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    Summary
    EudraCT Number:2018-002734-20
    Sponsor's Protocol Code Number:R3918-PNH-1852
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2021-05-25
    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 ConcernedItaly - Italian Medicines Agency
    A.2EudraCT number2018-002734-20
    A.3Full title of the trial
    An Open Label, Single Arm Study to Evaluate the Efficacy and Safety of REGN3918 in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Are Complement Inhibitor Naive or Have Not Recently Received Complement Inhibitor Therapy
    Studio in aperto, a braccio singolo per valutare l’efficacia e la sicurezza di REGN3918 in pazienti affetti da emoglobinuria parossistica notturna (EPN) naive al trattamento con inibitori del complemento o che non hanno ricevuto di recente una terapia con inibitori del complemento
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A trial with REGN3918 in patients Paroxysmal Nocturnal Hemoglobinuria (PNH) who have not been treated previously with a Complement Inhibitor or Have Not Recently Received Complement Inhibitor Therapy
    Studio con REGN3918 in pazienti affetti da emoglobinuria parossistica notturna (EPN) che non hanno ricevuto di recente una terapia con inibitori del complemento
    A.3.2Name or abbreviated title of the trial where available
    -
    -
    A.4.1Sponsor's protocol code numberR3918-PNH-1852
    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 SponsorREGENERON PHARMACEUTICALS, INC.
    B.1.3.4CountryUnited States
    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 supportRegeneron Pharmaceuticals, Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationRegeneron Pharmaceuticals, Inc.
    B.5.2Functional name of contact pointClinical Trial Information
    B.5.3 Address:
    B.5.3.1Street Address777 Old Saw Mill River Road
    B.5.3.2Town/ cityTarrytown, NY
    B.5.3.3Post code10591
    B.5.3.4CountryUnited States
    B.5.6E-mailclinicaltrials@regeneron.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 nameREGN3918
    D.3.2Product code [REGN3918]
    D.3.4Pharmaceutical form Lyophilisate for solution for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    Subcutaneous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNREGN3918
    D.3.9.2Current sponsor codeREGN3918
    D.3.9.3Other descriptive nameREGN3918
    D.3.9.4EV Substance CodeSUB184871
    D.3.10 Strength
    D.3.10.1Concentration unit mg/kg milligram(s)/kilogram
    D.3.10.2Concentration typeup to
    D.3.10.3Concentration number265
    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 Information not present in EudraCT
    D.3.11.3.2Gene therapy medical product Information not present in EudraCT
    D.3.11.3.3Tissue Engineered Product Information not present in EudraCT
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D.3.11.3.5Committee 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 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
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Paroxysmal nocturnal hemoglobinuria
    emoglobinuria parossistica notturna
    E.1.1.1Medical condition in easily understood language
    Paroxysmal nocturnal hemoglobinuria (PNH) is characterized by the destruction of red blood cells.
    emoglobinuria parossistica notturna è caratterizzatat dalle distruzione dei globuli rossi nel sangue
    E.1.1.2Therapeutic area Diseases [C] - Blood and lymphatic diseases [C15]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 21.1
    E.1.2Level LLT
    E.1.2Classification code 10055629
    E.1.2Term Paroxysmal nocturnal hemoglobinuria
    E.1.2System Organ Class 100000004857
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    The primary objective of the study is to demonstrate a reduction in intravascular hemolysis by REGN3918 over 26 weeks of treatment in patients with active PNH who are treatment naive to complement inhibitor therapy or have not recently received complement inhibitor therapy
    L’obiettivo primario dello studio è dimostrare una riduzione dell’emolisi intravascolare ottenuta con REGN3918 nel corso di 26 settimane di trattamento in pazienti con EPN attiva che sono naïve al trattamento con inibitori del complemento o che non hanno recentemente ricevuto una terapia con inibitori del complemento.
    E.2.2Secondary objectives of the trial
    The secondary objectives of the study are:
    •To evaluate the safety and tolerability of REGN3918.
    •To evaluate the effect of REGN3918 on parameters of intravascular hemolysis
    •To assess the concentrations of total REGN3918 in serum.
    •To evaluate the incidence of treatment emergent anti drug antibodies to REGN3918.
    •To evaluate the effect of REGN3918 on patient reported outcomes (PROs) measuring fatigue and health related quality of life
    Gli obiettivi secondari dello studio consistono nel:
    - Valutare la sicurezza e la tollerabilità di REGN3918.
    - Valutare l’effetto di REGN3918 sui parametri dell’emolisi intravascolare
    - Valutare le concentrazioni totali di REGN3918 nel siero.
    - Valutare l’incidenza degli anticorpi antifarmaco a REGN3918 emergenti dal trattamento.
    - Valutare l’effetto di REGN3918 sugli esiti riferiti dal paziente (Patient-Reported Outcomes, PRO) che misurano l’affaticamento e la qualità della vita correlata alla salute
    E.2.3Trial contains a sub-study Yes
    E.2.3.1Full title, date and version of each sub-study and their related objectives

    Other types of substudies
    Specify title, date and version of each substudy with relative objectives: There are optional future biomedical research and pharmacogenomic
    analysis sub-studies . Subjects will be required to sign separate substudy
    ICFs before collection of samples. There is no separate protocol.
    The purpose of the genomic analyses is to identify genomic associations
    with clinical or biomarker response, other clinical outcome measures and
    possible AEs. In addition, associations between genomic variants and
    prognosis or progression of as well as other diseases may also be
    studied.
    In addition, PROs such as fatigue or quality of life in a subset of patients
    at participating sites will be assessed (ie, sub study).
    There will not be separate sub-study protocols.

    Altre tipologie di sottostudi
    specificare il titolo, la data e la versione di ogni sottostudio con i relativi obiettivi: Esistono due sottostudi: ricerche future biomediche opzionali e farmacogenomica
    I soggetti saranno tenuti a firmare consensi informati separati
    prima della raccolta dei campioni. Non esiste un protocollo separato.
    Lo scopo delle analisi genomiche è di identificare le associazioni genomiche
    con la risposta clinica o i biomarker, altre outcome measure ed eventi avversi possibili. Inoltre, associazioni tra varianti genomiche e prognosi o la progressione di altre malattie in
    studio.
    Inoltre, saranno valutati come la fatica o la qualità della vita in un sottogruppo di pazienti
    ai centri partecipanti (ad esempio, sub-studio).
    Non ci saranno protocolli di sub-studio separati.
    E.3Principal inclusion criteria
    • Male or female = 18 years of age or legal age of majority at screening, whichever is greater
    •Diagnosis of paroxysmal nocturnal hemoglobinuria (PNH) confirmed by high sensitivity flow cytometry
    •Active disease, as defined by the presence of 1 or more PNH related signs or symptoms or history of red blood cell (RBC) transfusion due to PNH within 3 months of screening.
    •Lactate dehydrogenase (LDH) level = 2 × upper limit of normal (ULN) at screening visit.
    •Type III PNH granulocytes >10% at screening visit.
    Uomo o donna di = 18 anni di età o maggiorenne allo screening, a seconda di quale età sia più alta
    - Diagnosi di emoglobinuria parossistica notturna (EPN) confermata da citometria a flusso ad alta sensibilità
    - Malattia attiva, definita dalla presenza di 1 o più segni o sintomi correlati all’EPN o anamnesi di trasfusione di globuli rossi (RBC) dovuta a EPN entro 3 mesi dallo screening.
    - Livello di lattato deidrogenasi (LDH) = 2 volte il limite superiore alla norma (ULN) alla visita di screening.
    - Granulociti EPN di tipo III >10% alla visita di screening.
    E.4Principal exclusion criteria
    •Prior treatment with a complement inhibitor either within 6 months prior to screening visit or at any time where the patient was refractory to complement inhibitor therapy, in the opinion of the investigator (with the exception of eculizumab refractory patients due to the C5 variant R885H/C)
    Trattamento precedente con un inibitore del complemento entro i 6 mesi precedenti alla visita di screening o in qualsiasi momento in cui il paziente sia stato refrattario alla terapia con inibitori del complemento a giudizio dello sperimentatore (ad eccezione dei pazienti refrattari a eculizumab per via della variante in C5 R885H/C)
    E.5 End points
    E.5.1Primary end point(s)
    The co primary endpoints are:
    •The proportion of patients achieving adequate control of their intravascular hemolysis, defined as LDH = 1.5 x ULN at every scheduled time point between week 4 and week 26, inclusive
    •The proportion of patients achieving transfusion avoidance defined as no post baseline transfusion of RBCs per protocol over 26 weeks
    Gli endpoint co-primari sono:
    - Percentuale di pazienti che ottengono un controllo adeguato dell’emolisi intravascolare, definito come LDH = 1,5 ULN a ogni momento definito programmato tra la settimana 4 e 26 (incluse)
    - Percentuale di pazienti che ottengono interruzione delle trasfusioni, definita come nessuna trasfusione post-basale di RBC da protocollo nel corso di 26 settimane
    E.5.1.1Timepoint(s) of evaluation of this end point
    26 weeks
    26 settimane
    E.5.2Secondary end point(s)
    The secondary endpoints include:
    •The rate of breakthrough hemolysis over 26 weeks, defined as the measurement of LDH = 2 x ULN concomitant with associated signs or symptoms at any time subsequent to an initial achievement of disease control (ie, LDH = 1.5 x ULN)
    •The proportion of patients achieving normalization of their intravascular hemolysis, defined as LDH = 1.0 x ULN at every scheduled time point between week 4 through week 26, inclusive
    •Time to first LDH = 1.5 x ULN
    •Percentage of days with LDH = 1.5 x ULN between week 4 and week 26, inclusive.
    •Change and percent change in LDH levels from baseline to week 26
    Gli endpoint secondari sono:
    - Tasso di emolisi importante nel corso di 26 settimane, definito come misurazione di LDH = 2 x ULN concomitante con segni o sintomi associati in qualsiasi momento successivo a un controllo iniziale della malattia (ovvero, LDH = 1,5 x ULN)
    - Percentuale di pazienti che ottengono un controllo adeguato dell’emolisi intravascolare, definito come LDH = 1,0 x ULN a ogni momento definito programmato tra la settimana 4 e 26 (incluse)
    - Tempo al primo LDH = 1,5 x ULN
    - Percentuale di giorni con LDH = 1,5 x ULN tra la settimana 4 e 26 (incluse)
    - Variazione e variazione percentuale dei livelli di LDH dal basale alla settimana 26
    Tasso e numero di unità trasfusionali di RBC nel corso di 26 settimane
    - Variazione dei livelli di emoglobina RBC dal basale alla settimana 26
    - Variazione dei livelli di emoglobina libera dal basale alla settimana 26
    - Variazione e variazione percentuale del dosaggio dell’attività emolitica del complemento totale (CH50) dal basale alla settimana 26
    - Variazione degli esiti riferiti dal paziente (affaticamento misurato dalla scala FACIT-Affaticamento e qualità della vita correlata alla salute misurata dai questionari EORTC-QLQ-30 ed EQ-5D-3L del Gruppo cooperativo orientale di oncologia [European Cooperative Oncology Group]) dal basale alla settimana 26
    - Incidenza e gravità degli eventi avversi emergenti dal trattamento (Treatment-Emergent Adverse Event, TEAE) e di altre variabili di sicurezza nel corso di 26 settimane
    - Concentrazioni totali di REGN3918 nel siero valutate per tutta la durata dello studio
    - Incidenza degli anticorpi antifarmaco a REGN3918 emergenti dal trattamento nei pazienti nel tempo
    E.5.2.1Timepoint(s) of evaluation of this end point
    26 weeks
    26 settimane
    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 Yes
    E.6.7Pharmacodynamic Yes
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic Yes
    E.6.12Pharmacoeconomic No
    E.6.13Others No
    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 No
    E.8.1.1Randomised No
    E.8.1.2Open No
    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 Yes
    E.8.4 The trial involves multiple sites in the Member State concerned No
    E.8.4.1Number of sites anticipated in Member State concerned1
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA24
    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
    Canada
    Hong Kong
    Korea, Democratic People's Republic of
    Malaysia
    Singapore
    South Africa
    Taiwan
    United States
    Germany
    Hungary
    Italy
    Netherlands
    Poland
    Romania
    United Kingdom
    E.8.7Trial has a data monitoring committee No
    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
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years3
    E.8.9.1In the Member State concerned months3
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years3
    E.8.9.2In all countries concerned by the trial months3
    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: 30
    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 Yes
    F.3.3.2Women of child-bearing potential using contraception No
    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 21
    F.4.2.2In the whole clinical trial 42
    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 will be offered the opportunity to enter a separate open label extension trial with REGN3918
    Ai pazienti sarà data la possibilità di partecipare in uno studio di estensione in aperto con REGN3918
    G. Investigator Networks to be involved in the Trial
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2019-05-16
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2019-04-18
    P. End of Trial
    P.End of Trial StatusCompleted
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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
    As of 31 January 2023, all EU/EEA initial clinical trial applications must be submitted through CTIS . Updated EudraCT trials information and information on PIP/Art 46 trials conducted exclusively in third countries continues to be submitted through EudraCT and published on this website.

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