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    Clinical Trial Results:
    A Prospective, Multicenter, Open Enrollment Study of Human Plasma-Derived Factor XIII Concentrate in Subjects With Congenital Factor XIII Deficiency

    Summary
    EudraCT number
    2014-003764-20
    Trial protocol
    Outside EU/EEA  
    Global end of trial date
    22 Aug 2011

    Results information
    Results version number
    v1(current)
    This version publication date
    13 Jul 2016
    First version publication date
    06 Aug 2015
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    BI71023_3002
    Additional study identifiers
    ISRCTN number
    -
    US NCT number
    NCT00945906
    WHO universal trial number (UTN)
    -
    Sponsors
    Sponsor organisation name
    CSL Behring LLC
    Sponsor organisation address
    1020 First Avenue, King of Prussia, United States, 19406-0901
    Public contact
    Clinical Trial Disclosure Manager, CSL Behring, clinicaltrials@cslbehring.com
    Scientific contact
    Clinical Trial Disclosure Manager, CSL Behring, clinicaltrials@cslbehring.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?
    Yes
    Results analysis stage
    Analysis stage
    Final
    Date of interim/final analysis
    20 Dec 2011
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    22 Aug 2011
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    The objectives of the study are to provide FXIII Concentrate (Human) to patients in the United States until the product becomes commercially available in the United States as well as to collect additional long-term safety data in this population.
    Protection of trial subjects
    This study was carried out in accordance with the International Conference on Harmonisation (ICH) Good Clinical Practice guidelines, and standard operating procedures for clinical research and development at CSL Behring (CSLB). The study protocol and all amendments were approved by the Independent Ethics Committee(s) (IECs) / Institutional Review Board(s) (IRBs) of the participating centers. Before undergoing screening procedures for possible enrollment into the study, subjects were informed, in an understandable form, about the nature, scope, and possible consequences of the study. The investigator was responsible for obtaining a subject’s written informed consent to participate in the study.
    Background therapy
    -
    Evidence for comparator
    -
    Actual start date of recruitment
    18 Sep 2009
    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: 61
    Worldwide total number of subjects
    61
    EEA total number of subjects
    0
    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)
    4
    Children (2-11 years)
    15
    Adolescents (12-17 years)
    15
    Adults (18-64 years)
    27
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    Subjects who were enrolled in pharmacokinetic (PK) Study BI71023_2002 or Phase 3b Study B171023_3001 were offered enrollment in this study. Enrollment was also offered to subjects who were enrolled in a clinical study conducted under BB-IND 5986 or not currently participating in any other study.

    Pre-assignment
    Screening details
    Eligible subjects were males or females of any age with documented congenital Factor XIII deficiency that required prophylactic treatment with a Factor XIII-containing product. Sixty-one subjects were screened, enrolled, and received at least 1 dose of Factor XIII Concentrate (Human) in the study.

    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
    Factor XIII
    Arm description
    Subjects were administered FXIII Concentrate (Human) by intravenous (IV) infusion approximately every 28 days to maintain a trough FXIII level of approximately 5 to 20%.
    Arm type
    Experimental

    Investigational medicinal product name
    Factor XIII Concentrate (Human)
    Investigational medicinal product code
    BI71023
    Other name
    Cluvot, Fibrogammin®, Corifact®
    Pharmaceutical forms
    Powder and solvent for solution for injection/infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    Subjects who did not complete PK Study BI71023_2002 or receive at least 3 doses of the investigational medicinal product in the Phase 3b Study BI71023_3001 received Factor XIII Concentrate (Human) at a dose of 40 U/kg by intravenous infusion. Subjects who were enrolled in a clinical study conducted under BB-IND 5986 received Factor XIII Concentrate at a dose of 40 U/kg by intravenous infusion. For all other subjects and all doses after Baseline (Day 0), the dose was guided by the individual subject’s most recent (pre-infusion) trough Factor XIII activity levels, with the objective of dosing every 28 days (4 weeks) to maintain a trough Factor XIII activity level of approximately 5 to 20%.

    Number of subjects in period 1
    Factor XIII
    Started
    61
    Completed
    54
    Not completed
    7
         Consent withdrawn by subject
    1
         'Did not want to return for last visit '
    2
         Moved to another country
    2
         Unable to return for last visit
    2

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Factor XIII
    Reporting group description
    Subjects were administered FXIII Concentrate (Human) by intravenous (IV) infusion approximately every 28 days to maintain a trough FXIII level of approximately 5 to 20%.

    Reporting group values
    Factor XIII Total
    Number of subjects
    61 61
    Age categorical
    Units: Subjects
        < 16 years
    29 29
        16 to <65 years
    32 32
        >= 65 years
    0 0
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    18.5 ( 12.48 ) -
    Gender categorical
    Units: Subjects
        Female
    26 26
        Male
    35 35

    End points

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    End points reporting groups
    Reporting group title
    Factor XIII
    Reporting group description
    Subjects were administered FXIII Concentrate (Human) by intravenous (IV) infusion approximately every 28 days to maintain a trough FXIII level of approximately 5 to 20%.

    Primary: Adverse Events

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    End point title
    Adverse Events [1]
    End point description
    Number of subjects with any treatment-emergent adverse event (AE), treatment-related AE or serious AE (SAE). Treatment-related AEs are defined as AEs whose relationship to treatment is related, or possibly related and AEs with missing relationship.
    End point type
    Primary
    End point timeframe
    After the first infusion until study completion. Study completion is up to 2 years or until Factor XIII Concentrate (Human) is commercially available in the USA.
    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: Statistical analyses were not performed in this study.
    End point values
    Factor XIII
    Number of subjects analysed
    61
    Units: subjects
        Any treatment-emergent AE
    42
        Treatment-emergent and related AE
    2
        Serious AE
    2
    No statistical analyses for this end point

    Secondary: Hematology and Chemistry Testing

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    End point title
    Hematology and Chemistry Testing
    End point description
    Number of participants with treatment-emergent clinically significant hematology and/or chemistry laboratory parameter values.
    End point type
    Secondary
    End point timeframe
    After the first infusion and at the end-of-study (or withdrawal) visit.
    End point values
    Factor XIII
    Number of subjects analysed
    61
    Units: subjects
        Clinically significant hematology test result
    1
        Clinically significant chemistry test result
    1
    No statistical analyses for this end point

    Secondary: Factor XIII Antibody Testing

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    End point title
    Factor XIII Antibody Testing
    End point description
    Number of participants with serum Factor XIII antibodies.
    End point type
    Secondary
    End point timeframe
    Before the first infusion, then every 48 weeks, at the end-of-study (or withdrawal) visit and after a bleeding episode requiring treatment with a Factor XIII -containing product.
    End point values
    Factor XIII
    Number of subjects analysed
    61
    Units: subjects
        Subjects with Factor XIII antibodies
    1
        Subjects without Factor XIII antibodies
    60
    No statistical analyses for this end point

    Secondary: Factor XIII Concentration

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    End point title
    Factor XIII Concentration
    End point description
    Trough Factor XIII concentration.
    End point type
    Secondary
    End point timeframe
    Before the first infusion, at 24 and 48 weeks after the first infusion, and at the end-of-study (or withdrawal) visit.
    End point values
    Factor XIII
    Number of subjects analysed
    61
    Units: IU/mL
    arithmetic mean (standard deviation)
        Baseline (n=35)
    0.0987 ( 0.03695 )
        Week 4 (n = 28)
    0.1177 ( 0.03484 )
        Week 8 (n = 8)
    0.1238 ( 0.02973 )
        Week 12 (n = 8)
    0.1075 ( 0.03196 )
        Week 16 (n = 4)
    0.1025 ( 0.04573 )
        Week 20 (n = 5)
    0.116 ( 0.03578 )
        Week 24 (n = 41)
    0.1341 ( 0.03346 )
        Week 28 (n = 3)
    0.1667 ( 0.06658 )
        Week 32 (n = 2)
    0.095 ( 0.02121 )
        Week 36 (n = 3)
    0.1083 ( 0.07489 )
        Week 40 (n = 3)
    0.0933 ( 0.04509 )
        Week 44 (n = 1)
    0.05 ( 0 )
        Week 48 (n = 13)
    0.1246 ( 0.02961 )
        Week 72 (n = 2)
    0.14 ( 0.01414 )
    No statistical analyses for this end point

    Secondary: Number of Subjects With at Least One Bleeding Episode

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    End point title
    Number of Subjects With at Least One Bleeding Episode
    End point description
    Number of subjects with at least one bleeding episode at any time after the first infusion in the study, and the number of subjects with at least one bleeding episode requiring Factor XIII treatment.
    End point type
    Secondary
    End point timeframe
    After the first infusion until study completion. Study completion is up to 2 years or until Factor XIII Concentrate (Human) is commercially available in the USA.
    End point values
    Factor XIII
    Number of subjects analysed
    61
    Units: subjects
        At least one bleeding episode (after treatment)
    10
        At least one bleeding episode requiring treatment
    1
    No statistical analyses for this end point

    Secondary: Total Number of Bleeding Episodes

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    End point title
    Total Number of Bleeding Episodes
    End point description
    Number of bleeding episodes at any time after the first infusion in the study.
    End point type
    Secondary
    End point timeframe
    After the first infusion until study completion. Study completion is up to 2 years or until Factor XIII Concentrate (Human) is commercially available in the USA.
    End point values
    Factor XIII
    Number of subjects analysed
    61
    Units: Bleeding episodes
    14
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    After the first infusion until study completion. Study completion is up to 2 years or until Factor XIII Concentrate (Human) is commercially available in the USA.
    Assessment type
    Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    MedDRA
    Dictionary version
    12.0
    Reporting groups
    Reporting group title
    Factor XIII
    Reporting group description
    Subjects were administered FXIII Concentrate (Human) by intravenous (IV) infusion approximately every 28 days to maintain a trough FXIII level of approximately 5 to 20%.

    Serious adverse events
    Factor XIII
    Total subjects affected by serious adverse events
         subjects affected / exposed
    2 / 61 (3.28%)
         number of deaths (all causes)
    0
         number of deaths resulting from adverse events
    0
    Blood and lymphatic system disorders
    Factor XIII inhibition
         subjects affected / exposed
    1 / 61 (1.64%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    General disorders and administration site conditions
    Fever
         subjects affected / exposed
    1 / 61 (1.64%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Infections and infestations
    Pelvic inflammatory disease
         subjects affected / exposed
    1 / 61 (1.64%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Pyelonephritis
         subjects affected / exposed
    1 / 61 (1.64%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 4%
    Non-serious adverse events
    Factor XIII
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    19 / 61 (31.15%)
    Injury, poisoning and procedural complications
    Bruising
         subjects affected / exposed
    4 / 61 (6.56%)
         occurrences all number
    5
    Nervous system disorders
    Headache
         subjects affected / exposed
    4 / 61 (6.56%)
         occurrences all number
    4
    General disorders and administration site conditions
    Fever
         subjects affected / exposed
    6 / 61 (9.84%)
         occurrences all number
    7
    Gastrointestinal disorders
    Vomiting
         subjects affected / exposed
    3 / 61 (4.92%)
         occurrences all number
    4
    Respiratory, thoracic and mediastinal disorders
    Cough
         subjects affected / exposed
    5 / 61 (8.20%)
         occurrences all number
    5
    Infections and infestations
    Upper respiratory infection
         subjects affected / exposed
    4 / 61 (6.56%)
         occurrences all number
    5

    More information

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    Substantial protocol amendments (globally)

    Were there any global substantial amendments to the protocol? No

    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
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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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