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    Summary
    EudraCT Number:2020-002917-16
    Sponsor's Protocol Code Number:ARGX-113-1905
    National Competent Authority:Spain - AEMPS
    Clinical Trial Type:EEA CTA
    Trial Status:Prematurely Ended
    Date on which this record was first entered in the EudraCT database:2020-09-30
    Trial results View 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 number2020-002917-16
    A.3Full title of the trial
    An Open-Label, Multicenter, Follow-up Trial of ARGX-113-1904 to Evaluate the Safety, Tolerability, and Efficacy of Efgartigimod PH20 SC in Patients with Pemphigus
    Ensayo de seguimiento del ARGX-113-1904, abierto y multicéntrico para evaluar la seguridad, la tolerabilidad y la eficacia de efgartigimod PH20 SC en pacientes con pénfigo
    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 PH20 SC in adults with Pemphigus (Vulgaris or Foliaceus)
    Un estudio para evaluar la seguridad y la eficacia de una formulación sub-cutánea de efgartigimod PH20 SC en adultos con con pénfigo (Vulgar o Folicáceo)
    A.3.2Name or abbreviated title of the trial where available
    ADDRESS+
    ADDRESS +
    A.4.1Sponsor's protocol code numberARGX-113-1905
    A.7Trial is part of a Paediatric Investigation Plan No
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of Sponsorargenx BV
    B.1.3.4CountryBelgium
    B.3.1 and B.3.2Status of the sponsorCommercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportargenx BV
    B.4.2CountryBelgium
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationargenx BV
    B.5.2Functional name of contact pointRegulatory
    B.5.3 Address:
    B.5.3.1Street AddressIndustriepark Zwijnaarde 7
    B.5.3.2Town/ cityZwijnaarde
    B.5.3.3Post codeB-9052
    B.5.3.4CountryBelgium
    B.5.4Telephone 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
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Pemphigus Vulgaris or Pemphigus Foliaceus
    Pénfigo vulgar o pénfigo foliáceo
    E.1.1.1Medical condition in easily understood language
    Blistering autoimmune diseases that affect the skin and mucous membranes
    Enfermedades autoinmunes ampollosas que afectan la piel y las
    membranas mucosas
    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 20.0
    E.1.2Level LLT
    E.1.2Classification code 10052802
    E.1.2Term Pemphigus vulgaris
    E.1.2System Organ Class 100000004858
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10057069
    E.1.2Term Pemphigus foliaceus
    E.1.2System Organ Class 100000004858
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To assess the safety of extended treatment and re-treatment with efgartigimod PH20 SC in patients with PV or PF
    Evaluar la seguridad del tratamiento ampliado y el retratamiento con efgartigimod PH20 SC en pacientes con PV o PF
    E.2.2Secondary objectives of the trial
    To evaluate the efficacy of efgartigimod PH20 SC treatment in PV and PF
    To evaluate the effects of efgartigimod PH20 SC on quality of life (QoL) in patients with PV or PF
    To evaluate the pharmacokinetics (PK) of efgartigimod PH20 SC in patients with PV or PF
    To evaluate the PD of efgartigimod PH20 SC in patients with PV or PF
    To evaluate the immunogenicity of efgartigimod PH20 SC in patients with PV or PF
    Evaluar la eficacia del tratamiento de efgartigimod PH20 SC en PV y PF
    Evaluar los efectos de efgartigimod PH20 SC en la calidad de vida de pacientes con PV o PF
    Evaluar la farmacocinética (FC) de efgartigimod PH20 SC en pacientes con PV o PF
    Evaluar la FD de efgartigimod PH20 SC en pacientes con PV o PF
    Evaluar la inmunogenicidad de efgartigimod PH20 SC en pacientes con PV o PF
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Participants must meet all of the following 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), willingness and ability to comply with the trial protocol procedures (including required trial visits).
    2. The patient participated in trial ARGX-113-1904 and completed the study or has the defined criteria for rollover.
    3. Women of childbearing potential:
    a. Must have a negative urine pregnancy test at baseline before trial medication can be administered.
    b. Must be on a stable regimen for at least 1 month of at least 1 highly effective method of contraception (ie, failure rate of less than 1% per year) during the trial and for 90 days after the last administration of IMP.
    4. Non-sterilized male patients who are sexually active with a female partner of childbearing potential must use effective contraception from first administration of IMP through 90 days after the last administration of the IMP. Male patients practicing true sexual abstinence (as consistent with preferred and usual life style) can be included. Sterilized male patients who have had a vasectomy and with documented absence of sperm post-procedure can be included. Male patients are not allowed to donate sperm from first administration of IMP through 90 days after the last dose of IMP.
    Los participantes deben cumplir con todos los siguientes criterios de inclusión:
    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) y disposición y capacidad para cumplir los procedimientos del protocolo del ensayo (incluidas las visitas del ensayo exigidas)
    2.El paciente ha participado en el ensayo ARGX-113-1904 y ha completado el estudio o tiene los criterios definidos para la transferencia.
    3.Mujeres en edad fértil:
    a.Prueba de embarazo en orina negativa en el momento basal antes de que pueda administrarse la medicación del ensayo
    b.Deben estar recibiendo una pauta estable durante 1 mes como mínimo de al menos un método anticonceptivo muy eficaz (es decir, tasa de fallos inferior al 1% anual) durante el ensayo y durante 90 días después de la última administración del MEI
    4.Los varones no esterilizados que mantengan relaciones sexuales con una pareja en edad fértil deberán usar un método anticonceptivo eficaz desde la primera administración del MEI hasta 90 días después de la última dosis. Podrán participar varones que practiquen una abstinencia sexual real (compatible con el modo de vida preferido y habitual). Podrán participar varones esterilizados que se hayan sometido a una vasectomía con ausencia de espermatozoides documentada después del procedimiento. Los varones no podrán donar semen desde la primera administración del MEI hasta 90 días después de la última dosis del MEI.
    E.4Principal exclusion criteria
    Participants are excluded from the trial if any of the following criteria apply:
    1. Pregnant and lactating women and those intending to become pregnant during the trial or within 90 days after the last administration of IMP.
    2. Patients with clinical evidence of other significant serious disease or patients who recently underwent or have planned a major surgery during the period of the trial, or any other condition in the opinion of the investigator, that could confound the results of the trial or put the patient at undue risk.
    3. Known hypersensitivity to any of the components of the administered treatments.
    Los participantes quedan excluidos del ensayo si se aplica alguno de los siguientes criterios:
    1.Mujeres embarazadas y lactantes y mujeres que pretendan quedarse embarazadas durante el ensayo o en los 90 días siguientes a la última administración del MEI.
    2.Pacientes con signos clínicos de otra enfermedad grave importante o pacientes que se hayan sometido recientemente o tengan previsto someterse a una intervención de cirugía mayor durante el período del ensayo, o cualquier otro proceso que, en opinión del investigador, pueda confundir los resultados del ensayo o suponer un riesgo excesivo para el paciente.
    3.Hipersensibilidad conocida a cualquiera de los componentes de los tratamientos administrados
    E.5 End points
    E.5.1Primary end point(s)
    1. Incidence and severity of treatment-emergent adverse events (TEAEs), adverse events of special interest (AESIs), and serious adverse events (SAEs) by System Organ Class (SOC) and Preferred Term (PT)
    2. Vital signs, physical examination, electrocardiogram (ECG), and clinical laboratory safety evaluations
    1.Incidencia de acontecimientos adversos surgidos durante el tratamiento (AAST), acontecimientos adversos de interés especial (AAIE) y AAG por clase de órgano y sistema (SOC) y término preferente (TP).
    2.Constantes vitales, exploración física, electrocardiograma(ECG), y evaluaciones de seguridad del laboratorio clínico
    E.5.1.1Timepoint(s) of evaluation of this end point
    Up to 60 weeks
    Hasta 60 semanas
    E.5.2Secondary end point(s)
    1. Proportion of PV patients who achieve CR on minimal prednisone therapy
    2. Proportion of PV and PF patients who achieve CR on minimal prednisone therapy in patients with PV and PF 3.
    3. Time to DC
    4. Time to CR
    5. Time to CR on minimal prednisone therapy
    6. Time to CR off therapy
    7. Time to flare
    8. Rate of treatment failure
    9. Rate of flare
    10. Cumulative prednisone dose over the trial
    11. Pemphigus Disease Area Index (PDAI) at each visit
    12. EuroQol 5-Dimension 5-Level (EQ-5D-5L) score
    13. Autoimmune Bullous Disease Quality of Life (ABQOL) score
    14. Efgartigimod serum concentrations
    15. Total IgG and subtype (IgG1, IgG2, IgG3, IgG4) serum levels
    16. Anti-Dsg-1 and -3 autoantibodies serum levels
    17. Anti-drug antibodies (ADAs) to efgartigimod (serum levels) and rHuPH20 (plasma levels)
    1.Proporción de pacientes con PV que obtengan una RC con la dosis mínima de prednisona
    2.Proporción de pacientes con PV y PF que obtengan una RC con la dosis mínima de prednisona
    3.Tiempo hasta el CE
    4.Tiempo hasta la RC
    5.Tiempo hasta la RC con la dosis mínima de prednisona
    6.Tiempo hasta la RC sin tratamiento
    7.Tiempo hasta el brote
    8.Tasa de fracasos del tratamiento
    9.Tasa de brotes
    10.Dosis acumulada de prednisona durante el ensayo
    11.PDAI en cada visita
    12.Puntuación en escala EuroQol de cinco dimensiones (EQ-5D-5L)
    13.Puntuación de ABQOL (Calidad de vida con enfermedad autoinmunitaria ampollosa)
    14.Concentraciones séricas de efgartigimod
    15.Concentraciones séricas de IgG total y subtipo (IgG1, IgG2, IgG3, IgG4)
    16.Concentraciones séricas de autoanticuerpos anti-Dsg-1 y -3
    17.Anticuerpos (ACF) contra efgartigimod (niveles séricos) y rHuPH20 (niveles de plasma)
    E.5.2.1Timepoint(s) of evaluation of this end point
    1+2: up to 52 weeks treatment period
    3,4,5,6,7,8,9: up to 60 weeks
    10,11,12,13: up to 52 weeks treatment period
    14,15,16, 17: up to 60 weeks
    1+2:periodo de tratamiento de hasta 52 semanas
    3,4,5,6,7,8,9: hasta 60 semanas
    10,11,12,13: periodo de tratamiento de hasta 52 semanas
    14,15,16,17: hasta 60 semanas
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis No
    E.6.3Therapy No
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic Yes
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others Yes
    E.6.13.1Other scope of the trial description
    Immunogenicity
    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) No
    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 concerned6
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA51
    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
    Australia
    Bulgaria
    France
    Georgia
    Germany
    Greece
    Hungary
    India
    Israel
    Italy
    Japan
    Poland
    Romania
    Russian Federation
    Spain
    Turkey
    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
    LVLS
    UVUP
    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 months9
    E.8.9.1In the Member State concerned days25
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months9
    E.8.9.2In all countries concerned by the trial days25
    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: 105
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 45
    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 state16
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 100
    F.4.2.2In the whole clinical trial 150
    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)
    Under certain conditions described in the protocol, the patients may be eligible to roll over to the open-label extension trial ARGX-113-1905. For patients, randomized to the efgartigimod PH20 SC or placebo treatment arm in ARGX-113-1904, trial ARGX-113-1905 provides extended or first treatment and re-treatment options in the respective treatment arms. argenx is currently assessing the appropriateness and possibility of making efgartigimod PH20 SC available for trial participants post-trial.
    Según condiciones descritas en protocolo,los pacientes pueden ser elegibles para pasar al ensayo de extensión abiertoARGX-1131905.Para pacientes randomizados al brazo de tratam con efgartigimod PH20SC o placebo en ARGX-113-1904,ARGX-113-1905 proporciona opciones de tratam prolongado o de 1er tratam y retratam en los respectivos brazos.argenx está evaluando actualmente conveniencia y posibilidad de hacer que efgartigimod PH20SC esté disponible para ls participantes del ensayo después del ensayo
    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 Decision2020-12-26
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2020-12-23
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
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