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    Clinical Trial Results:
    EFFICACY AND SAFETY STUDY OF WILFACTIN ADMINISTERED BY CONTINUOUS INFUSION IN PATIENTS WITH SEVERE VON WILLEBRAND DISEASE UNDERGOING MAJOR SURGICAL PROCEDURES

    Summary
    EudraCT number
    		 2007-004116-32
    Trial protocol
    		 FR   BE  
    Global end of trial date
    07 Mar 2013

    Results information
    Results version number
    		 v1(current)
    This version publication date
    30 Jun 2016
    First version publication date
    22 Jul 2015
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    Protocol WIL1-0609
    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 - LES ULIS, COURTABOEUF, France, 91930
    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
    03 Feb 2014
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    07 Mar 2013
    Global end of trial reached?
    Yes
    Global end of trial date
    07 Mar 2013
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    The primary objective is to evaluate the efficacy of continuous infusion of WILFACTIN for the prevention of perioperative bleeding in subjects with severe von Willebrand Disease undergoing elective major surgery.
    Protection of trial subjects
    Blood sampling usually done for laboratory testing presents a potential discomfort and the associated risks are slight pain at the site, feeling light-headed, bruising and, exceptionally, local infection as well as bleeding from the site of the puncture. However, all precautionary measures will be taken to minimizes potential side effects. 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.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    08 Dec 2008
    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: 2
    Country: Number of subjects enrolled
    France: 4
    Worldwide total number of subjects
    6
    EEA total number of subjects
    6
    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)
    5
    From 65 to 84 years
    1
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 In total, six (6) subjects have been enrolled by in 3 centers in France (Besancon, Lyon, and Rennes) and 1 center in Poland (Warsaw) between 08 December 2008 and 23 January 2013

    Pre-assignment
    Screening details
    		 During the “pre-surgical period”, informed consent was to be obtained and screening procedures conducted to confirm eligibility. Screening was to be done no longer than 8 weeks prior to the surgery. Main medical and surgical history, including bleeding history was recorded.

    Pre-assignment period milestones
    Number of subjects started
    		 6
    Number of subjects completed
    		 6

    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
    		 WILFACTIN
    Arm description
    		 -
    Arm type
    		 Experimental

    Investigational medicinal product name
    WILFACTIN
    Investigational medicinal product code
    vWF SD-35-DH
    Other name
    Pharmaceutical forms
    Powder and solvent for solution for infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    Single dose of 60 IU VWF:RCo/kg by bolus injection.

    Number of subjects in period 1
    		WILFACTIN
    Started
    6
    Completed
    6
    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
    		 WILFACTIN
    Arm description
    		 -
    Arm type
    		 Experimental

    Investigational medicinal product name
    WILFACTIN
    Investigational medicinal product code
    vWF SD-35-DH
    Other name
    Pharmaceutical forms
    Powder and solvent for solution for infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    The pre-Continuous infusion period started with: ▪ An extra bolus injection of vWF SD-35-DHD 12-24 hours before surgery (on D-1) if the subject's baseline FVIII:C levels was < 0.5 IU/mL. ▪ A loading dose 2 hours before the surgery on D0 in all subjects The Continuous Infusion (CI) period started immediately after the end of loading dose (H0) and lasted until the day of satisfactory healing of the surgical wound (Dn). This period lasted at least 6 days (144 hours) and could be longer depending on the type of surgery and its outcome. .

    Number of subjects in period 2
    		WILFACTIN
    Started
    6
    Completed
    6

    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
    		 6 6
    Age categorical
    Units: Subjects
        Adults (18-75 years)
    		 6 6
    Age continuous
    Units: years
        median (full range (min-max))
    		 49.5 (32 to 75) -
    Gender categorical
    Units: Subjects
        Female
    		 3 3
        Male
    		 3 3
    Subject analysis sets

    Subject analysis set title
    TTS (Total Treated Set)
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    All patients included in the study analysed in the TTS

    Subject analysis sets values
    		 TTS (Total Treated Set)
    Number of subjects
    6
    Age categorical
    Units: Subjects
        Adults (18-75 years)
    6
    Age continuous
    Units: years
        median (full range (min-max))
    49.5 (32 to 75)
    Gender categorical
    Units: Subjects
        Female
    3
        Male
    3

    End points

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

    Subject analysis set title
    TTS (Total Treated Set)
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    All patients included in the study analysed in the TTS

    Primary: Overall hemostatic efficacy assessment

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    End point title
    		 Overall hemostatic efficacy assessment [1]
    End point description
    End point type
    Primary
    End point timeframe
    The primary endpoint was the overall hemostatic efficacy assessment by the investigator after 6 days (6 × 24 hours = 144 hours) of CI (at D7) using a 4-point scale.
    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)
    Number of subjects analysed
    6
    Units: 4-point scale
        Excellent
    100
        Good
    0
        Moderate
    0
        None
    0
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Clinical safety was assessed throughout the study.
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    11.0
    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 / 6 (33.33%)
         number of deaths (all causes)
    0
         number of deaths resulting from adverse events
    0
    Infections and infestations
    Pseudomonal sepsis
    Additional description: Pseudomonas sepsis with abdominal entrance 6 days following orthopaedic surgery. The AE resolved without sequelae.
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Device related infection
    Additional description: Fortuitis discovery of infection (staphylococcus epidermidis) on the defectice knee prosthesis (device) to be removed for re-alloplasty. The AE resolved without sequelae.
         subjects affected / exposed
    1 / 6 (16.67%)
         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
    		 Total Treated Set
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    6 / 6 (100.00%)
    Investigations
    Blood pressure increased
    Additional description: 1 blood pressure increased assessed as not related to the study drug 1 blood pressure increased assessed as possibly related to the study drug
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    2
    Transaminases increased
    Additional description: Not related to the study drug
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Injury, poisoning and procedural complications
    Anaemia postoperative
    Additional description: Not related to the study drug
         subjects affected / exposed
    3 / 6 (50.00%)
         occurrences all number
    3
    Vascular disorders
    Haematoma
    Additional description: Not related to the study drug
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Nervous system disorders
    Sciatica
    Additional description: Not related to te study drug
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    General disorders and administration site conditions
    Infusion site reaction
    Additional description: Probable relationship with the study drug
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Venipuncture site inflammation
    Additional description: Possibly related to the study drug
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Vessel puncture site haematoma
    Additional description: Not related to the study drug
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Gastrointestinal disorders
    Diarrhoea
    Additional description: Not related to the study drug
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Dyspepsia
    Additional description: Not related to the study drug
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Psychiatric disorders
    Insomnia
    Additional description: Not related to the study drug
         subjects affected / exposed
    2 / 6 (33.33%)
         occurrences all number
    3

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    19 May 2011
    -To extend the study duration, -To modulate the use of the corrective factor at initiation of continuous infusion (dose for safety margin), as newly describe in the European "Guideline on the clinical investigation of recombinant and human plasma-derived-factor VIII products", EMEA/CHMP/BPWP/144533/2009 (23 July 2009) -To specify change of Director of Medical and Development Director. -To updated the French Patient information and Consent Form sheets by providing the latest information of French regulation regarding the reference methodology for personal data processing in biomedical clinical studies.

    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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