Health Condition 1: L102- Pemphigus foliaceous Health Condition 2: L100- Pemphigus vulgaris
Conditions
Interventions
Intervention1: ARGX-113 (Efgartigimod PH20 SC): Efgartigimod PH20 will be administered subcutaneously either weekly 1000 mg or 2000 mg on day 1 and day 8, followed by weekly 1000 mg with treatment bei
Sponsors
argenx BV
Eligibility
Inclusion criteria
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 patient participated in trial ARGX-113-1904 and completed the study or has the defined criteria for rollover. 3. Women of childbearing potential: a. Must have a negative urine pregnancy test at baseline before trial medication can be administered. b. Must be on a stable regimen for at least 1 month of at least 1 highly effective method of contraception (ie, failure rate of less than 1% per year) during the trial and for 90 days after the last administration of IMP. 4. Non-sterilized male patients who are sexually active with a female partner of childbearing potential must use effective contraception from first administration of IMP through 90 days after the last administration of the IMP. Male patients practicing true sexual abstinence (as consistent with preferred and usual life style) can be included. Sterilized male patients who have had a vasectomy and with documented absence of sperm post-procedure can be included. Male patients are not allowed to donate sperm from first administration of IMP through 90 days after the last dose of IMP.
Exclusion criteria
Exclusion criteria: 1. Pregnant and lactating women and those intending to become pregnant during the trial or within 90 days after the last administration of IMP. 2. Patients with clinical evidence of other significant serious disease or patients 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 patient at undue risk. 3. Known hypersensitivity to any of the components of the administered treatments.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| To assess the safety of extended treatment and retreatment with efgartigimod PH20 SC in patients with PV or PFTimepoint: a. 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)- Upto 60 weeks. b. Vital sign measurements, physical examinations, electrocardiograms (ECGs), and clinical laboratory safety evaluations- Upto 60 weeks | — |
Secondary
| Measure | Time frame |
|---|---|
| To evaluate the effects of efgartigimod PH20 SC on quality of life (QoL) in patients with PV or PFTimepoint: a. EuroQol 5-Dimension 5-Level (EQ-5D-5L) score- Up to 52 weeks treatment period b. Autoimmune Bullous Disease Quality of Life (ABQOL) score- Up to 52 weeks treatment period;To evaluate the efficacy of efgartigimod PH20 SC treatment in PV and PFTimepoint: a. Proportion of PV patients who achieve CR on minimal therapy- Up to 52 weeks treatment period b. Proportion of PV and PF patients who achieve CR on minimal therapy- Up to 52 weeks treatment period c. Time to DC- Upto 60 weeks d. Time to CR- Upto 60 weeks e. Time to CR on minimal therapy- Upto 60 weeks f. Time to CR off therapy- Upto 60 weeks g. Time to flare- Upto 60 weeks;To evaluate the efficacy of efgartigimod PH20 SC treatment in PV and PF (..continued)Timepoint: a. Rate of treatment failure- Upto 60 weeks. b. Rate of flare- Upto 60 weeks c. Cumulative prednisone dose over the trial- Upto 52 weeks treatment period d. Pemphigus Disease Area Index (PDAI) at each visit- Upto 52 weeks treatment period ;To evaluate the immunogenicity of efgartigimod PH20 SC in patients with PV or PFTimepoint: Anti-drug antibodies (ADAs) to efgartigimod (serum levels) and rHuPH20 (plasma levels)- Upto 60 weeks;To evaluate the PD of efgartigimod PH20 SC in patients with PV or PFTimepoint: a. Total IgG and subtype (IgG1, IgG2, IgG3, IgG4) serum levels- Upto 60 weeks b. Anti-Dsg-1 and -3 autoantibodies serum levels- Upto 60 weeks;To evaluate the pharmacokinetics (PK) of efgartigimod PH20 SC in patients with PV or PFTimepoint: Efgartigimod serum concentrations- Upto 60 weeks | — |
Countries
Australia, Bulgaria, France, Georgia, Germany, Greece, Hungary, India, Israel, Italy, Japan, Poland, Romania, Russian Federation, Spain, Turkey, Ukraine, United Kingdom, United States of America
Contacts
Public ContactRashmi Chitgupi
PPD Pharmaceutical Development India Private Limited
Outcome results
None listed