Pemphigus VulgarisPhase 2NCT00429533
Efficacy of Dapsone as a Steroid Sparing Agent in Pemphigus Vulgaris
A Phase 2 study testing Dapsone in people with Pemphigus Vulgaris, run by Jacobus Pharmaceutical at 8 sites in United States.
Trial summary
The purpose of this 12-month study was to determine the efficacy of dapsone as a glucocorticoid-sparing agent in maintenance phase pemphigus vulgaris. Read more about this study
- Who is it for?
- Ages 18 to 80 years
- Conditions the study is for
- Pemphigus Vulgaris
- What is being tested?
- Dapsone
- How many people
- 48 participants
- Where
- 8 sitesUnited States
- Study dates
- Nov 1996to Feb 2004
- Phase
- Phase 2
- 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 to 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Histologic evidence compatible with pemphigus vulgaris and direct immunofluorescence evidence of pemphigus vulgaris.
- Chronic disease that has been controlled with steroids and/or cytotoxics, e.g. maintenance phase.
- On prednisone 15 or more mg/day to around 40 mg/day or on prednisone 15 or more mg every other day (qod) to around 40 mg qod.
- Failure to taper steroids below a range of 15 mg/day to around 40 mg/day or 15 mg/qod to around 40 qod without flaring the disease.
- The steroid dosage at which the most recent flare occurred should not be less than 85% of the last (within 30 days) dosage which controlled the disease, i.e. 85% of the baseline steroid dosage. This is to ensure that patients will not have had a recent acute flare at the time of entry into the study, and be in the rapid steroid taper portion of their disease after such a flare.
- Two baseline steroid dosages as determined by prior flares. It is common that patients will be repetitively unable to taper below a certain baseline steroid dose without experiencing a mild flare of their disease. This baseline dose will be determined on two occasions during attempted tapers, and the baseline number then averaged to determine the dose of steroid the patient is on at the time of entry into the study.
- No pulse steroids, pulse cyclosphosphamide, or plasmapheresis within two months of beginning the protocol. This will exclude patients who had recent acute flares of their disease and may be on the rapid steroid taper portion of their disease. The patient must be in maintenance phase, as defined in the criteria listed in e.
- Patient understands the procedures and agrees to participate in the study program by giving written informed consent.
Who cannot join
- Patients able to taper steroids without recurrence of disease.
- Patients with early, severe disease that have not responded to high doses of prednisone, cytotoxics, plasmapheresis, or other modalities.
- Contraindications to the use of Dapsone, including severe anemia or G6PD deficiency.
- Patient has behavioral problems that might interfere with compliance.
- Pregnancy or breast-feeding.
- Younger than 18 or older than 80 years of age. Since PV is rare in patients younger than 18, it was decided to exclude this potentially different population. It is unlikely that this will exclude many patients. Dapsone induces a hemolytic anemia, which would be a particular problem for patients over age 80, who are more likely to have ischemic heart disease or other atherosclerotic vascular disease.
- History of allergy to dapsone.
- Ischemic heart disease
Trial locations and contacts
8 sites in 1 country.
8 sites in 1 country
United States8 sites
Cooper Hospital/University Medical Center
Camden, New Jersey, 08103
Northwestern University Medical Center
Chicago, Illinois, 60611-3010
Rush-Presbyterian-St. Luke's Medical Center
Chicago, Illinois, 60612
Case Western Reserve University School of Medicine
Cleveland, Ohio, 44106
University of Texas
Dallas, Texas, 75235
Henry Ford Hospital
Detroit, Michigan, 48202
The New York VA Medical Center, New York University
New York, New York, 10010
University of Pennsylvania
Philadelphia, Pennsylvania, 19104
About this study
Official title: A Prospective Randomized Placebo-Controlled Clinical Trial of Dapsone as a Glucocorticoid-Sparing Agent in Maintenance Phase Pemphigus Vulgaris
The purpose of this 12-month study was to determine the efficacy of dapsone as a glucocorticoid-sparing agent in maintenance phase pemphigus vulgaris.
Read the detailed description
Patients were entered into the trial on steroids in combination with cytotoxic agents as needed. The steroid dose was the lowest dose at which the patient's disease was controlled before the last flare (see eligibility criteria). The patients were randomized to receive either Dapsone or placebo. Treatment was to be started at a dose of 50 mg and increased by 25 mg increments each week once the hemoglobin was shown not to have dropped by more than 2 gm/dl. The target dose was 150 mg and patients who did not respond could be advanced to 200 mg daily. After beginning treatment, a standardized steroid taper was commenced. A standardized steroid taper was suggested with tapering by 10 mg/wk for doses above 40 mg/day or more slowly if warranted. A slower taper thereafter or an every other day dosing schedule would be elected according to the individual investigator's preference. Flares were treated by increasing the dose of steroids - in the case of a mild flare to the last dose preceding the flare, in the case of a moderate flare by 20 mg/day and in the case of a severe flare by 40 mg/day. Tapering was to be resumed once the disease had stabilized. Disease activity was assessed by a simple scoring system for skin, mucosa, and sites involved. Laboratory assessments initially weekly became monthly once the study medication dosage was stabilized.
- Conditions
- Pemphigus Vulgaris
- Being tested
- Dapsone
- Medical subject headings
- Pemphigus
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
DapsoneDrug
What is being measured
The primary outcome is the main question the study is designed to answer.
The ability of patients to taper to ≤7.5mg/day within one year of reaching the maximum dosage of the study drug.
Secondary outcomes (1)
Steroid dosage reduced by more than 25% within 4 months after completing the upward titration of the study drug.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted31 Jan 2007 (estimated)
- Study startNov 1996
- Study completionFeb 2004
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Pemphigus VulgarisStudies of the same condition, open and under way.
- 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
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Double blind
- Enrolment
- 48 participants
Sponsor and investigators
- Lead sponsor
- Jacobus Pharmaceutical
- Sponsor type
- Industry (pharma, biotech, devices)
- Investigators
- Victoria P. Werth, MD, Study chair, University of PennsylvaniaVictoria P. Werth, MD, Principal investigator, University of PennsylvaniaDiana Chen, MD, Principal investigator, Northwestern UniversityWarren R Heymann, MD, Principal investigator, Cooper Hospital/University Medical CenterNeil Korman, MD, Principal investigator, Case Western Reserve University School of MedicineAmit Pandya, MD, Principal investigator, University of TexasM J Rico, MD, Principal investigator, The New York VA Medical Center - New York UniversityMichael D Tharp, MD, Principal investigator, Rush University Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00429533
- Sponsor's protocol ID
- 51,988
- Status last verified
- Feb 2007
- First submitted
- 29 Jan 2007
- Last updated
- 5 Feb 2007
- Results posted
- No
Publications
Werth VP, Fivenson D, Pandya AG, Chen D, Rico MJ, Albrecht J, Jacobus D. Multicenter randomized, double-blind, placebo-controlled, clinical trial of dapsone as a glucocorticoid-sparing agent in maintenance-phase pemphigus vulgaris. Arch Dermatol. 2008 Jan;144(1):25-32. doi: 10.1001/archderm.144.1.25.
PubMed 18209165
Study at a glance
From the registry record, last updated by the sponsor on 5 Feb 2007. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Prospective Randomized Placebo-Controlled Clinical Trial of Dapsone as a Glucocorticoid-Sparing Agent in Maintenance Phase Pemphigus Vulgaris
- ClinicalTrials.gov ID
- NCT00429533
- Acronym / sponsor ID
- 51,988
- Conditions
- Pemphigus Vulgaris
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Terminated
- Enrolment
- 48 participants
- Lead sponsor
- Jacobus PharmaceuticalIndustry (pharma, biotech, devices)
- Study start
- Nov 1996
- Study completion
- Feb 2004
- First posted
- 31 Jan 2007 (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.
The purpose of this 12-month study was to determine the efficacy of dapsone as a glucocorticoid-sparing agent in maintenance phase pemphigus vulgaris.
Detailed description
Patients were entered into the trial on steroids in combination with cytotoxic agents as needed. The steroid dose was the lowest dose at which the patient's disease was controlled before the last flare (see eligibility criteria). The patients were randomized to receive either Dapsone or placebo. Treatment was to be started at a dose of 50 mg and increased by 25 mg increments each week once the hemoglobin was shown not to have dropped by more than 2 gm/dl. The target dose was 150 mg and patients who did not respond could be advanced to 200 mg daily. After beginning treatment, a standardized steroid taper was commenced. A standardized steroid taper was suggested with tapering by 10 mg/wk for doses above 40 mg/day or more slowly if warranted. A slower taper thereafter or an every other day dosing schedule would be elected according to the individual investigator's preference. Flares were treated by increasing the dose of steroids - in the case of a mild flare to the last dose preceding the flare, in the case of a moderate flare by 20 mg/day and in the case of a severe flare by 40 mg/day. Tapering was to be resumed once the disease had stabilized. Disease activity was assessed by a simple scoring system for skin, mucosa, and sites involved. Laboratory assessments initially weekly became monthly once the study medication dosage was stabilized.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Double blind
Who may qualify
- Ages
- 18 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- Histologic evidence compatible with pemphigus vulgaris and direct immunofluorescence evidence of pemphigus vulgaris.
- Chronic disease that has been controlled with steroids and/or cytotoxics, e.g. maintenance phase.
- On prednisone 15 or more mg/day to around 40 mg/day or on prednisone 15 or more mg every other day (qod) to around 40 mg qod.
- Failure to taper steroids below a range of 15 mg/day to around 40 mg/day or 15 mg/qod to around 40 qod without flaring the disease.
- The steroid dosage at which the most recent flare occurred should not be less than 85% of the last (within 30 days) dosage which controlled the disease, i.e. 85% of the baseline steroid dosage. This is to ensure that patients will not have had a recent acute flare at the time of entry into the study, and be in the rapid steroid taper portion of their disease after such a flare.
Key exclusion criteria (8 listed)
- Patients able to taper steroids without recurrence of disease.
- Patients with early, severe disease that have not responded to high doses of prednisone, cytotoxics, plasmapheresis, or other modalities.
- Contraindications to the use of Dapsone, including severe anemia or G6PD deficiency.
- Patient has behavioral problems that might interfere with compliance.
- Pregnancy or breast-feeding.
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 |
|---|---|---|
| Dapsone | Drug | – |
Primary outcome
The ability of patients to taper to ≤7.5mg/day within one year of reaching the maximum dosage of the study drug.
1 secondary outcome
Steroid dosage reduced by more than 25% within 4 months after completing the upward titration of the study drug.
Results and safety
The study was stopped early. Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Feb 2007.
- Derived from this study: PubMed 18209165Werth VP, Fivenson D, Pandya AG, Chen D, Rico MJ, Albrecht J, Jacobus D. Multicenter randomized, double-blind, placebo-controlled, clinical trial of dapsone as a glucocorticoid-sparing agent in maintenance-phase pemphigus vulgaris. Arch Dermatol. 2008 Jan;144(1):25-32. doi: 10.1001/archderm.144.1.25.
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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.
Victoria P. Werth, MD study chair
University of Pennsylvania
Victoria P. Werth, MD principal investigator
University of Pennsylvania
Diana Chen, MD principal investigator
Northwestern University
8 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Dapsone in people with Pemphigus Vulgaris, run by Jacobus Pharmaceutical at 8 sites in United States.
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 8 inclusion and 8 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: stopped early, as of the sponsor's last update on 5 Feb 2007. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 8 sites in United States. 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 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.
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 was stopped early. Results are not available in the source record.
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 ability of patients to taper to ≤7.5mg/day within one year of reaching the maximum dosage of the study drug.. 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 NCT00429533, 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 5 Feb 2007.