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

University of Sao Paulo

Status and phase: Completed; Phase 2; Phase 1

Conditions: Type 1 Diabetes Mellitus

Treatments: Procedure: Immunosuppression and autologous stem cell transplantation

Study type: Interventional

Funder types: Other; Industry

Identifiers

NCT00315133

HCFMRPUSP

Details and patient eligibility

About

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.

Full 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 (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.

Enrollment: 25 patients

Sex: All

Ages: 12 to 35 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

---

25 participants in 1 patient group

Transplant arm

Experimental group

Description:

This is a single arm study. The intervention is immunosuppression and autologous stem cell transplantation.

Treatment:

Procedure: Immunosuppression and autologous stem cell transplantation