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

The recruitment status of this study is unknown because the information has not been verified recently.
Verified February 2011 by Osiris Therapeutics.
Recruitment status was  Active, not recruiting
Sponsor:
Collaborator:
Juvenile Diabetes Research Foundation
Information provided by:
Osiris Therapeutics
ClinicalTrials.gov Identifier:
NCT00690066
First received: June 2, 2008
Last updated: February 7, 2011
Last verified: February 2011
  Purpose

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 Intervention Phase
Type 1 Diabetes Mellitus
Diabetes Mellitus, Type 1
Diabetes Mellitus, Insulin-Dependent
Drug: PROCHYMAL®
Drug: Placebo
Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, 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 the Treatment of Recently Diagnosed Type 1 Diabetes Mellitus (T1DM)

Resource links provided by NLM:


Further study details as provided by Osiris Therapeutics:

Primary Outcome Measures:
  • C-peptide AUC response (MMTT) [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Peak C-peptide response (MMTT) [ Designated as safety issue: No ]
  • Basal C-peptide response [ Designated as safety issue: No ]
  • Total daily insulin dose (units/kg) [ Designated as safety issue: No ]
  • Glycosylated hemoglobin (HbA1c) levels [ Designated as safety issue: No ]
  • Number of severe and documented hypoglycemic events [ Designated as safety issue: No ]
  • Changes in levels of GAD or IA-2 autoantibodies [ Designated as safety issue: No ]

Estimated Enrollment: 60
Study Start Date: June 2008
Estimated Primary Completion Date: December 2010 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: A
PROCHYMAL®
Drug: PROCHYMAL®
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
Placebo
Drug: Placebo
Intravenous infusion of excipients of PROCHYMAL®

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.

  Eligibility

Ages Eligible for Study:   12 Years to 35 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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
  Contacts and Locations
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, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00690066

Locations
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
Osiris Therapeutics
Juvenile Diabetes Research Foundation
  More Information

Additional Information:
No publications provided

Responsible Party: Nancy Massoni, Osiris Therapeutics, Inc.
ClinicalTrials.gov Identifier: NCT00690066     History of Changes
Other Study ID Numbers: 901
Study First Received: June 2, 2008
Last Updated: February 7, 2011
Health Authority: United States: Food and Drug Administration

Keywords provided by Osiris Therapeutics:
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

Additional relevant MeSH terms:
Diabetes Mellitus
Diabetes Mellitus, Type 1
Glucose Metabolism Disorders
Metabolic Diseases
Endocrine System Diseases
Autoimmune Diseases
Immune System Diseases

ClinicalTrials.gov processed this record on August 19, 2014