Safety and Efficacy Study of Autologous Stem Cell Transplantation for Early Onset Type I Diabetes Mellitus
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ClinicalTrials.gov Identifier: NCT00315133 |
Recruitment Status :
Completed
First Posted : April 17, 2006
Last Update Posted : January 18, 2017
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Condition or disease | Intervention/treatment | Phase |
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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) |
Primary Purpose: | Treatment |
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 |

Arm | Intervention/treatment |
---|---|
Experimental: Transplant arm
This is a single arm study. The intervention is immunosuppression and autologous stem cell transplantation.
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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

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Ages Eligible for Study: | 12 Years to 35 Years (Child, Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- Type 1 diabetes mellitus diagnosed by clinical/metabolic parameters and positive anti-GAD antibodies
- Less than 12 weeks from diagnosis
Exclusion Criteria:
- Previous diabetic ketoacidosis
- Pregnancy
- Severe psychiatric disorder
- Severe organic impairment (renal, hepatic, cardiac, pulmonary)
- Active infectious disease
- Previous or present neoplastic disease

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
Brazil | |
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 |
Additional Information:


Publications of Results:
Other Publications:
Responsible Party: | Julio C. Voltarelli, University of Sao Paulo |
ClinicalTrials.gov Identifier: | NCT00315133 History of Changes |
Other Study ID Numbers: |
HCFMRPUSP |
First Posted: | April 17, 2006 Key Record Dates |
Last Update Posted: | January 18, 2017 |
Last Verified: | January 2017 |
Individual Participant Data (IPD) Sharing Statement: | |
Plan to Share IPD: | Undecided |
Plan Description: | IPD may be shared if the responsible investigators feel it may increase understanding, reliability and reproducibility of the present study. |
Keywords provided by Julio C. Voltarelli, University of Sao Paulo:
Diabetes mellitus Stem cells Autologous stem cell transplantation Autoimmune diseases |
Additional relevant MeSH terms:
Diabetes Mellitus Diabetes Mellitus, Type 1 Glucose Metabolism Disorders Metabolic Diseases |
Endocrine System Diseases Autoimmune Diseases Immune System Diseases |