European Trial of Adjuvant Oral Glucocorticoid Pulse Therapy in Pemphigus

Pemphigus Clinical Trial

Official title:

European Randomised Placebo-Controlled Trial of Adjuvant Oral Glucocorticoid Pulse Therapy in Pemphigus (Pempuls Trial)

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00127764
• Other study ID #: 754309
• Status: Completed
• Phase: Phase 2/Phase 3
• First received: August 4, 2005
• Last updated: August 29, 2007
• Start date: January 2001
• Est. completion date: April 2005

Study information

• Verified date: August 2004
• Source: University Medical Center Groningen
• Contact: n/a
• Is FDA regulated: No
• Health authority: Netherlands: The Central Committee on Research Involving Human Subjects (CCMO)
• Study type: Interventional

Clinical 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). 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 60
• Est. completion date: April 2005
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 18 Years and older

Eligibility

Inclusion Criteria:

• New patients with diagnosis of pemphigus vulgaris

Exclusion Criteria:

• 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.

Study Design

Allocation: Randomized, Endpoint Classification: Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double-Blind, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Pemphigus

Intervention

Drug:
• dexamethasone (50mg 1dd6, 3 consecutive days/month)

Locations

Country	Name	City	State
Netherlands	Department of Dermatology, University Medical Center Groningen	Groningen

Sponsors (1)

Lead Sponsor	Collaborator
University Medical Center Groningen

Country where clinical trial is conducted

Netherlands, 

References & Publications (5)

Mentink LF, Mackenzie MW, Tóth 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. — View Citation

Tóth GG, Jonkman MF. Therapy of pemphigus. Clin Dermatol. 2001 Nov-Dec;19(6):761-7. Review. — View Citation

Tóth GG, Kloosterman C, Uges DR, Jonkman MF. Pharmacokinetics of high-dose oral and intravenous dexamethasone. Ther Drug Monit. 1999 Oct;21(5):532-5. — View Citation

Tóth GG, van de Meer JB, Jonkman MF. Dexamethasone pulse therapy in pemphigus. J Eur Acad Dermatol Venereol. 2002 Nov;16(6):607-11. — View Citation

Tóth 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. — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	The rate of complete remission with pulse therapy
Primary	Number of weeks in complete remission after 12 months
Secondary	Time from start of pulse therapy until initial control
Secondary	Number of weeks in remission until 12 months
Secondary	Cumulative prednisolone use until 12 months
Secondary	Number of adverse events reported