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    Summary
    EudraCT Number:2019-003076-39
    Sponsor's Protocol Code Number:ARGX-113-1802
    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:2020-02-28
    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 number2019-003076-39
    A.3Full title of the trial
    A Phase 2 Trial to Investigate the Efficacy, Safety, and Tolerability of Efgartigimod PH20 SC in Adult Patients with Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)
    Ensayo de fase 2 para investigar la eficacia, la seguridad y la tolerabilidad de efgartigimod PH20 SC en pacientes adultos con polineuropatía desmielinizante inflamatoria crónica (PDIC)
    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 in adults with chronic inflammatory demyelinating polyneuropathy (an autoimmune disorder that affects the peripheral nerves)
    Estudio para evaluar la seguridad y la eficacia de una formulación subcutánea de efgartigimod en adultos con polineuropatía desmielinizante inflamatoria crónica (un trastorno autoinmune que afecta los nervios periféricos
    A.4.1Sponsor's protocol code numberARGX-113-1802
    A.7Trial is part of a Paediatric Investigation Plan No
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of Sponsorargenx BVBA
    B.1.3.4CountryBelgium
    B.3.1 and B.3.2Status of the sponsorCommercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportargenx BVBA
    B.4.2CountryBelgium
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationargenx BVBA
    B.5.2Functional name of contact pointRegulatory
    B.5.3 Address:
    B.5.3.1Street AddressIndustriepark Zwijnaarde 7
    B.5.3.2Town/ cityZwijnaarde
    B.5.3.3Post codeB-9052
    B.5.3.4CountryBelgium
    B.5.4Telephone number0034900834223
    B.5.6E-mailRegistroEspanolDeEstudiosClinicos@druginfo.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.3.9.3Other 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.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
    Chronic Inflammatory Demyelinating Polyneuropathy
    Polineuropatía desmielinizante inflamatoria crónica
    E.1.1.1Medical condition in easily understood language
    Inflammation of peripheral nervous system
    Inflamación del sistema nervioso periférico.
    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
    Stage A: To assess the activity of efgartigimod PH20 SC (efgartigimod co-formulated with recombinant human hyaluronidase PH20 [rHuPH20]) based on the percentage of patients classified as treatment responders.

    Stage B: To determine the efficacy of efgartigimod PH20 SC compared to placebo based on the time needed for the occurrence of the first evidence of clinical deterioration.
    Etapa A: Evaluar la actividad de efgartigimod PH20 SC (efgartigimod coformulado con hialuronidasa humana recombinante PH20 [rHuPH20]) en base al porcentaje de pacientes clasificados como con respuesta al tratamiento.

    Etapa B: Determinar la eficacia de efgartigimod PH20 SC en comparación con placebo de acuerdo con el tiempo necesario para la aparición de los primeros signos de deterioro clínico.
    E.2.2Secondary objectives of the trial
    Stage A
    - To assess the time to clinical improvement.
    -To determine the treatment effect of efgartigimod PH20 SC based on clinical functional assessments of motor function and muscle strength.
    -To assess the short-term safety and tolerability of efgartigimod PH20 SC.
    -To assess the pharmacokinetics (PK) of efgartigimod PH20 SC.
    -To assess the pharmacodynamic (PD) effect of efgartigimod PH20 SC.
    -To assess the immunogenicity of efgartigimod and rHuPH20.

    Stage B
    -To determine the efficacy of efgartigimod PH20 SC compared to placebo based on clinical functional assessments of disease disability and motor function and muscle strength.
    -To assess the safety and tolerability of efgartigimod PH20 SC.
    -To assess the PK of efgartigimod PH20 SC.
    -To assess the PD effect of efgartigimod PH20 SC.
    -To assess the immunogenicity of efgartigimod and rHuPH20.
    Etapa A
    - Evaluar el tiempo hasta la mejoría clínica.
    - Determinar el efecto del tratamiento con efgartigimod PH20 SC de acuerdo con las evaluaciones funcionales clínicas de la función motora y la fuerza muscular.
    - Evaluar la seguridad y la tolerabilidad a corto plazo de efgartigimod PH20 SC.
    - Evaluar la farmacocinética (FC) de efgartigimod PH20 SC.
    - Evaluar el efecto farmacodinámico (FD) de efgartigimod PH20 SC.
    - Evaluar la inmunogenicidad de efgartigimod y rHuPH20.

    Etapa B
    - Determinar la eficacia de efgartigimod PH20 SC en comparación con placebo de acuerdo con las evaluaciones funcionales clínicas de la discapacidad de la enfermedad y de la función motora y la fuerza muscular.
    - Evaluar la seguridad y la tolerabilidad de efgartigimod PH20 SC.
    - Evaluar la FC de efgartigimod PH20 SC.
    - Evaluar el efecto FD de efgartigimod PH20 SC.
    - Evaluar la inmunogenicidad de efgartigimod y rHuPH20.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Patients are eligible to be included in the trial only if all of the following criteria apply:
    1. Ability to understand the requirements of the trial, provide written informed consent (include 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. Male or female patient aged 18 years or older, at the time of signing the informed consent.

    3. Diagnosed with probable or definite CIDP according to criteria of the European Federation of Neurological Societies/Peripheral Nerve Society (EFNS/PNS 2010), progressing or relapsing forms.

    4. CIDP Disease Activity Status (CDAS) score ≥2 at screening

    5. INCAT score ≥2 , with a scrore of 2 exclusively form leg disability, at the first run-in visit (RI-VI; for patients entering run-in) or stage A baseline (A-VI; for treatment-naïve patients with documented evidence for worsening on the total adjusted INCAT disability score within 3 months prior to screening)

    6. Fulfilling any of the following treatment conditions:
    -Currently treated with pulsed corticosteroids, oral corticosteroids equivalent to prednisolone/prednisone ≤10mg/day, and/or IVIg or SCIg, if this treatment has been started within the last 5 years before screening, and the patient is willing to discontinue this treatment at the first run-in visit (RI-VI); or
    -Without previous treatment (treatment-naive); or
    -Treatment with corticosteroids and/or IVIg or SCIg discontinued at least 6 months prior to screening
    Note: Patients not treated with monthly or daily corticosteroids, IVIg or SCIg for at least 6 months prior to screening are considered as equal to treatment-naïve patients.

    7. Women of childbearing potential who have a negative pregnancy test at screening and a negative urine pregnancy test up to Stage A baseline (D1A).

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

    9. 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 screening 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 screening to 90 days after the last administration of IMP
    Únicamente podrán ser incluidos en el estudio los pacientes que cumplan todos los criterios siguientes:
    1. Capacidad para comprender los requisitos del ensayo, otorgar el consentimiento informado por escrito (incluido el consentimiento para el uso y la divulgación de información médica relacionada con la investigación), disposición y capacidad para cumplir los procedimientos del protocolo del ensayo (incluidas las visitas del ensayo exigidas).

    2. Paciente varón o mujer, de 18 años o más en el momento de firmar el documento de consentimiento informado.

    3. Diagnóstico de PDIC probable o confirmado según los criterios de la European Federation of Neurological Societies/Peripheral Nerve Society (EFNS/PNS, 2010)(11), formas en progresión o recidiva.

    4. Puntuación del Estado de Actividad de la Enfermedad de PDIC (CDAS)(14) ≥ 2 en la selección.

    5. Puntuación INCAT ≥ 2, con una puntuación de 2 exclusivamente de discapacidad en las piernas en la primera visita de preinclusión (PI-V1; en los pacientes que se incorporen a la preinclusión) o en el momento basal de la Etapa A (A-V1; en los pacientes no tratados previamente con datos documentados de empeoramiento de la puntuación total ajustada de discapacidad INCAT en los 3 meses previos a la selección).

    6. Cumplimiento de cualquiera de las siguientes situaciones relativas al tratamiento:
    - Tratamiento actual con corticosteroides en pulsos, corticosteroides orales equivalentes a prednisolona/prednisona ≤ 10 mg/día o IgIV o IgSC, si este tratamiento se ha iniciado en los 5 años previos a la selección y el paciente está dispuesto a suspender este tratamiento en la primera visita de preinclusión (PI-V1); O
    - Sin tratamiento previo; O
    - El tratamiento con corticosteroides, IgIV o IgSC se suspendió al menos 6 meses antes de la selección.
    Nota: Se considera que los pacientes no tratados con corticosteroides mensuales o diarios, IgIV o IgSC durante al menos 6 meses antes de la selección son iguales a los pacientes sin tratamiento previo.

    7. Mujeres en edad fértil con una prueba de embarazo en suero negativa en la selección y una prueba de embarazo en orina negativa hasta el momento basal de la Etapa A (D1A).

    8. Las mujeres en edad fértil deberán utilizar un método anticonceptivo altamente eficaz (tasa de fracasos inferior al 1 % anual) desde la selección hasta 90 días después de la última administración del PEI.

    9. Los pacientes varones no esterilizados que mantengan relaciones sexuales con una pareja femenina en edad fértil deberán utilizar preservativo y su pareja deberá utilizar un método anticonceptivo altamente eficaz (tasa de fracasos inferior al 1 % anual) desde la selección hasta 90 días después de la última administración del PEI. Podrán participar pacientes varones que practiquen una abstinencia sexual verdadera (cuando esté en consonancia con el modo de vida preferido y habitual del participante). Podrán participar pacientes varones esterilizados que se hayan sometido a una vasectomía con aspermia documentada después del procedimiento. Además, los pacientes varones no podrán donar semen desde la selección hasta 90 días después de la última administración del PEI.
    E.4Principal exclusion criteria
    Patients are excluded from the trial if any of the following criteria apply:
    1. Pure sensory atypical CIDP (EFNS/PNS definition(11)).

    2. Polyneuropathy of other causes, including the following:
    -Multifocal motor neuropathy;
    -Monoclonal gammopathy of uncertain significance with anti-myelin-associated glycoprotein immunoglobulin M (IgM) antibodies;
    -Hereditary demyelinating neuropathy;
    -Polyneuropathy, organomegaly, endocrinopathy, monoclonal protein and skin change syndromes;
    -Lumbosacral radiculoplexus neuropathy;
    -Polyneuropathy most likely due to diabetes mellitus;
    -Polyneuropathy most likely due to systemic illnesses;
    -Drug- or toxin-induced polyneuropathy.

    3. Any other disease that could better explain the patient’s signs and symptoms.

    4. Any history of myelopathy or evidence of central demyelination.

    5. Current or past history (within 12 months of screening) of alcohol, drug or medication abuse.

    6. Severe psychiatric disorder (such as severe depression, psychosis, bipolar disorder), history of suicide attempt, or current suicidal ideation that in the opinion of the investigator could create undue risk to the patient or could affect adherence with the trial protocol.

    7. Patients with clinically significant active or chronic uncontrolled bacterial, viral, or fungal infection at screening, including patients who test positive for an active viral infection at screening with:
    − Active Hepatitis B Virus (HBV): serologic panel test results indicative of an active (acute or chronic) infection;
    − Active Hepatitis C Virus (HCV): serology positive for HCV-Ab;
    − Human Immunodeficiency Virus (HIV) positive serology associated with an Acquired Immune Deficiency Syndrome (AIDS)-defining condition or with a cluster of differentiation 4 (CD4) count ≤200 cells/mm3.

    8. Total IgG level <6 g/L at screening.

    9. Treatment with the following:
    -Within 3 months (or 5 half-lives of the drug, whichever is longer) before screening: plasma exchange or immunoadsorption, any concomitant Fc-containing therapeutic agents or other biological, or any other investigational product;
    -Within 6 months before screening: rituximab, alemtuzumab, any other monoclonal antibody, cyclophosphamide, interferon, tumor necrosis factor–alpha inhibitors, fingolimod, methotrexate, azathioprine, mycophenolate, any other immunomodulating or immunosuppressive medications, and oral daily corticosteroids >10 mg/day.
    Note: Patients using IVIg, SCIg, pulsed corticosteroids, and oral daily corticosteroids ≤10 mg/day can be included.

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

    11. Patients with any other known autoimmune disease that, in the opinion of the investigator, would interfere with an accurate assessment of clinical symptoms of CIDP.

    12. Use of an investigational drug within 3 months or 5 half-lives of the drug (whichever is longer) prior to first IMP administration.

    13. Patients who have a history of malignancy unless deemed cured by adequate treatment with no evidence of recurrence for ≥3 years before the first IMP administration. Patients with the following cancer can be included anytime:
    -Adequately treated basal cell or squamous cell skin cancer,
    -Carcinoma in situ of the cervix,
    -Carcinoma in situ of the breast, or
    -Incidental histological finding of Prostate cancer (TNM [tumor, nodes, and metastases classification] stage T1a or T1b).

    14. Patients who previously participated in a trial with efgartigimod and have received at least one administration of IMP.

    15. Patients with known medical history of hypersensitivity to any of the ingredients of IMP.

    16. 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.
    Se excluirá del ensayo a los pacientes que cumplan alguno de los criterios siguientes:
    1. PDIC atípica sensitiva pura (definición de EFNS/PNS(11)).

    2. Polineuropatía de otras causas, como las siguientes:
    - Neuropatía multifocal motora
    - Gammapatía monoclonal de significado incierto con anticuerpos contra la glucoproteína inmunoglobulina M (IgM) asociada a la mielina;
    - Neuropatía desmielinizante hereditaria;
    - Síndromes de polineuropatía, organomegalia, endocrinopatía, proteína monoclonal y alteraciones cutáneas;
    - Neuropatía por radiculoplexo lumbosacro;
    - Polineuropatía probablemente debida a diabetes mellitus;
    - Polineuropatía probablemente debida a enfermedades sistémicas;
    - Polineuropatía inducida por fármacos o toxinas.

    3. Cualquier otra enfermedad que pueda explicar mejor los signos y síntomas del paciente.

    4. Antecedentes de mielopatía o signos de desmielinización central.

    5. Antecedentes actuales o pasados (en los 12 meses previos a la selección) de abuso de alcohol, drogas o medicamentos.

    6. Trastorno psiquiátrico grave (como depresión grave, psicosis, trastorno bipolar), antecedentes de intento de suicidio o ideación suicida actual que, en opinión del investigador, pueda suponer un riesgo indebido para el paciente o afectar al cumplimiento del protocolo del ensayo.

    7. Pacientes con infección bacteriana, vírica o fúngica activa o crónica no controlada clínicamente significativa en la selección, incluidos los pacientes que den positivo para una infección vírica activa en la selección con:
    - Virus de la hepatitis B (VHB) activo: resultados de pruebas serológicas indicativos de una infección activa (aguda o crónica);
    - Virus de la hepatitis C (VHC) activo: serología positiva para Ac-VHC;
    - Serología positiva para el virus de la inmunodeficiencia humana (VIH) asociada a una enfermedad definitoria del síndrome de inmunodeficiencia adquirida (SIDA) o con un recuento de linfocitos CD4 (grupo de diferenciación 4) ≤ 200 células/mm3.

    8. Concentración de IgG total < 6 g/l en la selección.

    9. Tratamiento con lo siguiente:
    - En los 3 meses (o 5 semividas del fármaco, lo que sea más largo) previos a la selección: recambio o inmunoadsorción de plasma, cualquier agente terapéutico concomitante que contenga fragmentos cristalizables (Fc) u otro producto biológico, o cualquier otro producto en investigación;
    - En los 6 meses previos a la selección: rituximab, alemtuzumab, cualquier otro anticuerpo monoclonal, ciclofosfamida, interferón, inhibidores del factor de necrosis tumoral alfa, fingolimod, metotrexato, azatioprina, micofenolato, cualquier otro medicamento inmunomodulador o inmunodepresor y corticosteroides orales diarios >10 mg/día.
    Nota: Podrán participar los pacientes que utilicen IgIV, IgSC, corticosteroides en pulsos y corticosteroides orales diarios a dosis ≤ 10 mg/día.

    10. Mujeres embarazadas y lactantes y mujeres que pretendan quedarse embarazadas durante el ensayo o en los 90 días siguientes a la última administración del PEI.

    11. Pacientes con cualquier otra enfermedad autoinmunitaria conocida que, en opinión del investigador, interferiría en una evaluación exacta de los síntomas clínicos de la PDIC.

    12. Uso de un fármaco en investigación en los 3 meses o 5 semividas del fármaco (lo que suponga más tiempo) previos a la primera administración del PEI.

    13. Pacientes con antecedentes de neoplasias malignas, a menos que se consideren curadas con un tratamiento adecuado sin signos de recidiva durante ≥ 3 años antes de la primera administración del PEI. Los pacientes con el siguiente cáncer pueden incluirse en cualquier momento:
    - Carcinoma de piel basocelular o epidermoide de piel correctamente tratados,
    - Carcinoma in situ de cuello uterino,
    - Carcinoma in situ de mama o
    - Hallazgo histológico fortuito de cáncer de próstata (estadio TNM [tumor, ganglios y metástasis] T1a o T1b).

    14. Pacientes que hayan participado previamente en un ensayo con efgartigimod y hayan recibido al menos una administración del PEI.

    15. Pacientes con antecedentes médicos conocidos de hipersensibilidad a cualquiera de los componentes del PEI.

    16. Pacientes con signos clínicos de otra enfermedad grave importante o pacientes que se hayan sometido recientemente o tengan programada una intervención de cirugía mayor, o cualquier otro motivo que pueda confundir los resultados del ensayo o suponer un riesgo indebido para el paciente.
    E.5 End points
    E.5.1Primary end point(s)
    Stage A
    Percentage of patients with confirmed evidence of clinical improvement (ECI).

    Stage B
    Stage B: Time to first ajusted INCAT deterioration compared to Stage B baseline
    Etapa A
    Porcentaje de pacientes con signos de mejora clínica confirmados (SMC).

    Etapa B
    Tiempo transcurrido hasta el primer deterioro ajustado de la INCAT en comparación con el momento basal de la Etapa B.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Stage A
    Up to 12 weeks (with option 1 additional week for confirmation of ECI) during the open-label stage A

    Stage B
    Every Stage B visit
    Etapa A
    Hasta 12 semanas ( con opcion de 1 semana adicional para confirmar los signos de mejora clínica) durante el periodo de regimen abierto de la Etapa A

    Etapa B
    Cada visita de la Etapa B
    E.5.2Secondary end point(s)
    Stage A
    - Time to initial confirmed ECI
    - Change from Stage A baseline (D1A) over time in adjusted INCAT score
    - Change from Stage A baseline (D1A) over time in Medical Research Council (MRC) Sum score
    - Change from Stage A baseline (D1A) over time in 24-item Inflammatory Rasch-built Overall Disability Scale (I-RODS) disability scores
    - Change from Stage A baseline (D1A) over time in Timed Up-and-go (TUG) score
    - Change from Stage A baseline (D1A) over time in Mean grip strength
    - Exposure adjusted occurrence of treatment-emergent adverse events and serious adverse events
    - Incidence of clinically significant laboratory abnormalities
    - Pre-dosing efgartigimod serum concentrations over time
    - Changes of serum IgG levels (total and IgG subtypes) over time
    - Percentage of patients with and titers of binding antibodies towards efgartigimod and/or rHuPH20 and the presence of neutralizing antibodies against efgartigimod and/or rHuPH20


    Stage B
    - Time to CIDP disease progression.
    Note: Time to CIDP disease progression is defined by the time from first dose of double-blind IMP to the first I-RODS score decrease ≥4 points compared to Stage B baseline using the centile metric.
    - Percentage of patients with improved functional level compared to Stage B baseline as measured by an increase in the 24-item I-RODS score up to Week 48
    - Change from Stage B baseline over time in adjusted INCAT score
    - Change from Stage B baseline over time in MRC Sum score
    - Change from Stage B baseline over time in 24-item I-RODS disability score
    - Change from Stage B baseline over time in TUG score
    - Change from Stage B baseline over time in mean grip strength
    - Time to 10% decrease in the 24-item I-RODS
    - Exposure adjusted occurrence of treatment-emergent adverse events and serious adverse events
    - Incidence of clinically significant laboratory abnormalities
    - Pre-dosing efgartigimod serum concentrations over time
    - Changes of serum IgG levels (total and IgG subtypes) over time
    - Percentage of patients with and titers of binding antibodies towards efgartigimod and/or rHuPH20 and the presence of neutralizing antibodies against efgartigimod and/or rHuPH20
    Etapa A
    − Tiempo transcurrido hasta los SMC confirmados iniciales.
    − Variación con respecto al momento basal de la Etapa A (D1A) a lo largo del tiempo de la puntuación INCAT ajustada
    − Variación con respecto al momento basal de la Etapa A (D1A) a lo largo del tiempo de la puntuación de Suma del Medical Research Council (MRC).
    − Variación con respecto al momento basal de la Etapa A (D1A) a lo largo del tiempo de las puntuaciones de discapacidad según la Escala de Discapacidad Global inflamatoria de Rasch de 24 apartados (I-RODS).
    − Variación con respecto al momento basal de la Etapa A (D1A) a lo largo del tiempo de la puntuación cronometrada de levantarse y caminar (Timed Up and go - TUG).
    − Variación con respecto al momento basal de la Etapa A (D1A) a lo largo del tiempo de la fuerza media de prensión evaluada con un vigorímetro de Martin.
    − Incidencia ajustada respecto a la exposición de acontecimientos adversos aparecidos durante el tratamiento (AAAT) y acontecimientos adversos graves (AAG)
    − Incidencia de anomalías analíticas clínicamente significativas.
    − Concentraciones séricas de efgartigimod antes de la administración a lo largo del tiempo.
    − Porcentaje de pacientes con títulos de anticuerpos de unión (AcU) contra efgartigimod o rHuPH20 y la presencia de anticuerpos neutralizantes (AcN) contra efgartigimod y títulos de AcN contra rHuPH20.

    Etapa B
    − Tiempo hasta la progresión de la PDIC.
    Nota: El tiempo hasta la progresión de la enfermedad PDIC se define como el tiempo transcurrido entre la primera dosis del PEI en régimen doble ciego y la primera disminución de la puntuación I-RODS ≥ 4 puntos en comparación con el momento basal de la Etapa B, utilizando la métrica centil.
    − Porcentaje de pacientes con mejoría del nivel funcional en comparación con el momento basal de la Etapa B, determinada por un aumento de la puntuación I-RODS de 24 apartados hasta la semana 48.
    − Variación con respecto al momento basal de la Etapa B a lo largo del tiempo de la puntuación INCAT ajustada
    − Variación con respecto al momento basal de la Etapa B a lo largo del tiempo de la puntuación total del MRC
    − Variación con respecto al momento basal de la Etapa B a lo largo del tiempo de las puntuaciones de discapacidad I-RODS de 24 apartados.
    − Variación con respecto al momento basal de la Etapa B a lo largo del tiempo de la puntuación TUG.
    − Variación con respecto al momento basal de la Etapa B a lo largo del tiempo de la fuerza media de prensión evaluada con un vigorímetro de Martin.
    − Tiempo hasta una disminución del 10 % de la puntuación I-RODS de 24 apartados.
    − Incidencia ajustada respecto a la exposición de acontecimientos adversos aparecidos durante el tratamiento y acontecimientos adversos graves
    − Incidencia de anomalías analíticas clínicamente significativas.
    − Concentraciones séricas de efgartigimod antes de la administración a lo largo del tiempo.
    − Variaciones de las concentraciones séricas de IgG (subtipos total e IgG) a lo largo del tiempo.
    − Porcentaje de pacientes con títulos de AcU contra efgartigimod o rHuPH20 y la presencia de AcN contra efgartigimod y títulos de AcN contra rHuPH20.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Stage A
    Up to 12 weeks (with option 1 additional week for confirmation of ECI) during the open-label stage A

    Stage B
    Up to 48 weeks during the randomized placebo-controlled stage B
    Etapa A
    Hasta12 semanas ( con opcion de 1 semana adicional para confirmar los signos de mejora clínica) durante el periodo de regimen abierto de la Etapa A

    Etapa B
    Hasta 48 semanas durante el periodo de tratamienco aleatorizado y controlado con placebo en la Etapa B
    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
    Inmunogenicidad
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) No
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E.7.1.3.1Other trial type description
    E.7.2Therapeutic exploratory (Phase II) Yes
    E.7.3Therapeutic confirmatory (Phase III) No
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled 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 concerned6
    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 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
    Belgium
    Bulgaria
    Canada
    Czech Republic
    Denmark
    France
    Georgia
    Germany
    Hungary
    Israel
    Italy
    Japan
    Netherlands
    Poland
    Romania
    Russian Federation
    Serbia
    Spain
    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
    The end of trial is defined as the last patient last visit in the ARGX-113-1802 trial, ie, any of the following:
    1) the last visit before roll-over to the Open-Label Extension (OLE) trial,
    2) the follow-up visit 28 days after the Week-48 visit for patients not participating in the OLE trial or,
    3) the follow-up visit 28 days after early discontinuation, if applicable.
    El final del ensayo se define como la última visita del paciente en el ensayo ARGX-113-1802, es decir, cualquiera de los siguientes:
    1) la última visita antes de pasar al estudio de extensión de régimen abierto (OLE),
    2) la visita de seguimiento 28 días después de la visita de la semana 48 para pacientes que no participan en el ensayo OLE o,
    3) la visita de seguimiento 28 días después de la interrupción anticipada, si corresponde.
    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 months8
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years3
    E.8.9.2In all countries concerned by the trial months8
    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: 240
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 160
    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 state30
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 193
    F.4.2.2In the whole clinical trial 400
    F.5 Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)
    Patients who have clinical deterioration or who complete the Week-48 visit will have the possibility to enter the 4-week safety follow-up period and complete the ARGX-113-1802 trial or have the possibility to enroll for the OLE trial. Patients not eligible for OLE, or not willing to participate in the OLE trial, or have withdrawn/discontinued early, the most appropriate treatment will be provided in accordance with the trial site’s standard of care and generally accepted medical practice .
    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 Decision2020-05-25
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2020-03-25
    P. End of Trial
    P.End of Trial StatusOngoing
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