Flag of the European Union EU Clinical Trials Register Help

Clinical trials

The European Union Clinical Trials Register allows you to search for protocol and results information on:
  • interventional clinical trials that are conducted in the European Union (EU) and the European Economic Area (EEA);
  • clinical trials conducted outside the EU / EEA that are linked to European paediatric-medicine development.
  • Learn   more about the EU Clinical Trials Register   including the source of the information and the legal basis.


    The EU Clinical Trials Register currently displays   39233   clinical trials with a EudraCT protocol, of which   6427   are clinical trials conducted with subjects less than 18 years old.
    The register also displays information on   18700   older paediatric trials (in scope of Article 45 of the Paediatric Regulation (EC) No 1901/2006).
     
    Examples: Cancer AND drug name. Pneumonia AND sponsor name.
    How to search [pdf]
    Search Tips: Under advanced search you can use filters for Country, Age Group, Gender, Trial Phase, Trial Status, Date Range, Rare Diseases and Orphan Designation. For these items you should use the filters and not add them to your search terms in the text field.
    Advanced Search: Search tools
     

    < Back to search results

    Download PDF

    Clinical Trial Results:
    Transthyretin-Associated Amyloidosis Outcomes Survey (THAOS) – Optional Blood Sample Collection Sub-study.

    Summary
    EudraCT number
    2013-004090-28
    Trial protocol
    IT  
    Global end of trial date
    30 Apr 2015

    Results information
    Results version number
    v1(current)
    This version publication date
    14 May 2016
    First version publication date
    14 May 2016
    Other versions
    Summary report(s)
    B3461049 - Public Disclosure Synopsis

    Trial information

    Close Top of page
    Trial identification
    Sponsor protocol code
    B3461049 (Fx-R-001-S1)
    Additional study identifiers
    ISRCTN number
    -
    US NCT number
    -
    WHO universal trial number (UTN)
    -
    Sponsors
    Sponsor organisation name
    Pfizer Inc.
    Sponsor organisation address
    235 E 42nd Street, New York, United States, NY 10017
    Public contact
    Pfizer ClinicalTrials.gov Call Center, Pfizer Inc., 001 8007181021, ClinicalTrials.gov_Inquiries@pfizer.com
    Scientific contact
    Pfizer ClinicalTrials.gov Call Center, Pfizer Inc., 001 8007181021, ClinicalTrials.gov_Inquiries@pfizer.com
    Paediatric regulatory details
    Is trial part of an agreed paediatric investigation plan (PIP)
    No
    Does article 45 of REGULATION (EC) No 1901/2006 apply to this trial?
    No
    Does article 46 of REGULATION (EC) No 1901/2006 apply to this trial?
    No
    Results analysis stage
    Analysis stage
    Final
    Date of interim/final analysis
    15 Apr 2016
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    30 Apr 2015
    Global end of trial reached?
    Yes
    Global end of trial date
    30 Apr 2015
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    The objective of this study was to collect blood samples planned to assist in the development and validation of a biomarker assay for transthyretin (TTR) amyloidoses.
    Protection of trial subjects
    The study was in compliance with the ethical principles derived from the Declaration of Helsinki and in compliance with all International Council on Harmonization (ICH) Good Clinical Practice (GCP) Guidelines. All the local regulatory requirements pertinent to safety of trial subjects were followed.
    Background therapy
    -
    Evidence for comparator
    -
    Actual start date of recruitment
    06 Aug 2014
    Long term follow-up planned
    No
    Independent data monitoring committee (IDMC) involvement?
    No
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    United States: 2
    Country: Number of subjects enrolled
    Argentina: 5
    Country: Number of subjects enrolled
    Germany: 3
    Country: Number of subjects enrolled
    Italy: 2
    Country: Number of subjects enrolled
    Mexico: 5
    Country: Number of subjects enrolled
    Portugal: 15
    Worldwide total number of subjects
    32
    EEA total number of subjects
    20
    Number of subjects enrolled per age group
    In utero
    0
    Preterm newborn - gestational age < 37 wk
    0
    Newborns (0-27 days)
    0
    Infants and toddlers (28 days-23 months)
    0
    Children (2-11 years)
    0
    Adolescents (12-17 years)
    0
    Adults (18-64 years)
    29
    From 65 to 84 years
    3
    85 years and over
    0

    Subject disposition

    Close Top of page
    Recruitment
    Recruitment details
    -

    Pre-assignment
    Screening details
    This sub study of THAOS was a protocol to collect blood samples from enrolled subjects who had Val30Met, Glu89Gln, Phe64Leu, Thr60Ala, Ser50Arg, Ile107Val, Val20Ile, Ile68Leu, Val122Ile mutation, symptomatic ATTR disease, not treated with any medication which stabilizes or reduces the concentration of TTR,TTR monomer or oligomer in blood.

    Period 1
    Period 1 title
    Overall Study (overall period)
    Is this the baseline period?
    Yes
    Allocation method
    Not applicable
    Blinding used
    Not blinded

    Arms
    Arm title
    Entire Study Population
    Arm description
    Subject who had symptomatic transthyretin amyloidosis (ATTR) disease with documented Val30Met, Glu89Gln, Phe64Leu, Thr60Ala, Ser50Arg, Ile107Val, Val20Ile, Ile68Leu, or Val122Ile mutation in the TTR protein and were a part of THAOS were enrolled into this sub-study.
    Arm type
    Blood Sample Collection Substudy

    Investigational medicinal product name
    None
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Capsule
    Routes of administration
    Unknown use
    Dosage and administration details
    No investigational medicinal product was administered in this study.

    Number of subjects in period 1
    Entire Study Population
    Started
    32
    Completed
    32

    Baseline characteristics

    Close Top of page
    Baseline characteristics reporting groups
    Reporting group title
    Overall Study
    Reporting group description
    -

    Reporting group values
    Overall Study Total
    Number of subjects
    32 32
    Age categorical
    Units: Subjects
        Adults (18-64 years)
    29 29
        From 65-84 years
    3 3
        85 years and over
    0 0
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    49.23 ± 14.3 -
    Gender categorical
    Units: Subjects
        Female
    20 20
        Male
    12 12

    End points

    Close Top of page
    End points reporting groups
    Reporting group title
    Entire Study Population
    Reporting group description
    Subject who had symptomatic transthyretin amyloidosis (ATTR) disease with documented Val30Met, Glu89Gln, Phe64Leu, Thr60Ala, Ser50Arg, Ile107Val, Val20Ile, Ile68Leu, or Val122Ile mutation in the TTR protein and were a part of THAOS were enrolled into this sub-study.

    Primary: Number of Subjects With Their Blood Samples Collected To Assist in Development and Validation of Biomarker Assay for Transthyretin (TTR) Amyloidoses

    Close Top of page
    End point title
    Number of Subjects With Their Blood Samples Collected To Assist in Development and Validation of Biomarker Assay for Transthyretin (TTR) Amyloidoses [1]
    End point description
    Blood was collected from subjects who had the following mutations in the TTR protein: Val30Met, Glu89Gln, Thr60Ala, Ser50Arg, Val20Ile, and Val122Ile, had symptomatic ATTR disease, and had not been treated with any medication or treatment which stabilizes or reduces the concentration of TTR, TTR monomer, or TTR oligomer in the blood. These blood samples were collected to assist in the development and validation of a biomarker assay for TTR amyloidosis. Entire study population was analyzed.
    End point type
    Primary
    End point timeframe
    Baseline up to 268 days
    Notes
    [1] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: No statistical analysis was planned or conducted for this substudy.
    End point values
    Entire Study Population
    Number of subjects analysed
    32
    Units: Subjects
    32
    No statistical analyses for this end point

    Adverse events

    Close Top of page
    Adverse events information [1]
    Timeframe for reporting adverse events
    Time of consent up to 28 days after the last visit
    Assessment type
    Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    No dictionary used
    Dictionary version
    N/A
    Reporting groups
    Reporting group title
    Entire Study Population
    Reporting group description
    Subject who had symptomatic transthyretin amyloidosis (ATTR) disease with documented Val30Met, Glu89Gln, Phe64Leu, Thr60Ala, Ser50Arg, Ile107Val, Val20Ile, Ile68Leu, or Val122Ile mutation in the TTR protein and were a part of THAOS were enrolled into this sub-study.

    Serious adverse events
    Entire Study Population
    Total subjects affected by serious adverse events
         subjects affected / exposed
    0 / 32 (0.00%)
         number of deaths (all causes)
    0
         number of deaths resulting from adverse events
    Frequency threshold for reporting non-serious adverse events: 0%
    Non-serious adverse events
    Entire Study Population
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    0 / 32 (0.00%)
    Notes
    [1] - There are no non-serious adverse events recorded for these results. It is expected that there will be at least one non-serious adverse event reported.
    Justification: This was a blood draw study in which no study drug was administered and there were no adverse events and no safety issues reported.

    More information

    Close Top of page

    Substantial protocol amendments (globally)

    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    30 Aug 2013
    The protocol was updated to limit scope of blood collection for the purpose of development and validation of a biomarker assay and to remove the storage of samples for Biobanking, The amendment updated the sponsor name as FoldRx Pharmaceuticals, Inc had become a wholly-owned subsidiary of Pfizer, Inc. Pfizer protocol study number (B3461049) was inserted and protocol sections were renumbered to align with Pfizer protocol standards. Inclusion criteria and exclusion criteria were added to further clarify the substudy population. Language was added to clarify sample size determination and statistical analysis and to define study discontinuation criteria. There was a change in nomenclature whereby the term “patient” became “subject” and a change in the abbreviation ATTR-PN to TTR-FAP.

    Interruptions (globally)

    Were there any global interruptions to the trial? No

    Limitations and caveats

    Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data.
    None reported
    The status of studies in GB is no longer updated from 1.1.2021
    For the UK, as from 1.1.2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI
    EU Clinical Trials Register Service Desk: https://servicedesk.ema.europa.eu
    European Medicines Agency © 1995-2021 | Domenico Scarlattilaan 6, 1083 HS Amsterdam, The Netherlands
    Legal notice
    EMA HMA