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Study of Etanercept (Enbrel) in the Treatment of Pemphigus Vulgaris

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00135720
Recruitment Status : Completed
First Posted : August 26, 2005
Last Update Posted : June 3, 2015
Brigham and Women's Hospital
Stanford University
Information provided by:
Massachusetts General Hospital

Brief Summary:

The purpose of the study is to find out about the effectiveness and the safety of an investigational drug called etanercept (Enbrel) to treat pemphigus vulgaris. Pemphigus vulgaris is a potentially life threatening blistering condition that currently has no cure.

An investigational drug is one that has not been approved by the United States (US) Food and Drug Administration (FDA) to treat a particular condition or disease. Etanercept has been approved by the FDA to treat rheumatoid arthritis, juvenile rheumatoid arthritis, and psoriasis, but not pemphigus vulgaris.

Condition or disease Intervention/treatment Phase
Pemphigus Vulgaris Drug: Enbrel (Etanercept) Phase 2

Detailed Description:

People with pemphigus vulgaris have been found to have an increased amount of a substance called tumor-necrosis factor-alpha (TNF-alpha) in their blood. Etanercept may control pemphigus vulgaris by blocking the effect of TNF-alpha. Patients diagnosed with pemphigus vulgaris have been asked to participate in this research study.

Enrolled subjects will be given either etanercept or placebo. A placebo looks just like the study drug but contains no active medication. Placebos help study doctors find out if the effects of the study drug are due to the drug itself or just because of being in the study. This type of study design helps measure the true effectiveness of a treatment. Patients have 2 chances in 3 of receiving etanercept and 1 chance in 3 of receiving placebo.

The researchers expect to enroll 24 subjects in this study. Up to 16 subjects will be enrolled at Brigham and Women's Hospital (BWH) and Massachusetts General Hospital (MGH).

This study is sponsored by Amgen, Inc., the manufacturer of etanercept.

If taking part in another research study or have taken part in a research study in the last 28 days, patients will not be able to take part in this study. Patients cannot be on multiple studies simultaneously; however, exceptions are made for non-invasive, non-interventional, and observational studies.

This study will last about 16 weeks with a total of 6 study visits: screening, week 0 (baseline), weeks 2, 4, 8 and 16. The participants will be able to continue on your current medications for pemphigus throughout the study.

All participants must be at least 18 years of age with a clinical diagnosis of pemphigus vulgaris and at least 6 active blisters or erosions. Neither pregnant nor nursing women will be included in the study.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 12 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double
Primary Purpose: Treatment
Official Title: A Double-Blinded, Placebo-Controlled Study of Etanercept (Enbrel) in the Treatment of Pemphigus Vulgaris
Study Start Date : June 2004
Actual Study Completion Date : November 2007

Primary Outcome Measures :
  1. Determine the mean time (in days) it takes to reduce active lesions, including blisters and erosions, by 50%
  2. Determine whether etanercept is a possible steroid-sparing treatment option. This will be determined by tapering the patient's existing steroid dose once our primary endpoint has been reached.

Information from the National Library of Medicine

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, Learn About Clinical Studies.

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Ages Eligible for Study:   18 Years to 65 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Summary Inclusion Criteria:

  • Understand and sign the written informed consent form and be willing and able to comply with the procedures of the study
  • Have a clinical diagnosis of pemphigus vulgaris
  • Have at least 6 active pemphigus lesions (blisters or erosions)
  • Have no adverse effects due to combining etanercept with concurrent pemphigus medications
  • Be on stable dose of current pemphigus medications for at least one month
  • Females of child-bearing potential must have a negative urine pregnancy test prior to treatment and agree to practice an acceptable method of birth control (oral contraceptives, hormonal implant devices, Depo-Provera, condom with spermicide, diaphragm with contraceptive cream or foam, or intrauterine device) during the course of the study
  • Be at least 18 years old at time of enrollment
  • Be able to adhere to the study visit schedule and other protocol requirements

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT00135720

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United States, Massachusetts
Clinical Unit for Research Trials in Skin
Boston, Massachusetts, United States, 02114
Sponsors and Collaborators
Massachusetts General Hospital
Brigham and Women's Hospital
Stanford University
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Principal Investigator: Alexandra B Kimball, MD, MPH CURTIS - Clinical Unit for Research Trials in Skin at Partners (MGH and BWH)
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Responsible Party: Alexandra B. Kimball, Massachusetts General Hospital, Boston, MA 02114 Identifier: NCT00135720    
Other Study ID Numbers: 2004-P-002142
First Posted: August 26, 2005    Key Record Dates
Last Update Posted: June 3, 2015
Last Verified: May 2008
Keywords provided by Massachusetts General Hospital:
Pemphigus Vulgaris
Chronic Pemphigus
TNF-alpha antagonist
subcutaneous medication
Additional relevant MeSH terms:
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Skin Diseases, Vesiculobullous
Skin Diseases
Autoimmune Diseases
Immune System Diseases
Anti-Inflammatory Agents, Non-Steroidal
Analgesics, Non-Narcotic
Sensory System Agents
Peripheral Nervous System Agents
Physiological Effects of Drugs
Anti-Inflammatory Agents
Antirheumatic Agents
Gastrointestinal Agents
Immunosuppressive Agents
Immunologic Factors