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| Name | Class |
|---|---|
| Autoimmunity Centers of Excellence | OTHER |
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.
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| 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. |
|
| 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. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Infliximab | Drug | Chimeric IgG monoclonal antibody that binds to TNF-alpha, generally used to treat Crohn's disease, given in a dosage of 5 mg/kg |
|
| Measure | Description | Time Frame |
|---|---|---|
| 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. | 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. | Baseline to Week 18 |
| Measure | Description | Time Frame |
|---|---|---|
| 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. | Baseline to Week 18 |
| Participant Modified Response Status at Week 18 |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Russell P. Hall, MD | Division of Dermatology, Duke University Medical Center | Study Chair |
| E. William St. Clair, MD | Division of Rheumatology and Immunology, Duke University Medical Center | Study Chair |
| Garnett Kelsoe, DSci | Department of Immunology, Duke University | Principal Investigator |
| Victoria Werth, MD | Department of Dermatology, University of Pennsylvania School of Medicine | Principal Investigator |
| Janet Fairley, MD | Department of Dermatology, University of Iowa | Principal Investigator |
| David Woodley, MD | Department of Dermatology, Norris Cancer Center, University of Southern California | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Norris Cancer Center, University of Southern California | Los Angeles | California | 90033 | United States | ||
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 15243495 | Background | 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. | |
| 14973643 | Background | Drosou 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. |
| Label | URL |
|---|---|
| National Institute of Allergy and Infectious Diseases (NIAID) | View source |
| ID | Type | URL | Comment |
|---|---|---|---|
| SDY655 | Individual Participant Data Set | View IPD |
Each participant signed an informed consent before undergoing any screening procedures to assess eligibility. Refer to the Eligibility Section for further details.
Four study centers in the United States enrolled 20 subjects with pemphigus vulgaris (PV) who met entry criteria between August 2005 and December 2010.
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| ID | Title | Description |
|---|---|---|
| FG000 | Experimental: Infliximab (Remicade, Revellex) | Participants were 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 blinded (masked) fashion. Refer to section titled, "Detailed Description" for additional treatment information. |
| FG001 | Placebo Comparator: Placebo | Participants were 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 blinded (masked) fashion. Refer to section titled, "Detailed Description" for additional treatment information. |
| Title | Milestones | Reasons Not Completed | |||||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
|
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| ID | Title | Description |
|---|---|---|
| BG000 | Experimental: Infliximab (Remicade, Revellex) | Participants were 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 blinded (masked) fashion. Refer to section titled, "Detailed Description" for additional treatment information. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | 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. | Intent-to-Treat | Posted | Number | Participants | Baseline to Week 18 |
|
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All Adverse Events are included due to low sample size (20 participants).
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Infliximab | Subjects randomized to the Infliximab group |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Lymphopenia | Blood and lymphatic system disorders | MedDRA 11.0 | Systematic Assessment |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Anaemia | Blood and lymphatic system disorders | MedDRA 11.0 | Systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Associate Director, Clinical Research Program | DAIT/NIAID | 301-594-7669 | DAITClinicalTrialsGov@niaid.nih.gov |
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| ID | Term |
|---|---|
| D010392 | Pemphigus |
| D012871 | Skin Diseases |
| D001327 | Autoimmune Diseases |
| ID | Term |
|---|---|
| D012872 | Skin Diseases, Vesiculobullous |
| D017437 | Skin and Connective Tissue Diseases |
| D007154 | Immune System Diseases |
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| ID | Term |
|---|---|
| D000069285 | Infliximab |
| ID | Term |
|---|---|
| D000911 | Antibodies, Monoclonal |
| D000906 | Antibodies |
| D007136 | Immunoglobulins |
| D007162 | Immunoproteins |
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| Placebo Comparator | Other | Placebo administered in place of infliximab for control group |
|
|
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. |
| 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. | 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. | 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. | 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. | 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. | 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. | 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. | 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. | 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. | 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. | 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 | Baseline to Week 26 |
| University of Iowa Hospitals and Clinics |
| Iowa City |
| Iowa |
| 52242 |
| United States |
| Duke University Medical Center | Durham | North Carolina | 27710 | United States |
| University of Pennsylvania | Philadelphia | Pennsylvania | 19104 | United States |
| 16086769 | Background | Jacobi 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. |
| 16029365 | Background | Pardo 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. |
| 25123295 | Result | Hall 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. |
| Division of Allergy, Immunology, and Transplantation (DAIT) | View source |
Participant level data and additional relevant materials are available to the public in the Immunology Database and Analysis Portal (ImmPort), a long-term archive of clinical and mechanistic data from DAIT-funded grants and contracts. Data analysis tools are also available to researchers. |
| SDY655 | Study Protocol | View IPD | ImmPort study identifier is SDY655 |
| SDY655 | Study summary, -design, -adverse event(s), -summary of participant assessments, -interventions, -medications, -demographics, -lab tests, -mechanistic Assays, -study files et al. | View IPD | ImmPort study identifier is SDY655 |
| BG001 | Placebo Comparator: Placebo | Participants were 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 blinded (masked) fashion. Refer to section titled, "Detailed Description" for additional treatment information. |
| BG002 | Total | Total of all reporting groups |
| Participants |
|
| Age, Continuous | Mean | Standard Deviation | years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Region of Enrollment | Number | participants |
|
| PDAI Index Total Score | The Pemphigus Disease Area Index (PDAI) was developed by the International Pemphigus Committee and measures both activity of and damage due to pemphigus on the skin, scalp, and mucous membranes. Total scores can range from 0 to a possible 263 maximum score, with 250 points representing disease activity (120 points for skin activity, 10 points for scalp activity, and 120 points for mucosal activity) and 13 points representing disease damage. Higher scores reflect worse disease. | Mean | Standard Deviation | Total Activity Score |
|
| Pemphigus Vulgaris Disease Activity Score: Mucosal | The Pemphigus Vulgaris Disease Activity(PVDA)score was used to grade a participant's baseline 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 lesions and blisters present as well as total body surface area involved. Three types of disease activity were assessed: mucosal, cutaneous, and other organ system. A higher score indicates more severe disease activity. For each of the 3 types of disease activity, the number of participants who met the disease activity condition is shown. | Number | Participants |
|
| Pemphigus Vulgaris Disease Activity Score: Cutaneous | The Pemphigus Vulgaris Disease Activity(PVDA)score was used to grade a participant's baseline 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 lesions and blisters present as well as total body surface area involved. Three types of disease activity were assessed: mucosal, cutaneous, and other organ system. A higher score indicates more severe disease activity. For each of the 3 types of disease activity, the number of participants who met the disease activity condition is shown. | Number | Participants |
|
| Pemphigus Vulgaris Disease Activity Score: Other Organ System | The Pemphigus Vulgaris Disease Activity(PVDA)score was used to grade a participant's baseline 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 lesions and blisters present as well as total body surface area involved. Three types of disease activity were assessed: mucosal, cutaneous, and other organ system. A higher score indicates more severe disease activity. For each of the 3 types of disease activity, the number of participants who met the disease activity condition is shown. | Number | Participants |
|
Participants were 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 blinded (masked) fashion. Refer to section titled, "Detailed Description" for additional treatment information. |
|
|
|
| Primary | 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. | Safety Population | Posted | Number | Participants | Baseline to Week 18 |
|
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|
|
| Secondary | 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. | Per-protocol | Posted | Number | Participants | Baseline to Week 18 |
|
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|
|
| Secondary | 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. | Intent-to-Treat | Posted | Number | Participants | Baseline to Week 18 |
|
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|
|
| Secondary | 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. | Subset of Intent-to-Treat Who Experienced Cessation | Posted | Mean | Standard Deviation | Days | Baseline to Week 26 |
|
|
|
| Secondary | 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. | Subset of Intent-to-Treat Who Experienced Lesion Healing | Posted | Mean | Standard Deviation | Days | Baseline to Week 26 |
|
|
|
| Secondary | 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. | Subset of Intent-to-Treat Who Experienced Lesion Cessation | Posted | Mean | Standard Deviation | mg | Baseline to Week 26 |
|
|
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| Secondary | 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. | Subset of Intent-to-Treat Who Experienced Erosion Healing | Posted | Mean | Standard Deviation | mg | Baseline to Week 26 |
|
|
|
| Secondary | 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. | Intent-to-Treat with available data | Posted | Mean | Standard Deviation | units on a scale | Baseline to Week 18 |
|
|
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| Secondary | 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. | Intent-to-Treat with available data | Posted | Mean | Standard Deviation | units on a scale | Baseline to Week 18 |
|
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| Secondary | 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. | Participants in the Intent-to-Treat Population Who Were Responders at Week 18 | Posted | Number | Participants | Baseline to Week 26 |
|
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| Secondary | 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. | Safety Population | Posted | Number | Participants | Baseline to Week 26 |
|
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| Secondary | 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. | Safety Population | Posted | Number | Participants | Baseline to Week 26 |
|
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| Secondary | 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. | Safety Population | Posted | Number | Participants | Baseline to Week 26 |
|
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|
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| Secondary | 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 | Safety Population | Posted | Number | Participants | Baseline to Week 26 |
|
|
|
| 1 |
| 10 |
| 9 |
| 10 |
| EG001 | Placebo | Subjects randomized to the Placebo group | 2 | 10 | 10 | 10 |
| Syncope | Nervous system disorders | MedDRA 11.0 | Systematic Assessment |
|
| Laryngeal obstruction | Respiratory, thoracic and mediastinal disorders | MedDRA 11.0 | Systematic Assessment |
|
| Pulmonary embolism | Respiratory, thoracic and mediastinal disorders | MedDRA 11.0 | Systematic Assessment |
|
| Pemphigus | Skin and subcutaneous tissue disorders | MedDRA 11.0 | Systematic Assessment |
|
| Skin ulcer | Skin and subcutaneous tissue disorders | MedDRA 11.0 | Systematic Assessment |
|
| Eosinophilia | Blood and lymphatic system disorders | MedDRA 11.0 | Systematic Assessment |
|
| Lymphopenia | Blood and lymphatic system disorders | MedDRA 11.0 | Systematic Assessment |
|
| Bradycardia | Cardiac disorders | MedDRA 11.0 | Systematic Assessment |
|
| Tachycardia | Cardiac disorders | MedDRA 11.0 | Systematic Assessment |
|
| Vertigo | Ear and labyrinth disorders | MedDRA 11.0 | Systematic Assessment |
|
| Blepharitis | Eye disorders | MedDRA 11.0 | Systematic Assessment |
|
| Conjunctival haemorrhage | Eye disorders | MedDRA 11.0 | Systematic Assessment |
|
| Abdominal distension | Gastrointestinal disorders | MedDRA 11.0 | Systematic Assessment |
|
| Constipation | Gastrointestinal disorders | MedDRA 11.0 | Systematic Assessment |
|
| Diarrhoea | Gastrointestinal disorders | MedDRA 11.0 | Systematic Assessment |
|
| Dyspepsia | Gastrointestinal disorders | MedDRA 11.0 | Systematic Assessment |
|
| Haemorrhoids | Gastrointestinal disorders | MedDRA 11.0 | Systematic Assessment |
|
| Nausea | Gastrointestinal disorders | MedDRA 11.0 | Systematic Assessment |
|
| Vomiting | Gastrointestinal disorders | MedDRA 11.0 | Systematic Assessment |
|
| Fatigue | General disorders | MedDRA 11.0 | Systematic Assessment |
|
| Feeling hot | General disorders | MedDRA 11.0 | Systematic Assessment |
|
| Oedema peripheral | General disorders | MedDRA 11.0 | Systematic Assessment |
|
| Pitting oedema | General disorders | MedDRA 11.0 | Systematic Assessment |
|
| Pyrexia | General disorders | MedDRA 11.0 | Systematic Assessment |
|
| Cholelithiasis | Hepatobiliary disorders | MedDRA 11.0 | Systematic Assessment |
|
| Bronchitis | Infections and infestations | MedDRA 11.0 | Systematic Assessment |
|
| Candidiasis | Infections and infestations | MedDRA 11.0 | Systematic Assessment |
|
| Folliculitis | Infections and infestations | MedDRA 11.0 | Systematic Assessment |
|
| Fungal skin infection | Infections and infestations | MedDRA 11.0 | Systematic Assessment |
|
| Gastroenteritis viral | Infections and infestations | MedDRA 11.0 | Systematic Assessment |
|
| Influenza | Infections and infestations | MedDRA 11.0 | Systematic Assessment |
|
| Nasopharyngitis | Infections and infestations | MedDRA 11.0 | Systematic Assessment |
|
| Oral candidiasis | Infections and infestations | MedDRA 11.0 | Systematic Assessment |
|
| Oral herpes | Infections and infestations | MedDRA 11.0 | Systematic Assessment |
|
| Pharyngitis | Infections and infestations | MedDRA 11.0 | Systematic Assessment |
|
| Pneumonia | Infections and infestations | MedDRA 11.0 | Systematic Assessment |
|
| Pseudomonas infection | Infections and infestations | MedDRA 11.0 | Systematic Assessment |
|
| Rhinitis | Infections and infestations | MedDRA 11.0 | Systematic Assessment |
|
| Sinusitis | Infections and infestations | MedDRA 11.0 | Systematic Assessment |
|
| Staphylococcal skin infection | Infections and infestations | MedDRA 11.0 | Systematic Assessment |
|
| Tinea pedis | Infections and infestations | MedDRA 11.0 | Systematic Assessment |
|
| Upper respiratory tract infection | Infections and infestations | MedDRA 11.0 | Systematic Assessment |
|
| Vulval abscess | Infections and infestations | MedDRA 11.0 | Systematic Assessment |
|
| Vulvovaginal mycotic infection | Infections and infestations | MedDRA 11.0 | Systematic Assessment |
|
| Arthropod bite | Injury, poisoning and procedural complications | MedDRA 11.0 | Systematic Assessment |
|
| Back injury | Injury, poisoning and procedural complications | MedDRA 11.0 | Systematic Assessment |
|
| Contusion | Injury, poisoning and procedural complications | MedDRA 11.0 | Systematic Assessment |
|
| Excoriation | Injury, poisoning and procedural complications | MedDRA 11.0 | Systematic Assessment |
|
| Limb injury | Injury, poisoning and procedural complications | MedDRA 11.0 | Systematic Assessment |
|
| Sunburn | Injury, poisoning and procedural complications | MedDRA 11.0 | Systematic Assessment |
|
| Alanine aminotransferase increased | Investigations | MedDRA 11.0 | Systematic Assessment |
|
| Aspartate aminotransferase increased | Investigations | MedDRA 11.0 | Systematic Assessment |
|
| Haemoglobin decreased | Investigations | MedDRA 11.0 | Systematic Assessment |
|
| Monocyte count increased | Investigations | MedDRA 11.0 | Systematic Assessment |
|
| Neutrophil count increased | Investigations | MedDRA 11.0 | Systematic Assessment |
|
| Platelet count decreased | Investigations | MedDRA 11.0 | Systematic Assessment |
|
| Transaminases increased | Investigations | MedDRA 11.0 | Systematic Assessment |
|
| Gout | Metabolism and nutrition disorders | MedDRA 11.0 | Systematic Assessment |
|
| Hyperglycaemia | Metabolism and nutrition disorders | MedDRA 11.0 | Systematic Assessment |
|
| Hypoalbuminaemia | Metabolism and nutrition disorders | MedDRA 11.0 | Systematic Assessment |
|
| Hypoglycaemia | Metabolism and nutrition disorders | MedDRA 11.0 | Systematic Assessment |
|
| Hypokalaemia | Metabolism and nutrition disorders | MedDRA 11.0 | Systematic Assessment |
|
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA 11.0 | Systematic Assessment |
|
| Arthritis | Musculoskeletal and connective tissue disorders | MedDRA 11.0 | Systematic Assessment |
|
| Metatarsalgia | Musculoskeletal and connective tissue disorders | MedDRA 11.0 | Systematic Assessment |
|
| Neck pain | Musculoskeletal and connective tissue disorders | MedDRA 11.0 | Systematic Assessment |
|
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA 11.0 | Systematic Assessment |
|
| Tendonitis | Musculoskeletal and connective tissue disorders | MedDRA 11.0 | Systematic Assessment |
|
| Convulsion | Nervous system disorders | MedDRA 11.0 | Systematic Assessment |
|
| Dizziness | Nervous system disorders | MedDRA 11.0 | Systematic Assessment |
|
| Headache | Nervous system disorders | MedDRA 11.0 | Systematic Assessment |
|
| Migraine | Nervous system disorders | MedDRA 11.0 | Systematic Assessment |
|
| Anxiety | Psychiatric disorders | MedDRA 11.0 | Systematic Assessment |
|
| Hallucination, olfactory | Psychiatric disorders | MedDRA 11.0 | Systematic Assessment |
|
| Insomnia | Psychiatric disorders | MedDRA 11.0 | Systematic Assessment |
|
| Vulvovaginal dryness | Reproductive system and breast disorders | MedDRA 11.0 | Systematic Assessment |
|
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 11.0 | Systematic Assessment |
|
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA 11.0 | Systematic Assessment |
|
| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA 11.0 | Systematic Assessment |
|
| Rhinitis allergic | Respiratory, thoracic and mediastinal disorders | MedDRA 11.0 | Systematic Assessment |
|
| Throat irritation | Respiratory, thoracic and mediastinal disorders | MedDRA 11.0 | Systematic Assessment |
|
| Alopecia | Skin and subcutaneous tissue disorders | MedDRA 11.0 | Systematic Assessment |
|
| Decubitus ulcer | Skin and subcutaneous tissue disorders | MedDRA 11.0 | Systematic Assessment |
|
| Dermatitis bullous | Skin and subcutaneous tissue disorders | MedDRA 11.0 | Systematic Assessment |
|
| Eczema | Skin and subcutaneous tissue disorders | MedDRA 11.0 | Systematic Assessment |
|
| Exfoliative rash | Skin and subcutaneous tissue disorders | MedDRA 11.0 | Systematic Assessment |
|
| Heat rash | Skin and subcutaneous tissue disorders | MedDRA 11.0 | Systematic Assessment |
|
| Night sweats | Skin and subcutaneous tissue disorders | MedDRA 11.0 | Systematic Assessment |
|
| Pemphigus | Skin and subcutaneous tissue disorders | MedDRA 11.0 | Systematic Assessment |
|
| Petechiae | Skin and subcutaneous tissue disorders | MedDRA 11.0 | Systematic Assessment |
|
| Photosensitivity reaction | Skin and subcutaneous tissue disorders | MedDRA 11.0 | Systematic Assessment |
|
| Pruritus | Skin and subcutaneous tissue disorders | MedDRA 11.0 | Systematic Assessment |
|
| Rash | Skin and subcutaneous tissue disorders | MedDRA 11.0 | Systematic Assessment |
|
| Skin erosion | Skin and subcutaneous tissue disorders | MedDRA 11.0 | Systematic Assessment |
|
| Flushing | Vascular disorders | MedDRA 11.0 | Systematic Assessment |
|
| Hypertension | Vascular disorders | MedDRA 11.0 | Systematic Assessment |
|
| Hypotension | Vascular disorders | MedDRA 11.0 | Systematic Assessment |
|
Not provided
Not provided
| D001798 |
| Blood Proteins |
| D011506 | Proteins |
| D000602 | Amino Acids, Peptides, and Proteins |
| D012712 | Serum Globulins |
| D005916 | Globulins |
The primary endpoint analysis was replicated using the per-protocol population.
| Fisher Exact |
| 1.00 |
All secondary analyses are considered supplemental supportive analyses |
| Odds Ratio (OR) |
| 0.83 |
| 2-Sided |
| 90 |
| 0.02 |
| 38.35 |
| Superiority or Other |
| Odds Ratio (OR) |
| 0.43 |
| 2-Sided |
| 90 |
| 0.06 |
| 2.79 |
| Superiority or Other |
| Bodily Pain Scale |
|
| General Health Scale |
|
| Physical Component Scale |
|
| Vitality Scale |
|
| Social Functioning Scale |
|
| Role Emotional Scale |
|
| Mental Health Scale |
|
| Mental Component Scale |
|