Ignite Creation Date:
2025-12-24 @ 3:25 PM
Ignite Modification Date:
2026-01-03 @ 4:36 AM
Study NCT ID:
NCT00744692
Status:
COMPLETED
Last Update Posted:
2014-08-13
First Post:
2008-08-28
Is NOT Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
Reduced Intensity Conditioning for Umbilical Cord Blood Transplant in Pediatric Patients With Non-Malignant Disorders
Sponsor:
Organization:
Raw JSON
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{'id': 'D009750', 'term': 'Nutritional and Metabolic Diseases'}, {'id': 'D000745', 'term': 'Anemia, Hemolytic, Congenital'}, {'id': 'D000743', 'term': 'Anemia, Hemolytic'}, {'id': 'D000740', 'term': 'Anemia'}, {'id': 'D000081207', 'term': 'Primary Immunodeficiency Diseases'}, {'id': 'D007232', 'term': 'Infant, Newborn, Diseases'}, {'id': 'D049914', 'term': 'DNA Repair-Deficiency Disorders'}, {'id': 'D009083', 'term': 'Mucopolysaccharidoses'}, {'id': 'D002239', 'term': 'Carbohydrate Metabolism, Inborn Errors'}, {'id': 'D017520', 'term': 'Mucinoses'}, {'id': 'D003240', 'term': 'Connective Tissue Diseases'}, {'id': 'D017437', 'term': 'Skin and Connective Tissue Diseases'}, {'id': 'D038901', 'term': 'X-Linked Intellectual Disability'}, {'id': 'D008607', 'term': 'Intellectual Disability'}, {'id': 'D019954', 'term': 'Neurobehavioral Manifestations'}, {'id': 'D009461', 'term': 'Neurologic Manifestations'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}, {'id': 'D040181', 'term': 'Genetic Diseases, X-Linked'}, {'id': 'D020271', 'term': 'Heredodegenerative Disorders, Nervous System'}, {'id': 'D029502', 'term': 'Anemia, Hypoplastic, Congenital'}, {'id': 'D000741', 'term': 'Anemia, Aplastic'}, {'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'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'D000074323', 'term': 'Alemtuzumab'}, {'id': 'D006918', 'term': 'Hydroxyurea'}, {'id': 'C024352', 'term': 'fludarabine'}, {'id': 'D008558', 'term': 'Melphalan'}, {'id': 'D013852', 'term': 'Thiotepa'}], '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': 'D014508', 'term': 'Urea'}, {'id': 'D000577', 'term': 'Amides'}, {'id': 'D009930', 'term': 'Organic Chemicals'}, {'id': 'D009588', 'term': 'Nitrogen Mustard Compounds'}, {'id': 'D009150', 'term': 'Mustard Compounds'}, {'id': 'D006846', 'term': 'Hydrocarbons, Halogenated'}, {'id': 'D006838', 'term': 'Hydrocarbons'}, {'id': 'D010649', 'term': 'Phenylalanine'}, {'id': 'D024322', 'term': 'Amino Acids, Aromatic'}, {'id': 'D000598', 'term': 'Amino Acids, Cyclic'}, {'id': 'D000596', 'term': 'Amino Acids'}, {'id': 'D063088', 'term': 'Phosphoramides'}, {'id': 'D009943', 'term': 'Organophosphorus Compounds'}, {'id': 'D013721', 'term': 'Triethylenephosphoramide'}, {'id': 'D001388', 'term': 'Aziridines'}, {'id': 'D001389', 'term': 'Azirines'}, {'id': 'D006573', 'term': 'Heterocyclic Compounds, 1-Ring'}, {'id': 'D006571', 'term': 'Heterocyclic Compounds'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'suhag.parikh@dm.duke.edu', 'phone': '919-668-1100', 'title': 'Dr. Suhag Parikh', 'organization': 'Duke University Medical Center'}, 'certainAgreement': {'piSponsorEmployee': True}}, 'adverseEventsModule': {'description': 'Adverse events that were expected and non-serious were not collected in this study.', 'eventGroups': [{'id': 'EG000', 'title': 'RIC Cord Blood Transplant', 'description': 'Reduced Intensity Conditioning for Umbilical Cord Blood Transplant\n\nUnrelated Umbilical Cord Blood Transplant: Reduced Intensity Conditioning for unrelated umbilical cord blood transplant\n\nReduced Intensity Conditioning', 'otherNumAtRisk': 22, 'otherNumAffected': 0, 'seriousNumAtRisk': 22, 'seriousNumAffected': 18}], 'seriousEvents': [{'term': 'Respiratory Distress', 'stats': [{'groupId': 'EG000', 'numAtRisk': 22, 'numAffected': 2}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders'}, {'term': 'Fever', 'stats': [{'groupId': 'EG000', 'numAtRisk': 22, 'numAffected': 5}], 'organSystem': 'Investigations'}, {'term': 'Dehydration', 'stats': [{'groupId': 'EG000', 'numAtRisk': 22, 'numAffected': 2}], 'organSystem': 'Metabolism and nutrition disorders'}, {'term': 'Auto Immune Hemolytic Anemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 22, 'numAffected': 2}], 'organSystem': 'Blood and lymphatic system disorders'}, {'term': 'Infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 22, 'numAffected': 12}], 'organSystem': 'Infections and infestations'}, {'term': 'Hemolytic Transfusion Reaction', 'stats': [{'groupId': 'EG000', 'numAtRisk': 22, 'numAffected': 1}], 'organSystem': 'Blood and lymphatic system disorders'}, {'term': 'Graft Failure Post Transplant', 'stats': [{'groupId': 'EG000', 'numAtRisk': 22, 'numAffected': 2}], 'organSystem': 'Blood and lymphatic system disorders'}, {'term': 'Sepsis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 22, 'numAffected': 3}], 'organSystem': 'Infections and infestations'}, {'term': 'Abdominal Pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 22, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders'}, {'term': 'Nephrotic Syndrome', 'stats': [{'groupId': 'EG000', 'numAtRisk': 22, 'numAffected': 1}], 'organSystem': 'Renal and urinary disorders'}, {'term': 'VP Shunt Malfunction', 'stats': [{'groupId': 'EG000', 'numAtRisk': 22, 'numAffected': 1}], 'organSystem': 'Nervous system disorders'}, {'term': 'GI Distress', 'stats': [{'groupId': 'EG000', 'numAtRisk': 22, 'numAffected': 3}], 'organSystem': 'Gastrointestinal disorders'}, {'term': 'Multi System Organ Failure', 'stats': [{'groupId': 'EG000', 'numAtRisk': 22, 'numAffected': 2}], 'organSystem': 'General disorders'}, {'term': 'Seizure', 'stats': [{'groupId': 'EG000', 'numAtRisk': 22, 'numAffected': 1}], 'organSystem': 'Nervous system disorders'}, {'term': 'Respiratory Failure', 'stats': [{'groupId': 'EG000', 'numAtRisk': 22, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders'}, {'term': 'Graft Versus Host Disease', 'stats': [{'groupId': 'EG000', 'numAtRisk': 22, 'numAffected': 4}], 'organSystem': 'General disorders'}, {'term': 'Central Line Removal', 'stats': [{'groupId': 'EG000', 'numAtRisk': 22, 'numAffected': 1}], 'organSystem': 'Surgical and medical procedures'}, {'term': 'Deep Vein Thrombosis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 22, 'numAffected': 1}], 'organSystem': 'Blood and lymphatic system disorders'}, {'term': 'Hyperglycemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 22, 'numAffected': 1}], 'organSystem': 'Endocrine disorders'}, {'term': 'Pnematosis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 22, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders'}, {'term': 'Posterior Reversible Encephalopathy Syndrome', 'stats': [{'groupId': 'EG000', 'numAtRisk': 22, 'numAffected': 1}], 'organSystem': 'Nervous system disorders'}, {'term': 'Necrotic Ulcer (skin)', 'stats': [{'groupId': 'EG000', 'numAtRisk': 22, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders'}, {'term': 'Dental Rehabilitation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 22, 'numAffected': 1}], 'organSystem': 'Surgical and medical procedures'}], 'frequencyThreshold': '0'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Determine the Feasibility of Attaining Acceptable Rates of Donor Cell Engraftment (>25% Donor Cells at 180 Days) Following RIC Regimens in Children < 21 Years Receiving UCBT for Non-malignant Disorders.', 'denoms': [{'units': 'Participants', 'counts': [{'value': '18', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'RIC Cord Blood Transplant', 'description': 'Reduced Intensity Conditioning for Umbilical Cord Blood Transplant\n\nUnrelated Umbilical Cord Blood Transplant: Reduced Intensity Conditioning for unrelated umbilical cord blood transplant\n\nReduced Intensity Conditioning'}], 'classes': [{'categories': [{'measurements': [{'value': '88', 'groupId': 'OG000'}]}]}], 'paramType': 'NUMBER', 'timeFrame': '180 days post transplant', 'description': 'Determine the feasibility of attaining acceptable rates of donor cell engraftment (\\>25% donor cells at 180 days) following reduced intensity conditioning regimens in children \\< 21 years receiving cord blood transplant for non-malignant disorders.', 'unitOfMeasure': '% of participants', 'reportingStatus': 'POSTED', 'populationDescription': 'Of the 22 patients enrolled, 18 patients were alive at 180 days, the time-point for primary end point'}, {'type': 'SECONDARY', 'title': 'To Describe the Pace of Neutrophil Recovery', 'denoms': [{'units': 'Participants', 'counts': [{'value': '22', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'RIC Cord Blood Transplant', 'description': 'Reduced Intensity Conditioning for Umbilical Cord Blood Transplant\n\nUnrelated Umbilical Cord Blood Transplant: Reduced Intensity Conditioning for unrelated umbilical cord blood transplant\n\nReduced Intensity Conditioning'}], 'classes': [{'categories': [{'measurements': [{'value': '20', 'groupId': 'OG000', 'lowerLimit': '12', 'upperLimit': '28'}]}]}], 'paramType': 'MEDIAN', 'timeFrame': '42 days post transplant', 'description': 'Neutrophil recovery was defined as the first day of an absolute neutrophil count (ANC) more than 500/uL for 3 consecutive days not secondary to granulocyte infusions', 'unitOfMeasure': 'days', 'dispersionType': 'Full Range', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'To Evaluate the Pace of Immune Reconstitution.', 'denoms': [{'units': 'Participants', 'counts': [{'value': '15', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'RIC Cord Blood Transplant', 'description': 'Reduced Intensity Conditioning for Umbilical Cord Blood Transplant\n\nUnrelated Umbilical Cord Blood Transplant: Reduced Intensity Conditioning for unrelated umbilical cord blood transplant\n\nReduced Intensity Conditioning'}], 'classes': [{'categories': [{'measurements': [{'value': '805', 'groupId': 'OG000', 'lowerLimit': '6', 'upperLimit': '2013'}]}]}], 'paramType': 'MEDIAN', 'timeFrame': '1 year post transplant', 'description': 'Immune reconstitution after RIC in UCBT was described. CD4 count is a standard measure of immune reconstitution and is described here. Additional data is available upon request.', 'unitOfMeasure': 'cells/uL', 'dispersionType': 'Full Range', 'reportingStatus': 'POSTED', 'populationDescription': 'Of the 22 patients at baseline, 5 patients died early before a year; and 2 additional patients had early graft failure. Thus 7 patients were not available for analysis of Immune reconstitution endpoint. CD4 count is reported here.'}, {'type': 'SECONDARY', 'title': 'To Determine the Overall Survival at day180 Post-transplant', 'denoms': [{'units': 'Participants', 'counts': [{'value': '22', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'RIC Cord Blood Transplant', 'description': 'Reduced Intensity Conditioning for Umbilical Cord Blood Transplant\n\nUnrelated Umbilical Cord Blood Transplant: Reduced Intensity Conditioning for unrelated umbilical cord blood transplant\n\nReduced Intensity Conditioning'}], 'classes': [{'categories': [{'measurements': [{'value': '81.8', 'groupId': 'OG000', 'lowerLimit': '58.5', 'upperLimit': '92.8'}]}]}], 'paramType': 'NUMBER', 'timeFrame': '180 days', 'description': 'To determine the overall survival at day180 post-transplant: determined by Kaplan Meier survival analysis', 'unitOfMeasure': 'percentage of participants', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'To Describe Incidence of Acute Graft Versus Host Disease (GVHD) (II - IV)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '22', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'RIC Cord Blood Transplant', 'description': 'Reduced Intensity Conditioning for Umbilical Cord Blood Transplant\n\nUnrelated Umbilical Cord Blood Transplant: Reduced Intensity Conditioning for unrelated umbilical cord blood transplant\n\nReduced Intensity Conditioning'}], 'classes': [{'categories': [{'measurements': [{'value': '22.7', 'groupId': 'OG000', 'lowerLimit': '5.3', 'upperLimit': '40.1'}]}]}], 'paramType': 'NUMBER', 'timeFrame': '100 days post transplant', 'description': 'To describe incidence of acute Graft Versus Host Disease (GVHD) (II - IV) : measured by cumulative incidence analysis', 'unitOfMeasure': 'percentage of participants', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'To Describe the Incidence of Grade 3-4 Organ Toxicity', 'denoms': [{'units': 'Participants', 'counts': [{'value': '22', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'RIC Cord Blood Transplant', 'description': 'Reduced Intensity Conditioning for Umbilical Cord Blood Transplant\n\nUnrelated Umbilical Cord Blood Transplant: Reduced Intensity Conditioning for unrelated umbilical cord blood transplant\n\nReduced Intensity Conditioning'}], 'classes': [{'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}], 'paramType': 'NUMBER', 'timeFrame': '2 years post transplant', 'unitOfMeasure': 'participants', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'To Evaluate Long-term Complications, Such as Sterility, Endocrinopathy, and Growth Failure', 'denoms': [{'units': 'Participants', 'counts': [{'value': '15', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'RIC Cord Blood Transplant', 'description': 'Reduced Intensity Conditioning for Umbilical Cord Blood Transplant\n\nUnrelated Umbilical Cord Blood Transplant: Reduced Intensity Conditioning for unrelated umbilical cord blood transplant\n\nReduced Intensity Conditioning'}], 'classes': [{'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'at least 2 years post transplant', 'unitOfMeasure': 'percentage of patients', 'reportingStatus': 'POSTED', 'populationDescription': 'Of the 22 patients at baseline, 5 patients died early before a year; and 2 additional patients had early graft failure. Thus 7 patients were not available for analysis of this 2 year late effects endpoint.'}, {'type': 'SECONDARY', 'title': 'To Evaluate the Incidence of Late Graft Failures at 2 Years Post-transplant', 'denoms': [{'units': 'Participants', 'counts': [{'value': '15', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'RIC Cord Blood Transplant', 'description': 'Reduced Intensity Conditioning for Umbilical Cord Blood Transplant\n\nUnrelated Umbilical Cord Blood Transplant: Reduced Intensity Conditioning for unrelated umbilical cord blood transplant\n\nReduced Intensity Conditioning'}], 'classes': [{'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}], 'paramType': 'NUMBER', 'timeFrame': '2 years post transplant', 'unitOfMeasure': 'participants', 'reportingStatus': 'POSTED', 'populationDescription': 'Of the 22 patients at baseline, 5 patients died early before a year; and 2 additional patients had early graft failure. Thus 7 patients were not available for analysis of this 2 year late graft failure endpoint.'}, {'type': 'SECONDARY', 'title': 'To Describe the Pace of Platelet Recovery', 'denoms': [{'units': 'Participants', 'counts': [{'value': '22', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'RIC Cord Blood Transplant', 'description': 'Reduced Intensity Conditioning for Umbilical Cord Blood Transplant\n\nUnrelated Umbilical Cord Blood Transplant: Reduced Intensity Conditioning for unrelated umbilical cord blood transplant\n\nReduced Intensity Conditioning'}], 'classes': [{'categories': [{'measurements': [{'value': '48', 'groupId': 'OG000', 'lowerLimit': '33', 'upperLimit': '144'}]}]}], 'paramType': 'MEDIAN', 'timeFrame': '180 days post transplant', 'description': 'Platelet engraftment was defined as the first day of platelet counts more than 50,000/uL for 7 consecutive days without transfusions', 'unitOfMeasure': 'days', 'dispersionType': 'Full Range', 'reportingStatus': 'POSTED'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'RIC Cord Blood Transplant', 'description': 'Reduced Intensity Conditioning for Umbilical Cord Blood Transplant\n\nUnrelated Umbilical Cord Blood Transplant: Reduced Intensity Conditioning for unrelated umbilical cord blood transplant\n\nReduced Intensity Conditioning'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '22'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '22'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}]}]}]}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '22', 'groupId': 'BG000'}]}], 'groups': [{'id': 'BG000', 'title': 'RIC Cord Blood Transplant', 'description': 'Reduced Intensity Conditioning for Umbilical Cord Blood Transplant\n\nUnrelated Umbilical Cord Blood Transplant: Reduced Intensity Conditioning for unrelated umbilical cord blood transplant\n\nReduced Intensity Conditioning'}], 'measures': [{'title': 'Age, Continuous', 'classes': [{'categories': [{'measurements': [{'value': '2.83', 'groupId': 'BG000', 'lowerLimit': '0.3', 'upperLimit': '8'}]}]}], 'paramType': 'MEDIAN', 'description': 'Age (in years) on the day of transplant', 'unitOfMeasure': 'years', 'dispersionType': 'FULL_RANGE'}, {'title': 'Age, Categorical', 'classes': [{'categories': [{'title': '<=18 years', 'measurements': [{'value': '22', 'groupId': 'BG000'}]}, {'title': 'Between 18 and 65 years', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': '>=65 years', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'description': 'Age (years) on the day of transplant', 'unitOfMeasure': 'Participants'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '9', 'groupId': 'BG000'}]}, {'title': 'Male', 'measurements': [{'value': '13', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}], 'populationDescription': '22 subjects undergoing umbilical cord blood transplant'}}, 'protocolSection': {'designModule': {'phases': ['PHASE1'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NON_RANDOMIZED', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 22}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2008-10'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2014-07', 'completionDateStruct': {'date': '2014-04', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2014-07-23', 'studyFirstSubmitDate': '2008-08-28', 'resultsFirstSubmitDate': '2014-07-23', 'studyFirstSubmitQcDate': '2008-08-29', 'lastUpdatePostDateStruct': {'date': '2014-08-13', 'type': 'ESTIMATED'}, 'resultsFirstSubmitQcDate': '2014-07-23', 'studyFirstPostDateStruct': {'date': '2008-09-01', 'type': 'ESTIMATED'}, 'resultsFirstPostDateStruct': {'date': '2014-08-13', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2012-12', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Determine the Feasibility of Attaining Acceptable Rates of Donor Cell Engraftment (>25% Donor Cells at 180 Days) Following RIC Regimens in Children < 21 Years Receiving UCBT for Non-malignant Disorders.', 'timeFrame': '180 days post transplant', 'description': 'Determine the feasibility of attaining acceptable rates of donor cell engraftment (\\>25% donor cells at 180 days) following reduced intensity conditioning regimens in children \\< 21 years receiving cord blood transplant for non-malignant disorders.'}], 'secondaryOutcomes': [{'measure': 'To Describe the Pace of Neutrophil Recovery', 'timeFrame': '42 days post transplant', 'description': 'Neutrophil recovery was defined as the first day of an absolute neutrophil count (ANC) more than 500/uL for 3 consecutive days not secondary to granulocyte infusions'}, {'measure': 'To Evaluate the Pace of Immune Reconstitution.', 'timeFrame': '1 year post transplant', 'description': 'Immune reconstitution after RIC in UCBT was described. CD4 count is a standard measure of immune reconstitution and is described here. Additional data is available upon request.'}, {'measure': 'To Determine the Overall Survival at day180 Post-transplant', 'timeFrame': '180 days', 'description': 'To determine the overall survival at day180 post-transplant: determined by Kaplan Meier survival analysis'}, {'measure': 'To Describe Incidence of Acute Graft Versus Host Disease (GVHD) (II - IV)', 'timeFrame': '100 days post transplant', 'description': 'To describe incidence of acute Graft Versus Host Disease (GVHD) (II - IV) : measured by cumulative incidence analysis'}, {'measure': 'To Describe the Incidence of Grade 3-4 Organ Toxicity', 'timeFrame': '2 years post transplant'}, {'measure': 'To Evaluate Long-term Complications, Such as Sterility, Endocrinopathy, and Growth Failure', 'timeFrame': 'at least 2 years post transplant'}, {'measure': 'To Evaluate the Incidence of Late Graft Failures at 2 Years Post-transplant', 'timeFrame': '2 years post transplant'}, {'measure': 'To Describe the Pace of Platelet Recovery', 'timeFrame': '180 days post transplant', 'description': 'Platelet engraftment was defined as the first day of platelet counts more than 50,000/uL for 7 consecutive days without transfusions'}]}, 'oversightModule': {'oversightHasDmc': False}, 'conditionsModule': {'keywords': ['Immunodeficiencies', 'Congenital Marrow Failures', 'Hemoglobinopathies', 'Inborn Errors of Metabolism', 'SCIDS', 'Wiskott Aldrich', 'FEL', 'HLH', 'IPEX', 'LAD', 'Sickle Cell', 'Thalassemia', "Omenn's Syndrome", "Hurler's Syndrome", 'MLD', 'ALD', 'Sanfilippo', 'Krabbe', "Hunter's syndrome", 'TaySachs', 'Diamond Blackfan Anemia', 'transplant', 'MPS', 'Gaucher'], 'conditions': ['Non Malignant Disorders', 'Immunodeficiencies', 'Congenital Marrow Failures', 'Hemoglobinopathies', 'Inborn Errors of Metabolism', 'Sickle Cell', 'Thalassemia', 'Lysosomal Storage Disease']}, 'referencesModule': {'references': [{'pmid': '24296492', 'type': 'DERIVED', 'citation': 'Parikh SH, Mendizabal A, Benjamin CL, Komanduri KV, Antony J, Petrovic A, Hale G, Driscoll TA, Martin PL, Page KM, Flickinger K, Moffet J, Niedzwiecki D, Kurtzberg J, Szabolcs P. A novel reduced-intensity conditioning regimen for unrelated umbilical cord blood transplantation in children with nonmalignant diseases. Biol Blood Marrow Transplant. 2014 Mar;20(3):326-36. doi: 10.1016/j.bbmt.2013.11.021. Epub 2013 Dec 1.'}]}, 'descriptionModule': {'briefSummary': 'The primary objective is to determine the feasibility of attaining acceptable rates of donor cell engraftment (\\>25% donor chimerism at 180 days) following reduced intensity conditioning (RIC) regimens in pediatric patients \\< 21 years receiving cord blood transplantation for non-malignant disorders.', 'detailedDescription': 'Myeloablative doses of chemotherapy and/or radiation therapy are employed with the primary purpose of eradicating malignant cells. Additionally, these regimens exert varying degree of immunosuppression/immunoablation that aids in reducing the likelihood of rejection by host hematopoietic cells. However, myeloablative /immunoablative regimens have also been associated with significant regimen related toxicity (RRT) and regimen related mortality (RRM) that may cause death in up to 20% of patients and significantly higher rate of severe organ dysfunction or failure. While most of these RRT occur typically in the first 100 days \\[ e.g. VOD (veno occlusive disease), pulmonary or intracranial hemorrhage, multiorgan failure (MOF)\\], there are significant long term toxicities of TBI and/or chemotherapy including growth impairment, gonadal dysfunction/failure, hypothyroidism, cataracts, neurocognitive impairment, and second malignancies.\n\nThe primary objective is to determine the feasibility of attaining acceptable rates of donor cell engraftment (\\>25% donor chimerism at 180 days) following reduced intensity conditioning (RIC) regimens in pediatric patients \\< 21 years receiving cord blood transplantation for non-malignant disorders.\n\nThe secondary objectives are:\n\n* To describe the pace of neutrophil and platelet recovery\n* To evaluate the pace of immune reconstitution.\n* To determine the treatment related mortality, overall survival and disease free survival by days 100 and 180 post-transplant\n* To describe incidence of acute Graft Versus Host Disease (GVHD) (II - IV) and chronic extensive GVHD\n* To describe the incidence of grade 3-4 organ toxicity\n* To evaluate long-term complications, such as sterility, endocrinopathy, and growth failure\n* To evaluate the incidence of late graft failures at 2 years post-transplant'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT'], 'maximumAge': '21 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* 0-21 years of age with a diagnosis of a immunodeficiency, congenital marrow failure syndrome, inborn error of metabolism, or hereditary anemia\n* Appropriately matched related or unrelated umbilical cord blood unit with a cell dose ≥ 3 x 10e7cells/kg\n* Performance score (lansky or karnofsky) greater than or equal to 70\n* Adequate organ function (Creatinine ≤ 2.0 mg/dl and creatinine clearance ≥ 50 ml/min/1.73 m2; Hepatic transaminases (ALT/AST) ≤ 4 x normal; Shortening fraction \\>26% or ejection fraction \\>40% or \\> 80% of normal value for age; Pulmonary function tests demonstrating CVC or FEV1/FVC of \\>60% of predicted for age.)\n* Informed consent\n* Not pregnant or breast feeding\n* Minimum life expectancy of at least 6 months\n* HIV negative\n* No uncontrolled infections at the time of cytoreduction\n* Disease specific inclusion criteria\n\nExclusion Criteria:\n\n* Patients with hemoglobinopathies \\> 3 years of age\n* UCB unit with a total nucleated cell count \\< 3 x 10e7/kg or \\> 2 antigen mismatching\n* Available HLA-matched related living donor able to donate without previous UCB donation\n* Allogeneic hematopoietic stem cell transplant within the previous 6 months\n* Any active malignancy, MDS, or any history of malignancy\n* Severe acquired aplastic anemia\n* DLCO \\< 60% of normal value for age; requirement for supplemental oxygen\n* Uncontrolled bacterial, viral or fungal infection (currently taking medication and progression of clinical symptoms)\n* Pregnancy or nursing mother\n* HIV/HTLV seropositive, Hep B surface antigen positive, or HCV RNA positive by PCR\n* Any condition that precludes serial follow-up'}, 'identificationModule': {'nctId': 'NCT00744692', 'briefTitle': 'Reduced Intensity Conditioning for Umbilical Cord Blood Transplant in Pediatric Patients With Non-Malignant Disorders', 'organization': {'class': 'OTHER', 'fullName': 'Duke University'}, 'officialTitle': 'A Pilot Study of Reduced Intensity Conditioning in Pediatric Patients <21 Years of Age With Non-Malignant Disorders Undergoing Umbilical Cord Blood Transplantation', 'orgStudyIdInfo': {'id': 'Pro00008753'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'RIC Cord Blood Transplant', 'description': 'Reduced Intensity Conditioning for Umbilical Cord Blood Transplant', 'interventionNames': ['Biological: Unrelated Umbilical Cord Blood Transplant', 'Drug: Reduced Intensity Conditioning']}], 'interventions': [{'name': 'Unrelated Umbilical Cord Blood Transplant', 'type': 'BIOLOGICAL', 'description': 'Reduced Intensity Conditioning for unrelated umbilical cord blood transplant', 'armGroupLabels': ['RIC Cord Blood Transplant']}, {'name': 'Reduced Intensity Conditioning', 'type': 'DRUG', 'otherNames': ['Campath', 'Hydroxyurea', 'Fludarabine', 'Melphalan', 'Thiotepa'], 'armGroupLabels': ['RIC Cord Blood Transplant']}]}, 'contactsLocationsModule': {'locations': [{'zip': '27705', 'city': 'Durham', 'state': 'North Carolina', 'country': 'United States', 'facility': 'Duke University Medical Center Pediatric Blood and Marrow Transplant Program', 'geoPoint': {'lat': 35.99403, 'lon': -78.89862}}], 'overallOfficials': [{'name': 'Suhag Parikh, MD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Duke Pediatric Blood and Marrow Transplant'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Duke University', 'class': 'OTHER'}, 'responsibleParty': {'type': 'SPONSOR'}}}}