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    Summary
    EudraCT Number:2017-002333-40
    Sponsor's Protocol Code Number:ARGX-113-1701
    National Competent Authority:Germany - PEI
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2017-07-14
    Trial results View results
    Index
    A. PROTOCOL INFORMATION
    B. SPONSOR INFORMATION
    C. APPLICANT IDENTIFICATION
    D. IMP IDENTIFICATION
    D.8 INFORMATION ON PLACEBO
    E. GENERAL INFORMATION ON THE TRIAL
    F. POPULATION OF TRIAL SUBJECTS
    G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
    N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
    P. END OF TRIAL
    Expand All   Collapse All
    A. Protocol Information
    A.1Member State ConcernedGermany - PEI
    A.2EudraCT number2017-002333-40
    A.3Full title of the trial
    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 or Foliaceus)
    Offene, nicht kontrollierte Phase-II-Studie zur Bewertung der Sicherheit, Pharmakodynamik, Pharmakokinetik, Wirksamkeit und Anwendungsbedingungen von ARGX-113 bei Patienten mit leichtem bis mittelschwerem Pemphigus (vulgaris oder foliaceus).
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A study to evaluate the safety and efficacy of ARGX-113 in patients with Mild to Moderate Pemphigus Vulgaris or Foliaceus)
    A.4.1Sponsor's protocol code numberARGX-113-1701
    A.7Trial is part of a Paediatric Investigation Plan No
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of Sponsorargenx BVBA
    B.1.3.4CountryBelgium
    B.3.1 and B.3.2Status of the sponsorCommercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportargenx BVBA
    B.4.2CountryBelgium
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationargenx BVBA
    B.5.2Functional name of contact pointregulatory
    B.5.3 Address:
    B.5.3.1Street AddressIndustriepark Zwijnaarde 7
    B.5.3.2Town/ cityZwijnaarde
    B.5.3.3Post codeB-9052
    B.5.3.4CountryBelgium
    B.5.4Telephone number+32 09310 3400
    B.5.5Fax number+32 09310 3499
    B.5.6E-mailregulatory@argenx.com
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameARGX-113
    D.3.2Product code ARGX-113
    D.3.4Pharmaceutical form Solution for infusion
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNefgartigimod
    D.3.9.1CAS number 1821402-21-4
    D.3.9.2Current sponsor codeARGX-113
    D.3.9.4EV Substance CodeSUB180001
    D.3.10 Strength
    D.3.10.1Concentration unit mg/kg milligram(s)/kilogram
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number25
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin No
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) Yes
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product Yes
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.8 Information on Placebo
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Pemphigus (Vulgaris or Foliaceus)
    E.1.1.1Medical condition in easily understood language
    Pemphigus (Vulgaris or Foliaceus)
    E.1.1.2Therapeutic area Diseases [C] - Skin and Connective Tissue Diseases [C17]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10052802
    E.1.2Term Pemphigus vulgaris
    E.1.2System Organ Class 100000004858
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10057069
    E.1.2Term Pemphigus foliaceus
    E.1.2System Organ Class 100000004858
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To evaluate the safety and tolerability of ARGX-113 in PV and PF patients
    E.2.2Secondary objectives of the trial
    - 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.
    E.2.3Trial contains a sub-study No
    E.3Principal 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 positive indirect immunofluorescence and/or enzyme-linked immunosorbent assay (ELISA).
    3. Mild to moderate disease severity (Pemphigus Disease Area Index [PDAI] < 45).
    4.Newly diagnosed patients or relapsing patients off therapy; 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 antigen 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).
    E.4Principal 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:
    a. Aspartate aminotransferase (AST) or alanine aminotransferase (ALT) > 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 < 50 mL/min (using the Chronic Kidney Disease Epidemiology -Creatinine formula)
    d. Hemoglobin (Hb) ≤ 9 g/dL
    e. International normalized ratio (INR) > 1.5 or activated partial thromboplastin time (aPTT) > 1.5 x ULN
    f. Total immunoglobulin G (IgG) level < 6 g/L
    g. Presence of > 1 + proteinuria dipstick
    17. Patient having participated in another interventional study within the last 3 months prior to Baseline visit.
    E.5 End points
    E.5.1Primary 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.
    E.5.1.1Timepoint(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, at V1-EOT

    -ECG: screening, v5, v7 to EOT
    E.5.2Secondary 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.
    -In additional cohort 4 and optional cohort 5, time to end of
    consolidation, defined as the time at which no new lesions have
    developed for a minimum of 2 weeks, and approximately 80% of lesions
    have healed.
    -In additional cohort 4 and optional cohort 5, time to complete clinical
    remission, as defined by the absence of new lesions and established
    lesions completely healed (except post-inflammatory hyperpigmentation
    or erythema from resolving lesions).
    -In additional cohort 4 and optional cohort 5, time to complete clinical
    remission under minimal therapy, as defined as a prednisone dose of 10
    mg/day or less for at least 8 weeks.
    -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 each visit as
    per schedule of assessments.
    E.5.2.1Timepoint(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
    -time to end of consolidation
    -time to complete clinical remission,, defined as the time at which no
    new lesions have developed for a minimum of 2 weeks
    -time to complete clinical remission , as defined by the absence of new
    lesions and established lesions completely healed
    -time to complete clinical remission under minimal therapy, as defined
    as a prednisone dose of 10 mg/day or less for at least 8 weeks
    - at Baseline then at each Visit
    - at each visit as per schedule of assessments
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis No
    E.6.3Therapy Yes
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic Yes
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others No
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) No
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E.7.1.3.1Other trial type description
    E.7.2Therapeutic exploratory (Phase II) Yes
    E.7.3Therapeutic confirmatory (Phase III) No
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled No
    E.8.1.1Randomised No
    E.8.1.2Open Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group No
    E.8.1.6Cross over No
    E.8.1.7Other No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo No
    E.8.2.3Other No
    E.8.3 The trial involves single site in the Member State concerned No
    E.8.4 The trial involves multiple sites in the Member State concerned Yes
    E.8.4.1Number of sites anticipated in Member State concerned4
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA8
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA Yes
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    Germany
    Hungary
    Israel
    Italy
    Romania
    Ukraine
    E.8.7Trial has a data monitoring committee No
    E.8.8 Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years3
    E.8.9.1In the Member State concerned months3
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years3
    E.8.9.2In all countries concerned by the trial months3
    E.8.9.2In all countries concerned by the trial days0
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 No
    F.1.1.1In Utero No
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.3Newborns (0-27 days) No
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.5Children (2-11years) No
    F.1.1.6Adolescents (12-17 years) No
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 10
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 2
    F.2 Gender
    F.2.1Female Yes
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers No
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations Yes
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception Yes
    F.3.3.3Pregnant women No
    F.3.3.4Nursing women No
    F.3.3.5Emergency situation No
    F.3.3.6Subjects incapable of giving consent personally No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state5
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 18
    F.4.2.2In the whole clinical trial 22
    F.5 Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)
    Patients will return to standard of care treatment.
    G. Investigator Networks to be involved in the Trial
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2017-11-09
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2018-01-31
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2020-10-28
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