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

Primary Purpose

Pemphigus Vulgaris

Status
Completed
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
Enbrel (Etanercept)
Sponsored by
Massachusetts General Hospital
About
Eligibility
Locations
Outcomes
Full info

About this trial

This is an interventional treatment trial for Pemphigus Vulgaris focused on measuring Pemphigus Vulgaris, Chronic Pemphigus, Etanercept, Enbrel, anti-TNF-alpha, TNF-alpha antagonist, biologic, immunomodulator, subcutaneous medication

Eligibility Criteria

18 Years - 65 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • Clinical Unit for Research Trials in Skin

Outcomes

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.

Secondary Outcome Measures

Full Information

First Posted
August 24, 2005
Last Updated
June 2, 2015
Sponsor
Massachusetts General Hospital
Collaborators
Brigham and Women's Hospital, Stanford University
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1. Study Identification

Unique Protocol Identification Number
NCT00135720
Brief Title
Study of Etanercept (Enbrel) in the Treatment of Pemphigus Vulgaris
Official Title
A Double-Blinded, Placebo-Controlled Study of Etanercept (Enbrel) in the Treatment of Pemphigus Vulgaris
Study Type
Interventional

2. Study Status

Record Verification Date
May 2008
Overall Recruitment Status
Completed
Study Start Date
June 2004 (undefined)
Primary Completion Date
undefined (undefined)
Study Completion Date
November 2007 (Actual)

3. Sponsor/Collaborators

Name of the Sponsor
Massachusetts General Hospital
Collaborators
Brigham and Women's Hospital, Stanford University

4. Oversight

Data Monitoring Committee
Yes

5. Study Description

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.
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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Pemphigus Vulgaris
Keywords
Pemphigus Vulgaris, Chronic Pemphigus, Etanercept, Enbrel, anti-TNF-alpha, TNF-alpha antagonist, biologic, immunomodulator, subcutaneous medication

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
Double
Allocation
Randomized
Enrollment
12 (Anticipated)

8. Arms, Groups, and Interventions

Intervention Type
Drug
Intervention Name(s)
Enbrel (Etanercept)
Primary Outcome Measure Information:
Title
Determine the mean time (in days) it takes to reduce active lesions, including blisters and erosions, by 50%
Title
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.

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
65 Years
Accepts Healthy Volunteers
No
Eligibility 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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Alexandra B Kimball, MD, MPH
Organizational Affiliation
CURTIS - Clinical Unit for Research Trials in Skin at Partners (MGH and BWH)
Official's Role
Principal Investigator
Facility Information:
Facility Name
Clinical Unit for Research Trials in Skin
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02114
Country
United States

12. IPD Sharing Statement

Citations:
Citation
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.
Results Reference
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Citation
Goldstein BG and Goldstein AO. Pemphigus and bullous pemphigoid. UpToDate.
Results Reference
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PubMed Identifier
7503585
Citation
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. doi: 10.1001/archderm.131.11.1339. No abstract available.
Results Reference
background
PubMed Identifier
11756956
Citation
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. doi: 10.1067/mjd.2001.119100.
Results Reference
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PubMed Identifier
15551718
Citation
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.
Results Reference
background
PubMed Identifier
11845451
Citation
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.
Results Reference
background
PubMed Identifier
9459618
Citation
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.
Results Reference
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Study of Etanercept (Enbrel) in the Treatment of Pemphigus Vulgaris

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