Bullous Pemphigoid MedDRA version: 21.1 Level: LLT Classification code 10006567 Term: Bullous pemphigoid System Organ Class: 10040785 - Skin and subcutaneous tissue disorders
Conditions
Interventions
Product Name: Efgartigimod PH20 SC
Pharmaceutical Form: Solution for injection
INN or Proposed INN: Efgartigimod Alfa
CAS Number: 1821402-21-4
Current Sponsor code: ARGX-113
Other descriptive name: Ef
Sponsors
argenx BV
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Has completed the week 36 visit of ARGX-113-2009 2. Is capable of providing signed informed consent and complying with protocol requirements 3. Agrees to use contraceptive measures consistent with local regulations and the following: a. Male participants: An acceptable method of contraception is a condom. All nonsterilized male participants must use this method from signing of the ICF until the date of the last dose of IMP. b. Women of childbearing potential must have a negative urine pregnancy test at baseline before receiving IMP. WOCBP must use one of the contraception methods described in the protocol from signing the ICF until the last dose of IMP 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: 1. Clinically significant disease, recent major surgery (within 3 months of baseline), or intention to have surgery during the study; or any other medical condition that, in the investigator’s opinion would confound the results of the study or put the participant at undue risk 2. Known hypersensitivity to IMP or 1 of its excipients 3. Permanently discontinued IMP in ARGX-113-2009 due to an AE considered related to IMP and for whom the benefit/risk balance is not considered positive
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To assess the long-term safety and tolerability of treatment with efgartigimod PH20 SC in participants with BP;Secondary Objective: To assess the long-term efficacy and durability of response with efgartigimod PH20 SC treatment in participants with BP To evaluate the impact of efgartigimod PH20 SC treatment in reducing long-term glucocorticoid-associated toxicity in participants with BP To evaluate the impact of efgartigimod PH20 SC treatment on QoL in participants with BP To evaluate the PD and immunogenicity of efgartigimod PH20 SC in participants with BP;Primary end point(s): Incidence and severity of treatment-emergent adverse events (TEAEs), serious AEs (SAEs), and AEs of special interest (AESIs) Rate of treatment discontinuation because of safety concerns ;Timepoint(s) of evaluation of this end point: Up to wk 56 | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): 1. Proportions of participants achieving: - complete remission (CR) while off oral corticosteroids (OCS) for =8 weeks - CR or partial remission (PR) while off OCS for =8 weeks - CR while on minimal OCS therapy for =8 weeks. (Minimal OCS therapy is defined as =0.10 mg/kg/day of prednisone [or an equivalent dose of another OCS]) - CR while off both OCS and efgartigimod PH20 SC for =8 weeks - CR or PR while off both OCS and efgartigimod PH20 SC for =8 weeks 2. Duration of sustained remission 3. Proportion of participants who relapse 4. Time to relapse 5. Incidence and severity of relapse 6. Bullous Pemphigoid Disease Area Index (BPDAI) activity scores, Investigator Global Assessment of Bullous Pemphigoid (IGA-BP) scores, and itch numerical rating scale (NRS) over time 7. Rate of treatment failure 8. GTI-related scores, including the GTI-AIS, GTI CWS, and GTI SL over time 9. EQ-5D-5L, DLQI, and ABQoL scores over time 10. Percent change from baseline over time for anti BP180 and anti BP230 antibody levels 11. Incidence and prevalence of ADA against efgartigimod (serum levels);Timepoint(s) of evaluation of this end point: 1-5 & 7: Up to wk 56 6: At wks 0, 2, 4, 8, 16, 24, 32, 40, 48 & 56 when EFG is NA. When EFG continues/starts its evaluated at wks 0, 2, 4, 8, then every 4 wks to EFG stop, every 8 wks after & at wks 48, 52 & 56 8 & 9: At wks 0, 24 & 48 when EFG is NA. When EFG continues/starts its evaluated at wks 0, 8, every 16 wks up to & after EFG stop, then at wk 48 10: At wks 0, 2, 4, 8, 16, 24, 32, 40, 48 & 56 when EFG is NA. When EFG continues/starts its evaluated at wks 0, 2, 4, 8, then every 4 wks to EFG stop, every 8 wks after, then at wks 48, 52 & 56 11: At wks 0, 2, 4, 8, 16, 24, 32, 40, 48 & 56 when EFG is NA. When EFG continues/starts its evaluated at wks 0, 4, 8, then every 8 wks to EFG stop, every 8 wks after, then at wks 48, 52 & 56 | — |
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