Ignite Creation Date:
2025-12-24 @ 2:33 PM
Ignite Modification Date:
2026-02-23 @ 12:21 PM
Study NCT ID:
NCT02179359
Status:
TERMINATED
Last Update Posted:
2025-03-03
First Post:
2014-06-27
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Hematopoietic Stem Cell Transplant for High Risk Hemoglobinopathies
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D000755', 'term': 'Anemia, Sickle Cell'}, {'id': 'D017086', 'term': 'beta-Thalassemia'}, {'id': 'D029503', 'term': 'Anemia, Diamond-Blackfan'}, {'id': 'D006457', 'term': 'Hemoglobinuria, Paroxysmal'}, {'id': 'D013915', 'term': 'Thrombasthenia'}, {'id': 'C537592', 'term': 'Neutropenia, Severe Congenital, Autosomal Recessive 3'}, {'id': 'D000081003', 'term': 'Shwachman-Diamond Syndrome'}], 'ancestors': [{'id': 'D000745', 'term': 'Anemia, Hemolytic, Congenital'}, {'id': 'D000743', 'term': 'Anemia, Hemolytic'}, {'id': 'D000740', 'term': 'Anemia'}, {'id': 'D006402', 'term': 'Hematologic Diseases'}, {'id': 'D006425', 'term': 'Hemic and Lymphatic Diseases'}, {'id': 'D006453', 'term': 'Hemoglobinopathies'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D013789', 'term': 'Thalassemia'}, {'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'}, {'id': 'D009190', 'term': 'Myelodysplastic Syndromes'}, {'id': 'D025861', 'term': 'Blood Coagulation Disorders, Inherited'}, {'id': 'D001778', 'term': 'Blood Coagulation Disorders'}, {'id': 'D001791', 'term': 'Blood Platelet Disorders'}, {'id': 'D006474', 'term': 'Hemorrhagic Disorders'}, {'id': 'D010188', 'term': 'Exocrine Pancreatic Insufficiency'}, {'id': 'D010182', 'term': 'Pancreatic Diseases'}, {'id': 'D004066', 'term': 'Digestive System Diseases'}, {'id': 'D008052', 'term': 'Lipid Metabolism, Inborn Errors'}, {'id': 'D052439', 'term': 'Lipid Metabolism Disorders'}, {'id': 'D008659', 'term': 'Metabolic Diseases'}, {'id': 'D009750', 'term': 'Nutritional and Metabolic Diseases'}, {'id': 'D008068', 'term': 'Lipomatosis'}]}}, 'protocolSection': {'designModule': {'phases': ['NA'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NON_RANDOMIZED', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 38}}, 'statusModule': {'whyStopped': 'Replaced by another study', 'overallStatus': 'TERMINATED', 'startDateStruct': {'date': '2014-09-02'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-02', 'completionDateStruct': {'date': '2024-11-21', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2025-02-27', 'studyFirstSubmitDate': '2014-06-27', 'studyFirstSubmitQcDate': '2014-06-27', 'lastUpdatePostDateStruct': {'date': '2025-03-03', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2014-07-01', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2024-11-21', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'incidence of graft failure', 'timeFrame': '42 days'}], 'secondaryOutcomes': [{'measure': 'overall survival', 'timeFrame': '6 months, 1 and 2 years'}, {'measure': 'disease free survival', 'timeFrame': '6 months, 1 and 2 years', 'description': 'patient no longer needing red blood cell transfusion and/or a hemoglobin S level at that of the donor ( sickle cell disease only)'}]}, 'oversightModule': {'isUsExport': False, 'oversightHasDmc': True, 'isFdaRegulatedDrug': False, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['Stem Cell Transplant'], 'conditions': ['Sickle Cell Disease', 'Transfusion Dependent Alpha- or Beta- Thalassemia', 'Diamond Blackfan Anemia', 'Paroxysmal Nocturnal Hemoglobinuria', 'Glanzmann Thrombasthenia', 'Severe Congenital Neutropenia', 'Shwachman-Diamond Syndrome', 'Non-Malignant Hematologic Disorders']}, 'descriptionModule': {'briefSummary': 'This is a study to collect the outcomes of stem cell transplantation for patients with hematologic diseases other than cancer.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT'], 'maximumAge': '55 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Diagnosis of Sickle Cell Disease, Thalassemia, Diamond Blackfan Anemia or other non-malignant hematologic disorders for which a stem cell transplant is indicated\n* Acceptable stem cell source identified\n* Performance status of ≥ 70% (Karnofsky),or ≥ 70 (Lansky play score)\n* Creatinine \\<2.0 mg/dl for adults or glomerular filtration rate \\> 50 ml/min for children\n* Bilirubin, Aspartate Aminotransferase, Alkaline phosphatase \\<5 times the upper limit of institutional normal\n* Absence of decompensated congestive heart failure, or uncontrolled arrhythmia and left ventricular ejection fraction \\> 40%\n\nExclusion Criteria:\n\n* active, uncontrolled infection\n* pregnant or breastfeeding\n* HIV positive'}, 'identificationModule': {'nctId': 'NCT02179359', 'briefTitle': 'Hematopoietic Stem Cell Transplant for High Risk Hemoglobinopathies', 'organization': {'class': 'OTHER', 'fullName': 'Masonic Cancer Center, University of Minnesota'}, 'officialTitle': 'MT2014-10C: Allogeneic Hematopoietic Stem Cell Transplant for Patients With High Risk Hemoglobinopathies and Other Red Cell Transfusion Dependent Disorders', 'orgStudyIdInfo': {'id': '2014OC034'}, 'secondaryIdInfos': [{'id': 'MT2014-10C', 'type': 'OTHER', 'domain': 'Masonic Cancer Center, University of Minnesota'}]}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Reduced Toxicity Ablative Regimen', 'description': 'For use in patients with a matched sibling donor or unrelated UCB donor and DBA patients who are \\<12 years and/or have mild/moderate iron exposure.', 'interventionNames': ['Drug: Reduced Toxicity Ablative Regimen']}, {'type': 'EXPERIMENTAL', 'label': 'Reduced Intensity Preparative Regimen', 'description': 'For use in patients with unrelated donor bone marrow and for DBA patients who are \\>12 years and/or have significant iron exposure.', 'interventionNames': ['Drug: Reduced Intensity Preparative Regimen']}, {'type': 'EXPERIMENTAL', 'label': 'Myeloablative Preparative Regimen', 'description': 'For use in patients with a matched sibling donor, unrelated umbilical cord blood and in those with severe thalassemia.', 'interventionNames': ['Drug: Myeloablative Preparative Regimen']}], 'interventions': [{'name': 'Reduced Toxicity Ablative Regimen', 'type': 'DRUG', 'description': '* Anti-thymocyte Globulin (ATG)\n* Fludarabine\n* Busulfan\n* Stem Cell Infusion Day 0', 'armGroupLabels': ['Reduced Toxicity Ablative Regimen']}, {'name': 'Reduced Intensity Preparative Regimen', 'type': 'DRUG', 'description': '* Alemtuzumab\n* Cyclophosphamide\n* Fludarabine\n* Total Body Irradiation (TBI)\n* Stem Cell Infusion Day 0', 'armGroupLabels': ['Reduced Intensity Preparative Regimen']}, {'name': 'Myeloablative Preparative Regimen', 'type': 'DRUG', 'description': '* Alemtuzumab\n* Cyclophosphamide\n* Busulfan\n* Stem Cell Infusion Day 0', 'armGroupLabels': ['Myeloablative Preparative Regimen']}]}, 'contactsLocationsModule': {'locations': [{'zip': '55455', 'city': 'Minneapolis', 'state': 'Minnesota', 'country': 'United States', 'facility': 'University of Minnesota Medical Center, Fairview', 'geoPoint': {'lat': 44.97997, 'lon': -93.26384}}], 'overallOfficials': [{'name': 'Ashish Gupta, MBBS, MPH', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Masonic Cancer Center, University of Minnesota'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Masonic Cancer Center, University of Minnesota', 'class': 'OTHER'}, 'responsibleParty': {'type': 'SPONSOR'}}}}