Study of Etanercept (Enbrel) in the Treatment of Pemphigus Vulgaris
Information source: Massachusetts General Hospital
Information obtained from ClinicalTrials.gov on June 20, 2008
Link to the current ClinicalTrials.gov record.
Condition(s) targeted: Pemphigus Vulgaris
Intervention: Enbrel (Etanercept) (Drug)
Phase: Phase 2
Sponsored by: Massachusetts General Hospital
Official(s) and/or principal investigator(s):
Alexandra B Kimball, MD, MPH, Principal Investigator, Affiliation: CURTIS - Clinical Unit for Research Trials in Skin at Partners (MGH and BWH)
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.
Official title: A Double-Blinded, Placebo-Controlled Study of Etanercept (Enbrel) in the Treatment of Pemphigus Vulgaris
Study design: Treatment, Randomized, Double-Blind, Placebo Control, Parallel Assignment, Safety/Efficacy Study
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.
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.
Minimum age: 18 Years.
Maximum age: 65 Years.
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
- 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
Locations and Contacts
Clinical Unit for Research Trials in Skin, Boston, Massachusetts 02114, United States
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.
Starting date: June 2004
Ending date: November 2007
Last updated: May 19, 2008