Safety and Efficacy Study of Autologous Stem Cell Transplantation for Early Onset Type I Diabetes Mellitus
Recruitment status was Recruiting
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Purpose
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.
| Condition | Intervention | Phase |
|---|---|---|
|
Type 1 Diabetes Mellitus |
Procedure: Immunosuppression and autologous stem cell transplantation Procedure: Autologous hematopoietic stem cell transplantation |
Phase 1 Phase 2 |
| Study Type: | Interventional |
| Study Design: | Allocation: Non-Randomized Endpoint Classification: Safety/Efficacy Study Intervention Model: Parallel Assignment Masking: Open Label Primary Purpose: Treatment |
| Official Title: | Autologous Hematopoietic Stem Cell Transplantation for Early Onset Type 1 Diabetes Mellitus- a Phase I/II Study |
- Exogenous insulin dose [ Time Frame: Daily ] [ Designated as safety issue: Yes ]
- C-peptide levels [ Time Frame: Each 6 months ] [ Designated as safety issue: No ]
- Hemoglobin A1c [ Time Frame: Each 3 months ] [ Designated as safety issue: No ]
- Quality of life [ Time Frame: Each 6 months ] [ Designated as safety issue: Yes ]
- Anti-GAD titres [ Time Frame: Each 6 months ] [ Designated as safety issue: No ]
- Immunologic reconstitution parameters [ Time Frame: Yearly ] [ Designated as safety issue: No ]
| Estimated Enrollment: | 24 |
| Study Start Date: | December 2003 |
| Estimated Study Completion Date: | December 2012 |
| Estimated Primary Completion Date: | December 2010 (Final data collection date for primary outcome measure) |
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Procedure: Immunosuppression and autologous stem cell transplantation
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, 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.
Eligibility| Ages Eligible for Study: | 12 Years to 35 Years |
| Genders Eligible for Study: | Both |
| 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
Contacts and Locations| Contact: Julio C Voltarelli, MD | 55-16-2101-9369 | jcvoltar@fmrp.usp.br |
| Contact: Eduardo B Couri, MD | 55-16-9149-5151 | ce.couri@yahoo.com.br |
| Brazil | |
| Hospital das Clínicas da Faculdade de Medicina de Ribeirão Preto da Universidade de São Paulo | Recruiting |
| Ribeirão Preto, Brazil, 14048-900 | |
| Contact: Julio C Voltarelli, MD PhD 55-16-2101-9369 jcvoltar@fmrp.usp.br | |
| Principal Investigator: Julio C Voltarelli, MD PhD | |
| Principal Investigator: | Julio C Voltarelli, MD PhD | University Hospital, School of Medicine of Ribeirão Preto, Brazil |
More Information
Additional Information:
Publications:
Additional publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
| Responsible Party: | Julio Voltarelli MD PhD, University of São Paulo, School of Medicine of Ribeirão Preto |
| ClinicalTrials.gov Identifier: | NCT00315133 History of Changes |
| Other Study ID Numbers: | HCFMRPUSP |
| Study First Received: | April 13, 2006 |
| Last Updated: | November 15, 2010 |
| Health Authority: | Brazil: Ministry of Health |
Keywords provided by 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 |
ClinicalTrials.gov processed this record on May 23, 2013