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    Clinical Trial Results:
    A multicenter, prospective, open-label, clinical study to assess the effect of using a new risk score approach to select the most appropriate prophylaxis regimen for reaching a favorable outcome, when hemophilia A patients switch from standard half-life (SHL) products to damoctocog alfa pegol (Jivi)

    Summary
    EudraCT number
    		 2021-006191-16
    Trial protocol
    		 Outside EU/EEA  
    Global end of trial date
    17 Oct 2024

    Results information
    Results version number
    		 v1(current)
    This version publication date
    09 Apr 2025
    First version publication date
    09 Apr 2025
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    BAY94-9027/21924
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT05036278
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Bayer HealthCare Pharmaceuticals Inc.
    Sponsor organisation address
    100 Bayer Boulevard, P.O. Box 915, Whippany, United States,
    Public contact
    Bayer Clinical Trials Contact, Bayer AG, clinical-trials-contact@bayer.com
    Scientific contact
    Bayer Clinical Trials Contact, 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?
    No
    Results analysis stage
    Analysis stage
    Final
    Date of interim/final analysis
    17 Jan 2025
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    17 Oct 2024
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    To assess the effect of using a baseline risk score, based on a participant’s phenotypic and biologic variables, to select the most appropriate prophylaxis regimen for reaching a favorable outcome, when switching from a SHL product to Jivi
    Protection of trial subjects
    The conduct of this clinical study met all local legal and regulatory requirements. The study was conducted in accordance with ethical principles that have their origin in the Declaration of Helsinki and the International Council for Harmonization guideline E6: Good Clinical Practice. Before entering the study, the informed consent was read by and explained to all the subjects. Participating subjects 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 Jul 2022
    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
    United States: 21
    Worldwide total number of subjects
    21
    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)
    0
    Adolescents (12-17 years)
    3
    Adults (18-64 years)
    17
    From 65 to 84 years
    1
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 The study was conducted at 14 study centers in the US between 28 July 2022 (first particpant first visit) and 03 September 2024 (last participant last visit).

    Pre-assignment
    Screening details
    		 A total of 21 participants were enrolled. 21 participants were assigned to treatment.

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

    Arms
    Arm title
    		 overall
    Arm description
    		 all participants enrolled
    Arm type
    		 Experimental

    Investigational medicinal product name
    Jivi
    Investigational medicinal product code
    Other name
    BAY94-9027, Damoctocog alfa pegol, recombinant coagulation FVIII
    Pharmaceutical forms
    Powder for solution for infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    vial sizes of 500 and 2000 IU international unit(s) All eligible participants will start treatment 2x/week (40 IU/kg/dose) with Jivi for 4 weeks. Treatment will then continue according to their assignment to 1 of the 3 following prophylaxis regimens: • Participants with a high risk score (> 4) continue on prophylaxis 2x/week (40 IU/kg/dose) • Participants with a medium risk score (2 to 4) will switch after 4 weeks to prophylaxis Q5D (50 IU/kg/dose) • Participants with a low risk score (< 2) will switch after 4 weeks to prophylaxis Q5D (50 IU/kg/dose) and then after 4 weeks to a less frequent (e.g. Q7D) regimen (60 IU/kg/dose)

    Number of subjects in period 1
    		overall
    Started
    21
    Completed
    19
    Not completed
    2
         Consent withdrawn by subject
    1
         Adverse event, non-fatal
    1

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    overall
    Reporting group description
    		 -

    Reporting group values
    		 overall Total
    Number of subjects
    		 21 21
    Age categorical
    Units: Subjects
        Adolescents (12-17 years)
    		 3 3
        Adults (18-64 years)
    		 17 17
        From 65-84 years
    		 1 1
    Gender categorical
    Units: Subjects
        Female
    		 2 2
        Male
    		 19 19
    Subject analysis sets

    Subject analysis set title
    Modified intent to treat set
    Subject analysis set type
    		 Modified intention-to-treat
    Subject analysis set description
    All participants enrolled and followed for at least 4 months

    Subject analysis set title
    Safety analysis set
    Subject analysis set type
    		 Safety analysis
    Subject analysis set description
    all participants enrolled, who received at least one dose of Jivi

    Subject analysis set title
    Censored modified intent to treat set
    Subject analysis set type
    		 Modified intention-to-treat
    Subject analysis set description
    all patients enrolled, followed for at least 4 months and not having switched from the score assigned regimen

    Subject analysis sets values
    		 Modified intent to treat set Safety analysis set Censored modified intent to treat set
    Number of subjects
    19
    21
    17
    Age categorical
    Units: Subjects
        Adolescents (12-17 years)
    3
    3
    3
        Adults (18-64 years)
    15
    17
    13
        From 65-84 years
    1
    1
    1
    Age continuous
    Units:
        
    ( )
    ( )
    ( )
    Gender categorical
    Units: Subjects
        Female
    2
    2
    2
        Male
    17
    19
    15

    End points

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    End points reporting groups
    Reporting group title
    overall
    Reporting group description
    		 all participants enrolled

    Subject analysis set title
    Modified intent to treat set
    Subject analysis set type
    		 Modified intention-to-treat
    Subject analysis set description
    All participants enrolled and followed for at least 4 months

    Subject analysis set title
    Safety analysis set
    Subject analysis set type
    		 Safety analysis
    Subject analysis set description
    all participants enrolled, who received at least one dose of Jivi

    Subject analysis set title
    Censored modified intent to treat set
    Subject analysis set type
    		 Modified intention-to-treat
    Subject analysis set description
    all patients enrolled, followed for at least 4 months and not having switched from the score assigned regimen

    Primary: Occurrence of favorable outcome on the score selected dosing regimen

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    End point title
    		 Occurrence of favorable outcome on the score selected dosing regimen [1]
    End point description
    To assess the effect of using a baseline risk score, based on a participant's phenotypic and biologic variables, to select the most appropriate prophylaxis regimen for reaching a favorable outcome, when switching from a standard half-life (SHL) product to Jivi. t_140201 Table 14.2/3 (censored modified intention to treat set)
    End point type
    Primary
    End point timeframe
    up to 6 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: Only descriptive statistics were applied in this study due to low number of participants. As this is a single arm study, no comparative statistics were applied.
    End point values
    		 Censored modified intent to treat set
    Number of subjects analysed
    17
    Units: participants with favorable outcome
    12
    No statistical analyses for this end point

    Secondary: Annualized bleeding rate (ABR) (total, joint, spontaneous)

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    End point title
    		 Annualized bleeding rate (ABR) (total, joint, spontaneous)
    End point description
    To assess the efficacy of Jivi compared to a previous SHL treatment. t_140207
    End point type
    Secondary
    End point timeframe
    up to 6 months
    End point values
    		 Censored modified intent to treat set
    Number of subjects analysed
    17
    Units: bleeds per year
    arithmetic mean (standard deviation)
        total bleeds
    4.172 ( 5.516 )
        joint bleeds
    2.800 ( 4.160 )
        spontaneous bleeds
    1.788 ( 3.968 )
    No statistical analyses for this end point

    Secondary: Change in total ABR from pre-study

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    End point title
    		 Change in total ABR from pre-study
    End point description
    To assess the efficacy of Jivi compared to a previous SHL treatment. Table 14.2/6.1
    End point type
    Secondary
    End point timeframe
    up to 6 months
    End point values
    		 Censored modified intent to treat set
    Number of subjects analysed
    17
    Units: annual bleeding rate
        arithmetic mean (standard deviation)
    -8.005 ( 15.242 )
    No statistical analyses for this end point

    Secondary: Change in the frequency of pre-study SHL treatment to the frequency of Jivi administration (infusions/month)

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    End point title
    		 Change in the frequency of pre-study SHL treatment to the frequency of Jivi administration (infusions/month)
    End point description
    To assess the frequency of Jivi administration. t_140116 Table 14.1/16
    End point type
    Secondary
    End point timeframe
    up to 6 months
    End point values
    		 Censored modified intent to treat set
    Number of subjects analysed
    17
    Units: infusions per month
        arithmetic mean (standard deviation)
    -6.876 ( 4.7557 )
    No statistical analyses for this end point

    Secondary: Occurrence of participants with 0 and ≤ 1 spontaneous bleeds

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    End point title
    		 Occurrence of participants with 0 and ≤ 1 spontaneous bleeds
    End point description
    To assess the proportion of participants with 0 and ≤ 1 spontaneous bleeds. Table 14.2/9
    End point type
    Secondary
    End point timeframe
    up to 6 months
    End point values
    		 Modified intent to treat set
    Number of subjects analysed
    19
    Units: participants
        0 spontaneous bleed
    13
        <=1 spontaneous bleed
    13
    No statistical analyses for this end point

    Secondary: Change in Haemophilia Quality of Life Questionnaire (Haem-A-QoL or Haemo-QoL)

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    End point title
    		 Change in Haemophilia Quality of Life Questionnaire (Haem-A-QoL or Haemo-QoL)
    End point description
    To assess participant quality of life (QoL) and physical activity, as measured by Patient Reported Outcomes (PROs). Table 14.2/13
    End point type
    Secondary
    End point timeframe
    up to 6 months
    End point values
    		 Modified intent to treat set
    Number of subjects analysed
    10 [2]
    Units: Score
    arithmetic mean (standard deviation)
        Visit 2 (Baseline)
    31.5 ( 16.38 )
        Visit 6 (Change from Baseline)
    -4.7 ( 11.7 )
    Notes
    [2] - for visit 2, 14 participants, for visit 6, 10 participants
    No statistical analyses for this end point

    Secondary: Patient's Global Impression of Change (PGI-C)

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    End point title
    		 Patient's Global Impression of Change (PGI-C)
    End point description
    To assess participant quality of life (QoL) and physical activity, as measured by Patient Reported Outcomes (PROs). Table 14.2/15
    End point type
    Secondary
    End point timeframe
    up to 6 months
    End point values
    		 Modified intent to treat set
    Number of subjects analysed
    19 [3]
    Units: Patient reported outcomes
        status minimally improved
    1
        status minimally worse
    1
        status much improved
    2
        status not changed
    4
        status very much improved
    1
    Notes
    [3] - Not all participants completed this PRO.
    No statistical analyses for this end point

    Secondary: EuroQoL 5 Dimensions (EQ-5D-5L) questionnaire

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    End point title
    		 EuroQoL 5 Dimensions (EQ-5D-5L) questionnaire
    End point description
    To assess participant quality of life (QoL) and physical activity, as measured by Patient Reported Outcomes (PROs). Change to baseline means changes between Visit 2 and Visit 6 Table 14.2/16
    End point type
    Secondary
    End point timeframe
    up to 6 months
    End point values
    		 Modified intent to treat set
    Number of subjects analysed
    19
    Units: Patient reported outcome
        (walking) improved, less impaired
    1
        (walking) no change
    5
        (walking) worsened, more impaired
    5
        (self-care) improved, less impaired
    1
        (self-care) no change
    10
        (self-care) worsened, more impaired
    0
        (usual activities) improved, less impaired
    0
        (usual activities) no change
    11
        (usual activities) worsened, more impaired
    0
        (pain or discomfort) improved, less impaired
    0
        (pain or discomfort) no change
    9
        (pain or discomfort) worsened, more impaired
    2
        (anxious or depressed) improved, less impaired
    2
        (anxious or depressed) no change
    6
        (anxious or depressed) worsened, more impaired
    3
    No statistical analyses for this end point

    Secondary: Treatment Satisfaction Questionnaire for Medication (TSQM)

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    End point title
    		 Treatment Satisfaction Questionnaire for Medication (TSQM)
    End point description
    To assess participant quality of life (QoL) and physical activity, as measured by Patient Reported Outcomes (PROs). Score ranges from 10 (lowest satisfaction) to 100 (greatest satisfaction). Table 14.2/17
    End point type
    Secondary
    End point timeframe
    up to 6 months
    End point values
    		 Modified intent to treat set
    Number of subjects analysed
    5 [4]
    Units: score
    arithmetic mean (standard deviation)
        Visit 2 (baseline)
    37.9 ( 7.44 )
        Visit 6 (change from baseline)
    -1.8 ( 6.42 )
    Notes
    [4] - At visit 2, 11 participants, at visit 6, 5 participants
    No statistical analyses for this end point

    Secondary: Work Productivity and Activity Impairment (WPAI) questionnaire scores

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    End point title
    		 Work Productivity and Activity Impairment (WPAI) questionnaire scores
    End point description
    To assess participant quality of life (QoL) and physical activity, as measured by Patient Reported Outcomes (PROs). Baseline = Visit 2, Change from Baseline = Visit 6 Table 14.2/18
    End point type
    Secondary
    End point timeframe
    up to 6 months
    End point values
    		 Modified intent to treat set
    Number of subjects analysed
    4 [5]
    Units: score
    arithmetic mean (standard deviation)
        Percent Work Time Missed (baseline)
    1.8 ( 4.64 )
        Percent Work Time Missed (change from baseline)
    19.4 ( 24.21 )
        Percent Working Impairment (baseline)
    26.7 ( 22.51 )
        Percent Working Impairment (change from baseline)
    10.0 ( 20.00 )
        Percent Class Time Missed (baseline)
    5.0 ( 10.00 )
        Percent Class Time Missed (change from baseline)
    1.4 ( 1.96 )
        Percent Class Attending Impairment (baseline)
    15.0 ( 19.15 )
        Percent Class Attending Impairment (change)
    0 ( 28.28 )
        Percent Daily Activities Impairment (baseline)
    33.3 ( 29.34 )
        Percent Daily Activities Impairment (change)
    -7.1 ( 32.00 )
        Average Impairment Score (baseline)
    32.9 ( 27.01 )
        Average Impairment Score (change from baseline)
    -10.7 ( 23.53 )
    Notes
    [5] - Participants' number varied depending on parameter between 2 and 7 participants
    No statistical analyses for this end point

    Secondary: Number of target joints and change in target joint status from baseline

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    End point title
    		 Number of target joints and change in target joint status from baseline
    End point description
    To assess target joint status, per modified International Society on Thrombosis and Haemostasis (ISTH) guidelines 14.2/11
    End point type
    Secondary
    End point timeframe
    up to 6 months
    End point values
    		 Modified intent to treat set
    Number of subjects analysed
    19
    Units: target joints
        zero (baseline)
    8
        one (baseline)
    4
        two (baseline)
    2
        three (baseline)
    3
        four (baseline)
    2
        minus four (change from baseline)
    1
        minus three (change from baseline)
    4
        minus two (change from baseline)
    2
        minus one (change from baseline)
    4
        zero (change from baseline)
    8
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    All Adverse events were collected from the first study intervention up to 14 days after the end of study intervention.
    Adverse event reporting additional description
    Treatment Emergent AEs for Safety Population
    Assessment type
    		 Non-systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    27
    Reporting groups
    Reporting group title
    Safety Analysis Set
    Reporting group description
    		 all participants who have received at least one study intervention

    Serious adverse events
    		 Safety Analysis Set
    Total subjects affected by serious adverse events
         subjects affected / exposed
    2 / 21 (9.52%)
         number of deaths (all causes)
    0
         number of deaths resulting from adverse events
    0
    Musculoskeletal and connective tissue disorders
    Haemorrhage
         subjects affected / exposed
    2 / 21 (9.52%)
         occurrences causally related to treatment / all
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 0%
    Non-serious adverse events
    		 Safety Analysis Set
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    7 / 21 (33.33%)
    Nervous system disorders
    Headache
         subjects affected / exposed
    2 / 21 (9.52%)
         occurrences all number
    2
    Dizziness
         subjects affected / exposed
    1 / 21 (4.76%)
         occurrences all number
    1
    Taste disorder
         subjects affected / exposed
    1 / 21 (4.76%)
         occurrences all number
    1
    General disorders and administration site conditions
    Injection site bruising
         subjects affected / exposed
    1 / 21 (4.76%)
         occurrences all number
    1
    Peripheral swelling
         subjects affected / exposed
    1 / 21 (4.76%)
         occurrences all number
    1
    Gastrointestinal disorders
    Abdominal pain
         subjects affected / exposed
    1 / 21 (4.76%)
         occurrences all number
    1
    Diarrhoea
         subjects affected / exposed
    1 / 21 (4.76%)
         occurrences all number
    1
    Skin and subcutaneous tissue disorders
    Acne
         subjects affected / exposed
    1 / 21 (4.76%)
         occurrences all number
    1
    Urticaria
         subjects affected / exposed
    1 / 21 (4.76%)
         occurrences all number
    1
    Musculoskeletal and connective tissue disorders
    Pain in extremity
         subjects affected / exposed
    1 / 21 (4.76%)
         occurrences all number
    1
    Joint swelling
         subjects affected / exposed
    1 / 21 (4.76%)
         occurrences all number
    1
    Arthralgia
         subjects affected / exposed
    1 / 21 (4.76%)
         occurrences all number
    1
    Infections and infestations
    Nasopharyngitis
         subjects affected / exposed
    1 / 21 (4.76%)
         occurrences all number
    1
    Staphylococcal infection
         subjects affected / exposed
    1 / 21 (4.76%)
         occurrences all number
    1
    Pharyngitis streptococcal
         subjects affected / exposed
    1 / 21 (4.76%)
         occurrences all number
    1

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    17 Sep 2021
    adjustment to new safety reporting/assessment standards
    19 Aug 2022
    Accommodation of inclusion of additional countries Exclusion of participants with diagnosis of von Willebrand disease

    Interruptions (globally)
    Were there any global interruptions to the trial? No

    Limitations and caveats
    Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data.
    None reported
    For support, Contact us.
    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
    As of 31 January 2023, all EU/EEA initial clinical trial applications must be submitted through CTIS . Updated EudraCT trials information and information on PIP/Art 46 trials conducted exclusively in third countries continues to be submitted through EudraCT and published on this website.

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