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    Summary
    EudraCT Number:2019-002100-41
    Sponsor's Protocol Code Number:ARGX-113-1801
    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:2019-11-11
    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 number2019-002100-41
    A.3Full title of the trial
    A Phase 3, Multicenter, Randomized, Double-Blinded, Placebo-Controlled Trial to Evaluate the Efficacy and Safety of Efgartigimod (ARGX-113) 10 mg/kg Intravenous in Adult Patients with Primary Immune Thrombocytopenia
    Ensayo en fase III multicéntrico, aleatorizado, doble ciego y controlado con placebo para evaluar la eficacia y la seguridad de efgartigimod (ARGX 113) 10 mg/kg por vía intravenosa en pacientes adultos con trombocitopenia inmune primaria
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A study to assess the efficacy and safety of efgartigimod in adult patients with primary immune thrombocytopenia (an autoimmune disorder that destructs platelets, blood cells that help with clotting, and can lead to easy or excessive bruising and bleeding)
    Estudio para evaluar la eficacia y la seguridad del efgartigimod en pacientes adultos con trombocitopenia inmune primaria (un trastorno autoinmunitario que destruye las plaquetas, los glóbulos sanguíneos que ayudan a coagular la sangre, y puede provocar un exceso de hematomas o hemorragias o facilidad para sufrirlos)
    A.3.2Name or abbreviated title of the trial where available
    ADVANCE
    A.4.1Sponsor's protocol code numberARGX-113-1801
    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 number0034932483341
    B.5.5Fax number+32 9 310 3499
    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
    D.3.2Product code ARGX-113
    D.3.4Pharmaceutical form Concentrate for solution for infusion
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous 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 number20
    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 placeboConcentrate for solution for infusion
    D.8.4Route of administration of the placeboIntravenous use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Primary immune thrombocytopenia
    Trombocitopenia inmune primaria
    E.1.1.1Medical condition in easily understood language
    Disorder that can lead to easy or excessive bruising and bleeding due low levels of the cells that help blood clot
    El trastorno puede provocar un exceso de hematomas y hemorragias o facilidad para sufrirlos debido a una baja concentración de las células que ayudan a coagular la sangre
    E.1.1.2Therapeutic area Diseases [C] - Immune System Diseases [C20]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.1
    E.1.2Level LLT
    E.1.2Classification code 10050245
    E.1.2Term Autoimmune thrombocytopenia
    E.1.2System Organ Class 100000004851
    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 efficacy of efgartigimod compared to placebo in achieving a sustained platelet count response in patients with primary chronic immune thrombocytopenia (ITP), with a sustained platelet count response defined as platelet counts of at least 50×10^9/L for at least 4 of the 6 visits between visits 19 and 24 of the trial.
    Evaluar la eficacia de efgartigimod en comparación con placebo para lograr una respuesta sostenida del recuento plaquetario en pacientes con trombocitopenia inmune (TPI) primaria crónica; la respuesta sostenida del recuento plaquetario se define como recuentos plaquetarios de como mínimo 50×10exp9/l durante al menos 4 de las 6 visitas comprendidas entre las visitas 19 y 24 del ensayo.
    E.2.2Secondary objectives of the trial
    - To evaluate the efficacy of efgartigimod compared to placebo in overall platelet count response.
    - To evaluate the safety and tolerability of efgartigimod administered IV weekly or biweekly.
    - To evaluate the incidence and severity of bleeding events while receiving treatment with efgartigimod compared to placebo.
    - To evaluate the use of rescue treatment and changes in concurrent ITP therapy while receiving treatment with efgartigimod compared to placebo.
    - To evaluate the effects of efgartigimod treatment on quality-of-life (QoL) measures and patient-reported outcomes (PRO) compared to placebo.
    - To assess the immunogenicity of efgartigimod.
    - To assess the PK of efgartigimod.
    - To assess the PD effects of efgartigimod.
    - Evaluar la eficacia de efgartigimod en comparación con placebo en la respuesta global del recuento plaquetario.
    - Evaluar la seguridad y la tolerabilidad de efgartigimod administrado por vía intravenosa (i.v.) cada semana o cada dos semanas.
    - Evaluar la incidencia y la intensidad de los episodios hemorrágicos producidos durante el tratamiento con efgartigimod en comparación con el placebo.
    - Evaluar el uso de tratamiento de rescate y los cambios realizados en el tratamiento simultáneo para la TPI durante el tratamiento con efgartigimod en comparación con el placebo.
    - Evaluar los efectos del tratamiento con efgartigimod sobre las mediciones de la calidad de vida (CdV) y los resultados notificados por el paciente (RNP) en comparación con el placebo.
    - Evaluar la inmunogenicidad de efgartigimod.
    - Evaluar la farmacocinética (FC) de efgartigimod.
    - Evaluar los efectos farmacodinámicos (FD) de efgartigimod.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Ability to understand the requirements of the trial, to provide written informed consent (including consent for the use and disclosure of research-related health information), and to comply with the trial protocol procedures (including required trial visits).
    2. Male or female patient aged ≥18 years.
    3. Confirmed ITP diagnosis, at least 3 months before randomization and according to the ASH Criteria 2011, and no known other etiology for thrombocytopenia.
    4. Diagnosis supported by a response to a prior ITP therapy (other than TPO-RAs), in the opinion of the investigator.
    5. Mean platelet count of <30×10^9/L (and no single platelet count of >35×10^9/L) from 3 qualifying counts, 2 during the screening period and the pre-dose platelet count at visit 1. The 3 platelet counts must be over the course of 7 to 14 days, with at least 2 days between any 2 counts.
    6. At the start of the trial, the patient is either on concurrent ITP treatment(s) and has received at least 1 prior therapy for ITP in the past, or the patient is not on treatment for ITP but has received at least 2 prior treatments for ITP. Patients receiving permitted concurrent ITP treatment(s) at baseline, must have been stable in dose and frequency for at least 4 weeks prior to randomization.
    Permitted concurrent ITP medications include oral corticosteroids, oral immunosuppressants, dapsone/danazol, and/or eltrombopag.
    Patients not receiving concurrent ITP therapy are also eligible for the trial if they have not received prior ITP therapy for at least 4 weeks prior to baseline, and 6 months in case of prior ITP therapy with an anti-CD20 therapy (e.g. rituximab).
    7. Women of childbearing potential must have a negative serum pregnancy test at the screening visit and a negative urine pregnancy test at baseline before study medication (infusion) can be administered. Women are considered of childbearing potential unless they are post-menopausal (defined by continuous amenorrhea) for at least 1 year with an FSH of >40 IU/L or are surgically sterilized (i.e. women who had a hysterectomy, both ovaries surgically removed, or have a documented permanent female sterilization procedure including tubal ligation). Follicle-stimulating hormone can be used to confirm post-menopausal status in amenorrheic patients not on hormonal replacement therapy.
    8. Women of childbearing potential should use a highly effective method of contraception (i.e. pregnancy rate of less than 1% per year) during the trial and for 90 days after the last administration of the IMP. They must be on a stable regimen, for at least 1 month:
    • combined (estrogen and progestogen containing) hormonal contraception associated with inhibition of ovulation:
    -oral
    -intravaginal
    - transdermal
    • progestogen-only hormonal contraception associated with inhibition of ovulation:
    - oral
    - injectable
    - implantable
    • intrauterine device (IUD)
    • intrauterine hormone-releasing system
    • bilateral tubal occlusion
    • vasectomized partner (provided that the partner is the sole sexual partner of the trial participant and documented aspermia post procedure)
    • continuous abstinence from heterosexual sexual contact. Sexual abstinence is only allowable if it is the preferred and usual lifestyle of the patient. Periodic abstinence (calendar, symptothermal, post-ovulation methods) is not acceptable.
    9. Non-sterilized male patients who are sexually active with a female partner of childbearing potential must use effective double contraception, being a condom for male patients and a highly effective form of contraception for the female partner of childbearing potential (same as for female patients described in inclusion criterion 8). Male patients practicing true sexual abstinence (when this is in line with the preferred and usual lifestyle 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 during this period from signing of ICF, throughout the duration of the trial, and for 90 days after the last administration of IMP.
    1. Capacidad para comprender los requisitos del ensayo, otorgar su consentimiento informado por escrito (incluido consentimiento para uso y publicación de información sanitaria relacionada con la investigación) y para cumplir con procedimientos del protocolo (incluidas las visitas del ensayo necesarias).
    2. Paciente de sexo masculino o femenino de >=18 años de edad
    3. Diagnóstico confirmado de TPI al menos 3 meses antes de aleatorización según criterios de Sociedad Estadounidense Hematología de 2011, y ausencia de cualquier otra etiología conocida para la trombocitopenia.
    4. Diagnóstico respaldado por la respuesta a un tratamiento previo para la TPI (distinto de agonistas del receptor de trombopoyetina [AR-TPO]), en opinión del investigador.
    5. Recuento plaquetario medio <30×10exp9/l (y ningún recuento plaquetario >35×10exp9/l) en 3 recuentos de valoración, 2 durante el periodo de selección y el recuento plaquetario previo a la dosis en la visita 1. Los 3 recuentos plaquetarios deben haberse obtenido en un plazo de entre 7 y 14 días, dejando al menos 2 días entre 2 recuentos cualesquiera.
    6. Al comienzo del ensayo, el paciente recibe tratamiento(s) simultáneo(s) para la TPI y ha recibido al menos 1 tratamiento previo para la TPI en el pasado o el paciente no está recibiendo tratamiento para la TPI, pero ha recibido al menos 2 tratamientos previos para la TPI. Los pacientes que reciban tratamiento(s) simultáneo(s) permitido(s) para la TPI en el inicio deben haber mantenido una dosis y una frecuencia estables al menos durante las 4 semanas anteriores a la aleatorización.
    Los medicamentos simultáneos permitidos para la TPI son corticoesteroides por vía oral, inmunodepresores por vía oral, dapsona/danazol, y/o eltrombopag.
    Los pacientes que no reciban tratamientos simultáneos para la TPI también serán aptos para el ensayo si no han recibido antes tratamiento para la TPI durante al menos 4 semanas antes del inicio, y 6 meses en el caso de los tratamientos previos anti-CD20 para la TPI (p. ej., rituximab).
    7. Para poder recibir el medicamento (infusión) del ensayo, las mujeres con capacidad de concebir deben presentar una prueba de embarazo en suero con resultado negativo en la visita de selección y una en orina con resultado negativo en el inicio. Las mujeres se consideran con capacidad de concebir a menos que sean posmenopáusicas (definidas como mujeres con amenorrea continua) durante al menos 1 año con unas concentraciones de hormona foliculoestimulante (FSH) >40 UI/l o que estén esterilizadas quirúrgicamente (es decir, mujeres sometidas a histerectomía, a extirpación quirúrgica de ambos ovarios o a un procedimiento documentado de esterilización femenina permanente, incluida la ligadura de trompas). La hormona foliculoestimulante se puede usar para confirmar el estado posmenopáusico en pacientes amenorreicas que no reciban tratamiento de reposición hormonal.
    8. Las mujeres con capacidad de concebir deben utilizar un método anticonceptivo muy eficaz (es decir, con una tasa de embarazos inferior al 1 % anual) durante el ensayo y hasta 90 días después de la última administración del PEI. Deben estar recibiendo, durante al menos 1 mes, una pauta posológica estable de:
    • anticonceptivos hormonales combinados (con estrógeno y gestágeno) asociados a inhibición de la ovulación
    -orales
    -intravaginales
    -transdérmicos
    • anticonceptivos hormonales solo de gestágeno asociados a inhibición de la ovulación
    -orales
    -inyectables
    -implantables
    • dispositivo intrauterino (DIU)
    • sistema intrauterino de liberación hormonal
    • ligadura de trompas bilateral
    • vasectomía de la pareja (siempre que esta sea la única pareja sexual de la participante en el ensayo y se haya documentado la aspermia tras el procedimiento)
    • abstinencia continua de relaciones heterosexuales. La abstinencia sexual solo será aceptable si constituye el estilo de vida habitual y preferido del paciente. La abstinencia periódica (métodos del calendario, sintotérmico o de posovulación) no es aceptable.
    9. Los pacientes varones no esterilizados que sean sexualmente activos con una pareja de sexo femenino con capacidad de concebir deben utilizar un método anticonceptivo doble eficaz consistente en el preservativo para los pacientes de sexo masculino y un método anticonceptivo muy eficaz para la pareja de sexo femenino con capacidad de concebir (los mismos que se describen en el criterio de inclusión 8 para las pacientes del ensayo). Se puede incluir a los pacientes varones que practiquen una abstinencia sexual real (si ello concuerda con el estilo de vida habitual y preferido del participante).
    También se podrá incluir a los pacientes varones esterilizados que se hayan sometido a una vasectomía con azoospermia documentada posterior a la intervención. Además, los pacientes varones no podrán donar semen durante este periodo desde la firma del FCI, mientras dure el ensayo y hasta 90 días después de la última administración del PEI.
    E.4Principal exclusion criteria
    1. ITP/thrombocytopenia associated with another condition, e.g. lymphoma, chronic lymphocytic leukemia, viral infection, autoimmune disorders, thyroid disease, human immunodeficiency virus (HIV), hepatitis, induced or alloimmune thrombocytopenia, or thrombocytopenia associated with myeloid dysplasia.
    2. Use of anticoagulants (e.g. vitamin K antagonists, direct oral anticoagulants) within 4 weeks prior to randomization.
    3. Use of any transfusions within 4 weeks prior to randomization.
    4. Use of IVIg, SC or intramuscular route, or plasmapheresis (PLEX), 4 weeks prior to randomization.
    5. Use of anti-CD20 therapy (e.g. rituximab) within 6 months prior to randomization.
    6. Use of romiplostim within 4 weeks prior to randomization.
    7. Use of fostamatinib within 4 weeks prior to randomization.
    8. Undergone splenectomy less than 4 weeks prior to randomization.
    9. Use of any other investigational drug within 3 months or 5 half-lives of the drug (whichever is longer) prior to randomization.
    10. Use of monoclonal antibodies or Fc fusion proteins, other than those previously indicated, within 3 months prior to randomization.
    11. At the screening visit, clinically significant laboratory abnormalities as below:
    - Hemoglobin ≤9 g/dL.
    OR
    - International normalized ratio >1.5 or activated partial thromboplastin time >1.5×ULN.
    OR
    - Total IgG level <6 g/L.
    12. Patients who have a history of malignancy, including malignant thymoma, or myeloproliferative or lymphoproliferative disorders, unless deemed cured by adequate treatment with no evidence of recurrence for ≥3 years before screening. Patients with completely excised non-melanoma skin cancer (such as basal cell carcinoma or squamous cell carcinoma) or cervical carcinoma in situ would be permitted at any time.
    13. Uncontrolled hypertension, defined as a repeated elevated blood pressure exceeding 160 mmHg (systolic) and/or 100 mmHg (diastolic) despite appropriate treatments.
    14. History of any thrombotic or embolic event within 12 months prior to randomization.
    15. History of coagulopathy or hereditary thrombocytopenia or a family history of thrombocytopenia.
    16. History of a recent or planned major surgery (that involves major organs e.g. brain, heart, lung, liver, bladder, or gastrointestinal tract) within 4 weeks of randomization.
    17. Patients with known serum-positivity or who test positive for an active viral infection at screening with: Hepatitis B Virus (HBV) (except patients who are anti HBs Ab positive because of HBV vaccination), Hepatitis C Virus, HIV.
    18. Clinical evidence of significant unstable or uncontrolled acute or chronic diseases other than ITP (e.g. cardiovascular, pulmonary, hematologic, gastrointestinal, endocrine, hepatic, renal, neurological, malignancy, infectious diseases, uncontrolled diabetes) despite appropriate treatments which could put the patient at undue risk.
    19. Patients with known medical history of hypersensitivity to any of the ingredients of the IMP.
    20. Patients who previously participated in a clinical trial with efgartigimod.
    21. Pregnant or lactating females.
    22. Employees of the investigator or trial center, with direct involvement in the proposed trial or other trials under the direction of that investigator or trial center, as well as family of the employees or the investigator.
    1. TPI/trombocitopenia asociadas a otra afección, como p. ej., linfoma, leucemia linfocítica crónica, infección vírica, trastornos autoinmunitarios, enfermedades tiroideas, virus de inmunodeficiencia humana (VIH), hepatitis, trombocitopenia inducida o aloinmune, o trombocitopenia asociada a mielodisplasia.
    2. Uso de anticoagulantes (p. ej., antagonistas de la vitamina K, anticoagulantes directos por vía oral) dentro de las 4 semanas anteriores a la aleatorización.
    3. Uso de cualquier transfusión dentro de las 4 semanas anteriores a la aleatorización.
    4. Uso de IgIV por vía subcutánea o intramuscular, o de plasmaféresis (PLEX), 4 semanas antes de la aleatorización.
    5. Uso de un tratamiento anti-CD20 (p. ej., rituximab) dentro de los 6 meses anteriores a la aleatorización.
    6. Uso de romiplostim dentro de las 4 semanas anteriores a la aleatorización.
    7. Uso de fostamatinib dentro de las 4 semanas anteriores a la aleatorización.
    8. Haberse sometido a una esplenectomía menos de 4 semanas antes de la aleatorización.
    9. Uso de cualquier otro fármaco en investigación dentro de los 3 meses o las 5 semividas del fármaco (lo que suponga más tiempo) previos a la aleatorización.
    10. Uso de anticuerpos monoclonales o de proteínas de fusión de fragmentos cristalizables (Fc) distintos de los indicados anteriormente dentro de los 3 meses anteriores a la aleatorización.
    11. En la visita de selección, las anomalías analíticas de importancia clínica que se indican a continuación:
    -Hemoglobina <=9 g/dl.
    - O BIEN -
    - Índice internacional normalizado >1,5 o tiempo de tromboplastina parcial activada >1,5 × LSN.
    - O BIEN -
    - Concentración total de IgG <6 g/l.
    12. Pacientes con antecedentes de neoplasia maligna, incluido el timoma maligno, o trastornos mieloproliferativos o linfoproliferativos, a menos que se consideren curados con un tratamiento adecuado sin indicios de recurrencia durante >=3 años antes de la selección. Se admitirá en todo momento a los pacientes con cáncer de piel no melanocítico (como carcinoma basocelular o carcinoma epidermoide) o carcinoma cervicouterino in situ extirpados por completo.
    13. Hipertensión incontrolada, definida como una elevación reiterada de la presión arterial por encima de los 160 mmHg (sistólica) o los 100 mmHg (diastólica), a pesar de recibir tratamientos adecuados.
    14. Antecedentes de cualquier episodio trombótico o embólico dentro de los 12 meses anteriores a la aleatorización.
    15. Antecedentes de coagulopatía o de trombocitopenia hereditaria, o antecedentes familiares de trombocitopenia.
    16. Antecedentes de cirugía mayor reciente o programada (que afecte a órganos importantes, como cerebro, corazón, pulmón, hígado, vejiga o tubo digestivo) dentro de las 4 semanas anteriores a la aleatorización.
    17. Pacientes con seropositividad conocida o con un resultado positivo en la prueba de infección vírica activa en el momento de la selección por: Virus de la hepatitis B (VHB) (excepto los pacientes positivos para Ac anti-HB por vacunación contra el VHB), virus de la hepatitis C, VIH.
    18. Indicios clínicos de enfermedades importantes agudas o crónicas inestables o incontroladas distintas de la TPI (p. ej., enfermedades cardiovasculares, pulmonares, hematológicas, gastrointestinales, endocrinas, hepáticas, renales, neurológicas, neoplasias malignas, enfermedades infecciosas, diabetes incontrolada) a pesar de recibir tratamientos adecuados y que puedan poner al paciente en riesgo indebido.
    19. Pacientes con antecedentes médicos conocidos de hipersensibilidad a alguno de los ingredientes del PEI.
    20. Pacientes que hayan participado anteriormente en un ensayo clínico con efgartigimod.
    22. Mujeres embarazadas o en periodo de lactancia.
    23. Empleados del investigador o del centro del ensayo con participación directa en el ensayo propuesto o en otros bajo la dirección de dicho investigador o centro del ensayo, así como familiares de los empleados o del investigador.
    E.5 End points
    E.5.1Primary end point(s)
    Proportion of patients with chronic ITP with a sustained platelet count response defined as achieving platelet counts of at least 50×10^9/L for at least 4 of the 6 visits between visits 19 and 24 of the trial.
    Proporción de pacientes con TPI crónica con una respuesta sostenida del recuento plaquetario, definida como recuentos plaquetarios de como mínimo 50×10exp9/l durante al menos 4 de las 6 visitas comprendidas entre las visitas 19 y 24 del ensayo.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Between visits 19 and 24 of the trial.
    Entre las visitas 19 y 24 del estudio.
    E.5.2Secondary end point(s)
    Key Secondary Efficacy Endpoints Subject to Alpha Control:
    1. Extent of disease control defined as the number of cumulative weeks over the planned 24-week treatment period with platelet counts of ≥50×10^9/L in the chronic ITP population.
    2. Proportion of patients in the overall population (chronic and persistent ITP) with a sustained platelet count response defined as achieving platelet counts of at least 50×10^9/L for at least 4 of the 6 visits between visits 19 and 24 of the trial.
    3. Incidence and severity of the WHO-classified bleeding events.
    4. Proportion of patients in the overall population achieving platelet counts of at least 50×10^9/L for at least 6 of the 8 visits between visits 17 and 24 of the trial.
    Criterios de valoración secundarios clave de la eficacia sujetos a control de alfa:

    1. Grado de control de la enfermedad, definido como el número de semanas acumuladas durante el periodo de tratamiento previsto de 24 semanas con recuentos plaquetarios>=50 × 10exp9/l en la población con TPI crónica.
    2. Proporción de pacientes de la población global (TPI crónica y persistente) con una respuesta sostenida del recuento plaquetario, definida como recuentos plaquetarios de como mínimo 50×10exp9/l durante al menos 4 de las 6 visitas comprendidas entre las visitas 19 y 24 del ensayo.
    3. Incidencia e intensidad de los episodios hemorrágicos según la clasificación de la OMS.


    4. Proporción de pacientes de la población global con recuentos plaquetarios de como mínimo 50 × 10exp9/l durante al menos 6 de las 8 visitas comprendidas entre las visitas 17 y 24 del ensayo.
    E.5.2.1Timepoint(s) of evaluation of this end point
    1. Over the 24-week treatment
    2. Between visits 19 and 24
    3. At each visit
    4. Between visits 17 and 24
    1. Durante el tratamiento previsto de 24 semanas
    2. Entre las visitas 19 y 24 del estudio
    3. En cada visita
    4. Entre las visitas 17 y 24
    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
    Tolerability, immunogenicity of efgartigimod
    Tolerabilidad e inmunogenicidad del efgartigimod
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) No
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E.7.1.3.1Other trial type description
    E.7.2Therapeutic exploratory (Phase II) No
    E.7.3Therapeutic confirmatory (Phase III) Yes
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled 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 concerned8
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA75
    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
    Austria
    Belgium
    Bulgaria
    Canada
    Czech Republic
    France
    Germany
    Hungary
    Italy
    Japan
    Netherlands
    Poland
    Russian Federation
    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
    End-of-Trial is defined as last patient last visit in the trial.
    El final del estudio se define como la última visita del último paciente del estudio.
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years1
    E.8.9.1In the Member State concerned months7
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years1
    E.8.9.2In all countries concerned by the trial months7
    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: 140
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 16
    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 state25
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 140
    F.4.2.2In the whole clinical trial 156
    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 completing the 24 week randomized trial period will perform the End-of-Treatment visit and can enter the open-label extension trial (ARGX 113 1803) to receive efgartigimod 10 mg/kg IV.
    Los pacientes que finalicen el periodo de estudio de 24 semanas aleatorizado realizarán una visita de Fin de Tratamiento y podrán participar en el ensayo de ampliación abierto (ARGX-113-1803) para recibir efgartigimod en dosis de 10 mg/kg i.v.
    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 Decision2019-12-16
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2019-12-12
    P. End of Trial
    P.End of Trial StatusOngoing
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