PemphigusPhase 2/3NCT00127764
European Trial of Adjuvant Oral Glucocorticoid Pulse Therapy in Pemphigus
A Phase 2/3 study testing dexamethasone (50mg 1dd6, 3 consecutive days/month) in people with Pemphigus, run by University Medical Center Groningen at 1 site in Netherlands.
Trial summary
By this study, the investigators want to test if the value of adjuvant pulse glucocorticoid therapy for pemphigus can be determined by comparing an experimental arm (dexamethasone pulse therapy, prednisolone, and azathioprine) with a control arm (placebo pulse therapy, prednisolone, and azathioprine). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Pemphigus
- What is being tested?
- dexamethasone (50mg 1dd6, 3 consecutive days/month)
- How many people
- 60 participants
- Where
- 1 siteNetherlands
- Study dates
- Jan 2001to Apr 2005
- Phase
- Phase 2/3
- Are there results?
- None posted in the registry
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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- New patients with diagnosis of pemphigus vulgaris
Who cannot join
- Diagnosis of pemphigus different from vulgaris type
- Current use of adjuvant treatment other than azathioprine. A wash out period of at least 2 weeks is required before inclusion is acceptable.
- Presence of contra-indications for the use of high dose steroids
- No availability for follow-up.
- Concomitant diseases treated with oral steroids.
- Glucocorticoids administered for pemphigus vulgaris longer than two months.
- Presence of contra-indications for the use of azathioprine.
Trial locations and contacts
1 site in 1 country.
Netherlands1 site
Department of Dermatology, University Medical Center Groningen
Groningen, 9713 GZ
About this study
Official title: European Randomised Placebo-Controlled Trial of Adjuvant Oral Glucocorticoid Pulse Therapy in Pemphigus (Pempuls Trial)
By this study, the investigators want to test if the value of adjuvant pulse glucocorticoid therapy for pemphigus can be determined by comparing an experimental arm (dexamethasone pulse therapy, prednisolone, and azathioprine) with a control arm (placebo pulse therapy, prednisolone, and azathioprine). The investigators will determine the rate of complete remission with dexamethasone pulse therapy; the time needed for complete remission; and the duration of remission, and compare these data with those of the placebo arm.
Read the detailed description
Pemphigus is a severe chronic dermatological disease which appears to be incurable. Oral glucocorticoid now forms the cornerstone of treatment. From previous uncontrolled studies it has been suggested, that the use of high dose intravenous glucocorticoid pulse therapy may lead to complete remission without further therapy. Prospective placebo-controlled studies of adjuvant pulse therapy for pemphigus are lacking. This prospective, multi-centre, randomised, double-blind, placebo-controlled trial assesses the efficacy and steroid-sparing effect of oral high-dose glucocorticoid pulse therapy as adjuvant added to the standard combination therapy of prednisone and azathioprine in the treatment of pemphigus vulgaris, in terms of complete remission rate (primary endpoint), initial control, disease control, and adverse events. In the experimental arm glucocorticoid pulse therapy is given, comprising monthly pulses with 300mg oral dexamethasone on three consecutive days. In addition the patients receive daily prednisone and azathioprine treatment, the latter dose adjusted to the thiopurine-methyltransferase level. In the control arm the glucocorticoid pulse therapy is replaced double blindly by monthly oral placebo capsules on three consecutive days. in addition the patients receive the same daily combination schedule with prednisone and azathioprine as in the experimental arm. This study design requires 30 patients in each arm, with one year follow-up. Only new patients with pemphigus vulgaris and/or pemphigus oris are included.
- Conditions
- Pemphigus
- Being tested
- dexamethasone (50mg 1dd6, 3 consecutive days/month)
- Keywords
- pemphigus, dexamethasone, glucocorticoids, pulse, therapy, randomised, placebo-controlled
- Medical subject headings
- Pemphigus
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
dexamethasone (50mg 1dd6, 3 consecutive days/month)Drug
What is being measured
The primary outcome is the main question the study is designed to answer.
The rate of complete remission with pulse therapy
Number of weeks in complete remission after 12 months
Secondary outcomes (4)
Time from start of pulse therapy until initial control
Number of weeks in remission until 12 months
Cumulative prednisolone use until 12 months
Number of adverse events reported
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Aug 2005 (estimated)
- Study startJan 2001
- Study completionApr 2005
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (5)Papers the sponsor has linked to this study.
- Other trials for PemphigusStudies of the same condition, open and under way.
- More trials run by University Medical Center GroningenThe 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 2/3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Enrolment
- 60 participants
Sponsor and investigators
- Lead sponsor
- University Medical Center Groningen
- Sponsor type
- University, hospital or other
- Investigators
- Marcel F Jonkman, MD, PhD, Principal investigator, Department of Dermatology, University Medical Center Groningen
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00127764
- Sponsor's protocol ID
- 754309
- Status last verified
- Aug 2004
- First submitted
- 4 Aug 2005
- Last updated
- 30 Aug 2007
- Results posted
- No
Publications
Toth GG, van de Meer JB, Jonkman MF. Dexamethasone pulse therapy in pemphigus. J Eur Acad Dermatol Venereol. 2002 Nov;16(6):607-11. doi: 10.1046/j.1468-3083.2002.00413.x.
PubMed 12482045Toth GG, Westerlaken BO, Eilders M, Laseur M, Jonkman MF, Uges DR. Dexamethasone pharmacokinetics after high-dose oral therapy for pemphigus. Ann Pharmacother. 2002 Jun;36(6):1108-9. doi: 10.1345/aph.1A047. No abstract available.
PubMed 12058710Toth GG, Kloosterman C, Uges DR, Jonkman MF. Pharmacokinetics of high-dose oral and intravenous dexamethasone. Ther Drug Monit. 1999 Oct;21(5):532-5. doi: 10.1097/00007691-199910000-00007.
PubMed 10519450Toth GG, Jonkman MF. Therapy of pemphigus. Clin Dermatol. 2001 Nov-Dec;19(6):761-7. doi: 10.1016/s0738-081x(00)00190-5. No abstract available.
PubMed 11705686Mentink LF, Mackenzie MW, Toth GG, Laseur M, Lambert FP, Veeger NJ, Cianchini G, Pavlovic MD, Jonkman MF. Randomized controlled trial of adjuvant oral dexamethasone pulse therapy in pemphigus vulgaris: PEMPULS trial. Arch Dermatol. 2006 May;142(5):570-6. doi: 10.1001/archderm.142.5.570.
PubMed 16702494
Study at a glance
From the registry record, last updated by the sponsor on 30 Aug 2007. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- European Randomised Placebo-Controlled Trial of Adjuvant Oral Glucocorticoid Pulse Therapy in Pemphigus (Pempuls Trial)
- ClinicalTrials.gov ID
- NCT00127764
- Acronym / sponsor ID
- 754309
- Conditions
- Pemphigus
- Keywords
- pemphigus, dexamethasone, glucocorticoids, pulse, therapy, randomised, placebo-controlled
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 60 participants
- Lead sponsor
- University Medical Center GroningenUniversity, hospital or other
- Study start
- Jan 2001
- Study completion
- Apr 2005
- First posted
- 8 Aug 2005 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
By this study, the investigators want to test if the value of adjuvant pulse glucocorticoid therapy for pemphigus can be determined by comparing an experimental arm (dexamethasone pulse therapy, prednisolone, and azathioprine) with a control arm (placebo pulse therapy, prednisolone, and azathioprine). The investigators will determine the rate of complete remission with dexamethasone pulse therapy; the time needed for complete remission; and the duration of remission, and compare these data with those of the placebo arm.
Detailed description
Pemphigus is a severe chronic dermatological disease which appears to be incurable. Oral glucocorticoid now forms the cornerstone of treatment. From previous uncontrolled studies it has been suggested, that the use of high dose intravenous glucocorticoid pulse therapy may lead to complete remission without further therapy. Prospective placebo-controlled studies of adjuvant pulse therapy for pemphigus are lacking. This prospective, multi-centre, randomised, double-blind, placebo-controlled trial assesses the efficacy and steroid-sparing effect of oral high-dose glucocorticoid pulse therapy as adjuvant added to the standard combination therapy of prednisone and azathioprine in the treatment of pemphigus vulgaris, in terms of complete remission rate (primary endpoint), initial control, disease control, and adverse events. In the experimental arm glucocorticoid pulse therapy is given, comprising monthly pulses with 300mg oral dexamethasone on three consecutive days. In addition the patients receive daily prednisone and azathioprine treatment, the latter dose adjusted to the thiopurine-methyltransferase level. In the control arm the glucocorticoid pulse therapy is replaced double blindly by monthly oral placebo capsules on three consecutive days. in addition the patients receive the same daily combination schedule with prednisone and azathioprine as in the experimental arm. This study design requires 30 patients in each arm, with one year follow-up. Only new patients with pemphigus vulgaris and/or pemphigus oris are included.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (1 listed)
- New patients with diagnosis of pemphigus vulgaris
Key exclusion criteria (7 listed)
- Diagnosis of pemphigus different from vulgaris type
- Current use of adjuvant treatment other than azathioprine. A wash out period of at least 2 weeks is required before inclusion is acceptable.
- Presence of contra-indications for the use of high dose steroids
- No availability for follow-up.
- Concomitant diseases treated with oral steroids.
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 |
|---|---|---|
| dexamethasone (50mg 1dd6, 3 consecutive days/month) | Drug | – |
Primary outcome
The rate of complete remission with pulse therapy
Number of weeks in complete remission after 12 months
4 secondary outcomes
Time from start of pulse therapy until initial control
Number of weeks in remission until 12 months
Cumulative prednisolone use until 12 months
Number of adverse events reported
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 Aug 2007.
- Background: PubMed 12482045Toth GG, van de Meer JB, Jonkman MF. Dexamethasone pulse therapy in pemphigus. J Eur Acad Dermatol Venereol. 2002 Nov;16(6):607-11. doi: 10.1046/j.1468-3083.2002.00413.x.
- Background: PubMed 12058710Toth GG, Westerlaken BO, Eilders M, Laseur M, Jonkman MF, Uges DR. Dexamethasone pharmacokinetics after high-dose oral therapy for pemphigus. Ann Pharmacother. 2002 Jun;36(6):1108-9. doi: 10.1345/aph.1A047. No abstract available.
- Background: PubMed 10519450Toth GG, Kloosterman C, Uges DR, Jonkman MF. Pharmacokinetics of high-dose oral and intravenous dexamethasone. Ther Drug Monit. 1999 Oct;21(5):532-5. doi: 10.1097/00007691-199910000-00007.
- Background: PubMed 11705686Toth GG, Jonkman MF. Therapy of pemphigus. Clin Dermatol. 2001 Nov-Dec;19(6):761-7. doi: 10.1016/s0738-081x(00)00190-5. No abstract available.
- Results publication: PubMed 16702494Mentink LF, Mackenzie MW, Toth GG, Laseur M, Lambert FP, Veeger NJ, Cianchini G, Pavlovic MD, Jonkman MF. Randomized controlled trial of adjuvant oral dexamethasone pulse therapy in pemphigus vulgaris: PEMPULS trial. Arch Dermatol. 2006 May;142(5):570-6. doi: 10.1001/archderm.142.5.570.
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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.
Marcel F Jonkman, MD, PhD principal investigator
Department of Dermatology, University Medical Center Groningen
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing dexamethasone (50mg 1dd6, 3 consecutive days/month) in people with Pemphigus, run by University Medical Center Groningen at 1 site in Netherlands.
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 1 inclusion and 7 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 30 Aug 2007. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Netherlands. 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. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
The rate of complete remission with pulse therapy. The study lists 2 primary outcomes in all. 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 NCT00127764, which also lists 5 linked publications.
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 30 Aug 2007.