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    Summary
    EudraCT Number:2013-004090-28
    Sponsor's Protocol Code Number:Fx-R-001-S1/B3461049
    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:2014-03-31
    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 number2013-004090-28
    A.3Full title of the trial
    TRANSTHYRETIN-ASSOCIATED AMYLOIDOSES OUTCOMES SURVEY
    (THAOS) – OPTIONAL BLOOD SAMPLE COLLECTION SUBSTUDY
    Sondaggio Sugli Esiti dell’Amiloidosi da Transtiretina (Transthyretin-Associated Amyloidosis Outcome Survey, THAOS) - Sottostudio per la Raccolta di Campioni di Sangue Opzionali
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    The purpose of this substudy is to describe the collection of blood
    samples to be used for development and validation of a biomarker assay.
    Lo scopo di questo sottostudio è di descrivere la raccolta di campioni di sangue da utilizzare per lo sviluppo e la validazione di un saggio sui biomarcatori.
    A.4.1Sponsor's protocol code numberFx-R-001-S1/B3461049
    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 SponsorFold RX Pharmaceuticals, a Pfizer Company
    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 supportPfizer, Inc
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationPfizer, Inc
    B.5.2Functional name of contact pointClinician
    B.5.3 Address:
    B.5.3.1Street AddressMS 8260-2606, Eastern Pt. Rd.
    B.5.3.2Town/ cityGroton, CT
    B.5.3.3Post code06340
    B.5.3.4CountryUnited States
    B.5.4Telephone number0108604411542
    B.5.5Fax number0108606865350
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP Role
    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 Information not present in EudraCT
    D.2.1.1.1Trade name Not applicable. No drug used.
    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.3.4Pharmaceutical form
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMP
    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) No
    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 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
    Transthyretin (TTR) a 127-amino acid, tetrameric protein, primarily
    synthesized in the liver, is a secreted protein present in the blood and cerebrospinal fluid and is a carrier of thyroxine and retinol-binding protein-retinol (vitamin A) complex. Transthyretinassociated
    amyloidoses are diseases caused by dissociation of the transthyretin
    tetramer into monomers, which misfold, ultimately forming amyloid deposits in various organs.
    Transtiretina (TTR) un acido ammino-127, proteina tetramerica, principalmente sintetizzata nel fegato, è una proteina secreta presente nel sangue e nel fluido cerebrospinale ed è un vettore di tiroxina e proteina-retinolo legame al retinolo (vitamina A) complesso.
    Amiloidosi da Transtiretina è una malattia causata dalla dissociazione del tetramero transthyretin in monomeri, che misfold, formando infine depositi di amiloide in vari organi..
    E.1.1.1Medical condition in easily understood language
    Transthyretin-associated amyloidoses are diseases caused by dissociation of the transthyretin tetramer into monomers, which misfold, ultimately forming amyloid deposits in various organs.
    Amiloidosi da Transtiretina è una malattia causata dalla dissociazione del tetramero transthyretin in monomeri, che misfold, formando infine depositi di amiloide in vari organi.
    E.1.1.2Therapeutic area Diseases [C] - Nervous System Diseases [C10]
    MedDRA Classification
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    The objective of this study is to collect blood samples which will be
    used to assist in development and validation of a biomarker assay for TTR amyloidoses.
    Lo scopo di questo sottostudio è di descrivere la raccolta di campioni di sangue da utilizzare per lo sviluppo e la validazione di un saggio sui biomarcatori.
    E.2.2Secondary objectives of the trial
    Not Applicable
    Non applicabile
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Subject has symptomatic ATTR disease with documented Val30Met,
    Glu89Gln,
    Phe64Leu, Thr60Ala, Ser50Arg, Ile107Val, Val20Ile, Ile68Leu, or
    Val122Ile
    mutation in the TTR gene.
    2. Must be able to be a participant in the THAOS registry and are either
    currently
    XML File Identifier: HMmrmo7rsZGmYHxB4NUacap1qIE=
    Page 7/14 21/01/2014
    enrolled or will be enrolled in THAOS prior to entry into this substudy.
    3. Subject provides written informed consent to participate in this
    substudy. Subject
    must have already provided written informed consent to participate in
    the THAOS
    registry.
    1. Il soggetto presenta malattia ATTR sintomatica con mutazione documentata di Val30Met, Glu89Gln, Phe64Leu, Thr60Ala, Ser50Arg, Ile107Val, Val20Ile, Ile68Leu, o Val122Ile nel gene TTR.

    2. Deve essere in grado di partecipare al registro THAOS ed è attualmente arruolato, o sarà arruolato, nel registro THAOS prima dell’ingresso in questo sottostudio.

    3. Il soggetto fornisce un consenso informato scritto per la partecipazione a questo sottostudio. Il soggetto deve aver già fornito il consenso informato scritto per la partecipazione al registro THAOS.
    E.4Principal exclusion criteria
    1. Subject has a diagnosis of primary or secondary amyloidosis.
    2. Subject has a documented mutation in the TTR gene other than
    Val30Met, Glu89Gln,
    Phe64Leu, Thr60Ala, Ser50Arg, Ile107Val, Val20Ile, Ile68Leu, or
    Val122Ile.
    3. Subject has received a liver transplant.
    4. Subject does not have symptomatic ATTR disease.
    5. Subject has received treatment with tafamidis prior to entry into this
    substudy.
    6. Subjects using non-steroidal anti-inflammatory drugs (NSAIDs) that
    are not
    permitted in the protocol within 2 weeks prior to blood collection.
     The following NSAIDs are permitted: acetylsalicylic acid, etodolac,
    ibuprofen,
    indomethicin, ketoprofen, nabumetone, naproxen, nimesulide,
    piroxicam, and
    sulindac.
    7. Subject received an investigational drug in another clinical
    investigational study
    within 30 days (or as determine by the local requirements, whichever
    is longer) or
    5 half-lives prior to substudy enrollment and blood collection.
    8. Participation in other studies (other than the THAOS survey) that
    include medication
    treatment within 6 months prior to substudy enrollment and blood
    collection.
    9. Subjects who are investigational site staff members directly involved
    in the conduct
    of the trial and their family members, site staff members otherwise
    supervised by the
    Investigator, or subjects who are Pfizer employees directly involved in
    the conduct
    1. Il soggetto presenta una diagnosi di amiloidosi primaria o secondaria.
    2. Il soggetto presenta una mutazione documentata del gene TTR diversa da Val30Met, Glu89Gln, Phe64Leu, Thr60Ala, Ser50Arg, Ile107Val, Val20Ile, Ile68Leu o Val122Ile.
    3. Il soggetto è stato sottoposto a trapianto di fegato.
    4. Il soggetto non è affetto da ATTR sintomatica.
    5. Il soggetto ha ricevuto un trattamento a base di tafamidis prima dell’ingresso nel presente sottostudio.
    6. I soggetti che utilizzano farmaci antinfiammatori non steroidei (FANS) non consentiti dal protocollo nelle 2 settimane precedenti al prelievo di sangue.
    - I FANS consentiti sono i seguenti: acido acetilsalicilico, etodolac, ibuprofene, indometacina, ketoprofene, nabumetone, naprossene, nimesulide, piroxicam e sulindac.

    7. Il soggetto ha ricevuto un farmaco sperimentale in un altro studio clinico sperimentale nei 30 giorni precedenti (o come determinato dai requisiti locali, il termine più lungo dei due) o 5 emivite prima dell’arruolamento al sottostudio e al prelievo di sangue.
    8. Partecipazione ad altri studi (diversi dal sondaggio THAOS) che prevedono il trattamento farmacologico nei 6 mesi che precedono l’arruolamento al sottostudio e al prelievo di sangue.
    9. Soggetti membri del personale del Centro di sperimentazione direttamente coinvolti nella conduzione della sperimentazione e membri delle rispettive famiglie, membri del personale del Centro altrimenti supervisionati dallo sperimentatore o soggetti dipendenti di Pfizer direttamente coinvolti nella conduzione della sperimentazione.
    E.5 End points
    E.5.1Primary end point(s)
    The objective of this study is to collect blood samples which will be
    used to assist in development and validation of a biomarker assay for TTR amyloidoses.
    L’obiettivo di questo studio è di raccogliere campioni di sangue da utilizzare per contribuire allo sviluppo e alla validazione di un saggio sui biomarcatori per le amiloidosi da TTR.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Samples will be retained until they are no longer needed for the
    development and validation of a biomarker assay of TTR.
    I campioni saranno conservati fino a quando non saranno più necessari per lo sviluppo e la validazione di un saggio sui biomarcatori per le amiloidosi da TTR.
    E.5.2Secondary end point(s)
    Not applicable
    Non applicabile
    E.5.2.1Timepoint(s) of evaluation of this end point
    Not applicable
    Non applicabile
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis Yes
    E.6.2Prophylaxis No
    E.6.3Therapy No
    E.6.4Safety No
    E.6.5Efficacy No
    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
    The purpose of this substudy is to describe the collection of blood
    samples to be used for development and validation of a biomarker assay.
    L’obiettivo di questo studio è di raccogliere campioni di sangue da utilizzare per contribuire allo sviluppo e alla validazione di un saggio sui biomarcatori per le amiloidosi da TTR.
    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) No
    E.7.4Therapeutic use (Phase IV) Yes
    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.8.3 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 No
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA No
    E.8.6.2Trial being conducted completely outside of the EEA Yes
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    Argentina
    Brazil
    United States
    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
    ultima visita dell'ultimo paziente
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years
    E.8.9.1In the Member State concerned months9
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years0
    E.8.9.2In all countries concerned by the trial months9
    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: 15
    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 No
    F.3.3.1Women of childbearing potential not using contraception No
    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.4.2.2In the whole clinical trial 45
    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)
    Not applicable. Normal Treatment.
    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 Decision2014-05-08
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2014-05-13
    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
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