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

The recruitment status of this study is unknown because the information has not been verified recently.
Verified November 2010 by University of Sao Paulo.
Recruitment status was  Recruiting
Sponsor:
Collaborators:
Northwestern University
Genzyme, a Sanofi Company
Information provided by:
University of Sao Paulo
ClinicalTrials.gov Identifier:
NCT00315133
First received: April 13, 2006
Last updated: November 15, 2010
Last verified: November 2010

April 13, 2006
November 15, 2010
December 2003
December 2010   (final data collection date for primary outcome measure)
  • 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 ]
  • Exogenous insulin dose
  • C-peptide levels
  • Hemoglobin A1c
  • Quality of life
Complete list of historical versions of study NCT00315133 on ClinicalTrials.gov Archive Site
  • Anti-GAD titres [ Time Frame: Each 6 months ] [ Designated as safety issue: No ]
  • Immunologic reconstitution parameters [ Time Frame: Yearly ] [ Designated as safety issue: No ]
  • 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, 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
Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Open Label
Primary Purpose: Treatment
Type 1 Diabetes Mellitus
  • Procedure: Immunosuppression and autologous stem cell transplantation
    Mobilization of hematopoietic stem cells (HSC) with cyclophosphamide (2 g/m2) and G-CSF (10 ug/kg/d), followed by collection and freezing of HSC and conditioning with cyclophosphamide (200 mg/kg) plus rabbit ATG (4,5 mg/kg).
  • Procedure: Autologous hematopoietic stem cell transplantation
    Mobilization of hematopoietic stem cells (HSC) to peripheral blood with G-CSF (10 ug/kg/d) and cyclophosphamide (2 g/m2) followed by collection and freezing of HSC and high dose chemotherapy with cyclophosphamide again (200 mg/kg) plus rabbit antithymocyte globulin (4,5 mg/kg).
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruiting
24
December 2012
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
Both
12 Years to 35 Years
No
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
 
NCT00315133
HCFMRPUSP
Not Provided
Julio Voltarelli MD PhD, University of São Paulo, School of Medicine of Ribeirão Preto
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
University of Sao Paulo
November 2010

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