Pemphigus Vulgaris (PV)Phase 3NCT00683930
A Study to Assess the Effect of CellCept (Mycophenolate Mofetil) and Reduced Corticosteroids in Patients With Active Pemphigus Vulgaris (PV)
A Phase 3 study testing Mycophenolate Mofetil 2 g/Day and Mycophenolate Mofetil (MMF) 3 g/Day in people with Pemphigus Vulgaris (PV), run by Hoffmann-La Roche at 26 sites in 8 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 70 years
- Conditions the study is for
- Pemphigus Vulgaris (PV)
- What is being tested?
- Mycophenolate Mofetil 2 g/Day and Mycophenolate Mofetil (MMF) 3 g/Day
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 96 participants
- Where
- 26 sitesin 8 countries
- Study dates
- May 2004to Oct 2008
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 70 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Adult patients 18 to 70 years of age
- Diagnosis of mild to moderate pemphigus vulgaris within the past 12 months, requiring high dose corticosteroids
Who cannot join
- 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
Trial locations and contacts
26 sites in 8 countries.
26 sites in 8 countries
Germany6 sites
Study site
Cologne, 50937
Study site
Frankfurt am Main, 60596
Study site
Heidelberg, 69115
Study site
Mainz, 55131
Study site
Münster, 48149
Study site
Ulm, 89081
United States5 sites
Study site
Atlanta, Georgia, 30033
Study site
Baltimore, Maryland, 21205
Study site
Los Angeles, California, 90095
Study site
New York, New York, 10010
Study site
St Louis, Missouri, 63110
Ukraine5 sites
Study site
Crimea, 95006
Study site
Donetsk, 83099
Study site
Kiev, 252151
Study site
Lugnansk
Study site
Uzhhorod, 88011
Turkey (Türkiye)3 sites
Study site
Ankara, 6100
Study site
Ankara
Study site
Istanbul
Canada2 sites
Study site
Montreal, Quebec, H3G 1C6
Study site
Toronto, Ontario, M5T 3A9
United Kingdom2 sites
Study site
Leicester, LE1 5WW
Study site
London, SE1 7EH
About this study
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
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.
- Conditions
- Pemphigus Vulgaris (PV)
- Being tested
- Mycophenolate Mofetil 2 g/Day, Mycophenolate Mofetil (MMF) 3 g/Day, Placebo
- Medical subject headings
- Pemphigus
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
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
| Study group | Role | What they receive |
|---|---|---|
| Mycophenolate Mofetil (MMF) 2 g/Day | Experimental | Mycophenolate mofetil 500 mg tablets; 4 tablets twice daily for 52 weeks Receives: Drug: Mycophenolate Mofetil 2 g/Day |
| Mycophenolate Mofetil (MMF) 3 g/Day | Experimental | Mycophenolate mofetil 500 mg tablets; 6 tablets twice daily for 52 weeks Receives: Drug: Mycophenolate Mofetil (MMF) 3 g/Day |
| Placebo | Placebo comparator | Not described Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: 52 weeks
Secondary outcomes (3)
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)
Measured: 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.
Measured: 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.
Measured: 52 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 May 2008 (estimated)
- Study startMay 2004
- Primary completionOct 2008
- Study completionOct 2008
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Pemphigus Vulgaris (PV)Studies of the same condition, open and under way.
- More trials run by Hoffmann-La RocheThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 96 participants (actual)
Sponsor and investigators
- Lead sponsor
- Hoffmann-La Roche
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Aspreva Pharmaceuticals
- Investigators
- Clinical Trials, Study director, Hoffmann-La Roche
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00683930
- Sponsor's protocol ID
- WX17796
- Status last verified
- May 2011
- First submitted
- 19 May 2008
- Last updated
- 22 Jun 2011
- Results posted
- Yes, on ClinicalTrials.gov
Publications
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.
PubMed 20410913
Study at a glance
From the registry record, last updated by the sponsor on 22 Jun 2011. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT00683930
- Acronym / sponsor ID
- WX17796
- Conditions
- Pemphigus Vulgaris (PV)
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 96 participants (actual)
- Lead sponsor
- Hoffmann-La RocheIndustry (pharma, biotech, devices)
- Collaborators
- Aspreva Pharmaceuticals
- Study start
- May 2004
- Primary completion
- Oct 2008 (actual)
- Study completion
- Oct 2008 (actual)
- First posted
- 26 May 2008 (estimated)
- Results
- Posted 22 Jun 2011
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Mycophenolate Mofetil (MMF) 2 g/Day | Experimental | Drug: Mycophenolate Mofetil 2 g/DayMycophenolate mofetil 500 mg tablets; 4 tablets twice daily for 52 weeks |
| Mycophenolate Mofetil (MMF) 3 g/Day | Experimental | Drug: Mycophenolate Mofetil (MMF) 3 g/DayMycophenolate mofetil 500 mg tablets; 6 tablets twice daily for 52 weeks |
| Placebo | Placebo comparator | Drug: Placebo |
Who may qualify
- Ages
- 18 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (2 listed)
- Adult patients 18 to 70 years of age
- Diagnosis of mild to moderate pemphigus vulgaris within the past 12 months, requiring high dose corticosteroids
Key exclusion criteria (5 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Mycophenolate Mofetil 2 g/DayMycophenolate mofetil 500 mg tablets; 4 tablets twice daily for 52 weeks | Drug | Mycophenolate Mofetil (MMF) 2 g/Day |
| Mycophenolate Mofetil (MMF) 3 g/DayMycophenolate mofetil 500 mg tablets; 6 tablets twice daily for 52 weeks | Drug | Mycophenolate Mofetil (MMF) 3 g/Day |
| PlaceboPlacebo 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 | Drug | Placebo |
Primary outcome
Percentage of Patients Achieving Responder Status at Week 52
Time frame: 52 weeks
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
3 secondary outcomes
Time to Initial Response
Time frame: up to 52 weeks
Time to Sustained Response
Time frame: up to 52 weeks
Duration of Prednisone Maintenance Dosing
Time frame: 52 weeks
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Jun 2011.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 20410913Beissert 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Clinical Trials study director
Hoffmann-La Roche
26 sites in 8 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Mycophenolate Mofetil 2 g/Day and Mycophenolate Mofetil (MMF) 3 g/Day in people with Pemphigus Vulgaris (PV), run by Hoffmann-La Roche at 26 sites in 8 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 22 Jun 2011. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 26 sites in Germany, United States, Ukraine, Turkey (Türkiye), Canada and 3 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 22 Jun 2011). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Percentage of Patients Achieving Responder Status at Week 52 (time frame: 52 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00683930, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 22 Jun 2011.