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

Hoffmann-La Roche96 enrolled

Overview

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.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

DOUBLE

Enrollment

Conditions

Pemphigus Vulgaris (PV)

Interventions

Mycophenolate Mofetil 2 g/DayDRUG

Mycophenolate mofetil 500 mg tablets; 4 tablets twice daily for 52 weeks

Mycophenolate Mofetil (MMF) 3 g/DayDRUG

Mycophenolate mofetil 500 mg tablets; 6 tablets twice daily for 52 weeks

PlaceboDRUG

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

Eligibility

Sex: ALLMin age: 18 YearsMax age: 70 Years

Medical Language ↔ Plain English

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

Locations (26)

Unnamed facility

Los Angeles, California, United States

Unnamed facility

Atlanta, Georgia, United States

Outcomes

Primary Outcomes

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

Time frame: 52 weeks

Secondary Outcomes

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)

Time frame: 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.

Time frame: 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.

Time frame: 52 weeks

Data from ClinicalTrials.gov

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