Bullous Pemphigoid MedDRA version: 21.1 Level: LLT Classification code 10006567 Term: Bullous pemphigoid System Organ Class: 100000004858
Conditions
Sponsors
argenx BV
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: The participant is willing and able to do the following: a)understand the requirements of the study b)provide written informed consent c)comply with the study protocol procedures. * The participant is male or female, has reached the local legal age of consent at the time of signing the informed consent form (ICF). * Participants have clinical signs of BP. * The participant agrees to use contraceptive measures consistent with the local regulations. WOCBP must have a negative Serum pregnancy test at screening and a negative urine pregancy test at baseline before receiving IMP. The full list of inclusion criteria can be found in the protocol. Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 40 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 120
Exclusion criteria
Exclusion criteria: • Other forms of pemphigoid or other autoimmune bullous diseases (AIBDs). • 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 oral corticosteroids (OCS), topical corticosteroids (TCS), conventional immunosuppressants or dapsone. • Known contraindication to OCS therapy. • Active, or chronic or latent infection at screening. • Positive Covid-19 test result at screening (testing performed if required per local regulations) • 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: o Basal cell or squamous cell skin cancer o Carcinoma in situ of the cervix o Carcinoma in situ of the breast o Incidental histological finding of prostate cancer • 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 • Use of an investigational product within 3 months before the first dose of IMP • Previously participated in a clinical study with efgartigimod or currently participating in another interventrional clinical study • Known hypersensitivity to any of the components of the administered treatments • Positive serum test at screening for an active infection: o HBV o HCV o HIV • Current or history (ie, within 12 months of screening) of alcohol, drug, or medication abuse as assessed by the Investigator • Pregnant or lactating females and those who intend to become pregnant during the study • Live or live-attenuated vaccine received < 4weeks before baseline visit The full list of exclusion criteria can be found in the protocol.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: Part A and B: To evaluate the efficacy of efgartigimod PH20 SC on achieving sustained remission in the treatment of participants with bullous pemphigoid (BP).;Secondary Objective: Parts A and B, To: • evaluate the corticosteroid-sparing effects of efgartigimod PH20 SC in participants with BP • characterize the overall efficacy of efgartigimod PH20 SC in the treatment of participants with BP • evaluate the efficacy of efgartigimodPH20 SC in preventing relapse of BP • evaluate the effect of efgartigimod PH20 SC on pruritus in participants with BP • assess the safety and tolerability of efgartigimod PH20 SC administered to participants with BP • assess glucocorticoid-associated morbidity and evaluate the impact of efgartigimodPH20 SC on reducing glucocorticoid toxicity • evaluate the effects of efgartigimod PH20 SC on the quality of life of participants with BP • evaluate the PK of efgartigimod PH20 SC in participants with BP • evaluate the PD of efgartigimod PH20 SC in participants with BP • evaluate the immunogenicity of efgartigimod PH20 SC in participants with BP • evaluate the competency of participants or caregivers to (self-)administer EFG PH20 SC;Primary end point(s): Proportion of participants who are in CR while receiving efgartigimod PH20 SC or placebo and have been off OCS therapy for =8 weeks at week 36. ;Timepoint(s) of evaluation of this end point: At week 36. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Part A and B: 1. Cumulative dose of oral corticosteroid (OCS) from baseline to week 36 2. Proportion of participants who achieve an IGA-BP score of 0 while receiving efgartigimod PH20 SC or placebo and have been off OCS therapy for =8 weeks at week 36 therapy for =8 weeks at week 36 3. Proportion of participants who achieve an IGA-BP score of 0 or 1 while receiving efgartigimod PH20 SC or placebo and have been off OCS therapy for =8 weeks at week 36 4. Proportion of participants who achieve an IGA-BP score of 0 or 1 while receiving efgartigimod PH20 SC or placebo at any time through week 36 5. Changes from baseline in the BPDAI activity score 6. 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 =0.1 mg/kg/day of prednisone [or an equivalent dose of another OCS].) 7. Time to achieve the following: -CDA -CR -CR while being on minimal OCS therapy for =8 weeks -CR/PR while being off OCS therapy for =8 weeks -CR while being off OCS therapy for =8 weeks -Relapse 8. Cumulative OCS dose for the participant at the timepoints when they exhibit the following: -CDA -CR -CR while being on minimal OCS therapy for =8 weeks -CR/PR while being off OCS therapy for =8 weeks -CR while being off OCS therapy for =8 weeks -Relapse 9. Proportion of participants who receive rescue therapy before week 36 10. Proportion of participants who achieve CDA while receiving efgartigimod PH20 SC or placebo and remain free of relapse through week 36 11. Changes from baseline in the 24-hour average itch score from the Itch NRS 12. Changes from baseline in the 24-hour worst itch score from the Itch NRS 13. Incidence and severity of TEAEs, AESIs, and SAEs 14. The Aggregate Improvement Score (AIS) from the Glucocorticoid Toxicity Index (GTI) 15. The Cumulative Worsening Score (CWS) from the GTI 16. The GTI specific List (GTI-SL) 17. EQ-5D- | — |
Countries
Australia, Bulgaria, Canada, China, Croatia, Czechia, France, Germany, Greece, Hungary, Israel, Italy, Japan, Latvia, Netherlands, Poland, Romania, Serbia, Slovakia, Spain, United Kingdom, United States
Contacts
Public ContactRegulatory
argenx BV
Outcome results
None listed