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PROCHYMAL® (Human Adult Stem Cells) for the Treatment of Recently Diagnosed Type 1 Diabetes Mellitus (T1DM)

Primary Purpose

Type 1 Diabetes Mellitus, Type 1 Diabetes, Diabetes Mellitus, Insulin-Dependent

Status
Completed
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
PROCHYMAL®
Placebo
Sponsored by
Mesoblast, Inc.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Type 1 Diabetes Mellitus focused on measuring T1DM, Type 1 Diabetes Mellitus, Type 1 Diabetes, Diabetes Mellitus, Insulin-Dependent, Juvenile Diabetes, Adult Human Stem Cells, Mesenchymal Stem Cells, MSCs, Insulin, Osiris, Prochymal

Eligibility Criteria

12 Years - 35 Years (Child, Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  • Participant must have a diagnosis of type 1 diabetes mellitus based on the American Diabetes Association (ADA) criteria.
  • Participant must be screened between 2 and 20 weeks from initial T1DM diagnosis
  • Participants must be between the ages of 12 and 35 (inclusive).
  • Participant must have at least one diabetes-related autoantibody present (either GAD or IA-2).
  • Participant must have some beta cell function as determined by C-peptide testing (at least 0.2 pmol/mL (0.6 ng/mL) during MMTT.
  • Participants must be willing to comply with "intensive diabetes management" as directed by the Investigator with the goal of maintaining blood glucose as close to normal as possible (i.e., glycosylated hemoglobin A1c (HbA1c) value of ≤ 7.0%).
  • Participants must be willing to comply with the schedule of study visits and protocol requirements.

Exclusion Criteria:

  • Participant has Body Mass Index (BMI) ≥ 30.
  • Participant has evidence of retinopathy at baseline.
  • Participant has abnormally high lipid levels.
  • Participant has abnormal blood pressure.
  • Participant has an abnormal serum creatinine.
  • Participant has evidence of clinically significant proteinuria.
  • Participant has diabetic ketoacidosis.
  • Participant is being treated for a severe active infection of any type.
  • A female participant who is breast-feeding, pregnant, or intends to become pregnant during the study.
  • Participant with clinically relevant uncontrolled medical condition not associated with diabetes (e.g. hematologic, renal, hepatic, neurologic, cardiac, or respiratory).
  • Participant has received an investigational drug (not approved by the FDA) for any indication 30 days prior to the screening visit.
  • Participant is allergic to bovine or porcine products.
  • Participant has evidence of active malignancy or prior history of active malignancy that has not been in remission for at least 5 years.
  • Participant has any medical condition, which in the opinion of the Investigator, rendered his/her participation in this study unsuitable.

Sites / Locations

  • University of Alabama, Division of Endocrinology & Metabolism
  • Scripps Whittier Diabetes Institute
  • Stanford University
  • University of Florida
  • Diabetes Research Institute
  • University of Kentucky
  • University of Minnesota
  • Desert Endocrinology CRC
  • Nevada Alliance Against Diabetes
  • University of North Carolina Diabetes Care Center
  • American Health Research, Inc.
  • The Lindner Clinical Trial Center
  • Providence Health Partners - Center for Clinical Research
  • Cumberland Valley Endocrinology
  • AM Diabetes & Endocrinology Center
  • The University of Texas Southwestern Medical Center
  • Optimum Clinical Research, Inc.
  • The Strelitz Diabetes Center, Eastern VA Medical School
  • University of Wisconsin Health- West Clinic
  • Clinical and Transitional Science Institute

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Placebo Comparator

Arm Label

Prochymal

Placebo

Arm Description

PROCHYMAL®

Placebo

Outcomes

Primary Outcome Measures

C-peptide area under the concentration curve (AUC) response (MMTT)

Secondary Outcome Measures

Peak C-peptide response (MMTT)
Basal C-peptide response
Total daily insulin dose (units/kg)
Glycosylated hemoglobin (HbA1c) levels
Number of severe and documented hypoglycemic events
Changes in levels of glutamic acid decarboxylase (GAD) or islet antigen 2 (IA-2) autoantibodies

Full Information

First Posted
June 2, 2008
Last Updated
December 20, 2021
Sponsor
Mesoblast, Inc.
Collaborators
Juvenile Diabetes Research Foundation
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1. Study Identification

Unique Protocol Identification Number
NCT00690066
Brief Title
PROCHYMAL® (Human Adult Stem Cells) for the Treatment of Recently Diagnosed Type 1 Diabetes Mellitus (T1DM)
Official Title
A Phase II, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of PROCHYMAL® (Ex Vivo Cultured Adult Human Mesenchymal Stem Cells) for the Treatment of Recently Diagnosed Type 1 Diabetes Mellitus
Study Type
Interventional

2. Study Status

Record Verification Date
December 2021
Overall Recruitment Status
Completed
Study Start Date
June 11, 2008 (Actual)
Primary Completion Date
December 12, 2010 (Actual)
Study Completion Date
December 19, 2011 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Mesoblast, Inc.
Collaborators
Juvenile Diabetes Research Foundation

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. Study Description

Brief Summary
The purpose of this study is to establish the safety and efficacy of multiple administrations of PROCHYMAL® in participants recently diagnosed with type 1 diabetes mellitus.
Detailed Description
Diabetes mellitus refers to disorders in which the body has trouble controlling its blood glucose levels. There are two main types of diabetes: type 1 and type 2. Type 1 diabetes mellitus (T1DM), which is being studied in this trial, is an autoimmune disorder in which the body's own immune system attacks and destroys the cells that make insulin. These cells are called beta cells. As beta cells are destroyed, less insulin can be made. This causes blood sugar levels to increase above normal and can cause life-threatening hypo- and hyper-glycemic reactions. For this reason, people with type 1 diabetes must take insulin to help control their blood sugar levels. Over time, poorly controlled diabetes can lead to a variety of serious health conditions, including heart disease, stroke, blindness, amputations, kidney disease, and nerve damage. Insulin is the primary method of controlling diabetes by regulating blood glucose levels, but it may not reverse or prevent disease progression. The active ingredient in PROCHYMAL® is adult human mesenchymal stem cells (MSCs). MSCs have been shown to interact with the immune cells in the body, reducing inflammation and assisting in tissue repair. This study will help determine whether MSCs can protect normal pancreatic tissue from autoimmune attack and repair damaged pancreatic tissue, leading to an increase in insulin production and decrease in circulating blood glucose. The characteristics and biologic activity of PROCHYMAL®, along with a good safety profile in human trials to date, suggest that PROCHYMAL® may be a good candidate for addressing Type 1 Diabetes.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Type 1 Diabetes Mellitus, Type 1 Diabetes, Diabetes Mellitus, Insulin-Dependent, Juvenile Diabetes
Keywords
T1DM, Type 1 Diabetes Mellitus, Type 1 Diabetes, Diabetes Mellitus, Insulin-Dependent, Juvenile Diabetes, Adult Human Stem Cells, Mesenchymal Stem Cells, MSCs, Insulin, Osiris, Prochymal

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
63 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Prochymal
Arm Type
Experimental
Arm Description
PROCHYMAL®
Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Description
Placebo
Intervention Type
Drug
Intervention Name(s)
PROCHYMAL®
Other Intervention Name(s)
ex vivo cultured adult human mesenchymal stem cells, Prochymal
Intervention Description
Intravenous infusion of ex vivo cultured adult human mesenchymal stem cells
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
Intravenous infusion of excipients of PROCHYMAL®
Primary Outcome Measure Information:
Title
C-peptide area under the concentration curve (AUC) response (MMTT)
Time Frame
1 year
Secondary Outcome Measure Information:
Title
Peak C-peptide response (MMTT)
Time Frame
2 years
Title
Basal C-peptide response
Time Frame
2 years
Title
Total daily insulin dose (units/kg)
Time Frame
2 years
Title
Glycosylated hemoglobin (HbA1c) levels
Time Frame
2 years
Title
Number of severe and documented hypoglycemic events
Time Frame
2 years
Title
Changes in levels of glutamic acid decarboxylase (GAD) or islet antigen 2 (IA-2) autoantibodies
Time Frame
2 years

10. Eligibility

Sex
All
Minimum Age & Unit of Time
12 Years
Maximum Age & Unit of Time
35 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Participant must have a diagnosis of type 1 diabetes mellitus based on the American Diabetes Association (ADA) criteria. Participant must be screened between 2 and 20 weeks from initial T1DM diagnosis Participants must be between the ages of 12 and 35 (inclusive). Participant must have at least one diabetes-related autoantibody present (either GAD or IA-2). Participant must have some beta cell function as determined by C-peptide testing (at least 0.2 pmol/mL (0.6 ng/mL) during MMTT. Participants must be willing to comply with "intensive diabetes management" as directed by the Investigator with the goal of maintaining blood glucose as close to normal as possible (i.e., glycosylated hemoglobin A1c (HbA1c) value of ≤ 7.0%). Participants must be willing to comply with the schedule of study visits and protocol requirements. Exclusion Criteria: Participant has Body Mass Index (BMI) ≥ 30. Participant has evidence of retinopathy at baseline. Participant has abnormally high lipid levels. Participant has abnormal blood pressure. Participant has an abnormal serum creatinine. Participant has evidence of clinically significant proteinuria. Participant has diabetic ketoacidosis. Participant is being treated for a severe active infection of any type. A female participant who is breast-feeding, pregnant, or intends to become pregnant during the study. Participant with clinically relevant uncontrolled medical condition not associated with diabetes (e.g. hematologic, renal, hepatic, neurologic, cardiac, or respiratory). Participant has received an investigational drug (not approved by the FDA) for any indication 30 days prior to the screening visit. Participant is allergic to bovine or porcine products. Participant has evidence of active malignancy or prior history of active malignancy that has not been in remission for at least 5 years. Participant has any medical condition, which in the opinion of the Investigator, rendered his/her participation in this study unsuitable.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Mahboob Rahman, MD
Organizational Affiliation
Mesoblast, Inc.
Official's Role
Study Director
Facility Information:
Facility Name
University of Alabama, Division of Endocrinology & Metabolism
City
Birmingham
State/Province
Alabama
ZIP/Postal Code
35294
Country
United States
Facility Name
Scripps Whittier Diabetes Institute
City
La Jolla
State/Province
California
ZIP/Postal Code
92037
Country
United States
Facility Name
Stanford University
City
Stanford
State/Province
California
ZIP/Postal Code
94305
Country
United States
Facility Name
University of Florida
City
Gainesville
State/Province
Florida
ZIP/Postal Code
32610
Country
United States
Facility Name
Diabetes Research Institute
City
Miami
State/Province
Florida
ZIP/Postal Code
33136
Country
United States
Facility Name
University of Kentucky
City
Lexington
State/Province
Kentucky
ZIP/Postal Code
40502
Country
United States
Facility Name
University of Minnesota
City
Minneapolis
State/Province
Minnesota
ZIP/Postal Code
55455
Country
United States
Facility Name
Desert Endocrinology CRC
City
Henderson
State/Province
Nevada
ZIP/Postal Code
89052
Country
United States
Facility Name
Nevada Alliance Against Diabetes
City
Las Vegas
State/Province
Nevada
ZIP/Postal Code
89101
Country
United States
Facility Name
University of North Carolina Diabetes Care Center
City
Chapel Hill
State/Province
North Carolina
ZIP/Postal Code
27599
Country
United States
Facility Name
American Health Research, Inc.
City
Charlotte
State/Province
North Carolina
ZIP/Postal Code
28207
Country
United States
Facility Name
The Lindner Clinical Trial Center
City
Cincinnati
State/Province
Ohio
ZIP/Postal Code
45219
Country
United States
Facility Name
Providence Health Partners - Center for Clinical Research
City
Dayton
State/Province
Ohio
ZIP/Postal Code
45439
Country
United States
Facility Name
Cumberland Valley Endocrinology
City
Carlisle
State/Province
Pennsylvania
ZIP/Postal Code
17015
Country
United States
Facility Name
AM Diabetes & Endocrinology Center
City
Bartlett
State/Province
Tennessee
ZIP/Postal Code
38133
Country
United States
Facility Name
The University of Texas Southwestern Medical Center
City
Dallas
State/Province
Texas
ZIP/Postal Code
75390
Country
United States
Facility Name
Optimum Clinical Research, Inc.
City
Salt Lake City
State/Province
Utah
ZIP/Postal Code
84102
Country
United States
Facility Name
The Strelitz Diabetes Center, Eastern VA Medical School
City
Norfolk
State/Province
Virginia
ZIP/Postal Code
23510
Country
United States
Facility Name
University of Wisconsin Health- West Clinic
City
Madison
State/Province
Wisconsin
ZIP/Postal Code
53717
Country
United States
Facility Name
Clinical and Transitional Science Institute
City
Milwaukee
State/Province
Wisconsin
ZIP/Postal Code
53226
Country
United States

12. IPD Sharing Statement

Learn more about this trial

PROCHYMAL® (Human Adult Stem Cells) for the Treatment of Recently Diagnosed Type 1 Diabetes Mellitus (T1DM)

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