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

NCT04206553 | Completed Phase 2 Results DMC FDA Drug

• 3 other identifiers: R668-BP-19022019-003520-202024-510745-34-00
• Study Type: interventional
• Target: 106
• Locations: 9 countries
• Sites: 53

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

Conditions

Bullous Pemphigoid

Outcome Measures

Primary Outcomes (1)

• Percent of Participants Achieving Sustained Remission 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.

  At Week 36

Secondary Outcomes (47)

• Total Cumulative Dose of Oral Corticosteroids (OCS) From Baseline to Week 36

  Week 36

• Percent Change in Weekly Average of Daily Peak Pruritus Numerical Rating Scale (NRS) Score From Baseline to Week 36

  Baseline, Week 36

• Percent of Participants With Improvement (Reduction) of Weekly Average of Daily Peak Pruritus NRS ≥4 From Baseline to Week 36

  Baseline, Week 36

• Percent Change in Bullous Pemphigoid Disease Area Index (BPDAI) Activity Score From Baseline to Week 36

  Baseline, Week 36

• Time to First Use of Rescue Medication Up to Week 36

  Up to Week 36

Other Outcomes (1)

• Supplemental Analysis of Total Cumulative Dose of OCS From Baseline to Week 36

  Week 36

Study Arms (2)

dupilumab

EXPERIMENTAL

Drug: dupilumab; Drug: Oral corticosteroids (OCS)

Matching placebo

EXPERIMENTAL

Drug: Matching Placebo; Drug: Oral corticosteroids (OCS)

Interventions

dupilumab DRUG

Loading dose administered subcutaneous (SC), followed by SC once every 2 weeks (Q2W) dosing.

Also known as: Dupixent®, REGN668, SAR231893

dupilumab

Matching Placebo DRUG

Matching dupilumab without active substance

Matching placebo

Oral corticosteroids (OCS) DRUG

Prednisone or prednisolone per standard of care to obtain control of disease activity.

Matching placebo; dupilumab

Eligibility Criteria

• Age: 18 Years - 90 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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

Sponsors & Collaborators

• Regeneron Pharmaceuticals: lead
• Sanofi: collaborator

Study Sites (53)

Regeneron study Site

Birmingham, Alabama, 35233, United States

Location

Regeneron Study Site

Scottsdale, Arizona, 85259, United States

Location

Regeneron Study Site

Redwood City, California, 94063, United States

Location

Regeneron Study Site

Farmington, Connecticut, 06030, United States

Location

Regeneron Study Site

Miami, Florida, 33125, United States

Location

Regeneron study Site

Orlando, Florida, 32827, United States

Location

Regeneron Study Site

Iowa City, Iowa, 52242, United States

Location

Regeneron Study Site

Boston, Massachusetts, 02116, United States

Location

Regeneron study Site

Ann Arbor, Michigan, 48109-5314, United States

Location

Regeneron Study Site

Chapel Hill, North Carolina, 27516, United States

Location

Regeneron Study Site

Portland, Oregon, 97239, United States

Location

Regeneron Study Site

Philadelphia, Pennsylvania, 19104, United States

Location

Regeneron study Site

Providence, Rhode Island, 02908, United States

Location

Regeneron Study Site

Murray, Utah, 84107, United States

Location

Regeneron study Site

Charlottesville, Virginia, 22903, United States

Location

Regeneron Study Site

Kogarah, New South Wales, 2217, Australia

Location

Regeneron Study Site

Box Hill, Victoria, 3128, Australia

Location

Regeneron Study Site

Bobigny, 93009, France

Location

Regeneron Study Site

Bordeaux, 33000, France

Location

Regeneron Study Site

Lille, 59037, France

Location

Regeneron Study Site

Nice, 06200, France

Location

Regeneron Study Site

Paris, 75010, France

Location

Regeneron Study Site

Rouen, 76031, France

Location

Regeneron Study Site

Münster, North Rhine-Westphal, 48149, Germany

Location

Regeneron Study Site

Mainz, Rhineland-Palatinate, 55131, Germany

Location

Regeneron Study Site 2

Dresden, Saxony, 01307, Germany

Location

Regeneron Study Site

Lübeck, Schleswig-Holstein, 23538, Germany

Location

Regeneron Study Site

Berlin, 10117, Germany

Location

Regeneron Study Site

Buxtehude, 21614, Germany

Location

Regeneron Study Site

Erlangen, 91054, Germany

Location

Regeneron Study Site

Freiburg im Breisgau, 79104, Germany

Location

Regeneron Study site'

Magdeburg, 39120, Germany

Location

Regeneron Study site

Marburg, 35043, Germany

Location

Regeneron Study Site

Munich, 80802, Germany

Location

Regeneron Study Site

Stuttgart, 70178, Germany

Location

Regeneron study Site

Afula, 1834111, Israel

Location

Regeneron study Site

Petah Tikva, 49100, Israel

Location

Regeneron study Site

Tel Aviv, 6423906, Israel

Location

Regeneron Study site

Kurume, Hukuoka, 830-0011, Japan

Location

Regeneron Study Site

Hirosaki, 036-8563, Japan

Location

Regeneron Study Site

Ichinomiya, 491-8558, Japan

Location

Regeneron Study Site

Osaka, 545-8586, Japan

Location

Regeneron Study Site

Sapporo, 060-8648, Japan

Location

Regeneron Study Site

Tokyo, 160-8582, Japan

Location

Regeneron Study Site

Krakow, Lesser Poland Voivodeship, 31-147, Poland

Location

Regeneron Study site'

Ossy, 42-624, Poland

Location

Regeneron Study site'

Wroclaw, 50566, Poland

Location

Regeneron Study Site

Badalona, Barcelona, 08916, Spain

Location

Regeneron Study Site

Madrid, 28034, Spain

Location

Regeneron study Site

Madrid, 28046, Spain

Location

Regeneron study Site

Pamplona, 31008, Spain

Location

Regeneron study Site

Taipei, Zhongzheng District, 10002, Taiwan

Location

Regeneron study Site

Taoyuan, 33305, Taiwan

Location

Related Publications (1)

• Murrell DF, Joly P, Werth VP, Ujiie H, Worm M, Mangold AR, Avetisova E, Maloney J, Laws E, Mortensen E, Dubost-Brama A, Shabbir A. Study Design of a Phase 2/3 Randomized Controlled Trial of Dupilumab in Adults with Bullous Pemphigoid: LIBERTY-BP ADEPT. Adv Ther. 2024 Jul;41(7):2991-3002. doi: 10.1007/s12325-024-02810-3. Epub 2024 Mar 5.

  PMID: 38443648 DERIVED

MeSH Terms

Conditions

Pemphigoid, Bullous

Interventions

dupilumab; Adrenal Cortex Hormones

Condition Hierarchy (Ancestors)

Skin Diseases, Vesiculobullous; Skin Diseases; Skin and Connective Tissue Diseases; Autoimmune Diseases; Immune System Diseases

Intervention Hierarchy (Ancestors)

Hormones; Hormones, Hormone Substitutes, and Hormone Antagonists

Results Point of Contact

• Title: Clinical Trials Administrator
• Organization: Regeneron Pharmaceuticals, Inc.

clinicaltrials@regeneron.com

844-734-6643

Study Officials

• Clinical Trial Management

  Regeneron Pharmaceuticals

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 18, 2019
• First Posted: December 20, 2019
• Study Start: October 28, 2020
• Primary Completion: July 12, 2024
• Study Completion: January 5, 2025
• Last Updated: March 6, 2026
• Results First Posted: March 6, 2026

Record last verified: 2026-02

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ICF, CSR, ANALYTIC CODE
• Time Frame: When Regeneron has: * received marketing authorization from major health authorities (e.g., FDA, European Medicines Agency (EMA), Pharmaceuticals and Medical Devices Agency (PMDA), etc.) for the product and indication or has globally discontinued development of the product for all indications on or after April 2020 and has no plans for future development * made the study results publicly available (e.g., scientific publication, scientific conference, clinical trial registry) * the legal authority to share the data, and * ensured the ability to protect participant privacy
• Access Criteria: Qualified researchers can submit a proposal for access to individual patient or aggregate level data from a Regeneron-sponsored clinical trial through Vivli. Regeneron's Independent Research Request Evaluation Criteria can be found at: https://www.regeneron.com/sites/default/files/Regeneron-External-Data-Sharing-Policy-and-Independent-Research-Request-Evaluation-Criteria.pdf

Locations

IL (3); US (15); JP (6); PL (3); TW (2); FR (6); ES (4); AU (2); DE (12)