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    Summary
    EudraCT Number:2021-002460-46
    Sponsor's Protocol Code Number:ARGX-113-2008
    National Competent Authority:Spain - AEMPS
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2022-02-02
    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 number2021-002460-46
    A.3Full title of the trial
    A Long-term, Single-Arm, Open-label, Multicenter, Follow-on Trial of ARGX113-2006 to Evaluate Safety of Efgartigimod Administered Intravenously in Children With Generalized Myasthenia Gravis.
    Ensayo de seguimiento a largo plazo, de un solo brazo, abierto, multicéntrico de ARGX-113-2006 para evaluar la seguridad de Efgartigimod administrado por vía intravenosa en niños con miastenia grave generalizada.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Evaluating Long-term Safety of Efgartigimod Administered Intravenously in Children With Generalized Myasthenia Gravis.
    Evaluación de la seguridad a largo plazo de efgartigimod administrado por vía intravenosa en niños con miastenia grave generalizada.
    A.3.2Name or abbreviated title of the trial where available
    ADAPT JR.+
    ADAPT JR.+
    A.4.1Sponsor's protocol code numberARGX-113-2008
    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 code9052
    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 Yes
    D.2.5.1Orphan drug designation numberEMA/OD/245/17
    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.3.9.4EV Substance CodeSUB198780
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration 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
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Generalized Myasthenia Gravis
    Miastenia Grave Generalizada
    E.1.1.1Medical condition in easily understood language
    Myasthenia Gravis in patients aged 2 to 18 years who have generalized
    Muscle Weakness
    Miastenia Grave en pacientes de 2 a 18 años que tienen debilidad
    muscular generalizada
    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 21.1
    E.1.2Level PT
    E.1.2Classification code 10028417
    E.1.2Term Myasthenia gravis
    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 efgartigimod IV
    Evaluar la seguridad y la tolerabilidad de efgartigimod IV
    E.2.2Secondary objectives of the trial
    To evaluate the immunogenicity of efgartigimod IV
    Evaluar la inmunogenicidad de efgartigimod IV
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. The participant completed ARGX-113-2006, defined as:
    a. The participant reached EoT in trial ARGX-113-2006 and agreed to participate in the ARGX-113-2008 trial.
    b. The participant qualifies for retreatment in trial ARGX-113-2006, but cannot complete a Treatment Period (TP) and the required IP visits within the ARGX-113-2006 trial's timeframe (ie, between TPnV1 to IPnV4).
    2. Either the participant or the participant’s legally acceptable representative (LAR) can understand the requirements of the trial and provide written informed consent/assent (including consent/assent for the use and disclosure of research-related health information), and demonstrates a willingness and ability to comply with the trial protocol procedures (including attending the required trial visits).
    3. Contraceptive use by males and females must be consistent with local regulations for those participating in clinical trials and:
    a. Male participants agree not to donate sperm from the time the informed consent form (ICF) is signed until the end of the trial.
    b. Female adolescents of childbearing potential (FAOCBP; defined in Section 10.4.1.1 of the protocol ) must have a negative urine pregnancy test at trial entry (TP1V1 or IP0V1).
    The contraceptive requirements for male participants are described in Section 10.4.2.2. The contraceptive requirements for FAOCBP are described in Section 10.4.2.1.
    1. El participante ha completado el estudio ARGX-113-2006, lo que se define como:
    a. El participante alcanzó el FDE en el ensayo ARGX-113-2006 y accedió a participar en el ensayo ARGX-113-2008.
    b. El participante cumple los requisitos para repetir el tratamiento en el ensayo ARGX-113-2006, pero no puede completar un período de tratamiento (PT) ni las visitas IP obligatorias dentro del plazo del ensayo ARGX-113-2006 (es decir, entre TPnV1 e IPnV4).
    2. El participante o su representante legal pueden comprender los requisitos del ensayo y otorgar su consentimiento/asentimiento informado por escrito (incluido el consentimiento/asentimiento para el uso y la comunicación de información médica relacionada con la investigación) y muestran disposición y capacidad para cumplir los procedimientos del protocolo del ensayo (incluida la asistencia a las visitas obligatorias del ensayo).
    3. El uso de anticonceptivos por varones y mujeres deberá cumplir la normativa local para participantes en ensayos clínicos y:
    a. Los varones participantes se comprometen a no donar semen desde el momento de la firma del documento de consentimiento informado (DCI) hasta el final del ensayo.
    b. Las adolescentes en edad fértil (definición en la sección 10.4.1.1 del protocolo) deberán tener una prueba de embarazo en orina negativa en el momento de incorporarse al ensayo (TP1V1 o IP0V1).
    Los requisitos sobre anticonceptivos para los varones participantes se describen en la sección 10.4.2.2. Los requisitos sobre anticonceptivos para las adolescentes en edad fértil se describen en la sección 10.4.2.1.
    E.4Principal exclusion criteria
    Participants are excluded from the trial if any of the following criteria apply:
    1. FAOCBP: Pregnancy or lactation, or the participant intends to become pregnant during the trial or within 90 days after the last dose of investigational medicinal product (IMP)
    2. Discontinued early from ARGX-113-2006 treatment due to: pregnancy, receiving prohibited medication, participating in another trial with an investigational product, or the occurrence of a life-threatening or an investigational medicinal product-related AE, as assessed by the investigator
    3. A known hypersensitivity reaction to efgartigimod or any of its excipients
    4. Received a live-attenuated vaccine fewer than 4 weeks before trial entry.
    5. Any of the following medical conditions:
    a. Clinically significant uncontrolled chronic bacterial, viral, or fungal infection at trial entry
    b. Any other known autoimmune disease that, in the opinion of the investigator, would interfere with accurate assessment of clinical symptoms of gMG or put the participant at undue risk
    Se excluirá del estudio a los participantes que cumplan alguno de los criterios siguientes:
    1. Adolescentes en edad fértil: Embarazo o lactancia, o intención de quedarse embarazada durante el ensayo o en los 90 días siguientes a la última dosis del producto en investigación (PEI).
    2. Suspensión prematura del tratamiento con ARGX-113-2006 por embarazo, recepción de un medicamento no permitido, participación en otro ensayo con un producto en investigación o aparición de un AA potencialmente mortal o relacionado con el producto en investigación, según la evaluación del investigador.
    3. Reacción de hipersensibilidad conocida a efgartigimod o a cualquiera de sus excipientes.
    4. Recepción de una vacuna atenuada con microbios vivos menos de 4 semanas antes de la incorporación al ensayo.
    5. Cualquiera de las siguientes afecciones:
    a. Infección bacteriana, viral o micótica crónica, no controlada y clínicamente significativa al incorporarse al ensayo.
    b. Cualquier otra enfermedad autoinmunitaria conocida que, en opinión del investigador, podría interferir en una evaluación exacta de los síntomas clínicos de la MGg o suponer un riesgo excesivo para el participante.
    E.5 End points
    E.5.1Primary end point(s)
    - Incidence and severity of adverse events (AEs)
    - Incidence and severity of serious adverse events (SAEs)
    - Incidence and severity of adverse events of special interest (AESIs)
    - Changes in laboratory test results, vital signs, height and weight, and
    electrocardiogram (ECG) results
    - Incidencia e intensidad de los acontecimientos adversos (AA)
    - Incidencia e intensidad de los acontecimientos adversos graves (AAG)
    - Incidencia e intensidad de los acontecimientos adversos de interés especial (AAIE)
    - Variaciones de los resultados analíticos, las constantes vitales, la estatura y el peso y los resultados del electrocardiograma (ECG)
    E.5.1.1Timepoint(s) of evaluation of this end point
    Once a month
    Una vez al mes
    E.5.2Secondary end point(s)
    Incidence and prevalence of antidrug antibodies (ADAs) against
    efgartigimod
    Incidencia y prevalencia de anticuerpos contra el fármaco (ACF) dirigidos contra efgartigimod
    E.5.2.1Timepoint(s) of evaluation of this end point
    No specific timepoints, during the entire study
    No hay momentos específicos; durante todo el estudio
    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 Yes
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic No
    E.6.7Pharmacodynamic No
    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
    - Tolerabilidad
    - 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) Yes
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled No
    E.8.1.1Randomised No
    E.8.1.2Open Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group No
    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 No
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial1
    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 concerned2
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA21
    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
    France
    Georgia
    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
    The end of the trial is defined as the date of the last visit of the last
    participant in the ARGX-113-2008 trial. A participant is considered to
    have completed the trial if he/she completed the end of trial (EoT) visit
    and safety follow-up (SFU) visit
    El final del ensayo se define como la fecha de la última visita del último participante en el ensayo ARGX-113-2008. Se considera que un participante ha finalizado el ensayo si completa la visita de final del ensayo (FDE) y la visita de seguimiento de la seguridad (SS).
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years4
    E.8.9.1In the Member State concerned months
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years4
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 Yes
    F.1.1Number of subjects for this age range: 12
    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) Yes
    F.1.1.5.1Number of subjects for this age range: 6
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 6
    F.1.2Adults (18-64 years) No
    F.1.3Elderly (>=65 years) No
    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 state2
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 6
    F.4.2.2In the whole clinical trial 12
    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)
    At the end of the study trial, argenx will comply with all local laws and regulations to ensure access to the drug before commercial availability or until another option to access efgartigimod is available.
    Al final del ensayo, argenx cumplirá todas las leyes y reglamentos locales para garantizar el acceso al fármaco antes de su comercialización o hasta que se disponga de otra opción de acceso a efgartigimod.
    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 Decision2022-04-04
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2022-02-28
    P. End of Trial
    P.End of Trial StatusOngoing
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    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
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