Currently there is no globally approved immunosuppressant therapy for PV based on data generated from well-controlled clinical trials.  Therefore, there is a need for a prospective, randomized, controlled study to obtain scientifically valid information for potential indication-labeling by the FDA.

Background 

Pemphigus vulgaris (PV) is a rare autoimmune blistering disease of the skin and/or mucous membrane.  Burn-like lesions or blisters which are very painful can cover a significant area of the skin and the inside of the mouth.  Current treatment is largely based on systemic immunosuppression using corticosteroids.  These corticosteroid therapies have proven beneficial, however they have limited long-term utility because of their potential to induce kidney and/or liver dysfunction, bone marrow suppression and other adverse effects.

Several academic centers in the United States are conducting a study using Mycophenolate mofetil (MMF) in the treatment of pemphigus.  MMF has the potential to serve as a therapeutic treatment in pemphigus, although currently it is not approved by any regulatory body for such an indication.  This investigational drug has been approved by the United States Food and Drug Administration for the management of kidney, heart, and liver transplant patients.     

Purpose of Trial     

Currently there is no globally approved immunosuppressant therapy for PV based on data generated from well-controlled clinical trials.  Therefore, there is a need for a prospective, randomized, controlled study to obtain scientifically valid information for potential indication-labeling by the FDA.  Such a trial would show whether adjunctive MMF can act as an important steroid-sparing and immunosuppressant agent for the treatment of PV.

Study Design

This study is designed as a prospective, randomized, double-blind, placebo-controlled, parallel group, international multi-center, 52 week trial.  After screening and determination of eligibility, subjects will be randomized to receive either MMF (1.5g or 1.0g twice a day) or matching placebo orally for 52 weeks.  All subjects will also receive oral prednisone and dosed according to ideal body weight.  Subjects may initiate prednisone treatment at the required dose at enrollment, or, if already being treated with prednisone, will adjust their dose to the required dose level.  Subjects will return to the clinic at Week 2, Week 4, and every 4 weeks thereafter until Week 52.  Prednisone dose will be reduced at these visits when the following criteria are fulfilled: no new persistent lesions and existing lesions demonstrate healing according to investigator judgment.

Inclusion Criteria

• Subjects of either sex, aged 18 to 70 years (inclusive); subjects over 70 may be eligible with the agreement of the Medical Monitor
• Diagnosis of Pemphigus Vulgaris within the past 24 months
• Mild to moderate disease state

Exclusion Criteria

• Known hypersensitivity or contraindication to MMF, mycophenolic acid (MPA), any component of the drug product, or corticosteroids
• Pregnancy, breastfeeding, or lactating or use of a non-reliable method of contraception
• History of unresponsiveness to a prior adequate trial of corticosteroid treatment in the judgment of the investigator
• Receiving regularly scheduled intravenous immunoglobulin (IVIG) treatment or plasma exchange (PE) or receiving IVIG/PE treatment within 8 weeks prior to randomization
• History of MMF or other immunosuppressant therapy, except corticosteroids, (including cyclosporine, cyclophosphamide, azathioprine or methotrexate) exceeding 4 weeks total duration and within 8 weeks prior to randomization
• Use of PV therapies other than those noted above (e.g., tetracycline, dapsone, gold) within 4 weeks prior to randomization
• Use of bile acid sequestrants within 4 weeks prior to randomization
• Evidence of paraneoplastic pemphigus or other autoimmune blistering disease other than PV
• Other known clinically significant active medical conditions

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