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

NCT ID: NCT00690066

Last Updated: 2021-12-23

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 63 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2008-06-11
• Study Completion Date: 2011-12-19

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.

Conditions

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

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

Prochymal

PROCHYMAL®

Group Type: EXPERIMENTAL

PROCHYMAL®

Intervention Type: DRUG

Intravenous infusion of ex vivo cultured adult human mesenchymal stem cells

Placebo

Placebo

Group Type: PLACEBO_COMPARATOR

Placebo

Intervention Type: DRUG

Intravenous infusion of excipients of PROCHYMAL®

Interventions

PROCHYMAL®

Intravenous infusion of ex vivo cultured adult human mesenchymal stem cells

Intervention Type: DRUG

Placebo

Intravenous infusion of excipients of PROCHYMAL®

Intervention Type: DRUG

Other Intervention Names

ex vivo cultured adult human mesenchymal stem cells; Prochymal

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.

• Minimum Eligible Age: 12 Years
• Maximum Eligible Age: 35 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Juvenile Diabetes Research Foundation

OTHER

Sponsor Role: collaborator

Mesoblast, Inc.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Mahboob Rahman, MD

Role: STUDY_DIRECTOR

Mesoblast, Inc.

Locations

University of Alabama, Division of Endocrinology & Metabolism

Birmingham, Alabama, United States

Scripps Whittier Diabetes Institute

La Jolla, California, United States

Stanford University

Stanford, California, United States

University of Florida

Gainesville, Florida, United States

Diabetes Research Institute

Miami, Florida, United States

University of Kentucky

Lexington, Kentucky, United States

University of Minnesota

Minneapolis, Minnesota, United States

Desert Endocrinology CRC

Henderson, Nevada, United States

Nevada Alliance Against Diabetes

Las Vegas, Nevada, United States

University of North Carolina Diabetes Care Center

Chapel Hill, North Carolina, United States

American Health Research, Inc.

Charlotte, North Carolina, United States

The Lindner Clinical Trial Center

Cincinnati, Ohio, United States

Providence Health Partners - Center for Clinical Research

Dayton, Ohio, United States

Cumberland Valley Endocrinology

Carlisle, Pennsylvania, United States

AM Diabetes & Endocrinology Center

Bartlett, Tennessee, United States

The University of Texas Southwestern Medical Center

Dallas, Texas, United States

Optimum Clinical Research, Inc.

Salt Lake City, Utah, United States

The Strelitz Diabetes Center, Eastern VA Medical School

Norfolk, Virginia, United States

University of Wisconsin Health- West Clinic

Madison, Wisconsin, United States

Clinical and Transitional Science Institute

Milwaukee, Wisconsin, United States

Countries

United States

Other Identifiers

901

Identifier Type: -

Identifier Source: org_study_id