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 )

June 2, 2008
June 4, 2008
December 4, 2014
June 2008
December 2010   (Final data collection date for primary outcome measure)
C-peptide AUC response (MMTT) [ Time Frame: 1 year ]
C-peptide AUC response (MMTT)
Complete list of historical versions of study NCT00690066 on Archive Site
  • Peak C-peptide response (MMTT) [ Time Frame: 2 years ]
  • Basal C-peptide response [ Time Frame: 2 years ]
  • Total daily insulin dose (units/kg) [ Time Frame: 2 years ]
  • Glycosylated hemoglobin (HbA1c) levels [ Time Frame: 2 years ]
  • Number of severe and documented hypoglycemic events [ Time Frame: 2 years ]
  • Changes in levels of GAD or IA-2 autoantibodies [ Time Frame: 2 years ]
  • 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 GAD or IA-2 autoantibodies
Not Provided
Not Provided
PROCHYMAL® (Human Adult Stem Cells) for the Treatment of Recently Diagnosed Type 1 Diabetes Mellitus (T1DM)
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
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.
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.
Phase 2
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
  • Type 1 Diabetes Mellitus
  • Type 1 Diabetes
  • Diabetes Mellitus, Insulin-Dependent
  • Juvenile Diabetes
  • Drug: PROCHYMAL®
    Intravenous infusion of ex vivo cultured adult human mesenchymal stem cells
    Other Names:
    • ex vivo cultured adult human mesenchymal stem cells
    • Prochymal
  • Drug: Placebo
    Intravenous infusion of excipients of PROCHYMAL®
  • Experimental: A
    Intervention: Drug: PROCHYMAL®
  • Placebo Comparator: B
    Intervention: Drug: Placebo
Not Provided

*   Includes publications given by the data provider as well as publications identified by Identifier (NCT Number) in Medline.
December 2011
December 2010   (Final data collection date for primary outcome measure)

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
Sexes Eligible for Study: All
12 Years to 35 Years   (Child, Adult)
Contact information is only displayed when the study is recruiting subjects
United States
Not Provided
Not Provided
Mesoblast, Ltd. ( Mesoblast International Sàrl )
Mesoblast International Sàrl
Juvenile Diabetes Research Foundation
Not Provided
Mesoblast, Ltd.
December 2014

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP