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    Summary
    EudraCT Number:2021-003302-50
    Sponsor's Protocol Code Number:ARGX-117-2002
    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:2021-11-02
    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 number2021-003302-50
    A.3Full title of the trial
    A Phase 2, Randomized, Double-Blinded, Placebo-Controlled, Parallel-Group, Multicenter Trial to Evaluate the Safety and Tolerability, Efficacy, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of 2 Dose Regimens of ARGX-117 in Adults With Multifocal Motor Neuropathy
    Ensayo de fase 2, aleatorizado, doble ciego, controlado con placebo, de grupos paralelos, multicéntrico para evaluar la seguridad y la tolerabilidad, la eficacia, la farmacocinética, la farmacodinámica y la inmunogenicidad de dos pautas posológicas de ARGX-117 en adultos con neuropatía motora multifocal
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A clinical trial to investigate the safety and tolerability, efficacy pharmacokinetics, pharmacodynamics, and immunogenicity of 2 dose regimens of ARGX-117 in adults with multifocal motor neuropathy
    Ensayo clínico para evaluar la seguridad y la tolerabilidad, la eficacia, la farmacocinética, la farmacodinámica y la inmunogenicidad de dos pautas posológicas de ARGX-117 en adultos con neuropatía motora multifocal
    A.3.2Name or abbreviated title of the trial where available
    ARDA
    ARDA
    A.4.1Sponsor's protocol code numberARGX-117-2002
    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 (Ghent)
    B.5.3.3Post codeB-9052
    B.5.3.4CountryBelgium
    B.5.4Telephone number+34900834223
    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 nameARGX-117 IV
    D.3.2Product code ARGX-117 IV
    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 INNnot defined
    D.3.9.3Other descriptive nameARGX-117
    D.3.9.4EV Substance CodeSUB206651
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number120
    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 No
    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
    Multifocal Motor Neuropathy
    Neuropatía Motora Multifocal
    E.1.1.1Medical condition in easily understood language
    Multifocal Motor Neuropathy
    Neuropatía Motora Multifocal
    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 21.1
    E.1.2Level PT
    E.1.2Classification code 10065579
    E.1.2Term Multifocal motor neuropathy
    E.1.2System Organ Class 10029205 - Nervous system 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 safety and tolerability of ARGX-117 compared to placebo in adult participants previously stabilized with IVIg
    Evaluar la seguridad y la tolerabilidad de ARGX-117 en comparación con placebo en participantes adultos previamente estabilizados con IgIV
    E.2.2Secondary objectives of the trial
    - To evaluate the efficacy of ARGX-117 compared to placebo on muscle strength and/or motor function in adult participants previously stabilized with IVIg
    - To evaluate the efficacy of ARGX-117 on functional ability, arm and hand function, quality of life, and fatigue in adult participants with MMN
    - To evaluate the effect of ARGX-117 on health-related productivity and work productivity
    - To evaluate medication treatment satisfaction
    - To assess the PK, PD, and immunogenicity of ARGX-117
    • Evaluar la eficacia de ARGX-117 en comparación con placebo en la fuerza muscular o la función motora en participantes adultos previamente estabilizados con IgIV
    • Evaluar la eficacia de ARGX-117 sobre la capacidad funcional, la función de los brazos y las manos, la calidad de vida y la fatiga en participantes adultos con NMM
    • Evaluar el efecto de ARGX-117 sobre la productividad laboral y relacionada con la salud
    • Evaluar la satisfacción con la medicación.
    • Evaluar la FC, la FD y la inmunogenicidad de ARGX-117
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Capable of giving signed informed consent which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol (including consent for the use and disclosure of research related health information). Participants must be able to read and write and be willing and able to comply with the trial protocol procedures (including attending the required trial visits)
    2. Male/female at least 18 years of age at the time the ICF is signed
    3. Probable or definite MMN according to the EFNS/PNS 2010 guidelines at screening confirmed by the MCC
    4. Receiving a stable IVIg regimen before screening and both of the following:
    a) IVIg treatment interval of 2 to 5 weeks
    b) IVIg dose of 0.4 to 2.0 g per kg body weight and infusion
    5. IVIg treatment dependency confirmation by the MCC at screening or at IMV1, based on 1 of the following:
    a) Recently initiated IVIg treatment (less than 3 months):
    - Clinical improvement following IVIg initiation documented in the participant’s medical record
    b) Maintenance therapy with IVIg (longer than 3 months), based on 1 of the following:
    - Clinical deterioration following IVIg withdrawal, IVIg dose reduction, or IVIg delayed administration within 12 months prior to screening (documented in the participant’s medical record)
    - Clinical deterioration following IVIg delayed administration during the IVDP
    6. Immunization with the first meningococcal vaccine and pneumococcal vaccine, and the single Haemophilus influenza type B vaccine must be performed at least 14 days before IMP administration at V1 according to local country-specific immunization schedules. A documented history of vaccination against Neisseria meningitides, Haemophilus influenza type B, and streptococcus pneumonia will be permitted
    7. Contraceptive use by men and women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies
    a) Male participants must agree to not donate sperm from the time the ICF is signed until 12 months after the last IMP administration
    b) Women of childbearing potential (WOCBP) must have a negative serum pregnancy test at screening and a negative urine pregnancy test at baseline before IMP can be administered
    1.Capacidad para otorgar el consentimiento informado firmado, lo que incluye el cumplimiento de los requisitos y restricciones que se recogen en el documento de consentimiento informado (DCI) y en este protocolo (incluido el consentimiento para el uso y la divulgación de información médica relacionada con la investigación). Los participantes deben ser capaces de leer y escribir y estar dispuestos y ser capaces de cumplir los procedimientos del protocolo del ensayo (incluida la asistencia a las visitas del ensayo exigidas).
    2. Varón o mujer de 18 años o más de edad en el momento de la firma del DCI
    3. NMM probable o definitiva según las directrices de la EFNS/PNS de 2010 en la selección confirmada por el CCN
    4. Estar recibiendo una pauta estable de IgIV antes de la selección y cumplir las dos condiciones siguientes:
    a) Intervalo de tratamiento con IgIV de 2 a 5 semanas.
    b) Dosis de IgIV de 0,4 a 2,0 gramos por kg de peso corporal e infusión
    5. Confirmación de la dependencia del tratamiento con IgIV por el CCN en la selección o en la VVI1, basada en una de las circunstancias siguientes:
    a) Inicio reciente del tratamiento con IgIV (menos de 3 meses):
    - Mejoría clínica tras el inicio de la IgIV documentada en la historia clínica del participante.
    b) Tratamiento de mantenimiento con IgIV (más de 3 meses), basado en 1 de los siguientes criterios:
    - Deterioro clínico tras la retirada de la IgIV, la reducción de la dosis de IgIV o el retraso de la administración de IgIV en los 12 meses previos a la selección (documentado en la historia clínica del participante).
    - Deterioro clínico tras la administración diferida de IgIV durante el período de dependencia de IgIV.
    6. La vacunación con la primera vacuna antimeningocócica y la vacuna antineumocócica, y la vacuna única contra Haemophilus influenzae de tipo B, deberá realizarse al menos 14 días antes de la administración del MEI en la V1 conforme a los calendarios de vacunación específicos de cada país. Se permitirán los antecedentes documentados de vacunación contra Neisseria meningitidis, Haemophilus influenzae tipo B y Streptococcus pneumoniae
    7. El uso de anticonceptivos en varones y mujeres debe cumplir la normativa local sobre métodos anticonceptivos para participantes en estudios clínicos.
    a) Los varones participantes deberán comprometerse a no donar semen desde el momento de la firma del DCI hasta 12 meses después de la última administración del MEI.
    b) Las mujeres en edad fértil (MEF) deberán tener una prueba de embarazo en suero negativa en la selección y una prueba de embarazo en orina negativa en el momento basal antes de poder administrar el MEI
    E.4Principal exclusion criteria
    1. Any coexisting condition which may interfere with the outcome assessments (eg, diabetic neuropathy, CIDP, inflammatory arthritis, or osteoarthritis affecting the hand)
    2. Clinical signs or symptoms suggestive for neuropathies other than MMN such as motor neuron disease (eg, bulbar signs or brisk reflexes) or other inflammatory neuropathies (eg, sensory neuropathy)
    3. 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 participant or could affect adherence with the trial protocol.
    4. Clinically significant uncontrolled active or chronic bacterial, viral, or fungal infection during the screening and/or IVMP.
    5. Any other known autoimmune disease that, in the opinion of the investigator, would interfere with an accurate assessment of clinical symptoms of MMN or put the participant at undue risk (eg, SLE).
    6. History of malignancy unless resolved by adequate treatment with no evidence of recurrence for ≥3 years before the first administration of the IMP. Participants with the following carcinomas will be eligible:
    a. Adequately treated basal cell or squamous cell skin cancer
    b. Carcinoma in situ of the cervix
    c. Carcinoma in situ of the breast
    or
    d. Incidental histological finding of prostate cancer (TNM stage T1a or T1b)
    7. Clinical evidence of other significant serious diseases, have had a recent major surgery, or who have any other condition in the opinion of the investigator, that could confound the results of the trial or put the participant at undue risk
    8. Prior/concomitant therapy
    a. Cyclophosphamide and/or rituximab and/or eculizumab and/or mycophenolate mofetil within 3 months prior to screening
    b. Use of an investigational product within 3 months or 5 half-lives (whichever is longer) before the first dose of the IMP.
    9. Positive serum test at screening for an active viral infection with any of the following conditions:
    a. Hepatitis B virus (HBV) that is indicative of an acute or chronic infection (https://www.cdc.gov/hepatitis/HBV/PDFs/SerologicChartv8.pdf)
    b. Hepatitis C virus (HCV) based on HCV antibody assay
    c. HIV based on test results that are associated with an AIDS-defining condition or a CD4 count <200 cells/mm3
    10. Current or history of (ie, within 12 months of screening) alcohol, drug, or medication abuse
    11. Known hypersensitivity reaction to 1 of the components of the IMP or any of its excipients
    12. Female participants with a positive serum or urine pregnancy test, lactating females, and those who intend to become pregnant during the trial or within 9 months after last dose of the IMP
    1. Cualquier enfermedad coexistente que pueda interferir en las evaluaciones del resultado (p. ej., neuropatía diabética, polineuropatía desmielinizante idiopática crónica, artritis inflamatoria o artrosis que afecte a la mano).
    2. Signos o síntomas clínicos indicativos de neuropatías distintas de la NMM, como enfermedad de las motoneuronas (p. ej., signos bulbares o hiperreflexia) u otras neuropatías inflamatorias (p. ej., neuropatía sensitiva).
    3. 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 excesivo para el participante o afectar al cumplimiento del protocolo del ensayo.
    4. Infección bacteriana, viral o fúngica activa o crónica, clínicamente significativa y no controlada durante la selección y/o el período de vigilancia de IgIV.
    5. Cualquier otra enfermedad autoinmunitaria conocida que, en opinión del investigador, pueda interferir en una evaluación exacta de los síntomas clínicos de la NMM o suponer un riesgo excesivo para el participante (p. ej., LES).
    6. Antecedentes de neoplasias malignas, a menos que se consideren resueltas con un tratamiento adecuado sin signos de recidiva durante un mínimo de tres años antes de la primera administración del MEI. Podrán incluirse en el estudio participantes con los siguientes carcinomas:
    a. Carcinoma basocelular o epidermoide cutáneo tratado adecuadamente.
    b. Carcinoma in situ de cuello uterino.
    c. Carcinoma in situ de mama o
    d. Hallazgo histológico fortuito de cáncer de próstata (estadio TNM T1a o T1b).
    7. Evidencia clínica de otras enfermedades graves significativas, haberse sometido recientemente a una intervención de cirugía mayor, o presencia de cualquier otro proceso que, en opinión del investigador, podría confundir los resultados del ensayo o suponer un riesgo excesivo para el participante.
    8. Tratamiento previo y concomitante.
    a. Ciclofosfamida, rituximab, eculizumab o micofenolato mofetilo en los 3 meses previos a la selección.
    b. Uso de un producto en investigación en los tres meses, o el período correspondiente a cinco semividas (lo que suponga más tiempo), previos a la primera dosis del MEI.
    9. Infección vírica activa en el período de selección según una prueba en suero positiva y alguna de las circunstancias siguientes:
    a. Virus de la hepatitis B (VHB) que es indicativo de una infección aguda o crónica (https://www.cdc.gov/hepatitis/HBV/PDFs/SerologicChartv8.pdf).
    b. Virus de la hepatitis C (VHC), según un análisis de anticuerpos contra el VHC.
    c. VIH según los resultados de los análisis que se asocia a una enfermedad definitoria de SIDA o a un recuento de CD4 < 200 células/mm3.
    10. Presencia o antecedentes (en los 12 meses previos a la selección) de abuso de alcohol, drogas o medicamentos.
    11. Reacción de hipersensibilidad conocida a 1 de los componentes del MEI o a cualquiera de sus excipientes.
    12. Mujeres participantes con una prueba de embarazo positiva en suero u orina, mujeres lactantes y aquellas que pretendan quedarse embarazadas durante el ensayo o en los 9 meses siguientes a la última dosis del MEI.
    E.5 End points
    E.5.1Primary end point(s)
    Safety outcomes based on adverse event (AE) monitoring and other safety assessments
    Variables de seguridad basadas en la vigilancia de acontecimientos adversos (AA) y otras evaluaciones de la seguridad
    E.5.1.1Timepoint(s) of evaluation of this end point
    Days 1, 4, 8, 15, 22, 29, 43, 57, 71, 78, 85, 99, 102 and 113 + continuous monitoring (CM)
    Días 1, 4, 8, 15, 22, 29, 43, 57, 71, 78, 85, 99, 102 y 113 + monitorización contínua (MC)
    E.5.2Secondary end point(s)
    A. Time to the first retreatment with IVIg since the final IVIg treatment of the IVIg monitoring period
    B. Difference between the time to the first retreatment with IVIg (cycle) and the second time to retreatment with IVIg
    C. AUC of the change from baseline in mMRC-10 sum score
    D. Change from baseline in the 2 most important muscle groups as assessed by the mMRC-14 sum score
    E. Value and change from baseline in the mMRC-14 sum score
    F. Proportion of participants showing a deterioration of 1 or more points in at least 2 muscle groups as assessed by the mMRC-14 sum score
    G. Percentage of time with no deterioration in 2 or more muscle groups as assessed by mMRC-14 sum score
    H. AUC of the change from baseline in GS
    I. Proportion of participants with a GS decrease of 8 kilopascal (kPa) or more over 3 consecutive days
    J. Values, change, and percent change from baseline in GS
    K. Values and change from baseline in the Rasch-built overall disability scale for MMN (MMN‐RODS©)
    L. Values and change from baseline in upper extremity (arm and hand) function (9-Hole Peg Test [9-HPT], or timed Peg Board Test)
    M. Values and change from baseline in quality of life using the Euro-Quality of Life 5 Dimensions 5 Levels (EQ-5D-5L) scale
    N. Values and change from baseline in the chronic acquired polyneuropathy patient-reported index (CAP-PRI)
    O. Values and change from baseline in the Patient Global Impression Change (PGIC) scale
    P. Values and change from baseline in the 9-item Fatigue Severity Scale (FSS)
    Q. Values and change from baseline in the Health-Related Productivity Questionnaire (HRPQ)
    R. Values and change from baseline in the Treatment Satisfaction 14-I tem Questionnaire for Medication (TSQM)
    S. Serum concentrations and PK parameters for ARGX-117
    T. Values and change from baseline in free C2, total C2, functional complement activity (CH50)
    U. Serum titer levels of binding antibodies (BAbs) against ARGX-117
    A. Tiempo hasta el primer retratamiento con IgIVa desde el tratamiento final con IgIV del período de vigilancia de IgIV.
    B. Diferencia entre el tiempo hasta el primer retratamiento con IgIV (ciclo) y el tiempo hasta el segundo retratamiento con IgIV.
    C. Área bajo la curva (AUC) de la variación de la puntuación acumulada de la escala mMRC-10 con respecto al valor basal.
    D. Variación con respecto al momento basal en los 2 grupos musculares más importantes, evaluados mediante la puntuación acumulada de la escala mMRC-14.
    E. Valor y variación con respecto al momento basal de la puntuación acumulada de la escala mMRC-14.
    F. Proporción de participantes que presenten un deterioro de 1 o más puntos en al menos 2 grupos musculares, evaluados mediante la puntuación acumulada de la escala mMRC-14.
    G. Porcentaje de tiempo sin deterioro en 2 o más grupos musculares, evaluados mediante la puntuación acumulada de la escala mMRC-14.
    H. AUC de la variación de la FP con respecto al valor basal.
    I. Proporción de participantes con una disminución de la FP de 8 kilopascales (kPa) o más durante 3 días consecutivos.
    J. Valores, variación y variación porcentual de la FP con respecto al valor basal.
    K. Valores y variación con respecto al momento basal de la escala de discapacidad global basada en el modelo de Rasch para la NMM (MMN‐RODS©, Multifocal Motor Neuropathy-Rasch-built overall disability scale).
    L. Valores y variación con respecto al momento basal de la función de las extremidades superiores (brazo y mano) (prueba del tablero con 9 orificios [9-HPT, 9-Hole Peg Test] o prueba del tablero cronometrada).
    M. Valores y variación con respecto al momento basal de la calidad de vida según la escala Euro-Quality of Life de 5 dimensiones y 5 niveles (EQ-5D-5L).
    N. Valores y variación con respecto al valor basal del Índice de polineuropatía adquirida crónica comunicado por el paciente (CAP-PRI, chronic acquired polyneuropathy patient-reported index).
    O. Valores y variación con respecto al momento basal de la escala de Impresión global del paciente-Cambio, (PGIC, Patient Global Impression-Change).
    P. Valores y variación con respecto al momento basal de la Escala de intensidad de la fatiga de 9 apartados (FSS, Fatigue Severity Scale).
    Q. Valores y variación con respecto al valor basal del Cuestionario de productividad relacionada con la salud (HRPQ, Health-Related Productivity Questionnaire).
    R. Valores y variación con respecto al momento basal del Cuestionario de satisfacción con el tratamiento para medicamentos de 14 apartados (TSQM, Treatment Satisfaction 14-Item Questionnaire for Medication).
    S. Concentraciones séricas y parámetros FC de ARGX-117.
    T. Valores y variación con respecto al momento basal de C2 libre, C2 total y actividad funcional del complemento (CH50).
    U. Títulos séricos de anticuerpos de unión (AcU) contra ARGX-117.
    E.5.2.1Timepoint(s) of evaluation of this end point
    A & S: Days 1, 4, 8, 15, 22, 29, 43, 57, 71, 78, 85, 99, 102 and 113
    B: Days 4, 8, 15, 22, 29, 43, 57, 71, 78, 85, 99, 102 and 113
    C, D, E, F, G: Days 1, 8, 15, 22, 29, 43, 57, 71, 85, 99 and 113
    H, I & J: Days 1, 4, 8, 15, 22, 29, 43, 57, 71, 78, 85, 99, 102 and 113 + cm
    K & L: Days 1, 8, 15, 22, 29, 43, 57, 71, 85, 99, and 113
    M, N & Q: Days 1, 15, 29, 57, 85 and 113
    O: Days 1, 15, 29, 43, 57, 71, 85, 99 and 113
    P: Days 1, 15, 29, 43, 57, 71, 85, 99 and 113
    R: Days 1, 15, 29, 43, 57, 71, 85, 99, 113
    T: Days 1, 8, 15, 22, 29, 43, 57, 85 and 113
    U: Days 1 and 113
    A & S: Días 1, 4, 8, 15, 22, 29, 43, 57, 71, 78, 85, 99, 102 y 113
    B: Días 4, 8, 15, 22, 29, 43, 57, 71, 78, 85, 99, 102 y 113
    C, D, E, F, G: Días 1, 8, 15, 22, 29, 43, 57, 71, 85, 99 y 113
    H, I & J: Días 1, 4, 8, 15, 22, 29, 43, 57, 71, 78, 85, 99, 102 y 113 + cm
    K & L: Días 1, 8, 15, 22, 29, 43, 57, 71, 85, 99, y 113
    M, N & Q: Días 1, 15, 29, 57, 85 y 113
    O: Días 1, 15, 29, 43, 57, 71, 85, 99 y 113
    P: Días 1, 15, 29, 43, 57, 71, 85, 99 y 113
    R: Días 1, 15, 29, 43, 57, 71, 85, 99, 113
    T: Días 1, 8, 15, 22, 29, 43, 57, 85 y 113
    U: Días 1 y 113
    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 Yes
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others Yes
    E.6.13.1Other scope of the trial description
    Immunogenicity, Quality of Life
    Inmunogenicidad, Calidad de Vida
    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 concerned3
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA23
    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
    Canada
    Denmark
    France
    Germany
    Italy
    Netherlands
    Poland
    Spain
    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
    LVLS
    Ultima visita del último paciente
    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 months11
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months2
    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: 36
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 12
    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 state6
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 32
    F.4.2.2In the whole clinical trial 48
    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 completing the double-blinded treatment period, participants who meet the inclusion and exclusion criteria of may enroll in a longterm extension study ARGX-117-2003, and receive ARGX-117.
    Después de completar el período de tratamiento doble ciego, los participantes que cumplan con los criterios de inclusión y exclusión pueden inscribirse en un estudio de extensión a largo plazo ARGX-117-2003 y recibir ARGX-117
    G. Investigator Networks to be involved in the Trial
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2022-03-08
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2022-03-04
    P. End of Trial
    P.End of Trial StatusOngoing
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