Try the modernized ClinicalTrials.gov beta website. Learn more about the modernization effort.
Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu
Trial record 1 of 3 for:    argenx | Pemphigus Vulgaris
Previous Study | Return to List | Next Study

A Study to Evaluate the Safety, PD, PK and Efficacy of ARGX-113 in Patients With Pemphigus

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03334058
Recruitment Status : Completed
First Posted : November 7, 2017
Last Update Posted : December 14, 2020
Sponsor:
Information provided by (Responsible Party):
argenx

Tracking Information
First Submitted Date  ICMJE October 23, 2017
First Posted Date  ICMJE November 7, 2017
Last Update Posted Date December 14, 2020
Actual Study Start Date  ICMJE October 18, 2017
Actual Primary Completion Date October 28, 2020   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: March 1, 2019)
Safety and tolerability as measured by the incidence and severity of treatment-emergent (serious) adverse events over the study. [ Time Frame: Up to 6 months ]
Original Primary Outcome Measures  ICMJE
 (submitted: November 6, 2017)
To evaluate the safety and tolerability of ARGX 113 in patients with Pemphigus Vulgaris (PV) [ Time Frame: Up to 17 weeks ]
Incidence and severity of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs)
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: August 28, 2019)
  • Evaluation of serum levels of total IgG and subtypes (IgG1, IgG2, IgG3, IgG4) [ Time Frame: Up to 6 months ]
  • Evaluation of serum levels of anti Dsg-1 and -3 autoantibodies [ Time Frame: Up to 6 months ]
  • Pemphigus Disease Area Index (PDAI) [ Time Frame: Up to 6 months ]
    The score has a range from 0 to 263, the higher the score, the more severe the disease.
  • Time to disease control (DC), control being defined as the absence of new lesions and established lesions beginning to heal [ Time Frame: Up to 6 months ]
  • 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 [ Time Frame: Up to 6 months ]
  • Pharmacokinetic parameters of ARGX 113: Tmax [ Time Frame: Up to 6 months ]
  • Pharmacokinetic parameters of ARGX 113: Cmax [ Time Frame: Up to 6 months ]
  • Incidence of anti-drug antibodies (ADA) to ARGX 113 [ Time Frame: Up to 6 months ]
Original Secondary Outcome Measures  ICMJE Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE A Study to Evaluate the Safety, PD, PK and Efficacy of ARGX-113 in Patients With Pemphigus
Official Title  ICMJE An Open-label, Non-controlled, Phase II Study to Evaluate the Safety, Pharmacodynamics, Pharmacokinetics, Efficacy and Conditions of Use of ARGX-113 in Patients With Mild to Moderate Pemphigus (Vulgaris and Foliaceus)
Brief Summary

The proposed study is an open-label, non-controlled, adaptive-design Phase II study to evaluate the safety, pharmacodynamics, pharmacokinetics, efficacy, and conditions of use (dosage, frequency of administration at maintenance) of ARGX-113 in patients with mild to moderate Pemphigus (Vulgaris or Foliaceus), either newly diagnosed or relapsing.

The total study duration for each patient is less than 6 months. It consists of a Screening period, an Induction, a maintenance treatment period followed by a treatment-free Follow-up (FU) period.

Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE
  • Pemphigus Vulgaris
  • Pemphigus Foliaceus
Intervention  ICMJE Drug: ARGX-113
human IgG1-derived Fc fragment that binds to human neonatal Fc receptor (FcRn)
Study Arms  ICMJE Experimental: ARGX-113
Intervention: Drug: ARGX-113
Publications * Goebeler M, Bata-Csörgő Z, De Simone C, Didona B, Remenyik E, Reznichenko N, Stoevesandt J, Ward ES, Parys W, de Haard H, Dupuy P, Verheesen P, Schmidt E, Joly P; ARGX-113-1701 Investigator Study Group. Treatment of pemphigus vulgaris and foliaceus with efgartigimod, a neonatal Fc receptor inhibitor: a phase II multicentre, open-label feasibility trial. Br J Dermatol. 2021 Oct 5. doi: 10.1111/bjd.20782. [Epub ahead of print]

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: June 30, 2020)
34
Original Estimated Enrollment  ICMJE
 (submitted: November 6, 2017)
12
Actual Study Completion Date  ICMJE October 28, 2020
Actual Primary Completion Date October 28, 2020   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  1. Male or female patients aged ≥ 18 years.
  2. Clinical diagnosis of PV or PF, that has been confirmed by positive direct immunofluorescence and by positive indirect immunofluorescence and/or ELISA.
  3. Mild to moderate disease severity (PDAI < 45).
  4. Newly diagnosed patients or relapsing patients off therapy with or without a first course of prednisone of maximum 4 weeks, and for whom an initial period of ARGX-113 monotherapy is judged clinically acceptable; or newly diagnosed patients or relapsing patients off therapy on a first course of oral prednisone at stable dose for at least 2 weeks and for whom ARGX-113 monotherapy is considered not clinically acceptable; or patients who relapse despite oral prednisone at tapered dose +/- a conventional immunosuppressant (e.g. azathioprine, mycophenolate mofetil).
  5. Identified serum levels of autoantibodies directed against Dsg 3 and/or Dsg-1 antigens at screening, using indirect immunofluorescence or ELISA.
  6. Ability to understand the requirements of the study, provide written informed consent (including consent for the use and disclosure of research-related health information), and comply with the study protocol procedures (including required study visits).

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 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 or known 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 < 60%; see Appendix 14.2).
  16. At Screening, have clinically significant laboratory abnormalities as below:

    1. Aspartate aminotransferase (AST) or alanine aminotransferase (ALT) > 3 × upper limit of normal (ULN)
    2. 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)
    3. Serum creatinine of > 1.5 mg/dL or creatinine clearance < 50 mL/min (using the Chronic Kidney Disease Epidemiology Creatinine formula)
    4. Hemoglobin (Hb) ≤ 9 g/dL
    5. International normalized ratio (INR) > 1.5 or activated partial thromboplastin time (aPTT) > 1.5 x ULN
    6. Total immunoglobulin G (IgG) level < 6 g/L
    7. Presence of > 1 + proteinuria dipstick
  17. Patient having participated in another interventional study within the last 3 months prior to Baseline visit.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Germany,   Hungary,   Israel,   Italy,   Ukraine
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03334058
Other Study ID Numbers  ICMJE ARGX-113-1701
2017-002333-40 ( EudraCT Number )
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE Not Provided
Responsible Party argenx
Study Sponsor  ICMJE argenx
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Patrick Dupuy, MD argenx
PRS Account argenx
Verification Date November 2020

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP