  
  Clinical Study in 
Bullous Pemphigoid (BP)

CLINICALTRIALS.GOV ID: NCT05267600

A Phase 2/3, Randomized, Double-Blinded, Placebo-Controlled, Parallel-Group Study to Investigate the Efficacy and Safety of Efgartigimod PH20 SC in Adult Participants With Bullous Pemphigoid

About Bullous Pemphigoid (BP)

Bullous Pemphigoid is caused by the disruption of the dermoepidermal junction by pathogenic autoantibodies resulting in large, tense bullae on the skin, progressing to epidermal erosion, crusting and urticarial plaque formation.

Classification of moderate to severe BP - presence of bullae with or without the presence of urticarial (hives), eczematous, or erythematous plaques, and with or without pruritus.

Moderate: Multiple (10 to 29) blisters or erosions and/or multiple erythematous, eczematous or urticarial lesions (involving 10-30% of the body surface area) and/or at the most, 2 large erosive areas (>5 cm) or large blisters (>5 cm).
Severe: Extensive (≥30) blisters or erosions and/or extensive erythematous, eczematous or urticarial lesions (involving >30% of the body surface area) and/or 3 or more large erosive areas (>5 cm) or large blisters (>5 cm).

About the Investigational Therapy

Efgartigimod is an investigational drug currently being studied in a variety of disease states. Efgartigimod is currently not approved for use in Bullous Pemphigoid by any regulatory agency, as efficacy and safety have not been established.

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A Phase 2/3, Global, Multi-Center, Randomized, Double-Blinded, Placebo-Controlled, Parallel-Group Study

STUDY DESIGN

PRIMARY ENDPOINT:

Complete remission while receiving efgartigimod PH20 SC or placebo and have been off oral corticosteroid (OCS) therapy for ≥8 weeks

at week 36.

DURATION:

Screening Period: 2-3 weeks
Treatment Period: 36 weeks
Follow up Period: 7 weeks OR roll over to Open-Label Extension Study

TREATMENT:

1:1 randomization to efgartigimod PH20 SC or placebo
2 injections on weeks 1 & 2 then 1 injection once a week
Concurrent treatment with daily oral prednisone (or an equivalent OCS)

VISITS:

Some visits take place at the study center

Some visits may take place at the patient’s home with a trained nurse, depending on disease activity

MONITORING:

Throughout the study, participants will be monitored to determine if prednisone doses may be reduced

Key Eligibility Criteria

INCLUSION

Adults (≥18 years old) with a diagnosis of BP confirmed by positive histopathology and DIF before randomization to treatment assignment, and by positive serology (by IIF, CLEIA, or ELISA, according to local practice) at screening.
Clinical signs of BP (ie, presence of bullae), with or without the presence of urticarial/eczematous/erythematous plaques or pruritus at the screening and baseline visits.
Investigator Global Assessment of Bullous Pemphigoid (IGA-BP) score of 3 or 4 at screening and baseline, indicating moderate to severe BP.
Karnofsky performance status of at least 60% at screening.
No male contraception is required.
Females: must agree to use a highly effective or acceptable contraception method from the time that the ICF is signed until the date of their last dose of IMP.

EXCLUSION

Drug-induced BP or other forms of pemphigoid/autoimmune bullous diseases.
Received unstable dose of treatments known to cause or exacerbate BP for at least 4 weeks prior to the baseline visit.
Use of BP treatments other than OCS such as topical corticosteroids, conventional immunosuppressants, or dapsone.
History of malignancy unless deemed cured for ≥3 years except basal/squamous cell carcinoma, carcinoma in situ of cervix/breast, or prostate cancer (T1a or b) may be included at any time.
Other serious diseases or recent surgery.
Previously participated in a clinical study with efgartigimod.
Active, chronic, or latent infection at screening.
Total IgG serum levels <4 g/L at screening.
Pregnant or lactating females.

PROHIBITED TREATMENTS DURING THE STUDY

Topical corticosteroids, conventional immunosuppressants (e.g. azathioprine, cyclophosphamide, methotrexate, mycophenolate mofetil), or dapsone must be discontinued at the baseline visit; no washout required.
Tetracyclines with or without nicotinamide at doses higher than the recommended daily allowance/dietary reference intake must be discontinued within 2 weeks of the baseline visit.
Complementary therapies, such as traditional Chinese medicines, herbs, or procedures (e.g. acupuncture), must be discontinued within 4 weeks (or 5 half-lives) of the baseline visit, if the investigator determines that such therapies may interfere with the study’s efficacy assessments and/or potentially risk the safety of the participant.
Sulfasalazine, IVIg, subcutaneous administration of immunoglobulin, immunoadsorption or plasma exchange must be discontinued within 8 weeks of the baseline visit.
Other investigational product must be discontinued within 3 months (or 5 half-lives) before the first dose of IMP.
Any monoclonal antibody (including rituximab or another anti-CD20 biologic) must be discontinued within 6 months of the baseline visit.