Clinical trial NCT03484741: Mesenchymal Stem Cell Therapy for Type 1 Diabetes Mellitus Patients
Official title: A Preliminary Safety and Efficacy Evaluation of Mesenchymal Stem Cell Transplantation for Type 1 Diabetes Mellitus (T1DM) Patients
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE1
PHASE2
Conditions: Type 1 Diabetes Mellitus
Lead sponsor: Van Hanh General Hospital
Sponsor class: OTHER
Collaborators: name: University of Science Ho Chi Minh City
First posted: 2018-04-02
Enrollment: 15
Sex: ALL
Minimum age (years): 18
Maximum age (years): 45
Standard ages: ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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

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.

Eligibility criteria: 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.

Primary outcomes:
measure: Fasting blood glucose
timeFrame: every month in the course of 6 months
measure: Hemoglobin A1c (HbA1c) level
timeFrame: 1 month, 3 months and 6 months after transplantation
measure: Adverse events
timeFrame: during the course of 6 months
Secondary outcomes:
measure: Insulin dose
timeFrame: during the course of 6 months
measure: C-peptide
timeFrame: every month in the course of 6 months
measure: Blood insulin level
timeFrame: every month in the course of 6 months
Other outcomes:
Locations:
city: Ho Chi Minh City
country: Vietnam
facility: Van Hanh Geral Hospital
state: Ho Chi Minh
Study officials:
affiliation: Stem Cell Unit, Van Hanh General Hospital
name: Phuong Le, MSc-MD
role: PRINCIPAL_INVESTIGATOR
affiliation: Stem Cell Institute, University of Science Ho Chi Minh City
name: Ngoc Phan, MSc
role: PRINCIPAL_INVESTIGATOR
References: