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

NCT00135720 | Completed Phase 2 DMC

• 2 other identifiers: 2004-P-00214279639;
• Study Type: interventional
• Target: 12
• Locations: 1 country
• Sites: 1

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.

Conditions

Pemphigus Vulgaris

Keywords

Pemphigus Vulgaris; Chronic Pemphigus; Etanercept; Enbrel; anti-TNF-alpha; TNF-alpha antagonist; biologic; immunomodulator; subcutaneous medication

Outcome Measures

Primary Outcomes (2)

• 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.

Interventions

Enbrel (Etanercept) DRUG

Eligibility Criteria

• Age: 18 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

Sponsors & Collaborators

• Massachusetts General Hospital: lead
• Brigham and Women's Hospital: collaborator
• Stanford University: collaborator

Study Sites (1)

Clinical Unit for Research Trials in Skin

Boston, Massachusetts, 02114, United States

Location

Related Publications (7)

• 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.

  BACKGROUND

• Goldstein BG and Goldstein AO. Pemphigus and bullous pemphigoid. UpToDate.

  BACKGROUND

• 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.

  PMID: 7503585 BACKGROUND

• 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.

  PMID: 11756956 BACKGROUND

• 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.

  PMID: 15551718 BACKGROUND

• 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.

  PMID: 11845451 BACKGROUND

• 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.

  PMID: 9459618 BACKGROUND

MeSH Terms

Conditions

Pemphigus

Interventions

Etanercept

Condition Hierarchy (Ancestors)

Skin Diseases, Vesiculobullous; Skin Diseases; Skin and Connective Tissue Diseases; Autoimmune Diseases; Immune System Diseases

Intervention Hierarchy (Ancestors)

Immunoglobulin Fc Fragments; Immunoglobulin Fragments; Peptide Fragments; Peptides; Amino Acids, Peptides, and Proteins; Immunoglobulin Constant Regions; Immunoglobulins; Immunoproteins; Blood Proteins; Proteins; Serum Globulins; Globulins; Receptors, Tumor Necrosis Factor; Receptors, Cytokine; Receptors, Immunologic; Receptors, Cell Surface; Membrane Proteins

Study Officials

• Alexandra B Kimball, MD, MPH

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

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER

Study Record Dates

• First Submitted: August 24, 2005
• First Posted: August 26, 2005
• Study Start: June 1, 2004
• Study Completion: November 1, 2007
• Last Updated: June 3, 2015

Record last verified: 2008-05

Locations

US (1)