 'populationDescription': 'Four participants used immunosuppressants. Four used steroids and 2 of the 4 also used non-steroid. Duration of use for 3 participants using steroids was not recorded. One participant using non-steroid was not recorded.'}, {'type': 'SECONDARY', 'title': 'Survival', 'denoms': [{'units': 'Participants', 'counts': [{'value': '32', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Haploidentical BMT With PTCy for Acute Leukemias and MDS', 'description': 'Patients with AML and MDS:\n\nDays -6 through -3: Busulfan q 5-6h IV q24h x 4 days\n\nDays -2 and -1: Cyclophosphamide 50mg/kg/day IV x 2 days+ Mesna 40 mg/kg/day IV\n\nFor patients with ALL and lymphoblastic lymphoma:\n\nDays -5 through -4: Cyclophosphamide 50mg/kg/day IV q24h x 2 days+Mesna 40 mg/kg/day IV\n\nDays -3 through -1: TBI 200 Centigray (cGy) twice a day for 3 days\n\nAll patients Day 0: Infuse unmanipulated bone marrow\n\nDay +3 and +4: Cyclophosphamide 50 mg/kg/day IV + Mesna 40 mg/kg IBW/day IV\n\nDay +5: Begin tacrolimus 0.015mg/kg IBW/dose IV over 4 hours q 12h and mycophenolate mofetil (MMF)15mg/kg po/IV tid with maximum daily dose 3 gm/day\n\nDay +30: Assess chimerism and disease status in bone marrow\n\nDay +35: Discontinue MMF\n\nDay +60: Assess chimerism and disease status in bone marrow\n\nDay 180: Discontinue tacrolimus\n\nCyclophosphamide: Chemotherapy administration\n\nTBI: Radiation Therapy\n\nBusulfan: Chemotherapy Administered\n\nUnmanipulated Bone Marrow: Bone Marrow Transplant\n\nTacrolimus: Immunosuppressive Drug Administered\n\nMycophenolate mofetil: Immunosuppressive Drug Administered'}], 'classes': [{'title': 'overall survival (OS)', 'categories': [{'measurements': [{'value': '77', 'groupId': 'OG000'}]}]}, {'title': 'progression-free survival (PFS)', 'categories': [{'measurements': [{'value': '68', 'groupId': 'OG000'}]}]}, {'title': 'disease-free survival (DFS)', 'categories': [{'measurements': [{'value': '68', 'groupId': 'OG000'}]}]}, {'title': 'event-free survival', 'categories': [{'measurements': [{'value': '68', 'groupId': 'OG000'}]}]}, {'title': 'Relapse-free GVHD-free survival (GRFS)', 'categories': [{'measurements': [{'value': '65', 'groupId': 'OG000'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'up to 1 years', 'description': 'Estimate incidence of overall survival (OS), progression-free survival (PFS), disease-free survival (DFS), event-free survival, and relapse-free GVHD-free survival (GRFS) in patients receiving myeloablative, HLA-mismatched BMT for patients with high risk hematologic malignancies at 1 year. Incidence as a percentage.', 'unitOfMeasure': 'percent', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Survival', 'denoms': [{'units': 'Participants', 'counts': [{'value': '32', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Haploidentical BMT With PTCy for Acute Leukemias and MDS', 'description': 'Patients with AML and MDS:\n\nDays -6 through -3: Busulfan q 5-6h IV q24h x 4 days\n\nDays -2 and -1: Cyclophosphamide 50mg/kg/day IV x 2 days+ Mesna 40 mg/kg/day IV\n\nFor patients with ALL and lymphoblastic lymphoma:\n\nDays -5 through -4: Cyclophosphamide 50mg/kg/day IV q24h x 2 days+Mesna 40 mg/kg/day IV\n\nDays -3 through -1: TBI 200 Centigray (cGy) twice a day for 3 days\n\nAll patients Day 0: Infuse unmanipulated bone marrow\n\nDay +3 and +4: Cyclophosphamide 50 mg/kg/day IV + Mesna 40 mg/kg IBW/day IV\n\nDay +5: Begin tacrolimus 0.015mg/kg IBW/dose IV over 4 hours q 12h and mycophenolate mofetil (MMF)15mg/kg po/IV tid with maximum daily dose 3 gm/day\n\nDay +30: Assess chimerism and disease status in bone marrow\n\nDay +35: Discontinue MMF\n\nDay +60: Assess chimerism and disease status in bone marrow\n\nDay 180: Discontinue tacrolimus\n\nCyclophosphamide: Chemotherapy administration\n\nTBI: Radiation Therapy\n\nBusulfan: Chemotherapy Administered\n\nUnmanipulated Bone Marrow: Bone Marrow Transplant\n\nTacrolimus: Immunosuppressive Drug Administered\n\nMycophenolate mofetil: Immunosuppressive Drug Administered'}], 'classes': [{'title': 'overall survival (OS)', 'categories': [{'measurements': [{'value': '73', 'groupId': 'OG000'}]}]}, {'title': 'progression-free survival (PFS)', 'categories': [{'measurements': [{'value': '64', 'groupId': 'OG000'}]}]}, {'title': 'disease-free survival (DFS)', 'categories': [{'measurements': [{'value': '64', 'groupId': 'OG000'}]}]}, {'title': 'event-free survival', 'categories': [{'measurements': [{'value': '64', 'groupId': 'OG000'}]}]}, {'title': 'Relapse-free GVHD-free survival (GRFS)', 'categories': [{'measurements': [{'value': '52', 'groupId': 'OG000'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'up to 2 years', 'description': 'Estimate incidence of overall survival (OS), progression-free survival (PFS), disease-free survival (DFS), event-free survival, and relapse-free GVHD-free survival (GRFS) in patients receiving myeloablative, HLA-mismatched BMT for patients with high risk hematologic malignancies at 2 years. Incidence as a percentage.', 'unitOfMeasure': 'percent', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Immune Reconstitution', 'denoms': [{'units': 'Participants', 'counts': [{'value': '0', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Haploidentical BMT With PTCy for Acute Leukemias and MDS', 'description': 'Patients with acute myelogenous leukemia (AML) and myelodysplastic syndrome (MDS):\n\nDays -6 through -3: Busulfan q 5-6h IV q24h x 4 days Days -2 and -1: Cyclophosphamide 50mg/kg/day IV x 2 days+ Mesna 40 mg/kg/day IV\n\nFor patients with acute lymphocytic leukemia (ALL) and lymphoblastic lymphoma:\n\nDays -5 through -4: Cyclophosphamide 50mg/kg/day IV q24h x 2 days+Mesna 40 mg/kg/day IV Days -3 through -1: total body irradiation (TBI) 200 Centigray (cGy) twice a day for 3 days All patients Day 0: Infuse unmanipulated bone marrow Day +3 and +4: Cyclophosphamide 50 mg/kg/day IV + Mesna 40 mg/kg IBW/day IV Day +5: Begin tacrolimus 0.015mg/kg IBW/dose IV over 4 hours q 12h and mycophenolate mofetil (MMF)15mg/kg po/IV tid with maximum daily dose 3 gm/day Day +30: Assess chimerism and disease status in bone marrow Day +35: Discontinue MMF Day +60: Assess chimerism and disease status in bone marrow Day 180: Discontinue tacrolimus Cyclophosphamide: Chemotherapy administration TBI: Radiation Therapy Busulfan: Chemotherapy Administered Unmanipulated Bone Marrow: Bone Marrow Transplant Tacrolimus: Immunosuppressive Drug Administered Mycophenolate mofetil: Immunosuppressive Drug Administered'}], 'timeFrame': 'Two Years', 'description': 'Characterize immune reconstitution post myeloablative haploidentical BMT with Post transplantation cyclophosphamide (PT/Cy).', 'reportingStatus': 'POSTED', 'populationDescription': 'Blood samples collected were not analyzed due to lack of funding. No data was generated.'}, {'type': 'SECONDARY', 'title': 'Time to Neutrophil and Platelet Recovery', 'denoms': [{'units': 'Participants', 'counts': [{'value': '32', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Haploidentical BMT With PTCy for Acute Leukemias and MDS', 'description': 'Patients with AML and MDS:\n\nDays -6 through -3: Busulfan q 5-6h IV q24h x 4 days\n\nDays -2 and -1: Cyclophosphamide 50mg/kg/day IV x 2 days+ Mesna 40 mg/kg/day IV\n\nFor patients with ALL and lymphoblastic lymphoma:\n\nDays -5 through -4: Cyclophosphamide 50mg/kg/day IV q24h x 2 days+Mesna 40 mg/kg/day IV\n\nDays -3 through -1: TBI 200 Centigray (cGy) twice a day for 3 days\n\nAll patients Day 0: Infuse unmanipulated bone marrow\n\nDay +3 and +4: Cyclophosphamide 50 mg/kg/day IV + Mesna 40 mg/kg IBW/day IV\n\nDay +5: Begin tacrolimus 0.015mg/kg IBW/dose IV over 4 hours q 12h and mycophenolate mofetil (MMF)15mg/kg po/IV tid with maximum daily dose 3 gm/day\n\nDay +30: Assess chimerism and disease status in bone marrow\n\nDay +35: Discontinue MMF\n\nDay +60: Assess chimerism and disease status in bone marrow\n\nDay 180: Discontinue tacrolimus\n\nCyclophosphamide: Chemotherapy administration\n\nTBI: Radiation Therapy\n\nBusulfan: Chemotherapy Administered\n\nUnmanipulated Bone Marrow: Bone Marrow Transplant\n\nTacrolimus: Immunosuppressive Drug Administered\n\nMycophenolate mofetil: Immunosuppressive Drug Administered'}], 'classes': [{'title': 'neutrophil', 'categories': [{'measurements': [{'value': '22', 'groupId': 'OG000', 'lowerLimit': '14', 'upperLimit': '58'}]}]}, {'title': 'platelet', 'categories': [{'measurements': [{'value': '21', 'groupId': 'OG000', 'lowerLimit': '12', 'upperLimit': '44'}]}]}], 'paramType': 'MEDIAN', 'timeFrame': '100 days', 'description': 'Time to neutrophil and platelet recovery in median days', 'unitOfMeasure': 'days', 'dispersionType': 'Full Range', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Incidence of Donor Cell Engraftment', 'denoms': [{'units': 'Participants', 'counts': [{'value': '32', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Haploidentical BMT With PTCy for Acute Leukemias and MDS', 'description': 'Patients with AML and MDS:\n\nDays -6 through -3: Busulfan q 5-6h IV q24h x 4 days\n\nDays -2 and -1: Cyclophosphamide 50mg/kg/day IV x 2 days+ Mesna 40 mg/kg/day IV\n\nFor patients with ALL and lymphoblastic lymphoma:\n\nDays -5 through -4: Cyclophosphamide 50mg/kg/day IV q24h x 2 days+Mesna 40 mg/kg/day IV\n\nDays -3 through -1: TBI 200 Centigray (cGy) twice a day for 3 days\n\nAll patients Day 0: Infuse unmanipulated bone marrow\n\nDay +3 and +4: Cyclophosphamide 50 mg/kg/day IV + Mesna 40 mg/kg IBW/day IV\n\nDay +5: Begin tacrolimus 0.015mg/kg IBW/dose IV over 4 hours q 12h and mycophenolate mofetil (MMF)15mg/kg po/IV tid with maximum daily dose 3 gm/day\n\nDay +30: Assess chimerism and disease status in bone marrow\n\nDay +35: Discontinue MMF\n\nDay +60: Assess chimerism and disease status in bone marrow\n\nDay 180: Discontinue tacrolimus\n\nCyclophosphamide: Chemotherapy administration\n\nTBI: Radiation Therapy\n\nBusulfan: Chemotherapy Administered\n\nUnmanipulated Bone Marrow: Bone Marrow Transplant\n\nTacrolimus: Immunosuppressive Drug Administered\n\nMycophenolate mofetil: Immunosuppressive Drug Administered'}], 'classes': [{'categories': [{'measurements': [{'value': '84', 'groupId': 'OG000'}]}]}], 'paramType': 'NUMBER', 'timeFrame': '60 days', 'description': 'Incidence of donor cell engraftment measured as the percentage of donor cell engraftment.', 'unitOfMeasure': 'percentage of donor cell engraftment', 'reportingStatus': 'POSTED'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'Haploidentical BMT With PTCy for Acute Leukemias and MDS', 'description': 'Patients with acute myelogenous leukemia (AML) and myelodysplastic syndrome (MDS):\n\nDays -6 through -3: Busulfan q 5-6h IV q24h x 4 days Days -2 and -1: Cyclophosphamide 50mg/kg/day IV x 2 days+ Mesna 40 mg/kg/day IV\n\nFor patients with acute lymphocytic leukemia (ALL) and lymphoblastic lymphoma:\n\nDays -5 through -4: Cyclophosphamide 50mg/kg/day IV q24h x 2 days+Mesna 40 mg/kg/day IV Days -3 through -1: total body irradiation (TBI) 200 Centigray (cGy) twice a day for 3 days All patients Day 0: Infuse unmanipulated bone marrow Day +3 and +4: Cyclophosphamide 50 mg/kg/day IV + Mesna 40 mg/kg IBW/day IV Day +5: Begin tacrolimus 0.015mg/kg IBW/dose IV over 4 hours q 12h and mycophenolate mofetil (MMF)15mg/kg po/IV tid with maximum daily dose 3 gm/day Day +30: Assess chimerism and disease status in bone marrow Day +35: Discontinue MMF Day +60: Assess chimerism and disease status in bone marrow Day 180: Discontinue tacrolimus Cyclophosphamide: Chemotherapy administration TBI: Radiation Therapy Busulfan: Chemotherapy Administered Unmanipulated Bone Marrow: Bone Marrow Transplant Tacrolimus: Immunosuppressive Drug Administered Mycophenolate mofetil: Immunosuppressive Drug Administered'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '32'}]}, {'type': 'Completed 180 Days', 'achievements': [{'groupId': 'FG000', 'numSubjects': '30'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '24'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '8'}]}], 'dropWithdraws': [{'type': 'Death', 'reasons': [{'groupId': 'FG000', 'numSubjects': '8'}]}]}], 'preAssignmentDetails': "Three patients screen failed due to: One patient could not report on whether the marrow is cellular or at least 20 percent cellularity, one patient had poor liver function, and the one patient's Alanine aminotransferase (ALT) was over the study limit."}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '32', 'groupId': 'BG000'}]}], 'groups': [{'id': 'BG000', 'title': 'Haploidentical BMT With PTCy for Acute Leukemias and MDS', 'description': 'Patients with acute myelogenous leukemia (AML) and myelodysplastic syndrome (MDS):\n\nDays -6 through -3: Busulfan q 5-6h IV q24h x 4 days Days -2 and -1: Cyclophosphamide 50mg/kg/day IV x 2 days+ Mesna 40 mg/kg/day IV\n\nFor patients with acute lymphocytic leukemia (ALL) and lymphoblastic lymphoma:\n\nDays -5 through -4: Cyclophosphamide 50mg/kg/day IV q24h x 2 days+Mesna 40 mg/kg/day IV Days -3 through -1: total body irradiation (TBI) 200 Centigray (cGy) twice a day for 3 days All patients Day 0: Infuse unmanipulated bone marrow Day +3 and +4: Cyclophosphamide 50 mg/kg/day IV + Mesna 40 mg/kg IBW/day IV Day +5: Begin tacrolimus 0.015mg/kg IBW/dose IV over 4 hours q 12h and mycophenolate mofetil (MMF)15mg/kg po/IV tid with maximum daily dose 3 gm/day Day +30: Assess chimerism and disease status in bone marrow Day +35: Discontinue MMF Day +60: Assess chimerism and disease status in bone marrow Day 180: Discontinue tacrolimus Cyclophosphamide: Chemotherapy administration TBI: Radiation Therapy Busulfan: Chemotherapy Administered Unmanipulated Bone Marrow: Bone Marrow Transplant Tacrolimus: Immunosuppressive Drug Administered Mycophenolate mofetil: Immunosuppressive Drug Administered'}], 'measures': [{'title': 'Age, Continuous', 'classes': [{'categories': [{'measurements': [{'value': '12', 'groupId': 'BG000', 'lowerLimit': '1', 'upperLimit': '23'}]}]}], 'paramType': 'MEDIAN', 'unitOfMeasure': 'years', 'dispersionType': 'FULL_RANGE'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '13', 'groupId': 'BG000'}]}, {'title': 'Male', 'measurements': [{'value': '19', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Ethnicity (NIH/OMB)', 'classes': [{'categories': [{'title': 'Hispanic or Latino', 'measurements': [{'value': '11', 'groupId': 'BG000'}]}, {'title': 'Not Hispanic or Latino', 'measurements': [{'value': '21', 'groupId': 'BG000'}]}, {'title': 'Unknown or Not Reported', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Race (NIH/OMB)', 'classes': [{'categories': [{'title': 'American Indian or Alaska Native', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Asian', 'measurements': [{'value': '2', 'groupId': 'BG000'}]}, {'title': 'Native Hawaiian or Other Pacific Islander', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Black or African American', 'measurements': [{'value': '6', 'groupId': 'BG000'}]}, {'title': 'White', 'measurements': [{'value': '18', 'groupId': 'BG000'}]}, {'title': 'More than one race', 'measurements': [{'value': '4', 'groupId': 'BG000'}]}, {'title': 'Unknown or Not Reported', 'measurements': [{'value': '2', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Region of Enrollment', 'classes': [{'title': 'Canada', 'categories': [{'measurements': [{'value': '7', 'groupId': 'BG000'}]}]}, {'title': 'United States', 'categories': [{'measurements': [{'value': '25', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}]}}, 'documentSection': {'largeDocumentModule': {'largeDocs': [{'date': '2017-04-20', 'size': 1182898, 'label': 'Study Protocol and Statistical Analysis Plan', 'hasIcf': False, 'hasSap': True, 'filename': 'Prot_SAP_001.pdf', 'typeAbbrev': 'Prot_SAP', 'uploadDate': '2019-06-20T15:16', 'hasProtocol': True}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 35}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2015-07-02', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2021-11', 'completionDateStruct': {'date': '2020-10-01', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2021-11-23', 'studyFirstSubmitDate': '2014-04-16', 'resultsFirstSubmitDate': '2019-03-28', 'studyFirstSubmitQcDate': '2014-04-18', 'lastUpdatePostDateStruct': {'date': '2021-11-26', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2019-03-28', 'studyFirstPostDateStruct': {'date': '2014-04-22', 'type': 'ESTIMATED'}, 'resultsFirstPostDateStruct': {'date': '2019-04-18', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2018-06-15', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Cumulative Incidence of Non-relapse Mortality', 'timeFrame': 'Day 180', 'description': 'Cumulative incidence (measured as a percentage) of non-relapse mortality at 180 days following myeloablative, Human Leukocyte Antigen (HLA)-mismatched bone marrow transplant (BMT) for patients with high risk hematologic malignancies.'}], 'secondaryOutcomes': [{'measure': 'Number of Participants With Donor Cell Engraftment', 'timeFrame': 'Day 60', 'description': 'Number of Participants with Donor Cell Engraftment at Day 60 following myeloablative, HLA-mismatched BMT.'}, {'measure': 'Cumulative Incidence of Acute Graft Versus Host Disease (GVHD) Grades 2-4 and Grades 3-4', 'timeFrame': '100 days', 'description': 'Cumulative incidence (measured as a percentage) of acute GVHD grades 2-4 (overall) and grades 3-4 (severe).'}, {'measure': 'Cumulative Incidence of Chronic GVHD', 'timeFrame': '2 years', 'description': 'Cumulative incidence (measured as a percentage) of chronic graft versus host disease (GVHD).'}, {'measure': 'Primary and Secondary Graft Failure', 'timeFrame': '2 years', 'description': 'Incidence (measured as a percentage) of primary and secondary graft failure.'}, {'measure': 'Steroid and Non-steroid Immunosuppressants', 'timeFrame': 'Two Years', 'description': 'Number of participants who used steroid and non-steroid immunosuppressants to treat GVHD.'}, {'measure': 'Steroid and Non-steroid Immunosuppressants Use Duration', 'timeFrame': 'Two Years', 'description': 'Duration of use of steroid and non-steroid immunosuppressants (in months) to treat GVHD.'}, {'measure': 'Survival', 'timeFrame': 'up to 1 years', 'description': 'Estimate incidence of overall survival (OS), progression-free survival (PFS), disease-free survival (DFS), event-free survival, and relapse-free GVHD-free survival (GRFS) in patients receiving myeloablative, HLA-mismatched BMT for patients with high risk hematologic malignancies at 1 year. Incidence as a percentage.'}, {'measure': 'Survival', 'timeFrame': 'up to 2 years', 'description': 'Estimate incidence of overall survival (OS), progression-free survival (PFS), disease-free survival (DFS), event-free survival, and relapse-free GVHD-free survival (GRFS) in patients receiving myeloablative, HLA-mismatched BMT for patients with high risk hematologic malignancies at 2 years. Incidence as a percentage.'}, {'measure': 'Immune Reconstitution', 'timeFrame': 'Two Years', 'description': 'Characterize immune reconstitution post myeloablative haploidentical BMT with Post transplantation cyclophosphamide (PT/Cy).'}, {'measure': 'Time to Neutrophil and Platelet Recovery', 'timeFrame': '100 days', 'description': 'Time to neutrophil and platelet recovery in median days'}, {'measure': 'Incidence of Donor Cell Engraftment', 'timeFrame': '60 days', 'description': 'Incidence of donor cell engraftment measured as the percentage of donor cell engraftment.'}]}, 'oversightModule': {'oversightHasDmc': True}, 'conditionsModule': {'keywords': ['Acute lymphoblastic leukemia', 'Acute myeloid leukemia', 'Juvenile myelomonocytic leukemia', 'Treatment related leukemia', 'Biphenotypic leukemia'], 'conditions': ['Myeloablative Conditioning', 'HLA-mismatched Bone Marrow Transplantation', 'Graft Survival', 'Transplantation, Bone Marrow']}, 'referencesModule': {'references': [{'pmid': '37257193', 'type': 'DERIVED', 'citation': 'Fierro-Pineda JC, Tsai HL, Blackford A, Cluster A, Caywood E, Dalal J, Davis J, Egeler M, Huo J, Hudspeth M, Keating A, Kelly SS, Krueger J, Lee D, Lehmann L, Madden L, Oshrine B, Pulsipher MA, Fry T, Symons HJ. Prospective PTCTC trial of myeloablative haplo-BMT with posttransplant cyclophosphamide for pediatric acute leukemias. Blood Adv. 2023 Sep 26;7(18):5639-5648. doi: 10.1182/bloodadvances.2023010281.'}]}, 'descriptionModule': {'briefSummary': 'This is a multi-institutional phase II haploidentical T cell replete bone marrow transplant (BMT) study in children with high-risk leukemia. The myeloablative conditioning regimen prescribed will be Total body irradiation (TBI)-based for lymphoid leukemia and busulfan-based for myeloid leukemia. Our goal is to establish an easily exportable, inexpensive platform for haplotransplantation that has a safety profile equivalent to matched related and unrelated BMTs. The primary objective will be to estimate the incidence of 6-month non-relapse mortality (NRM), hypothesizing that NRM is \\< 18%.', 'detailedDescription': 'This is a phase II prospective study designed to evaluate the incidence of 6 month non- relapse mortality, safety, and feasibility of haploidentical bone marrow transplantation (BMT) after myeloablative conditioning with post-transplant Cy. Conditioning regimens include a total body irradiation (TBI)-based prep for lymphoid leukemias and a chemotherapy based prep for myeloid leukemias.\n\nTo estimate the incidence of non-relapse mortality at 180 days following myeloablative haploidentical BMT for children and young adults with high risk hematologic malignancies.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT'], 'maximumAge': '25 Years', 'minimumAge': '6 Months', 'healthyVolunteers': False, 'eligibilityCriteria': "Inclusion Criteria:\n\n* Patient age 0.5-25years\n* Patients must have a first-degree related donor or half-sibling who is at minimum HLA haploidentical. The donor and recipient must be identical at at least one allele of each of the following genetic loci: HLA-A, HLA-B, HLA-Cw, HLA-DRB1, and HLA-DQB1. A minimum match of 5/10 is therefore required, and will be considered sufficient evidence that the donor and recipient share one HLA haplotype.\n* An unrelated donor search is not required for a patient to be eligible for this protocol, or a donor search and donor mobilization may be abandoned if the clinical situation dictates an urgent transplant. Clinical urgency is defined as 6-8 weeks from referral to transplant or a low-likelihood of finding a matched, unrelated donor. Patients with an eligible HLA-matched RELATED should not be enrolled on this trial.\n* Patients must have at least one of the following high-risk conditions listed below:\n* Acute lymphocytic leukemia (ALL) in CR1\\* as defined by at least one of the following:\n\nhypodiploidy, induction failure,Minimal residual disease (MRD) after consolidation\n\n\\- Acute myeloid leukemia (AML) in CR1 with high risk features defined as: High allelic ratio FLT3/ITD+, Monosomy 7, Del (5q), Standard risk cytogenetics with positive minimal residual disease at the end of Induction I chemotherapy (for patients being treated on or according to Children's Oncology Group (COG) AAML1031 study who have had MRD studies sent to Seattle or performed at their local institution where the flow assay is sensitive enough to detect \\> 0.1% blasts)\n\n* Acute Leukemia in 2nd or subsequent CR (CR\\>2)\n* Mixed phenotype/Undifferentiated Leukemia in 1st or subsequent CR\\*\n* Secondary or therapy related leukemia in CR \\> 1\n* Natural Killer (NK) cell lymphoblastic leukemia CR \\> 1\n* Myelodysplastic syndrome (MDS)\n* Juvenile myelomonocytic leukemia (JMML) (patients are eligible if they are not eligible for COG1221 study)\n* Prior transplant eligible if \\< 18yo, \\>6 months has elapsed since BMT, and patient is off immunosuppression for \\> 3 months with no Graft versus host disease (GVHD)\n* No known active Central nervous system (CNS) involvement or extramedullary involvement by malignancy. Such disease treated into remission is permitted.\n* Acute Leukemia - Remission is defined as morphology with \\< 5% blasts with no morphological characteristics of acute leukemia (e.g., Auer Rods) in a bone marrow with \\> 20% cellularity.\n\nExclusion Criteria:\n\n* Poor cardiac function: left ventricular ejection fraction \\<45% as determined by Multigated acquisition scan (MUGA) or Echocardiogram (ECHO). For pediatric patients Left ventricular ejection fraction (LVEF) \\<45% or a shortening fraction below normal limits for age.\n* Symptomatic pulmonary disease. Poor pulmonary function: Forced expiratory volume (FEV1), Forced vital capacity (FVC), and Diffusing capacity for carbon monoxide (DLCO) \\<50% predicted (corrected for hemoglobin) for patients who have not received thoracic or mantle irradiation. For patients who have received thoracic or mantle irradiation, FEV1 and FVC \\<70% predicted or DLCO \\< 50 of predicted. For children unable to perform Pulmonary function tests (PFTs) because of developmental stage pulse oximetry \\< 92% on Room air (RA).\n* Poor liver function: bilirubin \\>2 mg/dl (not due to hemolysis, Gilbert's or primary malignancy). Alanine aminotransferase (ALT) or Aspartate transaminase (AST) \\> 3 x laboratory upper normal limits.\n* Poor renal function: Creatinine \\>2.0mg/dl or creatinine clearance (calculated creatinine clearance is permitted) \\< 60 mL/min based on Traditional Cockcroft-Gault formula: 140 - age (yrs) x Smaller of Actual Weight vs. Ideal Body Weight (kg) / 72 x Serum creatinine (mg/dl) Multiply by another factor of 0.85 if female Intended for ages 18-110, serum creatinine 0.6-7 mg/dl For patients \\<18 years: creatinine clearance (CrCl) will be estimated by the Schwartz formula. A measured CrCl or a Glomerular filtration rate (GFR) may be substituted to determine the subject's CrCl.\n* Schwartz equation: CrCl (ml/min/1.73m2)=\\[length (cm) x k\\] /serum creatinine K = 0.45 for infants 1 to 52 weeks old k = 0.55 for children 1 to 13 years old k = 0.55 for adolescent females 13-18 years old k = 0.7 for adolescent males 13-18 years old\n* HIV-positive\n* Positive leukocytotoxic crossmatch Specifically, complement dependent cytotoxicity and flow cytometric crossmatch assays must be negative, and the mean fluorescence intensity (MFI) of any anti-donor HLA antibody by solid phase immunoassay should be \\<3000. Consult with PI for the clinical significance of any anti-donor antibody.\n* Women of childbearing potential who currently are pregnant (HCG+) or who are not practicing adequate contraception or who are breastfeeding\n* Uncontrolled viral, bacterial, or fungal infections (currently taking medication and have progression of clinical symptoms)\n* Patients with symptoms consistent with Respiratory syncytial virus (RSV), influenza A, B, or parainfluenza at the time of enrollment will be assayed for the above viruses and if positive are not eligible for the trial until they are no longer symptomatic (patients may have continued assay positivity for a period of time post resolution of symptoms secondary to the nature of the assay"}, 'identificationModule': {'nctId': 'NCT02120157', 'briefTitle': 'Myeloablative Haploidentical BMT With Post-transplant Cyclophosphamide for Pediatric Patients With Hematologic Malignancies', 'organization': {'class': 'OTHER', 'fullName': 'Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins'}, 'officialTitle': 'Pediatric Blood & Marrow Transplant Consortium (PBMTC) Phase II Myeloablative Haploidentical BMT With Post-transplantation Cyclophosphamide for Pediatric Patients With Hematologic Malignancies', 'orgStudyIdInfo': {'id': 'J13161'}, 'secondaryIdInfos': [{'id': 'NA_00091665', 'type': 'OTHER', 'domain': 'JHM IRB'}]}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Haploidentical BMT with PTCy for acute leukemias and MDS', 'description': 'Patients with AML and MDS:\n\nDays -6 through -3: Busulfan q 5-6h IV q24h x 4 days\n\nDays -2 and -1: Cyclophosphamide 50mg/kg/day IV x 2 days+ Mesna 40 mg/kg/day IV\n\nFor patients with ALL and lymphoblastic lymphoma:\n\nDays -5 through -4: Cyclophosphamide 50mg/kg/day IV q24h x 2 days+Mesna 40 mg/kg/day IV\n\nDays -3 through -1: TBI 200 Centigray (cGy) twice a day for 3 days\n\nAll patients Day 0: Infuse unmanipulated bone marrow\n\nDay +3 and +4: Cyclophosphamide 50 mg/kg/day IV + Mesna 40 mg/kg IBW/day IV\n\nDay +5: Begin tacrolimus 0.015mg/kg IBW/dose IV over 4 hours q 12h and mycophenolate mofetil (MMF)15mg/kg po/IV tid with maximum daily dose 3 gm/day\n\nDay +30: Assess chimerism and disease status in bone marrow\n\nDay +35: Discontinue MMF\n\nDay +60: Assess chimerism and disease status in bone marrow\n\nDay 180: Discontinue tacrolimus', 'interventionNames': ['Drug: Cyclophosphamide', 'Radiation: TBI', 'Drug: Busulfan', 'Other: Unmanipulated Bone Marrow', 'Drug: Tacrolimus', 'Drug: Mycophenolate mofetil']}], 'interventions': [{'name': 'Cyclophosphamide', 'type': 'DRUG', 'otherNames': ['Cy'], 'description': 'Chemotherapy administration', 'armGroupLabels': ['Haploidentical BMT with PTCy for acute leukemias and MDS']}, {'name': 'TBI', 'type': 'RADIATION', 'description': 'Radiation Therapy', 'armGroupLabels': ['Haploidentical BMT with PTCy for acute leukemias and MDS']}, {'name': 'Busulfan', 'type': 'DRUG', 'otherNames': ['Bu'], 'description': 'Chemotherapy Administered', 'armGroupLabels': ['Haploidentical BMT with PTCy for acute leukemias and MDS']}, {'name': 'Unmanipulated Bone Marrow', 'type': 'OTHER', 'description': 'Bone Marrow Transplant', 'armGroupLabels': ['Haploidentical BMT with PTCy for acute leukemias and MDS']}, {'name': 'Tacrolimus', 'type': 'DRUG', 'otherNames': ['tacro'], 'description': 'Immunosuppressive Drug Administered', 'armGroupLabels': ['Haploidentical BMT with PTCy for acute leukemias and MDS']}, {'name': 'Mycophenolate mofetil', 'type': 'DRUG', 'otherNames': ['MMF'], 'description': 'Immunosuppressive Drug Administered', 'armGroupLabels': ['Haploidentical BMT with PTCy for acute leukemias and MDS']}]}, 'contactsLocationsModule': {'locations': [{'zip': '80045', 'city': 'Aurora', 'state': 'Colorado', 'country': 'United States', 'facility': "Children's Hospital of Colorado", 'geoPoint': {'lat': 39.72943, 'lon': -104.83192}}, {'zip': '19803', 'city': 'Wilmington', 'state': 'Delaware', 'country': 'United States', 'facility': 'Nemours Alfred I. DuPont Hospital for Children', 'geoPoint': {'lat': 39.74595, 'lon': -75.54659}}, {'zip': '33701', 'city': 'St. Petersburg', 'state': 'Florida', 'country': 'United States', 'facility': "All Children's Hospital Johns Hopkins Medicine", 'geoPoint': {'lat': 27.77086, 'lon': -82.67927}}, {'zip': '21287', 'city': 'Baltimore', 'state': 'Maryland', 'country': 'United States', 'facility': 'Johns Hopkins Hospital', 'geoPoint': {'lat': 39.29038, 'lon': -76.61219}}, {'zip': '63110', 'city': 'St Louis', 'state': 'Missouri', 'country': 'United States', 'facility': 'Washington University in St. Louis', 'geoPoint': {'lat': 38.62727, 'lon': -90.19789}}, {'zip': '28203', 'city': 'Charlotte', 'state': 'North Carolina', 'country': 'United States', 'facility': 'Levine Cancer Center', 'geoPoint': {'lat': 35.22709, 'lon': -80.84313}}, {'zip': '29423', 'city': 'Charleston', 'state': 'South Carolina', 'country': 'United States', 'facility': 'Medical University of South Carolina', 'geoPoint': {'lat': 32.77632, 'lon': -79.93275}}, {'zip': 'V6H 3V4', 'city': 'Vancouver', 'state': 'British Columbia', 'country': 'Canada', 'facility': "British Columbia Children's Hospital", 'geoPoint': {'lat': 49.24966, 'lon': -123.11934}}, {'zip': 'M5G 1X8', 'city': 'Toronto', 'state': 'Ontario', 'country': 'Canada', 'facility': 'Hospital for Sick Children', 'geoPoint': {'lat': 43.70643, 'lon': -79.39864}}], 'overallOfficials': [{'name': 'Heather Symons, MD, MHS', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'SKCCC Johns Hopkins Hospital'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins', 'class': 'OTHER'}, 'responsibleParty': {'type': 'SPONSOR'}}}}