Type 1 Diabetes MellitusPhase 2NCT00690066
PROCHYMAL® (Human Adult Stem Cells) for the Treatment of Recently Diagnosed Type 1 Diabetes Mellitus (T1DM)
A Phase 2 study testing PROCHYMAL® in people with Type 1 Diabetes Mellitus and Type 1 Diabetes, run by Mesoblast, Inc. at 20 sites in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 12 to 35 years
- Conditions the study is for
- Type 1 Diabetes Mellitus, Type 1 Diabetes, Diabetes Mellitus, Insulin-Dependent
- What is being tested?
- PROCHYMAL®
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 63 participants
- Where
- 20 sitesUnited States
- Study dates
- 11 Jun 2008to 19 Dec 2011
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 12 to 35 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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.
Who cannot join
- 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.
Show all 14Show fewer
- 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.
Trial locations and contacts
20 sites in 1 country.
20 sites in 1 country
United States20 sites
AM Diabetes & Endocrinology Center
Bartlett, Tennessee, 38133
University of Alabama, Division of Endocrinology & Metabolism
Birmingham, Alabama, 35294
Cumberland Valley Endocrinology
Carlisle, Pennsylvania, 17015
University of North Carolina Diabetes Care Center
Chapel Hill, North Carolina, 27599
American Health Research, Inc.
Charlotte, North Carolina, 28207
The Lindner Clinical Trial Center
Cincinnati, Ohio, 45219
The University of Texas Southwestern Medical Center
Dallas, Texas, 75390
Providence Health Partners - Center for Clinical Research
Dayton, Ohio, 45439
University of Florida
Gainesville, Florida, 32610
Desert Endocrinology CRC
Henderson, Nevada, 89052
Scripps Whittier Diabetes Institute
La Jolla, California, 92037
Nevada Alliance Against Diabetes
Las Vegas, Nevada, 89101
University of Kentucky
Lexington, Kentucky, 40502
University of Wisconsin Health- West Clinic
Madison, Wisconsin, 53717
Diabetes Research Institute
Miami, Florida, 33136
Clinical and Transitional Science Institute
Milwaukee, Wisconsin, 53226
University of Minnesota
Minneapolis, Minnesota, 55455
The Strelitz Diabetes Center, Eastern VA Medical School
Norfolk, Virginia, 23510
Optimum Clinical Research, Inc.
Salt Lake City, Utah, 84102
Stanford University
Stanford, California, 94305
About this study
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
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.
Read the 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
- Being tested
- PROCHYMAL®, Placebo
- 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
- Medical subject headings
- Diabetes Mellitus, Type 1, Insulin Resistance
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PROCHYMAL®Drug
Also called ex vivo cultured adult human mesenchymal stem cells, Prochymal
Intravenous infusion of ex vivo cultured adult human mesenchymal stem cells
PlaceboDrug
Intravenous infusion of excipients of PROCHYMAL®
| Study group | Role | What they receive |
|---|---|---|
| Prochymal | Experimental | PROCHYMAL® Receives: Drug: PROCHYMAL® |
| Placebo | Placebo comparator | Placebo Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
C-peptide area under the concentration curve (AUC) response (MMTT)
Measured: 1 year
Secondary outcomes (6)
Peak C-peptide response (MMTT)
Measured: 2 years
Basal C-peptide response
Measured: 2 years
Total daily insulin dose (units/kg)
Measured: 2 years
Glycosylated hemoglobin (HbA1c) levels
Measured: 2 years
Number of severe and documented hypoglycemic events
Measured: 2 years
Changes in levels of glutamic acid decarboxylase (GAD) or islet antigen 2 (IA-2) autoantibodies
Measured: 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Jun 2008 (estimated)
- Study start11 Jun 2008
- Primary completion12 Dec 2010
- Study completion19 Dec 2011
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Type 1 Diabetes MellitusStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 63 participants (actual)
Sponsor and investigators
- Lead sponsor
- Mesoblast, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Juvenile Diabetes Research Foundation
- Responsible party
- Sponsor
- Investigators
- Mahboob Rahman, MD, Study director, Mesoblast, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00690066
- Sponsor's protocol ID
- 901
- Status last verified
- Nov 2025
- First submitted
- 2 Jun 2008
- Last updated
- 11 Mar 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 11 Mar 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT00690066
- Acronym / sponsor ID
- 901
- Conditions
- 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
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 63 participants (actual)
- Lead sponsor
- Mesoblast, Inc.Industry (pharma, biotech, devices)
- Collaborators
- Juvenile Diabetes Research Foundation
- Study start
- 11 Jun 2008 (actual)
- Primary completion
- 12 Dec 2010 (actual)
- Study completion
- 19 Dec 2011 (actual)
- First posted
- 4 Jun 2008 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Prochymal | Experimental | Drug: PROCHYMAL®PROCHYMAL® |
| Placebo | Placebo comparator | Drug: PlaceboPlacebo |
Who may qualify
- Ages
- 12 to 35 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (7 listed)
- 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.
Key exclusion criteria (14 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| PROCHYMAL®Also: ex vivo cultured adult human mesenchymal stem cells, ProchymalIntravenous infusion of ex vivo cultured adult human mesenchymal stem cells | Drug | Prochymal |
| PlaceboIntravenous infusion of excipients of PROCHYMAL® | Drug | Placebo |
Primary outcome
C-peptide area under the concentration curve (AUC) response (MMTT)
Time frame: 1 year
6 secondary outcomes
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 glutamic acid decarboxylase (GAD) or islet antigen 2 (IA-2) autoantibodies
Time frame: 2 years
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 Mar 2026.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Mahboob Rahman, MD study director
Mesoblast, Inc.
20 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing PROCHYMAL® in people with Type 1 Diabetes Mellitus and Type 1 Diabetes, run by Mesoblast, Inc. at 20 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 14 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 11 Mar 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 20 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
C-peptide area under the concentration curve (AUC) response (MMTT) (time frame: 1 year). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 11 Mar 2026.