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    Clinical Trial Results:
    A Phase 3b Trial Investigating the Pharmacokinetics and Safety Profile of a Single Intravenous Dose of rFXIII in Paediatric (1 to less than 6 Years Old) Subjects with Congenital FXIII A-subunit Deficiency

    Summary
    EudraCT number
    		 2009-016869-28
    Trial protocol
    		 GB  
    Global end of trial date
    06 Jan 2012

    Results information
    Results version number
    		 v1(current)
    This version publication date
    15 Mar 2016
    First version publication date
    24 Jul 2015
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    F13CD-3760 (mentor™4)
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT01230021
    WHO universal trial number (UTN)
    		 U1111-1116-2533
    Sponsors
    Sponsor organisation name
    Novo Nordisk A/S
    Sponsor organisation address
    NOVO ALLE, Bagsvaerd, Denmark, 2880
    Public contact
    Global Clinical Registry, Novo Nordisk A/S, clinicaltrials@novonordisk.com
    Scientific contact
    Global Clinical Registry, Novo Nordisk A/S, clinicaltrials@novonordisk.com
    Paediatric regulatory details
    Is trial part of an agreed paediatric investigation plan (PIP)
    Yes
    EMA paediatric investigation plan number(s)
    		 EMEA-000185-PIP01-08
    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
    14 May 2012
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    06 Jan 2012
    Global end of trial reached?
    Yes
    Global end of trial date
    06 Jan 2012
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    To characterise the pharmacokinetics of rFXIII in paediatric subjects (1 to less than 6 years old) with congenital FXIII A-subunit deficiency following a single intravenous dose administration by measuring the area under the concentration vs. time curve (AUC0-30 Days) (IU×h/mL)
    Protection of trial subjects
    The trial was conducted in accordance with the Declaration of Helsinki and ICH Good Clinical Practice.
    Background therapy
    		 Monthly prophylactic treatment with a FXIII containing product (Fibrogammin®P/Corifact®) prior to inclusion in the trial
    Evidence for comparator
    		 Not applicable
    Actual start date of recruitment
    09 Nov 2010
    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 Kingdom: 3
    Country: Number of subjects enrolled
    United States: 2
    Country: Number of subjects enrolled
    Israel: 1
    Worldwide total number of subjects
    6
    EEA total number of subjects
    3
    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)
    1
    Children (2-11 years)
    5
    Adolescents (12-17 years)
    0
    Adults (18-64 years)
    0
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 Three countries were included in this trial−Israel(1 site), United Kingdom( 2 sites ) and the United States (2 sites).

    Pre-assignment
    Screening details
    		 Between screening and treatment with trial drug the children were assessed for eligibility. If eligible, the children were treated with one single dose of FXIII. The trial was not randomised.

    Period 1
    Period 1 title
    Overall period
    Is this the baseline period?
    		 Yes
    Allocation method
    Non-randomised - controlled
    Blinding used
    		 Not blinded
    Blinding implementation details
    Not Applicable

    Arms
    Arm title
    		 Recombinant factor XIII
    Arm description
    		 One single dose of 35 IU/kg rFXIII was administered as an intravenous (i.v.) injection to each child. The trial included one screening visit, one treatment visit (including pharmacokinetics (PK) assessments up to 24 hours) and 4 follow-up visits (at 7, 14, 21 and 30 days after dosing). Blood samples for PK assessments were drawn pre-dose and up to 30 days after dosing.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Recombinant Factor XIII (rFXIII)
    Investigational medicinal product code
    NN1841
    Other name
    Recombinant Factor XIII (rFXIII)
    Pharmaceutical forms
    Powder and solvent for solution for injection
    Routes of administration
    Intravenous use
    Dosage and administration details
    Intravenous injection of a single dose of recombinant factor XIII, 35 IU/kg bodyweight. The reconstituted and diluted rFXIII was given as a slow i.v. injection at a rate not exceeding 1−2 mL/min.

    Number of subjects in period 1
    		Recombinant factor XIII
    Started
    6
    Completed
    6

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Overall period
    Reporting group description
    		 One single dose of 35 IU/kg rFXIII was administered as an intravenous (i.v.) injection to each child. The trial included one screening visit, one treatment visit (including pharmacokinetics (PK) assessments up to 24 hours) and 4 follow-up visits (at 7, 14, 21 and 30 days after dosing). Blood samples for PK assessments were drawn pre-dose and up to 30 days after dosing

    Reporting group values
    		 Overall period Total
    Number of subjects
    		 6 6
    Age categorical
    Units: Subjects
        In utero
    		 0 0
        Preterm newborn infants (gestational age < 37 wks)
    		 0 0
        Newborns (0-27 days)
    		 0 0
        Infants and toddlers (28 days-23 months)
    		 1 1
        Children (2-11 years)
    		 5 5
        Adolescents (12-17 years)
    		 0 0
        Adults (18-64 years)
    		 0 0
        From 65-84 years
    		 0 0
        85 years and over
    		 0 0
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    		 2.67 ± 1.03 -
    Gender categorical
    Units: Subjects
        Female
    		 3 3
        Male
    		 3 3
    Ethnicity
    Units: Subjects
        Hispanic or Latino
    		 0 0
        Not Hispanic or Latino
    		 6 6
        Unknown or not reported
    		 0 0
    Race (NIH/OMB)
    Units: Subjects
        American Indian or Alaska Native
    		 0 0
        Asian
    		 3 3
        Native Hawaiian or other Pacific Islander
    		 0 0
        Black or African American
    		 1 1
        White
    		 2 2
        More than one race
    		 0 0
        Unknown or Not Reported
    		 0 0

    End points

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    End points reporting groups
    Reporting group title
    Recombinant factor XIII
    Reporting group description
    		 One single dose of 35 IU/kg rFXIII was administered as an intravenous (i.v.) injection to each child. The trial included one screening visit, one treatment visit (including pharmacokinetics (PK) assessments up to 24 hours) and 4 follow-up visits (at 7, 14, 21 and 30 days after dosing). Blood samples for PK assessments were drawn pre-dose and up to 30 days after dosing.

    Primary: Area Under the Concentration vs. Time Curve (AUC)

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    End point title
    		 Area Under the Concentration vs. Time Curve (AUC) [1]
    End point description
    A measure of the exposure. Blood samples for the PK assessment were drawn pre-dose and up to 30 days after dosing. The PK of FXIII in children was assessed after a single i.v. dose of rFXIII 35 IU/kg.
    End point type
    Primary
    End point timeframe
    At pre-dose, 30 minutes, 24 hours, 7, 14, 21 and 30 days after dosing
    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: There were no statistical analysis for this endpoint.The evaluation of the endpoint was descriptive through summary and listings.
    End point values
    		 Recombinant factor XIII
    Number of subjects analysed
    6
    Units: IU*h/mL
        arithmetic mean (standard deviation)
    250.25 ± 31.19
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Adverse events were collected and reported during the entire trial period; that is from day 0 (the treatment day) to day 30 (last follow-up visit).
    Adverse event reporting additional description
    Safety analysis population including all 6 children exposed to FXIII. All 6 children were exposed to one dose of trial product.
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    14.1
    Reporting groups
    Reporting group title
    Recombinant Factor XIII
    Reporting group description
    		 One single dose of 35 IU/kg rFXIII was administered as an intravenous (i.v.) injection to each child. The trial included one screening visit, one treatment visit (including pharmacokinetics (PK) assessments up to 24 hours) and 4 follow-up visits (at 7, 14, 21 and 30 days after dosing). Blood samples for PK assessments were drawn pre-dose and up to 30 days after dosing

    Serious adverse events
    		 Recombinant Factor XIII
    Total subjects affected by serious adverse events
         subjects affected / exposed
    0 / 6 (0.00%)
         number of deaths (all causes)
    0
         number of deaths resulting from adverse events
    0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 Recombinant Factor XIII
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    2 / 6 (33.33%)
    General disorders and administration site conditions
    Pyrexia
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Musculoskeletal and connective tissue disorders
    Pain in extremity
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    13 Apr 2011
    In this substantial protocol amendment: The title of the trial has been changed to include the name MentorTM 4. The name MentorTM 4 has been assigned to the trial to enhance recollection of the trial by accompanying the trial impact number with a name reference. In addition, a word mark will be placed on the front page.The protocol specifies a number of laboratory parameters to be assessed during the trial. The local laboratories for this trial operate according to standard operating procedures for each type of laboratory assessment. As a result of these standard procedures the local laboratories may provide analyses not requested in the protocol but produced in connection with the requested analyses. Such data will not be transferred to the trial database, but may be reported to the investigator according to specifications in the laboratory standard operating procedures and requirements. The investigator must review all laboratory results for concomitant illnesses and adverse events and report these according to the protocol. Consequently this discrepancy compromises neither subject safety, nor the safety reporting in the trial. This amendment will allow for these additional analyses to be reported to the investigator by the local laboratories. The text in Appendix C (Whole blood volume rule) regarding prioritization of blood samples has been deleted as this text is only applicable for the NN1841-3835 trial (safety extension trial to F13CD-3760 ) where pre- and post-dose blood samples are taken.

    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

    Online references
    http://www.ncbi.nlm.nih.gov/pubmed/23834599
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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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