TRIALNET: MMF/DZB Study

The MMF/DZB Study

(This study is no longer recruiting patients)

Type 1 diabetes is an autoimmune disease. This means your body's immune system mistakenly attacks and destroys your insulin-producing cells. As the beta cells, which make insulin, are destroyed, you develop diabetes. This study will see if it is possible to stop the immune system from destroying beta cells in new onset type 1 diabetes patients (within 3 months of diagnosis.)

Brief summary of study
Why are researchers interested in these medications?
What requirements must a patient meet to be in the study?
What factors must I not have if I want to be in this study?
What are the benefits of participating in this study?

Brief summary of study

The goal of this study is to find out if two medicines are able to stop the ongoing destruction of beta cells in your body. The two medicines are called Mycophenolate mofetil (MMF/CellCept®) and Daclizumab (DZB/Zenapax®). They work by making your immune system less active. TrialNet researchers hope that these medicines will help your beta cells keep the ability to make some insulin and keep your blood sugar (glucose) levels closer to normal. Even if the medicines work, you will still need to take insulin shots but your blood sugar may be easier to control. Better controlled blood sugar may help reduce long-term complications of diabetes such as blindness, kidney failure, nerve damage, heart attack and stroke.

For this study, participants will take MMF alone, MMF and DZB together, or a placebo. A placebo is a "pretend" medicine that looks real but is not active. The reason that some patients will take a placebo is to determine the benefit of the study medicine compared to not giving a medicine.

In this study, you will be randomly assigned to one of three groups:

Active MMF with DZB placebo
Active MMF with Active DZB
MMF placebo with DZB placebo

Random assignment means that a participant has an equal chance of being assigned to any group. This is done to make sure that the groups are as similar as possible at the beginning of the trial. Group assignment is decided by chance alone (similar to the toss of a coin). Also, this trial will be masked. This means that neither you nor the researchers know to which group you have been assigned.

If you decide to be in this study, you will be taking study medicines for two years. MMF or MMF placebo will be given as pills that you will take 2 or 3 times a day. DZB or DZB placebo will be given into a vein using an intravenous catheter (IV) twice during the first month of the study at a clinical center.

You will need to go to the clinical center for visits and tests. For the first month you will come in every week, then you will come in at month 2 and month 3. After the month 3 visit, visits will occur about every three months. At most visits, you will have blood drawn and meet with a doctor. Occasionally, you will be asked to do a longer test called a Mixed Meal Tolerance Test that involves having an IV inserted into your arm. You will then drink a special protein drink and have blood samples taken from the IV over the next 2 to 4 hours.

Why are researchers interested in these medications?

Researchers have made great strides in understanding how the immune system works and in changing the activity of immune cells with medicines called immunotherapies. Some immunotherapies work by making the immune system less active. Scientists have discovered that key immune cells, called T cells, help to cause type 1 diabetes. These T cells are largely responsible for attacking the beta cells that produce insulin. Doctors have found medicines that slow or suppress the activity of T cells.

It is hoped that these immunosuppressive medicines can help treat type 1 diabetes by stopping T cells before they destroy all of the beta cells. When the symptoms of type 1 diabetes appear, many (BUT NOT ALL) beta cells have already been destroyed. With fewer beta cells, the body cannot make enough insulin to control blood sugar (glucose). The common symptoms of diabetes--hunger, thirst and unexplained weight loss--are caused by high blood sugar. Researchers hope that immunotherapy may save the remaining beta cells in a new-onset patient. If this approach works, it is hoped that the disease will be easier to control and will cause fewer problems later in life.

Medicines that make the immune system less active have been developed and studied for other diseases. Mycophenolate mofetil (MMF) and Daclizumab (DZB) are two of these medicines. Their effects on the immune system are well understood. Researchers believe these medicines may lessen the immune system's destruction of beta cells. In addition, researcher hope the effect of these medicines will last longer than other therapies.

What requirements must a patient meet to be in the study?

To be eligible to participate, you must meet study guidelines. These guidelines help to identify appropriate participants for the study and help protect participant safety.

In order to be in this study, you must:

Have been diagnosed with type 1 diabetes within the past 3 months.
Be 12 to 35 years of age.
Have blood tests that show you are still making insulin. Have antibodies in your blood that are present in people with type 1 diabetes. (An antibody is a protein made by the immune system that fights infection. People with type 1 diabetes have antibodies against insulin and parts of the insulin producing cells.)
If female and sexually active, be willing to use birth control and undergo urine pregnancy tests at every visit.
Be willing, to the best of your ability, to make all scheduled visits to the clinic and follow your study instructions.
Be willing to accept assignment to any of the three treatment groups and be willing to stay in that group for the length of the study.
Be willing to comply with "intensive management" of your diabetes. The goal of this type of diabetes management is to keep your blood sugar as close to normal as possible. This involves checking your blood sugar 3 to 4 times each day and taking insulin by injections or an insulin pump to keep your blood sugar as close to normal as possible.

What factors must I not have if I want to be in this study?

In order to be eligible to be in this study there are certain factors that you must not have.

In order to be in this study, you must not:

Have any medical conditions that would make it difficult and unsafe for you to be in this study.
Have a Body Mass Index (BMI) greater than the 95th percentile for your age and gender. Your Body Mass Index (BMI) is a measure of your weight for your height.
Have certain infections like HIV, hepatitis, and tuberculosis.
Have had vaccinations within 6 weeks of starting the study.
Require medicines (like steroids) for other conditions that make your immune system less active.
Currently be pregnant or within 3 months of having been pregnant.
Currently be nursing or within 6 weeks of having stopped nursing.
Anticipate getting pregnant at any time during the study.

What are the benefits of participating in this study?

Researchers hope that MMF/DZB will help to protect remaining beta cells and their ability to produce insulin after the diagnosis of type 1 diabetes. If the study is successful, this could lead to better blood sugar control and reduced risk for long-term complications. The information gained from these studies may help other people at risk for type 1 diabetes.

If you wish to see if you might be eligible for this study, complete the online screening or call toll free anytime:

1- 800- HALT- DM1 (1-800-425-8361)

Or contact one of the participating clinical centers directly.

Information will be maintained in a confidential manner.

Participating Clinical Center: Regional Area
Barbara Davis Center for Childhood Diabetes University of Colorado Aurora, CO 800-572-3992: Colorado, Utah, Wyoming,
Missouri, and Kansas
Benaroya Research Institute at Virginia Mason Seattle, WA 800-888-4187: Washington, Oregon, Alaska,
Montana and Idaho
Children's Hospital Los Angeles Los Angeles, CA 888-835-3761 csuh@chla.usc.edu: Southern California and Arizona
The Childrens Mercy Hospital Kansas City, MO 816-234-1660: Kansas City, MO and surrounding areas
Columbia University Naomi Berrie Diabetes Center New York, NY 212-851-5425 emg25@columbia.edu: New York and Northern New Jersey
The Hospital for Sick Children Toronto, Ontario 866-699-1899: Canada
Riley Hospital for Children Indiana University Indianapolis, IN 866-230-8486 pedsdiab@iupui.edu: Indiana, Illinois, Kentucky and
Lower Peninsula Michigan
Joslin Diabetes Center Children's Hospital Boston Boston, MA 800-242-5836: Connecticut, Maine, Massachusetts,
New Hampshire, Rhode Island, and Vermont
Stanford University Medical Center Stanford, CA 877-232-5182 dwilson@stanford.edu: Central California and Nevada
University of California, San Francisco San Francisco, CA 415-353-9084 kfraserh@ucsf.edu: Northern California and Hawaii
University of Florida Gainesville, FL 800-749-7424, dial 1, Ext. 334-0857 DiabetesResearch@peds.uf.edu: Northern Florida, Georgia, South Carolina,
North Carolina and Tennessee
University of Minnesota Minneapolis, MN 800-688-5252, Ext. 58944 schmi094@umn.edu pete5601@umn.edu: Minnesota, North Dakota, Nebraska,
Wisconsin, South Dakota, Iowa,
and Upper Peninsula Michigan
Vita-Salute San Raffaele University Milan, Italy +39-02-2643 2818 grogan.pauline@hsr.it: Italy