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    Summary
    EudraCT Number:2021-001277-23
    Sponsor's Protocol Code Number:ARGX-113-2007
    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:2022-10-20
    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 number2021-001277-23
    A.3Full title of the trial
    A Phase 2/3, Randomized, Double-Blinded, Placebo-Controlled, Parallel-Group, 2-Arm, Multicenter, Operationally Seamless Study to Evaluate the Efficacy, Safety, Tolerability, Pharmacodynamics, Pharmacokinetics, and Immunogenicity of Efgartigimod PH20 SC in Participants Aged 18 Years and Older With Active Idiopathic Inflammatory Myopathy
    Studio di fase 2/3, randomizzato, in doppio cieco, controllato con placebo, a gruppi paralleli, a 2 bracci, multicentrico, operativamente senza interruzioni, per valutare l’efficacia, la sicurezza, la tollerabilità, la farmacodinamica, la farmacocinetica e l’immunogenicità di efgartigimod PH20 SC in partecipanti di età pari o superiore ai 18 anni affetti da miopatia infiammatoria idiopatica attiva
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A study to investigate the efficacy and safety of efgartigimod PH20 SC in adult participants with active idiopathic inflammatory myopathy
    Studio per valutare l’efficacia e la sicurezza di efgartigimod PH20 SC in partecipanti adulti affetti da miopatia infiammatoria idiopatica attiva
    A.3.2Name or abbreviated title of the trial where available
    alkivia
    alkivia
    A.4.1Sponsor's protocol code numberARGX-113-2007
    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.5.6E-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 [ARGX-113]
    D.3.4Pharmaceutical form Solution for injection/infusion
    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.3.9.4EV Substance CodeSUB198780
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number180
    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.8 Information on Placebo
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboSolution for injection/infusion
    D.8.4Route of administration of the placeboSubcutaneous use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Active Idiopathic Inflammatory Myopathy (IIM)
    Miopatia infiammatoria idiopatica (MII) attiva
    E.1.1.1Medical condition in easily understood language
    Idiopathic Inflammatory Myopathy (IIM), also referred to as myositis, is an inflammatory disorder of the skeletal muscle.
    La miopatia infiammatoria idiopatica (MII), detta anche miosite, è un disturbo infiammatorio del muscolo scheletrico
    E.1.1.2Therapeutic area Diseases [C] - Musculoskeletal Diseases [C05]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 24.1
    E.1.2Level PT
    E.1.2Classification code 10085970
    E.1.2Term Idiopathic inflammatory myopathy
    E.1.2System Organ Class 10028395 - Musculoskeletal and connective tissue disorders
    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 clinical improvement of efgartigimod PH20 SC treatment compared with placebo, in addition to standard of care immunomodulatory therapy
    Valutare il miglioramento clinico del trattamento con efgartigimod PH20 SC rispetto al placebo, oltre alla terapia immunomodulante standard di cura
    E.2.2Secondary objectives of the trial
    IMP = efgartigimod PH20 SC
    • Evaluate additional measures of efficacy of IMP in achieving clinical response
    • Evaluate effect of IMP on:
    - muscle strength
    - patient and physician global assessments of disease activity
    - physical function parameters
    • Evaluate steroid-sparing effect of IMP (phase 3 stage only)
    • Evaluate effect of IMP on:
    - extramuscular disease activity
    - additional measures of muscle strength
    - additional measures of patient and physician global assessments of disease activity
    - muscle endurance
    - pain
    - fatigue
    - additional physical functioning measures
    • Assess health impact of glucocorticoid use and evaluate steroid-sparing effect of IMP (phase 3 stage only)
    • Evaluate effect of IMP on:
    - health-related quality of life
    - muscle enzymes
    - preventing disease deterioration
    • Assess:
    - PK of IMP
    - PD effect on total IgG levels of IMP
    - immunogenicity of IMP and rHuPH20
    - safety and tolerability of IMP
    IMP = efgartigimod PH20 SC
    Valutare:
    •misure aggiuntive dell’efficacia dell’IMP in termini di ottenimento della risposta clinica
    •effetto dell’IMP su:forza muscolare;valutazioni globali del paziente e del medico relative all’attività della malattia;parametri di funzione fisica
    •effetto di risparmio steroideo dell’IMP (solo parte di fase 3)
    •effetto dell’IMP su:attività extramuscolare della malattia;ulteriori misure della forza muscolare;ulteriori misure della valutazione globale del paziente e del medico relativa all’attività della malattia;resistenza muscolare;dolore;affaticamento;ulteriori misure della funzione fisica
    •impatto sanitario dell’utilizzo di glucocorticoidi e effetto di risparmio steroideo dell’IMP (solo parte di fase 3)
    •l’effetto dell’IMP su:qualità della vita correlata alla salute;enzimi muscolari;prevenzione del peggioramento della malattia
    •PK dell’IMP;effetto della PD sui livelli di IgG totali dell’IMP;immunogenicità di IMP e rHuPH20;sicurezza e tollerabilità IMP
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    • Ability to consent in the jurisdiction in which the study is taking place and capable of giving signed informed consent.
    • A definite or probable clinical diagnosis of idiopathic inflammatory myopathy (IIM)
    • One of the following medical histories:
    a. Diagnosis of dermatomyositis (DM) or juvenile dermatomyositis (JDM), (age of disease onset <18 years of age). The diagnosis date for juvenile dermatomyositis should not be >5 years from the screening date.
    b. Diagnosis of polymyositis (PM) (including antisynthetase syndrome (ASyS))
    c. Diagnosis of immune-mediated necrotizing myopathy (IMNM)
    • Diagnosed with active disease as defined by the presence of at least 1 of the following criteria:
    a. Abnormal levels of at least 1 of the following enzymes: creatine kinase (CK), aldolase, lactate dehydrogenase, aspartate aminotransaminase (AST), alanine aminotransferase (ALT), based on central laboratory results
    b. Electromyography demonstrating active disease within the past 3 months
    c. Active dermatomyositis (DM) skin rash
    d. Muscle biopsy indicative of active idiopathic inflammatory myopathy (IIM) in the past 3 months
    e. Magnetic resonance imaging within the past 3 months indicative of active inflammation
    • Muscle weakness
    • Receiving a permitted background treatment for IIM. Permitted background treatment includes: oral corticosteroids; 1 antimalarial; or 1 of the following immunosuppressants: methotrexate, azathioprine, mycophenolate mofetil, mycophenolic acid, tacrolimus, cyclosporine, leflunomide, or mizoribine. Participants may receive a combination of either oral corticosteroids and up to 1 antimalarial or oral corticosteroids and up to 1 immunosuppressant.
    • Contraceptive use by nonsterilized male participants and women of childbearing potential will be consistent with local regulations, where available, for individuals participating in clinical studies. Women of childbearing potential must have a negative serum pregnancy test during screening and a negative urine pregnancy test at baseline to establish the nonpregnant state before receiving investigational medicinal product (IMP).

    The full list of inclusion criteria can be found in the protocol.
    • Capacità di prestare il consenso nella giurisdizione in cui viene condotto lo studio e capacità di prestare il consenso informato.
    • Diagnosi stabilita o probabile di miopatia infiammatoria idiopatica (MII).
    • Una delle seguenti anamnesi:
    a. diagnosi di dermatomiosite (DM) o dermatomiosite giovanile (JDM) (età dell’insorgenza <18 anni). La data di diagnosi della dermatomiosite giovanile non deve essere >5 anni dalla data di screening;
    b. diagnosi di polimiosite (PM) (inclusa sindrome da antisintetasi (ASyS));
    c. diagnosi di miopatia necrotizzante immunomediata (IMNM).
    • Diagnosi di malattia attiva come definito dalla presenza di almeno 1 dei seguenti criteri:
    a. livelli anormali di almeno 1 dei seguenti enzimi: creatinchinasi (CK), aldolasi, lattato deidrogenasi, aspartato aminotransaminasi (AST), alanina aminostransferasi (ALT), in base ai risultati del laboratorio centrale;
    b. elettromiografia che dimostri malattia attiva nei 3 mesi precedenti;
    c. eruzione cutanea da dermatomiosite attiva (DM);
    d. biopsia muscolare indicativa di miopatia infiammatoria idiopatica (MII) attiva negli ultimi 3 mesi;
    e. risonanza magnetica per immagini negli ultimi 3 mesi indicativa di infiammazione attiva.
    • Debolezza muscolare
    • Ricevimento di un trattamento di base permesso per la miopatia infiammatoria idiopatica. I trattamenti di base permessi includono: corticosteroidi orali, 1 antimalarico, o 1 dei seguenti immunosoppressori: metotrexato, azatioprina, micofenolato mofetil, acido micofenolico, tacrolimus, ciclosporina, leflunomide o mizoribina. I partecipanti possono ricevere una combinazione di corticosteroidi orali e fino a 1 antimalarico o corticosteroidi orali e fino a 1 immunosoppressore.
    • L’utilizzo di contraccettivi da parte dei partecipanti di sesso maschile non sterili e delle donne in età fertile sarà coerente con i regolamenti locali, se disponibili, per i soggetti che partecipano agli studi clinici. Le donne in età fertile devono avere un test di gravidanza sierico negativo allo screening e un test di gravidanza urinario negativo al basale per stabilire lo stato di non gravidanza prima di ricevere il medicinale sperimentale (IMP).

    Per la Lista completa dei criteri di inclusione si prega di riferirsi al protocollo
    E.4Principal exclusion criteria
    • A clinically significant uncontrolled active or chronic bacterial, viral, or fungal infection at screening
    • A COVID-19 polymerase chain reaction (PCR)-positive test before enrollment
    • Any other known autoimmune disease that, in the investigator’s opinion, would interfere with an accurate assessment of clinical symptoms of idiopathic inflammatory myopathy (IIM) or put the patient at undue risk
    • A history of malignancy unless considered cured by adequate treatment, with no evidence of recurrence for >= 3 years before the first administration of the investigational medicinal product (IMP). Adequately treated participants with the following cancers can be included at any time:
    a. Basal cell or squamous cell skin cancer
    b. Carcinoma in situ of the cervix
    c. Carcinoma in situ of the breast
    d. Incidental histological finding of prostate cancer
    • Severe muscle damage
    • Glucocorticoid-induced myopathy that the investigator considers the primary cause of muscle weakness or permanent weakness linked to a non-idiopathic inflammatory myopathy (IIM) cause
    • Juvenile myositis (JDM) diagnosed > 5 years from screening or juvenile myositis with extensive calcinosis or severe calcinosis.
    • Uncontrolled interstitial lung disease or any other uncontrolled idiopathic inflammatory myopathy (IIM) manifestation that, in the opinion of the investigator, would be likely to require treatment with prohibited medication during the study
    • Other inflammatory and noninflammatory myopathies: inclusion body myositis, overlap myositis, metabolic myopathies, muscle dystrophies or a family history of muscle dystrophy, drug-induced or endocrine induced myositis, and juvenile myositis (other than juvenile dermatomyositis (JDM))
    • Clinically significant disease, recent major surgery or intends to have surgery during the study, or has any other condition in the opinion of the investigator that could confound the results of the study or put the patient at undue risk
    • Known hypersensitivity reaction to investigational medicinal product (IMP) or 1 of its excipients
    • Received a live or live-attenuated vaccine less than 4 weeks before screening.
    • Lack of clinical response to plasmapheresis/plasma exchange (PLEX)
    • Positive serum test at screening for active viral infection with any of the following conditions:
    a. Hepatitis B virus (HBV)
    b. Hepatitis C virus (HCV)
    c. HIV
    • Any of the following prior therapy or procedures:
    a. Treatment within 2 weeks before screening: topical corticosteroids or topical immunomodulators (eg, tacrolimus) for idiopathic inflammatory myopathy (IIM) rash
    b. Treatment within 4 weeks before screening: local corticosteroid injections, anakinra, etanercept, Janus kinase (JAK) inhibitors, corticosteroids, plasmapheresis/plasma exchange, immunoadsorption
    c. Treatment within 8 weeks before screening: corticosteroid precursors
    d. Treatment within 12 weeks before screening: IVIg, SCIg, tocilizumab, abatacept, infliximab, adalimumab, golimumab, certolizumab, ustekinumab
    e. Treatment within 24 weeks before screening: rituximab or other anti-CD20 antibody, cyclophosphamide
    f. Use of a nonbiologic investigational product within 12 weeks before screening
    g. Use of a biologic therapy and/or monoclonal antibody within 24 weeks before screening
    h. Treatment with oral corticosteroids
    i. Treatment with >1 immunosuppressant, >1 antimalarial, or a combination of an immunosuppressant and an antimalarial
    • Participant has previously participated in an efgartigimod clinical study and received at least 1 dose of IMP
    • Participant is concurrently participating in any other clinical study, including a noninterventional study
    • Participant has a current or history (ie, within 12 months of screening) of alcohol, drug, or medication abuse
    • Participant is pregnant or lactating or intends to become pregnant during the study
    • Participant has severe renal impairment
    • Participant is institutionalized by a court or other governmental order or is in a dependent relationship with the sponsor or investigator
    The full list of exclusion criteria can be found in the protocol.
    •Un’infezione fungina, virale o batterica cronica o attiva non controllata, clinicamente significativa allo screening
    •Test positivo per reazione a catena della polimerasi (PCR) COVID-19 prima dell’arruolamento
    •Qualsiasi altra malattia autoimmune nota che, secondo il parere dello sperimentatore, interferirebbe con una valutazione accurata dei sintomi clinici della miopatia infiammatoria idiopatica (MII) o metterebbe inutilmente a rischio il/la paziente
    •Anamnesi di malignità, salvo se considerata curata con trattamento adeguato, senza evidenza di recidiva per >=3 anni prima della prima somministrazione del medicinale sperimentale (IMP).
    I partecipanti adeguatamente trattati con i seguenti tumori possono essere inclusi in qualsiasi momento:
    a.tumore cutaneo basocellulare o a cellule squamose;
    b.carcinoma in situ della cervice;
    c.carcinoma in situ della mammella;
    d.riscontro istologico accidentale di cancro della prostata.
    •Grave danno muscolare
    •Miopatia indotta da glucocorticoidi che, secondo il parere dello Sperimentatore, è la causa primaria di debolezza muscolare o debolezza permanente correlata a una causa di miopatia infiammatoria non idiopatica
    •Miosite giovanile (JDM) diagnosticata >5 anni dallo screening o miosite giovanile con calcinosi estesa o grave
    •Malattia polmonare interstiziale non controllata o qualsiasi altra manifestazione di miopatia infiammatoria idiopatica (MII) non controllata che, secondo il parere dello Sperimentatore, verosimilmente richiederebbe il trattamento con farmaci proibiti durante lo studio
    •Altre miopatie infiammatorie e non infiammatorie: miosite da corpi inclusi, miosite da overlap, miopatie metaboliche, distrofie muscolari o un’anamnesi famigliare di distrofia muscolare, miosite indotta da farmaco o associata al sistema endocrino e miosite giovanile (diversa dalla dermatomiosite giovanile (JDM))
    •Malattia clinicamente significativa, recente intervento chirurgico maggiore o intenzione di sottoporsi a intervento chirurgico durante lo studio, oppure presenza di un’altra condizione che, secondo il parere dello Sperimentatore, potrebbe confondere i risultati dello studio e o mettere inutilmente a rischio il/la paziente
    •Nota reazione da ipersensibilità al medicinale sperimentale (IMP) o a 1 dei suoi eccipienti
    •Il/La paziente ha ricevuto un vaccino vivo o attenuato vivo meno di 4 settimane prima dello screening.
    •Mancanza di risposta clinica a plasmaferesi/scambio plasmatico (PLEX)
    •Test sierico positivo allo screening per infezione virale attiva con una qualsiasi delle seguenti condizioni:
    a.virus dell’epatite B (HBV);
    b.virus dell’epatite C (HCV);
    c.HIV.
    •Una qualsiasi delle seguenti terapie o procedure precedenti:
    a.trattamento entro 2 settimane prima dello screening: corticosteroidi topici o immunomodulanti topici (es., tacrolimus) per eruzione cutanea da miopatia infiammatoria idiopatica (MII);
    b.trattamento entro 4 settimane prima dello screening: iniezioni di corticosteroidi orali, anakinra, etanercept, inibitori della Janus chinasi (JAK), corticosteroidi, plasmaferesi/scambio plasmatico, immunoassorbimento;
    c.trattamento entro 8 settimane prima dello screening: precursori dei corticosteroidi;
    d.trattamento entro 12 settimane prima dello screening: IVIg, SCIg, tocilizumab, abatacept, infliximab, adalimumab, golimumab, certolizumab, ustekinumab;
    e.trattamento entro 24 settimane prima dello screening: rituximab o altri anticorpi anti-CD20, ciclofosfamide;
    f.utilizzo di un medicinale sperimentale non biologico entro 12 settimane prima dello screening;
    g.utilizzo di un anticorpo monoclonale e/o terapia biologica entro 24 settimane prima dello screening;
    h.trattamento con corticosteroidi orali;
    i.trattamento con >1 immunosoppressore, >1 antimalarico o una combinazione di un immunosoppressore e un antimalarico.
    •Il/la partecipante ha già partecipato ad uno studio clinico di efgartigimod e ha ricevuto almeno 1 dose del medicinale sperimentale (IMP).
    •Il/la partecipante sta partecipando contemporaneamente a qualsiasi altro studio clinico, incluso uno studio non interventistico.
    •Il/la partecipante ha un’anamnesi attuale o precedente (cioè, entro 12 mesi dallo screening) di abuso di alcol, droghe o farmaci.
    •La partecipante è in gravidanza o sta allattando con latte materno o intende iniziare una gravidanza durante lo studio.
    •Il/la partecipante ha una compromissione renale grave
    •Il partecipante è istituzionalizzato da un tribunale o da un altro ordine governativoo ha una relazione di dipendenza con lo sponsor o lo sperimentatore

    L'elenco completo dei criteri di esclusione è disponibile nel protocollo.
    E.5 End points
    E.5.1Primary end point(s)
    Total improvement score (TIS)
    •Punteggio del miglioramento totale (TIS)
    E.5.1.1Timepoint(s) of evaluation of this end point
    Phase 2: up to 24 weeks; phase 3: up to 52 weeks
    Fase 2: fino a 24 settimane; Fase 3: fino a 52 settimane
    E.5.2Secondary end point(s)
    Key Secondary Endpoints
    • Time to reach TIS >= 20 (first "minimal clinical improvement")
    • Percentage of participants with TIS >= 20
    • Time to reach TIS > =40 (first "moderate clinical improvement")
    • Percentage of participants with TIS > =40
    • Change in manual muscle testing-8 (MMT8) score
    • Change in Patient Global Assessment of Disease Activity (PGA)
    • Change in Physician Global Assessment of Disease Activity (MDGA)
    • Change in Health Assessment Questionnaire Disability Index (HAQ-DI)
    • Change in steroid dose from baseline to last visit
    • Proportion of participants achieving target dose of <=5 mg (prednisone
    equivalent)
    • Change in the Extramuscular Global Assessment
    Other Secondary Endpoints
    • Change in abbreviated handheld dynamometry
    • Change in Patient Global Impression of Severity (PGI-S)
    • Patient Global Impression of Change (PGI-C)
    • Change in Clinical Global Impression of Severity (CGI-S)
    • Clinical Global Impression of Change (CGI-C)
    • Change in the Myositis Functional Index (FI-3)
    • Change in Patient Reported Outcomes Information System (PROMIS)
    Pain Interference 6a v1.0
    • Change in pain numeric rating scale (NRS) assessing worst pain in the past 7 days
    • Change in PROMIS Fatigue 7a v1.0
    • Change in Fatigue NRS assessing worst physical fatigue in the past 7 days
    • Change in PROMIS Physical Function 8b v2.0
    • Change in the Physical Functioning subscale and Physical Component Summary scores of the 36-Item Short Form Survey version 2 (SF-36v2)
    • Composite Glucocorticoid Toxicity Index (C-GTI) comprising the Aggregate Improvement Score (AIS) and the Cumulative Worsening
    Score (CWS)
    • Change in EQ-5D-5L utilities
    • Change in EQ-5D-5L VAS
    • Change in the most abnormal enzyme, in each participant: creatine kinase (CK), alanine aminotransferase (ALT), aspartate aminotransaminase (AST), and aldolase, or lactate dehydrogenase (LDH)
    • Proportion of participants who meet protocol-defined worsening criteria
    • Time to worsening
    • Efgartigimod serum concentrations
    • Absolute values, change from baseline, and percent reduction from baseline in total IgG levels
    • Incidence and prevalence of antidrug antibodies (ADA) against efgartigimod and antibodies against rHuPH20
    • Incidence and severity of treatment-emergent AEs (TEAEs), AESIs, and serious AEs (SAEs) presented by system organ class (SOC) and preferred term (PT)
    Endpoint secondari principali:
    • Tempo al raggiungimento di un TIS >=20 (primo “miglioramento clinico minimo”)
    • Percentuale di partecipanti con TIS >=20
    • Tempo al raggiungimento di un TIS >=40 (primo “miglioramento clinico moderato”)
    • Percentuale di partecipanti con TIS >=40
    •Cambiamento del punteggio MMT8 (test muscolare manuale 8)
    •Cambiamento della valutazione globale del paziente relativa all’attività della malattia (PGA)
    •Cambiamento della valutazione globale del medico relativa all’attività della malattia (MDGA)
    •Cambiamento del Questionario sullo stato di salute, Indice disabilità (HAQ-DI)
    •Cambiamento della dose di steroidi dal basale all’ultima visita
    •Proporzione di partecipanti che raggiungono una dose target <=5 mg (prednisone equivalente)
    •Cambiamento della valutazione globale extramuscolare
    Altri endpoint secondari
    •Cambiamento dei punteggi di dinamometria portatile abbreviata
    •Cambiamento dell’impressione globale della gravità da parte del paziente (PGI-S)
    •Impressione globale del cambiamento da parte del paziente (PGI-C)
    •Cambiamento dell’impressione clinica globale della gravità (CGI-S)
    •Impressione clinica globale del cambiamento (CGI-C)
    •Cambiamento dell’indice funzionale della miosite (FI-3)
    •Cambiamento dell’interferenza del dolore secondo il sistema informativo di misurazione degli esiti riportati dai pazienti (PROMIS) 6a v1.0
    •Cambiamento della valutazione del peggiore dolore negli ultimi 7 giorni secondo la scala di valutazione numerica del dolore (NRS)
    •Cambiamento del punteggio PROMIS affaticamento 7a v1.0
    •Cambiamento del punteggio NRS affaticamento che valuta il peggiore affaticamento fisico negli ultimi 7 giorni
    •Cambiamento del punteggio PROMIS funzione fisica 8b v2.0
    •Cambiamento della sottoscala funzione fisica e dei punteggi del riepilogo della componente fisica del 36-Item Short Form Survey versione 2 (SF-36v2)
    •Indice composito di tossicità da glucocorticoidi (C-GTI), compresi il punteggio di miglioramento aggregato (AIS) e il punteggio di peggioramento cumulativo (CWS)
    •Cambiamento del punteggio EQ-5D-5L utilities
    •Cambiamento del punteggio EQ-5D-5L VAS
    •Cambiamento dell’enzima più anomalo in ciascun partecipante: creatinchinasi (CK), alanina aminotransferasi (ALT), aspartato aminotransaminasi (AST) e aldolasi o lattato deidrogenasi (LDH)
    •Proporzione di partecipanti che soddisfano i criteri di peggioramento definiti dal protocollo
    •Tempo al peggioramento
    •Concentrazioni sieriche di efgartigimod
    •Valori assoluti, cambiamento dal basale e riduzione percentuale dal basale dei livelli di IgG totali
    •Incidenza e prevalenza di anticorpi antifarmaco (ADA) contro efgartigimod e di anticorpi contro rHuPH20
    •Incidenza e gravità di AE emergenti dal trattamento (TEAE), AESI e AE seri (SAE), presentati in base alla classificazione per sistemi e organi (SOC) e per termine preferito (PT)
    E.5.2.1Timepoint(s) of evaluation of this end point
    Phase 2: up to 24 weeks
    Phase 3: up to 52 weeks
    Fase 2: fino a 24 settimane
    Fase 3: fino a 52 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 and tolerability
    Immunogenicità e tollerabilità
    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) Yes
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled Yes
    E.8.1.1Randomised Yes
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind Yes
    E.8.1.5Parallel group Yes
    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 Yes
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial2
    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 EEA54
    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
    Argentina
    Australia
    Canada
    Israel
    Japan
    Korea, Republic of
    Mexico
    Peru
    Taiwan
    Thailand
    United States
    Austria
    France
    Lithuania
    Poland
    Sweden
    Bulgaria
    Netherlands
    Spain
    Switzerland
    Czechia
    Germany
    Greece
    Italy
    Belgium
    Denmark
    Georgia
    Hungary
    Ireland
    Portugal
    Slovakia
    Turkey
    United Kingdom
    Serbia
    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
    The end of the study is defined as the date of the last visit of the last participant in phase 3 of the study. Alternatively, it is defined as the date of the last participant visit following the sponsor's decision to terminate the study for any reason.
    La fine dello studio è definita come la data dell’ultima visita dell’ultimo partecipante nella fase 3 dello studio. In alternativa, è definita come la data della visita dell’ultimo partecipante a seguito dalla decisione dello Sponsor di interrompere lo studio per qualsiasi motivo.
    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 months4
    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 months4
    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: 220
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 20
    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 state9
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 83
    F.4.2.2In the whole clinical trial 240
    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 treatment period, participants will either enroll in the open-label extension study ARGX-113-2011 or complete a 56-day safety follow-up period.
    Dopo il periodo di trattamento, i partecipanti si arruoleranno nello studio 3 di estensione in aperto ARGX-113-2011 o completeranno il periodo di follow-up della sicurezza di 56 giorni.
    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 Decision2022-12-13
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2022-11-15
    P. End of Trial
    P.End of Trial StatusOngoing
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