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

Phase 1

• Conditions: Diabetes Mellitus, Insulin-Dependent
• Interventions: Biological: Intravenous Mesenchymal stem cell infusion

• Registration Number: NCT01322789
• Lead Sponsor: University of Sao Paulo

Brief Summary

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.

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. 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 Timeline

• Study Start: 2008-09
• Status Verified: 2010-11
• Study First Submitted: 2010-12-07
• Study First Submitted QC: 2011-03-24
• Last Update Submitted: 2011-03-24
• Study First Posted: 2011-03-25
• Last Update Posted: 2011-03-25
• Primary Completion: 2015-11
• Study Completion: 2015-12

Recruitment & Eligibility

• Status: UNKNOWN
• Sex: All
• Target Recruitment: 10

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Intravenous mesenchymal stem cell	Intravenous Mesenchymal stem cell infusion	This group wil receive 8 intravenous infusions of mesenchymal stem cells. Four infusions 1 week apart and 4 infusions a month apart

Primary Outcome Measures

Name	Time	Method
AUC C-peptide levels during mixed meal tolerance test	pré-treatment, 6 months, 12 months and then yearly (course of study 7 years)
Safety	Every 6 months until death	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.

Secondary Outcome Measures

Name	Time	Method
Daily insulin use	Daily (course of study is 7 years)
Hemoglobin A1C	3 months
Anti-GAD titres	Every 6 months
Immunologic reconstitution parameters	Yearly (course of study is 7 years)

Trial Locations

Locations (1)

Hospital das Clínicas da Faculdade de Medicina de Ribeirão Preto da Universidade de São Paulo; 🇧🇷 Ribeirão Preto, São Paulo, Brazil