Pemphigus Vulgaris or Pemphigus Foliaceus MedDRA version: 20.0 Level: LLT Classification code 10052802 Term: Pemphigus vulgaris System Organ Class: 10040785 - Skin and subcutaneous tissue disorders MedDRA version: 20.0 Level: LLT Classification code 10057069 Term: Pemphigus foliaceus 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: AR
Sponsors
argenx BV
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: Participants must meet all of the following inclusion criteria: 1. Ability to understand the requirements of the trial, to provide written informed consent (including consent for the use and disclosure of research-related health information), willingness and ability to comply with the trial protocol procedures (including required trial visits). 2. The participant participated in trial ARGX-113-1904 and completed the study or has the defined criteria for rollover. 3. Contraceptive use by men and women should be consistent with local regulations regarding the methods of contraception for those participating clinical trials and: Women of childbearing potential: a. Male participants: - Male participants must agree to use an acceptable method of contraception from signing the ICF until the last dose the study drug. b. Female participants: - Women of childbearing potential have a negative urine pregnancy test at baseline before the IMP can be administered. - agree to use a highly effective or acceptable contraception method, which should be maintained at minimum until after 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 153 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 27
Exclusion criteria
Exclusion criteria: Participants are excluded from the trial if any of the following criteria apply: 1. Pregnant and lactating women and those intending to become pregnant during the trial. 2. Participants with clinical evidence of other significant serious disease or participants who recently underwent or have planned a major surgery during the period of the trial, or any other condition in the opinion of the investigator, that could confound the results of the trial or put the participant at undue risk. 3. Known hypersensitivity to any of the components of the administered treatments.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To assess the safety of treatment, extended treatment and re-treatment with efgartigimod PH20 SC in participants with PV or PF;Secondary Objective: To evaluate the efficacy of efgartigimod PH20 SC treatment in PV and PF To assess the health impact of glucocorticoid (GC) use in participants with PV or PF To evaluate the effects of efgartigimod PH20 SC on quality of life (QoL) in participants with PV or PF To evaluate the pharmacokinetics (PK) of efgartigimod PH20 SC in participants with PV or PF To evaluate the PD of efgartigimod PH20 SC in participants with PV or PF To evaluate the immunogenicity of efgartigimod PH20 SC in participants with PV or PF To explore feasibility of self-administration of efgartigimod PH20 SC;Primary end point(s): 1. Incidence and severity of treatment-emergent adverse events (TEAEs), adverse events of special interest (AESIs), and serious adverse events (SAEs) by system organ class (SOC) and preferred term (PT) 2. Vital signs, physical examination, electrocardiogram (ECG), and clinical laboratory safety evaluations;Timepoint(s) of evaluation of this end point: Up to 60 weeks | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): 1. Proportion of PV participants who achieve CR min 2. Proportion of PV and PF participants who achieve CR min. 3. Time to DC 4. Time to CR 5. Time to CR min 6. Time to CR off 7. Time to flare 8. Rate of treatment failure 9. Rate of flare 10. Cumulative prednisone dose over the trial 11. Pemphigus Disease Area Index (PDAI) at each visit 12. Composite Glucocorticoid Toxicity Index (CGTI) comprising the Aggregate improvement Score (AIS) and the Cumulative Worsening Score (CWS) 13. EuroQol 5-Dimension 5-Level (EQ-5D-5L) score 14. Autoimmune Bullous Disease Quality of Life (ABQOL) score 15. Efgartigimod serum concentrations 16. Total IgG and subtype (IgG1, IgG2, IgG3, IgG4) serum levels 17. Anti-Dsg-1 and -3 autoantibodies serum levels 18. Immunogenicity to efgartigimod (serum levels) 19. Percentage of participants who performed self-administration 20. Percentage of caregivers who administered the injection to the participant. 21. Number of visits needed for the participant or caregiver to be competent to start administering efgartigimod PH20 SC 22. Frequency of self- or caregiver-supported administration at home ;Timepoint(s) of evaluation of this end point: 1,2,10,12,13,14,19,20,21 and 22: up to 52 weeks treatment period 3,4,5,6,7,8,9,11,15,16,17 and 18: up to 60 weeks | — |
Countries
Australia, Bulgaria, China, Croatia, France, Georgia, Germany, Greece, Hungary, India, Israel, Italy, Japan, Netherlands, Poland, Portugal, Romania, Russian Federation, Serbia, Spain, Türkiye, Ukraine, United Kingdom, United States
Contacts
Public ContactRegulatory
argenx BV
Outcome results
None listed