Ignite Creation Date:
2025-12-25 @ 3:28 AM
Ignite Modification Date:
2026-06-25 @ 1:01 AM
Study NCT ID:
NCT02833805
Status:
COMPLETED
Last Update Posted:
2022-07-12
First Post:
2016-07-12
Is NOT Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
NMA Haplo or MUD BMT for Newly Diagnosed Severe Aplastic Anemia
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D000741', 'term': 'Anemia, Aplastic'}, {'id': 'D000080983', 'term': 'Bone Marrow Failure Disorders'}], 'ancestors': [{'id': 'D000740', 'term': 'Anemia'}, {'id': 'D006402', 'term': 'Hematologic Diseases'}, {'id': 'D006425', 'term': 'Hemic and Lymphatic Diseases'}, {'id': 'D001855', 'term': 'Bone Marrow Diseases'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'C512542', 'term': 'thymoglobulin'}, {'id': 'D000961', 'term': 'Antilymphocyte Serum'}, {'id': 'C024352', 'term': 'fludarabine'}, {'id': 'C042382', 'term': 'fludarabine phosphate'}, {'id': 'D003520', 'term': 'Cyclophosphamide'}, {'id': 'D014916', 'term': 'Whole-Body Irradiation'}, {'id': 'D016559', 'term': 'Tacrolimus'}, {'id': 'D009173', 'term': 'Mycophenolic Acid'}], 'ancestors': [{'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': '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': 'D011878', 'term': 'Radiotherapy'}, {'id': 'D013812', 'term': 'Therapeutics'}, {'id': 'D008919', 'term': 'Investigative Techniques'}, {'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': 'adezerb1@jhmi.edu', 'phone': '410-502-7208', 'title': 'Dr. Amy DeZern', 'organization': 'Johns Hopkins University'}, 'certainAgreement': {'piSponsorEmployee': False, 'restrictiveAgreement': False}}, 'adverseEventsModule': {'timeFrame': 'Adverse events were monitored and collected from time of consent up to one year.', 'eventGroups': [{'id': 'EG000', 'title': 'Bone Marrow Transplant', 'description': 'Non-myeloablative bone marrow transplant with a Thymoglobulin (ATG), fludarabine (Flu), cyclophosphamide (Cy), total body irradiation (TBI) preparative regimen and post-transplant Cy, mycophenolate mofetil (MMF), and tacrolimus as GVHD prophylaxis.\n\nThymoglobulin: Day -9: 0.5 mg/kg Days -8 and -7: 2 mg/kg daily\n\nFludarabine: Days -6 through -2: 30 mg/m\\^2 IV daily\n\nCyclophosphamide: Days -6 and -5: 14.5 mg/kg IV daily Days 3 and 4: 50 mg/kg IV daily\n\nTotal body irradiation: Day -1: 200 centigray (cGy) in a single fraction\n\nTacrolimus: Start on Day 5 through Day 365\n\nMycophenolate mofetil: Days 5 through 35: 15 mg/kg PO three times daily (max 3 g/day)', 'otherNumAtRisk': 21, 'deathsNumAtRisk': 21, 'otherNumAffected': 0, 'seriousNumAtRisk': 21, 'deathsNumAffected': 2, 'seriousNumAffected': 2}], 'seriousEvents': [{'term': 'Gram negative rods Bacteremia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 21, 'numAffected': 2}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT'}], 'frequencyThreshold': '0'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Overall Survival and Engraftment at One Year', 'denoms': [{'units': 'Participants', 'counts': [{'value': '21', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Bone Marrow Transplant', 'description': 'Non-myeloablative bone marrow transplant with a Thymoglobulin (ATG), fludarabine (Flu), cyclophosphamide (Cy), total body irradiation (TBI) preparative regimen and post-transplant Cy, mycophenolate mofetil (MMF), and tacrolimus as GVHD prophylaxis.\n\nThymoglobulin: Day -9: 0.5 mg/kg Days -8 and -7: 2 mg/kg daily\n\nFludarabine: Days -6 through -2: 30 mg/m\\^2 IV daily\n\nCyclophosphamide: Days -6 and -5: 14.5 mg/kg IV daily Days 3 and 4: 50 mg/kg IV daily\n\nTotal body irradiation: Day -1: 200 centigray (cGy) in a single fraction\n\nTacrolimus: Start on Day 5 through Day 365\n\nMycophenolate mofetil: Days 5 through 35: 15 mg/kg PO three times daily (max 3 g/day)'}], 'classes': [{'categories': [{'measurements': [{'value': '19', 'groupId': 'OG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': '1 year', 'description': 'Number of enrolled participants who receive BMT, achieve engraftment, and are alive at one year post bone marrow transplant (BMT).', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Overall Survival at One Year', 'denoms': [{'units': 'Participants', 'counts': [{'value': '21', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Bone Marrow Transplant', 'description': 'Non-myeloablative bone marrow transplant with a Thymoglobulin (ATG), fludarabine (Flu), cyclophosphamide (Cy), total body irradiation (TBI) preparative regimen and post-transplant Cy, mycophenolate mofetil (MMF), and tacrolimus as GVHD prophylaxis.\n\nThymoglobulin: Day -9: 0.5 mg/kg Days -8 and -7: 2 mg/kg daily\n\nFludarabine: Days -6 through -2: 30 mg/m\\^2 IV daily\n\nCyclophosphamide: Days -6 and -5: 14.5 mg/kg IV daily Days 3 and 4: 50 mg/kg IV daily\n\nTotal body irradiation: Day -1: 200 centigray (cGy) in a single fraction\n\nTacrolimus: Start on Day 5 through Day 365\n\nMycophenolate mofetil: Days 5 through 35: 15 mg/kg PO three times daily (max 3 g/day)'}], 'classes': [{'categories': [{'measurements': [{'value': '19', 'groupId': 'OG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': '1 year', 'description': 'Number of participants alive at one year after BMT.', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Probability of Neutrophil Recovery as Assessed by the Number of Participants Who Have Recovered Neutrophil Counts', 'denoms': [{'units': 'Participants', 'counts': [{'value': '21', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Bone Marrow Transplant', 'description': 'Non-myeloablative bone marrow transplant with a Thymoglobulin (ATG), fludarabine (Flu), cyclophosphamide (Cy), total body irradiation (TBI) preparative regimen and post-transplant Cy, mycophenolate mofetil (MMF), and tacrolimus as GVHD prophylaxis.\n\nThymoglobulin: Day -9: 0.5 mg/kg Days -8 and -7: 2 mg/kg daily\n\nFludarabine: Days -6 through -2: 30 mg/m\\^2 IV daily\n\nCyclophosphamide: Days -6 and -5: 14.5 mg/kg IV daily Days 3 and 4: 50 mg/kg IV daily\n\nTotal body irradiation: Day -1: 200 centigray (cGy) in a single fraction\n\nTacrolimus: Start on Day 5 through Day 365\n\nMycophenolate mofetil: Days 5 through 35: 15 mg/kg PO three times daily (max 3 g/day)'}], 'classes': [{'categories': [{'measurements': [{'value': '21', 'groupId': 'OG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': '1 year', 'description': 'Probability of neutrophil recovery will be assessed by the number of participants who have recovered neutrophil counts at 1 year (\\>500 ANC).', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Probability of Platelet Recovery as Assessed by Number of Participants Who Have Recovered Platelet Counts', 'denoms': [{'units': 'Participants', 'counts': [{'value': '21', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Bone Marrow Transplant', 'description': 'Non-myeloablative bone marrow transplant with a Thymoglobulin (ATG), fludarabine (Flu), cyclophosphamide (Cy), total body irradiation (TBI) preparative regimen and post-transplant Cy, mycophenolate mofetil (MMF), and tacrolimus as GVHD prophylaxis.\n\nThymoglobulin: Day -9: 0.5 mg/kg Days -8 and -7: 2 mg/kg daily\n\nFludarabine: Days -6 through -2: 30 mg/m\\^2 IV daily\n\nCyclophosphamide: Days -6 and -5: 14.5 mg/kg IV daily Days 3 and 4: 50 mg/kg IV daily\n\nTotal body irradiation: Day -1: 200 centigray (cGy) in a single fraction\n\nTacrolimus: Start on Day 5 through Day 365\n\nMycophenolate mofetil: Days 5 through 35: 15 mg/kg PO three times daily (max 3 g/day)'}], 'classes': [{'categories': [{'measurements': [{'value': '20', 'groupId': 'OG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': '1 year', 'description': 'Probability of platelet recovery will be assessed by the number of participants who have recovered platelet counts at 1 year.', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Number of Participants Who Experience Primary Graft Failure', 'denoms': [{'units': 'Participants', 'counts': [{'value': '21', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Bone Marrow Transplant', 'description': 'Non-myeloablative bone marrow transplant with a Thymoglobulin (ATG), fludarabine (Flu), cyclophosphamide (Cy), total body irradiation (TBI) preparative regimen and post-transplant Cy, mycophenolate mofetil (MMF), and tacrolimus as GVHD prophylaxis.\n\nThymoglobulin: Day -9: 0.5 mg/kg Days -8 and -7: 2 mg/kg daily\n\nFludarabine: Days -6 through -2: 30 mg/m\\^2 IV daily\n\nCyclophosphamide: Days -6 and -5: 14.5 mg/kg IV daily Days 3 and 4: 50 mg/kg IV daily\n\nTotal body irradiation: Day -1: 200 centigray (cGy) in a single fraction\n\nTacrolimus: Start on Day 5 through Day 365\n\nMycophenolate mofetil: Days 5 through 35: 15 mg/kg PO three times daily (max 3 g/day)'}], 'classes': [{'categories': [{'measurements': [{'value': '21', 'groupId': 'OG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': '1 year', 'description': 'Number of participants who experience primary graft failure by one year after BMT.', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Number of Participants Who Experience Secondary Graft Failure', 'denoms': [{'units': 'Participants', 'counts': [{'value': '21', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Bone Marrow Transplant', 'description': 'Non-myeloablative bone marrow transplant with a Thymoglobulin (ATG), fludarabine (Flu), cyclophosphamide (Cy), total body irradiation (TBI) preparative regimen and post-transplant Cy, mycophenolate mofetil (MMF), and tacrolimus as GVHD prophylaxis.\n\nThymoglobulin: Day -9: 0.5 mg/kg Days -8 and -7: 2 mg/kg daily\n\nFludarabine: Days -6 through -2: 30 mg/m\\^2 IV daily\n\nCyclophosphamide: Days -6 and -5: 14.5 mg/kg IV daily Days 3 and 4: 50 mg/kg IV daily\n\nTotal body irradiation: Day -1: 200 centigray (cGy) in a single fraction\n\nTacrolimus: Start on Day 5 through Day 365\n\nMycophenolate mofetil: Days 5 through 35: 15 mg/kg PO three times daily (max 3 g/day)'}], 'classes': [{'categories': [{'measurements': [{'value': '19', 'groupId': 'OG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': '1 year', 'description': 'Number of participants who experience secondary graft failure by one year after BMT.', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Number of Participants Who Experience Grades II-IV Acute GVHD', 'denoms': [{'units': 'Participants', 'counts': [{'value': '21', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Bone Marrow Transplant', 'description': 'Non-myeloablative bone marrow transplant with a Thymoglobulin (ATG), fludarabine (Flu), cyclophosphamide (Cy), total body irradiation (TBI) preparative regimen and post-transplant Cy, mycophenolate mofetil (MMF), and tacrolimus as GVHD prophylaxis.\n\nThymoglobulin: Day -9: 0.5 mg/kg Days -8 and -7: 2 mg/kg daily\n\nFludarabine: Days -6 through -2: 30 mg/m\\^2 IV daily\n\nCyclophosphamide: Days -6 and -5: 14.5 mg/kg IV daily Days 3 and 4: 50 mg/kg IV daily\n\nTotal body irradiation: Day -1: 200 centigray (cGy) in a single fraction\n\nTacrolimus: Start on Day 5 through Day 365\n\nMycophenolate mofetil: Days 5 through 35: 15 mg/kg PO three times daily (max 3 g/day)'}], 'classes': [{'categories': [{'measurements': [{'value': '4', 'groupId': 'OG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'Day 100', 'description': 'Number of participants who experience grade II, III, or IV acute GVHD by Day 100.', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Number of Participants Who Experience Grades III-IV Acute GVHD', 'denoms': [{'units': 'Participants', 'counts': [{'value': '21', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Bone Marrow Transplant', 'description': 'Non-myeloablative bone marrow transplant with a Thymoglobulin (ATG), fludarabine (Flu), cyclophosphamide (Cy), total body irradiation (TBI) preparative regimen and post-transplant Cy, mycophenolate mofetil (MMF), and tacrolimus as GVHD prophylaxis.\n\nThymoglobulin: Day -9: 0.5 mg/kg Days -8 and -7: 2 mg/kg daily\n\nFludarabine: Days -6 through -2: 30 mg/m\\^2 IV daily\n\nCyclophosphamide: Days -6 and -5: 14.5 mg/kg IV daily Days 3 and 4: 50 mg/kg IV daily\n\nTotal body irradiation: Day -1: 200 centigray (cGy) in a single fraction\n\nTacrolimus: Start on Day 5 through Day 365\n\nMycophenolate mofetil: Days 5 through 35: 15 mg/kg PO three times daily (max 3 g/day)'}], 'classes': [{'categories': [{'measurements': [{'value': '2', 'groupId': 'OG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'Day 100', 'description': 'Number of participants who experience grade III or IV acute GVHD by Day 100.', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Number of Participants Who Experience Chronic GVHD', 'denoms': [{'units': 'Participants', 'counts': [{'value': '21', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Bone Marrow Transplant', 'description': 'Non-myeloablative bone marrow transplant with a Thymoglobulin (ATG), fludarabine (Flu), cyclophosphamide (Cy), total body irradiation (TBI) preparative regimen and post-transplant Cy, mycophenolate mofetil (MMF), and tacrolimus as GVHD prophylaxis.\n\nThymoglobulin: Day -9: 0.5 mg/kg Days -8 and -7: 2 mg/kg daily\n\nFludarabine: Days -6 through -2: 30 mg/m\\^2 IV daily\n\nCyclophosphamide: Days -6 and -5: 14.5 mg/kg IV daily Days 3 and 4: 50 mg/kg IV daily\n\nTotal body irradiation: Day -1: 200 centigray (cGy) in a single fraction\n\nTacrolimus: Start on Day 5 through Day 365\n\nMycophenolate mofetil: Days 5 through 35: 15 mg/kg PO three times daily (max 3 g/day)'}], 'classes': [{'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': '2 years', 'description': 'Number of participants who experience chronic GVHD by two years after BMT.', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Number of Participants With Full Donor Chimerism', 'denoms': [{'units': 'Participants', 'counts': [{'value': '21', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Bone Marrow Transplant', 'description': 'Non-myeloablative bone marrow transplant with a Thymoglobulin (ATG), fludarabine (Flu), cyclophosphamide (Cy), total body irradiation (TBI) preparative regimen and post-transplant Cy, mycophenolate mofetil (MMF), and tacrolimus as GVHD prophylaxis.\n\nThymoglobulin: Day -9: 0.5 mg/kg Days -8 and -7: 2 mg/kg daily\n\nFludarabine: Days -6 through -2: 30 mg/m\\^2 IV daily\n\nCyclophosphamide: Days -6 and -5: 14.5 mg/kg IV daily Days 3 and 4: 50 mg/kg IV daily\n\nTotal body irradiation: Day -1: 200 centigray (cGy) in a single fraction\n\nTacrolimus: Start on Day 5 through Day 365\n\nMycophenolate mofetil: Days 5 through 35: 15 mg/kg PO three times daily (max 3 g/day)'}], 'classes': [{'categories': [{'measurements': [{'value': '21', 'groupId': 'OG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'Day 60', 'description': 'Number of participants with full donor chimerism at Day 60.', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'GVHD-free Relapse-free Survival (GRFS)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '21', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Bone Marrow Transplant', 'description': 'Non-myeloablative bone marrow transplant with a Thymoglobulin (ATG), fludarabine (Flu), cyclophosphamide (Cy), total body irradiation (TBI) preparative regimen and post-transplant Cy, mycophenolate mofetil (MMF), and tacrolimus as GVHD prophylaxis.\n\nThymoglobulin: Day -9: 0.5 mg/kg Days -8 and -7: 2 mg/kg daily\n\nFludarabine: Days -6 through -2: 30 mg/m\\^2 IV daily\n\nCyclophosphamide: Days -6 and -5: 14.5 mg/kg IV daily Days 3 and 4: 50 mg/kg IV daily\n\nTotal body irradiation: Day -1: 200 centigray (cGy) in a single fraction\n\nTacrolimus: Start on Day 5 through Day 365\n\nMycophenolate mofetil: Days 5 through 35: 15 mg/kg PO three times daily (max 3 g/day)'}], 'classes': [{'categories': [{'measurements': [{'value': '19', 'groupId': 'OG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': '1 year', 'description': 'Number of participants alive, without relapse, and without GVHD at 1 year.', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Transplant-related Mortality', 'denoms': [{'units': 'Participants', 'counts': [{'value': '21', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Bone Marrow Transplant', 'description': 'Non-myeloablative bone marrow transplant with a Thymoglobulin (ATG), fludarabine (Flu), cyclophosphamide (Cy), total body irradiation (TBI) preparative regimen and post-transplant Cy, mycophenolate mofetil (MMF), and tacrolimus as GVHD prophylaxis.\n\nThymoglobulin: Day -9: 0.5 mg/kg Days -8 and -7: 2 mg/kg daily\n\nFludarabine: Days -6 through -2: 30 mg/m\\^2 IV daily\n\nCyclophosphamide: Days -6 and -5: 14.5 mg/kg IV daily Days 3 and 4: 50 mg/kg IV daily\n\nTotal body irradiation: Day -1: 200 centigray (cGy) in a single fraction\n\nTacrolimus: Start on Day 5 through Day 365\n\nMycophenolate mofetil: Days 5 through 35: 15 mg/kg PO three times daily (max 3 g/day)'}], 'classes': [{'categories': [{'measurements': [{'value': '2', 'groupId': 'OG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': '1 year', 'description': 'Number of participants deceased for reasons related to BMT at 1 year.', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'Bone Marrow Transplant', 'description': 'Non-myeloablative bone marrow transplant with a Thymoglobulin (ATG), fludarabine (Flu), cyclophosphamide (Cy), total body irradiation (TBI) preparative regimen and post-transplant Cy, mycophenolate mofetil (MMF), and tacrolimus as graft-versus-host disease (GVHD) prophylaxis.\n\nThymoglobulin: Day -9: 0.5 mg/kg Days -8 and -7: 2 mg/kg daily\n\nFludarabine: Days -6 through -2: 30 mg/m\\^2 IV daily\n\nCyclophosphamide: Days -6 and -5: 14.5 mg/kg IV daily Days 3 and 4: 50 mg/kg IV daily\n\nTotal body irradiation: Day -1: 200 centigray (cGy) in a single fraction\n\nTacrolimus: Start on Day 5 through Day 365\n\nMycophenolate mofetil: Days 5 through 35: 15 mg/kg PO three times daily (max 3 g/day)'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '21'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '21'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}]}]}]}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '21', 'groupId': 'BG000'}]}], 'groups': [{'id': 'BG000', 'title': 'Bone Marrow Transplant', 'description': 'Non-myeloablative bone marrow transplant with a Thymoglobulin (ATG), fludarabine (Flu), cyclophosphamide (Cy), total body irradiation (TBI) preparative regimen and post-transplant Cy, mycophenolate mofetil (MMF), and tacrolimus as GVHD prophylaxis.\n\nThymoglobulin: Day -9: 0.5 mg/kg Days -8 and -7: 2 mg/kg daily\n\nFludarabine: Days -6 through -2: 30 mg/m\\^2 IV daily\n\nCyclophosphamide: Days -6 and -5: 14.5 mg/kg IV daily Days 3 and 4: 50 mg/kg IV daily\n\nTotal body irradiation: Day -1: 200 centigray (cGy) in a single fraction\n\nTacrolimus: Start on Day 5 through Day 365\n\nMycophenolate mofetil: Days 5 through 35: 15 mg/kg PO three times daily (max 3 g/day)'}], 'measures': [{'title': 'Age, Categorical', 'classes': [{'categories': [{'title': '<=18 years', 'measurements': [{'value': '7', 'groupId': 'BG000'}]}, {'title': 'Between 18 and 65 years', 'measurements': [{'value': '14', 'groupId': 'BG000'}]}, {'title': '>=65 years', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Age, Continuous', 'classes': [{'categories': [{'measurements': [{'value': '25', 'groupId': 'BG000', 'lowerLimit': '3', 'upperLimit': '69'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'years', 'dispersionType': 'FULL_RANGE'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '8', 'groupId': 'BG000'}]}, {'title': 'Male', 'measurements': [{'value': '13', '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': '1', 'groupId': 'BG000'}]}, {'title': 'Native Hawaiian or Other Pacific Islander', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Black or African American', 'measurements': [{'value': '4', 'groupId': 'BG000'}]}, {'title': 'White', 'measurements': [{'value': '14', 'groupId': 'BG000'}]}, {'title': 'More than one race', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Unknown or Not Reported', 'measurements': [{'value': '2', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Race/Ethnicity, Customized', 'classes': [{'title': 'African American', 'categories': [{'measurements': [{'value': '4', 'groupId': 'BG000'}]}]}, {'title': 'Caucasian', 'categories': [{'measurements': [{'value': '14', 'groupId': 'BG000'}]}]}, {'title': 'Other', 'categories': [{'measurements': [{'value': '3', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Region of Enrollment', 'classes': [{'title': 'United States', 'categories': [{'measurements': [{'value': '21', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}]}}, 'documentSection': {'largeDocumentModule': {'largeDocs': [{'date': '2020-08-24', 'size': 861658, 'label': 'Study Protocol and Statistical Analysis Plan', 'hasIcf': False, 'hasSap': True, 'filename': 'Prot_SAP_000.pdf', 'typeAbbrev': 'Prot_SAP', 'uploadDate': '2022-04-26T15:06', 'hasProtocol': True}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 21}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2016-09', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2022-06', 'completionDateStruct': {'date': '2021-07', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2022-06-16', 'studyFirstSubmitDate': '2016-07-12', 'resultsFirstSubmitDate': '2022-05-05', 'studyFirstSubmitQcDate': '2016-07-13', 'lastUpdatePostDateStruct': {'date': '2022-07-12', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2022-06-16', 'studyFirstPostDateStruct': {'date': '2016-07-14', 'type': 'ESTIMATED'}, 'resultsFirstPostDateStruct': {'date': '2022-07-12', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2021-07', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Overall Survival and Engraftment at One Year', 'timeFrame': '1 year', 'description': 'Number of enrolled participants who receive BMT, achieve engraftment, and are alive at one year post bone marrow transplant (BMT).'}], 'secondaryOutcomes': [{'measure': 'Overall Survival at One Year', 'timeFrame': '1 year', 'description': 'Number of participants alive at one year after BMT.'}, {'measure': 'Probability of Neutrophil Recovery as Assessed by the Number of Participants Who Have Recovered Neutrophil Counts', 'timeFrame': '1 year', 'description': 'Probability of neutrophil recovery will be assessed by the number of participants who have recovered neutrophil counts at 1 year (\\>500 ANC).'}, {'measure': 'Probability of Platelet Recovery as Assessed by Number of Participants Who Have Recovered Platelet Counts', 'timeFrame': '1 year', 'description': 'Probability of platelet recovery will be assessed by the number of participants who have recovered platelet counts at 1 year.'}, {'measure': 'Number of Participants Who Experience Primary Graft Failure', 'timeFrame': '1 year', 'description': 'Number of participants who experience primary graft failure by one year after BMT.'}, {'measure': 'Number of Participants Who Experience Secondary Graft Failure', 'timeFrame': '1 year', 'description': 'Number of participants who experience secondary graft failure by one year after BMT.'}, {'measure': 'Number of Participants Who Experience Grades II-IV Acute GVHD', 'timeFrame': 'Day 100', 'description': 'Number of participants who experience grade II, III, or IV acute GVHD by Day 100.'}, {'measure': 'Number of Participants Who Experience Grades III-IV Acute GVHD', 'timeFrame': 'Day 100', 'description': 'Number of participants who experience grade III or IV acute GVHD by Day 100.'}, {'measure': 'Number of Participants Who Experience Chronic GVHD', 'timeFrame': '2 years', 'description': 'Number of participants who experience chronic GVHD by two years after BMT.'}, {'measure': 'Number of Participants With Full Donor Chimerism', 'timeFrame': 'Day 60', 'description': 'Number of participants with full donor chimerism at Day 60.'}, {'measure': 'GVHD-free Relapse-free Survival (GRFS)', 'timeFrame': '1 year', 'description': 'Number of participants alive, without relapse, and without GVHD at 1 year.'}, {'measure': 'Transplant-related Mortality', 'timeFrame': '1 year', 'description': 'Number of participants deceased for reasons related to BMT at 1 year.'}]}, 'oversightModule': {'oversightHasDmc': False}, 'conditionsModule': {'keywords': ['immunosuppression', 'nonmyeloablative', 'non-myeloablative', 'allogeneic', 'tacrolimus', 'bone marrow', 'bone marrow transplant', 'cyclophosphamide', 'thymoglobulin', 'ATG', 'fludarabine'], 'conditions': ['Severe Aplastic Anemia', 'Aplastic Anemia', 'Bone Marrow Failure', 'Immunosuppression']}, 'referencesModule': {'references': [{'pmid': '37084383', 'type': 'DERIVED', 'citation': 'DeZern AE, Zahurak M, Symons HJ, Cooke KR, Huff CA, Jain T, Swinnen LJ, Imus PH, Wagner-Johnston ND, Ambinder RF, Levis M, Luznik L, Bolanos-Meade J, Fuchs EJ, Jones RJ, Brodsky RA. Alternative donor BMT with posttransplant cyclophosphamide as initial therapy for acquired severe aplastic anemia. Blood. 2023 Jun 22;141(25):3031-3038. doi: 10.1182/blood.2023020435.'}, {'pmid': '32343796', 'type': 'DERIVED', 'citation': 'DeZern AE, Zahurak ML, Symons HJ, Cooke KR, Rosner GL, Gladstone DE, Huff CA, Swinnen LJ, Imus P, Borrello I, Wagner-Johnston N, Ambinder RF, Luznik L, Bolanos-Meade J, Fuchs EJ, Jones RJ, Brodsky RA. Haploidentical BMT for severe aplastic anemia with intensive GVHD prophylaxis including posttransplant cyclophosphamide. Blood Adv. 2020 Apr 28;4(8):1770-1779. doi: 10.1182/bloodadvances.2020001729.'}]}, 'descriptionModule': {'briefSummary': 'Our primary objective is to determine if it is feasible for previously untreated severe aplastic anemia (SAA) patients to be transplanted using non-myeloablative conditioning and post transplantation cyclophosphamide.', 'detailedDescription': 'This is a clinical trial of upfront bone marrow transplantation for patients with SAA who do not have a fully human leukocyte antigen (HLA) matched donor. The trial uses a conditioning regimen which has been successful in the refractory and relapsed setting to maximize engraftment and post transplant therapy to minimize graft versus host disease (GVHD). This would be used here in patients who have not yet undergone immunosuppressive therapy for their SAA or are thought to be unlikely to respond to immunosuppressive therapy for SAA.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'healthyVolunteers': False, 'eligibilityCriteria': "Inclusion Criteria:\n\n* Confirmed diagnosis of inherited or acquired severe aplastic anemia (SAA)\n* One of the following available donors:\n\n 1. HLA-haploidentical relative\n 2. If recipient is \\>= 40 years old, may use HLA-matched related donor\n 3. For recipients with inherited bone marrow failure syndromes (IBMFS) with clear evidence of same disorder in potential related donors, may use 10/10 matched unrelated donor\n* Recipient and/or legal guardian must sign protocol informed consent\n* Donor must be willing to donate bone marrow\n* Left ventricular ejection fraction (LVEF) \\>= 40%. For recipients \\< 13 years old, shortening fraction \\>= 26% may be used instead.\n* Bilirubin \\< 3 x upper limit of normal (ULN) for age, unless patient has Gilbert's disease\n* aspartate aminotransferase (AST) and alanine aminotransferase (ALT) \\< 5 x ULN for age\n* For patients \\>= 13 years old: estimated creatinine clearance \\> 50 mL/min using Cockcroft-Gault formula and actual body weight\n* For patients \\>= 1 but \\< 13 years old: glomerular filtration rate (GFR) estimated by updated Schwartz formula \\>= 90 mL/min/1.73 m\\^2. If estimated GFR is \\< 90 mL/min/1.73 m\\^2, 24-hour measured creatinine clearance must be \\> 50 mL/min/1.73 m\\^2.\n* For patients \\>= 8 years old, diffusing capacity of the lung for carbon monoxide (DLCO) (corrected for hemoglobin) \\> 40%; forced expiratory volume at one second (FEV1) \\> 50%; forced vital capacity (FVC) \\> 50%\n* For patients \\< 8 years old or unable to undergo pulmonary function testing: no evidence of dyspnea at rest; no need for supplemental oxygen; oxygen saturation \\> 92% on room air\n* Karnofsky/Lansky status (depending on age) \\>= 70%\n* Females and males of childbearing potential must agree to practice 2 effective methods of contraception at the same time. If unwilling, they must agree to complete abstinence.\n\nExclusion Criteria:\n\n* Previous administration of immunosuppressive therapy for SAA.\n* Fanconi anemia. At minimum, this diagnosis must be excluded by diepoxybutane (DEB) or equivalent testing on peripheral blood or marrow in patients \\< 30 years old.\n* Clonal cytogenetic abnormalities consistent with pre-myelodysplastic syndrome (pre-MDS) or MDS on bone marrow examination\n* Presence of anti-donor antibodies\n* Prior allogeneic stem cell transplant\n* Prior solid organ transplant\n* Uncontrolled bacterial, viral, or fungal infection\n* HIV seropositivity\n* Active hepatitis B or C infection determined by serology and/or nucleic acid testing (NAT)\n* Pregnancy or active breastfeeding\n* Prior malignancies except: resected basal carcinoma or treated cervical carcinoma in situ; cancer treated with curative intent \\> 5 years previously. Other prior cancers will not be allowed unless approved by the PI."}, 'identificationModule': {'nctId': 'NCT02833805', 'briefTitle': 'NMA Haplo or MUD BMT for Newly Diagnosed Severe Aplastic Anemia', 'organization': {'class': 'OTHER', 'fullName': 'Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins'}, 'officialTitle': 'A Phase II Trial of Non-Myeloablative (NMA) Conditioning and Transplantation of Partially HLA-Mismatched/Haploidentical Related or Matched Unrelated Donor (MUD) Bone Marrow for Newly Diagnosed Patients With Severe Aplastic Anemia', 'orgStudyIdInfo': {'id': 'J1688'}, 'secondaryIdInfos': [{'id': 'IRB00107139', 'type': 'OTHER', 'domain': 'JHMIRB'}]}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Bone marrow transplant', 'description': 'Non-myeloablative bone marrow transplant with a Thymoglobulin (ATG), fludarabine (Flu), cyclophosphamide (Cy), total body irradiation (TBI) preparative regimen and post-transplant Cy, mycophenolate mofetil (MMF), and tacrolimus as GVHD prophylaxis.', 'interventionNames': ['Drug: Thymoglobulin', 'Drug: Fludarabine', 'Drug: Cyclophosphamide', 'Radiation: Total body irradiation', 'Drug: Tacrolimus', 'Drug: Mycophenolate mofetil']}], 'interventions': [{'name': 'Thymoglobulin', 'type': 'DRUG', 'otherNames': ['Anti-thymocyte globulin', 'ATG'], 'description': 'Day -9: 0.5 mg/kg Days -8 and -7: 2 mg/kg daily', 'armGroupLabels': ['Bone marrow transplant']}, {'name': 'Fludarabine', 'type': 'DRUG', 'otherNames': ['Fludara'], 'description': 'Days -6 through -2: 30 mg/m\\^2 IV daily', 'armGroupLabels': ['Bone marrow transplant']}, {'name': 'Cyclophosphamide', 'type': 'DRUG', 'otherNames': ['Cytoxan', 'Cy', 'CTX'], 'description': 'Days -6 and -5: 14.5 mg/kg IV daily Days 3 and 4: 50 mg/kg IV daily', 'armGroupLabels': ['Bone marrow transplant']}, {'name': 'Total body irradiation', 'type': 'RADIATION', 'otherNames': ['TBI'], 'description': 'Day -1: 200 centigray (cGy) in a single fraction', 'armGroupLabels': ['Bone marrow transplant']}, {'name': 'Tacrolimus', 'type': 'DRUG', 'otherNames': ['FK-506', 'FK506', 'Prograf'], 'description': 'Start on Day 5 through Day 365', 'armGroupLabels': ['Bone marrow transplant']}, {'name': 'Mycophenolate mofetil', 'type': 'DRUG', 'otherNames': ['MMF', 'CellCept'], 'description': 'Days 5 through 35: 15 mg/kg PO three times daily (max 3 g/day)', 'armGroupLabels': ['Bone marrow transplant']}]}, 'contactsLocationsModule': {'locations': [{'zip': '21287', 'city': 'Baltimore', 'state': 'Maryland', 'country': 'United States', 'facility': 'Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins', 'geoPoint': {'lat': 39.29038, 'lon': -76.61219}}], 'overallOfficials': [{'name': 'Amy E DeZern, MD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Johns Hopkins University'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins', 'class': 'OTHER'}, 'responsibleParty': {'type': 'SPONSOR'}}}}