Mesenchymal Stem Cell Therapy for Type 1 Diabetes Mellitus Patients

A Preliminary Safety and Efficacy Evaluation of Mesenchymal Stem Cell Transplantation for Type 1 Diabetes Mellitus (T1DM) Patients

The purpose of this study is to evaluate the safety and efficacy of mesenchymal stem cells (MSCs) transplantation for type 1 Diabetes Mellitus patients.

Study Overview

• Status: Unknown
• Conditions: Type 1 Diabetes Mellitus
• Intervention / Treatment: Biological: MSC and PRP

Detailed Description

Mesenchymal stem cells (MSCs) are collected from autologous bone marrow mononuclear cells and allogeneic umbilical cord tissue (UC-MSC).

15 patients with type 1 Diabetes Mellitus will be enrolled and will receive MSCs by intravenous infusion. They were followed up for 6 months after transplantation.

Safety is to assess the occurrence of adverse events (AEs) during either stem cells infusion or by physician assessments.

The primary endpoint is to assess the improvement of patients' Fasting blood glucose, HbA1C, C-peptide, and blood insulin level.

• Study Type: Interventional
• Enrollment (Anticipated): 15
• Phase: Phase 2; Phase 1

Contacts and Locations

• Study Contact: Name: Phuong Le, MSc-MD; Phone Number: (+84)902742732; Email: drbphuong@gmail.com
• Study Contact Backup: Name: Stem Cell Unit, Van Hanh General Hospital; Email: tebaogocvanhanh@gmail.com

Study Locations

• Vietnam
  • Ho Chi Minh
    • Ho Chi Minh City, Ho Chi Minh, Vietnam, 700000
      • Recruiting
      • Van Hanh Geral Hospital
      • Contact: Phuong Le, MSc-MD; Phone Number: (+84)902742732; Email: drbphuong@gmail.com

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 16 years to 43 years (Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• Who is diagnosed with Type 1 Diabetes Mellitus according to the ADA, the patients meet at least one of the following criteria:

  • At least one autoantibody associated with Type 1 Diabetes Mellitus such as ICA or GAD.
  • Previously diagnosed at a medical facility with Type 1 Diabetes.
  • Having evidence of insulin depletion based on the test results during screening.
• Patients treated with fixed insulin dose for at least 3 months.
• Males and females between age 18 and 45 years at the screening.
• Patients able to read, write and understand ICF form

Exclusion Criteria:

• Uncontrolled blood pressure at the time of enrollment: systolic pressure >160 mmHg and/or diastolic blood pressure > 100 mmHg.

• Having evidence related to renal dysfunction:

  • creatinine > 1.5 mg/dl or (>133 mmol/L) for men.
  • creatinine > 1.4 mg/dl or (>124 mmol/L) for woman.
  • eGRF < 40 ml/ min
• Proteinuria > 300 mg/day
• Having evidence of ketoacidosis at the time of selection.
• Having evidence of ongoing or frequent hypoglycemia.
• Having severe infection
• Infected with hepatitis B virus or hepatitis C or tuberculosis. Positive results of HbsAg or Anti HCV or/and PCR tuberculosis are only acceptable in case of vaccination and without suspicious signs. All other cases are not accepted even in the absence of clinical signs.
• Diseases detected before/during screening such as cardiovascular disease, respiratory disease ( pulmonary, fibrosis, chronic respiratory failure), liver disease, cancer, neurology, metabolism.
• Having abnormalities in red blood cells such as sickle cells disease.
• Using alcohol and/or tobacco.
• Blood clotting disorders (INR > 1.5, PTT >40, PT > 15).
• Taking any anticoagulant.
• Taking systemic steroids.
• Participate in another clinical study involving experimenting drugs and/or medical equipment.
• Patients who are unable to perform the tests and assessments needed for the study (eg, patients who are unable to perform bone marrow transplantation) or patients who do not agree to participate in the study.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: MSC and PRP; 15 patients will be given autologous bone marrow-derived mesenchymal stem cells (BM-MSC) and mesenchymal stem cell from allogeneic umbilical cord tissue (UC-MSC) combined with platelet-rich plasma (PRP) by intravenous infusion.	Biological: MSC and PRP; MSC are collected from autologous bone marrow mononuclear cells (BMNC) and allogeneic umbilical cord tissue (UC-MSC) under sterile conditions and combined with activated platelet-rich plasma (PRP) to treat this disease.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Fasting blood glucose	Assess the changes in fasting blood glucose level after transplantation	every month in the course of 6 months
Hemoglobin A1c (HbA1c) level	Assess the changes in HbA1C level after transplantation	1 month, 3 months and 6 months after transplantation
Adverse events	Evaluate the safety of therapy by number record of adverse events (AEs)	during the course of 6 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Insulin dose	Reduction of insulin dose after transplantation	during the course of 6 months
C-peptide	Assess the improvement in C-peptide level after transplantation	every month in the course of 6 months
Blood insulin level	Assess the changes in blood insulin level after transplantation	every month in the course of 6 months

Collaborators and Investigators

• Sponsor: Van Hanh General Hospital
• Collaborators: University of Science Ho Chi Minh City
• Investigators: Principal Investigator: Phuong Le, MSc-MD, Stem Cell Unit, Van Hanh General Hospital; Principal Investigator: Ngoc Phan, MSc, Stem Cell Institute, University of Science Ho Chi Minh City

Study record dates

Study Major Dates

• Study Start (Actual): April 1, 2017
• Primary Completion (Anticipated): November 1, 2018
• Study Completion (Anticipated): November 1, 2018

Study Registration Dates

• First Submitted: March 16, 2018
• First Submitted That Met QC Criteria: March 25, 2018
• First Posted (Actual): April 2, 2018

Study Record Updates

• Last Update Posted (Actual): April 4, 2018
• Last Update Submitted That Met QC Criteria: April 2, 2018
• Last Verified: April 1, 2018

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Glucose Metabolism Disorders; Metabolic Diseases; Immune System Diseases; Autoimmune Diseases; Endocrine System Diseases; Diabetes Mellitus; Diabetes Mellitus, Type 1
• Other Study ID Numbers: DIME 1001 (Other Identifier: Ministry of Health, Viet Nam)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: Yes
• IPD Sharing Time Frame: 6 months after publication
• IPD Sharing Access Criteria: Type 1 Diabetes Mellitus
• IPD Sharing Supporting Information Type: Study Protocol; Clinical Study Report (CSR)

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No