Safety and Efficacy Study of Autologous Stem Cell Transplantation for Early Onset Type I Diabetes Mellitus
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT00315133|
Recruitment Status : Completed
First Posted : April 17, 2006
Last Update Posted : January 18, 2017
|Condition or disease||Intervention/treatment||Phase|
|Type 1 Diabetes Mellitus||Procedure: Immunosuppression and autologous stem cell transplantation||Phase 1 Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||25 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Autologous Hematopoietic Stem Cell Transplantation for Early Onset Type 1 Diabetes Mellitus- a Phase I/II Study|
|Study Start Date :||December 2003|
|Actual Primary Completion Date :||December 2010|
|Actual Study Completion Date :||December 2014|
Experimental: Transplant arm
This is a single arm study. The intervention is immunosuppression and autologous stem cell transplantation.
Procedure: Immunosuppression and autologous stem cell transplantation
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).
- C-peptide levels [ Time Frame: Every 6 months ]Stimulated C-peptide levels will be measured.
- Transplant-related toxicity [ Time Frame: Every 6 months or when reported ]
- Anti-GAD titres [ Time Frame: Every 6 months ]
- Exogenous insulin dose [ Time Frame: Every 6 months ]Number of international insulin units per kilogram per day in use will be registered.
- Hemoglobin A1C [ Time Frame: Every 6 months ]Hb A1C will be measured.
- Immunologic reconstitution parameters [ Time Frame: Yearly ]
- Quality of Life [ Time Frame: Every year ]SF-36 questionnaire
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00315133
|Hospital das Clínicas da Faculdade de Medicina de Ribeirão Preto da Universidade de São Paulo|
|Ribeirão Preto, Brazil, 14048-900|
|Principal Investigator:||Julio C Voltarelli, MD PhD||University Hospital, School of Medicine of Ribeirão Preto, Brazil|
|Study Chair:||Maria C Oliveira, MD PhD||University Hospital, Ribeirão Preto Medical School, Brazil|