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    Summary
    EudraCT Number:2020-002917-16
    Sponsor's Protocol Code Number:ARGX-113-1905
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2021-05-24
    Trial 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 ConcernedItaly - Italian Medicines Agency
    A.2EudraCT number2020-002917-16
    A.3Full title of the trial
    An Open-Label, Multicenter, Follow-up Trial of ARGX-113-1904 to Evaluate the Safety, Tolerability, and Efficacy of Efgartigimod PH20 SC in Patients with Pemphigus
    Studio di follow-up, in aperto, multicentrico su ARGX-113-1904 per valutare la sicurezza, la tollerabilità e l’efficacia di efgartigimod PH20 SC in pazienti affetti da pemfigo (ADDRESS+)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A study to assess the safety and efficacy of a subcutaneous formulation of efgartigimod PH20 SC in adults with Pemphigus (Vulgaris or Foliaceus)
    Studio per valutare la sicurezza e l’efficacia di una formulazione sottocutanea di efgartigimod PH20 SC negli adulti affetti da pemfigo (volgare o foliaceo)
    A.3.2Name or abbreviated title of the trial where available
    ADDRESS+
    ADDRESS+
    A.4.1Sponsor's protocol code numberARGX-113-1905
    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 BV
    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 BV
    B.4.2CountryBelgium
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationargenx BV
    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 number003293103400
    B.5.5Fax number000000
    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 nameEfgartigimod PH20 SC
    D.3.2Product code [na]
    D.3.4Pharmaceutical form Solution for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPSubcutaneous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNEFGARTIGIMOD ALFA
    D.3.9.1CAS number 1821402-21-4
    D.3.9.2Current sponsor codeARGX-113
    D.3.9.3Other descriptive nameARGX-113
    D.3.9.4EV Substance CodeSUB180001
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number165
    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 Information not present in EudraCT
    D.3.11.3.2Gene therapy medical product Information not present in EudraCT
    D.3.11.3.3Tissue Engineered Product Information not present in EudraCT
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product Information not present in EudraCT
    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.IMP: 2
    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 Yes
    D.2.1.1.1Trade name Prednison 5 mg GALEN
    D.2.1.1.2Name of the Marketing Authorisation holderGALENpharma GmbH
    D.2.1.2Country which granted the Marketing AuthorisationGermany
    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 namena
    D.3.2Product code [na]
    D.3.4Pharmaceutical form Tablet
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNPREDNISONE
    D.3.9.1CAS number 53-03-2
    D.3.9.2Current sponsor codena
    D.3.9.3Other descriptive namePREDNISONE
    D.3.9.4EV Substance CodeSUB10020MIG
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number5
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product Information not present in EudraCT
    D.3.11.3.2Gene therapy medical product Information not present in EudraCT
    D.3.11.3.3Tissue Engineered Product Information not present in EudraCT
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product Information not present in EudraCT
    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 No
    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.IMP: 3
    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 Yes
    D.2.1.1.1Trade name Prednison 10 mg GALEN
    D.2.1.1.2Name of the Marketing Authorisation holderGALENpharma GmbH
    D.2.1.2Country which granted the Marketing AuthorisationGermany
    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 namena
    D.3.2Product code [na]
    D.3.4Pharmaceutical form Tablet
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNPREDNISONE
    D.3.9.1CAS number 53-03-2
    D.3.9.2Current sponsor codena
    D.3.9.3Other descriptive namePREDNISONE
    D.3.9.4EV Substance CodeSUB10020MIG
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number10
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product Information not present in EudraCT
    D.3.11.3.2Gene therapy medical product Information not present in EudraCT
    D.3.11.3.3Tissue Engineered Product Information not present in EudraCT
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product Information not present in EudraCT
    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 No
    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.IMP: 4
    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 Yes
    D.2.1.1.1Trade name Prednison20 mg GALEN
    D.2.1.1.2Name of the Marketing Authorisation holderGALENpharma GmbH
    D.2.1.2Country which granted the Marketing AuthorisationGermany
    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 namena
    D.3.2Product code [na]
    D.3.4Pharmaceutical form Tablet
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNPREDNISONE
    D.3.9.1CAS number 53-03-2
    D.3.9.2Current sponsor codena
    D.3.9.3Other descriptive namePREDNISONE
    D.3.9.4EV Substance CodeSUB10020MIG
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number20
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product Information not present in EudraCT
    D.3.11.3.2Gene therapy medical product Information not present in EudraCT
    D.3.11.3.3Tissue Engineered Product Information not present in EudraCT
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product Information not present in EudraCT
    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 No
    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.IMP: 5
    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 Yes
    D.2.1.1.1Trade name Prednison 50 mg GALEN
    D.2.1.1.2Name of the Marketing Authorisation holderGALENpharma GmbH
    D.2.1.2Country which granted the Marketing AuthorisationGermany
    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 namena
    D.3.2Product code [na]
    D.3.4Pharmaceutical form Tablet
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNPREDNISONE
    D.3.9.1CAS number 53-03-2
    D.3.9.2Current sponsor codena
    D.3.9.3Other descriptive namePREDNISONE
    D.3.9.4EV Substance CodeSUB10020MIG
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number50
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product Information not present in EudraCT
    D.3.11.3.2Gene therapy medical product Information not present in EudraCT
    D.3.11.3.3Tissue Engineered Product Information not present in EudraCT
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product Information not present in EudraCT
    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 No
    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 Pemphigus Foliaceus
    Pemfigo volgare o pemfigo foliaceo
    E.1.1.1Medical condition in easily understood language
    Blistering autoimmune diseases that affect the skin and mucous membranes
    Malattie autoimmuni con formazione di vesciche che interessano la pelle e le membrane mucose
    E.1.1.2Therapeutic area Diseases [C] - Immune System Diseases [C20]
    MedDRA Classification
    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.1.2 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.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To assess the safety of extended treatment and re-treatment with efgartigimod PH20 SC in patients with PV or PF
    Valutare la sicurezza di un trattamento prolungato e del ri-trattamento con efgartigimod PH20 SC in pazienti affetti da PV o PF
    E.2.2Secondary objectives of the trial
    To evaluate the efficacy of efgartigimod PH20 SC treatment in PV and PF
    To evaluate the effects of efgartigimod PH20 SC on quality of life (QoL) in patients with PV or PF
    To evaluate the pharmacokinetics (PK) of efgartigimod PH20 SC in patients with PV or PF
    To evaluate the PD of efgartigimod PH20 SC in patients with PV or PF
    To evaluate the immunogenicity of efgartigimod PH20 SC in patients with PV or PF
    • Valutare l’efficacia del trattamento con efgartigimod PH20 SC in PV e PF
    • Valutare gli effetti di efgartigimod PH20 SC sulla qualità della vita (QoL) nei pazienti affetti da PV o PF
    • Valutare la PK di efgartigimod PH20 SC nei pazienti affetti da PV o PF
    • Valutare la PD di efgartigimod PH20 SC nei pazienti affetti da PV o PF
    • Valutare l’immunogenicità di efgartigimod PH20 SC nei pazienti affetti da PV o PF
    E.2.3Trial contains a sub-study Yes
    E.2.3.1Full title, date and version of each sub-study and their related objectives

    Other types of substudies
    Specify title, date and version of each substudy with relative objectives: Evaluation of biomarkers of pemphigus pathology from skin biopsis

    Altre tipologie di sottostudi
    specificare il titolo, la data e la versione di ogni sottostudio con i relativi obiettivi: Valutazione dei biomarcatori della patologia del pemfigo tramite biopsie cutanee
    E.3Principal inclusion criteria
    Participants must meet all of the following 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.
    I partecipanti devono soddisfare i seguenti criteri di inclusione:
    1. Capacità di comprendere i requisiti della sperimentazione, fornire un consenso informato scritto (compreso il consenso per l’utilizzo e la divulgazione di informazioni sanitarie correlate alla ricerca), volontà e capacità di attenersi alle procedure del protocollo di sperimentazione (comprese le visite previste della sperimentazione).
    2. Partecipazione del/la paziente alla sperimentazione ARGX-113-1904 e aver completato lo studio o possedere i criteri prestabiliti per il rollover.
    3. Donne in età fertile:
    a. Devono presentare un test di gravidanza sulle urine negativo alla visita basale prima che il farmaco sperimentale possa essere somministrato.
    b. Devono essere in un regime stabile da almeno 1 mese con minimo 1 metodo contraccettivo altamente efficace (ossia, tasso di fallimento inferiore all’1% annuo) durante la sperimentazione e per 90 giorni dopo l’ultima somministrazione dell’IMP.
    4. I pazienti di sesso maschile non sterilizzati, che sono sessualmente attivi con una partner di sesso femminile in età fertile, devono usare un metodo contraccettivo efficace dalla prima somministrazione dell’IMP fino a 90 giorni dopo l’ultima somministrazione dell’IMP. Potranno essere inclusi i pazienti di sesso maschile che pratichino realmente l’astinenza sessuale (se ciò è in linea con lo stile di vita preferito e abituale del partecipante). Potranno essere inclusi i pazienti di sesso maschile sterilizzati sottoposti a vasectomia e con aspermia post-procedurale documentata. Inoltre, ai pazienti di sesso maschile non sarà consentito donare lo sperma a partire dalla prima somministrazione dell’IMP e per 90 giorni dopo l’ultima dose dell’IMP.
    E.4Principal exclusion criteria
    Participants are excluded from the trial if any of the following criteria apply:
    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.
    I partecipanti saranno esclusi dalla sperimentazione se rientreranno in uno qualsiasi dei seguenti criteri:
    1. Le donne incinte e in fase di allattamento e quelle che intendono rimanere incinte durante la sperimentazione o entro i 90 giorni successivi all’ultima somministrazione dell’IMP.
    2. I pazienti con evidenza clinica di altra malattia significativa grave o i pazienti sottoposti di recente, o candidati, a intervento di chirurgia maggiore durante il periodo della sperimentazione o qualsiasi altra condizione che a giudizio dello sperimentatore potrebbe confondere i risultati della sperimentazione o comporterebbe un rischio eccessivo per il paziente.
    3. Ipersensibilità nota a uno qualsiasi dei componenti dei trattamenti somministrati.
    E.5 End points
    E.5.1Primary end point(s)
    1. 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)
    2. Vital signs, physical examination, electrocardiogram (ECG), and clinical laboratory safety evaluations
    • Incidenza e gravità di eventi avversi emergenti dal trattamento (TEAE), di eventi avversi di interesse particolare (AESI) e di eventi avversi seri (SAE) in base alla classificazione per sistemi e organi (SOC) e termine preferito (PT)
    • Misurazioni dei parametri vitali, esami obiettivi, elettrocardiogrammi (ECG) e valutazioni cliniche di laboratorio per la sicurezza
    E.5.1.1Timepoint(s) of evaluation of this end point
    Up to 60 weeks
    Fino a 60 settimane
    E.5.2Secondary end point(s)
    1. Proportion of PV patients who achieve CR on minimal prednisone therapy
    2. Proportion of PV and PF patients who achieve CR on minimal prednisone therapy in patients with PV and PF 3.
    3. Time to DC
    4. Time to CR
    5. Time to CR on minimal prednisone therapy
    6. Time to CR off therapy
    7. Time to flare
    8. Rate of treatment failure
    9. Rate of flare
    10. Cumulative prednisone dose over the trial
    11. Pemphigus Disease Area Index (PDAI) at each visit
    12. EuroQol 5-Dimension 5-Level (EQ-5D-5L) score
    13. Autoimmune Bullous Disease Quality of Life (ABQOL) score
    14. Efgartigimod serum concentrations
    15. Total IgG and subtype (IgG1, IgG2, IgG3, IgG4) serum levels
    16. Anti-Dsg-1 and -3 autoantibodies serum levels
    17. Anti-drug antibodies (ADAs) to efgartigimod (serum levels) and rHuPH20 (plasma levels)


    • Percentuale di pazienti che ottengono una CR con terapia minima trai i pazienti affetti da PV
    • Percentuale di pazienti che ottengono una CR con terapia minima tra i pazienti affetti da PV e PF
    • Tempo al DC
    • Tempo alla CR
    • Tempo alla CR con terapia minima con prednisone
    • Tempo alla CR senza terapia
    • Tempo alla riacutizzazione
    • Tasso di fallimento del trattamento
    • Tasso di riacutizzazione
    • Dose cumulativa di prednisone nell’arco della sperimentazione
    • Indice dell’area della malattia di pemfigo (PDAI) a ogni visita
    • Scala EuroQol a 5 dimensioni e 5 livelli (EQ-5D-5L)
    • Punteggio del Questionario sulla qualità della vita con malattia autoimmune bollosa (ABQOL)
    • Concentrazioni sieriche di efgartigimod
    • Livelli sierici di IgG totale e sottotipo (IgG1, IgG2, IgG3, IgG4)
    • Livelli sierici di autoanticorpi anti-Dsg-1 e -3
    • Anticorpi anti-farmaco (ADA) per efgartigimod (livelli sierici) e rHuPH20 (livelli plasmatici)
    E.5.2.1Timepoint(s) of evaluation of this end point
    1+2: up to 52 weeks treatment period
    3,4,5,6,7,8,9: up to 60 weeks
    10,11,12,13: up to 52 weeks treatment period
    14,15,16, 17: up to 60 weeks
    1+2: periodo di trattamento fino a 52 settimane
    3, 4, 5, 6, 7, 8, 9: fino a 60 settimane
    10, 11, 12, 13: periodo di trattamento fino a 52 settimane
    14, 15, 16, 17: fino a 60 settimane
    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 No
    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 Yes
    E.6.13.1Other scope of the trial description
    Immunogenicity
    immunogenicità
    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) No
    E.7.3Therapeutic confirmatory (Phase III) Yes
    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 No
    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 Yes
    E.8.1.7.1Other trial design description
    in aperto
    open
    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.8.2.4Number of treatment arms in the trial1
    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 concerned5
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA51
    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 Information not present in EudraCT
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    Australia
    Bulgaria
    France
    Georgia
    Germany
    Greece
    Hungary
    India
    Israel
    Italy
    Japan
    Poland
    Romania
    Russian Federation
    Spain
    Turkey
    Ukraine
    United Kingdom
    United States
    E.8.7Trial has a data monitoring committee Yes
    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
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years2
    E.8.9.1In the Member State concerned months9
    E.8.9.1In the Member State concerned days25
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months9
    E.8.9.2In all countries concerned by the trial days25
    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: 105
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 45
    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 state20
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 100
    F.4.2.2In the whole clinical trial 150
    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)
    Under certain conditions described in the protocol, the patients may be eligible to roll over to the open-label extension trial ARGX-113-1905. For patients, randomized to the efgartigimod PH20 SC or placebo treatment arm in ARGX-113-1904, trial ARGX-113-1905 provides extended or first treatment and re-treatment options in the respective treatment arms. argenx is currently assessing the appropriateness and possibility of making efgartigimod PH20 SC available for trial participants post-trial.
    In det condizioni descritte nel protocollo, i paz potrebbero essere idonei a passare allo studio di estensione in aperto ARGX-113-1905. Per i paz, randomizzati a efgartigimod PH20 SC o con placebo nello studio ARGX-113-1904, la speri. ARGX-113-1905 fornisce opzioni di trattamento prolungato o primo trattamento e ri-trattamento nei rispettivi bracci. Argenx sta valutando l'appropriatezza e la possibilità di rendere efgartigimod PH20 SC disponibile ai partecipanti alla speri. dopo la stessa.
    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 Decision2020-12-17
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2020-10-06
    P. End of Trial
    P.End of Trial StatusOngoing
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