Trial · NCT04206553
A Study to Evaluate the Efficacy and Safety of Dupilumab in Adult Patients With Bullous Pemphigoid
NCT04206553, Phase 2, Phase 3, Completed, Regeneron Pharmaceuticals; results first posted 2026-03-06
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. A Study to Evaluate the Efficacy and Safety of Dupilumab in Adult Patients With Bullous Pemphigoid. Trials, record NCT04206553. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04206553
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Study to Evaluate the Efficacy and Safety of Dupilumab in Adult Patients With Bullous Pemphigoid
- NCT number
- NCT04206553
- Status
- Completed
- Phases
- Phase 2, Phase 3
- Enrollment
- 106 participants, actual
- Lead sponsor
- Regeneron Pharmaceuticals
- Conditions
- Bullous Pemphigoid
- Interventions
- dupilumab, Matching Placebo, Oral corticosteroids (OCS)
- First posted
- 2019-12-20
- Last updated
- 2026-06-18
- Other registries' IDs
- eudract:2019-003520-20
- Results first posted
- 2026-03-06
Source ClinicalTrials.gov
Registered outcomes49
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Percent of Participants Achieving Sustained Remission at Week 36 | At Week 36 | Sustained remission at week 36, defined as: (1) Complete remission (absence of new lesions \& epithelialization of old lesions) \& off oral corticosteroids (OCS) no later than week 16 \& (2) Absence of disease relapse (appearance of ≥3 new lesions a month \[blisters, eczematous lesions, or urticarial plaques\] or at least 1 large eczematous lesion or urticarial plaque that does not heal within 1 week) after completion of OCS taper, no later than week 16, to week 36 \& (3) Absence of need for rescue therapy during 36 weeks (rescue therapy includes increase of OCS dose during the taper, re-initiation of OCS after completion of the OCS taper, or initiation of any BP-directed therapy). Participants with missing sustained remission data at week 36 due to discontinuation due to lack of efficacy, treatment-related AE, or death were considered non-responders \& those with missing data due to other reasons were handled using multiple imputation. |
| Secondary | Total Cumulative Dose of Oral Corticosteroids (OCS) From Baseline to Week 36 | Week 36 | All participants in both treatment groups were started on OCS at randomization (day 1) with taper beginning no later than week 6 with the objective to taper off by week 16. OCS could be used as rescue treatment for loss of disease control/ disease relapse during the study. Days after non-OCS systemic rescue treatment use were imputed by the worst observation carried forward (WOCF) method (i.e., the highest daily OCS dose prior to any non-OCS systemic rescue treatment use). Missing data after study discontinuation were imputed with the average daily OCS dose among week 36 completers in each treatment group. |
| Secondary | Percent Change in Weekly Average of Daily Peak Pruritus Numerical Rating Scale (NRS) Score From Baseline to Week 36 | Baseline, Week 36 | Peak pruritus NRS was used by participants to rate the intensity of pruritus during the past 24 hours, with 0 indicating no itch and 10 indicating worst itch possible. Participants were required to have a baseline peak pruritus NRS maximum intensity average of ≥4 during the 7 days prior to randomization for eligibility. Values after first rescue treatment were set to missing and were imputed with the worst value from the closest visit on or prior to rescue medication use and afterwards up to and including week 36. Missing values caused by discontinuation from the study due to lack of efficacy, treatment-related AE, or death were imputed by the WOCF method. Missing values due to other reasons were imputed by multiple imputation. |
| Secondary | Percent of Participants With Improvement (Reduction) of Weekly Average of Daily Peak Pruritus NRS ≥4 From Baseline to Week 36 | Baseline, Week 36 | Peak pruritus NRS was used by participants to rate the intensity of pruritus during the past 24 hours, with 0 indicating no itch and 10 indicating worst itch possible. Participants were required to have a baseline peak pruritus NRS maximum intensity average of ≥4 during the 7 days prior to randomization for eligibility. Participants were considered non-responders after rescue treatment use. Participants with missing values caused by discontinuation from the study before week 36 due to lack of efficacy, treatment-related AE, or death were considered non-responders. Missing values due to other reasons were imputed using multiple imputation of the continuous data of the weekly average of daily peak pruritus NRS. |
| Secondary | Percent Change in Bullous Pemphigoid Disease Area Index (BPDAI) Activity Score From Baseline to Week 36 | Baseline, Week 36 | BPDAI activity score is the arithmetic sum of 3 subcomponents: cutaneous blisters/erosions, cutaneous urticaria/erythema, and mucosal blisters/erosions. Scores can range from 0 to 360 for BPDAI total activity (maximum 240 for total skin activity and 120 for mucosal activity), with higher scores indicating greater disease activity. Participants were required to have a BPDAI activity score of ≥24 at the screening and baseline visits for eligibility. Values after first rescue treatment were set to missing and were imputed with the worst value from the closest visit on or prior to rescue medication use and afterwards up to and including week 36. Missing values caused by discontinuation from the study due to lack of efficacy, treatment-related AE, or death were imputed by the WOCF method. Missing values due to other reasons were imputed by multiple imputation. |
| Secondary | Time to First Use of Rescue Medication Up to Week 36 | Up to Week 36 | Participants were considered to have used rescue treatment at the time of discontinuation from the study due to lack of efficacy, treatment-related AE, or death. Participants were censored at the time of discontinuation from the study due to other reasons. Restricted mean event time and corresponding standard error (SE) are based on the Kaplan-Meier method. The restricted mean event time was calculated up to study day 253 (i.e., week 36). |
| Secondary | Duration of Complete Remission While Not Requiring OCS Up to Week 36 | Up to Week 36 | Complete remission was defined as absence of new lesions and epithelialization of old lesions. Participants were considered as not achieving complete remission after rescue treatment use. Participants with missing assessment of complete remission caused by discontinuation from the study before week 36 due to lack of efficacy, treatment-related AE, or death were considered as not achieving complete remission. Participants with missing assessment of complete remission due to other reasons were imputed using multiple imputation. |
| Secondary | Percent of Participants Who Did Not Achieve Control of Disease Activity Before Week 36 | Up to Week 36 | Control of disease activity was defined as absence of new lesions and existing lesions beginning to heal. Participants who achieved initial control of disease activity after rescue treatment use were considered to not have achieved control of disease activity before week 36. Participants whose response status could not be determined due to missing data were considered to not have achieved control of disease activity before week 36. |
| Secondary | Percent of Participants Who Relapsed After Achieving Control of Disease Activity Up to Week 36 | Up to Week 36 | Relapse was defined as the appearance of 3 or more new lesions a month (blisters, eczematous lesions, or urticarial plaques) or at least 1 large (\>10 centimeters \[cm\] diameter) eczematous lesion or urticarial plaque that does not heal within 1 week. Control of disease activity was defined as absence of new lesions and existing lesions beginning to heal. Participants who used rescue treatment were considered to have relapsed after achieving control of disease activity. Participants whose response status could not be determined due to missing data were considered to have relapsed after achieving control of disease activity. |
| Secondary | Percent of Participants Who Did Not Achieve Complete Remission Before Week 36 | Up to Week 36 | Complete remission was defined as absence of new lesions and epithelialization of old lesions. Participants who achieved initial complete remission after rescue treatment use were considered to not have achieved complete remission before week 36. Participants whose response status could not be determined due to missing data were considered to not have achieved complete remission before week 36. |
1 to 10 of 49Source ClinicalTrials.gov
See all 49 registered outcomesOutcomes50
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Percent of Participants Achieving Sustained Remission at Week 36 | At Week 36 | Estimated Percentage of Participants | NUMBER | |
| SECONDARY | Total Cumulative Dose of Oral Corticosteroids (OCS) From Baseline to Week 36 | Week 36 | milligrams (mg) | LEAST_SQUARES_MEAN | |
| SECONDARY | Percent Change in Weekly Average of Daily Peak Pruritus Numerical Rating Scale (NRS) Score From Baseline to Week 36 | Baseline, Week 36 | All participants with available baseline data were analyzed for this endpoint. | Percentage of Change | LEAST_SQUARES_MEAN |
| SECONDARY | Percent of Participants With Improvement (Reduction) of Weekly Average of Daily Peak Pruritus NRS ≥4 From Baseline to Week 36 | Baseline, Week 36 | All participants with available baseline data were analyzed for this endpoint. | Estimated Percentage of Participants | NUMBER |
| SECONDARY | Percent Change in Bullous Pemphigoid Disease Area Index (BPDAI) Activity Score From Baseline to Week 36 | Baseline, Week 36 | Percentage of Change | LEAST_SQUARES_MEAN | |
| SECONDARY | Time to First Use of Rescue Medication Up to Week 36 | Up to Week 36 | Days | MEAN | |
| SECONDARY | Duration of Complete Remission While Not Requiring OCS Up to Week 36 | Up to Week 36 | Days | LEAST_SQUARES_MEAN | |
| SECONDARY | Percent of Participants Who Did Not Achieve Control of Disease Activity Before Week 36 | Up to Week 36 | Percentage of Participants | NUMBER | |
| SECONDARY | Percent of Participants Who Relapsed After Achieving Control of Disease Activity Up to Week 36 | Up to Week 36 | Participants who achieved control of disease activity were analyzed for this endpoint. | Percentage of Participants | NUMBER |
| SECONDARY | Percent of Participants Who Did Not Achieve Complete Remission Before Week 36 | Up to Week 36 | Percentage of Participants | NUMBER |
1 to 10 of 50Source ClinicalTrials.gov
See all 50 outcomesResults by group108
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Percent of Participants Achieving Sustained Remission at Week 36 | Placebo | 4.0 | Estimated Percentage of Participants | ||
| Percent of Participants Achieving Sustained Remission at Week 36 | Dupilumab Q2W | 18.2 | Estimated Percentage of Participants | ||
| Total Cumulative Dose of Oral Corticosteroids (OCS) From Baseline to Week 36 | Placebo | 5121.15 | Standard Error 473.034 | milligrams (mg) | |
| Total Cumulative Dose of Oral Corticosteroids (OCS) From Baseline to Week 36 | Dupilumab Q2W | 4184.93 | Standard Error 454.733 | milligrams (mg) | |
| Percent Change in Weekly Average of Daily Peak Pruritus Numerical Rating Scale (NRS) Score From Baseline to Week 36 | Placebo | -26.7 | Standard Error 6.59 | Percentage of Change | |
| Percent Change in Weekly Average of Daily Peak Pruritus Numerical Rating Scale (NRS) Score From Baseline to Week 36 | Dupilumab Q2W | -51.8 | Standard Error 6.66 | Percentage of Change | |
| Percent of Participants With Improvement (Reduction) of Weekly Average of Daily Peak Pruritus NRS ≥4 From Baseline to Week 36 | Placebo | 10.5 | Estimated Percentage of Participants | ||
| Percent of Participants With Improvement (Reduction) of Weekly Average of Daily Peak Pruritus NRS ≥4 From Baseline to Week 36 | Dupilumab Q2W | 39.8 | Estimated Percentage of Participants | ||
| Percent Change in Bullous Pemphigoid Disease Area Index (BPDAI) Activity Score From Baseline to Week 36 | Placebo | -50.5 | Standard Error 7.04 | Percentage of Change | |
| Percent Change in Bullous Pemphigoid Disease Area Index (BPDAI) Activity Score From Baseline to Week 36 | Dupilumab Q2W | -76.7 | Standard Error 6.89 | Percentage of Change |
1 to 10 of 108Source ClinicalTrials.gov
See all 108 results by groupParticipant flow6
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Placebo | 53 | |
| Overall Study | STARTED | Dupilumab Q2W | 53 | |
| Overall Study | COMPLETED | Placebo | 32 | |
| Overall Study | COMPLETED | Dupilumab Q2W | 45 | |
| Overall Study | NOT COMPLETED | Placebo | 21 | |
| Overall Study | NOT COMPLETED | Dupilumab Q2W | 8 |
Source ClinicalTrials.gov
Adverse events204
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Nasopharyngitis | Infections and infestations | Placebo | 8 | 53 | 8 |
| other | Nasopharyngitis | Infections and infestations | Dupilumab Q2W | 8 | 53 | 10 |
| other | Urinary tract infection | Infections and infestations | Placebo | 2 | 53 | 2 |
| other | Urinary tract infection | Infections and infestations | Dupilumab Q2W | 5 | 53 | 5 |
| other | COVID-19 | Infections and infestations | Placebo | 4 | 53 | 4 |
| other | COVID-19 | Infections and infestations | Dupilumab Q2W | 4 | 53 | 4 |
| other | Conjunctivitis | Infections and infestations | Placebo | 0 | 53 | 0 |
| other | Conjunctivitis | Infections and infestations | Dupilumab Q2W | 5 | 53 | 5 |
| other | Upper respiratory tract infection | Infections and infestations | Placebo | 2 | 53 | 2 |
| other | Upper respiratory tract infection | Infections and infestations | Dupilumab Q2W | 3 | 53 | 4 |
1 to 10 of 204Source ClinicalTrials.gov
See all 204 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Placebo | 6 | 53 |
| Total, all-cause mortality | Dupilumab Q2W | 2 | 53 |
| Total, other adverse events | Placebo | 49 | 53 |
| Total, other adverse events | Dupilumab Q2W | 43 | 53 |
| Total, serious adverse events | Placebo | 21 | 53 |
| Total, serious adverse events | Dupilumab Q2W | 20 | 53 |
Source ClinicalTrials.gov
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 38443648 | DERIVED |
Source PubMed
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons8
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| Dupilumab Q2W vs Placebo: Number of Participants With At Least One Treatment-emergent Adverse Event (TEAE) Through Week 52 | Trial comparisons |
| Dupilumab Q2W vs Placebo: Number of Participants With At Least One TEAE Through Week 64 | Trial comparisons |
| Dupilumab Q2W vs Placebo: Number of Participants With At Least One Serious TEAE Through Week 52 | Trial comparisons |
| Dupilumab Q2W vs Placebo: Number of Participants With At Least One Serious TEAE Through Week 64 | Trial comparisons |
| Dupilumab Q2W vs Placebo: Number of Participants With At Least One TE AESI Through Week 64 | Trial comparisons |
| Dupilumab Q2W vs Placebo: deaths from any cause | Trial comparisons |
| Dupilumab Q2W vs Placebo: Number of Participants With At Least One TE Adverse Event of Special Interest (AESI) Through Week 52 | Trial comparisons |
| Dupilumab Q2W vs Placebo: serious adverse events | Trial comparisons |
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT04206553
GET https://conductscience.org/api/trials/v1/NCT04206553/registered?page=2