Beta Cell Rescue in New Onset Type 1 Diabetes With Efalizumab

NCT00737763 | Withdrawn Phase 2 DMC

• 2 other identifiers: IRB0007365BRiTE Trial for T1DM
• Study Type: interventional
• Target: N/A
• Locations: 0 countries
• Sites: N/A

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.

Conditions

Diabetes Mellitus, Type 1

Keywords

efalizumab; Raptiva; Diabetes; Type 1 Diabetes Mellitus

Outcome Measures

Primary Outcomes (1)

• 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.

  2 years

Study Arms (2)

A

EXPERIMENTAL

This group will receive weekly efalizumab injections for 6 months

Drug: efalizumab

B

PLACEBO COMPARATOR

This group will receive placebo injections for 6 months

Drug: placebo

Interventions

efalizumab DRUG

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

Also known as: Raptiva

A

placebo DRUG

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

Also known as: none applicable

B

Eligibility Criteria

• Age: 12 Years - 35 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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.

You may not qualify if:

• 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

Sponsors & Collaborators

• Emory University: lead
• Juvenile Diabetes Research Foundation: collaborator
• Genentech, Inc.: collaborator

MeSH Terms

Conditions

Diabetes Mellitus, Type 1; Diabetes Mellitus

Interventions

efalizumab

Condition Hierarchy (Ancestors)

Glucose Metabolism Disorders; Metabolic Diseases; Nutritional and Metabolic Diseases; Endocrine System Diseases; Autoimmune Diseases; Immune System Diseases

Study Officials

• 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

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Principal Investigator

Study Record Dates

• First Submitted: August 19, 2008
• First Posted: August 20, 2008
• Study Start: October 1, 2008
• Primary Completion: November 1, 2009
• Study Completion: November 1, 2009
• Last Updated: May 12, 2014

Record last verified: 2014-05