Working… Menu

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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00690066
Recruitment Status : Completed
First Posted : June 4, 2008
Last Update Posted : December 4, 2014
Juvenile Diabetes Research Foundation
Information provided by (Responsible Party):
Mesoblast, Ltd. ( Mesoblast International Sàrl )

Brief Summary:
The purpose of this study is to establish the safety and efficacy of multiple administrations of PROCHYMAL® in subjects recently diagnosed with type 1 diabetes mellitus.

Condition or disease Intervention/treatment Phase
Type 1 Diabetes Mellitus Type 1 Diabetes Diabetes Mellitus, Insulin-Dependent Juvenile Diabetes Drug: PROCHYMAL® Drug: Placebo Phase 2

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 ROCHYMAL® 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.

Layout table for study information
Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 63 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
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 Recently Diagnosed Type 1 Diabetes Mellitus
Study Start Date : June 2008
Actual Primary Completion Date : December 2010
Actual Study Completion Date : December 2011

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Diabetes Type 1

Arm Intervention/treatment
Experimental: A
Intravenous infusion of ex vivo cultured adult human mesenchymal stem cells
Other Names:
  • ex vivo cultured adult human mesenchymal stem cells
  • Prochymal

Placebo Comparator: B
Drug: Placebo
Intravenous infusion of excipients of PROCHYMAL®

Primary Outcome Measures :
  1. C-peptide AUC response (MMTT) [ Time Frame: 1 year ]

Secondary Outcome Measures :
  1. Peak C-peptide response (MMTT) [ Time Frame: 2 years ]
  2. Basal C-peptide response [ Time Frame: 2 years ]
  3. Total daily insulin dose (units/kg) [ Time Frame: 2 years ]
  4. Glycosylated hemoglobin (HbA1c) levels [ Time Frame: 2 years ]
  5. Number of severe and documented hypoglycemic events [ Time Frame: 2 years ]
  6. Changes in levels of GAD or IA-2 autoantibodies [ Time Frame: 2 years ]

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

Layout table for eligibility information
Ages Eligible for Study:   12 Years to 35 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Subject must have a diagnosis of type 1 diabetes mellitus based on the ADA criteria
  • Subject must be screened between 2 and 20 weeks from initial T1DM diagnosis
  • Subject must be between the ages of 12 and 35 (inclusive)
  • Subject must have at least one diabetes-related autoantibody present (either GAD or IA-2)
  • Subject must have some beta cell function as determined by C-peptide testing
  • Subject 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
  • Subject must be willing to comply with the schedule of study visits and protocol requirements

Exclusion Criteria:

  • Subject has Body Mass Index (BMI) ≥ 30
  • Subject has evidence of retinopathy at baseline
  • Subject has abnormally high lipid levels
  • Subject has abnormal blood pressure
  • Subject has abnormal serum creatinine
  • Subject has evidence of clinically significant proteinuria
  • Subject has diabetic ketoacidosis
  • Subject is being treated for severe active infection of any type
  • A female subject who is breast-feeding, pregnant, or intends to become pregnant during the study
  • Subject with clinically relevant uncontrolled medical condition not associated with diabetes (e.g. hematologic, renal, hepatic, neurologic, cardiac, or respiratory)
  • Subject is allergic to bovine or porcine products
  • Subject has evidence of active malignancy, or prior history of active malignancy that has not been in remission for at least 5 years

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT00690066

Layout table for location information
United States, Alabama
University of Alabama, Division of Endocrinology & Metabolism
Birmingham, Alabama, United States, 35294
United States, California
Scripps Whittier Diabetes Institute
LaJolla, California, United States, 92037
Stanford University
Stanford, California, United States, 94305
United States, Florida
University of Florida
Gainesville, Florida, United States, 32610
Diabetes Research Institute
Miami, Florida, United States, 33136
United States, Kentucky
University of Kentucky
Lexington, Kentucky, United States, 40502
United States, Minnesota
University of Minnesota
Minneapolis, Minnesota, United States, 55455
United States, Nevada
Desert Endocrinology CRC
Henderson, Nevada, United States, 89052
Nevada Alliance Against Diabetes
Las Vegas, Nevada, United States, 89101
United States, North Carolina
University of North Carolina Diabetes Care Center
Chapel Hill, North Carolina, United States, 27599
American Health Research, Inc.
Charlotte, North Carolina, United States, 28207
United States, Ohio
The Lindner Clinical Trial Center
Cincinnati, Ohio, United States, 45219
Providence Health Partners - Center for Clinical Research
Dayton, Ohio, United States, 45439
United States, Pennsylvania
Cumberland Valley Endocrinology
Carlisle, Pennsylvania, United States, 17015
United States, Tennessee
AM Diabetes & Endocrinology Center
Bartlett, Tennessee, United States
United States, Texas
The University of Texas Southwestern Medical Center
Dallas, Texas, United States, 75390
United States, Utah
Optimum Clinical Research, Inc.
Salt Lake City, Utah, United States, 84102
United States, Virginia
The Strelitz Diabetes Center, Eastern VA Medical School
Norfolk, Virginia, United States, 23510
United States, Wisconsin
University of Wisconsin Health- West Clinic
Madison, Wisconsin, United States, 53717
Clinical and Transitional Science Institute
Milwaukee, Wisconsin, United States, 53226
Sponsors and Collaborators
Mesoblast International Sàrl
Juvenile Diabetes Research Foundation

Layout table for additonal information
Responsible Party: Mesoblast International Sàrl Identifier: NCT00690066     History of Changes
Other Study ID Numbers: 901
First Posted: June 4, 2008    Key Record Dates
Last Update Posted: December 4, 2014
Last Verified: December 2014
Keywords provided by Mesoblast, Ltd. ( Mesoblast International Sàrl ):
Type 1 Diabetes Mellitus
Type 1 Diabetes
Diabetes Mellitus, Insulin-Dependent
Juvenile Diabetes
Adult Human Stem Cells
Mesenchymal Stem Cells
Additional relevant MeSH terms:
Layout table for MeSH terms
Diabetes Mellitus
Diabetes Mellitus, Type 1
Glucose Metabolism Disorders
Metabolic Diseases
Endocrine System Diseases
Autoimmune Diseases
Immune System Diseases
Hypoglycemic Agents
Physiological Effects of Drugs