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

This study has been completed.
Sponsor:
ClinicalTrials.gov Identifier:
NCT00315133
First Posted: April 17, 2006
Last Update Posted: January 18, 2017
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.
Collaborators:
Northwestern University
Genzyme, a Sanofi Company
Information provided by (Responsible Party):
Julio C. Voltarelli, University of Sao Paulo
April 13, 2006
April 17, 2006
January 18, 2017
December 2003
December 2010   (Final data collection date for primary outcome measure)
C-peptide levels [ Time Frame: Every 6 months ]
Stimulated C-peptide levels will be measured.
  • Exogenous insulin dose
  • C-peptide levels
  • Hemoglobin A1c
  • Quality of life
Complete list of historical versions of study NCT00315133 on ClinicalTrials.gov Archive Site
  • 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
  • Anti-GAD titres
  • Immunologic reconstitution parameters
Not Provided
Not Provided
 
Safety and Efficacy Study of Autologous Stem Cell Transplantation for Early Onset Type I Diabetes Mellitus
Autologous Hematopoietic Stem Cell Transplantation for Early Onset Type 1 Diabetes Mellitus- a Phase I/II Study
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.
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.
Interventional
Phase 1
Phase 2
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Type 1 Diabetes Mellitus
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).
Experimental: Transplant arm
This is a single arm study. The intervention is immunosuppression and autologous stem cell transplantation.
Intervention: Procedure: Immunosuppression and autologous stem cell transplantation

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Completed
25
December 2014
December 2010   (Final data collection date for primary outcome measure)

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
Sexes Eligible for Study: All
12 Years to 35 Years   (Child, Adult)
No
Contact information is only displayed when the study is recruiting subjects
Brazil
 
 
NCT00315133
HCFMRPUSP
Yes
Not Provided
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.
Julio C. Voltarelli, University of Sao Paulo
University of Sao Paulo
  • Northwestern University
  • Genzyme, a Sanofi Company
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
University of Sao Paulo
January 2017

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP