Flag of the European Union EU Clinical Trials Register Help

Clinical trials

The European Union Clinical Trials Register   allows you to search for protocol and results information on:
  • interventional clinical trials that were approved in the European Union (EU)/European Economic Area (EEA) under the Clinical Trials Directive 2001/20/EC
  • clinical trials conducted outside the EU/EEA that are linked to European paediatric-medicine development

    • EU/EEA interventional clinical trials approved under or transitioned to the Clinical Trial Regulation 536/2014 are publicly accessible through the
    Clinical Trials Information System (CTIS).

    The EU Clinical Trials Register currently displays   44334   clinical trials with a EudraCT protocol, of which   7366   are clinical trials conducted with subjects less than 18 years old.   The register also displays information on   18700   older paediatric trials (in scope of Article 45 of the Paediatric Regulation (EC) No 1901/2006).

    Phase 1 trials conducted solely on adults and that are not part of an agreed paediatric investigation plan (PIP) are not publicly available (see Frequently Asked Questions ).  
     
    Examples: Cancer AND drug name. Pneumonia AND sponsor name.
    How to search [pdf]
    Search Tips: Under advanced search you can use filters for Country, Age Group, Gender, Trial Phase, Trial Status, Date Range, Rare Diseases and Orphan Designation. For these items you should use the filters and not add them to your search terms in the text field.
    Advanced Search: Search tools
     

    < Back to search results

    Download PDF

    Clinical Trial Results:
    Efficacy and safety study of vWF SD-35-DH (WILFACTIN) in children under 6 years of age

    Summary
    EudraCT number
    		 2004-005051-34
    Trial protocol
    		 BE  
    Global end of trial date
    05 Aug 2014

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

    Trial information

    		 Close Top of page
    Trial identification
    Sponsor protocol code
    42-73-305
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 -
    WHO universal trial number (UTN)
    		 -
    Other trial identifiers
    		 WIL1-0305: WIL1-0305
    Sponsors
    Sponsor organisation name
    LFB Biotechnologies
    Sponsor organisation address
    3 Avenue des Tropiques , COURTABOEUF, France, 91930
    Public contact
    Françoise BRIDEY, LFB Biotechnologies, 33 169827010,
    Scientific contact
    Françoise BRIDEY, LFB Biotechnologies, 33 169827010,
    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
    04 Dec 2014
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    05 Aug 2014
    Global end of trial reached?
    Yes
    Global end of trial date
    05 Aug 2014
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    Main objective : to evaluate, in children presenting with inherited von Willebrand factor (VWF) deficiencies, the biological and clinical efficacy of WILFACTIN for the treatment of bleeding episodes and for the prevention of haemorrhages during surgery or invasive procedures when desmopressin is ineffective or contraindicated.
    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 in children.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    01 Feb 2006
    Long term follow-up planned
    Yes
    Long term follow-up rationale
    		 Safety, Efficacy
    Long term follow-up duration
    		 18 Months
    Independent data monitoring committee (IDMC) involvement?
    No
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    Poland: 3
    Country: Number of subjects enrolled
    Belgium: 1
    Country: Number of subjects enrolled
    Greece: 1
    Country: Number of subjects enrolled
    Tunisia: 4
    Worldwide total number of subjects
    9
    EEA total number of subjects
    5
    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)
    2
    Children (2-11 years)
    7
    Adolescents (12-17 years)
    0
    Adults (18-64 years)
    0
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

    		 Close Top of page
    Recruitment
    Recruitment details
    		 9 patients were included at 5 study centers in Belgium, Greece, Poland and Tunisia.

    Pre-assignment
    Screening details
    		 -

    Pre-assignment period milestones
    Number of subjects started
    		 10 [1]
    Number of subjects completed
    		 9

    Pre-assignment subject non-completion reasons
    Reason: Number of subjects
    		 selection criteria missing: 1
    Notes
    [1] - The number of subjects reported to have started the pre-assignment period are not the same as the worldwide number enrolled in the trial. It is expected that these numbers will be the same.
    Justification: 1 subject withdrawn after the pre-assigment period (1 selection criteria missing).
    Period 1
    Period 1 title
    Inclusion visit
    Is this the baseline period?
    		 Yes
    Allocation method
    Not applicable
    Blinding used
    		 Not blinded

    Arms
    Arm title
    		 Wilfactin treatment
    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
    No administration at inclusion.

    Number of subjects in period 1
    		Wilfactin treatment
    Started
    9
    Completed
    9
    Period 2
    Period 2 title
    Recovery study period
    Is this the baseline period?
    		 No
    Allocation method
    Not applicable
    Blinding used
    		 Not blinded

    Arms
    Arm title
    		 Wilfactin treatment
    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
    100 IU/kg by intravenous route

    Number of subjects in period 2
    		Wilfactin treatment
    Started
    9
    Completed
    9
    Period 3
    Period 3 title
    Efficacy period
    Is this the baseline period?
    		 No
    Allocation method
    Not applicable
    Blinding used
    		 Not blinded

    Arms
    Arm title
    		 Efficacy
    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
    For surgical procedures and treatment of bleeding episodes - when the recovery of von Willebrand factor is known: individualized dosing to provide plasma level of 100 % (VWF:RCo) , - when recovery information of von Willebrand factor was not available: 60-100 IU/kg The treatment duration depends on the clinical status of the subject and the baseline blood levels of VWF and factor VIII. For long-term prophylaxis, 50 IU/kg once per week to 30 IU/kg every other day to minimize spontaneous bleeding episodes.

    Number of subjects in period 3
    		Efficacy
    Started
    9
    Completed
    9

    Baseline characteristics

    		 Close Top of page
    Baseline characteristics reporting groups
    Reporting group title
    Inclusion visit
    Reporting group description
    		 -

    Reporting group values
    		 Inclusion visit Total
    Number of subjects
    		 9 9
    Age categorical
    Units: Subjects
        less than 6 years old
    		 9 9
    Age continuous
    Units: years
        median (full range (min-max))
    		 2 (0 to 5) -
    Gender categorical
    Units: Subjects
        Female
    		 4 4
        Male
    		 5 5
    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
    9
    Age categorical
    Units: Subjects
        less than 6 years old
    9
    Age continuous
    Units: years
        median (full range (min-max))
    2 (0 to 5)
    Gender categorical
    Units: Subjects
        Female
    4
        Male
    5

    End points

    		 Close Top of page
    End points reporting groups
    Reporting group title
    Wilfactin treatment
    Reporting group description
    		 -
    Reporting group title
    Wilfactin treatment
    Reporting group description
    		 -
    Reporting group title
    Efficacy
    Reporting group description
    		 -

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

    Primary: Percentage of Excellent/Good response

    		 Close Top of page
    End point title
    		 Percentage of Excellent/Good response [1]
    End point description
    End point type
    Primary
    End point timeframe
    Hemostasis in bleeding episodes and surgical/invasive procedures was evaluated by the investigator at the end of the bleeding episode or course of treatment
    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
    		 Efficacy
    Number of subjects analysed
    9
    Units: Four-point scale
        Excellent
    49
        Good
    43
        Moderate
    8
        None
    0
    No statistical analyses for this end point

    Adverse events

    		 Close Top of page
    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
    11.1
    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
    3 / 9 (33.33%)
         number of deaths (all causes)
    0
         number of deaths resulting from adverse events
    0
    General disorders and administration site conditions
    Gait disturbance
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Eye disorders
    Eyelid oedema
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Respiratory, thoracic and mediastinal disorders
    Lung disorder
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Infections and infestations
    Pneumonia
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Hordeolum
         subjects affected / exposed
    1 / 9 (11.11%)
         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
    9 / 9 (100.00%)
    Injury, poisoning and procedural complications
    Face injury
         subjects affected / exposed
    4 / 9 (44.44%)
         occurrences all number
    6
    Gingival injury
         subjects affected / exposed
    2 / 9 (22.22%)
         occurrences all number
    6
    Limb injury
         subjects affected / exposed
    3 / 9 (33.33%)
         occurrences all number
    6
    Traumatic haematoma
         subjects affected / exposed
    4 / 9 (44.44%)
         occurrences all number
    11
    Mouth injury
         subjects affected / exposed
    3 / 9 (33.33%)
         occurrences all number
    5
    Surgical and medical procedures
    Hepatitis A immunisation
         subjects affected / exposed
    7 / 9 (77.78%)
         occurrences all number
    7
    Infections and infestations
    Bronchitis
         subjects affected / exposed
    4 / 9 (44.44%)
         occurrences all number
    9
    Dental caries
         subjects affected / exposed
    3 / 9 (33.33%)
         occurrences all number
    5
    Nasopharyngitis
         subjects affected / exposed
    4 / 9 (44.44%)
         occurrences all number
    13
    Tonsillitis
         subjects affected / exposed
    2 / 9 (22.22%)
         occurrences all number
    5

    More information

    		 Close Top of page

    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    08 Jun 2007
    To prolong the study recuitment period. To add a recovery study to be performed after 6 months for children with type 3 VWD. To extand the number of centres including the participation of other countries.
    12 Nov 2007
    To change the name of the sponsor (LFB BIOTECHNOLOGIES ). To prolong the patient recruitment period (+ 6 month) and to add vWF inhibitor information in accordance with the guidelines on clinical investigation of human plasma derived von Willebrand factor products.
    12 Nov 2007
    To allow the patient to continue the IMP after 18 months follow-up under the same conditions if the product is registered but not yet commercialized. To specify the 2nd recovery study in the information patient sheet related to amendment n°4. To change the clinical project manager and extend the number of center and countries.
    05 Sep 2008
    To prolong the patient recruitment period; To update the procedure for SAE reporting; To add an interim analysis in Q2 2008.
    15 Mar 2011
    To prolong the recruitment period; To transfer storageand distribution to a sub-contractor; To clarify criteria for recovery study.

    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
    For support, Contact us.
    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.

    European Medicines Agency © 1995-Sun May 04 09:57:52 CEST 2025 | Domenico Scarlattilaan 6, 1083 HS Amsterdam, The Netherlands
    EMA HMA