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

Study Overview
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Brief Summary
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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
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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.

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

Conditions
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Diabetes Mellitus, Insulin-Dependent

Intervention / Treatment
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* Biological: Intravenous Mesenchymal stem cell infusion


Participation Criteria
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Eligibility Criteria
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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

Ages Eligible for Study
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Minimum Age: 12 Years
Maximum Age: 35 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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Accepts Healthy Volunteers

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Intravenous mesenchymal stem cell<br>This group wil receive 8 intravenous infusions of mesenchymal stem cells. Four infusions 1 week apart and 4 infusions a month apart | Biological: Intravenous Mesenchymal stem cell infusion<br>* Four consecutive intravenous infusions 1 week apart followed by 4 consecutive infusions 1 month apart<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| AUC C-peptide levels during mixed meal tolerance test |  | 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. | Every 6 months until death | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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) | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Diabetes mellitus, Stem cells, Autologous stem cell transplantation, Autoimmune diseases