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

Primary Purpose

Diabetes Mellitus, Type 1

Status

Withdrawn

Phase

Phase 2

Locations

Study Type

Interventional

Intervention

efalizumab

placebo

About this trial

This is an interventional treatment trial for Diabetes Mellitus, Type 1 focused on measuring efalizumab, Raptiva, Diabetes, Type 1 Diabetes Mellitus

Eligibility Criteria

12 Years - 35 Years (Child, Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Placebo Comparator

Arm Label

Arm Description

This group will receive weekly efalizumab injections for 6 months

This group will receive placebo injections for 6 months

Outcomes

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.

Secondary Outcome Measures

Full Information

NCT ID

NCT00737763

First Posted

August 19, 2008

Last Updated

May 9, 2014

Sponsor

Emory University

Collaborators

Juvenile Diabetes Research Foundation, Genentech, Inc.

1. Study Identification

Unique Protocol Identification Number

NCT00737763

Brief Title

Beta Cell Rescue in New Onset Type 1 Diabetes With Efalizumab

Acronym

BRiTE

Official Title

Beta Cell Rescue in New Onset Type 1 Diabetes Mellitus With the LFA-1 Antibody Efalizumab

Study Type

Interventional

2. Study Status

Record Verification Date

May 2014

Overall Recruitment Status

Withdrawn

Why Stopped

drug withdrawn from market

Study Start Date

October 2008 (undefined)

Primary Completion Date

November 2009 (Actual)

Study Completion Date

November 2009 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Principal Investigator

Name of the Sponsor

Emory University

Collaborators

Juvenile Diabetes Research Foundation, Genentech, Inc.

4. Oversight

Data Monitoring Committee

Yes

5. Study Description

Brief Summary

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.

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Diabetes Mellitus, Type 1

Keywords

efalizumab, Raptiva, Diabetes, Type 1 Diabetes Mellitus

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 2

Interventional Study Model

Parallel Assignment

Masking

ParticipantCare ProviderInvestigatorOutcomes Assessor

Allocation

Randomized

Enrollment

0 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Arm Type

Experimental

Arm Description

This group will receive weekly efalizumab injections for 6 months

Arm Title

Arm Type

Placebo Comparator

Arm Description

This group will receive placebo injections for 6 months

Intervention Type

Drug

Intervention Name(s)

efalizumab

Other Intervention Name(s)

Raptiva

Intervention Description

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

Intervention Type

Drug

Intervention Name(s)

placebo

Other Intervention Name(s)

none applicable

Intervention Description

Enrollees receiving placebo will be given a subcutaneous injection of equal volume and appearance to treatment on the same schedule.

Primary Outcome Measure Information:

Title

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

10. Eligibility

Sex

All

Minimum Age & Unit of Time

12 Years

Maximum Age & Unit of Time

35 Years

Accepts Healthy Volunteers

Eligibility 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

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Mark R Rigby, MD, PhD

Organizational Affiliation

Emory University, Children's Healthcare of Atlanta

Official's Role

Principal Investigator

First Name & Middle Initial & Last Name & Degree

Eric Felner, MD

Organizational Affiliation

Children's Healthcare of Atlanta, Emory University

Official's Role

Principal Investigator

First Name & Middle Initial & Last Name & Degree

Sol Jacobs, MD

Organizational Affiliation

Emory University

Official's Role

Principal Investigator

First Name & Middle Initial & Last Name & Degree

Christian Larsen, MD, DPhil

Organizational Affiliation

Emory University

Official's Role

Principal Investigator

12. IPD Sharing Statement

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Beta Cell Rescue in New Onset Type 1 Diabetes With Efalizumab

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