Safety Study of Mesenchymal Precursor Cells in Type 2 Diabetes

A Randomized, Placebo-Controlled Dose-Escalation Study to Assess the Safety and Tolerability of a Single Intravenous Infusion of Allogeneic Mesenchymal Precursor Cells (MPCs) in Patients With Type 2 Diabetes Sub-optimally Controlled on Metformin

This study is being conducted to assess the overall safety and tolerability of a single intravenous infusion of three doses of Mesenchymal Precursor Cells versus Placebo in subjects with Type 2 Diabetes inadequately controlled on Metformin.

Study Overview

• Status: Completed
• Conditions: Type 2 Diabetes
• Intervention / Treatment: Drug: Mesenchymal Precursor Cells (MPCs); Drug: Mesenchymal Precursor Cells (MPCs); Drug: Mesenchymal Precursor Cells (MPCs)

• Study Type: Interventional
• Enrollment (Actual): 61
• Phase: Phase 2; Phase 1

Contacts and Locations

Study Locations

• United States
  • California
    • Garden Grove, California, United States, 92844
      • SC Clinical Research
  • Florida
    • Miami, Florida, United States, 33136
      • Diabetes Research Institute
    • Orlando, Florida, United States, 32806
      • Compass Research
  • Louisiana
    • Baton Rouge, Louisiana, United States, 70808
      • Pennington Biomedical Research Center
    • New Orleans, Louisiana, United States, 70112
      • Tulane University Medical Center
  • Montana
    • Butte, Montana, United States, 59701
      • Big Sky Clinical Research
  • Nevada
    • Henderson, Nevada, United States, 89052
      • Desert Endocrinology Clinical Research Center-Henderson
    • Las Vegas, Nevada, United States, 89101
      • Alliance Against Diabetes/AAD Clinical Research
  • New Hampshire
    • Newington, New Hampshire, United States, 03801
      • Active Practices and Research
  • Ohio
    • Cincinnati, Ohio, United States, 45219
      • The Carl and Edyth Lindner Center for Research and Education at The Christ Hospital
    • Dayton, Ohio, United States, 45439
      • Providence Health Partners - Center for Clinical Reseach
  • Texas
    • Dallas, Texas, United States, 75230
      • Dallas Diabetes and Endocrine Center
    • Houston, Texas, United States, 77055
      • West Houston Clinical Research Services
    • San Antonio, Texas, United States, 78205
      • Paragon Research Center
  • Utah
    • Salt Lake City, Utah, United States, 84107
      • Wasatch Clinical Research
  • Virginia
    • Norfolk, Virginia, United States, 23502
      • National Clinical Research - Norfolk, Inc
    • Richmond, Virginia, United States, 23294
      • National Clinical Research - Richmond, Inc.
  • Washington
    • Olympia, Washington, United States, 98502
      • Capital Clinical Research Center

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 80 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Key Inclusion Criteria:

• Male and female subjects who are ≥ 18 and ≤ 80 years old
• Subjects diagnosed with type 2 diabetes at least one year prior to Screening and receiving a stable, therapeutic dose of metformin > 1500 mg/day according to local prescribing information for at least 3 months prior to Screening or the highest tolerated dose > 1000 mg/day documented in the subject's history
• HbA1c > 7.0% and < 10.5% at Screening
• C-peptide > 0.8 ng/mL at Screening
• Body mass index (BMI) > 22 and < 45 kg/m2 at Screening
• Body weight < 150 kg at Screening

Key Exclusion Criteria:

• Prior participation in any stem cell study
• Women who are pregnant, intending to become pregnant during the study period or currently lactating
• History of active substance abuse (including alcohol) within the past 2 years. Current alcohol abuse is defined as daily consumption of >3 alcoholic beverage. Current cigarette smoking > 10 cigarettes per day
• Severe hypoglycemia (defined as requiring third party assistance) or repeated and/or frequent hypoglycemia episodes (> 2 episodes/week) within one month prior to Screening
• Patients receiving treatment for type 2 diabetes with diet and exercise alone, insulin therapy within 6 months of Screening except if used transiently for < 7 days for intercurrent illness or any other anti-diabetic medication except metformin within 3 months of Screening
• Any concurrent medical condition/disorder or clinically symptomatic cardiovascular, gastrointestinal (including pancreatitis), renal, hematological, pulmonary, acute or chronic infectious disease, active retinal disease, or other disorder which in the Investigator's opinion would interfere with the subject's ability to complete the trial, would require administration of treatment that could affect the interpretation of the safety and efficacy variables or would preclude safe involvement in the study.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Single

Number of Arms

3

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Cohort 1; MPC dose 1 or Placebo	Drug: Mesenchymal Precursor Cells (MPCs); Single intravenous infusion of MPCs Dose 1; Drug: Mesenchymal Precursor Cells (MPCs); Single intravenous infusion of MPCs Dose 2; Drug: Mesenchymal Precursor Cells (MPCs); Single intravenous infusion of MPCs Dose 3
Experimental: Cohort 2; MPC dose 2 or Placebo	Drug: Mesenchymal Precursor Cells (MPCs); Single intravenous infusion of MPCs Dose 1; Drug: Mesenchymal Precursor Cells (MPCs); Single intravenous infusion of MPCs Dose 2; Drug: Mesenchymal Precursor Cells (MPCs); Single intravenous infusion of MPCs Dose 3
Experimental: Cohort 3; MPC dose 3 or Placebo	Drug: Mesenchymal Precursor Cells (MPCs); Single intravenous infusion of MPCs Dose 1; Drug: Mesenchymal Precursor Cells (MPCs); Single intravenous infusion of MPCs Dose 2; Drug: Mesenchymal Precursor Cells (MPCs); Single intravenous infusion of MPCs Dose 3

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Primary objective of the study is to assess the safety and tolerability of MPC therapy	Outcomes include the measurement of the following safety parameters: Adverse events and serious adverse events(including hypoglycemia); Vital signs (BP, HR, RR, O2 saturation); Physical examinations; Results of clinical laboratory tests (hematology, biochemistry, and urinalysis, flow cytometry Class I and Class II PRA % with specificity, antibovine and antimurine antibody analysis); Pulmonary function test; Electrocardiograms; Chest X-ray; Fundus oculi examination	116 Weeks

Collaborators and Investigators

• Sponsor: Mesoblast, Ltd.
• Investigators: Study Director: K Segal, PhD, Mesoblast, Ltd.

Publications and helpful links

General Publications

• Skyler JS, Fonseca VA, Segal KR, Rosenstock J; MSB-DM003 Investigators. Allogeneic Mesenchymal Precursor Cells in Type 2 Diabetes: A Randomized, Placebo-Controlled, Dose-Escalation Safety and Tolerability Pilot Study. Diabetes Care. 2015 Sep;38(9):1742-9. doi: 10.2337/dc14-2830. Epub 2015 Jul 7.

Study record dates

Study Major Dates

• Study Start: April 1, 2012
• Primary Completion (Actual): October 1, 2013
• Study Completion (Actual): October 1, 2015

Study Registration Dates

• First Submitted: April 9, 2012
• First Submitted That Met QC Criteria: April 10, 2012
• First Posted (Estimate): April 12, 2012

Study Record Updates

• Last Update Posted (Actual): June 2, 2020
• Last Update Submitted That Met QC Criteria: June 1, 2020
• Last Verified: June 1, 2020

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Glucose Metabolism Disorders; Metabolic Diseases; Endocrine System Diseases; Diabetes Mellitus; Diabetes Mellitus, Type 2
• Other Study ID Numbers: MSB-DM003