Ignite Creation Date:
2025-12-25 @ 4:28 AM
Ignite Modification Date:
2025-12-26 @ 3:31 AM
Study NCT ID:
NCT00638820
Status:
TERMINATED
Last Update Posted:
2017-12-28
First Post:
2008-03-11
Is NOT Gene Therapy:
False
Has Adverse Events:
True
Brief Title:
Reduced Intensity AlloTransplant For Osteopetrosis
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D010022', 'term': 'Osteopetrosis'}], 'ancestors': [{'id': 'D010026', 'term': 'Osteosclerosis'}, {'id': 'D010009', 'term': 'Osteochondrodysplasias'}, {'id': 'D001848', 'term': 'Bone Diseases, Developmental'}, {'id': 'D001847', 'term': 'Bone Diseases'}, {'id': 'D009140', 'term': 'Musculoskeletal Diseases'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'D036101', 'term': 'Cord Blood Stem Cell Transplantation'}, {'id': 'D016026', 'term': 'Bone Marrow Transplantation'}, {'id': 'D000074323', 'term': 'Alemtuzumab'}, {'id': 'D002066', 'term': 'Busulfan'}, {'id': 'D000077866', 'term': 'Clofarabine'}, {'id': 'D011878', 'term': 'Radiotherapy'}], 'ancestors': [{'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'}, {'id': 'D016378', 'term': 'Tissue Transplantation'}, {'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': 'D000227', 'term': 'Adenine Nucleotides'}, {'id': 'D011685', 'term': 'Purine Nucleotides'}, {'id': 'D011687', 'term': 'Purines'}, {'id': 'D006574', 'term': 'Heterocyclic Compounds, 2-Ring'}, {'id': 'D000072471', 'term': 'Heterocyclic Compounds, Fused-Ring'}, {'id': 'D006571', 'term': 'Heterocyclic Compounds'}, {'id': 'D001087', 'term': 'Arabinonucleosides'}, {'id': 'D009705', 'term': 'Nucleosides'}, {'id': 'D009706', 'term': 'Nucleic Acids, Nucleotides, and Nucleosides'}, {'id': 'D009711', 'term': 'Nucleotides'}, {'id': 'D012265', 'term': 'Ribonucleotides'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'orcha001@umn.edu', 'phone': '612-626-2313', 'title': 'Paul Orchard, M.D.', 'organization': 'Masonic Cancer Center, University of Minnesota'}, 'certainAgreement': {'piSponsorEmployee': True}, 'limitationsAndCaveats': {'description': 'Only 3 participants were treated in this study; all died by Day 100. Data for both treatment arms were combined due to low number. (Arm 1 utilized mobilized peripheral blood or marrow (related or unrelated). Arm 2 received cord blood transplants).'}}, 'adverseEventsModule': {'timeFrame': 'Adverse events were collected from Day 1 through Day 100 of the study.', 'eventGroups': [{'id': 'EG000', 'title': 'Patients With Osteopetrosis Who Received Transplant', 'description': 'All patients enrolled with osteopetrosis and received transplant.', 'otherNumAtRisk': 3, 'otherNumAffected': 3, 'seriousNumAtRisk': 3, 'seriousNumAffected': 3}], 'otherEvents': [{'term': 'Neurologic', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'CTCAE (3.0)'}, {'term': 'Renal', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Renal and urinary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'CTCAE (3.0)'}, {'term': 'Pulmonary', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 3, 'numAffected': 3}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'CTCAE (3.0)'}, {'term': 'Vascular', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Vascular disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'CTCAE (3.0)'}], 'seriousEvents': [{'term': 'Death, peri-transplant', 'notes': 'Death was related to receiving transplant.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 3, 'numAffected': 3}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'CTCAE (3.0)'}], 'frequencyThreshold': '5'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Number of Patients Achieving Donor Cell Engraftment', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Patients With Osteopetrosis Who Received Transplant', 'description': 'All patients enrolled with osteopetrosis and received transplant.'}], 'classes': [{'categories': [{'measurements': [{'value': '2', 'groupId': 'OG000'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'Day 100', 'description': 'Number of patients with persistent presence of donor-derived cells at Day 100', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'anticipatedPostingDate': '2009-07'}, {'type': 'SECONDARY', 'title': 'Number of Patients With Transplant Related Death', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Patients With Osteopetrosis Who Received Transplant', 'description': 'All patients enrolled with osteopetrosis and received transplant.'}], 'classes': [{'categories': [{'measurements': [{'value': '3', 'groupId': 'OG000'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'Day 100', 'description': 'Number of participants died during study by Day 100 and reason for death was related to transplant.', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'anticipatedPostingDate': '2009-07'}, {'type': 'SECONDARY', 'title': 'Number of Patients With Transplant Related Toxicity', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Patients With Osteopetrosis Who Received Transplant', 'description': 'All patients enrolled with osteopetrosis and received transplant.'}], 'classes': [{'title': 'Neurologic', 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}]}]}, {'title': 'Renal', 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}]}]}, {'title': 'Pulmonary', 'categories': [{'measurements': [{'value': '3', 'groupId': 'OG000'}]}]}, {'title': 'Peri-Transplant Mortality (Death by Day 100)', 'categories': [{'measurements': [{'value': '3', 'groupId': 'OG000'}]}]}, {'title': 'Vascular', 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'Day 100', 'description': 'Number of patients experiencing adverse effects due to transplant categorized by body system using Common Terminology Criteria for Adverse Events coding from the National Cancer Institute, Version 3.0.', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'anticipatedPostingDate': '2009-07'}, {'type': 'SECONDARY', 'title': 'Differential Imaging and Biologic Evaluations', 'denoms': [{'units': 'Participants', 'counts': [{'value': '0', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Patients With Osteopetrosis Who Received Transplant', 'description': 'All patients enrolled with osteopetrosis and received transplant.'}], 'timeFrame': 'Day 100, 6 months, 1, 2 and 5 years', 'description': 'These outcome measures were not assessed due to early study termination.', 'reportingStatus': 'POSTED', 'anticipatedPostingDate': '2009-07'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'Patients With Osteopetrosis Who Received Transplant', 'description': 'All patients enrolled with osteopetrosis and received transplant.'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '3'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '3'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}]}]}], 'preAssignmentDetails': 'This study was terminated early due to stopping rules. The first 3 patients enrolled died by Day 100 of study.'}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'BG000'}]}], 'groups': [{'id': 'BG000', 'title': 'Patients With Osteopetrosis Who Received Transplant', 'description': 'All patients enrolled with osteopetrosis and received transplant.'}], 'measures': [{'title': 'Age, Categorical', 'classes': [{'categories': [{'title': '<=18 years', 'measurements': [{'value': '2', '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': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Male', 'measurements': [{'value': '3', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Region of Enrollment', 'classes': [{'title': 'United States', 'categories': [{'measurements': [{'value': '3', 'groupId': 'BG000'}]}]}], 'paramType': 'NUMBER', 'unitOfMeasure': 'participants'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 3}}, 'statusModule': {'whyStopped': 'Excess toxicity', 'overallStatus': 'TERMINATED', 'startDateStruct': {'date': '2007-09'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2017-12', 'completionDateStruct': {'date': '2008-05', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2017-12-03', 'studyFirstSubmitDate': '2008-03-11', 'resultsFirstSubmitDate': '2009-08-26', 'studyFirstSubmitQcDate': '2008-03-18', 'lastUpdatePostDateStruct': {'date': '2017-12-28', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2010-01-18', 'studyFirstPostDateStruct': {'date': '2008-03-19', 'type': 'ESTIMATED'}, 'resultsFirstPostDateStruct': {'date': '2010-02-03', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2008-05', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Number of Patients Achieving Donor Cell Engraftment', 'timeFrame': 'Day 100', 'description': 'Number of patients with persistent presence of donor-derived cells at Day 100'}], 'secondaryOutcomes': [{'measure': 'Number of Patients With Transplant Related Death', 'timeFrame': 'Day 100', 'description': 'Number of participants died during study by Day 100 and reason for death was related to transplant.'}, {'measure': 'Number of Patients With Transplant Related Toxicity', 'timeFrame': 'Day 100', 'description': 'Number of patients experiencing adverse effects due to transplant categorized by body system using Common Terminology Criteria for Adverse Events coding from the National Cancer Institute, Version 3.0.'}, {'measure': 'Differential Imaging and Biologic Evaluations', 'timeFrame': 'Day 100, 6 months, 1, 2 and 5 years', 'description': 'These outcome measures were not assessed due to early study termination.'}]}, 'oversightModule': {'oversightHasDmc': True}, 'conditionsModule': {'keywords': ['osteopetrosis'], 'conditions': ['Osteopetrosis']}, 'descriptionModule': {'briefSummary': 'We believe that hematopoietic stem cell transplantation (HSCT) will help subjects with Osteopetrosis generate functioning osteoclasts, and by so doing assist in the resolution of the abnormal bone architecture, and the anemia and bone marrow failure that is also characteristic of this disease. However, we have found in past studies that approximately 30% of Osteopetrosis patients do not engraft. Therefore, in this study, we plan to use a different combination of pre-transplant drugs to try to make transplants safer for this disease, as well as to provide a second infusion of stem cells in patients with matched related or unrelated donors. The purpose of this research is to find a safer and more effective means of performing stem cell transplantation in patients with Osteopetrosis, using chemotherapy and radiation designed to bring about engraftment and lessen transplant mortality.', 'detailedDescription': 'This transplant protocol will test the following: 1) the ability to achieve engraftment with the reduced intensity protocol, 2) the mortality associated with transplant by day 100, 3) patient outcomes, based on differential imaging and biologic evaluations prior to transplantation and at designated points after transplantation (day 100, 6 months, 1, 2 and 5 years). Additional biologic studies will include microarray analysis, Campath levels just prior to the administration of the graft, and establishment of mesenchymal stem cell lines. In older patients, studies to evaluation osteoclast differentiation and function will also be offered.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT'], 'maximumAge': '45 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Patients eligible for transplantation under this protocol will be \\<45 years of age, and will be diagnosed with severe osteopetrosis. This will be defined as having the following manifestations of the disease.\n* Bones that are uniformly markedly dense based on skeletal survey\n* No history that would suggest autosomal dominant inheritance\n* Evidence of hematologic changes that are attributed to the underlying disease, including the need for ongoing transfusions, OR\n* the presence of progressive anemia or thrombocytopenia, OR a white blood cell differential with a predominance of immature forms and evidence of extramedullary hematopoiesis, OR\n* persistence of serious infectious complications that are thought to be due to the abnormal architecture of the bone that are resistant to surgical and medical interventions.\n\nExclusion Criteria:\n\n* Patients \\>45 years of age\n* Evidence of hepatic failure\n* pulmonary dysfunction sufficient to substantially increase the risk of transplant\n* Renal dysfunction with glomerular filtration rate (GFR) \\<30% of predicted.\n* Cardiac compromise sufficient to substantially increase the risk of transplantation\n* Severe, stable neurologic impairment.\n* Human immunodeficiency virus (HIV) positivity.\n* Pregnant or lactating females'}, 'identificationModule': {'nctId': 'NCT00638820', 'briefTitle': 'Reduced Intensity AlloTransplant For Osteopetrosis', 'organization': {'class': 'OTHER', 'fullName': 'Masonic Cancer Center, University of Minnesota'}, 'officialTitle': 'Reduced Intensity Allogeneic Transplantation For Severe Osteopetrosis Incorporating A Second Cd34 Selected Graft', 'orgStudyIdInfo': {'id': '0704M06581'}, 'secondaryIdInfos': [{'id': 'MT2007-06', 'type': 'OTHER', 'domain': 'Masonic Cancer Center, University of Minnesota'}]}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Intent-To-Treat', 'description': 'Patients enrolled and received study treatment.', 'interventionNames': ['Procedure: Stem Cell or Umbilical Cord Blood Transplantation', 'Drug: Campath, Busulfan, Clofarabine', 'Procedure: Total Lymphoid Irradiation']}], 'interventions': [{'name': 'Stem Cell or Umbilical Cord Blood Transplantation', 'type': 'PROCEDURE', 'otherNames': ['Bone Marrow Transplant'], 'description': 'Stem Cell (unrelated or matched related donor grafts (both peripheral blood and marrow) infusion on Day 0 and 42; Umbilical Cord Blood on Day 0 and 42', 'armGroupLabels': ['Intent-To-Treat']}, {'name': 'Campath, Busulfan, Clofarabine', 'type': 'DRUG', 'otherNames': ['Busulfex, Clolar,Alemtuzumab'], 'description': '* 12 Campath-1H 0.3 mg/kg intravenously (IV) over 2 hours\n* 11 Campath-1H 0.3 mg/kg intravenously over 2 hours\n* 10 Campath-1H 0.3 mg/kg intravenously over 2 hours\n* 9 Busulfan \\<12 kg: 2.2 mg/kg/dose IV every 12 hours \\>12 kg: 1.6 mg/kg/dose IV every 12 hours\n* 8 Busulfan \\<12 kg: 2.2 mg/kg/dose IV every 12 hours \\>12 kg: 1.6 mg/kg/dose IV every 12 hours\n* 7 "Rest"\n* 6 Clofarabine 40 mg/m2 intravenously over 2 hours\n* 5 Clofarabine 40 mg/m2 intravenously over 2 hours\n* 4 Clofarabine 40 mg/m2 intravenously over 2 hours\n* 3 Clofarabine 40 mg/m2 intravenously over 2 hours\n* 2 Clofarabine 40 mg/m2 intravenously over 2 hours', 'armGroupLabels': ['Intent-To-Treat']}, {'name': 'Total Lymphoid Irradiation', 'type': 'PROCEDURE', 'otherNames': ['Therapuetic radation, radiation therapy'], 'description': 'Dose 500 cGy via anteroposterior (AP) and posteroanterior (PA) fields (250 cGy AP and 250 cGy PA).', 'armGroupLabels': ['Intent-To-Treat']}]}, 'contactsLocationsModule': {'locations': [{'zip': '55455', 'city': 'Minneapolis', 'state': 'Minnesota', 'country': 'United States', 'facility': 'University of MInnesota, Fairview', 'geoPoint': {'lat': 44.97997, 'lon': -93.26384}}], 'overallOfficials': [{'name': 'Paul Orchard, MD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Masonic Cancer Center, University of Minnesota'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Masonic Cancer Center, University of Minnesota', 'class': 'OTHER'}, 'responsibleParty': {'oldNameTitle': 'Paul Orchard, M.D.', 'oldOrganization': 'University of Minnesota'}}}}