Ignite Creation Date:
2025-12-25 @ 2:44 AM
Ignite Modification Date:
2025-12-26 @ 1:23 AM
Study NCT ID:
NCT00315133
Status:
COMPLETED
Last Update Posted:
2017-01-18
First Post:
2006-04-13
Is NOT Gene Therapy:
False
Has Adverse Events:
False
Brief Title:
Safety and Efficacy Study of Autologous Stem Cell Transplantation for Early Onset Type I Diabetes Mellitus
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D003922', 'term': 'Diabetes Mellitus, Type 1'}, {'id': 'D003920', 'term': 'Diabetes Mellitus'}, {'id': 'D001327', 'term': 'Autoimmune Diseases'}], 'ancestors': [{'id': 'D044882', 'term': 'Glucose Metabolism Disorders'}, {'id': 'D008659', 'term': 'Metabolic Diseases'}, {'id': 'D009750', 'term': 'Nutritional and Metabolic Diseases'}, {'id': 'D004700', 'term': 'Endocrine System Diseases'}, {'id': 'D007154', 'term': 'Immune System Diseases'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'D007165', 'term': 'Immunosuppression Therapy'}], 'ancestors': [{'id': 'D007167', 'term': 'Immunotherapy'}, {'id': 'D056747', 'term': 'Immunomodulation'}, {'id': 'D001691', 'term': 'Biological Therapy'}, {'id': 'D013812', 'term': 'Therapeutics'}, {'id': 'D007158', 'term': 'Immunologic Techniques'}, {'id': 'D008919', 'term': 'Investigative Techniques'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE1', 'PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 25}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2003-12'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2017-01', 'completionDateStruct': {'date': '2014-12', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2017-01-13', 'studyFirstSubmitDate': '2006-04-13', 'studyFirstSubmitQcDate': '2006-04-13', 'lastUpdatePostDateStruct': {'date': '2017-01-18', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2006-04-17', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2010-12', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'C-peptide levels', 'timeFrame': 'Every 6 months', 'description': 'Stimulated C-peptide levels will be measured.'}], 'secondaryOutcomes': [{'measure': 'Transplant-related toxicity', 'timeFrame': 'Every 6 months or when reported'}, {'measure': 'Anti-GAD titres', 'timeFrame': 'Every 6 months'}, {'measure': 'Exogenous insulin dose', 'timeFrame': 'Every 6 months', 'description': 'Number of international insulin units per kilogram per day in use will be registered.'}, {'measure': 'Hemoglobin A1C', 'timeFrame': 'Every 6 months', 'description': 'Hb A1C will be measured.'}, {'measure': 'Immunologic reconstitution parameters', 'timeFrame': 'Yearly'}, {'measure': 'Quality of Life', 'timeFrame': 'Every year', 'description': 'SF-36 questionnaire'}]}, 'oversightModule': {'oversightHasDmc': True}, 'conditionsModule': {'keywords': ['Diabetes mellitus', 'Stem cells', 'Autologous stem cell transplantation', 'Autoimmune diseases'], 'conditions': ['Type 1 Diabetes Mellitus']}, 'referencesModule': {'references': [{'pmid': '12849006', 'type': 'BACKGROUND', 'citation': 'Burt RK, Oyama Y, Traynor A, Kenyon NS. Hematopoietic stem cell therapy for type 1 diabetes: induction of tolerance and islet cell neogenesis. Autoimmun Rev. 2002 May;1(3):133-8. doi: 10.1016/s1568-9972(02)00033-2.'}, {'pmid': '17426276', 'type': 'RESULT', 'citation': 'Voltarelli JC, Couri CE, Stracieri AB, Oliveira MC, Moraes DA, Pieroni F, Coutinho M, Malmegrim KC, Foss-Freitas MC, Simoes BP, Foss MC, Squiers E, Burt RK. Autologous nonmyeloablative hematopoietic stem cell transplantation in newly diagnosed type 1 diabetes mellitus. JAMA. 2007 Apr 11;297(14):1568-76. doi: 10.1001/jama.297.14.1568.'}, {'pmid': '19366777', 'type': 'RESULT', 'citation': 'Couri CE, Oliveira MC, Stracieri AB, Moraes DA, Pieroni F, Barros GM, Madeira MI, Malmegrim KC, Foss-Freitas MC, Simoes BP, Martinez EZ, Foss MC, Burt RK, Voltarelli JC. C-peptide levels and insulin independence following autologous nonmyeloablative hematopoietic stem cell transplantation in newly diagnosed type 1 diabetes mellitus. JAMA. 2009 Apr 15;301(15):1573-9. doi: 10.1001/jama.2009.470.'}]}, 'descriptionModule': {'briefSummary': 'The study evaluates the effect of inactivation of the immune system with chemotherapy and immunotherapy and infusion of bone marrow stem cells in early onset type 1 diabetes mellitus. We hypothesize that reprograming the immune system will stop immune aggression to the insulin producing cells allowing their regeneration and thus decreasing or eliminating the need of exogenous insulin.', 'detailedDescription': 'Patients from 12 to 35 years old with type I diabetes mellitus proved by anti-pancreatic beta cell antibodies and recently diagnosed (less than 6 weeks) will be included in this study. Peripheral blood hematopoietic stem cells will be mobilized from bone marrow of the patient with cyclophosphamide plus G-CSF (granulocyte-colony stimulating factor), collected by leukapheresis and cryopreserved. After 2-3 weeks, high dose immunosuppression is given (cyclophosphamide 200 mg/kg plus rabbit antithymocyte globulin 4.5 mg/kg) and stem cells are thawed and injected intravenously. This procedure is performed in isolated rooms at the Bone Marrow Transplantation Unit of the School of Medicine of Ribeirão Preto, University of São Paulo, Brazil. Patients are discharged from the hospital after engraftment and closely followed up to 2 months after transplantation (with at least weekly outpatient visits) and continue the followup for 5 years after transplantation. Clinical, hematological, metabolical and immunological evaluations are performed to analyse the effect of the transplant in the disease and in the hematopoetic and immunologic systems of the body. Patients fitting the inclusion criteria but not agreeing to perform the transplantation are the control group and they will be followed in parallel with transplanted patients.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT'], 'maximumAge': '35 Years', 'minimumAge': '12 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Type 1 diabetes mellitus diagnosed by clinical/metabolic parameters and positive anti-GAD antibodies\n* Less than 12 weeks from diagnosis\n\nExclusion Criteria:\n\n* Previous diabetic ketoacidosis\n* Pregnancy\n* Severe psychiatric disorder\n* Severe organic impairment (renal, hepatic, cardiac, pulmonary)\n* Active infectious disease\n* Previous or present neoplastic disease'}, 'identificationModule': {'nctId': 'NCT00315133', 'briefTitle': 'Safety and Efficacy Study of Autologous Stem Cell Transplantation for Early Onset Type I Diabetes Mellitus', 'organization': {'class': 'OTHER', 'fullName': 'University of Sao Paulo'}, 'officialTitle': 'Autologous Hematopoietic Stem Cell Transplantation for Early Onset Type 1 Diabetes Mellitus- a Phase I/II Study', 'orgStudyIdInfo': {'id': 'HCFMRPUSP'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Transplant arm', 'description': 'This is a single arm study. The intervention is immunosuppression and autologous stem cell transplantation.', 'interventionNames': ['Procedure: Immunosuppression and autologous stem cell transplantation']}], 'interventions': [{'name': 'Immunosuppression and autologous stem cell transplantation', 'type': 'PROCEDURE', 'description': 'Immunosuppression and autologous stem cell transplantation: Mobilization of hematopoietic stem cells (HSC) with cyclophosphamide (2 g/m2) and granulocyte-colony stimulating factor (G-CSF, 10 ug/kg/d), followed by collection and cryopreservation of unselected HSC and conditioning with cyclophosphamide (200 mg/kg) plus rabbit anti-thymocyte globulin (ATG 4.5 mg/kg).', 'armGroupLabels': ['Transplant arm']}]}, 'contactsLocationsModule': {'locations': [{'zip': '14048-900', 'city': 'Ribeirão Preto', 'state': 'Ribeirão Preto', 'country': 'Brazil', 'facility': 'Hospital das Clínicas da Faculdade de Medicina de Ribeirão Preto da Universidade de São Paulo', 'geoPoint': {'lat': -21.1775, 'lon': -47.81028}}], 'overallOfficials': [{'name': 'Julio C Voltarelli, MD PhD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'University Hospital, School of Medicine of Ribeirão Preto, Brazil'}, {'name': 'Maria C Oliveira, MD PhD', 'role': 'STUDY_CHAIR', 'affiliation': 'University Hospital, Ribeirão Preto Medical School, Brazil'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'UNDECIDED', 'description': 'IPD may be shared if the responsible investigators feel it may increase understanding, reliability and reproducibility of the present study.'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'University of Sao Paulo', 'class': 'OTHER'}, 'collaborators': [{'name': 'Northwestern University', 'class': 'OTHER'}, {'name': 'Genzyme, a Sanofi Company', 'class': 'INDUSTRY'}], 'responsibleParty': {'type': 'PRINCIPAL_INVESTIGATOR', 'investigatorTitle': 'Julio C. Voltarelli', 'investigatorFullName': 'Julio C. Voltarelli', 'investigatorAffiliation': 'University of Sao Paulo'}}}}