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   43857   clinical trials with a EudraCT protocol, of which   7284   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:
    A Prospective Study to Evaluate the Effect of rFVIII-FS in Different Prophylactic Regimens on Bleeding Events Frequency and Development of Arthropathy in Previously Treated and Minimally Treated Hemophilia A Pediatric Population

    Summary
    EudraCT number
    		 2014-005253-39
    Trial protocol
    		 Outside EU/EEA  
    Global end of trial date
    28 Sep 2009

    Results information
    Results version number
    		 v2(current)
    This version publication date
    07 Sep 2016
    First version publication date
    20 Jun 2015
    Other versions
    		 v1
    Version creation reason
    • New data added to full data set
    • Correction of full data set
    Bayer sponsor contact information to be updated

    Trial information

    		 Close Top of page
    Trial identification
    Sponsor protocol code
    BAY14-2222/12684
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT00632814
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Bayer AG
    Sponsor organisation address
    Kaiser-Wilhelm-Allee, D-51368, Leverkusen, Germany,
    Public contact
    Therapeutic Area Head, Bayer AG, clinical-trials-contact@bayer.com
    Scientific contact
    Therapeutic Area Head, Bayer AG, clinical-trials-contact@bayer.com
    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?
    Yes
    Results analysis stage
    Analysis stage
    Final
    Date of interim/final analysis
    02 Aug 2010
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    28 Sep 2009
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    To evaluate and compare the effect of 3 different prophylactic regimens (once per week, twice per week, and three times per week; dose escalation in case of insufficient bleeding protection) on frequency of joint bleeds in severe and moderate pediatric hemophilia A subjects.
    Protection of trial subjects
    The conduct of this clinical study met all local legal and regulatory requirements. The study was conducted in accordance with the ethical principles that have their origin in the Declaration of Helsinki and the International Conference on Harmonization guideline E6: Good Clinical Practice. Before entering the study, the informed consent form was read by and explained to all subjects and/or their legally authorized representatives. Participating subjects and/or their legally authorized representatives signed informed consent form and could withdraw from the study at any time without any disadvantage and without having to provide a reason for this decision. Only investigators qualified by training and experience were selected as appropriate experts to investigate the study drug.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    28 Jun 2007
    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
    Russian Federation: 32
    Worldwide total number of subjects
    32
    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)
    3
    Children (2-11 years)
    27
    Adolescents (12-17 years)
    2
    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
    		 The recruitment period took from first subject first visit 28 Jun 2007 to last subject first visit 26 Dec 2008. The study period took from first subject first visit 28 Jun 2007 to last subject last visit 28 Sep 2009. All 4 sites were medical clinics. Assignment to a group was based on subjects previous treatment schedule (non-randomized).

    Pre-assignment
    Screening details
    		 There was an indefinite time period between screening and baseline. Study treatment started at visit 2 (baseline).

    Period 1
    Period 1 title
    Overall Study (overall period)
    Is this the baseline period?
    		 Yes
    Allocation method
    Not applicable
    Blinding used
    		 Not blinded

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 rFVIII-FS (Kogenate FS, BAY14-2222), 70 IU/kg qw
    Arm description
    		 rFVIII-FS (Octocog-alfa, antihemophilic factor [recombinant]) 70 international units per kilogram (IU/kg), dosing by injection once per week (qw) (weekly on Day 7 + 1 after previous injection) for 9 months. Dose escalation was permitted due to joint bleeding (escalation to 35 IU/kg twice a week [biw] or further escalation to 25 IU/kg three times a week [tiw]).
    Arm type
    		 Experimental

    Investigational medicinal product name
    Recombinant FVIII formulated with sucrose (rFVIII-FS)
    Investigational medicinal product code
    BAY14-2222
    Other name
    Octocog alfa, Kogenate® FS
    Pharmaceutical forms
    Injection
    Routes of administration
    Intravenous use
    Dosage and administration details
    rFVIII-FS (Octocog-alfa, antihemophilic factor [recombinant]) 70 IU/kg, dosing by injection qw (weekly on Day 7 + 1 after previous injection) for 9 months. Dose escalation was permitted due to joint bleeding (escalation to 35 IU/kg biw or further escalation to 25 IU/kg tiw).

    Arm title
    		 rFVIII-FS (Kogenate FS, BAY14-2222), biw (30 IU/kg + 40 IU/kg)
    Arm description
    		 rFVIII-FS (Octocog-alfa, antihemophilic factor [recombinant]) 70 IU/kg, dosing by injection biw (30 IU/kg [day 1] + 40 IU/kg [day 4]) for 9 months. Dose escalation was permitted due to joint bleeding (escalation to 25 IU/kg tiw).
    Arm type
    		 Experimental

    Investigational medicinal product name
    Recombinant FVIII formulated with sucrose (rFVIII-FS)
    Investigational medicinal product code
    BAY14-2222
    Other name
    Octocog alfa, Kogenate® FS
    Pharmaceutical forms
    Injection
    Routes of administration
    Intravenous use
    Dosage and administration details
    rFVIII-FS (Octocog-alfa, antihemophilic factor [recombinant]) 70 IU/kg, dosing by injection biw (30 IU/kg [day 1] + 40 IU/kg [day 4]) for 9 months. Dose escalation was permitted due to joint bleeding (escalation to 25 IU/kg tiw).

    Arm title
    		 rFVIII-FS (Kogenate FS, BAY14-2222), tiw (3 x 25 IU/kg)
    Arm description
    		 rFVIII-FS (Octocog-alfa, antihemophilic factor [recombinant]) 75 IU/kg, dosing by injection tiw (3 x 25 IU/kg [day 1, 3, 5]) for 9 months. No escalation opportunity for subjects in this group.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Recombinant FVIII formulated with sucrose (rFVIII-FS)
    Investigational medicinal product code
    BAY14-2222
    Other name
    Octocog alfa, Kogenate® FS
    Pharmaceutical forms
    Injection
    Routes of administration
    Intravenous use
    Dosage and administration details
    rFVIII-FS (Octocog-alfa, antihemophilic factor [recombinant]) 75 IU/kg, dosing by injection tiw (3 x 25 IU/kg [day 1, 3, 5]) for 9 months. No escalation opportunity for subjects in this group.

    Number of subjects in period 1
    		rFVIII-FS (Kogenate FS, BAY14-2222), 70 IU/kg qw rFVIII-FS (Kogenate FS, BAY14-2222), biw (30 IU/kg + 40 IU/kg) rFVIII-FS (Kogenate FS, BAY14-2222), tiw (3 x 25 IU/kg)
    Started
    11
    13
    8
    Completed
    11
    13
    8

    Baseline characteristics

    		 Close Top of page
    Baseline characteristics reporting groups
    Reporting group title
    rFVIII-FS (Kogenate FS, BAY14-2222), 70 IU/kg qw
    Reporting group description
    		 rFVIII-FS (Octocog-alfa, antihemophilic factor [recombinant]) 70 international units per kilogram (IU/kg), dosing by injection once per week (qw) (weekly on Day 7 + 1 after previous injection) for 9 months. Dose escalation was permitted due to joint bleeding (escalation to 35 IU/kg twice a week [biw] or further escalation to 25 IU/kg three times a week [tiw]).

    Reporting group title
    rFVIII-FS (Kogenate FS, BAY14-2222), biw (30 IU/kg + 40 IU/kg)
    Reporting group description
    		 rFVIII-FS (Octocog-alfa, antihemophilic factor [recombinant]) 70 IU/kg, dosing by injection biw (30 IU/kg [day 1] + 40 IU/kg [day 4]) for 9 months. Dose escalation was permitted due to joint bleeding (escalation to 25 IU/kg tiw).

    Reporting group title
    rFVIII-FS (Kogenate FS, BAY14-2222), tiw (3 x 25 IU/kg)
    Reporting group description
    		 rFVIII-FS (Octocog-alfa, antihemophilic factor [recombinant]) 75 IU/kg, dosing by injection tiw (3 x 25 IU/kg [day 1, 3, 5]) for 9 months. No escalation opportunity for subjects in this group.

    Reporting group values
    		 rFVIII-FS (Kogenate FS, BAY14-2222), 70 IU/kg qw rFVIII-FS (Kogenate FS, BAY14-2222), biw (30 IU/kg + 40 IU/kg) rFVIII-FS (Kogenate FS, BAY14-2222), tiw (3 x 25 IU/kg) Total
    Number of subjects
    		 11 13 8
    Age categorical
    Units: Subjects
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    		 3.27 ± 3.2 6.08 ± 3.5 6 ± 3.12 -
    Gender Categorical
    Units: subjects
        Male
    		 11 13 8 32
    Ethnicity
    Units: Subjects
        Caucasian
    		 11 13 7 31
        Asian
    		 0 0 1 1
    Number of subjects on prophylaxis versus on-demand therapy prior to screening
    Number of subjects on prophylaxis versus on-demand therapy before screening
    Units: Subjects
        On demand
    		 9 6 5 20
        Prophylaxis
    		 2 7 3 12
    Number of subjects with different exposure days (ED)
    Units: Subjects
        0 ED
    		 1 0 0 1
        1 to <20 ED
    		 0 0 0 0
        20 to <100 ED
    		 5 2 4 11
        >=100 ED
    		 5 11 4 20
    Number of subjects with target joint present
    Units: Subjects
        Target joint present
    		 4 7 7 18
        Target joint absent
    		 7 6 1 14
    Number of subjects with bleeding rates in previous 6-9 Months
    Units: Subjects
        No bleedings
    		 1 2 0 3
        Any bleedings
    		 10 11 8 29
    Number of subjects with joint bleeding in previous 6-9 Months
    Units: Subjects
        Any joint bleeding
    		 9 11 6 26
        No joint bleeding
    		 2 2 2 6
    Body weight
    Units: kilograms
        arithmetic mean (standard deviation)
    		 17.48 ± 10.43 24.39 ± 11.35 25.45 ± 9.87 -
    Height
    Units: centimeters
        arithmetic mean (standard deviation)
    		 98.09 ± 22.88 120.31 ± 23.75 122 ± 20.74 -
    FVIII trough level at Baseline
    Units: percentage of FVIII activity
        arithmetic mean (standard deviation)
    		 1.43 ± 0.7 1.62 ± 1.79 0.78 ± 0.24 -
    Stockholm Joint Score
    The assessment of joint function using Stockholm Joint Score. The minimum value is 0 (the best condition), and the maximum value is 140 (the worst condition).
    Units: scores on a scale
        arithmetic mean (standard deviation)
    		 3.9 ± 4.8 5.9 ± 5.8 7.1 ± 4.6 -

    End points

    		 Close Top of page
    End points reporting groups
    Reporting group title
    rFVIII-FS (Kogenate FS, BAY14-2222), 70 IU/kg qw
    Reporting group description
    		 rFVIII-FS (Octocog-alfa, antihemophilic factor [recombinant]) 70 international units per kilogram (IU/kg), dosing by injection once per week (qw) (weekly on Day 7 + 1 after previous injection) for 9 months. Dose escalation was permitted due to joint bleeding (escalation to 35 IU/kg twice a week [biw] or further escalation to 25 IU/kg three times a week [tiw]).

    Reporting group title
    rFVIII-FS (Kogenate FS, BAY14-2222), biw (30 IU/kg + 40 IU/kg)
    Reporting group description
    		 rFVIII-FS (Octocog-alfa, antihemophilic factor [recombinant]) 70 IU/kg, dosing by injection biw (30 IU/kg [day 1] + 40 IU/kg [day 4]) for 9 months. Dose escalation was permitted due to joint bleeding (escalation to 25 IU/kg tiw).

    Reporting group title
    rFVIII-FS (Kogenate FS, BAY14-2222), tiw (3 x 25 IU/kg)
    Reporting group description
    		 rFVIII-FS (Octocog-alfa, antihemophilic factor [recombinant]) 75 IU/kg, dosing by injection tiw (3 x 25 IU/kg [day 1, 3, 5]) for 9 months. No escalation opportunity for subjects in this group.

    Primary: Percentage of Subjects With Less Than 2 Joint Bleeds During the 9-month Treatment Period

    		 Close Top of page
    End point title
    		 Percentage of Subjects With Less Than 2 Joint Bleeds During the 9-month Treatment Period [1]
    End point description
    End point type
    Primary
    End point timeframe
    Up to 9 months
    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 statistics were done, no inferential statistical analyses were performed.
    End point values
    		 rFVIII-FS (Kogenate FS, BAY14-2222), 70 IU/kg qw rFVIII-FS (Kogenate FS, BAY14-2222), biw (30 IU/kg + 40 IU/kg) rFVIII-FS (Kogenate FS, BAY14-2222), tiw (3 x 25 IU/kg)
    Number of subjects analysed
    11
    13
    8
    Units: Percentage of subjects
        number (not applicable)
    72.7
    84.6
    75
    No statistical analyses for this end point

    Secondary: Number of Bleeds per Subject During the 9-month Treatment Period

    		 Close Top of page
    End point title
    		 Number of Bleeds per Subject During the 9-month Treatment Period
    End point description
    End point type
    Secondary
    End point timeframe
    Up to 9 months
    End point values
    		 rFVIII-FS (Kogenate FS, BAY14-2222), 70 IU/kg qw rFVIII-FS (Kogenate FS, BAY14-2222), biw (30 IU/kg + 40 IU/kg) rFVIII-FS (Kogenate FS, BAY14-2222), tiw (3 x 25 IU/kg)
    Number of subjects analysed
    11
    13
    8
    Units: bleeds per subject
    median (full range (min-max))
        All bleeds
    3 (0 to 12)
    2 (0 to 6)
    1.5 (0 to 8)
        Joint bleeds
    0 (0 to 2)
    0 (0 to 3)
    0 (0 to 8)
    No statistical analyses for this end point

    Secondary: Number of Subjects With Bleeding Events During the 9-month Treatment Period

    		 Close Top of page
    End point title
    		 Number of Subjects With Bleeding Events During the 9-month Treatment Period
    End point description
    End point type
    Secondary
    End point timeframe
    Up to 9 months
    End point values
    		 rFVIII-FS (Kogenate FS, BAY14-2222), 70 IU/kg qw rFVIII-FS (Kogenate FS, BAY14-2222), biw (30 IU/kg + 40 IU/kg) rFVIII-FS (Kogenate FS, BAY14-2222), tiw (3 x 25 IU/kg)
    Number of subjects analysed
    11
    13
    8
    Units: subjects
    number (not applicable)
        no bleeds
    3
    4
    4
        bleeds at all
    8
    9
    4
    No statistical analyses for this end point

    Secondary: Number of Subjects With Joint Bleeds During the 9-month Treatment Period

    		 Close Top of page
    End point title
    		 Number of Subjects With Joint Bleeds During the 9-month Treatment Period
    End point description
    End point type
    Secondary
    End point timeframe
    Up to 9 months
    End point values
    		 rFVIII-FS (Kogenate FS, BAY14-2222), 70 IU/kg qw rFVIII-FS (Kogenate FS, BAY14-2222), biw (30 IU/kg + 40 IU/kg) rFVIII-FS (Kogenate FS, BAY14-2222), tiw (3 x 25 IU/kg)
    Number of subjects analysed
    11
    13
    8
    Units: subjects
    number (not applicable)
        no bleeds
    8
    9
    6
        bleeds at all
    3
    4
    2
    No statistical analyses for this end point

    Secondary: Number of Subjects in Each Group at the End of the Study

    		 Close Top of page
    End point title
    		 Number of Subjects in Each Group at the End of the Study
    End point description
    Subjects were allowed to switch treatment groups upon occurrence of joint bleed. Therefore, the number of subjects per group at the end of the study is different from the number of subjects per group at baseline.
    End point type
    Secondary
    End point timeframe
    Up to 9 months
    End point values
    		 rFVIII-FS (Kogenate FS, BAY14-2222), 70 IU/kg qw rFVIII-FS (Kogenate FS, BAY14-2222), biw (30 IU/kg + 40 IU/kg) rFVIII-FS (Kogenate FS, BAY14-2222), tiw (3 x 25 IU/kg)
    Number of subjects analysed
    11
    13
    8
    Units: Subjects
    8
    14
    10
    No statistical analyses for this end point

    Secondary: Actual Monthly rFVIII-FS Consumption

    		 Close Top of page
    End point title
    		 Actual Monthly rFVIII-FS Consumption
    End point description
    End point type
    Secondary
    End point timeframe
    Up to 9 months
    End point values
    		 rFVIII-FS (Kogenate FS, BAY14-2222), 70 IU/kg qw rFVIII-FS (Kogenate FS, BAY14-2222), biw (30 IU/kg + 40 IU/kg) rFVIII-FS (Kogenate FS, BAY14-2222), tiw (3 x 25 IU/kg)
    Number of subjects analysed
    11
    13
    8
    Units: IU/kg
        arithmetic mean (standard deviation)
    390.8 ± 106.9
    422.3 ± 138.2
    501.4 ± 241.2
    No statistical analyses for this end point

    Secondary: Change From Baseline in Stockholm Hemophilia Joint Score at 9 Months of Treatment

    		 Close Top of page
    End point title
    		 Change From Baseline in Stockholm Hemophilia Joint Score at 9 Months of Treatment
    End point description
    The assessment of joint function using Stockholm Joint Score. The minimum value is 0 (the best condition), and the maximum value is 140 (the worst condition).
    End point type
    Secondary
    End point timeframe
    baseline and 9 months
    End point values
    		 rFVIII-FS (Kogenate FS, BAY14-2222), 70 IU/kg qw rFVIII-FS (Kogenate FS, BAY14-2222), biw (30 IU/kg + 40 IU/kg) rFVIII-FS (Kogenate FS, BAY14-2222), tiw (3 x 25 IU/kg)
    Number of subjects analysed
    11
    13
    8
    Units: Scores on a scale
        arithmetic mean (standard deviation)
    -1.4 ± 2.4
    -2 ± 4.5
    -1.8 ± 2.2
    No statistical analyses for this end point

    Secondary: Haemo-Quality of Life (QoL) Standardized Total Score at 9 Months of Treatment (Completed by Subjects in the Total Group)

    		 Close Top of page
    End point title
    		 Haemo-Quality of Life (QoL) Standardized Total Score at 9 Months of Treatment (Completed by Subjects in the Total Group)
    End point description
    QoL was measured by the Haemo-QoL standardized total Score, which ranged from 0 (the best condition) to 100 (the worst condition).
    End point type
    Secondary
    End point timeframe
    9 months
    End point values
    		 rFVIII-FS (Kogenate FS, BAY14-2222), 70 IU/kg qw rFVIII-FS (Kogenate FS, BAY14-2222), biw (30 IU/kg + 40 IU/kg) rFVIII-FS (Kogenate FS, BAY14-2222), tiw (3 x 25 IU/kg)
    Number of subjects analysed
    3 [2]
    10 [3]
    7 [4]
    Units: Scores on a scale
        arithmetic mean (standard deviation)
    30.28 ± 13.09
    39.07 ± 23.5
    30.27 ± 11.95
    Notes
    [2] - Subjects who completed the questionnaire.
    [3] - subjects who completed the questionnaire.
    [4] - Subjects who completed the questionnaire.
    No statistical analyses for this end point

    Secondary: Haemo-Quality of Life (QoL) Standardized Total Score (Completed by Parents/Caregivers in the Total Group) at 9 Months of Treatment

    		 Close Top of page
    End point title
    		 Haemo-Quality of Life (QoL) Standardized Total Score (Completed by Parents/Caregivers in the Total Group) at 9 Months of Treatment
    End point description
    QoL was measured by the Haemo-QoL standardized total Score, which ranged from 0 (the best condition) to 100 (the worst condition).
    End point type
    Secondary
    End point timeframe
    9 months
    End point values
    		 rFVIII-FS (Kogenate FS, BAY14-2222), 70 IU/kg qw rFVIII-FS (Kogenate FS, BAY14-2222), biw (30 IU/kg + 40 IU/kg) rFVIII-FS (Kogenate FS, BAY14-2222), tiw (3 x 25 IU/kg)
    Number of subjects analysed
    3 [5]
    10 [6]
    7 [7]
    Units: Scores on a scale
        arithmetic mean (standard deviation)
    29.13 ± 17.4
    28.43 ± 10.27
    31.38 ± 13.06
    Notes
    [5] - Subjects who completed the questionnaire.
    [6] - subjects who completed the questionnaire.
    [7] - Subjects who completed the questionnaire.
    No statistical analyses for this end point

    Adverse events

    		 Close Top of page
    Adverse events information
    Timeframe for reporting adverse events
    From the start of study drug administration until the end of study (9 months +/- 2 weeks)
    Assessment type
    		 Non-systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    13.0
    Reporting groups
    Reporting group title
    rFVIII-FS (Kogenate FS, BAY14-2222), 70 IU/kg qw
    Reporting group description
    		 rFVIII-FS (Octocog-alfa, antihemophilic factor [recombinant]) 70 IU/kg, dosing by injection once per week [qw] (weekly on Day 7 + 1 after previous injection) for 9 months. Dose escalation was permitted due to joint bleeding (escalation to 35 IU/kg twice a week or further escalation to 25 IU/kg three times a week).

    Reporting group title
    rFVIII-FS (Kogenate FS, BAY14-2222), biw (30 IU/kg + 40 IU/kg)
    Reporting group description
    		 rFVIII-FS (Octocog-alfa, antihemophilic factor [recombinant]) 70 IU/kg, dosing by injection twice per week [biw] (30 IU/kg [day 1] + 40 IU/kg [day 4]) for 9 months. Dose escalation was permitted due to joint bleeding (escalation to 25 IU/kg three times a week).

    Reporting group title
    rFVIII-FS (Kogenate FS, BAY14-2222), tiw (3 x 25 IU/kg)
    Reporting group description
    		 rFVIII-FS (Octocog-alfa, antihemophilic factor [recombinant]) 75 IU/kg, dosing by injection three times per week [tiw] (3 x 25 IU/kg [day 1, 3, 5]) for 9 months. No escalation opportunity for subjects in this group.

    Serious adverse events
    		 rFVIII-FS (Kogenate FS, BAY14-2222), 70 IU/kg qw rFVIII-FS (Kogenate FS, BAY14-2222), biw (30 IU/kg + 40 IU/kg) rFVIII-FS (Kogenate FS, BAY14-2222), tiw (3 x 25 IU/kg)
    Total subjects affected by serious adverse events
         subjects affected / exposed
    1 / 11 (9.09%)
    2 / 13 (15.38%)
    0 / 8 (0.00%)
         number of deaths (all causes)
    0
    0
    0
         number of deaths resulting from adverse events
    0
    0
    0
    Blood and lymphatic system disorders
    FACTOR VIII INHIBITION
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 13 (0.00%)
    0 / 8 (0.00%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Gastrointestinal disorders
    ABDOMINAL PAIN
         subjects affected / exposed
    0 / 11 (0.00%)
    1 / 13 (7.69%)
    0 / 8 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    VOMITING
         subjects affected / exposed
    0 / 11 (0.00%)
    1 / 13 (7.69%)
    0 / 8 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Infections and infestations
    EAR INFECTION
         subjects affected / exposed
    0 / 11 (0.00%)
    1 / 13 (7.69%)
    0 / 8 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    RESPIRATORY TRACT INFECTION VIRAL
         subjects affected / exposed
    0 / 11 (0.00%)
    1 / 13 (7.69%)
    0 / 8 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 0%
    Non-serious adverse events
    		 rFVIII-FS (Kogenate FS, BAY14-2222), 70 IU/kg qw rFVIII-FS (Kogenate FS, BAY14-2222), biw (30 IU/kg + 40 IU/kg) rFVIII-FS (Kogenate FS, BAY14-2222), tiw (3 x 25 IU/kg)
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    4 / 11 (36.36%)
    3 / 13 (23.08%)
    3 / 8 (37.50%)
    Injury, poisoning and procedural complications
    LOWER LIMB FRACTURE
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 13 (0.00%)
    0 / 8 (0.00%)
         occurrences all number
    1
    0
    0
    Blood and lymphatic system disorders
    IRON DEFICIENCY ANAEMIA
         subjects affected / exposed
    0 / 11 (0.00%)
    0 / 13 (0.00%)
    1 / 8 (12.50%)
         occurrences all number
    0
    0
    1
    General disorders and administration site conditions
    CHILLS
         subjects affected / exposed
    0 / 11 (0.00%)
    1 / 13 (7.69%)
    1 / 8 (12.50%)
         occurrences all number
    0
    1
    1
    PYREXIA
         subjects affected / exposed
    0 / 11 (0.00%)
    0 / 13 (0.00%)
    1 / 8 (12.50%)
         occurrences all number
    0
    0
    4
    Respiratory, thoracic and mediastinal disorders
    EPISTAXIS
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 13 (0.00%)
    0 / 8 (0.00%)
         occurrences all number
    2
    0
    0
    LARYNGOSPASM
         subjects affected / exposed
    0 / 11 (0.00%)
    1 / 13 (7.69%)
    0 / 8 (0.00%)
         occurrences all number
    0
    1
    0
    RESPIRATORY DISORDER
         subjects affected / exposed
    0 / 11 (0.00%)
    1 / 13 (7.69%)
    0 / 8 (0.00%)
         occurrences all number
    0
    1
    0
    Skin and subcutaneous tissue disorders
    DERMATITIS ALLERGIC
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 13 (0.00%)
    0 / 8 (0.00%)
         occurrences all number
    1
    0
    0
    Infections and infestations
    ADENOIDITIS
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 13 (0.00%)
    0 / 8 (0.00%)
         occurrences all number
    1
    0
    0
    RESPIRATORY TRACT INFECTION VIRAL
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 13 (0.00%)
    1 / 8 (12.50%)
         occurrences all number
    1
    0
    1
    TONSILLITIS
         subjects affected / exposed
    0 / 11 (0.00%)
    0 / 13 (0.00%)
    1 / 8 (12.50%)
         occurrences all number
    0
    0
    1
    TUBERCULOSIS
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 13 (0.00%)
    0 / 8 (0.00%)
         occurrences all number
    1
    0
    0
    VARICELLA
         subjects affected / exposed
    0 / 11 (0.00%)
    0 / 13 (0.00%)
    1 / 8 (12.50%)
         occurrences all number
    0
    0
    1
    YERSINIA INFECTION
         subjects affected / exposed
    0 / 11 (0.00%)
    0 / 13 (0.00%)
    1 / 8 (12.50%)
         occurrences all number
    0
    0
    1

    More information

    		 Close Top of page

    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    12 Dec 2006
    Modification of the study design from a single-center study into a multicenter study with 3 study centers. 1. The number of subjects to be enrolled was increased from 20 to 40. 2. The planned enrolment period was extended from 3 months to 8 months. 3. An additional vial size containing 1000 IU FVIII/milliliter was introduced. 4. It was specified that the sterile water for injection was provided in a prefilled syringe in the treatment kit.
    22 Sep 2008
    1. The number of study centers was increased from 3 to 6. 2. The number of subjects to be enrolled was reduced from 40 to 36 in order to balance the sizes of the 3 treatment groups (12 per treatment group).

    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.
    Protocol deviations were not excluded: For example, a previously untreated subject developed the transitory inhibitor; change to a higher group did not always occur according protocol; there was temporary lack of smaller vial sizes at the centers.
    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-Wed Apr 24 00:59:45 CEST 2024 | Domenico Scarlattilaan 6, 1083 HS Amsterdam, The Netherlands
    EMA HMA