Pemphigus (Vulgaris or Foliaceus) MedDRA version: 20.0 Level: LLT Classification code 10052802 Term: Pemphigus vulgaris System Organ Class: 100000004858 MedDRA version: 20.0 Level: LLT Classification code 10057069 Term: Pemphigus foliaceus System Organ Class: 100000004858
Conditions
Sponsors
argenx BVBA
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Male or female patients aged = 18 years. 2. Clinical diagnosis of mucocutaneous PV or PF, that has been confirmed by positive direct immunofluorescence and positive indirect immunofluorescence and/or enzyme-linked immunosorbent assay (ELISA) 3. Mild to moderate disease severity (Pemphigus Disease Area Index [PDAI] =65 years) yes F.1.3.1 Number of subjects for this age range 2
Exclusion criteria
Exclusion criteria: 1. Pregnant and lactating women, and those intending to become pregnant during the study or within 90 days after the last dosing.Women of childbearing potential should have a negative serum pregnancy test at Screening and a negative urine pregnancy test at Baseline, prior to administration of IMP. 2. Male patients who are sexually active that do not intend to use effective methods of contraception during the study or within 90 days after the last dosing. 3. Confirmed diagnosis of pemphigus foliaceus, paraneoplastic pemphigus, drug-induced pemphigus or any other non-PV/non-PF autoimmune blistering disease. 4. History of refractory disease to active third line therapy (e.g. intravenous polyvalent human immunoglobulins [IVIg], rituximab, plasma exchange/ immunoadsorption). 5. Use of therapies other than oral prednisone and conventional immunosuppressants, that can interfere in the clinical course of the disease (e.g. intravenous [IV] prednisolone bolus, dapsone, sulfasalazine, tetracyclines, nicotinamide, plasmapheresis/ plasma exchange, immunoadsorption and IVIg) within 2 months prior to Baseline visit. 6. Use of rituximab and other CD20 target biologics within 6 months prior to Baseline visit. 7. History of anaphylactic reaction, or a known hypersensitivity reaction to one of the components of the IMP. 8. History of vaccination within the last 4 weeks prior to Baseline visit, or with a planned vaccination during the study, with the exception of seasonal vaccination (e.g. influenza vaccine). 9. Recent serious infection (i.e., requiring injectable antimicrobial therapy or hospitalization) within the 8 weeks prior to Baseline visit. 10. Known active or chronic viral infection with hepatitis B virus (HBV); refer to the Centers for Disease Control and Prevention (CDC) guidelines. 11. Known seropositive or active infection with hepatitis C virus (HCV). 12. Known history of known infection with viral infection with human immunodeficiency virus (HIV 1 and 2 antibodies). 13. Body Mass Index (BMI) at Screening > 35,0 kg/m2. 14. Clinical evidence of significant active, unstable or uncontrolled concomitant disease (e.g. cardiovascular, pulmonary, hematologic, gastrointestinal, endocrinological and metabolic, hepatic, renal, neurologic, malignancy, infectious diseases, coagulopathies, other autoimmune disease) or condition (lack of peripheral venous access, recent major surgery, etc), which, in the opinion of the investigator, puts the patient at undue risk or may affect the interpretation of the results. 15. Patients in general health condition not allowing study participation (Karnofsky index 3 × upper limit of normal (ULN) b. Total serum bilirubin of > 1.5 x ULN (except for Grade 1 hyperbilirubinemia as defined by the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI-CTCAE), due solely to a documented medical diagnosis of Gilbert’s syndrome) c. Serum creatinine of > 1.5 mg/dL or creatinine clearance 1.5 or activated partial thromboplastin time (aPTT) > 1.5 x ULN f. Total immunoglobulin G (IgG) level 1 + proteinuria dipstick 17. Patient having participated in another inte
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To evaluate the safety and tolerability of ARGX-113 in PV and PF patients ;Secondary Objective: - To define the therapeutic dose and the dose regimen through the pharmacodynamic (PD) and clinical efficacy findings; - To evaluate the serum levels of immunoglobulin (Ig) G (total IgG and subtypes) following ARGX-113 treatment; - To evaluate the serum levels of anti-desmoglein (Dsg)-1 and -3 autoantibodies following ARGX-113 treatment; - To assess the efficacy on cutaneous and mucosal pemphigus lesions; - To measure the pharmacokinetics (PK) of ARGX-113; - To investigate the immunogenicity of ARGX-113.;Primary end point(s): - Incidence and severity of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs) over the study. - Vital signs, electrocardiogram (ECG) parameters, physical examination abnormalities and clinical laboratory assessments.;Timepoint(s) of evaluation of this end point: -Treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs) will be monitored continuously from signing of informed consent until the last study-related activity -Vital signs, physical examination abnormalities and clinical laboratory assessments: screening, V1-9 -ECG: screening, v5, v7-9 | — |
Secondary
| Measure | Time frame |
|---|---|
| Timepoint(s) of evaluation of this end point: - at Baseline then at each visit - at Baseline then at each visit - at each visit - from Visit 2 until control is achieved - from any visit following DC - at Baseline then at each visit - at Baseline, Visit 3, then at Visits 5, all Maintenance treatment visits, FU-1, FU-2 and FU-3. ;Secondary end point(s): - Total IgG and subtypes, at Baseline then at each visit. -Serum levels of anti-Dsg-1 and -3 autoantibodies, at Baseline then at each visit. -PDAI, at each visit. -Time to disease control (DC), control being defined as the absence of new lesions and established lesions beginning to heal, evaluated from Visit 2 until control is achieved. -Time until relapse, relapse being defined as the appearance of 3 or more new lesions a month that do not heal spontaneously within 1 week, or as the extension of established lesions, evaluated from any visit following DC. -Pharmacokinetic parameters of ARGX-113, at Baseline then at each visit (pre- and post-dose when IMP is administered). -Incidence of anti-drug antibodies (ADA) to ARGX-113, at Baseline, Visit 3, then at Visits 5, all Maintenance treatment visits, FU-1, FU-2 and FU-3. | — |
Countries
Germany, Hungary, Israel, Italy, Romania, Ukraine
Contacts
Public Contactregulatory
argenx BVBA
Outcome results
None listed