A Study to Assess the Effect of CellCept (Mycophenolate Mofetil) and Reduced Corticosteroids in Patients With Active Pemphigus Vulgaris (PV)

• ClinicalTrials.gov Identifier: NCT00683930
• Recruitment Status: Completed
• First Posted: May 26, 2008
• Results First Posted: June 22, 2011
• Last Update Posted: June 22, 2011
• Sponsor: Hoffmann-La Roche
• Collaborator: Aspreva Pharmaceuticals
• Information provided by: Hoffmann-La Roche

Tracking Information

• First Submitted Date: May 19, 2008
• First Posted Date: May 26, 2008
• Results First Submitted Date: November 18, 2009
• Results First Posted Date: June 22, 2011
• Last Update Posted Date: June 22, 2011
• Study Start Date: May 2004
• Actual Primary Completion Date: October 2008 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: May 25, 2011)

Percentage of Patients Achieving Responder Status at Week 52 [ Time Frame: 52 weeks ]

The proportion of subjects achieving responder status (defined as no new persistent lesions and prednisone dose of not more than 10 mg/day from Week 48 until study termination at Week 52) in the two active treatment groups combined (2 g/day and 3 g/day mycophenolate mofetil) compared with the placebo group

• Original Primary Outcome Measures (submitted: May 23, 2008): Percentage of patients with responder status [ Time Frame: Week 52 ]

Current Secondary Outcome Measures (submitted: May 25, 2011)

• Time to Initial Response [ Time Frame: up to 52 weeks ]
  Time to initial response defined as the time that the subject first demonstrated responder status (defined as no new persistent lesions and prednisone dose of not more than 10 mg/day from Week 48 until study termination at Week 52)
• Time to Sustained Response [ Time Frame: up to 52 weeks ]
  Time to sustained response is defined as the week the subject first demonstrates both of the conditions of responder status provided the conditions are maintained through to study termination at Week 52. If a subject does not have a sustained response, time to sustained response is censored on the last day of the study.
• Duration of Prednisone Maintenance Dosing [ Time Frame: 52 weeks ]
  The duration of prednisone maintenance dosing was defined as the number of days that subjects maintained a prednisone dose of not more than 10 mg/day in the absence of new persistent lesions.

Original Secondary Outcome Measures (submitted: May 23, 2008)

• Number of days a patient maintains a prednisone dose of <= 10mg/dday, in the absence of a new persistent lesion [ Time Frame: Throughout study ]
• Time to relapse [ Time Frame: Event driven ]
• Time to Initial Response [ Time Frame: Event driven ]
• AEs, lab parameters, vital signs [ Time Frame: Throughout study ]

• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: A Study to Assess the Effect of CellCept (Mycophenolate Mofetil) and Reduced Corticosteroids in Patients With Active Pemphigus Vulgaris (PV)
• Official Title: A Prospective, Randomised, Double-blind, Placebo-controlled, Parallel Group, Mult-center, 52-week Trial to Assess the Efficacy and Safety of Adjunct Mycophenolate Mofetil (MMF) to Achieve Remission With Reduced Corticosteroid in Subjects With Pemphigus Vulgaris
• Brief Summary: This study was designed to assess the efficacy and safety of CellCept (1 g or 1.5 g orally twice daily for 52 weeks) in patients with pemphigus vulgaris receiving prednisone or other corticosteroids. During the study, patients had their corticosteroid dose gradually reduced if they responded to treatment. The anticipated time on study treatment was 12 months, and the target sample size was <100 individuals.
• Detailed Description: Not Provided
• Study Type: Interventional
• Study Phase: Phase 3
• Study Design: Allocation: Randomized; Intervention Model: Parallel Assignment; Masking: Double (Participant, Investigator); Primary Purpose: Treatment
• Condition: Pemphigus Vulgaris (PV)

Intervention

• Drug: Mycophenolate Mofetil 2 g/Day
  Mycophenolate mofetil 500 mg tablets; 4 tablets twice daily for 52 weeks
• Drug: Mycophenolate Mofetil (MMF) 3 g/Day
  Mycophenolate mofetil 500 mg tablets; 6 tablets twice daily for 52 weeks
• Drug: Placebo
  Placebo for MMF 2 g/day group: 4 tablets orally twice daily for 52 weeks; placebo for MMF 3 g/day group: 6 tablets orally twice daily for 52 weeks

Study Arms

• Experimental: Mycophenolate Mofetil (MMF) 2 g/Day
  Mycophenolate mofetil 500 mg tablets; 4 tablets twice daily for 52 weeks
  Intervention: Drug: Mycophenolate Mofetil 2 g/Day
• Experimental: Mycophenolate Mofetil (MMF) 3 g/Day
  Mycophenolate mofetil 500 mg tablets; 6 tablets twice daily for 52 weeks
  Intervention: Drug: Mycophenolate Mofetil (MMF) 3 g/Day
• Placebo Comparator: Placebo
  Intervention: Drug: Placebo

• Publications *: Beissert S, Mimouni D, Kanwar AJ, Solomons N, Kalia V, Anhalt GJ. Treating pemphigus vulgaris with prednisone and mycophenolate mofetil: a multicenter, randomized, placebo-controlled trial. J Invest Dermatol. 2010 Aug;130(8):2041-8. doi: 10.1038/jid.2010.91. Epub 2010 Apr 22.

Recruitment Information

• Recruitment Status: Completed
• Actual Enrollment (submitted: May 25, 2011): 96
• Original Estimated Enrollment (submitted: May 23, 2008): 64
• Actual Study Completion Date: October 2008
• Actual Primary Completion Date: October 2008 (Final data collection date for primary outcome measure)

Eligibility Criteria

Inclusion Criteria:

• Adult patients 18 to 70 years of age
• Diagnosis of mild to moderate pemphigus vulgaris within the past 12 months, requiring high dose corticosteroids

Exclusion Criteria:

• Female patients who are pregnant, breastfeeding, or lactating
• Regularly scheduled plasma exchange (PE) or intravenous immunoglobulin (IVIG) treatment, or PE or IVIG treatment within 8 weeks prior to randomization
• CellCept or other immunosuppressive therapy, except corticosteroids, exceeding 2 weeks total duration and within 8 weeks prior to randomization
• Use of PV therapies other than those noted above, within 4 weeks prior to randomization
• Use of topical corticosteroids within 2 weeks prior to randomization

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 18 Years to 70 Years (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: Canada, Germany, Israel, Switzerland, Turkey, Ukraine, United Kingdom, United States
• Removed Location Countries: India

Administrative Information

• NCT Number: NCT00683930
• Other Study ID Numbers: WX17796
• Has Data Monitoring Committee: Not Provided
• U.S. FDA-regulated Product: Not Provided
• IPD Sharing Statement: Not Provided
• Responsible Party: Disclosures Group, Hoffmann-La Roche
• Study Sponsor: Hoffmann-La Roche
• Collaborators: Aspreva Pharmaceuticals

Investigators

• Study Director: Clinical Trials; Hoffmann-La Roche

• PRS Account: Hoffmann-La Roche
• Verification Date: May 2011