Safety and Efficacy Study of Autologous Stem Cell Transplantation for Early Onset Type I Diabetes Mellitus - Full Text View

Sponsors and Collaborators:	University of Sao Paulo Northwestern University Genzyme
Information provided by:	University of Sao Paulo
ClinicalTrials.gov Identifier:	NCT00315133

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: Immunosuppresssion and autologous stem cell transplantation	Phase I Phase II

MedlinePlus related topics: Diabetes Diabetes Type 1

U.S. FDA Resources

Study Type:	Interventional
Study Design:	Treatment, Non-Randomized, Open Label, Active Control, Parallel Assignment, Safety/Efficacy Study
Official Title:	Autologous Hematopoietic Stem Cell Transplantation for Early Onset Type 1 Diabetes Mellitus- a Phse I/II Study

Further study details as provided by University of Sao Paulo:

Primary Outcome Measures:

Exogenous insulin dose
C-peptide levels
Hemoglobin A1c
Quality of life

Secondary Outcome Measures:

Anti-GAD titres
Immunologic reconstitution parameters

Estimated Enrollment:	24
Study Start Date:	December 2003
Estimated Study Completion Date:	December 2011

Detailed Description:

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 leukoapheresis 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

Criteria

Inclusion Criteria:

Type 1 diabetes mellitus diagnosed by clinical/metabolic parameters and positive anti-GAD antibodies
Less than 6 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 neoplasic disease

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00315133

Contacts

Contact: Julio C Voltarelli, MD	55-16-2101-9369	jcvoltar@fmrp.usp.br
Contact: Eduardo B Couri, MD	55-16-9149-5151	duducouri@uol.com.br

Locations

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

Sponsors and Collaborators

University of Sao Paulo

Northwestern University

Genzyme

Investigators

Principal Investigator:

Julio C Voltarelli, MD PhD

University Hospital, School of Medicine of Ribeirão Preto, Brazil

More Information

Additional Information:

General information about the institution where the trial is conducted This link exits the ClinicalTrials.gov site

General information about the blood and research center where stem cells are collected and where immunologic studies are performed This link exits the ClinicalTrials.gov site

Publications:

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. Review.

Additional publications automatically indexed to this study by National Clinical Trials Identifier (NCT ID):

Couri CE, Oliveira MC, Stracieri AB, Moraes DA, Pieroni F, Barros GM, Madeira MI, Malmegrim KC, Foss-Freitas MC, Simões 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.

Study ID Numbers:	HCFMRPUSP
Study First Received:	April 13, 2006
Last Updated:	April 13, 2006
ClinicalTrials.gov Identifier:	NCT00315133 History of Changes
Health Authority:	Brazil: Ministry of Health

Keywords provided by University of Sao Paulo:

Diabetes mellitus
Stem cells
Autologous stem cell transplantation
Autoimmune diseases

Study placed in the following topic categories:

Metabolic Diseases
Autoimmune Diseases
Diabetes Mellitus, Type 1
Diabetes Mellitus
Endocrine System Diseases

Diabetes Mellitus Type 1
Endocrinopathy
Glucose Metabolism Disorders
Metabolic Disorder

Additional relevant MeSH terms:

Autoimmune Diseases
Metabolic Diseases
Immune System Diseases
Diabetes Mellitus, Type 1

Diabetes Mellitus
Endocrine System Diseases
Glucose Metabolism Disorders

ClinicalTrials.gov processed this record on May 07, 2009