Beta Cell Rescue in New Onset Type 1 Diabetes With Efalizumab (BRiTE)
In this single-center therapeutic study, we will study the ability of efalizumab to protect remaining beta cells in teenagers and young adults who have been newly diagnosed with type 1 diabetes mellitus. Efalizumab is a monoclonal antibody which prevents the activation of antigen specific T lymphocytes to sites of inflammation. Efalizumab was approved by the FDA in 2003 for the treatment of psoriasis. It has been proven to be safe, well tolerated and effective in targeting T cell mediated disorders like those seen in autoimmunity.
|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:||Beta Cell Rescue in New Onset Type 1 Diabetes Mellitus With the LFA-1 Antibody Efalizumab|
- The primary endpoint for this study will be the difference from baseline in the body's ability to respond to a Mixed Meal Tolerance Test at 12 months after enrollment. [ Time Frame: 2 years ] [ Designated as safety issue: Yes ]
|Study Start Date:||October 2008|
|Estimated Study Completion Date:||September 2013|
|Estimated Primary Completion Date:||September 2013 (Final data collection date for primary outcome measure)|
This group will receive weekly efalizumab injections for 6 months
Enrollees randomized to efalizumab will receive the first dose of 0.7mg/kg subcutaneously given at enrollment, and 1.0 mg/kg subcutaneously weekly for 26 weeks self or family-administered after injection training. This is the FDA-approved initial and subsequent doses of efalizumab used for psoriasis treatment
Other Name: Raptiva
Placebo Comparator: B
This group will receive placebo injections for 6 months
Enrollees receiving placebo will be given a subcutaneous injection of equal volume and appearance to treatment on the same schedule.
Other Name: none applicable
Since there is data that shows that early intervention can prevent further destruction of insulin producing beta cells, the patients who will be enrolled in this study will have been diagnosed with Type 1 diabetes within 6 weeks of enrolling and starting therapy. Patients who meet the screening criteria will be randomized at a 2 to 1 ratio to either get weekly subcutaneous injections of efalizumab for 26 weeks versus a placebo injection. The researchers and patients will be blinded to the treatment group assignment. All patients will be followed for two years.
The primary endpoint for this study will be the difference from baseline in the body's ability to respond to a Mixed Meal Tolerance Test at 12 months after enrollment. The Mixed Meal Tolerance test will help test the production of insulin by the pancreas. By comparing the results of these tests between the treated group and the placebo group, we hope to be able to show preservation of beta cell function in the group treated with efalizumab.
|Principal Investigator:||Mark R Rigby, MD, PhD||Emory University, Children's Healthcare of Atlanta|
|Principal Investigator:||Eric Felner, MD||Children's Healthcare of Atlanta, Emory University|
|Principal Investigator:||Sol Jacobs, MD||Emory University|
|Principal Investigator:||Christian Larsen, MD, DPhil||Emory University|