PemphigusPhase 2NCT00283712
Use of Infliximab for the Treatment of Pemphigus Vulgaris
A Phase 2 study testing Infliximab in people with Pemphigus, run by National Institute of Allergy and Infectious Diseases (NIAID) at 4 sites in United States.
Trial summary
Pemphigus vulgaris (PV) is a rare skin disorder that causes blistering of the skin and mucous membranes. Infliximab is a man-made antibody used to treat certain types of immune system disorders, including rheumatoid arthritis and Crohn's disease. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Pemphigus
- What is being tested?
- Infliximab
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 20 participants
- Where
- 4 sitesUnited States
- Study dates
- Mar 2006to Mar 2011
- Phase
- Phase 2
- 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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Positive direct immunofluorescence of patient's skin showing IgG or complement C3 protein on cell surface with histopathology of lesional skin biopsies consistent with diagnosis of pemphigus vulgaris
- Failure to completely respond to standard steroid therapy (equivalent to prednisone 1 to 2 mg/kg/day followed by tapering)
- Systemic corticosteroid therapy of at least 20 mg prednisone daily and no more than 120 mg/day
- Inability to reduce systemic corticosteroid dosage below 20 mg/day for at least 8 weeks
- Stable dosage of prednisone for at least 2 weeks prior to study entry
- Oral/mucosal disease or skin disease. Detailed information about this criterion can be found in the protocol
- Willing to comply with the study protocol
- Willing to use acceptable means of contraception for the duration of the study and for 6 months after the end of the study
Who cannot join
- Positive tuberculosis (TB) test within 1 month prior to first administration of study drug
- History of latent or active TB prior to screening
- Signs or symptoms suggestive of TB disease by medical history or physical examination within 3 months prior to first administration of study drug
- Posterior/anterior/lateral chest radiograph within 3 months prior to screening showing evidence of cancer, infection, or abnormalities (apical scarring) suggestive of previous TB
- Serious infection, hospitalization for an infection, or treatment with intravenous (IV) antibiotics for an infection within 2 months prior to screening. Patients who have had less serious infections are eligible for this study at the discretion of the investigator.
- History or presence of opportunistic infections within 6 months prior to screening
Show all 33Show fewer
- History of receiving human/murine recombinant products
- Known allergy to murine products or other chimeric proteins
- Human immunodeficiency virus (HIV) infected
- Chronic hepatitis B or hepatitis C virus infection
- History of hepatitis C virus infection
- Cancer within the 5 years prior to study entry. Patients with completely resected non-melanoma skin cancers are not excluded.
- History or presence of congestive heart failure
- History or presence of seizure or demyelinating disorder
- History of latent or active granulomatous infection, including TB, histoplasmosis, or coccidioidomycosis
- Received a Bacillus Calmette-Guerin (BCG) vaccine within 12 months of screening
- History of lymphoproliferative disease, including lymphoma or signs and symptoms of possible lymphoproliferative disease, such as lymphadenopathy of unusual size or location or enlarged spleen
- Current signs or symptoms of severe progressive or uncontrolled kidney, liver, blood, gastrointestinal, endocrine, lung, heart, neurologic, or cerebral disease
- Have had chronic or recurrent infectious disease including, but not limited to, chronic kidney infection, chronic chest infection, sinusitis, recurrent urinary tract infection, infected skin wound, or ulcer
- Previous treatment with infliximab, other monoclonal antibodies, or antibody fragments
- Previous treatment with etanercept or other anti-tumor necrosis factor (TNF) agents in the 3 months prior to screening
- Treatment with methotrexate, azathioprine, mycophenolate mofetil, plasmapheresis, IV immunoglobulin, pulse systemic corticosteroids, or other systemic immunosuppressive agents within the 4 weeks prior to study entry
- History of alcohol or drug abuse within the 3 years prior to study entry
- History of noncompliance to medical regimens
- History of a systemic inflammatory disease other than pemphigus vulgaris
- History of a medical condition that would interfere with participation or increase the risk to the participant
- Unable or unwilling to undergo blood draws because of poor tolerability or lack of easy access
- Use of any investigational drug within 30 days prior to screening OR within 5 half-lives of the investigational agent, whichever is longer
- Participation in another investigative clinical trial
- Presence of transplanted solid organ. Participants who have received a corneal transplant more than 3 months prior to screening are not excluded.
- Require certain medications
- Other conditions or circumstances that could interfere with participant's adherence to the study requirements
- Pregnancy, breastfeeding, or plans to become pregnant
Trial locations and contacts
4 sites in 1 country.
United States4 sites
Duke University Medical Center
Durham, North Carolina, 27710
University of Iowa Hospitals and Clinics
Iowa City, Iowa, 52242
Norris Cancer Center, University of Southern California
Los Angeles, California, 90033
University of Pennsylvania
Philadelphia, Pennsylvania, 19104
About this study
Official title: A Randomized, Double-Blind, Placebo-Controlled Phase II Trial of Infliximab in Subjects With Pemphigus Vulgaris Receiving Prednisone
Pemphigus vulgaris (PV) is a rare skin disorder that causes blistering of the skin and mucous membranes. Infliximab is a man-made antibody used to treat certain types of immune system disorders, including rheumatoid arthritis and Crohn's disease. This study will determine if infliximab given in combination with prednisone is a safe and effective treatment for adults with PV.
Read the detailed description
PV involves blistering of the outer layer of skin and mucous membranes, causing a separation of epidermal cells. The disease occurs when the immune system produces antibodies to specific proteins in the skin and mucous membranes; the cause for production of these autoantibodies is unknown. Infliximab is a genetically engineered monoclonal antibody directed against tumor necrosis factor (TNF)-alpha, a chemical messenger that activates an immune response. Infliximab has been used to treat other autoimmune disorders, including rheumatoid arthritis, ankylosing spondylitis, and Crohn's disease. This study will evaluate the safety and efficacy of infliximab given in combination with prednisone for the treatment of adults with PV.
This study will last 26 weeks. At study entry, all patients will be taking a stable dose of prednisone (or an equivalent corticosteroid) of 20 to 120 mg/day for at least 2 weeks prior to study entry. Patients will be randomly assigned to one of two arms: experimental or placebo comparator. The experimental treatment arm will receive infusions of infliximab, and the control arm will receive placebo. Infusions will be given at study entry and Weeks 2, 6, and 14. Before the start of each infusion, a physical exam, vital signs measurement, medical and medication history, review of a disease activity log, a skin evaluation, and blood collection will occur. During each infusion and for 1 hour postinfusion, patients' vital signs will be monitored for any adverse events. Patients will need a responsible adult to take them home after they are discharged from the treatment facility; this person should remain with the patient overnight in case any problems arise from the treatment. The patient will be contacted by phone that night and the next morning after infusion and will be asked about any adverse effects they may have experienced. Those patients that experience adverse effects may be asked to return to the treatment facility for examination. Prednisone doses may be tapered by 15 percent every 2 weeks during the study at the investigator's discretion.
There will be a total of 9 study visits until Week 26: screening, study entry, Week 2, and every 4 weeks thereafter. Each study visit will include a physical exam, vital signs measurement, medical and medication history, a review of the disease activity log and adverse events experienced since the last visit, skin assessments, and blood collection; patients will also be asked to complete a tuberculosis (TB) questionnaire. Patients will be asked to complete quality of life questionnaires at study entry and Weeks 10, 18, and 26. Skin biopsies of unaffected skin will be done at study entry and Weeks 10, 18, and 26; if patients have PV-associated lesions, additional skin biopsies of affected skin will be done at study entry and Week 18.
- Conditions
- Pemphigus
- Being tested
- Infliximab, Placebo Comparator
- Keywords
- Skin Diseases, Autoimmune Diseases
- Medical subject headings
- Pemphigus, Skin Diseases, Autoimmune Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
InfliximabDrug
Also called Remicade®
Chimeric IgG monoclonal antibody that binds to TNF-alpha, generally used to treat Crohn's disease, given in a dosage of 5 mg/kg
Placebo ComparatorOther
Also called Control Arm
Placebo administered in place of infliximab for control group
| Study group | Role | What they receive |
|---|---|---|
| Infliximab | Experimental | Participants are randomized to receive intravenous infusions of infliximab (5mg/kg reconstituted in 10 mL of Sterile Water for Injection, USP ) at Weeks 0, 2, 6, and 14 over a time period of no less than two hours in a masked (blinded) fashion. Refer to section titled, "Detailed Description" for additional treatment information. Receives: Drug: Infliximab |
| Placebo Comparator | Placebo comparator | Participants are randomized to receive intravenous infusions of placebo (5 mg/kg comprised of a white lyophilized powder reconstituted in 10 mL of Sterile Water for Injection, USP) at Weeks 0, 2, 6, and 14 over a time period of no less than two hours in a masked (blinded) fashion. Refer to section titled, "Detailed Description" for additional treatment information. Receives: Other: Placebo Comparator |
What is being measured
The primary outcome is the main question the study is designed to answer.
Participant Response to Treatment at Week 18
Participants classified as responders at Week 18 had: 1. Achieved a prednisone dosage <= 25% of the initial starting dose or <= 10 mg/day (whichever is greater), and 2. Had no new blisters within the previous 4 weeks.
Measured: Baseline to Week 18
Treatment-Related Adverse Events >= Grade 3 On or Before Week 18
Grades were based on the National Cancer Institute (NCI), Common Terminology Criteria for Adverse Events (NCI-CTCAE), Version 3.0. An adverse event (AE) was considered treatment-related if it was classified as unlikely, possibly, probably, or definitely related to study treatment. Participants who experienced at least one treatment-related, grade 3 or higher AE were counted only once. AEs of skin including rash, skin ulceration, and chelitis as defined by the NCI-CTCAE V3.0 System Organ Class of "Skin and Subcutaneous Tissues Disorders" were excluded.
Measured: Baseline to Week 18
Secondary outcomes (13)
Participant Response to Treatment at Week 18
Participants classified as responders at Week 18 had: 1. Achieved a prednisone dosage <= 25% of the initial starting dose or <=10 mg/day (whichever is greater), and 2. Had no new blisters within the previous 4 weeks.
Measured: Baseline to Week 18
Participant Modified Response Status at Week 18
Modified responder status was defined as participants achieving a prednisone dosage <=25% of the initial starting dose or <=10 mg/day (whichever is greater) at Week 18 regardless of status on new blister formation during the previous 4 weeks.
Measured: Baseline to Week 18
Participant Time to Cessation of New Blisters
Time to cessation of new blisters was defined as the time from a participant's first treatment infusion date to the first date where that date and all subsequent dates had no new blisters. Participant diaries were used to assess new blister formation. To achieve cessation, participants had to be free of new blisters at least 3 weeks prior to their last assessment. In order to analyze missing or incomplete data, the data was censored at the date where a participant had no more data or on the date where 50% of the participant's data was missing past that point.
Measured: Baseline to Week 26
Time to 80% Lesion Healing
Time to 80% healing of existing erosions/ulcerations at time of enrollment was assessed using the SAGE II computerized burn-mapping system. The date of 80% healing of existing erosions/ulcerations at time of enrollment was defined as follows: the first date at which the percent of total body surface area (BSA) involved is at least 80% less than the percent of total BSA calculated at the time of enrollment, where the baseline percent of total BSA must be greater than zero percent. If a participant had missing post-baseline assessments, their data was censored at their last non-missing assessment date.
Measured: Baseline to Week 26
Total Prednisone Dosage Required for Participants to Achieve Cessation of New Blisters
Each participant's prednisone dose was summed from the time of enrollment until the date of cessation of new blisters. Actual prednisone use per day was computed as the average over all days in the week.
Measured: Baseline to Week 26
Total Prednisone Dosage Required for Participants to Achieve 80% Healing of Existing Erosions
Each participant's prednisone dose was summed from the time of enrollment until the date of 80% healing of existing erosions. Actual prednisone use per day was computed as the average over all days in the week.
Measured: Baseline to Week 26
Participant Health Related Quality of Life (Medical Outcome Study Short Form 36) Score Changes From Baseline to Week 18
The Medical Outcome Study Short Form 36 (MOS SF-36) measures health -related quality of life across multiple disease states. It has 36 questions with 8 subscale scores and 2 summary scores: PCS=physical functioning, role-physical, bodily pain, and general health; MCS=vitality, social functioning, role-emotional, and mental health. Scoring is done for both subscores and summary scores. For both, 0=worst score (or quality of life) and 100=best score. Change from baseline is computed as the value at Week 18 minus the baseline value. A positive value in change from Baseline indicates an improvement and a negative value worsening.
Measured: Baseline to Week 18
Participant Dermatology-Related Quality of Life Changes From Baseline to Week 18
The Dermatology Life Quality Index (DLQI) is a 10-question questionnaire with a weighted value to each question. The DLQI score was calculated by summing the score of each question, resulting in a maximum score of 30 and a minimum score of 0. The higher the score, the greater quality of life is impaired. Change from baseline values (defined as the visit value - baseline value) were calculated. A negative change indicates better quality of life; a positive change indicates poorer quality of life.
Measured: Baseline to Week 18
Participant Duration of Clinical Response
The primary efficacy endpoint of response to treatment at Week 18 was reassessed at study weeks 22 and 26 for participants who were responders at Week 18. Participants classified as responders had: 1. Achieved a prednisone dosage <= 25% of the initial starting dose or <= 10 mg/day (whichever is greater), and 2. had no new blisters within the previous 4 weeks.
Measured: Baseline to Week 26
Participants Who Experienced Severe Infusion Reactions
Participants who experienced severe infusion reactions of Grade 3 or greater based on the National Cancer Institute (NCI), Common Terminology Criteria for Adverse Events (NCI-CTCAE), Version 3.0 were assessed.
Measured: Baseline to Week 26
Participants Who Experienced Severe Infectious Complications
Serious and life-threatening infections of Grade 3 or greater based on the National Cancer Institute (NCI), Common Terminology Criteria for Adverse Events (NCI-CTCAE), Version 3.0 were assessed.
Measured: Baseline to Week 26
Adverse Events Resulting in Treatment Discontinuation
Adverse events experienced by participants resulting in study treatment discontinuation and assessed by the investigators as at least possibly related to treatment (i.e., possibly, probably, definitely) were assessed.
Measured: Baseline to Week 26
Participant Pemphigus Vulgaris Disease Activity Score
The Pemphigus Vulgaris Disease Activity (PVDA) score was used to grade a participant's disease activity using the SAGE II computerized burn mapping system, which calculated the total body surface area (BSA) involved. Scores were based on the number of new lesions and blisters present, old lesion history and BSA involved. Scores range from 0 to 3 (none to severe disease activity). A new disease activity score of 3 or an old lesion score of 3 indicates active disease. New disease activity scores of 3 for a 1-month duration or an old lesion score of 3 for 2 consecutive months was cause for removal from the study treatment
Measured: Baseline to Week 26
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 Jan 2006 (estimated)
- Study startMar 2006
- Primary completionDec 2010
- Study completionMar 2011
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 (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 National Institute of Allergy and Infectious Diseases (NIAID)The 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
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Care provider
- Enrolment
- 20 participants (actual)
Sponsor and investigators
- Sponsor type
- US National Institutes of Health
- Collaborators
- Autoimmunity Centers of Excellence
- Responsible party
- Sponsor
- Investigators
- Russell P. Hall, MD, Study chair, Division of Dermatology, Duke University Medical CenterE. William St. Clair, MD, Study chair, Division of Rheumatology and Immunology, Duke University Medical CenterGarnett Kelsoe, DSci, Principal investigator, Department of Immunology, Duke UniversityVictoria Werth, MD, Principal investigator, Department of Dermatology, University of Pennsylvania School of MedicineJanet Fairley, MD, Principal investigator, Department of Dermatology, University of IowaDavid Woodley, MD, Principal investigator, Department of Dermatology, Norris Cancer Center, University of Southern California
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00283712
- Sponsor's protocol ID
- DAIT APV01
- Status last verified
- Nov 2017
- First submitted
- 26 Jan 2006
- Last updated
- 6 Dec 2017
- Results posted
- Yes, on ClinicalTrials.gov
Publications
Anhalt GJ, Diaz LA. Pemphigus vulgaris--a model for cutaneous autoimmunity. J Am Acad Dermatol. 2004 Jul;51(1 Suppl):S20-1. doi: 10.1016/j.jaad.2004.01.011. No abstract available.
PubMed 15243495Drosou A, Kirsner RS, Welsh E, Sullivan TP, Kerdel FA. Use of infliximab, an anti-tumor necrosis alpha antibody, for inflammatory dermatoses. J Cutan Med Surg. 2003 Sep-Oct;7(5):382-6. doi: 10.1007/s10227-002-0134-1. Epub 2003 Sep 24.
PubMed 14973643Jacobi A, Shuler G, Hertl M. Rapid control of therapy-refractory pemphigus vulgaris by treatment with the tumour necrosis factor-alpha inhibitor infliximab. Br J Dermatol. 2005 Aug;153(2):448-9. doi: 10.1111/j.1365-2133.2005.06744.x. No abstract available.
PubMed 16086769Pardo J, Mercader P, Mahiques L, Sanchez-Carazo JL, Oliver V, Fortea JM. Infliximab in the management of severe pemphigus vulgaris. Br J Dermatol. 2005 Jul;153(1):222-3. doi: 10.1111/j.1365-2133.2005.06672.x. No abstract available.
PubMed 16029365Hall RP 3rd, Fairley J, Woodley D, Werth VP, Hannah D, Streilein RD, McKillip J, Okawa J, Rose M, Keyes-Elstein LL, Pinckney A, Overington A, Wedgwood J, Ding L, Welch B. A multicentre randomized trial of the treatment of patients with pemphigus vulgaris with infliximab and prednisone compared with prednisone alone. Br J Dermatol. 2015 Mar;172(3):760-8. doi: 10.1111/bjd.13350. Epub 2015 Feb 5.
PubMed 25123295
Study at a glance
From the registry record, last updated by the sponsor on 6 Dec 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Double-Blind, Placebo-Controlled Phase II Trial of Infliximab in Subjects With Pemphigus Vulgaris Receiving Prednisone
- ClinicalTrials.gov ID
- NCT00283712
- Acronym / sponsor ID
- DAIT APV01
- Conditions
- Pemphigus
- Keywords
- Skin Diseases, Autoimmune Diseases
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 20 participants (actual)
- Lead sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)US National Institutes of Health
- Collaborators
- Autoimmunity Centers of Excellence
- Study start
- Mar 2006
- Primary completion
- Dec 2010 (actual)
- Study completion
- Mar 2011 (actual)
- First posted
- 30 Jan 2006 (estimated)
- Results
- Posted 31 Jan 2013
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Pemphigus vulgaris (PV) is a rare skin disorder that causes blistering of the skin and mucous membranes. Infliximab is a man-made antibody used to treat certain types of immune system disorders, including rheumatoid arthritis and Crohn's disease. This study will determine if infliximab given in combination with prednisone is a safe and effective treatment for adults with PV.
Detailed description
PV involves blistering of the outer layer of skin and mucous membranes, causing a separation of epidermal cells. The disease occurs when the immune system produces antibodies to specific proteins in the skin and mucous membranes; the cause for production of these autoantibodies is unknown. Infliximab is a genetically engineered monoclonal antibody directed against tumor necrosis factor (TNF)-alpha, a chemical messenger that activates an immune response. Infliximab has been used to treat other autoimmune disorders, including rheumatoid arthritis, ankylosing spondylitis, and Crohn's disease. This study will evaluate the safety and efficacy of infliximab given in combination with prednisone for the treatment of adults with PV.
This study will last 26 weeks. At study entry, all patients will be taking a stable dose of prednisone (or an equivalent corticosteroid) of 20 to 120 mg/day for at least 2 weeks prior to study entry. Patients will be randomly assigned to one of two arms: experimental or placebo comparator. The experimental treatment arm will receive infusions of infliximab, and the control arm will receive placebo. Infusions will be given at study entry and Weeks 2, 6, and 14. Before the start of each infusion, a physical exam, vital signs measurement, medical and medication history, review of a disease activity log, a skin evaluation, and blood collection will occur. During each infusion and for 1 hour postinfusion, patients' vital signs will be monitored for any adverse events. Patients will need a responsible adult to take them home after they are discharged from the treatment facility; this person should remain with the patient overnight in case any problems arise from the treatment. The patient will be contacted by phone that night and the next morning after infusion and will be asked about any adverse effects they may have experienced. Those patients that experience adverse effects may be asked to return to the treatment facility for examination. Prednisone doses may be tapered by 15 percent every 2 weeks during the study at the investigator's discretion.
There will be a total of 9 study visits until Week 26: screening, study entry, Week 2, and every 4 weeks thereafter. Each study visit will include a physical exam, vital signs measurement, medical and medication history, a review of the disease activity log and adverse events experienced since the last visit, skin assessments, and blood collection; patients will also be asked to complete a tuberculosis (TB) questionnaire. Patients will be asked to complete quality of life questionnaires at study entry and Weeks 10, 18, and 26. Skin biopsies of unaffected skin will be done at study entry and Weeks 10, 18, and 26; if patients have PV-associated lesions, additional skin biopsies of affected skin will be done at study entry and Week 18.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, care provider)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Infliximab | Experimental | Drug: InfliximabParticipants are randomized to receive intravenous infusions of infliximab (5mg/kg reconstituted in 10 mL of Sterile Water for Injection, USP ) at Weeks 0, 2, 6, and 14 over a time period of no less than two hours in a masked (blinded) fashion. Refer to section titled, "Detailed Description" for additional treatment information. |
| Placebo Comparator | Placebo comparator | Other: Placebo ComparatorParticipants are randomized to receive intravenous infusions of placebo (5 mg/kg comprised of a white lyophilized powder reconstituted in 10 mL of Sterile Water for Injection, USP) at Weeks 0, 2, 6, and 14 over a time period of no less than two hours in a masked (blinded) fashion. Refer to section titled, "Detailed Description" for additional treatment information. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- Positive direct immunofluorescence of patient's skin showing IgG or complement C3 protein on cell surface with histopathology of lesional skin biopsies consistent with diagnosis of pemphigus vulgaris
- Failure to completely respond to standard steroid therapy (equivalent to prednisone 1 to 2 mg/kg/day followed by tapering)
- Systemic corticosteroid therapy of at least 20 mg prednisone daily and no more than 120 mg/day
- Inability to reduce systemic corticosteroid dosage below 20 mg/day for at least 8 weeks
- Stable dosage of prednisone for at least 2 weeks prior to study entry
Key exclusion criteria (33 listed)
- Positive tuberculosis (TB) test within 1 month prior to first administration of study drug
- History of latent or active TB prior to screening
- Signs or symptoms suggestive of TB disease by medical history or physical examination within 3 months prior to first administration of study drug
- Posterior/anterior/lateral chest radiograph within 3 months prior to screening showing evidence of cancer, infection, or abnormalities (apical scarring) suggestive of previous TB
- Serious infection, hospitalization for an infection, or treatment with intravenous (IV) antibiotics for an infection within 2 months prior to screening. Patients who have had less serious infections are eligible for this study at the discretion of the investigator.
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 |
|---|---|---|
| InfliximabAlso: Remicade®Chimeric IgG monoclonal antibody that binds to TNF-alpha, generally used to treat Crohn's disease, given in a dosage of 5 mg/kg | Drug | Infliximab |
| Placebo ComparatorAlso: Control ArmPlacebo administered in place of infliximab for control group | Other | Placebo Comparator |
Primary outcome
Participant Response to Treatment at Week 18
Time frame: Baseline to Week 18
Participants classified as responders at Week 18 had: 1. Achieved a prednisone dosage <= 25% of the initial starting dose or <= 10 mg/day (whichever is greater), and 2. Had no new blisters within the previous 4 weeks.
Treatment-Related Adverse Events >= Grade 3 On or Before Week 18
Time frame: Baseline to Week 18
Grades were based on the National Cancer Institute (NCI), Common Terminology Criteria for Adverse Events (NCI-CTCAE), Version 3.0. An adverse event (AE) was considered treatment-related if it was classified as unlikely, possibly, probably, or definitely related to study treatment. Participants who experienced at least one treatment-related, grade 3 or higher AE were counted only once. AEs of skin including rash, skin ulceration, and chelitis as defined by the NCI-CTCAE V3.0 System Organ Class of "Skin and Subcutaneous Tissues Disorders" were excluded.
13 secondary outcomes
Participant Response to Treatment at Week 18
Time frame: Baseline to Week 18
Participant Modified Response Status at Week 18
Time frame: Baseline to Week 18
Participant Time to Cessation of New Blisters
Time frame: Baseline to Week 26
Time to 80% Lesion Healing
Time frame: Baseline to Week 26
Total Prednisone Dosage Required for Participants to Achieve Cessation of New Blisters
Time frame: Baseline to Week 26
Total Prednisone Dosage Required for Participants to Achieve 80% Healing of Existing Erosions
Time frame: Baseline to Week 26
Participant Health Related Quality of Life (Medical Outcome Study Short Form 36) Score Changes From Baseline to Week 18
Time frame: Baseline to Week 18
Participant Dermatology-Related Quality of Life Changes From Baseline to Week 18
Time frame: Baseline to Week 18
Participant Duration of Clinical Response
Time frame: Baseline to Week 26
Participants Who Experienced Severe Infusion Reactions
Time frame: Baseline to Week 26
Participants Who Experienced Severe Infectious Complications
Time frame: Baseline to Week 26
Adverse Events Resulting in Treatment Discontinuation
Time frame: Baseline to Week 26
Participant Pemphigus Vulgaris Disease Activity Score
Time frame: Baseline to Week 26
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 Dec 2017.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Background: PubMed 15243495Anhalt GJ, Diaz LA. Pemphigus vulgaris--a model for cutaneous autoimmunity. J Am Acad Dermatol. 2004 Jul;51(1 Suppl):S20-1. doi: 10.1016/j.jaad.2004.01.011. No abstract available.
- Background: PubMed 14973643Drosou A, Kirsner RS, Welsh E, Sullivan TP, Kerdel FA. Use of infliximab, an anti-tumor necrosis alpha antibody, for inflammatory dermatoses. J Cutan Med Surg. 2003 Sep-Oct;7(5):382-6. doi: 10.1007/s10227-002-0134-1. Epub 2003 Sep 24.
- Background: PubMed 16086769Jacobi A, Shuler G, Hertl M. Rapid control of therapy-refractory pemphigus vulgaris by treatment with the tumour necrosis factor-alpha inhibitor infliximab. Br J Dermatol. 2005 Aug;153(2):448-9. doi: 10.1111/j.1365-2133.2005.06744.x. No abstract available.
- Background: PubMed 16029365Pardo J, Mercader P, Mahiques L, Sanchez-Carazo JL, Oliver V, Fortea JM. Infliximab in the management of severe pemphigus vulgaris. Br J Dermatol. 2005 Jul;153(1):222-3. doi: 10.1111/j.1365-2133.2005.06672.x. No abstract available.
- Results publication: PubMed 25123295Hall RP 3rd, Fairley J, Woodley D, Werth VP, Hannah D, Streilein RD, McKillip J, Okawa J, Rose M, Keyes-Elstein LL, Pinckney A, Overington A, Wedgwood J, Ding L, Welch B. A multicentre randomized trial of the treatment of patients with pemphigus vulgaris with infliximab and prednisone compared with prednisone alone. Br J Dermatol. 2015 Mar;172(3):760-8. doi: 10.1111/bjd.13350. Epub 2015 Feb 5.
- National Institute of Allergy and Infectious Diseases (NIAID)Link provided by the sponsor.
- Division of Allergy, Immunology, and Transplantation (DAIT)Link provided by the sponsor.
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Rituximab With Corticosteroids Versus Corticosteroids Plus Azathioprine for Moderate-to-Severe Pemphigus Vulgaris
Pemphigus Vulgaris, Rituximab (RTx)
RecruitingEarly Phase 1
A Study to Investigate Safety and Efficacy of KB111 for the Treatment of Hailey-Hailey Disease
Hailey Hailey Disease, Skin Diseases
Active, not recruitingPhase 3
ONO-4059 Study in Patients With Steroid-resistant Pemphigus
Pemphigus, Steroid-resistant Pemphigus
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.
Russell P. Hall, MD study chair
Division of Dermatology, Duke University Medical Center
E. William St. Clair, MD study chair
Division of Rheumatology and Immunology, Duke University Medical Center
Garnett Kelsoe, DSci principal investigator
Department of Immunology, Duke University
4 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 Infliximab in people with Pemphigus, run by National Institute of Allergy and Infectious Diseases (NIAID) at 4 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 33 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 6 Dec 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 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. 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 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?
Yes. Results are posted in the registry (first posted 31 Jan 2013). 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?
Participant Response to Treatment at Week 18 (time frame: Baseline to Week 18). 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 NCT00283712, 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 6 Dec 2017.