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    Clinical Trial Results:
    Safety, efficacy and exposure of subcutaneously administered NNC0365-3769 (Mim8) prophylaxis in children with haemophilia A with or without FVIII inhibitors

    Summary
    EudraCT number
    		 2020-003467-26
    Trial protocol
    		 NL   PL   IT   ES   PT   LT  
    Global end of trial date
    13 Nov 2024

    Results information
    Results version number
    		 v1(current)
    This version publication date
    28 May 2025
    First version publication date
    28 May 2025
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    NN7769-4516
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT05306418
    WHO universal trial number (UTN)
    		 U1111-1255-1540
    Other trial identifiers
    		 Japanese trial registration number: jRCT2031220670
    Sponsors
    Sponsor organisation name
    Novo Nordisk A/S
    Sponsor organisation address
    Novo Allé, 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)
    Yes
    EMA paediatric investigation plan number(s)
    		 EMEA-002762-PIP02-02
    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
    07 Jan 2025
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    13 Nov 2024
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    To investigate the safety of Mim8 prophylaxis in children with haemophilia A with or without coagulation factor VIII (FVIII) inhibitors.
    Protection of trial subjects
    This study was conducted in accordance with the protocol and consensus ethical principles derived from international guidelines including the Declaration of Helsinki, applicable International Council for Harmonization (ICH) Good Clinical Practice guidelines, and other applicable laws and regulations.
    Background therapy
    		 The products/treatment used for bleeds and surgery and prophylactic treatment during run-in period, treatment period and the follow-up period were regarded as non-investigational medicinal products (non-IMPs) in this trial. Prophylactic use of standard and extended FVIII products could continue for 1 week after Mim8 loading dose.
    Evidence for comparator
    		 Not applicable.
    Actual start date of recruitment
    04 Apr 2022
    Long term follow-up planned
    No
    Independent data monitoring committee (IDMC) involvement?
    Yes
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    Canada: 2
    Country: Number of subjects enrolled
    China: 10
    Country: Number of subjects enrolled
    India: 9
    Country: Number of subjects enrolled
    Israel: 2
    Country: Number of subjects enrolled
    Japan: 1
    Country: Number of subjects enrolled
    Italy: 2
    Country: Number of subjects enrolled
    Netherlands: 1
    Country: Number of subjects enrolled
    Poland: 9
    Country: Number of subjects enrolled
    Portugal: 3
    Country: Number of subjects enrolled
    South Africa: 7
    Country: Number of subjects enrolled
    Korea, Republic of: 9
    Country: Number of subjects enrolled
    Spain: 3
    Country: Number of subjects enrolled
    Switzerland: 1
    Country: Number of subjects enrolled
    Taiwan: 2
    Country: Number of subjects enrolled
    United Kingdom: 4
    Country: Number of subjects enrolled
    United States: 6
    Worldwide total number of subjects
    71
    EEA total number of subjects
    18
    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)
    10
    Children (2-11 years)
    61
    Adolescents (12-17 years)
    0
    Adults (18-64 years)
    0
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 The study was conducted at 29 sites that recruited subjects in Canada, China, India, Israel, Italy, Japan, Netherlands, Poland, Portugal, South Africa, South Korea, Spain, Switzerland, Taiwan, United Kingdom and United States.

    Pre-assignment
    Screening details
    		 Study included run-in period of at least 26 weeks for children previously treated on prophylaxis (PPX) which was followed by 52-week treatment period with part 1 + part 2, where all subjects received Mim8 and 21-week follow-up period unless the subject or parent(s)/caregiver(s) wanted to transfer to open-label extension study NN7769-4532.

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

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Part 1: Mim8 Once Weekly - Subjects 1-5 years
    Arm description
    		 Subjects aged 1-5 years subcutaneously received Mim8 once weekly based on their weight band until week-26 in part 1.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Mim8
    Investigational medicinal product code
    NNC0365 -3769 B
    Other name
    Pharmaceutical forms
    Solution for injection
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    A loading dose was administered once followed by once weekly maintenance doses in part 1. Loading doses were administered by 1 or 2 injections, using 1 or 2 cartridges of study intervention. Maintenance doses were administered by 1 injection using 1 cartridge of study intervention. 0.8 milliliter (mL) of study intervention was administered per injection. Dose amount was based on weight band of subject, whether it was a loading or maintenance dose, and the frequency of dosing: total loading dose (9.0 milligrams [mg] for less than 15 kilograms (kg), 24.0 mg for equals 15 kg - less than 45 kg and 55.0 mg for greater than equals 45 kg); total maintenance dose (1.6 mg for less than 15 kg, 4.0 mg for equals 15 kg - less than 45 kg and 9.0 mg greater than equals 45 kg).

    Arm title
    		 Part 1: Mim8 Once Weekly - Subjects 6-11 years
    Arm description
    		 Subjects aged 6-11 years subcutaneously received Mim8 once weekly subcutaneously based on their weight band until week-26 in part 1.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Mim8
    Investigational medicinal product code
    NNC0365 -3769 B
    Other name
    Pharmaceutical forms
    Solution for injection
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    A loading dose was administered once followed by once weekly maintenance doses in part 1. Loading doses were administered by 1 or 2 injections, using 1 or 2 cartridges of study intervention. Maintenance doses were administered by 1 injection using 1 cartridge of study intervention. 0.8 milliliter (mL) of study intervention was administered per injection. Dose amount was based on weight band of subject, whether it was a loading or maintenance dose, and the frequency of dosing: total loading dose (9.0 milligrams [mg] for less than 15 kilograms (kg), 24.0 mg for equals 15 kg - less than 45 kg and 55.0 mg for greater than equals 45 kg); total maintenance dose (1.6 mg for less than 15 kg, 4.0 mg for equals 15 kg - less than 45 kg and 9.0 mg greater than equals 45 kg).

    Number of subjects in period 1 [1]
    		Part 1: Mim8 Once Weekly - Subjects 1-5 years Part 1: Mim8 Once Weekly - Subjects 6-11 years
    Started
    36
    34
    Completed
    36
    34
    Notes
    [1] - The number of subjects reported to be in the baseline period are not the same as the worldwide number enrolled in the trial. It is expected that these numbers will be the same.
    Justification: The total of 71 subjects enrolled in the study. Out of 71 subjects, 1 subject withdrew during run-in period. Hence, data are reported for 70 participants.
    Period 2
    Period 2 title
    Treatment Period - Part 2
    Is this the baseline period?
    		 No
    Allocation method
    Not applicable
    Blinding used
    		 Not blinded

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Part 2: Mim8 Once Weekly - Subjects 1-5 years
    Arm description
    		 Subjects aged 1-5 years from part 1, decided at week-26 to continue on subcutaneously Mim8 once weekly (dose amount based on their weight band) until week-52 in part 2. The part 2 treatment period was followed by a 21-week follow-up period for all subjects unless the subjects or the parent(s)/caregiver(s) wanted to transfer to the open-label extension study NN7769-4532.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Mim8
    Investigational medicinal product code
    NNC0365 -3769 B
    Other name
    Pharmaceutical forms
    Solution for injection
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    Doses were administered once weekly in part 2. Doses were administered by 1 injection using 1 cartridge of study intervention. 0.8 mL of study intervention was administered per injection. Dose amount was based on weight band of subject, total dose (1.6 mg for less than 15 kg, 4.0 mg for equals 15 kg - less than 45 kg and 9.0 mg greater than equals 45 kg).

    Arm title
    		 Part 2: Mim8 Once Weekly - Subjects 6-11 years
    Arm description
    		 Subjects aged 6-11 years from part 1, decided at week-26 to continue on subcutaneously Mim8 once weekly (dose amount based on their weight band) until week-52 in part 2. The part 2 treatment period was followed by a 21-week follow-up period for all subjects unless the subjects or the parent(s)/caregiver(s) wanted to transfer to the open-label extension study NN7769-4532.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Mim8
    Investigational medicinal product code
    NNC0365 -3769 B
    Other name
    Pharmaceutical forms
    Solution for injection
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    Doses were administered once weekly in part 2. Doses were administered by 1 injection using 1 cartridge of study intervention. 0.8 mL of study intervention was administered per injection. Dose amount was based on weight band of subject, total dose (1.6 mg for less than 15 kg, 4.0 mg for equals 15 kg - less than 45 kg and 9.0 mg greater than equals 45 kg).

    Arm title
    		 Part 2: Mim8 Once Monthly - Subjects 1-5 years
    Arm description
    		 Subjects aged 1-5 years from part 1, decided at week-26 to switch to subcutaneously Mim8 once monthly (dose amount based on their weight band) until week-52 in part 2. The part 2 treatment period was followed by a 21-week follow-up period for all subjects unless the subjects or the parent(s)/caregiver(s) wanted to transfer to the open-label extension study NN7769-4532.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Mim8
    Investigational medicinal product code
    NNC0365 -3769 B
    Other name
    Pharmaceutical forms
    Solution for injection
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    Doses were administered once monthly in part 2. Doses were administered by 1 injection using 1 cartridge of study intervention. 0.8 mL of study intervention was administered per injection. Dose amount was based on weight band of subject, total dose (9.0 mg for less than 15 kg, 20.0 mg for equals 15 kg - less than 45 kg and 46.0 mg greater than equals 45 kg).

    Arm title
    		 Part 2: Mim8 Once Monthly - Subjects 6-11 years
    Arm description
    		 Subjects aged 6-11 years from part 1, decided at week-26 to switch to subcutaneously Mim8 once monthly (dose amount based on their weight band) until week-52 in part 2. The part 2 treatment period was followed by a 21-week follow-up period for all subjects unless the subjects or the parent(s)/caregiver(s) wanted to transfer to the open-label extension study NN7769-4532.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Mim8
    Investigational medicinal product code
    NNC0365 -3769 B
    Other name
    Pharmaceutical forms
    Solution for injection
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    Doses were administered once monthly in part 2. Doses were administered by 1 injection using 1 cartridge of study intervention. 0.8 mL of study intervention was administered per injection. Dose amount was based on weight band of subject, total dose (9.0 mg for less than 15 kg, 20.0 mg for equals 15 kg - less than 45 kg and 46.0 mg greater than equals 45 kg).

    Number of subjects in period 2
    		Part 2: Mim8 Once Weekly - Subjects 1-5 years Part 2: Mim8 Once Weekly - Subjects 6-11 years Part 2: Mim8 Once Monthly - Subjects 1-5 years Part 2: Mim8 Once Monthly - Subjects 6-11 years
    Started
    19
    19
    17
    15
    Completed
    19
    19
    17
    15

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Part 1: Mim8 Once Weekly - Subjects 1-5 years
    Reporting group description
    		 Subjects aged 1-5 years subcutaneously received Mim8 once weekly based on their weight band until week-26 in part 1.

    Reporting group title
    Part 1: Mim8 Once Weekly - Subjects 6-11 years
    Reporting group description
    		 Subjects aged 6-11 years subcutaneously received Mim8 once weekly subcutaneously based on their weight band until week-26 in part 1.

    Reporting group values
    		 Part 1: Mim8 Once Weekly - Subjects 1-5 years Part 1: Mim8 Once Weekly - Subjects 6-11 years Total
    Number of subjects
    		 36 34
    Age Categorical
    Units: Subjects
    Age Continuous
    Units: years
        arithmetic mean (standard deviation)
    		 2.9 ( 1.4 ) 8.4 ( 1.9 ) -
    Gender Categorical
    Units: subjects
        Male
    		 36 34 70

    End points

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    End points reporting groups
    Reporting group title
    Part 1: Mim8 Once Weekly - Subjects 1-5 years
    Reporting group description
    		 Subjects aged 1-5 years subcutaneously received Mim8 once weekly based on their weight band until week-26 in part 1.

    Reporting group title
    Part 1: Mim8 Once Weekly - Subjects 6-11 years
    Reporting group description
    		 Subjects aged 6-11 years subcutaneously received Mim8 once weekly subcutaneously based on their weight band until week-26 in part 1.
    Reporting group title
    Part 2: Mim8 Once Weekly - Subjects 1-5 years
    Reporting group description
    		 Subjects aged 1-5 years from part 1, decided at week-26 to continue on subcutaneously Mim8 once weekly (dose amount based on their weight band) until week-52 in part 2. The part 2 treatment period was followed by a 21-week follow-up period for all subjects unless the subjects or the parent(s)/caregiver(s) wanted to transfer to the open-label extension study NN7769-4532.

    Reporting group title
    Part 2: Mim8 Once Weekly - Subjects 6-11 years
    Reporting group description
    		 Subjects aged 6-11 years from part 1, decided at week-26 to continue on subcutaneously Mim8 once weekly (dose amount based on their weight band) until week-52 in part 2. The part 2 treatment period was followed by a 21-week follow-up period for all subjects unless the subjects or the parent(s)/caregiver(s) wanted to transfer to the open-label extension study NN7769-4532.

    Reporting group title
    Part 2: Mim8 Once Monthly - Subjects 1-5 years
    Reporting group description
    		 Subjects aged 1-5 years from part 1, decided at week-26 to switch to subcutaneously Mim8 once monthly (dose amount based on their weight band) until week-52 in part 2. The part 2 treatment period was followed by a 21-week follow-up period for all subjects unless the subjects or the parent(s)/caregiver(s) wanted to transfer to the open-label extension study NN7769-4532.

    Reporting group title
    Part 2: Mim8 Once Monthly - Subjects 6-11 years
    Reporting group description
    		 Subjects aged 6-11 years from part 1, decided at week-26 to switch to subcutaneously Mim8 once monthly (dose amount based on their weight band) until week-52 in part 2. The part 2 treatment period was followed by a 21-week follow-up period for all subjects unless the subjects or the parent(s)/caregiver(s) wanted to transfer to the open-label extension study NN7769-4532.

    Subject analysis set title
    Part 1+2: Mim8 Once Weekly - Subjects 1-5 years
    Subject analysis set type
    		 Sub-group analysis
    Subject analysis set description
    Subjects aged 1-5 years subcutaneously received Mim8 once weekly until week-26 in part 1. For the part 2, a subgroup of patients decided at week-26 to continue on Mim8 once weekly until week-52 in part 2. The part 2 treatment period was followed by a 21-week follow-up period for all subjects unless the subjects or the parent(s)/caregiver(s) wanted to transfer to the open-label extension study NN7769-4532.

    Subject analysis set title
    Part 1+2: Mim8 Once Weekly - Subjects 6-11 years
    Subject analysis set type
    		 Sub-group analysis
    Subject analysis set description
    Subjects aged 6-11 years subcutaneously received Mim8 once weekly until week-26 in part 1. For the part 2, a subgroup of patients decided at week-26 to continue on Mim8 once weekly until week-52 in part 2. The part 2 treatment period was followed by a 21-week follow-up period for all subjects unless the subjects or the parent(s)/caregiver(s) wanted to transfer to the open-label extension study NN7769-4532.

    Subject analysis set title
    Run-in: Previous PPX + Previous On Demand Subjects 1-5 years
    Subject analysis set type
    		 Sub-group analysis
    Subject analysis set description
    Subjects aged 1-5 years previously treated on prophylaxis (standard/extended half-life FVIII or bypassing agent) or on demand participated in run-in period.

    Subject analysis set title
    Run-in: Previous PPX + Previous On Demand Subjects 6-11 years
    Subject analysis set type
    		 Sub-group analysis
    Subject analysis set description
    Subjects aged 6-11 years previously treated on prophylaxis (standard/extended half-life FVIII or bypassing agent) or on demand participated in run-in period.

    Primary: Number of treatment emergent adverse events (TEAEs)

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    End point title
    		 Number of treatment emergent adverse events (TEAEs) [1]
    End point description
    AE is any untoward medical occurrence in a subject that is temporally associated with the use of an IMP, whether or not considered related to IMP. Number of TEAEs is presented. AEs were evaluated based on data from on-treatment period which is given as time period in which subject was exposed to trial product and started at the date of first dose of product and ended at the first date of any of the following: end of treatment visit if they enrolled in the study NN7769-4532 or follow-up visit and last date on trial product +21 weeks. Safety analysis set (SAS) included all subjects exposed to at least 1 dose of trial product and were analysed according to treatment they actually received. As defined in protocol section 9, analysis was planned to be performed for once weekly and once monthly treatment regimens. Hence, all of subjects during part 1 as well as that decided to continue on Mim8 once weekly in part 2 are combined and represented in part 1+2 once weekly reporting group.
    End point type
    Primary
    End point timeframe
    From treatment initiation to follow up visit (week 0 to week 72)
    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: The primary endpoint investigated safety and was analysed using descriptive statistics, and thus no statistical analysis was performed.
    End point values
    		 Part 2: Mim8 Once Monthly - Subjects 1-5 years Part 2: Mim8 Once Monthly - Subjects 6-11 years Part 1+2: Mim8 Once Weekly - Subjects 1-5 years Part 1+2: Mim8 Once Weekly - Subjects 6-11 years
    Number of subjects analysed
    17
    15
    36
    34
    Units: events
    20
    27
    99
    85
    No statistical analyses for this end point

    Secondary: Number of treated bleeds

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    End point title
    		 Number of treated bleeds
    End point description
    A bleed is considered treated if a coagulation factor product is administered to stop the bleed. Number of treated bleeds are presented. The endpoint was evaluated based on in-study period. The in-study period started at the first dose of Mim8 or screening (for run-in) and ended at the end of treatment period or discontinuation of Mim8. Analysis population (full analysis set [FAS]) included all subjects exposed to at least 1 dose of trial product. Subjects were analysed according to the treatment they actually received. As defined in protocol section 9, analysis was planned to be performed for once weekly and once monthly treatment regimens. Hence, all of subjects during part 1 as well as that decided to continue on Mim8 once weekly in part 2 are combined and represented in part 1+2 once weekly reporting group.
    End point type
    Secondary
    End point timeframe
    From treatment initiation to end of treatment (week 0 to week 52)
    End point values
    		 Part 2: Mim8 Once Monthly - Subjects 1-5 years Part 2: Mim8 Once Monthly - Subjects 6-11 years Part 1+2: Mim8 Once Weekly - Subjects 1-5 years Part 1+2: Mim8 Once Weekly - Subjects 6-11 years
    Number of subjects analysed
    17
    15
    36
    34
    Units: bleeds
    2
    2
    15
    20
    No statistical analyses for this end point

    Secondary: Number of treated spontaneous bleeds

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    End point title
    		 Number of treated spontaneous bleeds
    End point description
    A bleed is considered treated if a coagulation factor product is administered to stop the bleed. A spontaneous bleed refers bleeding instance that occur without a clear cause or not linked to a specific, known event or activity. Number of treated spontaneous bleeds are presented. The endpoint was evaluated based on in-study period. The in-study period started at the first dose of Mim8 or screening (for run-in) and ended at the end of treatment period or discontinuation of Mim8. FAS included all subjects exposed to at least 1 dose of trial product. Subjects were analysed according to the treatment they actually received. As defined in protocol section 9, analysis was planned to be performed for once weekly and once monthly treatment regimens. Hence, all of subjects during part 1 as well as that decided to continue on Mim8 once weekly in part 2 are combined and represented in part 1+2 once weekly reporting group.
    End point type
    Secondary
    End point timeframe
    From treatment initiation to end of treatment (week 0 to week 52)
    End point values
    		 Part 2: Mim8 Once Monthly - Subjects 1-5 years Part 2: Mim8 Once Monthly - Subjects 6-11 years Part 1+2: Mim8 Once Weekly - Subjects 1-5 years Part 1+2: Mim8 Once Weekly - Subjects 6-11 years
    Number of subjects analysed
    17
    15
    36
    34
    Units: bleeds
    0
    1
    2
    3
    No statistical analyses for this end point

    Secondary: Number of treated joint bleeds

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    End point title
    		 Number of treated joint bleeds
    End point description
    A bleed is considered treated if a coagulation factor product is administered to stop the bleed. A joint bleed refers bleeding instance that is caused in joints. Number of treated joints bleeds are presented. The endpoint was evaluated based on in-study period. The in-study period started at the first dose of Mim8 or screening (for run-in) and ended at the end of treatment period or discontinuation of Mim8. FAS included all subjects exposed to at least 1 dose of trial product. Subjects were analysed according to the treatment they actually received. As defined in protocol section 9, analysis was planned to be performed for once weekly and once monthly treatment regimens. Hence, all of subjects during part 1 as well as that decided to continue on Mim8 once weekly in part 2 are combined and represented in part 1+2 once weekly reporting group.
    End point type
    Secondary
    End point timeframe
    From treatment initiation to end of treatment (week 0 to week 52)
    End point values
    		 Part 2: Mim8 Once Monthly - Subjects 1-5 years Part 2: Mim8 Once Monthly - Subjects 6-11 years Part 1+2: Mim8 Once Weekly - Subjects 1-5 years Part 1+2: Mim8 Once Weekly - Subjects 6-11 years
    Number of subjects analysed
    17
    15
    36
    34
    Units: bleeds
    0
    2
    3
    12
    No statistical analyses for this end point

    Secondary: Number of treated traumatic bleeds

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    End point title
    		 Number of treated traumatic bleeds
    End point description
    A bleed is considered treated if a coagulation factor product is administered to stop the bleed. A traumatic bleed refers bleeding instance that is caused by a specific, known event or activity (e.g. injury or exercise). Number of treated traumatic bleeds are presented. The endpoint was evaluated based on in-study period. The in-study period started at the first dose of Mim8 or screening (for run-in) and ended at the end of treatment period or discontinuation of Mim8. FAS included all subjects exposed to at least 1 dose of trial product. Subjects were analysed according to the treatment they actually received. As defined in protocol section 9, analysis was planned to be performed for once weekly and once monthly treatment regimens. Hence, all of subjects during part 1 as well as that decided to continue on Mim8 once weekly in part 2 are combined and represented in part 1+2 once weekly reporting group.
    End point type
    Secondary
    End point timeframe
    From treatment initiation to end of treatment (week 0 to week 52)
    End point values
    		 Part 2: Mim8 Once Monthly - Subjects 1-5 years Part 2: Mim8 Once Monthly - Subjects 6-11 years Part 1+2: Mim8 Once Weekly - Subjects 1-5 years Part 1+2: Mim8 Once Weekly - Subjects 6-11 years
    Number of subjects analysed
    17
    15
    36
    34
    Units: bleeds
    2
    1
    12
    16
    No statistical analyses for this end point

    Secondary: Number of treated target joint bleeds

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    End point title
    		 Number of treated target joint bleeds
    End point description
    A bleed is considered treated if a coagulation factor product is administered to stop the bleed. A target joint is defined as a joint in which 3 or more spontaneous bleeding episodes have occurred within 6 months before the date of the assessment. Number of treated target joints bleeds are presented. The endpoint was evaluated based on in-study period. The in-study period started at the first dose of Mim8 or screening (for run-in) and ended at the end of treatment period or discontinuation of Mim8. FAS included all subjects exposed to at least 1 dose of trial product. Subjects were analysed according to the treatment they actually received. As defined in protocol section 9, analysis was planned to be performed for once weekly and once monthly treatment regimens. Hence, all of subjects during part 1 as well as that decided to continue on Mim8 once weekly in part 2 are combined and represented in part 1+2 once weekly reporting group.
    End point type
    Secondary
    End point timeframe
    From treatment initiation to end of treatment (week 0 to week 52)
    End point values
    		 Part 2: Mim8 Once Monthly - Subjects 1-5 years Part 2: Mim8 Once Monthly - Subjects 6-11 years Part 1+2: Mim8 Once Weekly - Subjects 1-5 years Part 1+2: Mim8 Once Weekly - Subjects 6-11 years
    Number of subjects analysed
    17
    15
    36
    34
    Units: bleeds
    0
    0
    0
    2
    No statistical analyses for this end point

    Secondary: Number of injection site reactions

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    End point title
    		 Number of injection site reactions
    End point description
    Injection site reactions were assessed based on subject feedback and visual inspection of injection site. Number of injection site reactions are presented. Endpoint was evaluated based on data from on-treatment period. The on-treatment period represented the time period in which a subject was considered exposed to trial product and started at the date of first dose of trial product and ended at the first date of any of the following: end of treatment visit if they enrolled in the study NN7769-4532 or the follow-up visit and the last date on trial product + 21 weeks. SAS included all subjects exposed to at least 1 dose of trial product and were analysed according to the treatment they actually received. As defined in protocol section 9, analysis was planned to be performed for once weekly and once monthly treatment regimens. Hence, all of subjects during part 1 as well as that decided to continue on Mim8 once weekly in part 2 are combined and represented in part 1+2 once weekly group.
    End point type
    Secondary
    End point timeframe
    From treatment initiation to end of treatment (week 0 to week 52)
    End point values
    		 Part 2: Mim8 Once Monthly - Subjects 1-5 years Part 2: Mim8 Once Monthly - Subjects 6-11 years Part 1+2: Mim8 Once Weekly - Subjects 1-5 years Part 1+2: Mim8 Once Weekly - Subjects 6-11 years
    Number of subjects analysed
    17
    15
    36
    34
    Units: site reactions
    0
    5
    6
    20
    No statistical analyses for this end point

    Secondary: Occurrence of anti-Mim8 antibodies

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    End point title
    		 Occurrence of anti-Mim8 antibodies
    End point description
    Number of subjects with anti-Mim8 antibodies are presented. The endpoint was evaluated based on data from on-treatment period. The on-treatment period represented the time period in which a subject was considered exposed to trial product and started at the date of first dose of trial product and ended at the first date of any of the following: end of treatment visit if they enrolled in the study NN7769-4532 or the follow-up visit and the last date on trial product + 21 weeks. SAS included all subjects exposed to at least 1 dose of trial product. Subjects were analysed according to the treatment they actually received. As defined in protocol section 9, analysis was planned to be performed for once weekly and once monthly treatment regimens. Hence, all of subjects during part 1 as well as that decided to continue on Mim8 once weekly in part 2 are combined and represented in part 1+2 once weekly reporting group.
    End point type
    Secondary
    End point timeframe
    From treatment initiation to end of treatment (week 0 to week 52)
    End point values
    		 Part 2: Mim8 Once Monthly - Subjects 1-5 years Part 2: Mim8 Once Monthly - Subjects 6-11 years Part 1+2: Mim8 Once Weekly - Subjects 1-5 years Part 1+2: Mim8 Once Weekly - Subjects 6-11 years
    Number of subjects analysed
    17
    15
    36
    34
    Units: subjects
    0
    0
    1
    4
    No statistical analyses for this end point

    Secondary: Consumption of factor product per bleed treatment (number of injections per bleed)

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    End point title
    		 Consumption of factor product per bleed treatment (number of injections per bleed)
    End point description
    Consumption of factor product per bleed treatment was calculated as number of injections used in the time period from the start of bleed to the stop of bleed. The mean number of injections required to stop the bleed is presented. The analysis was based on the total number of bleeds. The endpoint was evaluated based on in-study period. The in-study period starts at the first dose of Mim8 or screening (for run-in) and ends at the end of treatment period or discontinuation of Mim8. For subjects with run-in, the in-study period starts at screening and ends at the end of treatment period or discontinuation of Mim8. FAS included all subjects exposed to at least 1 dose of trial product. Subjects were analysed according to the treatment they actually received.
    End point type
    Secondary
    End point timeframe
    From run-in initiation to end of treatment (week -26 to week 52)
    End point values
    		 Part 1: Mim8 Once Weekly - Subjects 1-5 years Part 2: Mim8 Once Weekly - Subjects 1-5 years Part 1: Mim8 Once Weekly - Subjects 6-11 years Part 2: Mim8 Once Weekly - Subjects 6-11 years Part 2: Mim8 Once Monthly - Subjects 1-5 years Part 2: Mim8 Once Monthly - Subjects 6-11 years Run-in: Previous PPX + Previous On Demand Subjects 1-5 years Run-in: Previous PPX + Previous On Demand Subjects 6-11 years
    Number of subjects analysed
    36
    19
    34
    19
    17
    15
    19
    19
    Units: injections per bleed
        arithmetic mean (standard deviation)
    1.0 ( 0.0 )
    1.0 ( 0.0 )
    1.4 ( 0.8 )
    1.3 ( 0.6 )
    1.0 ( 0.0 )
    1.5 ( 0.7 )
    1.1 ( 0.3 )
    1.3 ( 0.8 )
    No statistical analyses for this end point

    Secondary: Mim8 plasma concentration

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    End point title
    		 Mim8 plasma concentration
    End point description
    The endpoint was evaluated based on in-study period. The in-study period started at the first dose of Mim8 or screening (for run-in) and ended at the end of treatment period or discontinuation of Mim8. FAS included all subjects exposed to at least 1 dose of trial product. Subjects were analysed according to the treatment they actually received. Here, "n" represents number of subjects who evaluable at specific time point for respective reporting group and "99999" indicates not applicable data point.
    End point type
    Secondary
    End point timeframe
    From treatment initiation to end of treatment (week 0 to week 52)
    End point values
    		 Part 1: Mim8 Once Weekly - Subjects 1-5 years Part 2: Mim8 Once Weekly - Subjects 1-5 years Part 1: Mim8 Once Weekly - Subjects 6-11 years Part 2: Mim8 Once Weekly - Subjects 6-11 years Part 2: Mim8 Once Monthly - Subjects 1-5 years Part 2: Mim8 Once Monthly - Subjects 6-11 years
    Number of subjects analysed
    36
    19
    34
    19
    17
    15
    Units: microgram per milliliter (μg/mL)
    arithmetic mean (standard deviation)
        Week 0(n=33,34,0,0,0,0)
    0.0 ( 0.0 )
    99999 ( 99999 )
    0.0 ( 0.0 )
    99999 ( 99999 )
    99999 ( 99999 )
    99999 ( 99999 )
        Week 26 (n=35,34,0,0,0,0)
    6.81 ( 2.43 )
    99999 ( 99999 )
    5.07 ( 1.98 )
    99999 ( 99999 )
    99999 ( 99999 )
    99999 ( 99999 )
        Week 36 (n=0,0,19,19,17,15)
    99999 ( 99999 )
    6.77 ( 2.41 )
    99999 ( 99999 )
    4.36 ( 1.40 )
    6.97 ( 3.02 )
    5.22 ( 1.62 )
        Week 52 (n=0,0,19,19,16,15)
    99999 ( 99999 )
    6.18 ( 2.02 )
    99999 ( 99999 )
    4.13 ( 1.63 )
    4.01 ( 1.33 )
    4.03 ( 1.88 )
    No statistical analyses for this end point

    Secondary: Treatment preference for Mim8 versus previous treatment using Caregiver Haemophilia Patient Preference Questionnaire (Caregiver H-PPQ)

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    End point title
    		 Treatment preference for Mim8 versus previous treatment using Caregiver Haemophilia Patient Preference Questionnaire (Caregiver H-PPQ)
    End point description
    The questionnaire assesses "Overall which treatment do you prefer?”, with the response options “current”, “previous” or “no preference”, and "How strong is that preference", with the response options “very strong”, “fairly strong”, and “not very strong”. Percentage of subjects with treatment preference for Mim8 versus previous treatment using caregiver H-PPQ is presented. The caregiver H-PPQ is only relevant for previously treated subjects. The endpoint was evaluated based on in-study period. The in-study period started at the first dose of Mim8 or screening (for run-in) and ended at the end of treatment period or discontinuation of Mim8. FAS included all subjects exposed to at least 1 dose of trial product. Subjects were analysed according to the treatment they actually received. Here, "number of subjects analysed" included subjects who had responded the questionnaire within a 1 day interval and applicable for previously treated subjects at week 26.
    End point type
    Secondary
    End point timeframe
    Once during treatment (week 26)
    End point values
    		 Part 1: Mim8 Once Weekly - Subjects 1-5 years Part 1: Mim8 Once Weekly - Subjects 6-11 years
    Number of subjects analysed
    30
    30
    Units: percentage of subjects
    number (not applicable)
        Current treatment: Very strong
    76.7
    70.0
        Current treatment: Fairly strong
    20.0
    23.3
        Current treatment: Not very strong
    3.3
    3.3
        Previous treatment
    0
    0
        No preference
    0
    3.3
    No statistical analyses for this end point

    Secondary: Change in physical function domain of Paediatric Quality of Life inventory (PEDS-QL) Generic Core Scales

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    End point title
    		 Change in physical function domain of Paediatric Quality of Life inventory (PEDS-QL) Generic Core Scales
    End point description
    PedsQL assesses quality of life, including domains like physical functioning. Higher scores indicate better quality of life and physical functioning with range from 0-100. Positive change indicates improvement and negative change indicates worsening. Questionnaire is for 5 to 11 years. Change in PEDS-QL from week 0 to week 52 is presented. Endpoint based on in-study period which started at first dose of Mim8 or screening (for run-in) and ended at end of treatment or discontinuation of Mim8. FAS included subjects exposed to at least 1 dose of trial product and analysed based on treatment they actually received. In protocol section 9, analysis was planned to be performed for once weekly and once monthly treatment regimens. Hence, all of subjects during part 1 as well as that decided to continue on Mim8 once weekly in part 2 are combined and represented in part 1+2 once weekly group. Here, "subject analysed" included responders at week 0 and week 52 and "99999" indicates not applicable.
    End point type
    Secondary
    End point timeframe
    From treatment initiation to end of treatment (week 0 to week 52)
    End point values
    		 Part 2: Mim8 Once Monthly - Subjects 1-5 years Part 2: Mim8 Once Monthly - Subjects 6-11 years Part 1+2: Mim8 Once Weekly - Subjects 1-5 years Part 1+2: Mim8 Once Weekly - Subjects 6-11 years
    Number of subjects analysed
    1
    12
    3
    13
    Units: score on a scale
        arithmetic mean (standard deviation)
    0.0 ( 99999 )
    7.0 ( 13.1 )
    -1.0 ( 1.8 )
    23.8 ( 18.4 )
    No statistical analyses for this end point

    Secondary: Change in subjects’ treatment burden using the Haemophilia treatment experience measure (Hemo TEM)

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    End point title
    		 Change in subjects’ treatment burden using the Haemophilia treatment experience measure (Hemo TEM)
    End point description
    Child Hemo-TEM (caregiver reported) measures treatment burden. Change in subjects’ treatment burden using Hemo TEM from week 0 to week 52 is presented. The score ranges from 0-100 where lower score indicates lower treatment burden. The caregiver H-PPQ is only relevant for previously treated subjects. Endpoint was evaluated based on in-study period. The in-study period started at the first dose of Mim8 or screening (run-in) and ended at the end of treatment period or discontinuation of Mim8. FAS included all subjects exposed to at least 1 dose of trial product. Subjects were analysed according to the treatment they actually received. In protocol section 9, analysis was planned to be performed for once weekly and once monthly treatment regimens. Hence, all of subjects during part 1 as well as that decided to continue on Mim8 once weekly in part 2 are combined and represented in part 1+2 once weekly reporting group. Here, "subject analysed" includes responders at week 0 and week 52.
    End point type
    Secondary
    End point timeframe
    From treatment initiation to end of treatment (week 0 to week 52)
    End point values
    		 Part 2: Mim8 Once Monthly - Subjects 1-5 years Part 2: Mim8 Once Monthly - Subjects 6-11 years Part 1+2: Mim8 Once Weekly - Subjects 1-5 years Part 1+2: Mim8 Once Weekly - Subjects 6-11 years
    Number of subjects analysed
    10
    12
    14
    13
    Units: score on a scale
        arithmetic mean (standard deviation)
    -14.6 ( 14.3 )
    -19.6 ( 15.3 )
    -13.0 ( 25.0 )
    -26.9 ( 28.7 )
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    From treatment initiation to follow up visit (week 0 to week 72)
    Adverse event reporting additional description
    Based on on-treatment period - period started at the date of first dose and ended at the first date of any of following: end of treatment if they enrolled in NN7769-4532 or follow-up and last date on trial product +21 weeks. SAS included all subjects exposed to at least 1 dose of trial product and based on treatment they actually received.
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    27.1
    Reporting groups
    Reporting group title
    Part 1+2: Mim8 - Subjects 6-11 years
    Reporting group description
    		 All subjects aged 6-11 years received subcutaneously Mim8 once weekly in part 1 and part 2 or once weekly in part 1 and once monthly in part 2 from week 0 until week 52. The part 2 treatment period was followed by a 21-week follow-up period for all subjects unless the subjects or the parent(s)/caregiver(s) wanted to transfer to the open-label extension study NN7769-4532.

    Reporting group title
    Part 1+2: Mim8 - Subjects 1-5 years
    Reporting group description
    		 All subjects aged 1-5 years received subcutaneously Mim8 once weekly in part 1 and part 2 or once weekly in part 1 and once monthly in part 2 from week 0 until week 52. The part 2 treatment period was followed by a 21-week follow-up period for all subjects unless the subjects or the parent(s)/caregiver(s) wanted to transfer to the open-label extension study NN7769-4532.

    Serious adverse events
    		 Part 1+2: Mim8 - Subjects 6-11 years Part 1+2: Mim8 - Subjects 1-5 years
    Total subjects affected by serious adverse events
         subjects affected / exposed
    4 / 34 (11.76%)
    1 / 36 (2.78%)
         number of deaths (all causes)
    0
    0
         number of deaths resulting from adverse events
    0
    0
    Injury, poisoning and procedural complications
    Head injury
         subjects affected / exposed
    0 / 34 (0.00%)
    1 / 36 (2.78%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Musculoskeletal and connective tissue disorders
    Myositis
         subjects affected / exposed
    0 / 34 (0.00%)
    1 / 36 (2.78%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Infections and infestations
    Pneumonia
         subjects affected / exposed
    1 / 34 (2.94%)
    0 / 36 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Mycoplasma infection
         subjects affected / exposed
    1 / 34 (2.94%)
    0 / 36 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Pneumonia influenzal
         subjects affected / exposed
    1 / 34 (2.94%)
    0 / 36 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Pharyngitis streptococcal
         subjects affected / exposed
    1 / 34 (2.94%)
    0 / 36 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Pneumonia haemophilus
         subjects affected / exposed
    1 / 34 (2.94%)
    0 / 36 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 Part 1+2: Mim8 - Subjects 6-11 years Part 1+2: Mim8 - Subjects 1-5 years
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    23 / 34 (67.65%)
    30 / 36 (83.33%)
    Investigations
    Blood alkaline phosphatase increased
         subjects affected / exposed
    2 / 34 (5.88%)
    0 / 36 (0.00%)
         occurrences all number
    3
    0
    Prothrombin fragment 1.2 increased
         subjects affected / exposed
    6 / 34 (17.65%)
    3 / 36 (8.33%)
         occurrences all number
    7
    4
    Fibrin D dimer increased
         subjects affected / exposed
    0 / 34 (0.00%)
    2 / 36 (5.56%)
         occurrences all number
    0
    2
    Injury, poisoning and procedural complications
    Fall
         subjects affected / exposed
    2 / 34 (5.88%)
    2 / 36 (5.56%)
         occurrences all number
    3
    2
    Skin laceration
         subjects affected / exposed
    2 / 34 (5.88%)
    1 / 36 (2.78%)
         occurrences all number
    2
    1
    Product communication issue
         subjects affected / exposed
    2 / 34 (5.88%)
    1 / 36 (2.78%)
         occurrences all number
    2
    1
    Nervous system disorders
    Headache
         subjects affected / exposed
    1 / 34 (2.94%)
    2 / 36 (5.56%)
         occurrences all number
    1
    2
    General disorders and administration site conditions
    Injection site reaction
         subjects affected / exposed
    2 / 34 (5.88%)
    5 / 36 (13.89%)
         occurrences all number
    25
    6
    Pyrexia
         subjects affected / exposed
    5 / 34 (14.71%)
    9 / 36 (25.00%)
         occurrences all number
    5
    10
    Gastrointestinal disorders
    Dental caries
         subjects affected / exposed
    0 / 34 (0.00%)
    2 / 36 (5.56%)
         occurrences all number
    0
    2
    Vomiting
         subjects affected / exposed
    2 / 34 (5.88%)
    1 / 36 (2.78%)
         occurrences all number
    2
    1
    Respiratory, thoracic and mediastinal disorders
    Cough
         subjects affected / exposed
    4 / 34 (11.76%)
    5 / 36 (13.89%)
         occurrences all number
    4
    5
    Skin and subcutaneous tissue disorders
    Dermatitis contact
         subjects affected / exposed
    0 / 34 (0.00%)
    2 / 36 (5.56%)
         occurrences all number
    0
    2
    Infections and infestations
    Bronchitis
         subjects affected / exposed
    0 / 34 (0.00%)
    4 / 36 (11.11%)
         occurrences all number
    0
    4
    Upper respiratory tract infection
         subjects affected / exposed
    6 / 34 (17.65%)
    9 / 36 (25.00%)
         occurrences all number
    6
    10
    Influenza
         subjects affected / exposed
    1 / 34 (2.94%)
    3 / 36 (8.33%)
         occurrences all number
    1
    3
    Nasopharyngitis
         subjects affected / exposed
    4 / 34 (11.76%)
    2 / 36 (5.56%)
         occurrences all number
    5
    5
    Respiratory tract infection
         subjects affected / exposed
    0 / 34 (0.00%)
    2 / 36 (5.56%)
         occurrences all number
    0
    2
    Viral upper respiratory tract infection
         subjects affected / exposed
    0 / 34 (0.00%)
    3 / 36 (8.33%)
         occurrences all number
    0
    4
    Viral infection
         subjects affected / exposed
    2 / 34 (5.88%)
    1 / 36 (2.78%)
         occurrences all number
    2
    1
    Varicella
         subjects affected / exposed
    0 / 34 (0.00%)
    3 / 36 (8.33%)
         occurrences all number
    0
    3

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    25 Jun 2021
    Amended to extend the follow-up period from 16 to 21 weeks after last dose; to clarify inclusion criterion 4 - it will be assessed by checking if the subject had a prescription of FVIII concentrate or bypassing agent; correction to align with the study design, as subjects treated on demand have the option to have a run-in period; Correction to specify that the target joints only need be assessed at week 4; to specify that clinical safety data from the phase 1 study will be summarised before this study will be initiated and that the summary will be submitted to regulatory authorities if and as required.
    26 Aug 2021
    Amended to add statement to clarify children under which age group are allowed for the first 10 subjects to be dosed in the study; in order to describe the involvement of data monitoring committee (DMC) in decisions regarding study and confirmation of the role of DMC in the study.
    22 Aug 2022
    Amended to include the dose for subjects based on the multiple ascending dose (MAD) part of study NN7769-4513 and on study NN7769-4882; exclusion criterion updated regarding requirement of participation in any interventional clinical study prior to this study; exclusion criterion updated regarding exposure to non-factor haemostatic products; to comply with section about concomitant medication; updated sections based on interim CTR from study NN7769-4513; to specify an aim to obtain equal distribution of the subjects in the study.

    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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