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    EudraCT Number:2019-003107-35
    Sponsor's Protocol Code Number:ARGX-113-1902
    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:2021-06-17
    Trial results
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    A. Protocol Information
    A.1Member State ConcernedItaly - Italian Medicines Agency
    A.2EudraCT number2019-003107-35
    A.3Full title of the trial
    Open-label Extension of the ARGX-113-1802 Trial to Investigate the Long-term Safety, Tolerability, and Efficacy of Efgartigimod PH20 SC in Patients with Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)
    Estensione in aperto della sperimentazione ARGX-113-1802 per valutare la sicurezza, la tollerabilità e l’efficacia a lungo termine di efgartigimod PH20 SC in pazienti affetti da polineuropatia demielinizzante infiammatoria cronica (CIDP)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A study to assess the long-term safety and efficacy of a subcutaneous formulation of efgartigimod in adults with chronic inflammatory demyelinating polyneuropathy (an autoimmune disorder that affects the peripheral nerves)
    Studio per valutare la sicurezza e l’efficacia a lungo termine di una formulazione sottocutanea di efgartigimod in adulti con polineuropatia demielinizzante infiammatoria cronica (disturbo autoimmune che colpisce i nervi periferici)
    A.3.2Name or abbreviated title of the trial where available
    A.4.1Sponsor's protocol code numberARGX-113-1902
    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.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.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 code9052
    B.5.4Telephone number003293103400
    B.5.5Fax number000000
    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/ml milligram(s)/millilitre
    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. cell therapy medicinal product Information not present in EudraCT
    D. therapy medical product Information not present in EudraCT
    D. Engineered Product Information not present in EudraCT
    D. ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D. 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.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
    Chronic Inflammatory Demyelinating Polyneuropathy
    Polineuropatia demielinizzante infiammatoria cronica
    E.1.1.1Medical condition in easily understood language
    Inflammation of peripheral nervous system
    Infiammazione del sistema nervoso periferico
    E.1.1.2Therapeutic area Diseases [C] - Nervous System Diseases [C10]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10077384
    E.1.2Term Chronic inflammatory demyelinating polyneuropathy
    E.1.2System Organ Class 100000004852
    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 long-term safety and tolerability of efgartigimod PH20 SC (efgartigimod co-formulated with recombinant human hyaluronidase PH20 [rHuPH20] for subcutaneous [SC] administration)
    Valutare la sicurezza e la tollerabilità a lungo termine di efgartigimod PH20 SC (efgartigimod co-formulato con ialuronidasi umana ricombinante PH20 [rHuPH20] per somministrazione sottocutanea [SC])
    E.2.2Secondary objectives of the trial
    - To determine the long-term efficacy
    - To evaluate the immunogenicity (anti-drug antibodies [ADA]) of efgartigimod and rHuPH20
    - To evaluate the pharmacokinetics (PK) of efgartigimod PH20 SC
    - To evaluate the pharmacodynamic (PD) effect of efgartigimod PH20 SC (ie, immunoglobulin G [IgG] levels)
    - To evaluate additional patient-reported outcomes (PROs) (including patient-reported quality of life and satisfaction with treatment)
    - To explore self-administration of the treatment
    - Determinare l’efficacia a lungo termine
    - Valutare l’immunogenicità (anticorpi anti-farmaco [ADA]) di efgartigimod e rHuPH20
    - Valutare la farmacocinetica (PK) di efgartigimod PH20 SC
    - Valutare l’effetto farmacodinamico (PD) di efgartigimod PH20 SC (ovvero i livelli di immunoglobulina G [IgG])
    - Valutare gli ulteriori esiti riferiti dal paziente (PRO) (compresa la qualità della vita riferita dal paziente e la soddisfazione per il trattamento)
    - Esaminare l’autosomministrazione del trattamento
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Ability to understand the requirements of the trial, 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) of this trial.

    2. Male or female patient with one of the following options:
    - Have completed the Week-48 visit of Stage B of the ARGX-113-1802 trial and are considered to be eligible for treatment with efgartigimod PH20 SC; or
    - Have deteriorated during Stage B of the ARGX-113-1802 trial and are considered to be eligible for treatment with efgartigimod PH20 SC, or
    - Have been offered the participation in the OLE trial due to early termination of the ARGX-113-1802 trial (because sufficient events for the primary endpoint analysis of the that trial have been reached and it is stopped) and are considered to be eligible for treatment with efgartigimod PH20 SC treatment; or
    - Have completed the Week-48 visit of the previous cycle of the OLE trial and are considered to be eligible to continue with efgartigimod PH20 SC treatment.

    3. Women of childbearing potential who have a negative urine pregnancy test at baseline before IMP administration.

    4. Women of childbearing potential must use a highly effective method of contraception (failure rate of less than 1% per year) from baseline to 90 days after the last administration of IMP.

    5. Non-sterilized male patients who are sexually active with a female partner of childbearing potential must use a condom and his partner must use a highly effective method of contraception (failure rate of less than 1% per year) from baseline to 90 days after the last administration of IMP. Male patients practicing true sexual abstinence (when this is in line with the preferred and usual life style of the participant) can be included. Sterilized male patients who have had vasectomy with documented aspermia post-procedure can be included. In addition, male patients are not allowed to donate sperm from baseline to 90 days after the last administration of IMP.
    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 (comprese le visite obbligatorie dello studio) della presente sperimentazione.

    2. Paziente di sesso maschile o femminile con una delle seguenti opzioni:
    - Avere completato la visita della Settimana 48 della Parte B della sperimentazione ARGX-113-1802 ed essere ritenuto idoneo per il trattamento con efgartigimod PH20 SC; oppure
    - Essere peggiorato durante la Parte B della sperimentazione ARGX-113-1802 ed essere ritenuto idoneo per il trattamento con efgartigimod PH20 SC; oppure
    - Avere la possibilità di partecipare alla sperimentazione OLE a causa dell’interruzione anticipata della sperimentazione ARGX-113-1802 (perché è stato raggiunto un numero sufficiente di eventi per l’analisi dell’endpoint primario della sperimentazione e quest’ultima è stata interrotta) ed essere ritenuto idoneo per il trattamento con efgartigimod PH20 SC; oppure
    - Avere completato la visita della Settimana 48 del ciclo precedente della sperimentazione OLE ed essere ritenuto idoneo a proseguire il trattamento con efgartigimod PH20 SC.

    3. Le donne in età fertile che presentano un test di gravidanza sulle urine negativo al basale prima della somministrazione di IMP.

    4. Le donne in età fertile dovranno utilizzare un metodo contraccettivo altamente efficace (tasso di fallimento inferiore all’1% annuo) dal basale fino a 90 giorni dopo l’ultima somministrazione di IMP.

    5. I pazienti di sesso maschile non sterilizzati e sessualmente attivi con una partner di sesso femminile in età fertile dovranno usare un profilattico e la partner dovrà utilizzare un metodo contraccettivo altamente efficace (tasso di fallimento inferiore all’1% annuo) dal basale fino a 90 giorni dopo l’ultima somministrazione di IMP. Potranno essere inclusi i pazienti di sesso maschile che pratichino realmente l’astinenza sessuale (quando ciò sia in linea con lo stile di vita preferito e abituale del partecipante). Potranno essere inclusi i pazienti di sesso maschile sterilizzati sottoposti a vasectomia con aspermia post-procedurale documentata. Inoltre, ai pazienti di sesso maschile non sarà consentito donare il seme a partire dal basale e per 90 giorni dopo l’ultima somministrazione di IMP.
    E.4Principal exclusion criteria
    1. Week-48/ED visit in the ARGX-113-1802 trial or the Week-48 visit of the previous OLE participation occurred more than 14 days prior to SD1 of the OLE trial or the start of a new treatment cycle in the OLE trial and more than 21 days since the last dose of IMP.

    2. Pregnant and lactating women and those intending to become pregnant during the trial or within 90 days after last IMP administration.

    3. Patients with clinical evidence of other significant serious disease or patients who underwent a recent or have a planned major surgery, or any other reason which could confound the results of the trial or put the patient at undue risk.
    1. La visita della Settimana 48/di interruzione anticipata (ED) della sperimentazione ARGX-113-1802 o la visita della Settimana 48 della partecipazione OLE precedente, si è verificata più di 14 giorni prima dell’SD1 nella sperimentazione OLE o dell’inizio di un nuovo ciclo di trattamento nella sperimentazione OLE e più di 21 giorni dall’ultima dose di IMP.

    2. Le donne incinte e in fase di allattamento e coloro che intendono rimanere incinte durante la sperimentazione o entro i 90 giorni successivi all’ultima somministrazione di IMP.

    3. I pazienti con evidenza clinica di altra malattia seria o i pazienti sottoposti di recente, o candidati, a intervento di chirurgia maggiore o qualsiasi altro motivo che potrebbe confondere i risultati della sperimentazione o comporterebbe un rischio eccessivo per il paziente.
    E.5 End points
    E.5.1Primary end point(s)
    - Incidence of TEAEs and SAEs by system organ class (SOC) and preferred term (PT).
    - Incidence of clinically significant laboratory abnormalities.
    - L’incidenza di TEAE e SAE mediante la classificazione per sistemi e organi (SOC) e termine preferito (PT).
    - L’incidenza di anomalie di laboratorio clinicamente significative.
    E.5.1.1Timepoint(s) of evaluation of this end point
    TEAEs and SAEs are monitored throughout the entire study duration; Blood sampling for laboratory analysis is taken at every study visit
    TEAE e SAE sono monitorati per tutta la durata dello studio; il prelievo di campioni di sangue per l'analisi di laboratorio viene effettuato in occasione di ogni visita dello studio
    E.5.2Secondary end point(s)

    Change from baseline over time in the following scores and measurements:
    - Adjusted INCAT score;
    - MRC Sum score;
    - 24-item I-RODS disability scores;
    - Mean grip strength assessed by Martin vigorimeter;
    - TUG score.
    Percentage of patients without clinical deterioration over time, defined by adjusted INCAT increase =1 point compared to baseline.

    - Percentage of patients with and titers of binding antibodies (BAb) towards efgartigimod and/or rHuPH20; and the presence of neutralizing antibodies (NAb) against efgartigimod and titers of NAb against rHuPH20.

    - Efgartigimod serum concentrations over time during the trial.

    - Changes from baseline over time of serum IgG levels (total and IgG subtypes)

    Additional Patient-reported Outcome;
    Change from baseline over time in:
    - Health-related quality-of-life questionnaire (EQ-5D-5L);
    - Brief Pain Inventory – Short Form (BPI-SF);
    - 9-item Treatment Satisfaction Questionnaire for Medication (TSQM-9);
    - Rasch-transformed-Fatigue Severity Scale (RT-FSS);
    - Hospital Anxiety and Depression Scale (HADS).

    Variazione rispetto al basale nel tempo dei seguenti punteggi e misurazioni:
    - Punteggio INCAT rettificato;
    - Punteggio totale MRC;
    - Punteggi di invalidità alla scala I-RODS a 24 voci;
    - Forza media di presa valutata mediante vigorimetro di Martin;
    - Punteggio TUG.
    Percentuale di pazienti senza deterioramento clinico nel tempo, definito da un aumento del punteggio INCAT rettificato =1 punto rispetto al basale.

    - Percentuale di pazienti con titoli di anticorpi leganti (BAb) verso efgartigimod e/o rHuPH20; presenza di anticorpi neutralizzanti (NAb) contro efgartigimod e titoli di NAb contro rHuPH20.

    - Concentrazioni sieriche di efgartigimod nel tempo nel corso della sperimentazione.

    - Variazioni dal basale nel tempo dei livelli sierici di IgG (totale e sottotipi IgG)

    Ulteriori esiti riferiti dal paziente;
    Variazione dal basale nel tempo di:
    - Questionario per misurare la qualità della vita correlata alla salute (EQ-5D-5L);
    - Forma abbreviata dell’Inventario breve sul dolore (Brief Pain Inventory - Short Form, BPI-SF);
    - Questionario a 9 voci sulla soddisfazione del trattamento con i farmaci (Treatment Satisfaction Questionnaire for Medication, TSQM-9);
    - Scala di gravità dell’affaticamento trasformata Rasch (Rasch-transformed Fatigue Severity Scale, RT-FSS);
    - Scala della depressione e dell’ansia da ospedale (HADS).
    E.5.2.1Timepoint(s) of evaluation of this end point
    Efficacy, immunogenicity, pharmacokinetic and pharmacodynamic endpoints are assessed at every study visit.
    Patient report outcomes are assessed at every study visit with the exception of visit 2 (week 4)
    Gli endpoint di efficacia, immunogenicità, farmacodinamica e farmacocinetica sono valutati a ogni visita dello studio.
    Gli esiti riferiti dal/dalla paziente vengono valutati a ogni visita dello studio, fatta eccezione per la visita 2 (Settimana 4)
    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 No
    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
    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. 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 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 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.8.2.4Number 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 concerned9
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA61
    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
    Russian Federation
    United States
    United Kingdom
    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
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years4
    E.8.9.1In the Member State concerned months0
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years4
    E.8.9.2In all countries concerned by the trial months0
    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: 216
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 144
    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 state15
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 155
    F.4.2.2In the whole clinical trial 360
    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)
    After the end of the trial, patients will no longer receive IMP and will be treated as considered appropriate by the investigator.
    Dopo la fine della sperimentazione, i pazienti non riceveranno più IMP e saranno trattati come ritenuto opportuno dallo sperimentatore.
    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 Decision2020-06-01
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2020-04-15
    P. End of Trial
    P.End of Trial StatusOngoing
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