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    Clinical Trial Results:
    EFFICACY AND SAFETY STUDY OF FACTANE 200 IU/ml ADMINISTERED BY CONTINUOUS INFUSION IN SEVERE HAEMOPHILIA A PATIENTS DURING MAJOR SURGICAL PROCEDURES

    Summary
    EudraCT number
    		 2010-023666-46
    Trial protocol
    		 PL  
    Global end of trial date
    31 Jul 2012

    Results information
    Results version number
    		 v1(current)
    This version publication date
    06 Jan 2017
    First version publication date
    06 Jan 2017
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    F8VR-1006
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 -
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    LFB Biotechnologies
    Sponsor organisation address
    3 Avenue des Tropiques, 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)
    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
    27 Feb 2013
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    31 Jul 2012
    Global end of trial reached?
    Yes
    Global end of trial date
    31 Jul 2012
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    To evaluate the efficacy of FACTANE 200 IU/ml administered by continuous infusion for the perioperative and postoperative prophylaxis of bleeding during major surgical procedures in severe haemophilia A patients.
    Protection of trial subjects
    LFB has developed a new concentration to improve patient comfort by supplying the product in a smaller volume: FACTANE 200 IU/mL contains approximately 1000 IU of factor VIII in 5 mL of solution. Continuous infusion, through a catheter placed in a vein in the arm, is also advantageous in that it avoids repeat injections and minimises pain and injury in the vein due to many needle punctures. 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
    12 Mar 2012
    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
    Poland: 4
    Worldwide total number of subjects
    4
    EEA total number of subjects
    4
    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)
    4
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 In total, four (4) subjects have been enrolled by in single center in Poland (Warsaw) between 12 March 2012 and 28 May 2012

    Pre-assignment
    Screening details
    		 -

    Pre-assignment period milestones
    Number of subjects started
    		 4
    Number of subjects completed
    		 4

    Period 1
    Period 1 title
    Preoperative pharmacokinetic study
    Is this the baseline period?
    		 Yes
    Allocation method
    Not applicable
    Blinding used
    		 Not blinded

    Arms
    Arm title
    		 FACTANE
    Arm description
    		 -
    Arm type
    		 Active comparator

    Investigational medicinal product name
    FACTANE
    Investigational medicinal product code
    F8VR
    Other name
    Pharmaceutical forms
    Solution for infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    Pharmacokinetic study before the surgical procedure: Single weight-adjusted dose (50 IU/kg) administered by bolus at a rate of 4mL/min

    Number of subjects in period 1
    		FACTANE
    Started
    4
    Completed
    4
    Period 2
    Period 2 title
    Surgery by continuous infusion
    Is this the baseline period?
    		 No
    Allocation method
    Not applicable
    Blinding used
    		 Not blinded

    Arms
    Arm title
    		 FACTANE
    Arm description
    		 -
    Arm type
    		 Experimental

    Investigational medicinal product name
    FACTANE
    Investigational medicinal product code
    F8VR
    Other name
    Pharmaceutical forms
    Solution for infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    - Preoperative loading dose administered by IV bolus at a rate of 4 mL/min. The loading dose was to be administered to reach circulating FVIII levels between 80 and 120 IU/dL according to the following formula: Loading dose (IU) = patient's weight (kg) × desired level (IU/dL) × 1 / patient's incremental recovery at T15 min ([IU/dL]/[IU/kg]). - Continuous infusion dose immediately after bolus injection: H0 (D0). The infusion rate was calculated based on the patient's clearance and the desired FVIII level as follows: Infusion rate (IU/kg/h) = clearance (mL/h/kg) × desired FVIII level (IU/mL). The desired FVIII levels were to be: 80 to 120 IU/dL from D0 to D6 and 30 to 80 IU/dL from D7 to the end of the continuous infusion (Dn).

    Number of subjects in period 2
    		FACTANE
    Started
    4
    Completed
    4
    Period 3
    Period 3 title
    Follow-up period
    Is this the baseline period?
    		 No
    Allocation method
    Not applicable
    Blinding used
    		 Not blinded

    Arms
    Arm title
    		 FACTANE
    Arm description
    		 -
    Arm type
    		 Active comparator

    Investigational medicinal product name
    Factor 8
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Solution for infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    after continuous infusion, the patient had to be treated with his usual factor VIII concentrate.

    Number of subjects in period 3
    		FACTANE
    Started
    4
    Completed
    4

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Preoperative pharmacokinetic study
    Reporting group description
    		 -

    Reporting group values
    		 Preoperative pharmacokinetic study Total
    Number of subjects
    		 4 4
    Age categorical
    Units: Subjects
        Adults (18-64 years)
    		 4 4
    Age continuous
    Units: years
        median (full range (min-max))
    		 39 (37 to 43) -
    Gender categorical
    Units: Subjects
        Female
    		 0 0
        Male
    		 4 4
    Subject analysis sets

    Subject analysis set title
    TTS
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    Total Treated Set

    Subject analysis sets values
    		 TTS
    Number of subjects
    4
    Age categorical
    Units: Subjects
        Adults (18-64 years)
    4
    Age continuous
    Units: years
        median (full range (min-max))
    39 (37 to 43)
    Gender categorical
    Units: Subjects
        Female
    0
        Male
    4

    End points

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

    Subject analysis set title
    TTS
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    Total Treated Set

    Primary: Hemostatic efficacy

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    End point title
    		 Hemostatic efficacy [1]
    End point description
    Efficacy was assessed based on the number of ‘responder’ patients. A patient was considered as a 'responder' if he obtained an 'excellent' or 'good' score in 3 successive haemostasis assessments: • 6 hours after the start of the surgery (by the surgeon), • on the day of drain tube removal (or on D2 if there was no drain) (by the surgeon), • on D7 (by the Investigator). Haemostasis was evaluated using the following scale: 'excellent', 'good', 'moderate', 'none'. - ‘excellent’: haemostasis similar to that of a non-bleeding disorder patient during the same surgery - ‘good’: slight oozing at surgical incision, - ‘moderate’: mild, but controlled bleeding, - ‘none’: severe, uncontrolled bleeding.
    End point type
    Primary
    End point timeframe
    Haemostatic efficacy was rated at 3 time-points after start of surgery (D0): H6, day of drain removal, D7
    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
    Number of subjects analysed
    4
    Units: number of 'responder' patients
        excellent/good
    4
        moderate/none
    0
    No statistical analyses for this end point

    Secondary: patient with inhibitor

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    End point title
    		 patient with inhibitor
    End point description
    End point type
    Secondary
    End point timeframe
    during the study
    End point values
    		 TTS
    Number of subjects analysed
    4
    Units: number
    0
    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
    15.0
    Reporting groups
    Reporting group title
    Total treated Set
    Reporting group description
    		 -

    Serious adverse events
    		 Total treated Set
    Total subjects affected by serious adverse events
         subjects affected / exposed
    2 / 4 (50.00%)
         number of deaths (all causes)
    0
         number of deaths resulting from adverse events
    0
    Injury, poisoning and procedural complications
    postoperative anaemia
         subjects affected / exposed
    1 / 4 (25.00%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Vascular disorders
    circulatory collapse
         subjects affected / exposed
    1 / 4 (25.00%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 Total treated Set
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    4 / 4 (100.00%)
    Injury, poisoning and procedural complications
    Anaemia postoperative
    Additional description: Not related
         subjects affected / exposed
    3 / 4 (75.00%)
         occurrences all number
    3
    Vascular disorders
    Haematoma
    Additional description: Note related
         subjects affected / exposed
    2 / 4 (50.00%)
         occurrences all number
    2
    General disorders and administration site conditions
    Catheter site inflammation
    Additional description: Not related
         subjects affected / exposed
    3 / 4 (75.00%)
         occurrences all number
    4
    Pyrexia
    Additional description: Not related
         subjects affected / exposed
    2 / 4 (50.00%)
         occurrences all number
    3
    Musculoskeletal and connective tissue disorders
    Haemarthrosis
    Additional description: Note related
         subjects affected / exposed
    4 / 4 (100.00%)
         occurrences all number
    14

    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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