A Study to Evaluate the Efficacy and Safety of Dupilumab in Adult Patients With Bullous Pemphigoid (LIBERTY-BP)

Regeneron Pharmaceuticals

Status and phase: Completed; Phase 3; Phase 2

Conditions: Bullous Pemphigoid

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

Study type: Interventional

Funder types: Industry

Identifiers

NCT04206553

2024-510745-34-00 (Registry Identifier)

2019-003520-20 (EudraCT Number)

R668-BP-1902

Details and patient eligibility

About

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

Enrollment: 106 patients

Sex: All

Ages: 18 to 90 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion 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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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106 participants in 2 patient groups

dupilumab

Experimental group

Treatment:

Drug: Oral corticosteroids (OCS)

Drug: dupilumab

Matching placebo

Experimental group

Treatment:

Drug: Matching Placebo

Drug: Oral corticosteroids (OCS)