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

This study has been withdrawn prior to enrollment.
(drug withdrawn from market)
Sponsor:
Collaborators:
Juvenile Diabetes Research Foundation
Genentech
Information provided by (Responsible Party):
Mark Rigby, Emory University
ClinicalTrials.gov Identifier:
NCT00737763
First received: August 19, 2008
Last updated: May 9, 2014
Last verified: May 2014
  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:


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
Study Completion Date: November 2009
Primary Completion Date: November 2009 (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 Type 1Diabetes Mellitus, participant can be enrolled in the trial within 6 weeks of diagnosis.
  • Positive for at least one diabetes autoantibody. 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 skin test 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 Epstein-Barr Virus or Cytomegalovirus
  • 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
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

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 Rigby, Principal Investigator, Emory University
ClinicalTrials.gov Identifier: NCT00737763     History of Changes
Other Study ID Numbers: IRB0007365, BRiTE Trial for T1DM
Study First Received: August 19, 2008
Last Updated: May 9, 2014
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 August 28, 2014