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

This study has been completed.

Sponsor:

Collaborator:

Aspreva Pharmaceuticals

Information provided by:

Hoffmann-La Roche

ClinicalTrials.gov Identifier:

NCT00683930

First received: May 19, 2008

Last updated: May 25, 2011

Last verified: May 2011

History of Changes

Results First Received: November 18, 2009

Study Type:	Interventional
Study Design:	Allocation: Randomized; Endpoint Classification: Safety/Efficacy Study; Intervention Model: Parallel Assignment; Masking: Double Blind (Subject, Investigator); Primary Purpose: Treatment
Condition:	Pemphigus Vulgaris (PV)
Interventions:	Drug: Mycophenolate Mofetil 2 g/Day Drug: Mycophenolate Mofetil (MMF) 3 g/Day Drug: Placebo

Participant Flow

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Recruitment Details

Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
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Pre-Assignment Details

Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
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Reporting Groups

	Description
Placebo	Placebo for Mycophenolate Mofetil (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
MMF 2 g/Day	Mycophenolate mofetil (MMF) 500 mg tablets; 4 tablets twice daily for 52 weeks
MMF 3 g/Day	Mycophenolate mofetil (MMF) 500 mg tablets; 6 tablets twice daily for 52 weeks

Participant Flow: Overall Study

	Placebo	MMF 2 g/Day	MMF 3 g/Day
STARTED	36 ^[1]	21 ^[1]	37
COMPLETED	29	18	28
NOT COMPLETED	7	3	9

[1]	One subject not included: no study drug dispensed.

Baseline Characteristics

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Reporting Groups

	Description
Placebo	Placebo for Mycophenolate Mofetil (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
MMF 2 g/Day	Mycophenolate mofetil (MMF) 500 mg tablets; 4 tablets twice daily for 52 weeks
MMF 3 g/Day	Mycophenolate mofetil (MMF) 500 mg tablets; 6 tablets twice daily for 52 weeks
Total	Total of all reporting groups

Baseline Measures

	Placebo	MMF 2 g/Day	MMF 3 g/Day	Total
Number of Participants [units: participants]	36	21	37	94
Age ^[1] [units: years] Mean ± Standard Deviation	45.8 ± 12.18	41.0 ± 15.59	44.8 ± 13.47	44.33 ± 13.45
Gender ^[1] [units: participants]
Female	24	14	18	56
Male	12	7	19	38

[1]	Intent-to-treat population

Outcome Measures

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1. Primary:

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

Measure Type	Primary
Measure Title	Percentage of Patients Achieving Responder Status at Week 52
Measure 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
Safety Issue	No

Population Description

Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
Intent-to-treat population. Analysis population (AP): Placebo = 36; MMF 2 g/day or 3 g/day = 58.

Reporting Groups

	Description
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
MMF 2 g/Day and MMF 3 g/Day Groups Combined	MMF 2 g/day group: 500 mg tablets, 4 tablets twice daily for 52 weeks MMF 3 g/day group: 500 mg tablets, 6 tablets twice daily for 52 weeks

Measured Values

	Placebo	MMF 2 g/Day and MMF 3 g/Day Groups Combined
Number of Participants Analyzed [units: participants]	36	58
Percentage of Patients Achieving Responder Status at Week 52 [units: Percentage of Participants]	63.9	69.0

Statistical Analysis 1 for Percentage of Patients Achieving Responder Status at Week 52

Groups ^[1]	All groups
Method ^[2]	Fisher Exact
P Value ^[3]	0.6558
Treatment difference in response rate ^[4]	5.1
97.5% Confidence Interval	( -17.4 to 27.6 )

[1]	Additional details about the analysis, such as null hypothesis and power calculation:
	No text entered.
[2]	Other relevant information, such as adjustments or degrees of freedom:
	Alpha = 0.025
[3]	Additional information, such as whether or not the p-value is adjusted for multiple comparisons and the a priori threshold for statistical significance:
	No text entered.
[4]	Other relevant estimation information:
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2. Secondary:

Time to Initial Response [ Time Frame: up to 52 weeks ]

Measure Type	Secondary
Measure Title	Time to Initial Response
Measure 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
Safety Issue	No

Population Description

Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
Intent-to-treat population. Analysis population (AP): Placebo = 36, subjects censored = 7; MMF 2 g/day or 3 g/day = 58, subjects censored = 10.

Reporting Groups

	Description
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
MMF 2 g/Day and MMF 3 g/Day Groups Combined	MMF 2 g/day group: 500 mg tablets, 4 tablets twice daily for 52 weeks MMF 3 g/day group: 500 mg tablets, 6 tablets twice daily for 52 weeks

Measured Values

	Placebo	MMF 2 g/Day and MMF 3 g/Day Groups Combined
Number of Participants Analyzed [units: participants]	36	58
Time to Initial Response [units: Weeks] Median ( Inter-Quartile Range )	31.3 ( 25.0 to 36.4 )	24.1 ( 20.3 to 32.4 )

No statistical analysis provided for Time to Initial Response

3. Secondary:

Time to Sustained Response [ Time Frame: up to 52 weeks ]

Measure Type	Secondary
Measure Title	Time to Sustained Response
Measure 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
Safety Issue	No

Population Description

Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
Intent-to-treat population. Analysis population (AP): Placebo = 36, subjects censored = 20; MMF 2 g/day or 3 g/day = 58, subjects censored = 23.

Reporting Groups

	Description
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
MMF 2 g/Day and MMF 3 g/Day Groups Combined	MMF 2 g/day group: 500 mg tablets, 4 tablets twice daily for 52 weeks MMF 3 g/day group: 500 mg tablets, 6 tablets twice daily for 52 weeks

Measured Values

	Placebo	MMF 2 g/Day and MMF 3 g/Day Groups Combined
Number of Participants Analyzed [units: participants]	16	35
Time to Sustained Response [units: Weeks] Median ( Full Range )	46.0 ( 29.7 to NA ) ^[1]	32.1 ( 20.6 to NA ) ^[1]

[1]	Data not available. Median, Q1, and Q3 are calculated from Kaplan-Meier estimates.

No statistical analysis provided for Time to Sustained Response

4. Secondary:

Duration of Prednisone Maintenance Dosing [ Time Frame: 52 weeks ]

Measure Type	Secondary
Measure Title	Duration of Prednisone Maintenance Dosing
Measure 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
Safety Issue	No

Population Description

Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
Intent-to-treat population. Analysis population (AP): Placebo = 36; MMF 2 g/day = 21; MMF 3 g/day = 37; MMF Groups Combined (2 g/day or 3 g/day) = 58.

Reporting Groups

	Description
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
MMF 2 g/Day and MMF 3 g/Day Groups Combined	MMF 2 g/day group: 500 mg tablets, 4 tablets twice daily for 52 weeks MMF 3 g/day group: 500 mg tablets, 6 tablets twice daily for 52 weeks
Mycophenolate Mofetil 2 g/Day	MMF 500 mg tablets; 4 tablets twice daily for 52 weeks
Mycophenolate Mofetil 3 g/Day	MMF 500 mg tablets; 6 tablets twice daily for 52 weeks

Measured Values

	Placebo	MMF 2 g/Day and MMF 3 g/Day Groups Combined	Mycophenolate Mofetil 2 g/Day	Mycophenolate Mofetil 3 g/Day
Number of Participants Analyzed [units: participants]	36	58	21	37
Duration of Prednisone Maintenance Dosing [units: Days] Median ( Inter-Quartile Range )	136.5 ( 60.0 to 191.0 )	186.0 ( 79.0 to 244.0 )	185.0 ( 109.0 to 246.0 )	187.0 ( 75.0 to 225.0 )

No statistical analysis provided for Duration of Prednisone Maintenance Dosing

Serious Adverse Events

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Other Adverse Events

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More Information

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Certain Agreements:

Principal Investigators are NOT employed by the organization sponsoring the study.

There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.

The agreement is:

	The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is less than or equal to 60 days. The sponsor cannot require changes to the communication and cannot extend the embargo.
	The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is more than 60 days but less than or equal to 180 days. The sponsor cannot require changes to the communication and cannot extend the embargo.
	Other disclosure agreement that restricts the right of the PI to discuss or publish trial results after the trial is completed. Restriction Description: The Study being conducted under this Agreement is part of the Overall Study. Investigator is free to publish in reputable journals or to present at professional conferences the results of the Study, but only after the first publication or presentation that involves the Overall Study. The Sponsor may request that Confidential Information be deleted and/or the publication be postponed in order to protect the Sponsor's intellectual property rights.

Limitations and Caveats

Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
No text entered.

Results Point of Contact:

Name/Title: Medical Communications
Organization: Hoffmann-La Roche
phone: 800-821-8590

No publications provided by Hoffmann-La Roche

Publications automatically indexed to this study:

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. Epub 2010 Apr 22.

Responsible Party:	Disclosures Group, Hoffmann-La Roche
ClinicalTrials.gov Identifier:	NCT00683930 History of Changes
Obsolete Identifiers:	NCT00140127
Other Study ID Numbers:	WX17796
Study First Received:	May 19, 2008
Results First Received:	November 18, 2009
Last Updated:	May 25, 2011
Health Authority:	United States: Food and Drug Administration

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