Bullous Pemphigoid Lever's Pemphigoid
Conditions
Interventions
Participants with moderate-to- severe BP will be randomly assigned in a 1:1
ratio to 1 of the following study intervention groups:
Efgartigimod*PH20 SC or Placebo*PH20*SC
Efgartigimod PH20 SC or pla
Sponsors
PPD
Eligibility
Age
18 Years to 99 Years
Inclusion criteria
Inclusion criteria: Participants are eligible to be included in the study only if all of the following criteria apply: 1. The participant is willing and able to do the following: a. understand the requirements of the study b. provide written informed consent (including consent for the use and disclosure of research-related health information) c. comply with the study protocol procedures (including required study visits). 2. The participant is male or female and has reached the local legal age of consent at the time of signing the informed consent form (ICF). 3. Participants have 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. The diagnosis of BP must be 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 (Section 8.1.2). 4. The participant has an IGA BP score of 3 or 4 at screening and baseline. 5. The participant has a Karnofsky performance status of at least 60% at screening. 6. The participant agrees to use contraceptive measures consistent with local regulations. WOCBP must have a negative serum pregnancy test at screening and a negative urine pregnancy test at baseline before receiving IMP. See protocol for the full list of criteria
Exclusion criteria
Exclusion criteria: Participants are excluded from the study if any of the following criteria apply: 1. Other forms of pemphigoid (including but not limited to pemphigoid gestationis, drug induced BP that resolves after culprit-drug withdrawal, anti-p200 pemphigoid, mucous membrane pemphigoid, and cicatricial pemphigoid), or other AIBDs (including but not limited to epidermolysis bullosa acquisita, pemphigus vulgaris, and exfoliative erythroderma) 2. Received unstable dose of treatments known to cause or exacerbate BP (eg, angiotensin converting enzyme inhibitors, penicillamine, furosemide, phenacetin, dipeptidyl peptidase 4 inhibitor) for at least 4 weeks prior to the baseline visit 3. Use of BP treatments other than OCS, TCS, conventional immunosuppressants (eg, azathioprine, cyclophosphamide, methotrexate, mycophenolate mofetil), or dapsone, including the following: a. sulfasalazine, IVIg, subcutaneous administration of immunoglobulin (SCIg), immunoadsorption or plasma exchange within 8 weeks of the baseline visit b. tetracyclines with or without nicotinamide at doses higher than the recommended daily allowance (RDA)/dietary reference intake (DRI) within 2 weeks of the baseline visit c. any monoclonal antibody (including rituximab or another anti-CD20 biologic) within 6 months of the baseline visit d. complementary therapies*such as traditional Chinese medicines, herbs, or procedures (eg, acupuncture)*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 4. Known contraindication to OCS therapy 5. Active, chronic or latent infection at screening 6. Positive COVID-19 test result at screening (testing performed if required per local regulations) 7. Any other known autoimmune disease that, in the opinion of the investigator, would interfere with an accurate assessment of clinical symptoms of BP or put the participant at undue risk 8. History of malignancy unless deemed cured by adequate treatment with no evidence of recurrence for >=3 years before the first administration of the IMP. Participants with the following cancers can be included at any time, provided they are adequately treated prior to their participation in the study: a. Basal cell or squamous cell skin cancer b. Carcinoma in situ of the cervix c. Carcinoma in situ of the breast d. Incidental histological finding of prostate cancer (tumor-node metastasis [TNM] stage T1a or T1b) 9. Clinical evidence of other significant serious diseases, have had a recent surgery, or who have any other condition that, in the opinion of the investigator, could confound the results of the study or put the patient at undue risk or prevent participants from complying with protocol requirements. 10. Use of an investigational product within 3 months or 5 half-lives (whichever is longer) before the first dose of IMP 11. Previously participated in a clinical study with efgartigimod or currently participating in another interventional clinical study. 12. Known hypersensitivity to any of the components of the administered treatments 13. Positive serum test at screening for an active infection with any of the following conditions: a. HBV that is indicative of an acute or chronic infection, unles
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Parts A and B: Proportion of participants who are in complete remission (CR) while receiving efgartigimod PH20 SC or placebo and have been off oral corticosteroid (OCS) therapy for >=8 weeks at week 36 | — |
Secondary
| Measure | Time frame |
|---|---|
| Part B only: 1. Cumulative dose of OCS from baseline to week 36 2. Proportion of participants who achieve an Investigator Global Assessment of Bullous Pemphigoid (IGA BP) score of 0 while receiving efgartigimod PH20 SC or placebo and have been off OCS therapy for >=8 weeks at week 36 3. Proportion of participants who achieve control of disease activity (CDA) while receiving efgartigimod PH20 SC or placebo and remain free of relapse through week 36 4. Proportion of participants who are in CR while receiving efgartigimod PH20 SC or placebo and have been receiving minimal OCS therapy for >=8 weeks at week 36. (Minimal OCS therapy is defined as | — |
Countries
Netherlands
Outcome results
None listed