A Study to Evaluate the Efficacy and Safety of Dupilumab in Adult Patients With Bullous Pemphigoid

Phase 2

Completed

• Conditions: Bullous Pemphigoid
• Interventions: Drug: dupilumab; Drug: Matching Placebo; Drug: Oral corticosteroids (OCS)

• Registration Number: NCT04206553
• Lead Sponsor: Regeneron Pharmaceuticals

Brief 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

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2019-12-18
• Study First Submitted QC: 2019-12-18
• Study First Posted: 2019-12-20
• Study Start: 2020-10-28
• Primary Completion: 2024-07-12
• Status Verified: 2025-01
• Study Completion: 2025-01-05
• Last Update Submitted: 2025-02-03
• Last Update Posted: 2025-02-06

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 98

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
dupilumab	Oral corticosteroids (OCS)	-
dupilumab	dupilumab	-
Matching placebo	Oral corticosteroids (OCS)	-
Matching placebo	Matching Placebo	-

Primary Outcome Measures

Name	Time	Method
Proportion of patients achieving sustained remission	Week 36

Secondary Outcome Measures

Name	Time	Method
Time to first use of rescue medication	Up to week 36
Proportion of patients with improvement (reduction) of weekly average of daily peak pruritus NRS ≥4	Baseline to week 36
Percent change in Bullous Pemphigoid Disease Area Index Activity Score (BPDAI) activity score	Baseline to 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.
Percent change in weekly average of daily peak pruritus numerical rating score (NRS)	Baseline to week 36	Individual NRS used to rate the intensity of pruritus using an 11-point scale (0 to 10) in which 0 indicates no itch while 10 indicates worst itch possible.
Total cumulative dose of oral corticosteroids (OCS)	Baseline to week 36

Trial Locations

Locations (5)

Regeneron study Site; 🇨🇳 Taoyuan City, Taiwan

Regeneron Study Site 2; 🇩🇪 Dresden, Saxony, Germany

Regeneron Study site'; 🇵🇱 Wroclaw, Poland

Regeneron Study site; 🇯🇵 Kurume, Hukuoka, Japan

Regeneron Study Site; 🇪🇸 Madrid, Spain