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

Primary Purpose

Pemphigus Vulgaris (PV)

Status
Completed
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
Mycophenolate Mofetil 2 g/Day
Mycophenolate Mofetil (MMF) 3 g/Day
Placebo
Sponsored by
Hoffmann-La Roche
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Pemphigus Vulgaris (PV)

Eligibility Criteria

18 Years - 70 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm Type

Experimental

Experimental

Placebo Comparator

Arm Label

Mycophenolate Mofetil (MMF) 2 g/Day

Mycophenolate Mofetil (MMF) 3 g/Day

Placebo

Arm Description

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

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

Outcomes

Primary Outcome Measures

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

Secondary Outcome Measures

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 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.
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.

Full Information

First Posted
May 19, 2008
Last Updated
May 25, 2011
Sponsor
Hoffmann-La Roche
Collaborators
Aspreva Pharmaceuticals
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1. Study Identification

Unique Protocol Identification Number
NCT00683930
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
Study Type
Interventional

2. Study Status

Record Verification Date
May 2011
Overall Recruitment Status
Completed
Study Start Date
May 2004 (undefined)
Primary Completion Date
October 2008 (Actual)
Study Completion Date
October 2008 (Actual)

3. Sponsor/Collaborators

Name of the Sponsor
Hoffmann-La Roche
Collaborators
Aspreva Pharmaceuticals

4. Oversight

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Pemphigus Vulgaris (PV)

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Parallel Assignment
Masking
ParticipantInvestigator
Allocation
Randomized
Enrollment
96 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Mycophenolate Mofetil (MMF) 2 g/Day
Arm Type
Experimental
Arm Description
Mycophenolate mofetil 500 mg tablets; 4 tablets twice daily for 52 weeks
Arm Title
Mycophenolate Mofetil (MMF) 3 g/Day
Arm Type
Experimental
Arm Description
Mycophenolate mofetil 500 mg tablets; 6 tablets twice daily for 52 weeks
Arm Title
Placebo
Arm Type
Placebo Comparator
Intervention Type
Drug
Intervention Name(s)
Mycophenolate Mofetil 2 g/Day
Intervention Description
Mycophenolate mofetil 500 mg tablets; 4 tablets twice daily for 52 weeks
Intervention Type
Drug
Intervention Name(s)
Mycophenolate Mofetil (MMF) 3 g/Day
Intervention Description
Mycophenolate mofetil 500 mg tablets; 6 tablets twice daily for 52 weeks
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
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
Primary Outcome Measure Information:
Title
Percentage of Patients Achieving Responder Status at Week 52
Description
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 Outcome Measure Information:
Title
Time to Initial Response
Description
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
Title
Time to Sustained Response
Description
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
Title
Duration of Prednisone Maintenance Dosing
Description
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

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
70 Years
Accepts Healthy Volunteers
No
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Clinical Trials
Organizational Affiliation
Hoffmann-La Roche
Official's Role
Study Director
Facility Information:
City
Los Angeles
State/Province
California
ZIP/Postal Code
90095
Country
United States
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
30033
Country
United States
City
Baltimore
State/Province
Maryland
ZIP/Postal Code
21205
Country
United States
City
St Louis
State/Province
Missouri
ZIP/Postal Code
63110
Country
United States
City
New York
State/Province
New York
ZIP/Postal Code
10010
Country
United States
City
Toronto
State/Province
Ontario
ZIP/Postal Code
M5T 3A9
Country
Canada
City
Montreal
State/Province
Quebec
ZIP/Postal Code
H3G 1C6
Country
Canada
City
Frankfurt Am Main
ZIP/Postal Code
60596
Country
Germany
City
Heidelberg
ZIP/Postal Code
69115
Country
Germany
City
Köln
ZIP/Postal Code
50937
Country
Germany
City
Mainz
ZIP/Postal Code
55131
Country
Germany
City
Münster
ZIP/Postal Code
48149
Country
Germany
City
ULM
ZIP/Postal Code
89081
Country
Germany
City
Haifa
ZIP/Postal Code
31096
Country
Israel
City
Petach Tikva
ZIP/Postal Code
49100
Country
Israel
City
Zürich
ZIP/Postal Code
8091
Country
Switzerland
City
Ankara
ZIP/Postal Code
6100
Country
Turkey
City
Ankara
Country
Turkey
City
Istanbul
Country
Turkey
City
Crimea
ZIP/Postal Code
95006
Country
Ukraine
City
Donetsk
ZIP/Postal Code
83099
Country
Ukraine
City
Kiev
ZIP/Postal Code
252151
Country
Ukraine
City
Lugnansk
Country
Ukraine
City
Uzhgorod
ZIP/Postal Code
88011
Country
Ukraine
City
Leicester
ZIP/Postal Code
LE1 5WW
Country
United Kingdom
City
London
ZIP/Postal Code
SE1 7EH
Country
United Kingdom

12. IPD Sharing Statement

Citations:
PubMed Identifier
20410913
Citation
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.
Results Reference
derived
Links:
URL
http://www.roche-trials.com/studyResultGet.action?studyResultNumber=WX17796
Description
Clinical Study Report Synopsis

Learn more about this trial

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

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