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    Clinical Trial Results:
    An Evaluation of the Safety and Efficacy of On-Demand Treatment with Xyntha (B-Domain Deleted Recombinant Factor VIII, Albumin Free) in Chinese Subjects with Hemophilia A

    Summary
    EudraCT number
    2014-004106-15
    Trial protocol
    Outside EU/EEA  
    Global end of trial date
    04 Dec 2009

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

    Trial information

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    Trial identification
    Sponsor protocol code
    3082B2-3316
    Additional study identifiers
    ISRCTN number
    -
    US NCT number
    NCT00868530
    WHO universal trial number (UTN)
    -
    Other trial identifiers
    Alias: B1831015
    Sponsors
    Sponsor organisation name
    Pfizer Inc.
    Sponsor organisation address
    235 E 42nd Street, New York, United States, NY 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
    27 Jun 2010
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    04 Dec 2009
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    To describe the safety and clinical efficacy of Xyntha in previously treated Chinese subjects with hemophilia A.
    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
    18 Sep 2008
    Long term follow-up planned
    Yes
    Long term follow-up rationale
    Safety
    Long term follow-up duration
    1 Months
    Independent data monitoring committee (IDMC) involvement?
    Yes
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    China: 53
    Worldwide total number of subjects
    53
    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)
    6
    Adolescents (12-17 years)
    11
    Adults (18-64 years)
    36
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    Subjects were recruited in China from September 2008 to December 2009.

    Pre-assignment
    Screening details
    Overall, 55 subjects were screened, out of which 53 subjects were enrolled in the study.

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

    Arms
    Arm title
    Xyntha
    Arm description
    Subjects received on-demand treatments with Xyntha (which occurred each time a subject experienced bleeding episode during the active phase of the study) according to investigator’s prescription over a 6-month (calendar day) period.
    Arm type
    Experimental

    Investigational medicinal product name
    Xyntha
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Powder and solvent for solution for injection/infusion
    Routes of administration
    Intravenous bolus use
    Dosage and administration details
    A single 50 International Unit (IU)/kg (+/-5 IU/kg) intravenous (IV) bolus infusion of Xyntha was given for recovery assessments.

    Number of subjects in period 1
    Xyntha
    Started
    53
    Completed
    49
    Not completed
    4
         Adverse Event
    3
         Lost to follow-up
    1

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Xyntha
    Reporting group description
    Subjects received on-demand treatments with Xyntha (which occurred each time a subject experienced bleeding episode during the active phase of the study) according to investigator’s prescription over a 6-month (calendar day) period.

    Reporting group values
    Xyntha Total
    Number of subjects
    53 53
    Age categorical
    Units: Subjects
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    23.2 ± 10 -
    Gender categorical
    Units: Subjects
        Female
    0 0
        Male
    53 53

    End points

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    End points reporting groups
    Reporting group title
    Xyntha
    Reporting group description
    Subjects received on-demand treatments with Xyntha (which occurred each time a subject experienced bleeding episode during the active phase of the study) according to investigator’s prescription over a 6-month (calendar day) period.

    Primary: Investigator Hemostatic Efficacy Assessment 8 Hours Post Infusion

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    End point title
    Investigator Hemostatic Efficacy Assessment 8 Hours Post Infusion [1]
    End point description
    The Investigator Hemostatic Efficacy Assessment was based on a 4-point rating scale (Excellent = 1: definite pain relief or improvement in signs of bleeding, with no additional infusion, Good = 2: definite pain relief or improvement in signs of bleeding, Moderate = 3: probable or slight improvement, No Response = 4: no improvement at all between infusions). The Full Analysis Set (FAS) consisted of all subjects who were treated and had at least 1 evaluable efficacy assessment after treatment.
    End point type
    Primary
    End point timeframe
    8 hours post infusion
    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: Only descriptive data was planned to be reported for this end point.
    End point values
    Xyntha
    Number of subjects analysed
    51
    Units: Units on a scale
        arithmetic mean (standard deviation)
    1.86 ± 0.65
    No statistical analyses for this end point

    Primary: Investigator Hemostatic Efficacy Assessment 24 Hours Post Infusion

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    End point title
    Investigator Hemostatic Efficacy Assessment 24 Hours Post Infusion [2]
    End point description
    The Investigator Hemostatic Efficacy Assessment was based on a 4-point rating scale (Excellent = 1: definite pain relief or improvement in signs of bleeding, with no additional infusion, Good = 2: definite pain relief or improvement in signs of bleeding, Moderate = 3: probable or slight improvement, No Response = 4: no improvement at all between infusions). The FAS consisted of all subjects who were treated and had at least 1 evaluable efficacy assessment after treatment.
    End point type
    Primary
    End point timeframe
    24 hours post infusion
    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: Only descriptive data was planned to be reported for this end point.
    End point values
    Xyntha
    Number of subjects analysed
    51
    Units: Units on a scale
        arithmetic mean (standard deviation)
    1.74 ± 0.61
    No statistical analyses for this end point

    Primary: Number of Subjects With Factor VIII (FVIII) Inhibitor Development

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    End point title
    Number of Subjects With Factor VIII (FVIII) Inhibitor Development [3]
    End point description
    Incidence of FVIII inhibitor was defined as any result determined as positive at local laboratory, and confirmed at central laboratory. Incidence was stratified by subject exposure history: Minimally Treated Patients (MTPs): those who had received at least 1 prior FVIII infusion, and less than or equal to (<=) 100 documented Exposure Days (EDs), while Previously Treated Patients (PTPs): those who had received greater than (>) 100 documented prior EDs. When number of prior EDs for an individual was not known to be at least 100, subjects were included in the MTP population. The FAS consisted of all subjects who were treated and had at least 1 evaluable efficacy assessment after treatment.
    End point type
    Primary
    End point timeframe
    Day 1 and Month 6 or Early Termination Visit
    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: Only descriptive data was planned to be reported for this end point.
    End point values
    Xyntha
    Number of subjects analysed
    51
    Units: subjects
        MTP (n=34)
    6
        PTP (n=17)
    1
    No statistical analyses for this end point

    Primary: FVIII Recovery : Change From Baseline in FVIII Concentration

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    End point title
    FVIII Recovery : Change From Baseline in FVIII Concentration [4]
    End point description
    FVIII recovery was assessed by evaluating the change in FVIII concentration at 6 months compared to baseline. The FAS consisted of all subjects who were treated and had at least 1 evaluable efficacy assessment after treatment. Subjects with missing data were not included.
    End point type
    Primary
    End point timeframe
    Day 1 and Month 6 or Early Termination Visit
    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: Statistical analysis has been provided separately as an attachment.
    End point values
    Xyntha
    Number of subjects analysed
    42
    Units: IU/dL per IU/kg
        arithmetic mean (standard deviation)
    -0.11 ± 0.45
    Attachments
    Statistical Analyses for FVIII Recovery
    No statistical analyses for this end point

    Secondary: Number of Subjects With Less Than Expected Therapeutic Effect (LETE)

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    End point title
    Number of Subjects With Less Than Expected Therapeutic Effect (LETE)
    End point description
    The incidence of LETE, defined for on-demand treatment as no response after each of 2 successive infusions within 24 hours for the same bleeding event in the absence of confounding factors. The FAS consisted of all subjects who were treated and had at least 1 evaluable efficacy assessment after treatment.
    End point type
    Secondary
    End point timeframe
    24 hours after each of 2 successive infusion, up to 6 months
    End point values
    Xyntha
    Number of subjects analysed
    51
    Units: subjects
    0
    No statistical analyses for this end point

    Secondary: Number of Subjects With Thrombosis Allergic-Type Reactions

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    End point title
    Number of Subjects With Thrombosis Allergic-Type Reactions
    End point description
    The Safety Set (SS) consisted of all subjects who had taken at least 1 dose of investigational drug.
    End point type
    Secondary
    End point timeframe
    Baseline up to 6 months
    End point values
    Xyntha
    Number of subjects analysed
    53
    Units: subjects
    1
    No statistical analyses for this end point

    Secondary: Number of Subjects With Thrombosis

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    End point title
    Number of Subjects With Thrombosis
    End point description
    The SS consisted of all subjects who had taken at least 1 dose of investigational drug.
    End point type
    Secondary
    End point timeframe
    Baseline up to 6 months
    End point values
    Xyntha
    Number of subjects analysed
    53
    Units: subjects
    0
    No statistical analyses for this end point

    Other pre-specified: Frequency of Xyntha Infusions Required Per Hemorrhage

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    End point title
    Frequency of Xyntha Infusions Required Per Hemorrhage
    End point description
    The mean frequency of Xyntha infusions per hemorrhage was calculated as total number of injections throughout the study divided by total number of hemorrhagic events. The FAS consisted of all subjects who were treated and had at least 1 evaluable efficacy assessment after treatment.
    End point type
    Other pre-specified
    End point timeframe
    Day 1 to Month 6 or Early Termination Visit
    End point values
    Xyntha
    Number of subjects analysed
    51
    Units: Infusions
        arithmetic mean (standard deviation)
    1.16 ± 0.72
    No statistical analyses for this end point

    Other pre-specified: Average Dose of Xyntha Infusions Required Per Hemorrhage

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    End point title
    Average Dose of Xyntha Infusions Required Per Hemorrhage
    End point description
    The average dose of Xyntha per hemorrhagic event was calculated as total dose of Xyntha throughout the study (in IU) divided by total number of hemorrhage incidence. The FAS consisted of all subjects who were treated and had at least 1 evaluable efficacy assessment after treatment.
    End point type
    Other pre-specified
    End point timeframe
    Day 1 to Month 6 or Early Termination Visit
    End point values
    Xyntha
    Number of subjects analysed
    51
    Units: Dose/Bleed (IU)
        arithmetic mean (standard deviation)
    1226.28 ± 1208.49
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Adverse Events (AEs) and Serious AEs (SAEs) were collected from the signing of the informed consent form to approximately 30 days following the Month 6/Final/Early Termination visit
    Adverse event reporting additional description
    Safety population: who received at least 1 dose of Xyntha. Same event may appear as AE, SAE. However,what is presented are distinct events. An event may be categorized as serious in 1 subject and nonserious in another,or 1 subject may experience both SAE, Non-SAE. EU BR AE tables generated separately as per EU format using latest coding dictionary.
    Assessment type
    Non-systematic
    Dictionary used for adverse event reporting
    Dictionary name
    MedDRA
    Dictionary version
    17.1
    Reporting groups
    Reporting group title
    Xyntha
    Reporting group description
    Subjects received on-demand treatments with Xyntha (which occurred each time a subject experienced bleeding episode during the active phase of the study) according to investigator’s prescription over a 6-month (calendar day) period. A single 50 International Unit (IU)/kg (+/-5 IU/kg) intravenous (IV) bolus infusion of Xyntha was given for recovery assessments.

    Serious adverse events
    Xyntha
    Total subjects affected by serious adverse events
         subjects affected / exposed
    8 / 53 (15.09%)
         number of deaths (all causes)
    0
         number of deaths resulting from adverse events
    0
    Investigations
    Anti factor VIII antibody positive
         subjects affected / exposed
    6 / 53 (11.32%)
         occurrences causally related to treatment / all
    5 / 6
         deaths causally related to treatment / all
    0 / 0
    Investigation
         subjects affected / exposed
    2 / 53 (3.77%)
         occurrences causally related to treatment / all
    2 / 2
         deaths causally related to treatment / all
    0 / 0
    Gastrointestinal disorders
    Gastrointestinal haemorrhage
         subjects affected / exposed
    1 / 53 (1.89%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Gingival bleeding
         subjects affected / exposed
    1 / 53 (1.89%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 0%
    Non-serious adverse events
    Xyntha
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    12 / 53 (22.64%)
    Vascular disorders
    Haemorrhage
         subjects affected / exposed
    1 / 53 (1.89%)
         occurrences all number
    1
    Injury, poisoning and procedural complications
    Fall
         subjects affected / exposed
    1 / 53 (1.89%)
         occurrences all number
    1
    Injury
         subjects affected / exposed
    4 / 53 (7.55%)
         occurrences all number
    4
    Joint dislocation
         subjects affected / exposed
    1 / 53 (1.89%)
         occurrences all number
    1
    Joint injury
         subjects affected / exposed
    3 / 53 (5.66%)
         occurrences all number
    3
    Ligament sprain
         subjects affected / exposed
    1 / 53 (1.89%)
         occurrences all number
    2
    Limb injury
         subjects affected / exposed
    1 / 53 (1.89%)
         occurrences all number
    1
    Investigations
    Blood potassium decreased
         subjects affected / exposed
    2 / 53 (3.77%)
         occurrences all number
    2
    General disorders and administration site conditions
    Pyrexia
         subjects affected / exposed
    1 / 53 (1.89%)
         occurrences all number
    1
    Gastrointestinal disorders
    Diarrhoea
         subjects affected / exposed
    1 / 53 (1.89%)
         occurrences all number
    1
    Skin and subcutaneous tissue disorders
    Dermatitis allergic
         subjects affected / exposed
    1 / 53 (1.89%)
         occurrences all number
    2
    Musculoskeletal and connective tissue disorders
    Arthritis
         subjects affected / exposed
    1 / 53 (1.89%)
         occurrences all number
    1

    More information

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    Substantial protocol amendments (globally)

    Were there any global substantial amendments to the protocol? No

    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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