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    Clinical Trial Results:
    A multi-centre, open-label trial evaluating efficacy, safety and pharmacokinetics of nonacog beta pegol when used for treatment and prophylaxis of bleeding episodes in Chinese patients with haemophilia B.

    Summary
    EudraCT number
    		 2021-004947-25
    Trial protocol
    		 Outside EU/EEA  
    Global end of trial date
    11 May 2024

    Results information
    Results version number
    		 v1(current)
    This version publication date
    27 Nov 2024
    First version publication date
    27 Nov 2024
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    NN7999-4670
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT05365217
    WHO universal trial number (UTN)
    		 U1111-1260-0438
    Sponsors
    Sponsor organisation name
    Novo Nordisk A/S
    Sponsor organisation address
    Novo Alle, Bagsvaerd, Denmark, 2880
    Public contact
    Clinical Reporting Office (2834), Novo Nordisk A/S, clinicaltrials@novonordisk.com
    Scientific contact
    Clinical Reporting Office (2834), Novo Nordisk A/S, clinicaltrials@novonordisk.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
    28 Jun 2024
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    11 May 2024
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    To evaluate the clinical efficacy of nonacog beta pegol in haemostasis (treatment of bleeding episodes during on-demand and prophylaxis [PPX]) in Chinese patients aged 12-70 years with moderate to severe haemophilia B.
    Protection of trial subjects
    The trial was conducted in accordance with the Declaration of Helsinki (Oct 2013) and International Council for Humanization of Technical Requirements for Pharmaceuticals for Human Use (ICH) Good Clinical Practice, including archiving of essential documents (May 1996) and EN International Organisation for Standardisation (ISO) 14155 Part 1 and 2 and Food and Drug Administration (FDA) 21 US Code of Federal Regulations (CFR) 312.120.
    Background therapy
    		 NA
    Evidence for comparator
    		 NA
    Actual start date of recruitment
    18 May 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
    China: 30
    Worldwide total number of subjects
    30
    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)
    5
    Adults (18-64 years)
    25
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 This trial was conducted at 15 sites that enrolled subjects in 1 country (China mainland).

    Pre-assignment
    Screening details
    		 A total of 30 subjects were exposed to trial products, of which 15 subjects were in Arm A (on demand/Prophylaxis) treatment group and 15 in Arm B (Prophylaxis) treatment group.

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

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Arm A: Nonacog beta pegol (On-demand)
    Arm description
    		 Subjects received intravenous injections of nonacog beta pegol (on-demand treatment for 28 weeks during treatment period 1, followed by 40 international unit per kilogram (IU/kg) for mild or moderate bleeds and 80 IU/kg for severe bleeds, with additional doses as needed if the initial treatment showed no effect during treatment period 2) until 30 exposure days (EDs) to nonacog beta pegol in the entire trial were fulfilled.
    Arm type
    		 Experimental

    Investigational medicinal product name
    N9-GP
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Powder for solution for injection/infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    Subjects were administered nonacog beta pegol 40 IU/kg for mild or moderate bleeds and 80 IU/kg for severe bleeds.

    Arm title
    		 Arm B: Nonacog beta pegol (Prophylaxis)
    Arm description
    		 Subjects received intravenous injections of 40 IU/kg nonacog beta pegol once weekly (prophylactic treatment with nonacog beta pegol at a dose of 40 IU/kg weekly) until 50 EDs (including treatment of breakthrough bleeds) and 50 weeks in the entire trial were fulfilled.
    Arm type
    		 Experimental

    Investigational medicinal product name
    N9-GP
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Powder for solution for injection/infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    Subjects received prophylactic treatment with nonacog beta pegol at a dose of 40 IU/kg weekly.

    Number of subjects in period 1
    		Arm A: Nonacog beta pegol (On-demand) Arm B: Nonacog beta pegol (Prophylaxis)
    Started
    15
    15
    Completed
    14
    15
    Not completed
    1
    0
         Consent withdrawn by subject
    1
    -
    Period 2
    Period 2 title
    Treatment Period 2
    Is this the baseline period?
    		 No
    Allocation method
    Not applicable
    Blinding used
    		 Not blinded

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Arm A: Nonacog beta pegol (Prophylaxis)
    Arm description
    		 Subjects received intravenous injections of nonacog beta pegol (on-demand treatment for 28 weeks during treatment period 1, followed by 40 international unit per kilogram (IU/kg) for mild or moderate bleeds and 80 IU/kg for severe bleeds, with additional doses as needed if the initial treatment showed no effect during treatment period 2) until 30 EDs to nonacog beta pegol in the entire trial were fulfilled.
    Arm type
    		 Experimental

    Investigational medicinal product name
    N9-GP
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Powder for solution for injection/infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    Subjects received nonacog beta pegol weekly intravenous dose of 40 IU/kg to prevent bleeding episodes.

    Arm title
    		 Arm B: Nonacog beta pegol (Prophylaxis)
    Arm description
    		 Subjects received intravenous injections of 40 IU/kg nonacog beta pegol once weekly (prophylactic treatment with nonacog beta pegol at a dose of 40 IU/kg weekly) until 50 EDs (including treatment of breakthrough bleeds) and 50 weeks in the entire trial were fulfilled.
    Arm type
    		 Experimental

    Investigational medicinal product name
    N9-GP
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Powder for solution for injection/infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    Subjects received prophylactic treatment with nonacog beta pegol at a dose of 40 IU/kg weekly.

    Number of subjects in period 2
    		Arm A: Nonacog beta pegol (Prophylaxis) Arm B: Nonacog beta pegol (Prophylaxis)
    Started
    14
    15
    Completed
    14
    15

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Arm A: Nonacog beta pegol (On-demand)
    Reporting group description
    		 Subjects received intravenous injections of nonacog beta pegol (on-demand treatment for 28 weeks during treatment period 1, followed by 40 international unit per kilogram (IU/kg) for mild or moderate bleeds and 80 IU/kg for severe bleeds, with additional doses as needed if the initial treatment showed no effect during treatment period 2) until 30 exposure days (EDs) to nonacog beta pegol in the entire trial were fulfilled.

    Reporting group title
    Arm B: Nonacog beta pegol (Prophylaxis)
    Reporting group description
    		 Subjects received intravenous injections of 40 IU/kg nonacog beta pegol once weekly (prophylactic treatment with nonacog beta pegol at a dose of 40 IU/kg weekly) until 50 EDs (including treatment of breakthrough bleeds) and 50 weeks in the entire trial were fulfilled.

    Reporting group values
    		 Arm A: Nonacog beta pegol (On-demand) Arm B: Nonacog beta pegol (Prophylaxis) Total
    Number of subjects
    		 15 15 30
    Age Categorical
    Units: Subjects
        In utero
    		 0 0 0
        Preterm newborn infants (gestational age < 37 wks)
    		 0 0 0
        Newborns (0-27 days)
    		 0 0 0
        Infants and toddlers (28 days-23 months)
    		 0 0 0
        Children (2-11 years)
    		 0 0 0
        Adolescents (12-17 years)
    		 1 4 5
        Adults (18-64 years)
    		 14 11 25
        From 65-84 years
    		 0 0 0
        85 years and over
    		 0 0 0
    Age Continuous
    Units: years
        arithmetic mean (standard deviation)
    		 29.9 ( 7.7 ) 26.9 ( 9.7 ) -
    Gender Categorical
    Units: Subjects
        Female
    		 0 0 0
        Male
    		 15 15 30

    End points

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    End points reporting groups
    Reporting group title
    Arm A: Nonacog beta pegol (On-demand)
    Reporting group description
    		 Subjects received intravenous injections of nonacog beta pegol (on-demand treatment for 28 weeks during treatment period 1, followed by 40 international unit per kilogram (IU/kg) for mild or moderate bleeds and 80 IU/kg for severe bleeds, with additional doses as needed if the initial treatment showed no effect during treatment period 2) until 30 exposure days (EDs) to nonacog beta pegol in the entire trial were fulfilled.

    Reporting group title
    Arm B: Nonacog beta pegol (Prophylaxis)
    Reporting group description
    		 Subjects received intravenous injections of 40 IU/kg nonacog beta pegol once weekly (prophylactic treatment with nonacog beta pegol at a dose of 40 IU/kg weekly) until 50 EDs (including treatment of breakthrough bleeds) and 50 weeks in the entire trial were fulfilled.
    Reporting group title
    Arm A: Nonacog beta pegol (Prophylaxis)
    Reporting group description
    		 Subjects received intravenous injections of nonacog beta pegol (on-demand treatment for 28 weeks during treatment period 1, followed by 40 international unit per kilogram (IU/kg) for mild or moderate bleeds and 80 IU/kg for severe bleeds, with additional doses as needed if the initial treatment showed no effect during treatment period 2) until 30 EDs to nonacog beta pegol in the entire trial were fulfilled.

    Reporting group title
    Arm B: Nonacog beta pegol (Prophylaxis)
    Reporting group description
    		 Subjects received intravenous injections of 40 IU/kg nonacog beta pegol once weekly (prophylactic treatment with nonacog beta pegol at a dose of 40 IU/kg weekly) until 50 EDs (including treatment of breakthrough bleeds) and 50 weeks in the entire trial were fulfilled.

    Subject analysis set title
    Arm B: Nonacog beta pegol (Prophylaxis)
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    Subjects received intravenous injections of 40 IU/kg nonacog beta pegol once weekly (prophylactic treatment with nonacog beta pegol at a dose of 40 IU/kg weekly) until 50 EDs (including treatment of breakthrough bleeds) and 50 weeks in the entire trial were fulfilled.

    Primary: Haemostatic Effect of Nonacog beta pegol when used for Treatment of Bleeding Episodes During on Demand and Prophylaxis (PPX)

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    End point title
    		 Haemostatic Effect of Nonacog beta pegol when used for Treatment of Bleeding Episodes During on Demand and Prophylaxis (PPX) [1] [2]
    End point description
    Haemostatic effect of N8-GP for treatment of bleeding episodes was assessed by 4-point response scale: none, moderate, good or excellent. Evaluation during trial was done by subject and/or parent(s)/caregiver within approximately 8 hours after a single injection as follows: Excellent: Abrupt pain relief and/or clear improvement in objective signs of bleeding within approximately 8 hrs after a single injection; Good: Definite pain relief and/or improvement in signs of bleeding within approximately 8 hrs after a single injection, but possibly requiring more than one injection for complete resolution; Moderate: Probable or slight beneficial effect within approximately 8 hours after the first injection, but usually requiring more than one injection; None: No improvement, or worsening of symptoms. Results were based on the FAS which included all subjects exposed to N9-GP in this trial.
    End point type
    Primary
    End point timeframe
    From start of treatment (week 0) until end of treatment (up to week 50)
    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: Not all the arms were evaluated for this end point. Data is provided for the arms evaluated for this end point.
    [2] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: Not all the arms were evaluated for this end point. Data is provided for the arms evaluated for this end point.
    End point values
    		 Arm A: Nonacog beta pegol (On-demand) Arm A: Nonacog beta pegol (Prophylaxis) Arm B: Nonacog beta pegol (Prophylaxis)
    Number of subjects analysed
    15
    14
    15
    Units: Bleeding Episodes
        Excellent
    195
    11
    26
        Good
    8
    1
    16
        Moderate
    3
    0
    1
        None
    1
    0
    0
        Missing
    0
    0
    0
    No statistical analyses for this end point

    Secondary: Number of Treated Bleeding Episodes During Prophylaxis (PPX) Treatment (Arm B only)

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    End point title
    		 Number of Treated Bleeding Episodes During Prophylaxis (PPX) Treatment (Arm B only)
    End point description
    Number of bleeding episodes per year data is reported. Annualised bleeding rate (ABR) is the number of bleeding episodes per year. Results were based on the FAS which included all subjects exposed to N9-GP in this trial.
    End point type
    Secondary
    End point timeframe
    From start of treatment (week 0) until end of treatment (week 50)
    End point values
    		 Arm B: Nonacog beta pegol (Prophylaxis)
    Number of subjects analysed
    15
    Units: Bleeds per subject per year
        median (inter-quartile range (Q1-Q3))
    3.12 (0.00 to 4.23)
    No statistical analyses for this end point

    Secondary: FIX Trough Levels During Prophylaxis (PPX) Treatment (Arm B only)

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    End point title
    		 FIX Trough Levels During Prophylaxis (PPX) Treatment (Arm B only)
    End point description
    Trough levels of FVIII was reported for all participnats who received prophylaxis treatment. Chromogenic assay was performed with N8-GP product specific standard (PSS) as a calibrator. The analysis is based on a mixed model on the log transformed plasma FVIII activity with age group as fixed effect and subject as a random effect. The mean trough is presented back-transformed to the natural scale. Results were based on the FAS which included all subjects exposed to N9-GP in this trial.
    End point type
    Secondary
    End point timeframe
    From start of treatment (week 0) until end of treatment (week 50)
    End point values
    		 Arm B: Nonacog beta pegol (Prophylaxis)
    Number of subjects analysed
    15
    Units: International unit per milliliter(IU/mL)
        arithmetic mean (confidence interval 95%)
    0.298 (0.260 to 0.341)
    No statistical analyses for this end point

    Secondary: Consumption of Nonacog beta pegol for Prophylaxis (PPX) Treatment (Arm B only)

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    End point title
    		 Consumption of Nonacog beta pegol for Prophylaxis (PPX) Treatment (Arm B only)
    End point description
    The mean consumption of N9-GP for prophylaxis per year per subject was reported and it was measured in international units per kilogram per year (IU/kg/year). Results were based on the FAS which included all subjects exposed to N9-GP in this trial.
    End point type
    Secondary
    End point timeframe
    From start of treatment (week 0) until end of treatment (week 50)
    End point values
    		 Arm B: Nonacog beta pegol (Prophylaxis)
    Number of subjects analysed
    15
    Units: IU/kg per year
        arithmetic mean (standard deviation)
    2212.3 ( 26.0 )
    No statistical analyses for this end point

    Secondary: Consumption of Nonacog beta pegol for Treatment of Bleeding Episodes

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    End point title
    		 Consumption of Nonacog beta pegol for Treatment of Bleeding Episodes [3]
    End point description
    The mean number of injections of N9-GP used for treatment of a bleed from start to stop of a bleed was reported and it was measured in international units per kilogram per bleed (IU/kg/bleed). Results were based on the FAS which included all subjects exposed to N8-GP in this trial. Results were based on the FAS which included all subjects exposed to N9-GP in this trial.
    End point type
    Secondary
    End point timeframe
    From start of treatment (week 0) until end of treatment (week 50)
    Notes
    [3] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: Not all the arms were evaluated for this end point. Data is provided for the arms evaluated for this end point.
    End point values
    		 Arm A: Nonacog beta pegol (On-demand) Arm A: Nonacog beta pegol (Prophylaxis) Arm B: Nonacog beta pegol (Prophylaxis)
    Number of subjects analysed
    15
    14
    15
    Units: IU/kg per bleed
        arithmetic mean (standard deviation)
    42.5 ( 1.0 )
    41.4 ( 1.1 )
    41.6 ( 0.4 )
    No statistical analyses for this end point

    Secondary: Number of Subjects with Inhibitory Antibodies Against FIX Defined as Titre ≥0.6 Bethesda Units (BU)

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    End point title
    		 Number of Subjects with Inhibitory Antibodies Against FIX Defined as Titre ≥0.6 Bethesda Units (BU) [4]
    End point description
    Percentage of subjects who developed inhibitory antibodies (IA) against FVIII was presented. A subject was said to have FVIII-inhibitors if two consecutive tests, preferably within 2 weeks, were positive (greater than or equal to (≥) 0.6 bethesda unit (BU)). For the calculation of the inhibitor rate the numerator was included for all subjects with neutralising antibodies while the denominator was included for all subjects with a minimum of 50 exposures plus any subjects with less than 50 exposures but with neutralising inhibitor. Results were based on the FAS which included all subjects exposed to N9-GP in this trial.
    End point type
    Secondary
    End point timeframe
    From start of treatment (week 0) until end of treatment (week 50)
    Notes
    [4] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: Not all the arms were evaluated for this end point. Data is provided for the arms evaluated for this end point.
    End point values
    		 Arm A: Nonacog beta pegol (On-demand) Arm A: Nonacog beta pegol (Prophylaxis) Arm B: Nonacog beta pegol (Prophylaxis)
    Number of subjects analysed
    15
    14
    15
    Units: Number of subjects with IA
    0
    0
    0
    No statistical analyses for this end point

    Secondary: Number of Adverse Events (AEs)

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    End point title
    		 Number of Adverse Events (AEs) [5]
    End point description
    An adverse event (AE) was defined as any unfavorable and unintended sign (including an abnormal laboratory finding), symptom or disease temporally associated with the use of a product, whether or not considered related to the product. All presented AEs are treatment-emergent. A treatment-emergent adverse event was defined as an event with onset after first N8-GP administration. Results were based on the SAS which included all subjects exposed to N9-GP in this trial.
    End point type
    Secondary
    End point timeframe
    From start of treatment (week 0) until end of treatment (week 50)
    Notes
    [5] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: Not all the arms were evaluated for this end point. Data is provided for the arms evaluated for this end point.
    End point values
    		 Arm A: Nonacog beta pegol (On-demand) Arm A: Nonacog beta pegol (Prophylaxis) Arm B: Nonacog beta pegol (Prophylaxis)
    Number of subjects analysed
    15
    14
    15
    Units: Events
    16
    19
    29
    No statistical analyses for this end point

    Secondary: Number of Serious Adverse events (SAEs)

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    End point title
    		 Number of Serious Adverse events (SAEs) [6]
    End point description
    A serious adverse event (SAE) is defined as any untoward medical occurrence that at any dose results in death, or is life-threatening, or requires inpatient hospitalization or causes prolongation of existing hospitalization results in persistent or significant disability/incapacity, or may have caused a congenital anomaly/birth defect, or requires intervention to prevent permanent impairment or damage. All presented SAEs are treatment-emergent (any serious adverse events which occurred after trial product administration). Results were based on the SAS which included all subjects exposed to N9-GP in this trial.
    End point type
    Secondary
    End point timeframe
    From start of treatment (week 0) until end of treatment (week 50)
    Notes
    [6] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: Not all the arms were evaluated for this end point. Data is provided for the arms evaluated for this end point.
    End point values
    		 Arm A: Nonacog beta pegol (On-demand) Arm A: Nonacog beta pegol (Prophylaxis) Arm B: Nonacog beta pegol (Prophylaxis)
    Number of subjects analysed
    15
    14
    15
    Units: Events
    0
    1
    0
    No statistical analyses for this end point

    Secondary: Incremental Recovery (IR) (Arm B only)

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    End point title
    		 Incremental Recovery (IR) (Arm B only)
    End point description
    The incremental recovery was calculated by subtracting the FVIII activity (IU/mL) measured in plasma at time 0 from that measured at time 30 min after dosing and dividing this difference by the dose injected at time 0 expressed as IU/kg body weight. FVIII activity was measured with a chromogenic assay. The PK analysis set included sub-set of subjects from FAS who were included for PK assessments. Number analysed = Number of subjects with available data for specific timepoints.
    End point type
    Secondary
    End point timeframe
    Single-dose: 30±10 minutes post injection at week 0, Steady-state: 30±10 minutes post injection at week 12
    End point values
    		 Arm B: Nonacog beta pegol (Prophylaxis)
    Number of subjects analysed
    15
    Units: (IU/mL)/(IU/kg)
    geometric mean (geometric coefficient of variation)
        Week 0 (n=14)
    0.0182 ( 18.9550 )
        Week 12 (n=15)
    0.0192 ( 17.2668 )
    No statistical analyses for this end point

    Secondary: Terminal Half-life (t½) (Arm B only)

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    End point title
    		 Terminal Half-life (t½) (Arm B only)
    End point description
    Terminal half life was calculated as ln(2)/λz; where λz is the terminal elimination rate constant. The terminal elimination rate constant was estimated using linear regression on the terminal part of the log (activity) versus time profile. The PK analysis set included sub-set of subjects from FAS who were included for PK assessments. Number analysed = Number of subjects with available data for specific timepoints.
    End point type
    Secondary
    End point timeframe
    Single-dose: 0-168 hours post injection at week 0, Steady-state: 0-168 hours post injection at week 12
    End point values
    		 Arm B: Nonacog beta pegol (Prophylaxis)
    Number of subjects analysed
    15
    Units: hour
    geometric mean (geometric coefficient of variation)
        Week 0 (n= 13)
    90.868 ( 13.535 )
        Week 12 (n=15)
    90.738 ( 21.581 )
    No statistical analyses for this end point

    Secondary: Clearance (CL) (Arm B only)

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    End point title
    		 Clearance (CL) (Arm B only)
    End point description
    Clearance (CL) of drug after intravenous administration was reported. Clearance was calculated using the formula CL= Dose / AUC(0-inf) for single dose and CL= Dose / AUC(0-96) h for steady state. The PK analysis set included sub-set of subjects from FAS who were included for PK assessments. Number analysed = Number of subjects with available data for specific timepoints.
    End point type
    Secondary
    End point timeframe
    Single-dose: 0-168 hours post injection at week 0, Steady-state: 0-168 hours post injection at week 12
    End point values
    		 Arm B: Nonacog beta pegol (Prophylaxis)
    Number of subjects analysed
    15
    Units: mL/h/kg
    geometric mean (geometric coefficient of variation)
        Week 0 (n=13)
    0.536 ( 22.143 )
        Week 12 (n=15)
    0.487 ( 26.552 )
    No statistical analyses for this end point

    Secondary: Area Under the Curve (AUC) (Arm B only)

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    End point title
    		 Area Under the Curve (AUC) (Arm B only)
    End point description
    Area under the plasma activity versus time profile from time zero to 168 hours (AUC0-168h) was measured The PK analysis set included sub-set of subjects from FAS who were included for PK assessments. Number analysed = Number of subjects with available data for specific timepoints.
    End point type
    Secondary
    End point timeframe
    Single-dose: 0-168 hours post injection at week 0, Steady-state: 0-168 hours post injection at week 12
    End point values
    		 Arm B: Nonacog beta pegol (Prophylaxis)
    Number of subjects analysed
    15
    Units: h·IU/mL
    geometric mean (geometric coefficient of variation)
        Week 0 (n=14)
    51.856 ( 30.134 )
        Week 12 (n=15)
    92.914 ( 21.725 )
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    From start of treatment (Week 0) until end of trial (Week 54)
    Adverse event reporting additional description
    All presented AEs are treatment-emergent adverse events. A TEAE was defined as an event with onset after first N8-GP administration. Results were based on the SAS which included all subjects exposed to N9-GP in this trial.
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    27
    Reporting groups
    Reporting group title
    Arm A: Nonacog beta pegol (On-demand)
    Reporting group description
    		 Subjects received intravenous injections of nonacog beta pegol (on-demand treatment for 28 weeks during treatment period 1, followed by 40 international unit per kilogram (IU/kg) for mild or moderate bleeds and 80 IU/kg for severe bleeds, with additional doses as needed if the initial treatment showed no effect during treatment period 2) until 30 EDs to nonacog beta pegol in the entire trial were fulfilled.

    Reporting group title
    Arm B: Nonacog beta pegol (Prophylaxis)
    Reporting group description
    		 Subjects received intravenous injections of 40 IU/kg nonacog beta pegol once weekly (prophylactic treatment with nonacog beta pegol at a dose of 40 IU/kg weekly) until 50 EDs (including treatment of breakthrough bleeds) and 50 weeks in the entire trial were fulfilled.

    Reporting group title
    Arm A: Nonacog beta pegol (Prophylaxis)
    Reporting group description
    		 Subjects received intravenous injections of nonacog beta pegol (on-demand treatment for 28 weeks during treatment period 1, followed by 40 IU/kg for mild or moderate bleeds and 80 IU/kg for severe bleeds, with additional doses as needed if the initial treatment showed no effect during treatment period 2) until 30 EDs to nonacog beta pegol in the entire trial were fulfilled.

    Serious adverse events
    		 Arm A: Nonacog beta pegol (On-demand) Arm B: Nonacog beta pegol (Prophylaxis) Arm A: Nonacog beta pegol (Prophylaxis)
    Total subjects affected by serious adverse events
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 15 (0.00%)
    0 / 14 (0.00%)
         number of deaths (all causes)
    0
    0
    0
         number of deaths resulting from adverse events
    0
    0
    0
    Infections and infestations
    Haematoma infection
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 15 (0.00%)
    0 / 14 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 Arm A: Nonacog beta pegol (On-demand) Arm B: Nonacog beta pegol (Prophylaxis) Arm A: Nonacog beta pegol (Prophylaxis)
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    12 / 15 (80.00%)
    14 / 15 (93.33%)
    4 / 14 (28.57%)
    Vascular disorders
    Phlebitis superficial
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 15 (0.00%)
    0 / 14 (0.00%)
         occurrences all number
    1
    0
    0
    General disorders and administration site conditions
    Chest discomfort
         subjects affected / exposed
    0 / 15 (0.00%)
    0 / 15 (0.00%)
    1 / 14 (7.14%)
         occurrences all number
    0
    0
    1
    Pyrexia
         subjects affected / exposed
    2 / 15 (13.33%)
    1 / 15 (6.67%)
    0 / 14 (0.00%)
         occurrences all number
    2
    1
    0
    Influenza like illness
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 15 (0.00%)
    0 / 14 (0.00%)
         occurrences all number
    1
    0
    0
    Pain
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 15 (0.00%)
    0 / 14 (0.00%)
         occurrences all number
    1
    0
    0
    Respiratory, thoracic and mediastinal disorders
    Cough
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 15 (0.00%)
    0 / 14 (0.00%)
         occurrences all number
    1
    0
    0
    Epistaxis
         subjects affected / exposed
    0 / 15 (0.00%)
    1 / 15 (6.67%)
    0 / 14 (0.00%)
         occurrences all number
    0
    3
    0
    Pulmonary mass
         subjects affected / exposed
    0 / 15 (0.00%)
    1 / 15 (6.67%)
    0 / 14 (0.00%)
         occurrences all number
    0
    1
    0
    Investigations
    Blood fibrinogen decreased
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 15 (0.00%)
    0 / 14 (0.00%)
         occurrences all number
    1
    0
    0
    Fibrin D dimer increased
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 15 (0.00%)
    0 / 14 (0.00%)
         occurrences all number
    1
    0
    0
    Aspartate aminotransferase increased
         subjects affected / exposed
    0 / 15 (0.00%)
    1 / 15 (6.67%)
    0 / 14 (0.00%)
         occurrences all number
    0
    1
    0
    Blood fibrinogen increased
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 15 (0.00%)
    0 / 14 (0.00%)
         occurrences all number
    1
    0
    0
    C-reactive protein increased
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 15 (0.00%)
    0 / 14 (0.00%)
         occurrences all number
    1
    0
    0
    Fibrinogen degradation products increased
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 15 (0.00%)
    0 / 14 (0.00%)
         occurrences all number
    1
    0
    0
    Procalcitonin increased
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 15 (0.00%)
    0 / 14 (0.00%)
         occurrences all number
    1
    0
    0
    Weight increased
         subjects affected / exposed
    0 / 15 (0.00%)
    1 / 15 (6.67%)
    0 / 14 (0.00%)
         occurrences all number
    0
    1
    0
    White blood cell count increased
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 15 (0.00%)
    0 / 14 (0.00%)
         occurrences all number
    2
    0
    0
    Injury, poisoning and procedural complications
    Muscle strain
         subjects affected / exposed
    0 / 15 (0.00%)
    1 / 15 (6.67%)
    0 / 14 (0.00%)
         occurrences all number
    0
    1
    0
    Cardiac disorders
    Atrioventricular block
         subjects affected / exposed
    0 / 15 (0.00%)
    1 / 15 (6.67%)
    0 / 14 (0.00%)
         occurrences all number
    0
    1
    0
    Nervous system disorders
    Dizziness
         subjects affected / exposed
    0 / 15 (0.00%)
    0 / 15 (0.00%)
    1 / 14 (7.14%)
         occurrences all number
    0
    0
    1
    Neuritis
         subjects affected / exposed
    0 / 15 (0.00%)
    1 / 15 (6.67%)
    0 / 14 (0.00%)
         occurrences all number
    0
    1
    0
    Blood and lymphatic system disorders
    Anaemia
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 15 (0.00%)
    0 / 14 (0.00%)
         occurrences all number
    1
    0
    0
    Lymphadenitis
         subjects affected / exposed
    0 / 15 (0.00%)
    1 / 15 (6.67%)
    0 / 14 (0.00%)
         occurrences all number
    0
    1
    0
    Eye disorders
    Trichiasis
         subjects affected / exposed
    0 / 15 (0.00%)
    1 / 15 (6.67%)
    0 / 14 (0.00%)
         occurrences all number
    0
    1
    0
    Gastrointestinal disorders
    Diarrhoea
         subjects affected / exposed
    2 / 15 (13.33%)
    0 / 15 (0.00%)
    0 / 14 (0.00%)
         occurrences all number
    2
    0
    0
    Hepatobiliary disorders
    Hepatic function abnormal
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 15 (0.00%)
    0 / 14 (0.00%)
         occurrences all number
    1
    0
    0
    Skin and subcutaneous tissue disorders
    Acne
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 15 (0.00%)
    0 / 14 (0.00%)
         occurrences all number
    1
    0
    0
    Rash
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 15 (0.00%)
    0 / 14 (0.00%)
         occurrences all number
    1
    0
    0
    Renal and urinary disorders
    Proteinuria
         subjects affected / exposed
    0 / 15 (0.00%)
    0 / 15 (0.00%)
    1 / 14 (7.14%)
         occurrences all number
    0
    0
    1
    Musculoskeletal and connective tissue disorders
    Haemophilic arthropathy
         subjects affected / exposed
    0 / 15 (0.00%)
    1 / 15 (6.67%)
    0 / 14 (0.00%)
         occurrences all number
    0
    1
    0
    Synovitis
         subjects affected / exposed
    0 / 15 (0.00%)
    1 / 15 (6.67%)
    1 / 14 (7.14%)
         occurrences all number
    0
    1
    1
    Infections and infestations
    COVID-19
         subjects affected / exposed
    1 / 15 (6.67%)
    6 / 15 (40.00%)
    0 / 14 (0.00%)
         occurrences all number
    1
    6
    0
    Coronavirus infection
         subjects affected / exposed
    1 / 15 (6.67%)
    1 / 15 (6.67%)
    0 / 14 (0.00%)
         occurrences all number
    1
    1
    0
    Influenza
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 15 (0.00%)
    0 / 14 (0.00%)
         occurrences all number
    1
    0
    0
    Rhinitis
         subjects affected / exposed
    0 / 15 (0.00%)
    0 / 15 (0.00%)
    1 / 14 (7.14%)
         occurrences all number
    0
    0
    1
    Respiratory tract infection
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 15 (0.00%)
    1 / 14 (7.14%)
         occurrences all number
    1
    0
    1
    Periodontitis
         subjects affected / exposed
    2 / 15 (13.33%)
    0 / 15 (0.00%)
    0 / 14 (0.00%)
         occurrences all number
    2
    0
    0
    Pneumonia
         subjects affected / exposed
    0 / 15 (0.00%)
    1 / 15 (6.67%)
    0 / 14 (0.00%)
         occurrences all number
    0
    1
    0
    Upper respiratory tract infection
         subjects affected / exposed
    1 / 15 (6.67%)
    4 / 15 (26.67%)
    0 / 14 (0.00%)
         occurrences all number
    1
    5
    0
    Metabolism and nutrition disorders
    Hypoproteinaemia
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 15 (0.00%)
    0 / 14 (0.00%)
         occurrences all number
    1
    0
    0
    Hyperuricaemia
         subjects affected / exposed
    0 / 15 (0.00%)
    1 / 15 (6.67%)
    0 / 14 (0.00%)
         occurrences all number
    0
    2
    0

    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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    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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