Beta Cell Rescue in New Onset Type 1 Diabetes With Efalizumab - Full Text View

Purpose

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.

Condition	Intervention	Phase
Diabetes Mellitus, Type 1	Drug: efalizumab Drug: placebo	Phase 2

Study Type:	Interventional
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

Resource links provided by NLM:

Genetics Home Reference related topics: type 1 diabetes

MedlinePlus related topics: Diabetes Diabetes Type 1

U.S. FDA Resources

Further study details as provided by Emory University:

Primary Outcome Measures:

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 ]

Enrollment:	0
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)

Arms	Assigned Interventions
Experimental: A This group will receive weekly efalizumab injections for 6 months	Drug: efalizumab 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	Drug: placebo Enrollees receiving placebo will be given a subcutaneous injection of equal volume and appearance to treatment on the same schedule. Other Name: none applicable

Detailed Description:

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.

Eligibility

Ages Eligible for Study:	12 Years to 35 Years
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

Criteria

Inclusion Criteria:

Males or females 12-35 years old, no preference nor discrimination will be made based on ethnicity.
Recent diagnosis of T1DM, participant can be enrolled in the trial within 6 weeks of diagnosis.
Positive for at least one diabetes autoantibody (insulin, GAD65, IA2, ICA). Insulin autoantibody positivity will only be used as a selection criterion if insulin has not been used in at least the preceding 10 days.
Willingness to provide written informed consent (either the subject or the subject's legally authorized representative)
Have routine diabetic care under an endocrinologist and ability to follow study protocol for the duration of the 2-year study.
Although no preference or discrimination will be made based on ethnicity or gender, participants (and family and/or guardians when applicable) must demonstrate comprehension of the trial, including its obligations and potential risks.
If a female of childbearing potential, a negative pregnancy test and commitment to the use of two forms of effective contraception or abstinence for the duration of the study are necessary.
If a non-sterile male, commitment to the use of two forms of effective contraception (birth control) for the duration of the study is necessary.

Exclusion Criteria:

Severe allergic allergy or anaphylaxis to human monoclonal antibodies
Hospital admission for cardiac disease, stroke, or pulmonary disease within the past year
History of substance abuse within last 5 years
History of ongoing uncontrolled bacterial, viral, or fungal or atypical mycobacterium infections
History of opportunistic infections
Diagnosis with hepatic cirrhosis regardless of cause or severity
Diagnosis, history, or laboratory evidence of Hepatitis B or C infection
Hepatic enzymes 2 > times the upper limit of normal
History of active or treatment for tuberculosis or PPD positive
History of malignancy over the past 5 years
Recent initiation or change in treatment regimen of beta-blockers, angiotensin-converting enzyme inhibitors, interferons, quinidine anti-malarial drugs, or lithium in the past month
Seropositivity for human immunodeficiency virus (HIV)
Serologic or clinical evidence of recent or acute infection with EBV or CMV
Females who are pregnant, lactating, or planning on pregnancy during the 2 year study period
Progressive hearing loss
History of organ or bone marrow transplantation, sickle cell disease, cystic fibrosis, autoimmune anemia, seizures, autoimmune thrombocytopenia, leuko/lymphopenia, vasculitis, other autoimmune disease.
Current use of immunosuppressive medications
Plan or requirement of receiving new immunization of any type within the first 12 months of the study, or booster or completion vaccines with live or live-attenuated vaccines
Any condition that, in judgment of the investigator, could jeopardize the subject-safety following exposure to the drug.
Participation in another simultaneous medical investigation or trial

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00737763

Sponsors and Collaborators

Emory University

Juvenile Diabetes Research Foundation

Genentech

Investigators

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

More Information

No publications provided

Responsible Party:	Mark R. Rigby, MD, PhD, Emory University
ClinicalTrials.gov Identifier:	NCT00737763 History of Changes
Other Study ID Numbers:	BRiTE Trial for T1DM
Study First Received:	August 19, 2008
Last Updated:	May 18, 2009
Health Authority:	United States: Institutional Review Board

Keywords provided by Emory University:

efalizumab
Raptiva
Diabetes
Type 1 Diabetes Mellitus

Additional relevant MeSH terms:

Diabetes Mellitus
Diabetes Mellitus, Type 1
Glucose Metabolism Disorders
Metabolic Diseases

Endocrine System Diseases
Autoimmune Diseases
Immune System Diseases

ClinicalTrials.gov processed this record on May 21, 2013

Beta Cell Rescue in New Onset Type 1 Diabetes With Efalizumab (BRiTE)