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    Clinical Trial Results:
    Clinical pharmacology, efficacy and safety study of FGTW in paediatric patients with severe congenital fibrinogen deficiency

    Summary
    EudraCT number
    		 2013-000261-36
    Trial protocol
    		 FR   Outside EU/EEA  
    Global end of trial date
    11 Dec 2015

    Results information
    Results version number
    		 v1(current)
    This version publication date
    28 Sep 2016
    First version publication date
    28 Sep 2016
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    FGTW-1004
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT02094430
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    LFB Biotechnologies
    Sponsor organisation address
    3, Avenue des tropiques - BP 40305, COURTABOEUF, France, 91958
    Public contact
    Global Clinical Development Leader, LFB Biotechnologies, 33 169825656,
    Scientific contact
    Global Clinical Development Leader, LFB Biotechnologies, 33 169825656,
    Paediatric regulatory details
    Is trial part of an agreed paediatric investigation plan (PIP)
    Yes
    EMA paediatric investigation plan number(s)
    		 EMEA-000457-PIP02-10
    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
    29 Aug 2016
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    11 Dec 2015
    Global end of trial reached?
    Yes
    Global end of trial date
    11 Dec 2015
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    The primary objective of the study is to assess the efficacy of FGTW as a replacement therapy in paediatric patients with afibrinogenaemia or severe hypofibrinogenaemia: ▪ in preventing excessive bleeding in patients undergoing a surgical procedure, ▪ in treating bleeding of non-surgical origin
    Protection of trial subjects
    Blood analyses required by the study procedures will be adapted to the body weight of the patient to take into consideration the Ethical Considerations and ICH Harmonised Tripartite Guideline “Clinical Investigation of Medicinal Products in the Paediatric Population”. The protocol-related blood withdrawal will not exceed 3 % of the total blood volume of the patient during a period of four weeks and will not exceed 1% at any single time. The total volume of blood is estimated at 80 to 90 mL/kg body weight; 3% is 2.4 mL blood per kg of body weight. Patient confidentiality was maintained throughout the study. The study was conducted in accordance with the with the principles laid down in the Declaration of Helsinki, the ICH guidelines for Good Clinical Practice (GCP) and all applicable regulatory requirements.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    22 Jan 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
    Lebanon: 8
    Country: Number of subjects enrolled
    Turkey: 1
    Country: Number of subjects enrolled
    Morocco: 7
    Worldwide total number of subjects
    16
    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)
    1
    Children (2-11 years)
    14
    Adolescents (12-17 years)
    1
    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
    		 16 subjects were included in the TTS from 22/01/2014 to 11/12/2015 (LPO): 12 subjects participating in both PK and efficacy parts of the study and 4 subjects participating only in the efficacy part, all were analyzed for safety evaluation.

    Pre-assignment
    Screening details
    		 -

    Pre-assignment period milestones
    Number of subjects started
    		 16
    Number of subjects completed
    		 16

    Period 1
    Period 1 title
    Inclusion visit
    Is this the baseline period?
    		 Yes
    Allocation method
    Not applicable
    Blinding used
    		 Not blinded

    Arms
    Arm title
    		 Experimental
    Arm description
    		 -
    Arm type
    		 Experimental

    Investigational medicinal product name
    FibCLOT
    Investigational medicinal product code
    FGTW
    Other name
    Pharmaceutical forms
    Powder and solvent for solution for injection
    Routes of administration
    Intravenous use
    Dosage and administration details
    No administration at inclusion

    Number of subjects in period 1
    		Experimental
    Started
    16
    Completed
    16
    Period 2
    Period 2 title
    Pharmacology Study Period
    Is this the baseline period?
    		 No
    Allocation method
    Not applicable
    Blinding used
    		 Not blinded

    Arms
    Arm title
    		 Experimental
    Arm description
    		 -
    Arm type
    		 Experimental

    Investigational medicinal product name
    FibCLOT
    Investigational medicinal product code
    FGTW
    Other name
    Pharmaceutical forms
    Powder and solvent for solution for injection
    Routes of administration
    Intravenous use
    Dosage and administration details
    No administration at inclusion

    Number of subjects in period 2 [1]
    		Experimental
    Started
    12
    Completed
    12
    Notes
    [1] - The number of subjects starting the period is not consistent with the number completing the preceding period. It is expected the number of subjects starting the subsequent period will be the same as the number completing the preceding period.
    Justification: Pharmacokinetics at study entry was optional, and 12 of a total of 16 enrolled patients participated at PK study
    Period 3
    Period 3 title
    Efficacy Study Period
    Is this the baseline period?
    		 No
    Allocation method
    Not applicable
    Blinding used
    		 Not blinded

    Arms
    Arm title
    		 Experimental
    Arm description
    		 -
    Arm type
    		 Experimental

    Investigational medicinal product name
    FibCLOT
    Investigational medicinal product code
    FGTW
    Other name
    Pharmaceutical forms
    Powder and solvent for solution for injection
    Routes of administration
    Intravenous use
    Dosage and administration details
    No administration at inclusion

    Number of subjects in period 3
    		Experimental
    Started
    12
    Completed
    16
    Joined
    4
         patients without pharmacology period
    4

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Inclusion visit
    Reporting group description
    		 -

    Reporting group values
    		 Inclusion visit Total
    Number of subjects
    		 16 16
    Age categorical
    Units: Subjects
        Infants and toddlers (28 days-23 months)
    		 1 1
        Children (2-11 years)
    		 14 14
        Adolescents (12-17 years)
    		 1 1
    Age continuous
    Units: years
        median (full range (min-max))
    		 6.5 (1 to 12) -
    Gender categorical
    Units: Subjects
        Female
    		 6 6
        Male
    		 10 10

    End points

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    End points reporting groups
    Reporting group title
    Experimental
    Reporting group description
    		 -
    Reporting group title
    Experimental
    Reporting group description
    		 -
    Reporting group title
    Experimental
    Reporting group description
    		 -

    Subject analysis set title
    TTS (Total treated set) - Surgical procedures
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    the TTS was defined as all subjects who received at least one administration of FGTW for any part of the study protocol.

    Subject analysis set title
    TTS (Total treated Set) - Bleeding episodes
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    the TTS was defined as all subjects who received at least one administration of FGTW for any part of the study protocol.

    Primary: Proportion of Excellent/Good responses

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    End point title
    		 Proportion of Excellent/Good responses [1]
    End point description
    End point type
    Primary
    End point timeframe
    End point timeframe: Overall assessment of the hemostatic efficacy of FGTW done at least 6 hrs post-infusion for a bleeding episode/surgical procedure treated in an outpatient setting or on the day of hospital discharge when the subject was hospitalized
    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: Descriptive analysis
    End point values
    		 TTS (Total treated set) - Surgical procedures TTS (Total treated Set) - Bleeding episodes
    Number of subjects analysed
    10 [2]
    11 [3]
    Units: percentage
        Excellent/Good
    100
    97
        Moderate/None
    0
    3
    Notes
    [2] - 10 subjects underwent 11 surgical procedures. The statistical unit was the treated event with FGTW
    [3] - 11 subjects experienced 32 bleeding episodes. The statistical unit was the treated event with FGTW
    No statistical analyses for this end point

    Primary: Incremental Recovery

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    End point title
    		 Incremental Recovery [4]
    End point description
    End point type
    Primary
    End point timeframe
    Incremental Recovery one hour post-infusion for fibrinogen activity after single IV infusion 0.06 g/kg FGTW in subjects with afibrinogenaemia
    Notes
    [4] - 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: Descriptive analysis
    End point values
    		 Experimental
    Number of subjects analysed
    12
    Units: g/L per g/kg
        geometric mean (full range (min-max))
    19.1 (14 to 30.7)
    No statistical analyses for this end point

    Primary: Half-life

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    End point title
    		 Half-life [5]
    End point description
    End point type
    Primary
    End point timeframe
    Half-life of FGTW for fibrinogen activity after a single IV infusion of 0.06 g/kg FGTW in pediatric subjects of 12 years or less with afibrinogenaemia
    Notes
    [5] - 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: Descriptive analysis
    End point values
    		 Experimental
    Number of subjects analysed
    12
    Units: hours
        geometric mean (full range (min-max))
    49 (41.1 to 57.3)
    No statistical analyses for this end point

    Primary: Clearance

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    End point title
    		 Clearance [6]
    End point description
    End point type
    Primary
    End point timeframe
    Clearance of FGTW for fibrinogen activity after a single IV infusion of 0.06 g/kg FGTW in pediatric subjects of 12 years or less with afibrinogenaemia
    Notes
    [6] - 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: Descriptive analysis
    End point values
    		 Experimental
    Number of subjects analysed
    12
    Units: mL/h/kg
        geometric mean (full range (min-max))
    0.74 (0.47 to 1.03)
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Throughout the study
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    17.1
    Reporting groups
    Reporting group title
    Experimental
    Reporting group description
    		 -

    Serious adverse events
    		 Experimental
    Total subjects affected by serious adverse events
         subjects affected / exposed
    3 / 16 (18.75%)
         number of deaths (all causes)
    0
         number of deaths resulting from adverse events
    0
    Injury, poisoning and procedural complications
    Hand fracture
         subjects affected / exposed
    1 / 16 (6.25%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Hepatobiliary disorders
    Budd-Chiari syndrome
         subjects affected / exposed
    1 / 16 (6.25%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Musculoskeletal and connective tissue disorders
    Bone pain
         subjects affected / exposed
    1 / 16 (6.25%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 3%
    Non-serious adverse events
    		 Experimental
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    14 / 16 (87.50%)
    Injury, poisoning and procedural complications
    Limb injury
    Additional description: Not related to the study drug
         subjects affected / exposed
    8 / 16 (50.00%)
         occurrences all number
    20
    Joint injury
    Additional description: Not related to the study drug
         subjects affected / exposed
    4 / 16 (25.00%)
         occurrences all number
    5
    Limb crushing injury
    Additional description: Not related to the study drug
         subjects affected / exposed
    4 / 16 (25.00%)
         occurrences all number
    4
    Head injury
    Additional description: Not related to the study drug
         subjects affected / exposed
    3 / 16 (18.75%)
         occurrences all number
    5
    Buttock crushing
    Additional description: Not related to the study drug
         subjects affected / exposed
    3 / 16 (18.75%)
         occurrences all number
    4
    Nervous system disorders
    Headache
    Additional description: 2 adverse reactions in one subject
         subjects affected / exposed
    4 / 16 (25.00%)
         occurrences all number
    6
    Gastrointestinal disorders
    Abdominal pain
    Additional description: Not related to the study drug
         subjects affected / exposed
    2 / 16 (12.50%)
         occurrences all number
    4
    Musculoskeletal and connective tissue disorders
    Pain in extremity
    Additional description: Not related to the study drug
         subjects affected / exposed
    4 / 16 (25.00%)
         occurrences all number
    6
    Bone pain
    Additional description: Not related to the study drug
         subjects affected / exposed
    2 / 16 (12.50%)
         occurrences all number
    7
    Infections and infestations
    rhinitis
    Additional description: Not related to the study drug
         subjects affected / exposed
    5 / 16 (31.25%)
         occurrences all number
    10
    Tonsillitis
    Additional description: Not related to the study drug
         subjects affected / exposed
    3 / 16 (18.75%)
         occurrences all number
    7

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    23 Jul 2013
    The principal aim of this amendment was to summarise the changes between study protocol version 3.0 dated 06 May 2013 and study protocol version 5.0 dated 10 July 2013 made during the assessment of the clinical trial by the French Authority (ANSM).
    29 Sep 2014
    This amendment increased the number of enrolled subjects from 12 to 16. The definition of ‘’major bleeding in nonsurgical subjects’’ was revised to allow managing subjects in outpatient or inpatient setting. In previous protocol version, subjects were managed in inpatient setting only. The hospitalization was removed from the definition based on the current medical practice in fibrinogen congenital deficiency and the accurate definition from the ISTH Committee. The safety section “special rules for the study” was updated to capture any change in AE or SAE, in order to have as much information as possible on each AE or SAE.

    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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