Phase 3
N=106
A Study to Evaluate the Efficacy and Safety of Dupilumab in Adult Patients With Bullous Pemphigoid
Bullous Pemphigoid
Bottom Line
View on ClinicalTrials.gov: NCT04206553 ↗Enrolled (actual)
106
Serious AEs
38.7%
Results posted
Mar 2026
Primary outcome: Primary: Percent of Participants Achieving Sustained Remission at Week 36 — 4.0; 18.2 Estimated Percentage of Participants — p=0.0250
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- dupilumab (Drug); Matching Placebo (Drug); Oral corticosteroids (OCS) (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Regeneron Pharmaceuticals
- Primary completion
- Jul 2024
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Percent of Participants Achieving Sustained Remission at Week 36 |
4.0; 18.2 | 0.0250 sig |
| SECONDARY Total Cumulative Dose of Oral Corticosteroids (OCS) From Baseline to Week 36 |
5121.15; 4184.93 | 0.1092 |
| SECONDARY Percent Change in Weekly Average of Daily Peak Pruritus Numerical Rating Scale (NRS) Score From Baseline to Week 36 |
-26.7; -51.8 | 0.0025 sig |
| SECONDARY Percent of Participants With Improvement (Reduction) of Weekly Average of Daily Peak Pruritus NRS ≥4 From Baseline to Week 36 |
10.5; 39.8 | 0.0006 sig |
| SECONDARY Percent Change in Bullous Pemphigoid Disease Area Index (BPDAI) Activity Score From Baseline to Week 36 |
-50.5; -76.7 | 0.0021 sig |
| SECONDARY Time to First Use of Rescue Medication Up to Week 36 |
126.8; 168.9 | 0.0023 sig |
| SECONDARY Duration of Complete Remission While Not Requiring OCS Up to Week 36 |
12.9; 38.7 | 0.0112 sig |
| SECONDARY Percent of Participants Who Did Not Achieve Control of Disease Activity Before Week 36 |
7.5; 1.9 | 0.2414 |
| SECONDARY Percent of Participants Who Relapsed After Achieving Control of Disease Activity Up to Week 36 |
91.8; 73.1 | 0.0165 sig |
| SECONDARY Percent of Participants Who Did Not Achieve Complete Remission Before Week 36 |
50.9; 39.6 | 0.2758 |
| SECONDARY Percent of Participants Achieving a Reduction in BPDAI Activity Score of ≥50% From Baseline to Week 36 |
13.2; 41.0 | 0.0012 sig |
| SECONDARY Percent of Participants Achieving a Reduction in BPDAI Activity Score of ≥75% From Baseline to Week 36 |
12.5; 38.8 | 0.0024 sig |
| SECONDARY Percent of Participants Achieving a Reduction in BPDAI Activity Score of ≥90% From Baseline to Week 36 |
9.8; 38.6 | 0.0006 sig |
| SECONDARY Change in Autoimmune Bullous Disease Quality of Life (ABQOL) Score From Baseline to Week 36 |
-3.41; -7.09 | 0.0476 sig |
| SECONDARY Change in Percent Body Surface Area (BSA) of BP Involvement From Baseline to Week 36 |
-17.51; -24.07 | 0.0345 sig |
| SECONDARY Change in BP180 Immunoglobulin G (IgG) Autoantibody Titer From Baseline to Week 36 |
-137.57; -136.33 | 0.9857 |
| SECONDARY Change in BP230 IgG Autoantibody Titer From Baseline to Week 36 |
-33.91; -18.49 | 0.6680 |
| SECONDARY Percent of Participants Achieving Sustained Remission at Week 52 |
4.0; 14.2 | 0.0736 |
| SECONDARY Total Cumulative Dose of OCS From Baseline to Week 52 |
7159.09; 5133.03 | 0.0463 sig |
| SECONDARY Duration of Complete Remission While Not Requiring OCS Up to Week 52 |
24.7; 67.2 | 0.0198 sig |
| SECONDARY Percent of Participants Who Did Not Achieve Control of Disease Activity Before Week 52 |
7.5; 1.9 | 0.2414 |
| SECONDARY Percent of Participants Who Relapsed After Achieving Control of Disease Activity Up to Week 52 |
91.8; 76.9 | 0.0461 sig |
| SECONDARY Percent of Participants Who Did Not Achieve Complete Remission Before Week 52 |
50.9; 34.0 | 0.0843 |
| SECONDARY Percent Change in Weekly Average of Daily Peak Pruritus NRS From Baseline to Week 52 |
-23.4; -53.0 | 0.0002 sig |
| SECONDARY Percent of Participants With Improvement (Reduction) of Weekly Average of Daily Peak Pruritus NRS ≥4 From Baseline to Week 52 |
8.3; 36.4 | 0.0006 sig |
| SECONDARY Percent Change in BPDAI Activity Score From Baseline to Week 52 |
-49.1; -76.1 | 0.0014 sig |
| SECONDARY Percent of Participants Achieving a Reduction in BPDAI Activity Score of ≥50% From Baseline to Week 52 |
11.2; 37.2 | 0.0019 sig |
| SECONDARY Percent of Participants Achieving a Reduction in BPDAI Activity Score of ≥75% From Baseline to Week 52 |
10.6; 36.6 | 0.0016 sig |
| SECONDARY Percent of Participants Achieving a Reduction in BPDAI Activity Score of ≥90% From Baseline to Week 52 |
9.9; 34.4 | 0.0024 sig |
| SECONDARY Change in ABQOL Score From Baseline to Week 52 |
-3.27; -7.29 | 0.0311 sig |
| SECONDARY Change in Percent BSA of BP Involvement From Baseline to Week 52 |
-16.59; -23.89 | 0.0206 sig |
| SECONDARY Change in BP180 IgG Autoantibody Titer From Baseline to Week 52 |
-133.02; -137.60 | 0.9471 |
| SECONDARY Change in BP230 IgG Autoantibody Titer From Baseline to Week 52 |
-33.33; -18.83 | 0.6876 |
| SECONDARY Percent of Participants in Complete Remission and Off OCS No Later Than Week 16 |
26.6; 34.6 | 0.5283 |
| SECONDARY Percent Change in BPDAI Activity Score From Baseline to Week 16 |
-56.3; -78.5 | 0.0088 sig |
| SECONDARY Percent of Participants Achieving a Reduction in BPDAI Activity Score of ≥50% From Baseline to Week 16 |
45.2; 67.3 | 0.0250 sig |
| SECONDARY Percent of Participants Achieving a Reduction in BPDAI Activity Score of ≥75% From Baseline to Week 16 |
37.1; 59.4 | 0.0250 sig |
| SECONDARY Percent of Participants Achieving a Reduction in BPDAI Activity Score of ≥90% From Baseline to Week 16 |
32.7; 45.8 | 0.2089 |
| SECONDARY Percent Change in Weekly Average of Daily Peak Pruritus NRS From Baseline to Week 16 |
-36.4; -50.4 | 0.0767 |
| SECONDARY Percent of Participants With Improvement (Reduction) of Weekly Average of Daily Peak Pruritus NRS ≥4 From Baseline to Week 16 |
33.5; 50.9 | 0.0761 |
| SECONDARY Number of Participants With At Least One Treatment-emergent Adverse Event (TEAE) Through Week 52 |
51; 51 | — |
| SECONDARY Number of Participants With At Least One Serious TEAE Through Week 52 |
16; 12 | — |
| SECONDARY Number of Participants With At Least One TE Adverse Event of Special Interest (AESI) Through Week 52 |
0; 4 | — |
| SECONDARY Number of Participants With At Least One TEAE Through Week 64 |
23; 25 | — |
| SECONDARY Number of Participants With At Least One Serious TEAE Through Week 64 |
8; 8 | — |
| SECONDARY Number of Participants With At Least One TE AESI Through Week 64 |
0; 0 | — |
| SECONDARY Concentrations of Functional Dupilumab in Serum |
0; 50.2; 66.7; 71.0; 78.6; 8.73 | — |
| SECONDARY Number of Participants With TE Anti-drug Antibody (ADA) Response Per Maximum Titer Category |
0; 1; 0; 1; 0; 0 | — |
Summary
The main purpose of this study is to investigate whether dupilumab is effective and safe for the treatment of bullous pemphigoid. Dupilumab is a type of drug called a "monoclonal antibody". An antibody is a special kind of protein that the immune (defense) system normally makes to fight bacteria and viruses. Bullous pemphigoid is an autoimmune subepidermal blistering disease, predominately affecting the elderly (typical onset after age 60).
The study is looking at several other research questions, including:
* Side effects that may be experienced by people taking dupilumab
* How dupilumab works in the body and affects the body
* How dupilumab affects quality of life
* How much dupilumab is present in the blood
* To see if dupilumab works to wean the patient off oral corticosteroids
Eligibility Criteria
Key Inclusion Criteria
- Patients must have characteristic clinical features of bullous pemphigoid (BP) (eg, urticarial or eczematous or erythematous plaques, bullae, pruritus) at the screening and baseline visits.
- Study participants are required to have a confirmed diagnosis of BP based on histopathology, immunopathology, and serology at the baseline visit, as defined in the protocol.
- Bullous Pemphigoid Disease Area Index (BPDAI) activity score ≥24 at baseline and screening visits.
- Baseline peak pruritus NRS score for maximum itch intensity ≥4
- Karnofsky performance status score ≥50% at the screening visit.
Key Exclusion Criteria
- Forms of pemphigoid other than classic BP (eg, Brunsting-Perry cicatricial pemphigoid, anti-p200 pemphigoid, epidermolysis bullosa acquisita, or BP with concomitant pemphigus vulgaris)
- Patients who are receiving treatments known to cause or exacerbate BP (eg, angiotensin converting enzyme inhibitors, penicillamine, furosemide, phenacetin, dipeptidyl peptidase 4 inhibitor) who have not been on a stable dose of these medications for at least 4 weeks prior to the screening visit
- Have ever received treatment with an IL-4 or IL-13 antagonist such as dupilumab, tralokinumab, or lebrikizumab.
- Treatment with systemic corticosteroids within 7 days before the baseline visit
- Treatment with topical corticosteroids of medium potency or higher, topical calcineurin inhibitor, or topical crisaborole within 7 days before the baseline visit
- Treatment with non-steroidal immunosuppressive/immunomodulating drug(s) (eg, mycophenolate mofetil, azathioprine, or methotrexate) within 4 weeks before the baseline visit.
- Treatment with BP-directed biologics as follows:
- Any cell-depleting agents including but not limited to rituximab: within 12 months before the baseline visit, or until lymphocyte and CD 19+ lymphocyte count returns to normal, whichever is longer
- Other biologics (such as IL-5 inhibitors benralizumab or mepolizumab): within 5 half-lives (if known) or 16 weeks prior to the baseline visit, whichever is longer
- Intravenous immunoglobulin within 16 weeks prior to the baseline visit
NOTE: Other Protocol Defined Inclusion/Exclusion Criteria Apply
Data sourced from ClinicalTrials.gov (NCT04206553). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.