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

Study Overview
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Brief Summary
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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.

Official Title
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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

Conditions
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Pemphigus Vulgaris (PV)

Intervention / Treatment
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* Drug: Mycophenolate Mofetil 2 g/Day
* Drug: Mycophenolate Mofetil (MMF) 3 g/Day
* Drug: Placebo


Participation Criteria
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Eligibility Criteria
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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

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 70 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Mycophenolate Mofetil (MMF) 2 g/Day<br>Mycophenolate mofetil 500 mg tablets; 4 tablets twice daily for 52 weeks | Drug: Mycophenolate Mofetil 2 g/Day<br>* Mycophenolate mofetil 500 mg tablets; 4 tablets twice daily for 52 weeks<br>|
| Experimental: Mycophenolate Mofetil (MMF) 3 g/Day<br>Mycophenolate mofetil 500 mg tablets; 6 tablets twice daily for 52 weeks | Drug: Mycophenolate Mofetil (MMF) 3 g/Day<br>* Mycophenolate mofetil 500 mg tablets; 6 tablets twice daily for 52 weeks<br>|
| Placebo Comparator: Placebo<br> | Drug: Placebo<br>* 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<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Percentage of Patients Achieving Responder Status at Week 52 | 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 | 52 weeks | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Time to Initial Response | 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) | up to 52 weeks | 
| Time to Sustained Response | 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. | up to 52 weeks | 
| Duration of Prednisone Maintenance Dosing | 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. | 52 weeks |