Study of Etanercept (Enbrel) in the Treatment of Pemphigus Vulgaris - Full Text View

Sponsor:	Massachusetts General Hospital
Collaborators:	Brigham and Women's Hospital Stanford University
Information provided by:	Massachusetts General Hospital
ClinicalTrials.gov Identifier:	NCT00135720

Purpose

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	Intervention	Phase
Pemphigus Vulgaris	Drug: Enbrel (Etanercept)	Phase II

Study Type:	Interventional
Study Design:	Treatment, Randomized, Double-Blind, Placebo Control, Parallel Assignment, Safety/Efficacy Study
Official Title:	A Double-Blinded, Placebo-Controlled Study of Etanercept (Enbrel) in the Treatment of Pemphigus Vulgaris

Resource links provided by NLM:

MedlinePlus related topics: Pemphigus

Drug Information available for: Etanercept

U.S. FDA Resources

Further study details as provided by Massachusetts General Hospital:

Primary Outcome Measures:

Determine the mean time (in days) it takes to reduce active lesions, including blisters and erosions, by 50%
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.

Estimated Enrollment:	12
Study Start Date:	June 2004
Study Completion Date:	November 2007

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.

Eligibility

Ages Eligible for Study:	18 Years to 65 Years
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

Criteria

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

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00135720

Locations

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

Investigators

Principal Investigator:

Alexandra B Kimball, MD, MPH

CURTIS - Clinical Unit for Research Trials in Skin at Partners (MGH and BWH)

More Information

Additional Information:

CURTIS website This link exits the ClinicalTrials.gov site

Publications:

Stanley JR. Pemphigus. In Fizpatrick's Dermatology in General Medicine, 6th edition. Eds. Freedberg IM, Eisen AZ, Wolff K, Austen KF, et al. New York: McGraw-Hill, 2003. 558-567.

Alpsoy E, Yilmaz E, Basaran E, Yazar S, Cetin L. Is the combination of tetracycline and nicotinamide therapy alone effective in pemphigus? Arch Dermatol. 1995 Nov;131(11):1339-40. No abstract available.

Sacher C, Rubbert A, Konig C, Scharffetter-Kochanek K, Krieg T, Hunzelmann N. Treatment of recalcitrant cicatricial pemphigoid with the tumor necrosis factor alpha antagonist etanercept. J Am Acad Dermatol. 2002 Jan;46(1):113-5.

Berookhim B, Fischer HD, Weinberg JM. Treatment of recalcitrant pemphigus vulgaris with the tumor necrosis factor alpha antagonist etanercept. Cutis. 2004 Oct;74(4):245-7.

Alecu M, Alecu S, Coman G, Galatescu E, Ursaciuc C. ICAM-1, ELAM-1, TNF-alpha and IL-6 in serum and blister liquid of pemphigus vulgaris patients. Roum Arch Microbiol Immunol. 1999 Apr-Jul;58(2):121-30.

D'Auria L, Bonifati C, Mussi A, D'Agosto G, De Simone C, Giacalone B, Ferraro C, Ameglio F. Cytokines in the sera of patients with pemphigus vulgaris: interleukin-6 and tumour necrosis factor-alpha levels are significantly increased as compared to healthy subjects and correlate with disease activity. Eur Cytokine Netw. 1997 Dec;8(4):383-7.

Responsible Party:	Massachusetts General Hospital, Boston, MA 02114 ( Alexandra B. Kimball )
Study ID Numbers:	2004-P-002142, 79639;
Study First Received:	August 24, 2005
Last Updated:	May 19, 2008
ClinicalTrials.gov Identifier:	NCT00135720 History of Changes
Health Authority:	United States: Food and Drug Administration

Keywords provided by Massachusetts General Hospital:

Pemphigus Vulgaris
Chronic Pemphigus
Etanercept
Enbrel
anti-TNF-alpha

TNF-alpha antagonist
biologic
immunomodulator
subcutaneous medication

Additional relevant MeSH terms:

Anti-Inflammatory Agents
Autoimmune Diseases
Immunologic Factors
Skin Diseases, Vesiculobullous
Skin Diseases
Immune System Diseases
Physiological Effects of Drugs
Gastrointestinal Agents
TNFR-Fc fusion protein
Immunosuppressive Agents

Pharmacologic Actions
Pemphigus
Analgesics, Non-Narcotic
Sensory System Agents
Therapeutic Uses
Anti-Inflammatory Agents, Non-Steroidal
Analgesics
Peripheral Nervous System Agents
Antirheumatic Agents
Central Nervous System Agents

ClinicalTrials.gov processed this record on November 27, 2009