Safety and Efficacy of Mesenchymal Stem Cells in Newly-diagnosed Type 1 Diabetic Patients

University of Sao Paulo10 enrolled

Overview

Type 1 diabetes mellitus results from the autoimmune destruction of the insulin producing pancreatic β-cells. The autoimmune response begins months or even years before the presentation of hyperglycemic symptoms. Previous studies with other autoimmune diseases or acute inflammatory diseases testing the effect of the infusion of mesenchymal stem cells showed promising results in regulating immune system and promoting some degree of disease control. The aim of our study is to determine the safety and efficacy of intravenous infusions of mesenchymal stem cells in newly diagnosed type 1 diabetic patients.

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. First, bone marrow derived adult mesenchymal stem cells are collected from a first degree relative and cultured. After that, the patient receive 4 intravenous infusions 1 week apart followed by 4 infusion 4 months apart.

Study Type

INTERVENTIONAL

Allocation

NON_RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Diabetes Mellitus, Insulin-Dependent

Interventions

Eligibility

Sex: ALLMin age: 12 YearsMax age: 35 YearsHealthy volunteers:

Medical Language ↔ Plain English

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 neoplastic disease

Outcomes

Primary Outcomes

AUC C-peptide levels during mixed meal tolerance test

Time frame: pré-treatment, 6 months, 12 months and then yearly (course of study 7 years)

Safety

The process of analyzing safety data is made daily based on clinical interview, frequent physical examination and general laboratory findings weekly from the first stem cell infusion until 60 months after the last infusion. Chest X-ray will be performed in days 100, 180, 270, 360 after the lest infusion and then every 6 months. Fecal occult blood, alpha pheto protein, beta-human chorionic gonadotropin, carcino-embrionary antigen, abdomen ultrasound will be performed in month 6 and 12 after the last infusion and then yearly.

Time frame: Every 6 months until death

Secondary Outcomes

Daily insulin use

Time frame: Daily (course of study is 7 years)

Hemoglobin A1C

Time frame: 3 months

Anti-GAD titres

Time frame: Every 6 months

Immunologic reconstitution parameters

Time frame: Yearly (course of study is 7 years)