Ignite Creation Date:
2025-12-25 @ 2:28 AM
Ignite Modification Date:
2025-12-26 @ 1:03 AM
Study NCT ID:
NCT00301834
Status:
COMPLETED
Last Update Posted:
2017-09-28
First Post:
2006-03-09
Is NOT Gene Therapy:
False
Has Adverse Events:
True
Brief Title:
Alemtuzumab, Fludarabine, and Busulfan Followed By Donor Stem Cell Transplant in Treating Young Patients With Hematologic Disorders
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'C535982', 'term': 'Congenital amegakaryocytic thrombocytopenia'}, {'id': 'D029503', 'term': 'Anemia, Diamond-Blackfan'}, {'id': 'D007938', 'term': 'Leukemia'}, {'id': 'D009190', 'term': 'Myelodysplastic Syndromes'}, {'id': 'C537592', 'term': 'Neutropenia, Severe Congenital, Autosomal Recessive 3'}, {'id': 'D015466', 'term': 'Leukemia, Myeloid, Chronic-Phase'}], 'ancestors': [{'id': 'D029502', 'term': 'Anemia, Hypoplastic, Congenital'}, {'id': 'D000741', 'term': 'Anemia, Aplastic'}, {'id': 'D000740', 'term': 'Anemia'}, {'id': 'D006402', 'term': 'Hematologic Diseases'}, {'id': 'D006425', 'term': 'Hemic and Lymphatic Diseases'}, {'id': 'D012010', 'term': 'Red-Cell Aplasia, Pure'}, {'id': 'D000080984', 'term': 'Congenital Bone Marrow Failure Syndromes'}, {'id': 'D000080983', 'term': 'Bone Marrow Failure Disorders'}, {'id': 'D001855', 'term': 'Bone Marrow Diseases'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D009370', 'term': 'Neoplasms by Histologic Type'}, {'id': 'D009369', 'term': 'Neoplasms'}, {'id': 'D015464', 'term': 'Leukemia, Myelogenous, Chronic, BCR-ABL Positive'}, {'id': 'D007951', 'term': 'Leukemia, Myeloid'}, {'id': 'D009196', 'term': 'Myeloproliferative Disorders'}, {'id': 'D002908', 'term': 'Chronic Disease'}, {'id': 'D020969', 'term': 'Disease Attributes'}, {'id': 'D010335', 'term': 'Pathologic Processes'}, {'id': 'D013568', 'term': 'Pathological Conditions, Signs and Symptoms'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'D000074323', 'term': 'Alemtuzumab'}, {'id': 'D002066', 'term': 'Busulfan'}, {'id': 'D016572', 'term': 'Cyclosporine'}, {'id': 'C042382', 'term': 'fludarabine phosphate'}, {'id': 'D008727', 'term': 'Methotrexate'}, {'id': 'D008775', 'term': 'Methylprednisolone'}, {'id': 'D036102', 'term': 'Peripheral Blood Stem Cell Transplantation'}, {'id': 'D036101', 'term': 'Cord Blood Stem Cell Transplantation'}], 'ancestors': [{'id': 'D061067', 'term': 'Antibodies, Monoclonal, Humanized'}, {'id': 'D000911', 'term': 'Antibodies, Monoclonal'}, {'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': 'D002072', 'term': 'Butylene Glycols'}, {'id': 'D006018', 'term': 'Glycols'}, {'id': 'D000438', 'term': 'Alcohols'}, {'id': 'D009930', 'term': 'Organic Chemicals'}, {'id': 'D008698', 'term': 'Mesylates'}, {'id': 'D000476', 'term': 'Alkanesulfonates'}, {'id': 'D017738', 'term': 'Alkanesulfonic Acids'}, {'id': 'D000473', 'term': 'Alkanes'}, {'id': 'D006839', 'term': 'Hydrocarbons, Acyclic'}, {'id': 'D006838', 'term': 'Hydrocarbons'}, {'id': 'D013451', 'term': 'Sulfonic Acids'}, {'id': 'D013456', 'term': 'Sulfur Acids'}, {'id': 'D013457', 'term': 'Sulfur Compounds'}, {'id': 'D003524', 'term': 'Cyclosporins'}, {'id': 'D010456', 'term': 'Peptides, Cyclic'}, {'id': 'D047028', 'term': 'Macrocyclic Compounds'}, {'id': 'D011083', 'term': 'Polycyclic Compounds'}, {'id': 'D010455', 'term': 'Peptides'}, {'id': 'D000630', 'term': 'Aminopterin'}, {'id': 'D011622', 'term': 'Pterins'}, {'id': 'D011621', 'term': 'Pteridines'}, {'id': 'D006574', 'term': 'Heterocyclic Compounds, 2-Ring'}, {'id': 'D000072471', 'term': 'Heterocyclic Compounds, Fused-Ring'}, {'id': 'D006571', 'term': 'Heterocyclic Compounds'}, {'id': 'D011239', 'term': 'Prednisolone'}, {'id': 'D011246', 'term': 'Pregnadienetriols'}, {'id': 'D011245', 'term': 'Pregnadienes'}, {'id': 'D011278', 'term': 'Pregnanes'}, {'id': 'D013256', 'term': 'Steroids'}, {'id': 'D000072473', 'term': 'Fused-Ring Compounds'}, {'id': 'D018380', 'term': 'Hematopoietic Stem Cell Transplantation'}, {'id': 'D033581', 'term': 'Stem Cell Transplantation'}, {'id': 'D017690', 'term': 'Cell Transplantation'}, {'id': 'D064987', 'term': 'Cell- and Tissue-Based Therapy'}, {'id': 'D001691', 'term': 'Biological Therapy'}, {'id': 'D013812', 'term': 'Therapeutics'}, {'id': 'D014180', 'term': 'Transplantation'}, {'id': 'D013514', 'term': 'Surgical Procedures, Operative'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'mcowan@peds.ucsf.edu', 'phone': '4154762188', 'title': 'Dr. Morton Cowan', 'organization': 'University of California San Francisco'}, 'certainAgreement': {'piSponsorEmployee': True}}, 'adverseEventsModule': {'eventGroups': [{'id': 'EG000', 'title': 'Single Arm - Transplant Pre-conditioning Per Study Protocol', 'description': "All eligible patients received pre-transplant conditioning with Alemtuzumab, fludarabine and targeted busulfan followed by allogeneic transplant. The initial dose of busulfan was determined by performing PK (pharmacokinetics) analysis on a test dose of busulfan on the day prior to initiating conditioning. Based on the PK results a starting dose of busulfan was determined. A second PK study was done after the starting dose to ensure that the targeted dose was achieved. If it wasn't achieved, then a dose modification was made and a third PK study done.", 'otherNumAtRisk': 35, 'otherNumAffected': 33, 'seriousNumAtRisk': 35, 'seriousNumAffected': 7}], 'otherEvents': [{'term': 'Mucositis (All grades)', 'notes': '\\< Grade III - 17 participants; Grade III - 15 participants; Grade IV - 1 participant', 'stats': [{'groupId': 'EG000', 'numAtRisk': 35, 'numEvents': 33, 'numAffected': 33}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'aGVHD', 'notes': 'aGVHD \\< Grade IV', 'stats': [{'groupId': 'EG000', 'numAtRisk': 35, 'numEvents': 5, 'numAffected': 5}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'cGVHD (Chronic graft-versus-host disease)', 'stats': [{'groupId': 'EG000', 'numAtRisk': 35, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Infusion reaction to alemtuzumab', 'stats': [{'groupId': 'EG000', 'numAtRisk': 35, 'numEvents': 19, 'numAffected': 19}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}], 'seriousEvents': [{'term': 'aGVHD (acute Graft-Versus-Host Disease)', 'notes': 'acute Graft-Versus-Host Disease', 'stats': [{'groupId': 'EG000', 'numAtRisk': 35, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Respiratory distress associated with Cytomegalovirus infection', 'notes': 'After 4th unrelated donor transplant (off study), patient developed respiratory distress that was associated with a CMV infection. Respiratory distress worsened in spite of maximal therapy. The parents elected to withdraw therapy and he died.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 35, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT'}, {'term': 'Pancytopenia and splenomegaly', 'notes': 'Child presented with fever and pancytopenia, splenomegaly and was admitted for possible recurrent disease.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 35, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT'}, {'term': 'Repiratory failure following ideopathic pneumonia syndrome', 'notes': "Worsening respiratory status despite maximal support, at parent's request, patient was removed from mechanical ventilation, and the patient died quickly and peacefully.", 'stats': [{'groupId': 'EG000', 'numAtRisk': 35, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT'}, {'term': 'Thrombocytopenia', 'notes': 'Sudden onset of thrombocytopenia of unknown etiology.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 35, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT'}, {'term': 'Aspergillus pneumonia', 'notes': 'Patient admitted with cough and fever and diagnosed with Aspergillus pneumonia.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 35, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT'}, {'term': 'Renal Failure', 'notes': 'Patient developed renal failure and is required dialysis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 35, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Renal and urinary disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT'}, {'term': 'Graft rejection/failure', 'notes': 'initially, cells recovered normally with neutrophil count \\>500 by day +12 post-transplant. By day +20 they began a slow decline and by day +40 the neutrophil count was \\<500 with 99% host CD3+ (cluster of differentiation 3-positive) cells.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 35, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Viral Encephalitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 35, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT'}, {'term': 'Massive intracranial bleed', 'notes': "Child died from a massive intracranial bleed, probably due to Evan's syndrome and myeloablation; possible viral encephalitis possibly increased risk of intracranial hemorrhage.", 'stats': [{'groupId': 'EG000', 'numAtRisk': 35, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Vascular disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT'}], 'frequencyThreshold': '5'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Number of Participants Achieving Durable Engraftment (Presence of Donor Cells) at 6 Weeks Post Transplantation', 'denoms': [{'units': 'Participants', 'counts': [{'value': '34', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Single Arm - Transplant Pre-conditioning Per Study Protocol', 'description': 'Conditioning with Alemtuzumab, busulfan and fludarabine followed by allogeneic hematopoietic cell transplant.'}], 'classes': [{'categories': [{'measurements': [{'value': '31', 'groupId': 'OG000'}]}]}], 'paramType': 'NUMBER', 'timeFrame': '6 weeks post-transplant', 'description': 'Peripheral blood chimerism studies were performed by quantitative real time polymerase chain reaction (qPCR) evaluation of differential short tandem repeat DNA sequences', 'unitOfMeasure': 'participants', 'reportingStatus': 'POSTED', 'populationDescription': 'Participants evaluable for engraftment 6 weeks post-transplant; 1 patient died of transplant-related hemorrhage prior to 6 weeks and was not evaluated for this outcome'}, {'type': 'SECONDARY', 'title': 'Treatment-related Mortality at 100 Days and 1 Year Post Transplantation', 'denoms': [{'units': 'Participants', 'counts': [{'value': '35', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Single Arm - Transplant Pre-conditioning Per Study Protocol', 'description': 'Conditioning with Alemtuzumab, busulfan and fludarabine followed by allogeneic hematopoietic cell transplant.'}], 'classes': [{'title': 'Transplantation-related mortality 0-100 days', 'categories': [{'measurements': [{'value': '2', 'groupId': 'OG000'}]}]}, {'title': 'Transplantation-related mortality 100-365 days', 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}]}]}], 'paramType': 'NUMBER', 'timeFrame': '100 days and 1 year', 'unitOfMeasure': 'participants', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Toxicity Grade ≥ 3 From Start of Conditioning Through the First Year Post Transplantation', 'denoms': [{'units': 'Participants', 'counts': [{'value': '35', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Single Arm', 'description': 'Conditioning with Alemtuzumab, busulfan and fludarabine followed by allogeneic hematopoietic cell transplant.'}], 'classes': [{'title': 'Grade 3-4 acute Graft-Versus-Host Disease', 'categories': [{'measurements': [{'value': '2', 'groupId': 'OG000'}]}]}, {'title': 'Grade 3-4 mucositis', 'categories': [{'measurements': [{'value': '16', 'groupId': 'OG000'}]}]}], 'paramType': 'NUMBER', 'timeFrame': '1 year post-transplantation', 'unitOfMeasure': 'participants', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Cytomegalovirus (CMV) Viral Infection and Disease Symptoms', 'denoms': [{'units': 'Participants', 'counts': [{'value': '14', 'groupId': 'OG000'}, {'value': '17', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'CMV Seronegative Participants', 'description': '"seronegativity" means no detected presence of anti-CMV IgG, indicating no past infection by the virus'}, {'id': 'OG001', 'title': 'CMV Seropositive Participants', 'description': '"seropositivity" means the presence of anti-CMV IgG, indicating past infection by the virus'}], 'classes': [{'title': 'Positive CMV Viral Load Assay', 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}, {'value': '12', 'groupId': 'OG001'}]}]}, {'title': 'Symptomatic CMV disease', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'Up to one year post-transplant', 'description': 'polymerase chain reaction testing for presence of CMV weekly until at least day +100 then every 2 weeks until T-cell reconstitution as defined by cluster of differentiation 4 (CD4) \\> 200 cells/mm3. Median time to T-cell reconstitution was 6 months.', 'unitOfMeasure': 'participants', 'reportingStatus': 'POSTED', 'populationDescription': '4 participants were incapable of producing Ab or CMV testing result was indeterminate'}, {'type': 'SECONDARY', 'title': 'Disease-free Survival With Correction of Disease at One Year Post Transplantation', 'denoms': [{'units': 'Participants', 'counts': [{'value': '35', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Single Arm - Transplant Pre-conditioning Per Study Protocol', 'description': 'Conditioning with Alemtuzumab, busulfan and fludarabine followed by allogeneic hematopoietic cell transplant.'}], 'classes': [{'categories': [{'measurements': [{'value': '22', 'groupId': 'OG000'}]}]}], 'paramType': 'NUMBER', 'timeFrame': '1 year post-transplantation', 'description': 'Patients deemed "alive and well" at follow-up timepoint later than 1-year post-transplantation', 'unitOfMeasure': 'participants', 'reportingStatus': 'POSTED'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'Single Arm - Transplant Pre-conditioning Per Study Protocol', 'description': "All eligible patients received pre-transplant conditioning with Alemtuzumab, fludarabine and targeted busulfan followed by allogeneic transplant. The initial dose of busulfan was determined by performing PK (pharmacokinetics) analysis on a test dose of busulfan on the day prior to initiating conditioning. Based on the PK results a starting dose of busulfan was determined. A second PK study was done after the starting dose to ensure that the targeted dose was achieved. If it wasn't achieved, then a dose modification was made and a third PK study done."}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '35'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '34'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '1'}]}], 'dropWithdraws': [{'type': 'Death', 'reasons': [{'groupId': 'FG000', 'numSubjects': '1'}]}]}], 'recruitmentDetails': 'Subjects recruited from 9/2005 through 9/2010. Patients identified by the Pediatric BMT (Bone Marrow Transplant) Program and consented by study investigators as outpatients in the BMT clinic.', 'preAssignmentDetails': "Patients were ineligible if a cord blood was being used for the transplant or if they met any of the other ineligibility criteria in the protocol or did not meet eligibility criteria such as having Fanconi's Anemia."}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '35', 'groupId': 'BG000'}]}], 'groups': [{'id': 'BG000', 'title': 'Single Arm - Transplant Pre-conditioning Per Study Protocol', 'description': "All eligible patients received pre-transplant conditioning with Alemtuzumab, fludarabine and targeted busulfan followed by allogeneic transplant. The initial dose of busulfan was determined by performing PK (pharmacokinetics) analysis on a test dose of busulfan on the day prior to initiating conditioning. Based on the PK results a starting dose of busulfan was determined. A second PK study was done after the starting dose to ensure that the targeted dose was achieved. If it wasn't achieved, then a dose modification was made and a third PK study done."}], 'measures': [{'title': 'Age, Categorical', 'classes': [{'categories': [{'title': '<=18 years', 'measurements': [{'value': '34', 'groupId': 'BG000'}]}, {'title': 'Between 18 and 65 years', 'measurements': [{'value': '1', 'groupId': 'BG000'}]}, {'title': '>=65 years', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Age, Continuous', 'classes': [{'categories': [{'measurements': [{'value': '7.58', 'spread': '5.25', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'years', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '13', 'groupId': 'BG000'}]}, {'title': 'Male', 'measurements': [{'value': '22', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Region of Enrollment', 'classes': [{'title': 'United States', 'categories': [{'measurements': [{'value': '35', 'groupId': 'BG000'}]}]}], 'paramType': 'NUMBER', 'unitOfMeasure': 'participants'}], 'populationDescription': 'Thirty-five children undergoing allogeneic hemotopoietic stem cell transplants (HSCTs) for malignant and nonmalignant diseases; 12 with HLA-matched related donors (MRDs), 16 with 10 of 10 HLA allele-matched unrelated donors (MUDs), 7 with 9 of 10 allele MUDs.'}}, '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': '2005-01'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2017-08', 'completionDateStruct': {'date': '2011-09', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2017-08-30', 'studyFirstSubmitDate': '2006-03-09', 'resultsFirstSubmitDate': '2013-01-31', 'studyFirstSubmitQcDate': '2006-03-09', 'lastUpdatePostDateStruct': {'date': '2017-09-28', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2013-04-22', 'studyFirstPostDateStruct': {'date': '2006-03-13', 'type': 'ESTIMATED'}, 'resultsFirstPostDateStruct': {'date': '2013-05-16', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2011-09', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Number of Participants Achieving Durable Engraftment (Presence of Donor Cells) at 6 Weeks Post Transplantation', 'timeFrame': '6 weeks post-transplant', 'description': 'Peripheral blood chimerism studies were performed by quantitative real time polymerase chain reaction (qPCR) evaluation of differential short tandem repeat DNA sequences'}], 'secondaryOutcomes': [{'measure': 'Treatment-related Mortality at 100 Days and 1 Year Post Transplantation', 'timeFrame': '100 days and 1 year'}, {'measure': 'Toxicity Grade ≥ 3 From Start of Conditioning Through the First Year Post Transplantation', 'timeFrame': '1 year post-transplantation'}, {'measure': 'Cytomegalovirus (CMV) Viral Infection and Disease Symptoms', 'timeFrame': 'Up to one year post-transplant', 'description': 'polymerase chain reaction testing for presence of CMV weekly until at least day +100 then every 2 weeks until T-cell reconstitution as defined by cluster of differentiation 4 (CD4) \\> 200 cells/mm3. Median time to T-cell reconstitution was 6 months.'}, {'measure': 'Disease-free Survival With Correction of Disease at One Year Post Transplantation', 'timeFrame': '1 year post-transplantation', 'description': 'Patients deemed "alive and well" at follow-up timepoint later than 1-year post-transplantation'}]}, 'conditionsModule': {'keywords': ['de novo myelodysplastic syndromes', 'previously treated myelodysplastic syndromes', 'secondary myelodysplastic syndromes', 'childhood acute myeloid leukemia in remission', 'childhood chronic myelogenous leukemia', 'congenital amegakaryocytic thrombocytopenia', 'Diamond-Blackfan anemia', 'severe congenital neutropenia', 'secondary acute myeloid leukemia', 'chronic phase chronic myelogenous leukemia', 'childhood myelodysplastic syndromes'], 'conditions': ['Congenital Amegakaryocytic Thrombocytopenia', 'Diamond-blackfan Anemia', 'Leukemia', 'Myelodysplastic Syndromes', 'Severe Congenital Neutropenia']}, 'descriptionModule': {'briefSummary': "RATIONALE: Monoclonal antibodies, such as alemtuzumab, can block cancer growth in different ways. Some block the ability of cancer cells to grow and spread. Others find cancer cells and help kill them or carry cancer-killing substances to them. Drugs used in chemotherapy, such as fludarabine and busulfan, work in different ways to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. A peripheral stem cell, bone marrow , or umbilical cord blood transplant may be able to replace blood-forming cells that were destroyed by chemotherapy. Sometimes the transplanted cells from a donor can make an immune response against the body's normal cells. Giving cyclosporine together with methotrexate and methylprednisolone may stop this from happening.\n\nPURPOSE: This phase II trial is studying how well giving alemtuzumab together with fludarabine and busulfan works when given before donor stem cell transplant in treating young patients with hematologic disorders.", 'detailedDescription': 'OBJECTIVES:\n\nPrimary\n\n* Determine the engraftment rate with reduced toxicity ablative conditioning regimen comprising alemtuzumab, fludarabine, and busulfan followed by allogeneic stem cell transplantation in pediatric patients with stem cell defects, marrow failure syndromes, hemoglobinopathy, severe immunodeficiency syndromes (nonsevere combined immunodeficiency disorders), myelodysplastic syndromes, or myeloid leukemia.\n\nSecondary\n\n* Determine the acute reactions, incidence of infections, and rate of immune reconstitution in patients treated with this regimen.\n\nOUTLINE: This is a multicenter study.\n\n* Conditioning regimen: Patients receive alemtuzumab IV over 6 hours on days -12 to -10, high-dose busulfan IV over 2 hours 4 times daily on days -9 to -6, and fludarabine IV over 30 minutes on days -5 to -2.\n* Allogeneic stem cell transplantation: Two days after the completion of conditioning regimen, patients undergo allogeneic bone marrow, peripheral blood stem cell, or umbilical cord blood transplantation on day 0. Patients receive filgrastim (G-CSF) subcutaneously beginning on day 5 and continuing until blood counts recover.\n* Graft-vs-host disease (GVHD) prophylaxis:\n\n * Most transplantations (bone marrow or peripheral blood stem cell transplantation): Patients receive cyclosporine IV continuously beginning on day -1 until at least day 50 followed by a taper at either 2 months, 9 months, or 1 year in the absence of GVHD. Patients also receive methotrexate on days 1, 3, and 6.\n * Umbilical cord blood transplantation: Patients receive cyclosporine as in most transplantations, and methylprednisolone IV twice daily on days 0-21 followed by a weekly taper.\n\nAfter transplantation, patients are followed periodically for up to 20 years.\n\nPROJECTED ACCRUAL: A total of 35 patients will be accrued for this study.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT'], 'maximumAge': '21 Years', 'healthyVolunteers': False, 'eligibilityCriteria': "DISEASE CHARACTERISTICS:\n\n* Diagnosis of 1 of the following hematologic conditions:\n\n * Aplastic anemia with marrow aplasia, meeting all of the following criteria:\n\n * Absolute neutrophil count \\< 500/mm\\^3\n * Platelet and/or red cell transfusion dependent\n * Chronic aplastic anemia, meeting all of the following criteria:\n\n * Transfusion dependent\n * Unresponsive to immunosuppressive therapy\n * Alternative matched unrelated donor has been identified\n * Congenital marrow failure syndrome, including any of the following (with closely matched related or unrelated donor):\n\n * Primary red cell aplasia (Diamond-Blackfan syndrome)\n * Congenital neutropenia (Kostmann's syndrome)\n * Amegakaryocytic thrombocytopenia\n * Congenital dyserythropoietic anemias\n * Other severe acquired cytopenias in which a transplantation using a combined busulfan/cyclophosphamide conditioning regimen is indicated\n * Hemoglobinopathy (with closely matched related or unrelated donor)\n\n * β-thalassemia major\n * Sickle cell anemia\n * Hemoglobin E/β-thalassemia\n * Severe immunodeficiency disease\n\n * Chediak-Higashi disease\n * Wiskott-Aldrich syndrome\n * Combined immunodeficiency disease (Nezelof's)\n * Hyper immunoglobulin M (IgM) syndrome\n * Bare lymphocyte syndrome\n * Chronic granulomatous disease\n * Familial erythrohemophagocytic lymphohistiocytosis\n * Other stem cell defects (e.g., osteopetrosis)\n * Severe immune dysregulation/autoimmune disorders\n\n * Achieved a transient response to prior immunosuppressive therapy\n * Chronic myelogenous leukemia\n\n * Disease in first chronic phase\n * Acute myeloid leukemia\n\n * Disease in first remission\n * Myelodysplastic syndromes\n * Inborn errors of metabolism\n * Histiocytosis\n* No severe combined immunodeficiency disease\n* Matched related or unrelated donor available by high resolution DNA typing\n\n * Related donor, meeting both of the following criteria:\n\n * Matched at both human leukocyte antigen (HLA)-Drβ1 alleles\n * No more than 1 mismatch at the 4 HLA-A and -B alleles\n * Unrelated donor, meeting 1 of the following criteria:\n\n * Marrow matched at both HLA-Drβ1 alleles AND no more than 1 mismatch at the 4 HLA-A and -B alleles\n * Umbilical cord blood matched at 5/6 HLA-A, -B, and -DRβ1 alleles with at least 1 -DRβ1 match AND there are ≥ 3x10\\^5 CD34+ (Cluster of differentiation 34-positive) cells per kg body weight of recipient available at the time of cryopreservation\n\nPATIENT CHARACTERISTICS:\n\n* Cardiac ejection fraction ≥ 27%\n* Creatinine clearance ≥ 50 mL/min by 24-hour urine collection or glomerular filtration rate\n* DLCO (diffusion capacity of lung for carbon monoxide) ≥ 50% of predicted (corrected for anemia/lung volume)\n\nPRIOR CONCURRENT THERAPY:\n\n* No prior transplantation for leukemia from which patient remains engrafted and alemtuzumab is not needed as part of the conditioning regimen"}, 'identificationModule': {'nctId': 'NCT00301834', 'briefTitle': 'Alemtuzumab, Fludarabine, and Busulfan Followed By Donor Stem Cell Transplant in Treating Young Patients With Hematologic Disorders', 'organization': {'class': 'OTHER', 'fullName': 'University of California, San Francisco'}, 'officialTitle': 'Evaluation of Fludarabine, Busulfan and Alemtuzumab as a Reduced Toxicity Ablative Bone Marrow Stem Cell Transplant Regimen for Children With Stem Cell Defects, Marrow Failure Syndromes, or Myelodysplastic Syndrome (MDS)/Leukemia', 'orgStudyIdInfo': {'id': 'CDR0000462406'}, 'secondaryIdInfos': [{'id': 'UCSF-04152', 'type': 'OTHER', 'domain': 'UCSF Helen Diller Family Comprehensive Cancer Center'}, {'id': 'UCSF-00452', 'type': 'OTHER', 'domain': 'UCSF Helen Diller Family Comprehensive Cancer Center'}, {'id': 'UCSF-H411-25738-02', 'type': 'OTHER', 'domain': 'UCSF Committee on Human Research (CHR)'}]}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Single arm - conditioning and transplant', 'description': 'Alemtuzumab 0.5 mg/kg (maximum 15 mg) daily for 3 days; Busulfan i.v. every 6 hours from day -9 to day -6 for 16 total doses; Fludarabine phosphate from day -5 for 4 days at 1.3 mg/kg (if patient was less than 12 kg) or 40 mg/m\\*2 per dose; Cyclosporine continuous infusion 3 mg/kg/Day beginning day -1 for GVHD prophylaxis; Methotrexate at 15 mg/m\\*2 on day +1, 10 mg/m\\*2 on days +3, +6, and (only for MUDs) day +11 also for GVHD prophylaxis; Methylprednisolone only as required for GVHD prophylaxis; allogeneic bone marrow transplantation or allogeneic hematopoietic stem cell transplantation or peripheral blood stem cell transplantation or umbilical cord blood transplantation.', 'interventionNames': ['Biological: alemtuzumab', 'Drug: busulfan', 'Drug: cyclosporine', 'Drug: fludarabine phosphate', 'Drug: methotrexate', 'Drug: methylprednisolone', 'Procedure: allogeneic bone marrow transplantation', 'Procedure: allogeneic hematopoietic stem cell transplantation', 'Procedure: peripheral blood stem cell transplantation', 'Procedure: umbilical cord blood transplantation']}], 'interventions': [{'name': 'alemtuzumab', 'type': 'BIOLOGICAL', 'armGroupLabels': ['Single arm - conditioning and transplant']}, {'name': 'busulfan', 'type': 'DRUG', 'armGroupLabels': ['Single arm - conditioning and transplant']}, {'name': 'cyclosporine', 'type': 'DRUG', 'armGroupLabels': ['Single arm - conditioning and transplant']}, {'name': 'fludarabine phosphate', 'type': 'DRUG', 'armGroupLabels': ['Single arm - conditioning and transplant']}, {'name': 'methotrexate', 'type': 'DRUG', 'armGroupLabels': ['Single arm - conditioning and transplant']}, {'name': 'methylprednisolone', 'type': 'DRUG', 'armGroupLabels': ['Single arm - conditioning and transplant']}, {'name': 'allogeneic bone marrow transplantation', 'type': 'PROCEDURE', 'armGroupLabels': ['Single arm - conditioning and transplant']}, {'name': 'allogeneic hematopoietic stem cell transplantation', 'type': 'PROCEDURE', 'armGroupLabels': ['Single arm - conditioning and transplant']}, {'name': 'peripheral blood stem cell transplantation', 'type': 'PROCEDURE', 'armGroupLabels': ['Single arm - conditioning and transplant']}, {'name': 'umbilical cord blood transplantation', 'type': 'PROCEDURE', 'armGroupLabels': ['Single arm - conditioning and transplant']}]}, 'contactsLocationsModule': {'locations': [{'zip': '94115', 'city': 'San Francisco', 'state': 'California', 'country': 'United States', 'facility': 'UCSF Helen Diller Family Comprehensive Cancer Center', 'geoPoint': {'lat': 37.77493, 'lon': -122.41942}}, {'zip': '53792-6164', 'city': 'Madison', 'state': 'Wisconsin', 'country': 'United States', 'facility': 'University of Wisconsin Paul P. Carbone Comprehensive Cancer Center', 'geoPoint': {'lat': 43.07305, 'lon': -89.40123}}], 'overallOfficials': [{'name': 'Morton J. Cowan, MD', 'role': 'STUDY_CHAIR', 'affiliation': 'University of California, San Francisco'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'University of California, San Francisco', 'class': 'OTHER'}, 'collaborators': [{'name': 'National Cancer Institute (NCI)', 'class': 'NIH'}], 'responsibleParty': {'type': 'SPONSOR'}}}}