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    Clinical Trial Results:
    A Double-Blind, Randomized, Crossover Study of the Recovery of FACTANE 100 versus 200 IU/ml followed by an Open-Label Safety Study of FACTANE 200 IU/ml in Previously Treated Patients With Severe (FVIII:C<1%) Haemophilia A

    Summary
    EudraCT number
    		 2009-013227-28
    Trial protocol
    		 FR  
    Global end of trial date
    25 Nov 2010

    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-0624
    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
    30 May 2011
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    25 Nov 2010
    Global end of trial reached?
    Yes
    Global end of trial date
    25 Nov 2010
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    Recovery Study Part The primary objective of this clinical research part is to compare the recovery of FACTANE 200 IU/ml with FACTANE 100 IU/ml, when given as a 4 ml/minute intravenous bolus injection. Safety Study Part The primary objective of this clinical research part is to assess the safety of FACTANE 200 IU/ml for at least 9 injections or 3 months whichever is sooner during standard-of-care treatment (on-demand, prophylaxis).
    Protection of trial subjects
    Precautions taken to minimise inconveniencies and potential pain for patients during the recovery study part: - The patient is managed by his usual medical team. - The injections are performed by the nursing staff of the haemophilia treatment centre experienced in the management of haemophilia. - The number of blood samples is restricted to six (before and after each IMP injection during the 3 visits for the recovery tests). 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 Jan 2010
    Long term follow-up planned
    Yes
    Long term follow-up rationale
    		 Safety
    Long term follow-up duration
    		 3 Months
    Independent data monitoring committee (IDMC) involvement?
    No
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    France: 13
    Worldwide total number of subjects
    13
    EEA total number of subjects
    13
    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)
    12
    From 65 to 84 years
    1
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 13 patients with severe haemophilia A patients (FVIII <1 IU/dl (%)) were included between 12/01/2010 and 25/11/2010 in France.

    Pre-assignment
    Screening details
    		 -

    Pre-assignment period milestones
    Number of subjects started
    		 12 [1]
    Number of subjects completed
    		 12

    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: 12 subjects included in the recovery study part. 13 subjects included in the safety period.
    Period 1
    Period 1 title
    Recovery Study
    Is this the baseline period?
    		 Yes
    Allocation method
    Randomised - controlled
    Blinding used
    		 Double blind
    Roles blinded
    		 Subject, Investigator, Monitor, Data analyst, Carer, Assessor
    Blinding implementation details
    Randomisation by complete block was used to assign patients to one of the two sequences: FACTANE 100 IU/ml - FACTANE 200 IU/ml and FACTANE 200 IU/ml - FACTANE 100 IU/ml. The number of patients in each block was balanced.

    Arms
    Arm title
    		 FACTANE 100 or 200 IU/ml
    Arm description
    		 Patients received FACTANE 100 IU/ml then FACTANE 200 IU/ml during a crossover study with active experimental product
    Arm type
    		 Active comparator

    Investigational medicinal product name
    FACTANE
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Powder and solution for solution for injection
    Routes of administration
    Intravenous use
    Dosage and administration details
    PK cohort included a randomized, blinded dual crossover design, in which subjects received a single dose each of FACTANE 100 IU/ml (25-50 IU/kg) and FACTANE 200 IU/ml (25-50 IU/kg) in random order (3-day washout)

    Number of subjects in period 1 [2]
    		FACTANE 100 or 200 IU/ml
    Started
    12
    Completed
    12
    Notes
    [2] - The number of subjects reported to be in the baseline period are not the same as the worldwide number enrolled in the trial. It is expected that these numbers will be the same.
    Justification: 12 subjects included in the recovery study part. 13 subjects included in the safety period.
    Period 2
    Period 2 title
    Safety assessment period
    Is this the baseline period?
    		 No
    Allocation method
    Not applicable
    Blinding used
    		 Not blinded

    Arms
    Arm title
    		 FACTANE 200 IU/ml
    Arm description
    		 Safety study part all patients received FACTANE 200 IU/ml
    Arm type
    		 Experimental

    Investigational medicinal product name
    FACTANE 200 IU/ml
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Powder and solution for solution for injection
    Routes of administration
    Intravenous use
    Dosage and administration details
    FACTANE 200 IU/ml: dose varied by subject and therapeutic situation

    Number of subjects in period 2
    		FACTANE 200 IU/ml
    Started
    12
    Completed
    13
    Joined
    1
         no recovery period
    1

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Recovery Study
    Reporting group description
    		 -

    Reporting group values
    		 Recovery Study Total
    Number of subjects
    		 12 12
    Age categorical
    Units: Subjects
        Adults (18-64 years)
    		 12 12
        From 65-84 years
    		 0 0
    Age continuous
    Units: years
        median (full range (min-max))
    		 28.7 (22.2 to 60.8) -
    Gender categorical
    Units: Subjects
        Male
    		 12 12
    Subject analysis sets

    Subject analysis set title
    Efficacy population
    Subject analysis set type
    		 Per protocol
    Subject analysis set description
    Subjects included in the Efficacy population (Per Protocol Population) who received FACTANE 100 UI/ml and FACTANE 200 UI/ml in crossover study.

    Subject analysis set title
    Safety population (TTS)
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    Subjects in the safety population TTS (Total treated Set)

    Subject analysis sets values
    		 Efficacy population Safety population (TTS)
    Number of subjects
    12
    13
    Age categorical
    Units: Subjects
        Adults (18-64 years)
    12
    12
        From 65-84 years
    0
    1
    Age continuous
    Units: years
        median (full range (min-max))
    28.7 (22.2 to 60.8)
    29 (22.2 to 72.7)
    Gender categorical
    Units: Subjects
        Male
    12
    13

    End points

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    End points reporting groups
    Reporting group title
    FACTANE 100 or 200 IU/ml
    Reporting group description
    		 Patients received FACTANE 100 IU/ml then FACTANE 200 IU/ml during a crossover study with active experimental product
    Reporting group title
    FACTANE 200 IU/ml
    Reporting group description
    		 Safety study part all patients received FACTANE 200 IU/ml

    Subject analysis set title
    Efficacy population
    Subject analysis set type
    		 Per protocol
    Subject analysis set description
    Subjects included in the Efficacy population (Per Protocol Population) who received FACTANE 100 UI/ml and FACTANE 200 UI/ml in crossover study.

    Subject analysis set title
    Safety population (TTS)
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    Subjects in the safety population TTS (Total treated Set)

    Primary: FVIII incremental recovery

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    End point title
    		 FVIII incremental recovery [1]
    End point description
    The primary endpoint of the 'Recovery Study' part was determination of the equivalence of the test drug (FACTANE 200 IU/ml) and the reference drug (FACTANE 100 IU/ml) evaluated using the concentration at 15 minutes post injection (CT15) normalised by the dose injected (expressed by IU/kg or incremental recovery
    End point type
    Primary
    End point timeframe
    FVIII recovery for FVIII activity measured by one-stage clotting assay.
    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 population
    Number of subjects analysed
    12
    Units: %/IU/kg
    median (full range (min-max))
        FACTANE 100 IU/ml
    2.24 (1.76 to 2.89)
        FACTANE 200 IU/ml
    2.21 (1.66 to 2.88)
    No statistical analyses for this end point

    Primary: Long term tolerance of FACTANE 200 IU/ml (investigator's assessment scale)

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    End point title
    		 Long term tolerance of FACTANE 200 IU/ml (investigator's assessment scale) [2]
    End point description
    Investigator's assessment of the global safety of FACTANE 200IU/ml according to the same scale: "very good/good", "satisfactory/unsatisfactory".
    End point type
    Primary
    End point timeframe
    The primary objective of the safety part was to assess the long-term tolerance of FACTANE 200 IU/ml, when administered for at least 9 injections or 3 months whichever is sooner.
    Notes
    [2] - 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
    		 Safety population (TTS)
    Number of subjects analysed
    13
    Units: percentage
        very good /good
    100
        satisfactory / unsatisfactory
    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, during part I (recovery study) and part II (safety study).
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    9.1
    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
    0 / 13 (0.00%)
         number of deaths (all causes)
    0
         number of deaths resulting from adverse events
    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
    3 / 13 (23.08%)
    Nervous system disorders
    Headache
    Additional description: not related with the IMP.
         subjects affected / exposed
    2 / 13 (15.38%)
         occurrences all number
    3
    Gastrointestinal disorders
    Dysgeusia
    Additional description: possible relationship with the use of IMP (either FACTANE 100 IU/ml or FACTANE 200IU/ml)
         subjects affected / exposed
    1 / 13 (7.69%)
         occurrences all number
    14

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    05 Feb 2010
    The main objectives of this amendment are the following: - to extend the inclusion period, - to specify the changes of the Clinical Project Manager, - to homogenize information regarding the number of patients, - to describe the procedure for the management of non evaluable data (recovery part)

    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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