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    Summary
    EudraCT Number:2019-000130-20
    Sponsor's Protocol Code Number:R3918-PNH-1868
    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:2021-05-24
    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 number2019-000130-20
    A.3Full title of the trial
    An open-label extension study to evaluate the long-term safety, tolerability, and efficacy of REGN3918 in patients with paroxysmal nocturnal hemoglobinuria
    Studio di estensione in aperto atto a valutare la sicurezza a lungo termine, la tollerabilità e l’efficacia di REGN3918 in pazienti con emoglobinuria parossistica notturna
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    REGN3918 in patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) to evaluate its long term safety, efficacy and tolerability.
    REGN3918 in pazienti con emoglobinuria notturna parossistica (PNH) per valutare la sua sicurezza, efficacia e tollerabilità a lungo termine.
    A.3.2Name or abbreviated title of the trial where available
    R3918-PNH-1868
    R3918-PNH-1868
    A.4.1Sponsor's protocol code numberR3918-PNH-1868
    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 IMPSubcutaneous 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.3.9.4EV Substance CodeSUB184871
    D.3.10 Strength
    D.3.10.1Concentration unit mg/g milligram(s)/gram
    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 caratterizzata 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 evaluate the long-term safety,
    tolerability, and effect on intravascular hemolysis of REGN3918 in
    patients with paroxysmal nocturnal hemoglobinuria (PNH).
    L’obiettivo primario dello studio è valutare la sicurezza a lungo termine, la tollerabilità e l’effetto dell’emolisi intravascolare di REGN3918 in pazienti con emoglobinuria parossistica notturna (EPN).
    E.2.2Secondary objectives of the trial
    The secondary objectives of the study are:
    •To evaluate the long-term effect of REGN3918 on intravascular
    hemolysis
    •To assess the concentrations of total REGN3918 in serum
    •To evaluate the occurrence of the immunogenicity of REGN3918
    Gli obiettivi secondari dello studio consistono nel:
    • Valutare l’effetto a lungo termine di REGN3918 sull’emolisi intravascolare.
    • Valutare le concentrazioni totali di REGN3918 nel siero.
    • Valutare la comparsa di immunogenicità di REGN3918.
    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 . Participants will be required to sign separate sub-study ICFs before collection of samples. The purpose of the pharmacogenomic analyses is to identify genomic associations with clinical (safety or efficacy) or biomarker response to
    REGN3918, the complement pathway, PNH and related complement mediated diseases, clinical outcome measures, and possible AEs.

    Altre tipologie di sottostudi
    specificare il titolo, la data e la versione di ogni sottostudio con i relativi obiettivi: Ci sono sotto-studi di analisibiomediche future e farmacogenomiche facoltative . I partecipanti dovranno firmare un ICF separato per i sottostudi prima della raccolta di campioni.
    Lo scopo delle analisi farmacogenomiche è identificare l'associazione genomica con risposta clinica (sicurezza o efficacia) o biomarker a REGN3918, il pathway del complemento, PNH e le relative malattie mediate dal complemento, misure dell'esito clinico e possibili eventi avversi.
    E.3Principal inclusion criteria
    1. Patients with PNH who have completed, without discontinuation, study treatment in one of the parent studies in which they participated (either R3918-PNH-1852 or R3918-PNH-1853) Other protocol inclusion criteria apply
    1. Pazienti con EPN che hanno completato, senza interruzione, il trattamento di studio in uno degli studi principali a cui hanno partecipato (R3918-PNH-1852 o R3918-PNH-1853). Si applicano altri criteri di inclusione del protocollo
    E.4Principal exclusion criteria
    1. Significant protocol deviation(s) in the parent study based on the investigator's judgment and to the extent that these would (if continued) impact the study objectives and/or safety of the patient (for example, repetitive non-compliance with dosing by the patient)
    2. Any new condition or worsening of an existing condition which, in the opinion of the investigator, would make the patient unsuitable for enrollment or could interfere with the patient participating in or completing the study Other protocol defined exclusion criteria apply
    1. Deviazioni significative del protocollo nello studio principale in base al giudizio dello sperimentatore e nella misura in cui tali effetti (se continui) avrebbero un impatto sugli obiettivi dello studio e / o sulla sicurezza del paziente (ad esempio, la non conformità ripetitiva con la somministrazione del paziente)
    2. Qualsiasi nuova condizione o peggioramento di una condizione esistente che, secondo l'opinione dello sperimentatore, renderebbe il paziente inadatto all'arruolamento o potrebbe interferire con il paziente che partecipa o completa lo studio. Si applicano altri criteri di esclusione definiti dal protocollo
    E.5 End points
    E.5.1Primary end point(s)
    The primary safety endpoint is incidence and severity of treatmentemergent adverse events (TEAEs) up to week 104. TEAEs include adverse events (AEs), serious adverse events (SAEs), AEs of special interest (AESIs), laboratory data, vital signs, and electrocardiograms (ECGs)
    The primary efficacy endpoint is the proportion of patients achieving LDH =1.5×ULN up to week 26
    L'endpoint primario di sicurezza è l'incidenza e la gravità degli eventi avversi di trattamento (TEAE) fino alla settimana 104. I TEAE comprendono eventi avversi (EA), eventi avversi gravi (EA), eventi avversi di interesse speciale (EA), dati di laboratorio, segni vitali e elettrocardiogrammi (ECG)
    L'endpoint primario di efficacia è la percentuale di pazienti che raggiungono LDH = 1,5 × ULN fino alla settimana 26
    E.5.1.1Timepoint(s) of evaluation of this end point
    The primary efficacy endpoint is the proportion of patients achieving LDH
    =1.5×ULN up to week 26
    L'endpoint primario di efficacia è la percentuale di pazienti che raggiungono LDH = 1,5 × ULN fino alla settimana 26
    E.5.2Secondary end point(s)
    The secondary endpoints include:
    - Proportion of patients with breakthrough hemolysis up to week 26,
    78, and 104
    - Rate and number of units of transfusion up to week 26, 78, and 104
    - Proportions of patients who are transfusion-free (with Red blood cell
    [RBCs]) up to week 26, 78, and 104
    - Proportions of patients achieving adequate control of their
    intravascular
    hemolysis up to week 78 and 104
    - Proportions of patients achieving normalization of their intravascular
    hemolysis up to week 26, 78, and 104
    - Changes in LDH from baseline of the Open-label extension (OLE)
    study up to week 26, 78, and 104
    - Percent changes in LDH from baseline of the OLE study up to week 26,
    78, and 104
    - Changes in RBC hemoglobin levels from baseline of the OLE study up
    to week 26, 78, and 104
    - Changes in free hemoglobin levels from baseline of the OLE study up
    to week 26, 78, and 104
    - Concentrations of REGN3918 in serum up to week 104
    - Incidence of treatment-emergent anti-drug antibodies (ADA) to
    REGN3918 up to week 104
    Gli endpoint secondari includono:
    - Proporzione di pazienti con emolisi svolta fino alla settimana 26, 78 e 104
    - Velocità e numero di unità di trasfusione fino alla settimana 26, 78 e 104
    - Proporzioni di pazienti liberi da trasfusioni (con globuli rossi [RBCs]) fino alla settimana 26, 78 e 104
    - Proporzioni di pazienti che ottengono un controllo adeguato della loro intravascolare
    emolisi fino alla settimana 78 e 104
    - Proporzioni di pazienti che raggiungono la normalizzazione della loro emolisi intravascolare fino alla settimana 26, 78 e 104
    - Modifiche in LDH dal basale dello studio Open-label-Extention(OLE) studiare fino alla settimana 26, 78 e 104
    - Variazioni percentuali di LDH dal basale dello studio OLE fino alla settimana 26, 78 e 104
    - Cambiamenti nei livelli di emoglobina nei globuli rossi rispetto al basale dello studio OLE
    alla settimana 26, 78 e 104
    - Cambiamenti nei livelli di emoglobina libera dal basale dello studio OLE alla settimana 26, 78 e 104
    - Concentrazioni di REGN3918 nel siero fino alla settimana 104
    - Incidenza di anticorpi anti-farmaco (ADA) emergenti dal trattamento per REGN3918 fino alla settimana 104
    E.5.2.1Timepoint(s) of evaluation of this end point
    Please refer to E.5.2.
    Si prega di riferirsi alla sezione E5.2
    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) 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 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 Yes
    E.8.1.7.1Other trial design description
    Aperto
    open
    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, 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 years4
    E.8.9.1In the Member State concerned months0
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years4
    E.8.9.2In all countries concerned by the trial months0
    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: 111
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 36
    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 50
    F.4.2.2In the whole clinical trial 147
    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)
    none
    nessuno
    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 Decision2019-12-12
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2019-11-21
    P. End of Trial
    P.End of Trial StatusCompleted
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