Trial Outcomes & Findings for A Study to Assess the Effect of CellCept (Mycophenolate Mofetil) and Reduced Corticosteroids in Patients With Active Pemphigus Vulgaris (PV) (NCT NCT00683930)

Last Updated: 2011-06-22

Results Overview

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

Recruitment status

COMPLETED

Study phase

PHASE3

Target enrollment

96 participants

Primary outcome timeframe

52 weeks

Results posted on

2011-06-22

Participant Flow

Participant milestones

Participant milestones
Measure	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
Overall Study STARTED	36	21	37
Overall Study COMPLETED	29	18	28
Overall Study NOT COMPLETED	7	3	9

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

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

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Placebo n=36 Participants 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 n=21 Participants Mycophenolate mofetil (MMF) 500 mg tablets; 4 tablets twice daily for 52 weeks	MMF 3 g/Day n=37 Participants Mycophenolate mofetil (MMF) 500 mg tablets; 6 tablets twice daily for 52 weeks	Total n=94 Participants Total of all reporting groups
Age Continuous	45.8 years STANDARD_DEVIATION 12.18 • n=5 Participants	41.0 years STANDARD_DEVIATION 15.59 • n=7 Participants	44.8 years STANDARD_DEVIATION 13.47 • n=5 Participants	44.33 years STANDARD_DEVIATION 13.45 • n=4 Participants
Sex: Female, Male Female	24 Participants n=5 Participants	14 Participants n=7 Participants	18 Participants n=5 Participants	56 Participants n=4 Participants
Sex: Female, Male Male	12 Participants n=5 Participants	7 Participants n=7 Participants	19 Participants n=5 Participants	38 Participants n=4 Participants

PRIMARY outcome

Timeframe: 52 weeks

Population: Intent-to-treat population. Analysis population (AP): Placebo = 36; MMF 2 g/day or 3 g/day = 58.

Outcome measures

Outcome measures
Measure	Placebo n=36 Participants 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 n=58 Participants 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
Percentage of Patients Achieving Responder Status at Week 52	63.9 Percentage of Participants	69.0 Percentage of Participants	—	—

SECONDARY outcome

Timeframe: up to 52 weeks

Population: Intent-to-treat population. Analysis population (AP): Placebo = 36, subjects censored = 7; MMF 2 g/day or 3 g/day = 58, subjects censored = 10.

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)

Outcome measures

Outcome measures
Measure	Placebo n=36 Participants 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 n=58 Participants 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
Time to Initial Response	31.3 Weeks Interval 25.0 to 36.4	24.1 Weeks Interval 20.3 to 32.4	—	—

SECONDARY outcome

Timeframe: up to 52 weeks

Population: Intent-to-treat population. Analysis population (AP): Placebo = 36, subjects censored = 20; MMF 2 g/day or 3 g/day = 58, subjects censored = 23.

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.

Outcome measures

Outcome measures
Measure	Placebo n=16 Participants 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 n=35 Participants 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
Time to Sustained Response	46.0 Weeks Interval 29.7 to Data not available. Median, Q1, and Q3 are calculated from Kaplan-Meier estimates.	32.1 Weeks Interval 20.6 to Data not available. Median, Q1, and Q3 are calculated from Kaplan-Meier estimates.	—	—

SECONDARY outcome

Timeframe: 52 weeks

Population: 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.

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.

Outcome measures

Outcome measures
Measure	Placebo n=36 Participants 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 n=58 Participants 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 n=21 Participants MMF 500 mg tablets; 4 tablets twice daily for 52 weeks	Mycophenolate Mofetil 3 g/Day n=37 Participants MMF 500 mg tablets; 6 tablets twice daily for 52 weeks
Duration of Prednisone Maintenance Dosing	136.5 Days Interval 60.0 to 191.0	186.0 Days Interval 79.0 to 244.0	185.0 Days Interval 109.0 to 246.0	187.0 Days Interval 75.0 to 225.0

Adverse Events

Placebo

Serious events: 4 serious events

Other events: 31 other events

Deaths: 0 deaths

MMF 2 g/Day

Serious events: 1 serious events

Other events: 20 other events

Deaths: 0 deaths

MMF 3 g/Day

Serious events: 3 serious events

Other events: 32 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Placebo n=36 participants at risk 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 n=21 participants at risk Mycophenolate mofetil (MMF) 500 mg tablets; 4 tablets twice daily for 52 weeks	MMF 3 g/Day n=37 participants at risk Mycophenolate mofetil (MMF) 500 mg tablets; 6 tablets twice daily for 52 weeks
Gastrointestinal disorders Food Poisoning	2.8% 1/36 • AEs were monitored throughout the course of the study. Further follow-up was made at approximately 24 weeks (±7 days) after the Week 52 visit to record any AEs that occurred during the follow-up period. Safety analysis population. Note one patient from both the placebo group and MMF 2g/day group were excluded from the safety population as no study drug was dispensed.	0.00% 0/21 • AEs were monitored throughout the course of the study. Further follow-up was made at approximately 24 weeks (±7 days) after the Week 52 visit to record any AEs that occurred during the follow-up period. Safety analysis population. Note one patient from both the placebo group and MMF 2g/day group were excluded from the safety population as no study drug was dispensed.	0.00% 0/37 • AEs were monitored throughout the course of the study. Further follow-up was made at approximately 24 weeks (±7 days) after the Week 52 visit to record any AEs that occurred during the follow-up period. Safety analysis population. Note one patient from both the placebo group and MMF 2g/day group were excluded from the safety population as no study drug was dispensed.
Nervous system disorders Cerebrovascular Accident	0.00% 0/36 • AEs were monitored throughout the course of the study. Further follow-up was made at approximately 24 weeks (±7 days) after the Week 52 visit to record any AEs that occurred during the follow-up period. Safety analysis population. Note one patient from both the placebo group and MMF 2g/day group were excluded from the safety population as no study drug was dispensed.	4.8% 1/21 • AEs were monitored throughout the course of the study. Further follow-up was made at approximately 24 weeks (±7 days) after the Week 52 visit to record any AEs that occurred during the follow-up period. Safety analysis population. Note one patient from both the placebo group and MMF 2g/day group were excluded from the safety population as no study drug was dispensed.	0.00% 0/37 • AEs were monitored throughout the course of the study. Further follow-up was made at approximately 24 weeks (±7 days) after the Week 52 visit to record any AEs that occurred during the follow-up period. Safety analysis population. Note one patient from both the placebo group and MMF 2g/day group were excluded from the safety population as no study drug was dispensed.
Skin and subcutaneous tissue disorders Pemphigus	0.00% 0/36 • AEs were monitored throughout the course of the study. Further follow-up was made at approximately 24 weeks (±7 days) after the Week 52 visit to record any AEs that occurred during the follow-up period. Safety analysis population. Note one patient from both the placebo group and MMF 2g/day group were excluded from the safety population as no study drug was dispensed.	0.00% 0/21 • AEs were monitored throughout the course of the study. Further follow-up was made at approximately 24 weeks (±7 days) after the Week 52 visit to record any AEs that occurred during the follow-up period. Safety analysis population. Note one patient from both the placebo group and MMF 2g/day group were excluded from the safety population as no study drug was dispensed.	2.7% 1/37 • AEs were monitored throughout the course of the study. Further follow-up was made at approximately 24 weeks (±7 days) after the Week 52 visit to record any AEs that occurred during the follow-up period. Safety analysis population. Note one patient from both the placebo group and MMF 2g/day group were excluded from the safety population as no study drug was dispensed.
Pregnancy, puerperium and perinatal conditions Pregnancy	0.00% 0/36 • AEs were monitored throughout the course of the study. Further follow-up was made at approximately 24 weeks (±7 days) after the Week 52 visit to record any AEs that occurred during the follow-up period. Safety analysis population. Note one patient from both the placebo group and MMF 2g/day group were excluded from the safety population as no study drug was dispensed.	0.00% 0/21 • AEs were monitored throughout the course of the study. Further follow-up was made at approximately 24 weeks (±7 days) after the Week 52 visit to record any AEs that occurred during the follow-up period. Safety analysis population. Note one patient from both the placebo group and MMF 2g/day group were excluded from the safety population as no study drug was dispensed.	2.7% 1/37 • AEs were monitored throughout the course of the study. Further follow-up was made at approximately 24 weeks (±7 days) after the Week 52 visit to record any AEs that occurred during the follow-up period. Safety analysis population. Note one patient from both the placebo group and MMF 2g/day group were excluded from the safety population as no study drug was dispensed.
Pregnancy, puerperium and perinatal conditions Abortion Spontaneous	0.00% 0/36 • AEs were monitored throughout the course of the study. Further follow-up was made at approximately 24 weeks (±7 days) after the Week 52 visit to record any AEs that occurred during the follow-up period. Safety analysis population. Note one patient from both the placebo group and MMF 2g/day group were excluded from the safety population as no study drug was dispensed.	0.00% 0/21 • AEs were monitored throughout the course of the study. Further follow-up was made at approximately 24 weeks (±7 days) after the Week 52 visit to record any AEs that occurred during the follow-up period. Safety analysis population. Note one patient from both the placebo group and MMF 2g/day group were excluded from the safety population as no study drug was dispensed.	2.7% 1/37 • AEs were monitored throughout the course of the study. Further follow-up was made at approximately 24 weeks (±7 days) after the Week 52 visit to record any AEs that occurred during the follow-up period. Safety analysis population. Note one patient from both the placebo group and MMF 2g/day group were excluded from the safety population as no study drug was dispensed.
Vascular disorders Cardiovascular Insufficiency	2.8% 1/36 • AEs were monitored throughout the course of the study. Further follow-up was made at approximately 24 weeks (±7 days) after the Week 52 visit to record any AEs that occurred during the follow-up period. Safety analysis population. Note one patient from both the placebo group and MMF 2g/day group were excluded from the safety population as no study drug was dispensed.	0.00% 0/21 • AEs were monitored throughout the course of the study. Further follow-up was made at approximately 24 weeks (±7 days) after the Week 52 visit to record any AEs that occurred during the follow-up period. Safety analysis population. Note one patient from both the placebo group and MMF 2g/day group were excluded from the safety population as no study drug was dispensed.	0.00% 0/37 • AEs were monitored throughout the course of the study. Further follow-up was made at approximately 24 weeks (±7 days) after the Week 52 visit to record any AEs that occurred during the follow-up period. Safety analysis population. Note one patient from both the placebo group and MMF 2g/day group were excluded from the safety population as no study drug was dispensed.
Gastrointestinal disorders Duodenal Ulcer	2.8% 1/36 • AEs were monitored throughout the course of the study. Further follow-up was made at approximately 24 weeks (±7 days) after the Week 52 visit to record any AEs that occurred during the follow-up period. Safety analysis population. Note one patient from both the placebo group and MMF 2g/day group were excluded from the safety population as no study drug was dispensed.	0.00% 0/21 • AEs were monitored throughout the course of the study. Further follow-up was made at approximately 24 weeks (±7 days) after the Week 52 visit to record any AEs that occurred during the follow-up period. Safety analysis population. Note one patient from both the placebo group and MMF 2g/day group were excluded from the safety population as no study drug was dispensed.	0.00% 0/37 • AEs were monitored throughout the course of the study. Further follow-up was made at approximately 24 weeks (±7 days) after the Week 52 visit to record any AEs that occurred during the follow-up period. Safety analysis population. Note one patient from both the placebo group and MMF 2g/day group were excluded from the safety population as no study drug was dispensed.
Gastrointestinal disorders Pancreatitis	2.8% 1/36 • AEs were monitored throughout the course of the study. Further follow-up was made at approximately 24 weeks (±7 days) after the Week 52 visit to record any AEs that occurred during the follow-up period. Safety analysis population. Note one patient from both the placebo group and MMF 2g/day group were excluded from the safety population as no study drug was dispensed.	0.00% 0/21 • AEs were monitored throughout the course of the study. Further follow-up was made at approximately 24 weeks (±7 days) after the Week 52 visit to record any AEs that occurred during the follow-up period. Safety analysis population. Note one patient from both the placebo group and MMF 2g/day group were excluded from the safety population as no study drug was dispensed.	0.00% 0/37 • AEs were monitored throughout the course of the study. Further follow-up was made at approximately 24 weeks (±7 days) after the Week 52 visit to record any AEs that occurred during the follow-up period. Safety analysis population. Note one patient from both the placebo group and MMF 2g/day group were excluded from the safety population as no study drug was dispensed.
Musculoskeletal and connective tissue disorders Osteonecrosis	0.00% 0/36 • AEs were monitored throughout the course of the study. Further follow-up was made at approximately 24 weeks (±7 days) after the Week 52 visit to record any AEs that occurred during the follow-up period. Safety analysis population. Note one patient from both the placebo group and MMF 2g/day group were excluded from the safety population as no study drug was dispensed.	0.00% 0/21 • AEs were monitored throughout the course of the study. Further follow-up was made at approximately 24 weeks (±7 days) after the Week 52 visit to record any AEs that occurred during the follow-up period. Safety analysis population. Note one patient from both the placebo group and MMF 2g/day group were excluded from the safety population as no study drug was dispensed.	2.7% 1/37 • AEs were monitored throughout the course of the study. Further follow-up was made at approximately 24 weeks (±7 days) after the Week 52 visit to record any AEs that occurred during the follow-up period. Safety analysis population. Note one patient from both the placebo group and MMF 2g/day group were excluded from the safety population as no study drug was dispensed.
Infections and infestations Varicella	0.00% 0/36 • AEs were monitored throughout the course of the study. Further follow-up was made at approximately 24 weeks (±7 days) after the Week 52 visit to record any AEs that occurred during the follow-up period. Safety analysis population. Note one patient from both the placebo group and MMF 2g/day group were excluded from the safety population as no study drug was dispensed.	0.00% 0/21 • AEs were monitored throughout the course of the study. Further follow-up was made at approximately 24 weeks (±7 days) after the Week 52 visit to record any AEs that occurred during the follow-up period. Safety analysis population. Note one patient from both the placebo group and MMF 2g/day group were excluded from the safety population as no study drug was dispensed.	2.7% 1/37 • AEs were monitored throughout the course of the study. Further follow-up was made at approximately 24 weeks (±7 days) after the Week 52 visit to record any AEs that occurred during the follow-up period. Safety analysis population. Note one patient from both the placebo group and MMF 2g/day group were excluded from the safety population as no study drug was dispensed.
Eye disorders Cataract	2.8% 1/36 • AEs were monitored throughout the course of the study. Further follow-up was made at approximately 24 weeks (±7 days) after the Week 52 visit to record any AEs that occurred during the follow-up period. Safety analysis population. Note one patient from both the placebo group and MMF 2g/day group were excluded from the safety population as no study drug was dispensed.	0.00% 0/21 • AEs were monitored throughout the course of the study. Further follow-up was made at approximately 24 weeks (±7 days) after the Week 52 visit to record any AEs that occurred during the follow-up period. Safety analysis population. Note one patient from both the placebo group and MMF 2g/day group were excluded from the safety population as no study drug was dispensed.	0.00% 0/37 • AEs were monitored throughout the course of the study. Further follow-up was made at approximately 24 weeks (±7 days) after the Week 52 visit to record any AEs that occurred during the follow-up period. Safety analysis population. Note one patient from both the placebo group and MMF 2g/day group were excluded from the safety population as no study drug was dispensed.

Other adverse events

Additional Information

Medical Communications

Hoffmann-La Roche

Phone: 800-821-8590

Results disclosure agreements

Principal investigator is a sponsor employee 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.
Publication restrictions are in place

Restriction type: OTHER