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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register.
Register: ClinicalTrials.gov
Last refreshed on: 11 January 2021
Main ID:  NCT03334058
Date of registration: 23/10/2017
Prospective Registration: Yes
Primary sponsor: argenx
Public title: A Study to Evaluate the Safety, PD, PK and Efficacy of ARGX-113 in Patients With Pemphigus
Scientific title: 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)
Date of first enrolment: October 18, 2017
Target sample size: 34
Recruitment status: Completed
URL:  https://clinicaltrials.gov/show/NCT03334058
Study type:  Interventional
Study design:  Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).  
Phase:  Phase 2
Countries of recruitment
Germany Hungary Israel Italy Ukraine
Contacts
Name:     Patrick Dupuy, MD
Address: 
Telephone:
Email:
Affiliation:  argenx
Key inclusion & exclusion criteria

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.



Age minimum: 18 Years
Age maximum: N/A
Gender: All
Health Condition(s) or Problem(s) studied
Pemphigus Foliaceus
Pemphigus Vulgaris
Intervention(s)
Drug: ARGX-113
Primary Outcome(s)
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]
Secondary Outcome(s)
Evaluation of serum levels of anti Dsg-1 and -3 autoantibodies [Time Frame: Up to 6 months]
Evaluation of serum levels of total IgG and subtypes (IgG1, IgG2, IgG3, IgG4) [Time Frame: Up to 6 months]
Incidence of anti-drug antibodies (ADA) to ARGX 113 [Time Frame: Up to 6 months]
Pemphigus Disease Area Index (PDAI) [Time Frame: Up to 6 months]
Pharmacokinetic parameters of ARGX 113: Cmax [Time Frame: Up to 6 months]
Pharmacokinetic parameters of ARGX 113: Tmax [Time Frame: Up to 6 months]
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]
Secondary ID(s)
2017-002333-40
ARGX-113-1701
Source(s) of Monetary Support
Please refer to primary and secondary sponsors
Secondary Sponsor(s)
Ethics review
Results
Results available:
Date Posted:
Date Completed:
URL:
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