A Randomized, Double-Blind Study to Evaluate the Efficacy and Safety of Rituximab Versus MMF in Patients With Pemphigus Vulgaris
This study will evaluate the efficacy and safety of rituximab compared with mycophenolate mofetil (MMF) in patients with moderate-to-severely active pemphigus vulgaris (PV) requiring 60-120 mg/day oral prednisone (or equivalent).
Approximately 124 patients will be enrolled at up to 60 centers worldwide, with approximately 4 patients enrolled at this site. Patients will be randomized in a 1:1 ratio to receive either rituximab plus MMF placebo or rituximab placebo plus MMF. Randomization will be stratified by duration of illness.
The study will last approximately 24 months, with 14 study visits, and consists of three periods: a screening period of up to 28 days, a 52-week double-blind treatment period, and a 48-week safety follow up period that begins at the time of study treatment completion or discontinuation.
Study phase: III
Basic eligibility criteria:
Please contact the study coordinator for additional eligibility information.
- Age 18-75 years
- Confirmed diagnosis of PV within the previous 24 months
- Presence of moderate-to-severely active disease, defined as at least 6 lesions that last more than 1 week or at least 3% body surface involvement, including cutaneous and/or mucosal lesions
- Receiving standard-of-care corticosteroids consisting of 60-120 mg/day oral prednisone (or equivalent)
- Agreement to avoid excessive exposure to sunlight during study participation
- Diagnosis of pemphigus foliaceus or evidence of paraneoplastic pemphigus or other non-PV autoimmune blistering disease
- History of a severe allergic or anaphylactic reaction to humanized or murine monoclonal antibodies, or known hypersensitivity to any component of rituximab
- Pregnant or lactating, or intending to become pregnant during the study.
- Participated in another interventional clinical trial within 28 days prior to randomization
- Significant cardiovascular or pulmonary disease (including obstructive pulmonary disease)
- Known active infection of any kind (excluding fungal infections of nail beds) or any major episode of infection requiring hospitalization; entry into this study may be reconsidered once the infection has fully resolved.
- History of or current cancer, including solid tumors, hematologic malignancies, and carcinoma in situ (except complete excision of basal cell of the skin and squamous cell carcinoma of the skin that have been treated or excised and cured)
- Currently active alcohol or drug abuse, or history of alcohol or drug abuse within 24 weeks prior to screening
- Major surgery within 4 weeks prior to randomization, excluding diagnostic surgery
- Treatment with rituximab or a B cell-targeted therapy (e.g., anti-CD20, anti CD22, or anti-BLyS) within 12 months prior to randomization
- Treatment with a live vaccine within 28 days prior to randomization
- Positive test results for hepatitis B or hepatitis C virus at screening
Primary disease category: Dermatology
Sponsor: Hoffmann-La Roche Ltd
Protocol number: WA29330
Projected enrollment dates: May 2016 to May 2018
Official study title: A RANDOMIZED, DOUBLE-BLIND, DOUBLE-DUMMY, ACTIVE-COMPARATOR, MULTICENTER STUDY TO EVALUATE THE EFFICACY AND SAFETY OF RITUXIMAB VERSUS MMF IN PATIENTS WITH PEMPHIGUS VULGARIS