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    Summary
    EudraCT Number:2021-001277-23
    Sponsor's Protocol Code Number:ARGX-113-2007
    National Competent Authority:Spain - AEMPS
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2022-10-07
    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 ConcernedSpain - AEMPS
    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
    Estudio en fase II/III, aleatorizado, con enmascaramiento doble, comparativo con placebo, con grupos paralelos, de 2 grupos, multicéntrico y sin interrupciones operativas para evaluar la eficacia, la seguridad, la tolerabilidad, la farmacodinámica, la farmacocinética y la inmunogenicidad de efgartigimod PH20 s.c. en participantes de 18 años o más con miopatía inflamatoria idiopática activa
    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
    Estudio para investigar la eficacia y seguridad de efgartigimod PH20 SC en adultos con miopatía inflamatoria idiopática activa
    A.3.2Name or abbreviated title of the trial where available
    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 number+3293103400
    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.3.4Pharmaceutical 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.3.9.4EV 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 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
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboSolution for injection
    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)
    Miopatía inflamatoria idiopática activa (IIM)
    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 miopatía inflamatoria idiopática (IIM), también conocida como miositis, es un trastorno inflamatorio del músculo esquelético.
    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
    • Evaluar la mejora clínica del tratamiento con efgartigimod PH20 SC en comparación con el placebo, además del tratamiento inmunomodulador estándar.
    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 steroidsparing 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
    PEI = efgartigimod PH20 SC
    • Evaluar otros indicadores de la eficacia del PEI para lograr una respuesta clínica
    • Evaluar el efecto del PEI sobre:
    - la fuerza muscular
    • Evaluar el efecto ahorrador de corticoides del PEI (solo en la etapa de fase III)
    • Evaluar el efecto del PEI sobre:
    - la actividad extramuscular de la enfermedad
    - otros indicadores de las evaluaciones globales del médico y del paciente sobre la actividad de la enfermedad
    - la resistencia muscular
    - el dolor
    - la fatiga
    - otras variables del funcionamiento físico
    • Evaluar las repercusiones en la salud del uso de glucocorticoides, así como el efecto ahorrador de corticoides del PEI (solo en la etapa de fase III)
    • Evaluar el efecto del PEI sobre:
    - la calidad de vida relacionada con la salud
    - las enzimas musculares
    - la prevención del empeoramiento de la enfermedad
    • Evaluar:
    - la FC del PEI
    - el efecto FD del PEI en las concentraciones de IgG totales
    - la inmunogenia del PEI y la rHuPH20
    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 idiopathic inflammatory myopathy. 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.
    • Capacidad para prestar consentimiento en la jurisdicción en la que se está llevando a cabo el estudio y capacidad para otorgar el consentimiento informado firmado.
    • Diagnóstico clínico probable o definitivo de miopatía inflamatoria idiopática (MII)
    • Uno de los siguientes antecedentes médicos:
    a. Diagnóstico de dermatomiositis (DM) o dermatomiositis juvenil (DMJ) (edad de aparición de la enfermedad <18 años). La fecha de diagnóstico de la dermatomiositis juvenil no debe ser >5 años desde la fecha de la selección.
    b. Diagnóstico de polimiositis (PM) (incluido el síndrome antisintetasa [SAS])
    c. Diagnóstico de miopatía necrosante inmunitaria (MNI)
    • Diagnóstico de enfermedad activa, definida por la presencia de al menos 1 de los siguientes criterios:
    a. Concentraciones anómalas de al menos 1 de las siguientes enzimas: creatina-cinasa (CK), aldolasa, lactato-deshidrogenasa, aspartato-aminotransferasa (AST), alanina-aminotransferasa (ALT) de acuerdo con los resultados del laboratorio central
    b. Electromiografía que muestre enfermedad activa en los 3 últimos meses
    c. Erupción cutánea activa característica de la dermatomiositis (DM)
    d. Biopsia muscular indicativa de miopatía inflamatoria idiopática (MII) activa en los 3 últimos meses
    e. Resonancia magnética en los 3 últimos meses indicativa de inflamación activa
    • Debilidad muscular
    • Estar recibiendo un tratamiento de base permitido para la miopatía inflamatoria idiopática. Los tratamientos de base permitidos son: corticoesteroides orales, 1 antipalúdico o 1 de los siguientes inmunodepresores: metotrexato, azatioprina, micofenolato de mofetilo, ácido micofenólico, tacrólimus, ciclosporina, leflunomida o mizoribina. Los participantes pueden recibir una combinación de corticoesteroides orales y 1 antipalúdico o de corticoesteroides orales y 1 inmunodepresor.
    • El uso de anticonceptivos por parte de participantes varones no esterilizados y de participantes mujeres que puedan quedarse embarazadas debe cumplir las normativas locales, cuando las haya, para las personas que participan en ensayos clínicos. Las mujeres que puedan quedarse embarazadas deben obtener un resultado negativo en una prueba de embarazo en suero durante la selección y en una prueba de embarazo en orina en el momento basal para comprobar que no están embarazadas antes de recibir el medicamento en investigación (MI).

    La lista completa de criterios de inclusión puede consultarse en el protocolo.
    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 investigational medicinal product (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.
    • Infección bacteriana, vírica o fúngica activa o crónica no controlada y clínicamente significativa en la selección
    • Resultado positivo en la prueba de reacción en cadena de la polimerasa (PCR) de la COVID-19 antes de la inclusión
    • Cualquier otra enfermedad autoinmunitaria conocida que, en opinión del investigador, interferiría en la correcta evaluación de los síntomas clínicos de la miopatía inflamatoria idiopática (MII) o conllevaría un riesgo excesivo para el paciente
    • Antecedentes de cáncer, a menos que se considere curado mediante un tratamiento satisfactorio y sin indicios de recidiva durante un mínimo de 3 años antes de la primera administración del medicamento en investigación (MI)
    Podrá incluirse en cualquier momento a participantes con los siguientes tipos de cáncer tratados satisfactoriamente:
    a. Carcinoma basocelular o espinocelular
    b. Carcinoma de cuello uterino localizado
    c. Carcinoma de mama localizado
    d. Hallazgo histológico casual de cáncer de próstata
    • Daño muscular grave
    • Miopatía provocada por glucocorticoides considerada por el investigador como la causa principal de la debilidad muscular o debilidad permanente asociada a una causa no relacionada con la miopatía inflamatoria idiopática (MII)
    • Miositis juvenil (DMJ) diagnosticada >5 años antes de la selección o miositis juvenil con calcinosis generalizada o grave
    • Enfermedad pulmonar intersticial no controlada o cualquier otra manifestación de la miopatía inflamatoria idiopática (MII) no controlada que, en opinión del investigador, requeriría probablemente tratamiento con un medicamento prohibido durante el estudio
    • Otras miopatías de tipo inflamatorio y no inflamatorio: miositis por cuerpos de inclusión, miositis mixta, miopatías metabólicas, distrofias musculares o antecedentes familiares de distrofia muscular, miositis de origen medicamentoso o endocrino y miositis juvenil (que no sea la dermomiositis juvenil [DMJ])
    • Enfermedad clínicamente significativa, cirugía mayor reciente o intención de someterse a una intervención quirúrgica durante el estudio o cualquier otra enfermedad que, en opinión del investigador, podría dificultar la interpretación de los resultados del estudio o conllevar un riesgo excesivo para el paciente
    • Reacción de hipersensibilidad conocida al medicamento en investigación (MI) o a 1 de sus excipientes
    • Recepción de una vacuna con microbios vivos o atenuada menos de 4 semanas antes de la selección
    • Ausencia de respuesta clínica a la plasmaféresis (PLEX)
    • Resultado positivo en la selección en una prueba sérica de infección vírica activa con cualquiera de las siguientes enfermedades:
    a. Virus de la hepatitis B (VHB)
    b. Virus de la hepatitis C (VHC)
    c. VIH
    • Cualquiera de los siguientes tratamientos o procedimientos previos:
    a. Tratamiento en las 2 semanas anteriores a la selección: corticoesteroides tópicos o inmunomodulares tópicos (por ejemplo, el tracrólimus) para la erupción cutánea por miopatía inflamatoria idiopática (MII)
    b. Tratamiento en las 4 semanas anteriores a la selección: inyecciones de corticoesteroides locales, anakinra, etanercept, inhibidores de la cinasa Jano (JAK), corticosteroides, plasmaféresis, inmunoabsorción
    c. Tratamiento en las 8 semanas anteriores a la selección: precursores de corticosteroides
    d. Tratamiento en las 12 semanas anteriores a la selección: IgIV, IgSC, tocilizumab, abatacept, infliximab, adalimumab, golimumab, certolizumab, ustekinumab.
    e. Tratamiento en las 24 semanas anteriores a la selección: rituximab u otros anticuerpos anti-CD20, ciclofosfamida
    f. Uso de un producto en investigación no biológico en las 12 semanas anteriores a la selección
    g. Uso de un tratamiento biológico y/o un anticuerpo monoclonal en las 24 semanas anteriores a la selección
    h. Tratamiento con corticoesteroides orales
    i. Tratamiento con >1 inmunodepresor, >1 antipalúdico o una combinación de un inmunodepresor y un antipalúdico
    • Participantes que hayan participado anteriormente en un estudio con efgartigimod y recibido al menos 1 dosis del medicamento en investigación (MI)
    • Participantes que estén participando al mismo tiempo en otro estudio clínico, incluidos los estudios no intervencionistas
    • Participantes con abuso actual o pasado (es decir, en los 12 meses anteriores a la selección) de alcohol, drogas o medicamentos
    • Participantes embarazadas o en periodo de lactancia o que tengan intención de quedarse embarazadas durante el estudio
    • Participantes con insuficiencia renal grave
    • Participantes que estén internados por orden judicial u oficial o que mantengan una relación de dependencia con el promotor o el investigador

    La lista completa de criterios de exclusión puede consultarse en el protocolo.
    E.5 End points
    E.5.1Primary end point(s)
    Total improvement score (TIS)
    Puntuación de mejora total (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: hasta 24 semanas; fase 3: hasta 52 semanas
    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)
    • Porcentaje de participantes con TIS ≥40
    • Cambio en la puntuación de la prueba muscular manual-8 (MMT8)
    • Cambio en la evaluación global del paciente (PGA) sobre la actividad de la enfermedad
    • Cambio en la evaluación global del médico (MDGA) sobre la actividad de la enfermedad
    • Cambio en el índice de discapacidad del cuestionario para la evaluación de la salud (HAQ-DI)
    • Cambio en la dosis de corticoides desde el inicio hasta la última visita
    • Proporción de participantes que alcanzan la dosis objetivo ≤5 mg (equivalente de la prednisona)
    • Cambio en la evaluación global extramuscular

    Otros criterios de valoración secundarios
    • Cambio en la dinamometría manual abreviada
    • Cambio en la impresión global del paciente sobre la gravedad(PGI-S)
    • Impresión global del paciente sobre el cambio (PGI-C)
    • Cambio en la impresión global clínica de la gravedad (CGI-S)
    • Impresión global clínica del cambio (CGI-C)
    • Cambio en el índice de la funcionalidad para la miositis (FI-3)
    • Cambio en el sistema de información de resultados percibidos por el paciente (PROMIS), interferencia del dolor 6a v1.0
    • Cambio en la escala de valoración numérica (EVN) del dolor al evaluar el dolor más intenso en los últimos 7 días
    • Cambio en el PROMIS, fatiga 7a v1.0
    • Cambio en la EVN de la fatiga al evaluar la fatiga física más intensa en los últimos 7 días
    • Cambio en el PROMIS, funcionamiento físico 8b v2.0
    • Cambio en la subescala de funcionamiento físico y las puntuaciones de resumen del componente físico del formulario breve de 36 ítems, versión 2 (SF-36v2)
    • Índice compuesto de toxicidad de los glucocorticoides (C-GTI) formado por la puntuación total de la mejoría (AIS) y la puntuación acumulada del empeoramiento (CWS)
    • Cambio en las utilidades del EQ-5D-5L
    • Cambio en la EAV del EQ-5D-5L
    • Cambio en la enzima más anómala, en cada participante: creatina cinasa (CK), alanina transaminasa (ALT), aspartato transaminasa (AST), aldolasa o lactato deshidrogenasa (LDH)
    • Proporción de participantes que cumplan los criterios de empeoramiento de la enfermedad definidos en el protocolo
    • Tiempo hasta el empeoramiento
    • Concentraciones séricas de efgartigimod
    • Valores absolutos, cambio con respecto al inicio y reducción porcentual con respecto al inicio en las concentraciones de IgG totales
    • Incidencia y prevalencia de los anticuerpos antifármaco (AAF) contra el efgartigimod y de los anticuerpos contra la rHuPH20
    • Incidencia y gravedad de los AA surgidos durante el tratamiento (AAST), los AAIE y los AA graves (AAG) presentados por categoría de órgano, aparato o sistema (SOC) y término preferente (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: hasta 24 semanas
    Fase 3: hasta 52 semanas
    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
    Inmunogenicidad y tolerabilidad
    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 concerned10
    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 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
    Argentina
    Australia
    Canada
    Israel
    Japan
    Korea, Democratic People's Republic of
    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.
    El final del estudio se define como la fecha de la última visita del último participante en la fase 3 del estudio. Alternativamente, se define como la fecha de la última visita del participante luego de la decisión del patrocinador de terminar el estudio por cualquier 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 state11
    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 phase 3 open-label extension study ARGX-113-2011 or complete a 60-day safety follow-up period.
    Tras el periodo de tratamiento, se inscribirá a los participantes en el estudio de extensión abierto ARGX-113-2011 o bien estos completarán un periodo de seguimiento de la seguridad de 56 días.
    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 Decision2023-01-20
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2022-12-12
    P. End of Trial
    P.End of Trial StatusOngoing
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