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    EudraCT Number:2019-002100-41
    Sponsor's Protocol Code Number:ARGX-113-1801
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2021-06-18
    Trial results View results
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    A. Protocol Information
    A.1Member State ConcernedItaly - Italian Medicines Agency
    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
    Sperimentazione di fase 3, multicentrica, randomizzata, in doppio cieco, controllata con placebo, volta a valutare l’efficacia e la sicurezza di efgartigimod (ARGX-113) 10 mg/kg per via endovenosa in pazienti adulti con trombocitopenia immune 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)
    Studio volto a valutare l’efficacia e la sicurezza di efgartigimod in pazienti adulti con trombocitopenia immune primaria (un disturbo autoimmune che distrugge le piastrine, cellule del sangue che contribuiscono alla coagulazione, e può causare lividi e sanguinamenti che si manifestano con facilità o in numero eccessivo)
    A.3.2Name or abbreviated title of the trial where available
    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 BV
    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.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.5.4Telephone number+3293103400
    B.5.5Fax number+3293103499
    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 numberEU/3/19/2230
    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
    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 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. cell therapy medicinal product Information not present in EudraCT
    D. therapy medical product Information not present in EudraCT
    D. Engineered Product Information not present in EudraCT
    D. ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D. on Advanced therapies (CAT) has issued a classification for this product Information not present in EudraCT
    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 immune 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
    Disturbo che può causare lividi e sanguinamenti che si manifestano con facilità o in numero eccessivo, a causa dei bassi livelli delle cellule che contribuiscono alla coagulazione del sangue
    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 23.0
    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 week 19 and 24 of the trial.
    Valutare l’efficacia di efgartigimod rispetto al placebo nell’ottenere una risposta piastrinica sostenuta in pazienti con trombocitopenia immune primaria (ITP) cronica con una risposta piastrinica sostenuta definita come conta piastrinica di almeno 50×109/l ad almeno 4 delle 6 visite tra la settimana 19 e 24 della sperimentazione
    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 every other week (q2w).
    - 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.
    • Valutare l’efficacia di efgartigimod rispetto al placebo nella risposta piastrinica generale.
    • Valutare la sicurezza e la tollerabilità di efgartigimod somministrato per via endovenosa (EV) con frequenza settimanale o ogni 2 settimane.(q2w).
    • Valutare l’incidenza e la gravità degli eventi di sanguinamento durante il trattamento con efgartigimod rispetto al placebo.
    • Valutare l’uso di un trattamento di soccorso e di modifiche alla terapia concomitante per l’ITP durante il trattamento con efgartigimod rispetto al placebo.
    • Valutare gli effetti del trattamento con efgartigimod sulle misure della qualità della vita (QoL) e sugli esiti riferiti dal paziente (PRO) rispetto al placebo.
    • Valutare l’immunogenicità di efgartigimod.
    • Valutare la farmacocinetica (PK) di efgartigimod.
    • Valutare gli effetti farmacodinamici (PD) di 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 > and =18 years
    3. Confirmed ITP diagnosis, at least 3 months before randomization and according to the ASH Criteria, and no known other etiology for
    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 from 2 counts: 1 platelet count collected during the screening period and the pre-dose platelet count on the day of randomization (visit 1).
    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, fostamatinib and/or oral TPO-RAs. 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 (eg 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 (ie women who had a hysterectomy, a bilateral salpingectomy, both ovaries surgically removed, or have a documented permanent female sterilization procedure including tubal ligation). Follicle-stimulating hormone can be used to confirm postmenopausal status in amenorrheic patients on hormonal replacement therapy.
    8. Women of childbearing potential should use a highly effective or acceptable method of contraception 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
    o oral
    o intravaginal
    o transdermal
    - progestogen-only hormonal contraception associated with inhibition of ovulation:
    o oral
    o injectable
    o 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, postovulation methods) is not acceptable.
    Please refer to protocol for the full list.
    1. Capacità di comprendere i requisiti della sperimentazione, di fornire il consenso informato scritto (compreso il consenso per l’uso e la divulgazione delle informazioni sanitarie correlate alla ricerca) e di attenersi alle procedure del protocollo della sperimentazione (incluse le visite dello studio richieste).
    2. Paziente di sesso maschile o femminile di età > e =18 anni .
    3. Diagnosi confermata di ITP almeno 3 mesi prima della randomizzazione e in conformità con i criteri dell’American Society of Hematology e senza altre eziologie note per la trombocitopenia.
    4. Diagnosi supportata da una risposta a una precedente terapia per l’ITP (diversa dagli agonisti del recettore della trombopoietina [TPO-RAs]), a giudizio dello sperimentatore.
    5. Conta piastrinica media <30×109/l da 2 conte: 1 conta piastrinica raccolta durante il periodo di screening e la conta piastrinica pre-dose il giorno della randomizzazione (visita 1).
    6.Pazienti che all’inizio della sperimentazione stanno ricevendo un trattamento/i concomitante/i per l’ITP e che hanno ricevuto almeno 1 terapia precedente per l’ITP, o che non stanno ricevendo un trattamento per l’ITP, ma hanno ricevuto almeno 2 trattamenti precedenti per l’ITP. I pazienti che ricevono trattamento/i concomitanti consentiti per l’ITP al basale devono avere dose e frequenza stabili per almeno 4 settimane prima della randomizzazione.I farmaci concomitanti consentiti per l’ITP includono corticosteroidi orali, immunosoppressori orali, dapsone/danazolo, fostamatinib e/o TPO-RAs orali.
    Anche i pazienti che non ricevono una terapia concomitante per l’ITP sono idonei per la sperimentazione se non hanno ricevuto una precedente terapia per l’ITP per almeno 4 settimane prima del basale e per 6 mesi in caso di precedente terapia per l’ITP con una terapia anti-CD20 (per es. rituximab).
    7. Le donne in età fertile devono avere un test di gravidanza sul siero negativo alla visita di screening e un test di gravidanza sulle urine negativo al basale prima di poter somministrare l’infusione del farmaco in studio. Le donne sono considerate in età fertile a meno che non siano in post-menopausa (definita come amenorrea continua) da almeno 1 anno con valore dell’ormone follicolo-stimolante (FSH) >40 IU/l oppure chirurgicamente sterilizzate (ovvero, donne sottoposte a isterectomia, salpingectomia bilaterale, rimozione chirurgica di entrambe le ovaie o una procedura di sterilizzazione femminile permanente documentata, inclusa la legatura delle tube). L’ormone follicolo-stimolante può essere utilizzato per confermare lo stato postmenopausale nelle pazienti amenorroiche che assumono una terapia ormonale sostitutiva
    8.Le donne in età fertile devono impiegare un metodo contraccettivo altamente efficace o accettabile durante la sperimentazione e per 90 giorni dopo l’ultima somministrazione dell’IMP. Devono essere in un regime stabile, da almeno 1 mese:
    -contraccezione ormonale contenente estrogeni e progestinici associata a inibizione dell’ovulazione
    • orale
    • intravaginale
    • transdermico
    - contraccezione ormonale contenente solo progesterone associata a inibizione dell’ovulazione:
    o orale
    o iniettabile
    o impiantabile,
    -dispositivo intrauterino (IUD),
    -sistema intrauterino a rilascio di ormoni
    -occlusione bilaterale delle tube
    -compagno vasectomizzato (purché il compagno sia l’unico partner sessuale della partecipante allo studio e che l’azoospermia post vasectomia sia documentata)
    - l’astinenza continua dai rapporti sessuali eterosessuali. L’astinenza sessuale è consentita solo se si tratta dello stile di vita preferito e abituale della paziente. L’astinenza periodica (metodo del calendario, sintotermico, post-ovulazione) non è accettabile.
    Far riferimento al protocollo per la lista completa.
    E.4Principal exclusion criteria
    1. ITP/thrombocytopenia associated with another condition, eg lymphoma, chronic lymphocytic leukemia, viral infection, hepatitis, induced or alloimmune thrombocytopenia, or thrombocytopenia associated with myeloid dysplasia.
    2. Use of anticoagulants (eg 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 Ig (IV, SC or intramuscular route), or plasmapheresis (PLEX), 4 weeks prior to randomization.
    5. Use of anti-CD20 therapy (eg rituximab) within 6 months prior to randomization.
    6. Use of romiplostim within 4 weeks prior to randomization.
    7. Undergone splenectomy less than 4 weeks prior to randomization.
    8. Use of any other investigational drug within 3 months or 5 half-lives of the drug (whichever is longer) prior to randomization.
    9. Use of monoclonal antibodies or Fc fusion proteins, other than those previously indicated, within 3 months prior to randomization.
    10. At the screening visit, clinically significant laboratory abnormalities as below:
    - Hemoglobin = 9 g/dL.
    - International normalized ratio >1.5 or activated partial thromboplastin
    time >1.5×ULN.
    - Total IgG level <6 g/L.
    11. 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 > and= 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.
    12. Uncontrolled hypertension, defined as a repeated elevated blood pressure exceeding 160 mmHg (systolic) and/or 100 mmHg (diastolic) despite appropriate treatments.
    13. History of any major thrombotic or embolic event (eg, myocardial infarction, stroke, pulmonary embolism, deep venous thrombosis) within 12 months prior to randomization.
    14. History of coagulopathy or hereditary thrombocytopenia or a family history of thrombocytopenia.
    15. History of a recent or planned major surgery (that involves major organs eg brain, heart, lung, liver, bladder, or gastrointestinal tract) within 4 weeks of randomization.
    16. 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.
    17. Clinical evidence of significant unstable or uncontrolled acute or chronic diseases other than ITP (eg cardiovascular, pulmonary, hematologic, gastrointestinal, endocrine, hepatic, renal, neurological, malignancy, infectious diseases, uncontrolled diabetes) despite appropriate treatments which could put the patient at undue risk.
    18. Patients with known medical history of hypersensitivity to any of the ingredients of the IMP.
    19. Patients who previously participated in a clinical trial with efgartigimod and have received at least 1 administration of the IMP.
    20. Pregnant or lactating females.
    21. 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.
    22. Patients who received a live/lived-attenuated vaccine within 4 weeks before screening. The receipt of any inactivated, sub-unit, polysaccharide, or conjugate vaccine at any time before screening. The receipt of any inactivated, sub-unit, polysaccharide, or conjugate vaccine at any time before screening is not considered an exclusion criterion.
    1.ITP/trombocitopenia associata a un’altra patologia, per es linfoma, leucemia linfocitica cronica, infezione virale,epatite,trombocitopenia indotta o alloimmune o trombocitopenia associata a displasia mieloide.2.Uso di anticoagulanti (per es, antagonisti della vitamina K, anticoagulanti orali diretti) entro 4 settimane prima della randomiz.3.Uso di eventuali trasfusioni entro 4 settimane prima della randomiz.4.Uso di Ig (per via endovenosa, sottocutanea o intramuscolare),o plasmaferesi (PLEX) 4 sett prima della randomiz.5.Uso di terapia anti-CD20 (per es. rituximab) nei 6 mesi precedenti la randomiz.6.Uso di romiplostim entro 4 settimane prima della randomiz.7.Splenectomia meno di 4 settimane prima della randomiz. 8.Uso di qualsiasi altro farmaco sperimentale entro 3 mesi o 5 emivite del farmaco (a seconda di quale periodo sia più lungo) prima della randomiz.9.Uso di anticorpi monoclonali o proteine di fusione a frammento cristallizzato (Fc) diverse da quelle precedentemente indicate entro i 3 mesi precedenti alla randomizzazione.10.Alla visita di screening, anomalie di laboratorio clinicamente significative come le seguenti:• Emoglobina = 9 g/dl.- OPPURE• Rapporto internazionale normalizzato >1,5 o tempo di tromboplastina parziale attivata >1,5 volte il limite superiore della norma (ULN) - O • Livello totale di IgG <6 g/l.11.Pazienti con un’anamnesi di tumore maligno, incluso il timoma maligno, o disturbi mieloproliferativi o linfoproliferativi, a meno che non siano considerati curati con un trattamento adeguato senza alcuna evidenza di recidiva per > e = 3 anni prima dello screening. I pazienti con tumore della pelle non-melanoma (come il carcinoma basocellulare o il carcinoma a cellule squamose) o carcinoma in situ della cervice completamente escisso saranno consentiti in qualsiasi momento. 12. Ipertensione non controllata, definita come pressione sanguigna ripetutamente elevata superiore a 160 mmHg (sistolica) e/o 100 mmHg (diastolica) nonostante trattamenti adeguati. 13.Anamnesi di qualsiasi evento trombotico o embolico maggiore (per es.: infarto del miocardio, ictus, embolia polmonare, trombosi venosa profonda) entro i 12 mesi precedenti alla randomizzazione. 14.Anamnesi di coagulopatia o trombocitopenia ereditaria o anamnesi familiare di trombocitopenia. 15.Anamnesi di intervento chirurgico maggiore recente o programmato (che interessa gli organi principali, per es. cervello, cuore, polmone, fegato, vescica o tratto gastrointestinale) entro 4 settimane dalla randomizzazione. 16.Pazienti con sieropositività nota o che risultano positivi a un’infezione virale attiva allo screening con: virus dell’epatite B (HBV) (eccetto i pazienti positivi agli anticorpi anti-HBs a causa di vaccinazione anti-HBV), virus dell’epatite C, HIV.17.Evidenza clinica di malattie acute o croniche significative instabili o non controllate diverse dall’ITP (per es. malattie cardiovascolari, polmonari, ematologiche, gastrointestinali, endocrine, epatiche, renali, neurologiche, tumore maligno, malattie infettive, diabete non controllato), nonostante l’impiego di trattamenti appropriati, che potrebbero esporre il paziente a un rischio eccessivo.18.Pazienti con anamnesi nota di ipersensibilità a uno qualsiasi degli eccipienti dell’IMP. 19.Pazienti che hanno precedentemente partecipato a una sperimentazione clinica con efgartigimod e che hanno ricevuto almeno una somministrazione di IMP. 20.Donne in gravidanza o in allattamento. 21.Dipendenti dello sperimentatore o del centro di sperimentazione direttamente coinvolti nello studio proposto o coinvolti in altri studi sotto la direzione di tale sperimentatore o centro di sperimentazione, nonché familiari dei dipendenti o dello sperimentatore. 22.Pazienti che hanno ricevuto un vaccino vivo/vivo attenuato entro le 4 sett prec allo screening.La ricezione di qualsiasi vaccino coniugato o polisaccaridico a sub- unità, inattivato in qualsiasi momento prima dello screening non è considerata un crit di escl.
    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 week 19 and 24 of the trial.
    Percentuale di pazienti con ITP cronica con risposta piastrinica sostenuta definita come raggiungimento di una conta piastrinica di almeno 50×109/l ad almeno 4 delle 6 visite tra la settimana 19 e 24 della sperimentazione.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Between week 19 and 24 of the trial
    Tra la visita 19 e 24 della sperimentazione.
    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 week 19 and 24 of the trial.
    3.Incidence and severity of the WHO-classified bleeding events in the overll population.
    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 week 17 and 24 of the trial.
    Endpoint secondari di efficacia principali soggetti al controllo alfa:
    1.Misura di controllo della malattia definita come il numero di settimane cumulative durante il periodo di trattamento di 24 settimane pianificato con conta piastrinica =50×10^9/l nella popolazione con ITP cronica.
    2.Percentuale di pazienti nella popolazione generale (con ITP cronica e persistente) con risposta piastrinica sostenuta definita come raggiungimento di una conta piastrinica di almeno 50×10^9/l ad almeno 4 delle 6 visite tra la settimana 19 e 24 della sperimentazione.
    3.Incidenza e gravità degli eventi di sanguinamento classificati dall’OMS nella popolazione complessiva.
    4.Percentuale di pazienti nella popolazione generale che raggiungono una conta piastrinica di almeno 50×10^9 /l ad almeno 6 delle 8 visite tra la settimana 17 e 24 della sperimentazione.
    E.5.2.1Timepoint(s) of evaluation of this end point
    1. Over the 24-week treatment
    2. Between week 19 and 24
    3. At each visit
    4. Between week 17 and 24
    1.Durante il trattamento di 24 settimane,
    2.Tra la settimana 19 e 24
    3.A ogni visita,4
    4.Tra la settimana 17 e 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
    Tollerabilità, immunogenicità di 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. 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 concerned12
    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 Information not present in EudraCT
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    Russian Federation
    United States
    United Kingdom
    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
    Si definisce Fine dello studio l’ultima visita dell’ultimo/a paziente nella sperimentazione.
    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 state40
    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.
    I pazienti che completano il periodo di 24 settimane della sperimentazione randomizzata eseguiranno la visita di Fine trattamento e possono accedere alla sperimentazione di estensione in aperto (ARGX-113-1803) per ricevere efgartigimod 10 mg/kg EV.
    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 Decision2020-02-07
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2019-12-16
    P. End of Trial
    P.End of Trial StatusOngoing
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