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    Clinical Trial Results:
    An Open-Label, Single Dose Pharmacokinetic Study of BeneFIX (Nonacog Alfa, Recombinant Factor IX) in Male Chinese Subjects With Hemophilia B

    Summary
    EudraCT number
    2015-003027-61
    Trial protocol
    Outside EU/EEA  
    Global end of trial date
    08 Apr 2015

    Results information
    Results version number
    v1(current)
    This version publication date
    29 Jun 2016
    First version publication date
    29 Jun 2016
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    B1821048
    Additional study identifiers
    ISRCTN number
    -
    US NCT number
    NCT02213250
    WHO universal trial number (UTN)
    -
    Sponsors
    Sponsor organisation name
    Pfizer, Inc
    Sponsor organisation address
    235 E 42nd Street, New York, United States, 10017
    Public contact
    Pfizer ClinicalTrials.gov Call Center, Pfizer, Inc, 001 800-718-1021, ClinicalTrials.gov_Inquiries@pfizer.com
    Scientific contact
    Pfizer ClinicalTrials.gov Call Center, Pfizer, Inc, 001 800-718-1021, ClinicalTrials.gov_Inquiries@pfizer.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
    06 Jun 2016
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    08 Apr 2015
    Global end of trial reached?
    Yes
    Global end of trial date
    08 Apr 2015
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    The main objective was to characterize single dose pharmacokinetic profiles of BeneFIX (50 IU/kg) in male Chinese subjects with hemophilia B.
    Protection of trial subjects
    The study was in compliance with the ethical principles derived from the Declaration of Helsinki and in compliance with all International Conference on Harmonization (ICH) Good Clinical Practice (GCP) Guidelines. All the local regulatory requirements pertinent to safety of trial subjects were followed.
    Background therapy
    -
    Evidence for comparator
    -
    Actual start date of recruitment
    02 Mar 2015
    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
    China: 12
    Worldwide total number of subjects
    12
    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)
    0
    Children (2-11 years)
    4
    Adolescents (12-17 years)
    0
    Adults (18-64 years)
    8
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    Twelve (12) subjects that were age 6 years or older (weight ≥20 kg) with moderate severe to severe hemophilia B (FIX activity ≤2%) were enrolled. Four (4) subjects were at the younger age range of ≥6 and <12 years; remaining subjects were 12 years or older.

    Pre-assignment
    Screening details
    Subjects did not receive an infusion of any FIX products for at least 4 days and were required to be in a non-bleeding state before the administration of BeneFIX on Day 1.

    Pre-assignment period milestones
    Number of subjects started
    12
    Number of subjects completed
    12

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

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    BeneFIX 50 IU/kg; Age group: >=6 and <12 years
    Arm description
    Subjects received a single dose of 50 IU/kg BeneFIX administered by IV infusion within 10 minutes at approximately 0800 hours (±2 hours) on Day 1.
    Arm type
    Experimental

    Investigational medicinal product name
    BeneFIX
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Powder and solution for solution for injection
    Routes of administration
    Intravenous use
    Dosage and administration details
    a single of BeneFIX 50 IU/kg by intravenous infusion

    Arm title
    BeneFIX 50 IU/kg; Age group: >=12 years
    Arm description
    Subejcts received a single dose of 50 IU/kg BeneFIX administered by IV infusion within 10 minutes at approximately 0800 hours (±2 hours) on Day 1.
    Arm type
    Experimental

    Investigational medicinal product name
    BeneFIX
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Powder and solution for solution for injection
    Routes of administration
    Intravenous use
    Dosage and administration details
    a single dose of BeneFIX 50 IU/kg by intravenous infusion

    Number of subjects in period 1
    BeneFIX 50 IU/kg; Age group: >=6 and <12 years BeneFIX 50 IU/kg; Age group: >=12 years
    Started
    4
    8
    Completed
    4
    8

    Baseline characteristics

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

    Reporting group values
    Overall Study Total
    Number of subjects
    12 12
    Age Categorical
    Units: Subjects
        In utero
    0 0
        Preterm newborn infants (gestational age < 37 wks)
    0 0
        Newborns (0-27 days)
    0 0
        Infants and toddlers (28 days-23 months)
    0 0
        Children (2-11 years)
    4 4
        Adolescents (12-17 years)
    0 0
        Adults (18-64 years)
    8 8
        From 65-84 years
    0 0
        85 years and over
    0 0
    Age Continuous
    Units: years
        arithmetic mean (standard deviation)
    24.8 ± 15.4 -
    Gender Categorical
    Units: Subjects
        Male
    12 12
        Female
    0 0

    End points

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    End points reporting groups
    Reporting group title
    BeneFIX 50 IU/kg; Age group: >=6 and <12 years
    Reporting group description
    Subjects received a single dose of 50 IU/kg BeneFIX administered by IV infusion within 10 minutes at approximately 0800 hours (±2 hours) on Day 1.

    Reporting group title
    BeneFIX 50 IU/kg; Age group: >=12 years
    Reporting group description
    Subejcts received a single dose of 50 IU/kg BeneFIX administered by IV infusion within 10 minutes at approximately 0800 hours (±2 hours) on Day 1.

    Primary: Maximum Observed Plasma Concentration (Cmax)

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    End point title
    Maximum Observed Plasma Concentration (Cmax) [1]
    End point description
    The pharmacokinetics (PK) parameter analysis population was defined as all subjects enrolled and treated who had at least 1 of the PK parameters of primary interest. All subjects were included in the PK analysis population.
    End point type
    Primary
    End point timeframe
    Pre-dose, 0.25, 0.5, 1, 3, 6, 9, 24, 50, 72 and 96 hours post-dose
    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: No statistical analyses were done for this end point.
    End point values
    BeneFIX 50 IU/kg; Age group: >=6 and <12 years BeneFIX 50 IU/kg; Age group: >=12 years
    Number of subjects analysed
    4
    8
    Units: IU/milliliter (IU/mL)
        geometric mean (geometric coefficient of variation)
    0.388 ± 26
    0.4226 ± 16
    No statistical analyses for this end point

    Primary: Area Under the Concentration Time Curve From Time 0 to the Time of the Last Quantifiable Concentration (AUClast)

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    End point title
    Area Under the Concentration Time Curve From Time 0 to the Time of the Last Quantifiable Concentration (AUClast) [2]
    End point description
    The pharmacokinetics (PK) parameter analysis population was defined as all subjects enrolled and treated who had at least 1 of the PK parameters of primary interest. All subjects were included in the PK analysis population.
    End point type
    Primary
    End point timeframe
    Pre-dose, 0.25, 0.5, 1, 3, 6, 9, 24, 50, 72 and 96 hours post-dose
    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: No statistical analyses were done for this end point.
    End point values
    BeneFIX 50 IU/kg; Age group: >=6 and <12 years BeneFIX 50 IU/kg; Age group: >=12 years
    Number of subjects analysed
    4
    8
    Units: IU*hour/milliliter (IU*hr/mL)
        geometric mean (geometric coefficient of variation)
    6.93 ± 18
    9.707 ± 15
    No statistical analyses for this end point

    Primary: Primary: Area Under the Concentration Time Curve From Time 0 to Infinity (AUCinf)

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    End point title
    Primary: Area Under the Concentration Time Curve From Time 0 to Infinity (AUCinf) [3]
    End point description
    The pharmacokinetics (PK) parameter analysis population was defined as all subjects enrolled and treated who had at least 1 of the PK parameters of primary interest. All subjects were included in the PK analysis population.
    End point type
    Primary
    End point timeframe
    Pre-dose, 0.25, 0.5, 1, 3, 6, 9, 24, 50, 72 and 96 hours post-dose
    Notes
    [3] - 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: No statistical analyses were done for this end point.
    End point values
    BeneFIX 50 IU/kg; Age group: >=6 and <12 years BeneFIX 50 IU/kg; Age group: >=12 years
    Number of subjects analysed
    4
    8
    Units: IU*hr/ml
        geometric mean (geometric coefficient of variation)
    7.841 ± 16
    11.66 ± 15
    No statistical analyses for this end point

    Primary: Time to Reach Cmax (Tmax)

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    End point title
    Time to Reach Cmax (Tmax) [4]
    End point description
    The pharmacokinetics (PK) parameter analysis population was defined as all subjects enrolled and treated who had at least 1 of the PK parameters of primary interest. All subjects were included in the PK analysis population.
    End point type
    Primary
    End point timeframe
    Pre-dose, 0.25, 0.5, 1, 3, 6, 9, 24, 50, 72 and 96 hours post-dose
    Notes
    [4] - 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: No statistical analyses were done for this end point.
    End point values
    BeneFIX 50 IU/kg; Age group: >=6 and <12 years BeneFIX 50 IU/kg; Age group: >=12 years
    Number of subjects analysed
    4
    8
    Units: hours
        median (full range (min-max))
    0.5 (0.25 to 3)
    0.375 (0.25 to 3)
    No statistical analyses for this end point

    Primary: Volume of Distribution at Steady State (Vss)

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    End point title
    Volume of Distribution at Steady State (Vss) [5]
    End point description
    The pharmacokinetics (PK) parameter analysis population was defined as all subjects enrolled and treated who had at least 1 of the PK parameters of primary interest. All subjects were included in the PK analysis population.
    End point type
    Primary
    End point timeframe
    Pre-dose, 0.25, 0.5, 1, 3, 6, 9, 24, 50, 72 and 96 hours post-dose
    Notes
    [5] - 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: No statistical analyses were done for this end point.
    End point values
    BeneFIX 50 IU/kg; Age group: >=6 and <12 years BeneFIX 50 IU/kg; Age group: >=12 years
    Number of subjects analysed
    4
    8
    Units: milliliter/kilogram (mL/kg)
        geometric mean (geometric coefficient of variation)
    227.9 ± 20
    218.8 ± 19
    No statistical analyses for this end point

    Primary: Terminal Phase Rate Constant (Kel)

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    End point title
    Terminal Phase Rate Constant (Kel) [6]
    End point description
    The pharmacokinetics (PK) parameter analysis population was defined as all subjects enrolled and treated who had at least 1 of the PK parameters of primary interest. All subjects were included in the PK analysis population.
    End point type
    Primary
    End point timeframe
    Pre-dose, 0.25, 0.5, 1, 3, 6, 9, 24, 50, 72 and 96 hours post-dose
    Notes
    [6] - 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: No statistical analyses were done for this end point.
    End point values
    BeneFIX 50 IU/kg; Age group: >=6 and <12 years BeneFIX 50 IU/kg; Age group: >=12 years
    Number of subjects analysed
    4
    8
    Units: 1/hr
        geometric mean (geometric coefficient of variation)
    0.02509 ± 16
    0.01781 ± 20
    No statistical analyses for this end point

    Primary: Mean Residence Time (MRT)

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    End point title
    Mean Residence Time (MRT) [7]
    End point description
    The pharmacokinetics (PK) parameter analysis population was defined as all subjects enrolled and treated who had at least 1 of the PK parameters of primary interest. All subjects were included in the PK analysis population.
    End point type
    Primary
    End point timeframe
    Pre-dose, 0.25, 0.5, 1, 3, 6, 9, 24, 50, 72 and 96 hours post-dose
    Notes
    [7] - 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: No statistical analyses were done for this end point.
    End point values
    BeneFIX 50 IU/kg; Age group: >=6 and <12 years BeneFIX 50 IU/kg; Age group: >=12 years
    Number of subjects analysed
    4
    8
    Units: hours
        geometric mean (geometric coefficient of variation)
    35.78 ± 14
    51.01 ± 16
    No statistical analyses for this end point

    Primary: Plasma Decay Half-Life (t½)

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    End point title
    Plasma Decay Half-Life (t½) [8]
    End point description
    The pharmacokinetics (PK) parameter analysis population was defined as all subjects enrolled and treated who had at least 1 of the PK parameters of primary interest. All subjects were included in the PK analysis population.
    End point type
    Primary
    End point timeframe
    Pre-dose, 0.25, 0.5, 1, 3, 6, 9, 24, 50, 72 and 96 hours post-dose
    Notes
    [8] - 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: No statistical analyses were done for this end point.
    End point values
    BeneFIX 50 IU/kg; Age group: >=6 and <12 years BeneFIX 50 IU/kg; Age group: >=12 years
    Number of subjects analysed
    4
    8
    Units: hours
        arithmetic mean (standard deviation)
    27.88 ± 4.4903
    39.56 ± 7.3832
    No statistical analyses for this end point

    Primary: Systemic Clearance (CL)

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    End point title
    Systemic Clearance (CL) [9]
    End point description
    The pharmacokinetics (PK) parameter analysis population was defined as all subjects enrolled and treated who had at least 1 of the PK parameters of primary interest. All subjects were included in the PK analysis population.
    End point type
    Primary
    End point timeframe
    Pre-dose, 0.25, 0.5, 1, 3, 6, 9, 24, 50, 72 and 96 hours post-dose
    Notes
    [9] - 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: No statistical analyses were done for this end point.
    End point values
    BeneFIX 50 IU/kg; Age group: >=6 and <12 years BeneFIX 50 IU/kg; Age group: >=12 years
    Number of subjects analysed
    4
    8
    Units: mL/hr/kg
        geometric mean (geometric coefficient of variation)
    6.378 ± 16
    4.291 ± 15
    No statistical analyses for this end point

    Primary: Incremental Recovery

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    End point title
    Incremental Recovery [10]
    End point description
    The pharmacokinetics (PK) parameter analysis population was defined as all subjects enrolled and treated who had at least 1 of the PK parameters of primary interest. All subjects were included in the PK analysis population.
    End point type
    Primary
    End point timeframe
    Pre-dose, 0.25, 0.5 and 1 hour post-dose
    Notes
    [10] - 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: No statistical analyses were done for this end point.
    End point values
    BeneFIX 50 IU/kg; Age group: >=6 and <12 years BeneFIX 50 IU/kg; Age group: >=12 years
    Number of subjects analysed
    4
    8
    Units: (IU/dL)/(IU/Kg)
        geometric mean (geometric coefficient of variation)
    0.7759 ± 26
    0.8199 ± 17
    No statistical analyses for this end point

    Secondary: Number of Subjects With Abnormal Clinical Laboratory Measurements (Without Regard to Baseline Abnormality)

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    End point title
    Number of Subjects With Abnormal Clinical Laboratory Measurements (Without Regard to Baseline Abnormality)
    End point description
    Clinical laboratory analysis tests included hematology, serium chemistry, prothrombin time and urianalysis. Numbers of subjects with laboratory test abnormalities without regard to baseline abnormality were reported.
    End point type
    Secondary
    End point timeframe
    Screening, Day 0 and 96 hours post-dose (Day 5 or early termination)
    End point values
    BeneFIX 50 IU/kg; Age group: >=6 and <12 years BeneFIX 50 IU/kg; Age group: >=12 years
    Number of subjects analysed
    4
    8
    Units: subjects
    4
    8
    No statistical analyses for this end point

    Secondary: Number of Subjects With Vital Signs Post-Dose Data Met Criteria of Potential Clinical Concern (Without Regard to Baseline Abnormality)

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    End point title
    Number of Subjects With Vital Signs Post-Dose Data Met Criteria of Potential Clinical Concern (Without Regard to Baseline Abnormality)
    End point description
    The safety analysis population included All participants who received at least 1 dose of BeneFIX.
    End point type
    Secondary
    End point timeframe
    Screening, pre-dose (baseline) and 96 hours post-dose (Day 5 or early termination)
    End point values
    BeneFIX 50 IU/kg; Age group: >=6 and <12 years BeneFIX 50 IU/kg; Age group: >=12 years
    Number of subjects analysed
    4
    8 [11]
    Units: subjects
    1
    0
    Notes
    [11] - None of the vital signs values met the criteria for potential clinical concern.
    No statistical analyses for this end point

    Secondary: Number of Subjects with inhibitor development

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    End point title
    Number of Subjects with inhibitor development
    End point description
    The safety analysis population included All subjects who received at least 1 dose of BeneFIX.
    End point type
    Secondary
    End point timeframe
    Screening, Day 0 and 96 hours post-dose (Day 5 or early termination)
    End point values
    BeneFIX 50 IU/kg; Age group: >=6 and <12 years BeneFIX 50 IU/kg; Age group: >=12 years
    Number of subjects analysed
    4 [12]
    8 [13]
    Units: subjects
    0
    0
    Notes
    [12] - There were no subjects with inhibitor development.
    [13] - There were no subjects with inhibitor development.
    No statistical analyses for this end point

    Secondary: Number of Subjects with allergic reactions

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    End point title
    Number of Subjects with allergic reactions
    End point description
    The safety analysis population included all subjects who received at least 1 dose of BeneFIX.
    End point type
    Secondary
    End point timeframe
    Baseline up to 28 calendar days post the last administration of study drug
    End point values
    BeneFIX 50 IU/kg; Age group: >=6 and <12 years BeneFIX 50 IU/kg; Age group: >=12 years
    Number of subjects analysed
    4 [14]
    8 [15]
    Units: subjects
    0
    0
    Notes
    [14] - There were no subjects with allergic reactions.
    [15] - There were no subjects with allergic reactions.
    No statistical analyses for this end point

    Secondary: Number of subjects with thrombogenicity

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    End point title
    Number of subjects with thrombogenicity
    End point description
    The safety analysis population included all subjects who received at least 1 dose of BeneFIX.
    End point type
    Secondary
    End point timeframe
    Day 0 and 96 hours post-dose (Day 5 or early termination)
    End point values
    BeneFIX 50 IU/kg; Age group: >=6 and <12 years BeneFIX 50 IU/kg; Age group: >=12 years
    Number of subjects analysed
    4 [16]
    8 [17]
    Units: subjects
    0
    0
    Notes
    [16] - There were no subjects with thrombogenicity.
    [17] - There were no subjects with thrombogenicity.
    No statistical analyses for this end point

    Adverse events

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    Adverse events information [1]
    Timeframe for reporting adverse events
    Baseline up to 28 calendar days post the last administration of study drug (BeneFIX).
    Assessment type
    Non-systematic
    Dictionary used for adverse event reporting
    Dictionary name
    MedDRA
    Dictionary version
    17.1
    Reporting groups
    Reporting group title
    BeneFIX 50 IU/kg; Age group: >=12 years
    Reporting group description
    Subejcts received a single dose of 50 IU/kg BeneFIX administered by IV infusion within 10 minutes at approximately 0800 hours (±2 hours) on Day 1.

    Reporting group title
    BeneFIX 50 IU/kg; Age group: >=6 and <12 years
    Reporting group description
    Subjects received a single dose of 50 IU/kg BeneFIX administered by IV infusion within 10 minutes at approximately 0800 hours (±2 hours) on Day 1.

    Serious adverse events
    BeneFIX 50 IU/kg; Age group: >=12 years BeneFIX 50 IU/kg; Age group: >=6 and <12 years
    Total subjects affected by serious adverse events
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 4 (0.00%)
         number of deaths (all causes)
    0
    0
         number of deaths resulting from adverse events
    0
    0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    BeneFIX 50 IU/kg; Age group: >=12 years BeneFIX 50 IU/kg; Age group: >=6 and <12 years
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 4 (0.00%)
    Notes
    [1] - There are no non-serious adverse events recorded for these results. It is expected that there will be at least one non-serious adverse event reported.
    Justification: There were no adverse events reported in this study.

    More information

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    Substantial protocol amendments (globally)

    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    08 Aug 2014
    Schedule of activities, Section 3.1, Section 6.2.2 & 6.2.3, and Section 7.3.1: PK collection timpoints: 96 hours post dose is added for all subjects as it will provide additional information on the PK profile. 48 hour post dose is deleted for subjects who are 12 years old since the 48 hour and 50 hour are very close to each other and the concentrations observed in other trials at these two times are quite similar to each other. Section 6.2.2: The languages regarding the collection time of PK FIX activity samples relevant to infusion are revised from completion of the infusion to start of the infusion as the drug enters the blood at the start of the infusion, thus time ‘zero’ is the start of the infusion. Exclusion criterion #24, and Section 4.4.4: Contraception are deleted as there is no label restriction requiring contraception in males taking Benefix. Section 1.3.1: a statement regarding no contraception requirement on male subjects is added. Exclusion criteria 10, 11, 12 is combined as one criterion and standard language is added. Schedule of activities, Section 3.1 & Section 6.2.1: The requirement on stay in CRU is revised from completion of DAY4/early termination visit activities to completion of the 24 hours post dose PK sample collections, after that, it will be at investigator’s discretion on whether the subjects should stay in CRU or not. The visits that follow hour 24 could be conducted outpatient at investigator’s discretion. For Pediatric subjects, their parents/legal representatives will accompany them during the study if applicable. K–value is replaced by incremental recovery throughout the protocol as K–value is a past used term for incremental recovery and should not continue to be used. Updated Blood Volume table for subjects 6 <12 years old from 55.1 mL to 57.8 mL. Section 6.1.1: height is added to Demography. Section 7.3.3.: FIX inhibitor analysis is added. Pfizer protocol standard language update.

    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
    The status of studies in GB is no longer updated from 1.1.2021
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