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    Clinical Trial Results:
    A Phase IIIb, open-label, single-arm, single-dose, multicenter study to evaluate the safety, tolerability and efficacy of gene replacement therapy with intravenous OAV101 (AVXS-101) in pediatric patients with spinal muscular atrophy (SMA)

    Summary
    EudraCT number
    		 2020-005995-37
    Trial protocol
    		 FR   DE   BE   PT   IT  
    Global end of trial date
    13 Jun 2023

    Results information
    Results version number
    		 v2(current)
    This version publication date
    20 Jan 2024
    First version publication date
    24 Dec 2023
    Other versions
    		 v1
    Version creation reason

    Trial information

    		 Close Top of page
    Trial identification
    Sponsor protocol code
    COAV101A12306
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT04851873
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Novartis Pharma AG
    Sponsor organisation address
    Novartis Campus, Basel, Switzerland,
    Public contact
    Clinical Disclosure Office, Novartis Pharma AG, 41 613241111, Novartis.email@Novartis.com
    Scientific contact
    Clinical Disclosure Office, Novartis Pharma AG, 41 613241111, Novartis.email@Novartis.com
    Paediatric regulatory details
    Is trial part of an agreed paediatric investigation plan (PIP)
    Yes
    EMA paediatric investigation plan number(s)
    		 EMEA-002168-PIP01-17
    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
    13 Jun 2023
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    13 Jun 2023
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    The primary objective was to assess the safety and tolerability of intravenous (IV) OAV101 over a 12-month period in participants with SMA weighing ≥8.5 kg to ≤ 21 kg.
    Protection of trial subjects
    The study was in compliance with the ethical principles derived from the Declaration of Helsinki and the International Conference on Harmonization (ICH) Good Clinical Practice (GCP) guidelines. All the local regulatory requirements pertinent to safety of trial subjects were also followed during the conduct of the trial.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    08 Sep 2021
    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
    Australia: 3
    Country: Number of subjects enrolled
    Belgium: 2
    Country: Number of subjects enrolled
    Canada: 4
    Country: Number of subjects enrolled
    France: 2
    Country: Number of subjects enrolled
    United Kingdom: 3
    Country: Number of subjects enrolled
    Italy: 2
    Country: Number of subjects enrolled
    Portugal: 3
    Country: Number of subjects enrolled
    Taiwan: 3
    Country: Number of subjects enrolled
    United States: 2
    Worldwide total number of subjects
    24
    EEA total number of subjects
    9
    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)
    1
    Children (2-11 years)
    23
    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
    		 Participants took part in 13 investigative sites across 9 countries.

    Pre-assignment
    Screening details
    		 The screening period began after signature of the study informed consent. The study included a screening period of up to 45 days. On Day -1, participants were admitted to the hospital for pre-treatment baseline procedures. On Day 1, participants received the study treatment.

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

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 OAV101 1.1e14 vg/kg 8.5-13 kg
    Arm description
    		 8.5-13 kg
    Arm type
    		 Experimental

    Investigational medicinal product name
    Onasemnogene abeparvovec
    Investigational medicinal product code
    OAV101
    Other name
    AVXS-101; Zolgensma
    Pharmaceutical forms
    Solution for infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    OAV101 1.1e14 vg/kg

    Arm title
    		 OAV101 1.1e14 vg/kg >13-17 kg
    Arm description
    		 >13-17 kg
    Arm type
    		 Experimental

    Investigational medicinal product name
    Onasemnogene abeparvovec
    Investigational medicinal product code
    OAV101
    Other name
    AVXS-101; Zolgensma
    Pharmaceutical forms
    Solution for infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    OAV101 1.1e14 vg/kg

    Arm title
    		 OAV101 1.1e14 vg/kg >17-21 kg
    Arm description
    		 >17-21 kg
    Arm type
    		 Experimental

    Investigational medicinal product name
    Onasemnogene abeparvovec
    Investigational medicinal product code
    OAV101
    Other name
    AVXS-101; Zolgensma
    Pharmaceutical forms
    Solution for infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    OAV101 1.1e14 vg/kg

    Number of subjects in period 1
    		OAV101 1.1e14 vg/kg 8.5-13 kg OAV101 1.1e14 vg/kg >13-17 kg OAV101 1.1e14 vg/kg >17-21 kg
    Started
    7
    8
    9
    Completed
    7
    8
    9

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    OAV101 1.1e14 vg/kg 8.5-13 kg
    Reporting group description
    		 8.5-13 kg

    Reporting group title
    OAV101 1.1e14 vg/kg >13-17 kg
    Reporting group description
    		 >13-17 kg

    Reporting group title
    OAV101 1.1e14 vg/kg >17-21 kg
    Reporting group description
    		 >17-21 kg

    Reporting group values
    		 OAV101 1.1e14 vg/kg 8.5-13 kg OAV101 1.1e14 vg/kg >13-17 kg OAV101 1.1e14 vg/kg >17-21 kg Total
    Number of subjects
    		 7 8 9 24
    Age Categorical
    Units: Participants
        0 < 28 days
    		 0 0 0 0
        28 days - < 2 years
    		 1 0 0 1
        2 years - < 12 years
    		 6 8 9 23
        12 years - <18 years
    		 0 0 0 0
        >= 18 years
    		 0 0 0 0
    Age Continuous
    Units: years
        arithmetic mean (standard deviation)
    		 3.027 ± 1.1458 4.519 ± 1.1769 6.137 ± 1.6018 -
    Sex: Female, Male
    Units: Participants
        Female
    		 3 5 4 12
        Male
    		 4 3 5 12
    Race (NIH/OMB)
    Units: Subjects
        American Indian or Alaska Native
    		 0 0 0 0
        Asian
    		 1 5 3 9
        Native Hawaiian or Other Pacific Islander
    		 0 0 0 0
        Black or African American
    		 0 0 0 0
        White
    		 6 1 6 13
        More than one race
    		 0 0 0 0
        Unknown or Not Reported
    		 0 2 0 2

    End points

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    End points reporting groups
    Reporting group title
    OAV101 1.1e14 vg/kg 8.5-13 kg
    Reporting group description
    		 8.5-13 kg

    Reporting group title
    OAV101 1.1e14 vg/kg >13-17 kg
    Reporting group description
    		 >13-17 kg

    Reporting group title
    OAV101 1.1e14 vg/kg >17-21 kg
    Reporting group description
    		 >17-21 kg

    Subject analysis set title
    OAV101 1.1e14 vg/kg Overall
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    Overall

    Subject analysis set title
    OAV101 1.1e14 vg/kg >13-17kg
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    >13-17kg

    Subject analysis set title
    OAV101 1.1e14 vg/kg >17-21kg
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    >17-21kg

    Subject analysis set title
    OAV101 1.1e14 vg/kg Overall
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    Overall

    Subject analysis set title
    OAV101 1.1e14 vg/kg 8.5-13 kg Overall
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    Overall

    Primary: Number of participants with treatment emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) by weight bracket

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    End point title
    		 Number of participants with treatment emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) by weight bracket [1]
    End point description
    An AE is any untoward medical occurrence (e.g. any unfavorable and unintended sign [including abnormal laboratory findings], symptom or disease) in a clinical investigation participant after providing written informed consent for participation in the study.
    End point type
    Primary
    End point timeframe
    Up to Month 12
    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 applicable for a single arm study with a single open label dose, even if sorted by weight groups.
    End point values
    		 OAV101 1.1e14 vg/kg 8.5-13 kg OAV101 1.1e14 vg/kg >13-17 kg OAV101 1.1e14 vg/kg >17-21 kg OAV101 1.1e14 vg/kg Overall
    Number of subjects analysed
    7
    8
    9
    24
    Units: Participants
        Any treatment-emergent adverse events (AEs)
    7
    8
    9
    24
        Any treatment-emergent AEs related to OAV101
    7
    8
    9
    24
        Any severe treatment-emergent AEs
    1
    4
    3
    8
        Any serious treatment-emergent AEs
    3
    7
    5
    15
        Serious treatment-emergent AEs related to OAV101
    1
    4
    2
    7
        Treatment-emergent AEs leading to study disc.
    0
    0
    0
    0
        Treatment-emergent AEs leading to death
    0
    0
    0
    0
        Treatment-emergent AEs of special interest
    7
    7
    9
    23
    No statistical analyses for this end point

    Primary: Number of participants with important identified and important potential risks (Adverse Events of Special Interest (AESI)) by risk name and weight bracket

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    End point title
    		 Number of participants with important identified and important potential risks (Adverse Events of Special Interest (AESI)) by risk name and weight bracket [2]
    End point description
    Important identified and important potential risks included the following AESIs: Hepatotoxicity, Thrombocytopenia, Cardiac adverse events, Dorsal root ganglia toxicity and Thrombotic microangiopathy. These were assessed by the investigator.
    End point type
    Primary
    End point timeframe
    Up to Month 12
    Notes
    [2] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: Not applicable for a single arm study with a single open label dose, even if sorted by weight groups.
    End point values
    		 OAV101 1.1e14 vg/kg 8.5-13 kg OAV101 1.1e14 vg/kg >13-17 kg OAV101 1.1e14 vg/kg >17-21 kg OAV101 1.1e14 vg/kg Overall
    Number of subjects analysed
    7
    8
    9
    24
    Units: Participants
        Hepatotoxicity
    6
    5
    9
    20
        Transient thrombocytopenia
    4
    6
    7
    17
        Cardiac adverse events
    0
    2
    1
    3
        Thrombotic microangiopathy
    0
    0
    0
    0
        Dorsal root ganglia cell inflammation
    0
    0
    0
    0
    No statistical analyses for this end point

    Primary: Summary of participants meeting criteria for potentially clinically significant vital sign values by weight bracket - systolic and diastolic blood pressure

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    End point title
    		 Summary of participants meeting criteria for potentially clinically significant vital sign values by weight bracket - systolic and diastolic blood pressure [3] [4]
    End point description
    Change from baseline in vital signs measurements - systolic and diastolic blood pressure (mmHg). Systolic Blood Pressure-Low:<=5th percentile of the age(Any Age), High:>=90th percentile of the age, gender, and height group (<18 yrs). Diastolic Blood Pressure-High:>=90th percentile of the age, gender, and height group(<18 yrs).
    End point type
    Primary
    End point timeframe
    12 months
    Notes
    [3] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: Not applicable for a single arm study with a single open label dose, even if sorted by weight groups.
    [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 applicable for a single arm study with a single open label dose, even if sorted by weight groups.
    End point values
    		 OAV101 1.1e14 vg/kg 8.5-13 kg OAV101 1.1e14 vg/kg Overall OAV101 1.1e14 vg/kg >13-17kg OAV101 1.1e14 vg/kg >17-21kg
    Number of subjects analysed
    7
    24
    8
    9
    Units: Participants
        Systolic Blood Pressure (mmHg) Low
    1
    2
    0
    1
        Systolic Blood Pressure (mmHg) High
    7
    23
    8
    8
        Diastolic Blood Pressure (mmHg) High
    7
    22
    7
    8
    No statistical analyses for this end point

    Primary: Change from baseline in vital signs measurements - Systolic Blood Pressure (mmHg)

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    End point title
    		 Change from baseline in vital signs measurements - Systolic Blood Pressure (mmHg) [5] [6]
    End point description
    End point type
    Primary
    End point timeframe
    Baseline, Days 2 and 3, Weeks 1, 2, 3, 4, 6, 8, 10, 13, 26, 39 and 52
    Notes
    [5] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: Not applicable for a single arm study with a single open label dose, even if sorted by weight groups.
    [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 applicable for a single arm study with a single open label dose, even if sorted by weight groups.
    End point values
    		 OAV101 1.1e14 vg/kg 8.5-13 kg OAV101 1.1e14 vg/kg Overall OAV101 1.1e14 vg/kg >13-17kg OAV101 1.1e14 vg/kg >17-21kg
    Number of subjects analysed
    7
    24
    8
    9
    Units: mmHg
    arithmetic mean (standard deviation)
        Change from baseline at Day 2
    5.3 ± 12.65
    1.3 ± 11.75
    -1.3 ± 13.01
    0.4 ± 10.36
        Change from baseline at Day 3
    -2.3 ± 15.01
    -1.4 ± 11.62
    -5.6 ± 11.16
    3.0 ± 8.38
        Change from baseline at Week 1
    13.4 ± 10.53
    2.2 ± 15.18
    -7.4 ± 15.59
    2.0 ± 12.84
        Change from baseline at Week 2
    11.7 ± 20.33
    2.8 ± 16.45
    -3.8 ± 17.60
    1.8 ± 9.15
        Change from baseline at Week 3 (n=6,8,9,23)
    15.8 ± 8.04
    2.7 ± 12.33
    -3.5 ± 12.87
    -0.7 ± 7.25
        Change from baseline at Week 4 (n=6,8,8,22)
    12.3 ± 16.50
    4.5 ± 15.05
    -3.5 ± 15.89
    6.8 ± 10.02
        Change from baseline at Week 6 (n=7,8,8,23)
    8.1 ± 12.92
    3.4 ± 18.13
    -2.8 ± 24.15
    5.4 ± 15.39
        Change from baseline at Week 8 (n=7,7,9,23)
    3.9 ± 15.49
    2.3 ± 13.69
    -0.7 ± 14.57
    3.6 ± 12.83
        Change from baseline at Week 10
    10.0 ± 16.99
    2.8 ± 14.32
    -5.6 ± 13.19
    4.6 ± 10.06
        Change from baseline at Week 13
    6.1 ± 10.78
    0.8 ± 13.28
    -4.5 ± 17.50
    1.4 ± 9.93
        Change from baseline at Week 26
    -5.7 ± 9.05
    -8.5 ± 13.46
    -12.9 ± 15.34
    -6.9 ± 14.99
        Change from baseline at Week 39 (n=7,7,9,23)
    16.4 ± 29.70
    0.3 ± 21.98
    -10.3 ± 13.59
    -3.9 ± 3.56
        Change from baseline at Week 52
    5.0 ± 21.60
    -1.1 ± 15.67
    -7.8 ± 13.54
    0.1 ± 10.81
    No statistical analyses for this end point

    Primary: Change from baseline in vital signs measurements - Diastolic Blood Pressure (mmHg)

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    End point title
    		 Change from baseline in vital signs measurements - Diastolic Blood Pressure (mmHg) [7] [8]
    End point description
    End point type
    Primary
    End point timeframe
    Baseline, Days 2 and 3, Weeks 1, 2, 3, 4, 6, 8, 10, 13, 26, 39 and 52
    Notes
    [7] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: Not applicable for a single arm study with a single open label dose, even if sorted by weight groups.
    [8] - 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 applicable for a single arm study with a single open label dose, even if sorted by weight groups.
    End point values
    		 OAV101 1.1e14 vg/kg 8.5-13 kg OAV101 1.1e14 vg/kg Overall OAV101 1.1e14 vg/kg >13-17kg OAV101 1.1e14 vg/kg >17-21kg
    Number of subjects analysed
    7
    24
    8
    9
    Units: mmHg
    arithmetic mean (standard deviation)
        Change from baseline at Day 2 (n=6,8,9,23)
    1.7 ± 12.42
    1.3 ± 12.71
    4.8 ± 13.22
    -2.1 ± 13.04
        Change from baseline at Day 3 (n=6,8,9,23)
    -3.7 ± 12.29
    -1.1 ± 11.22
    -5.1 ± 13.91
    4.2 ± 5.45
        Change from baseline at Week 1
    7.4 ± 20.07
    4.2 ± 15.13
    1.8 ± 13.56
    3.8 ± 13.41
        Change from baseline at Week 2
    7.1 ± 16.07
    6.2 ± 12.05
    7.3 ± 11.30
    4.4 ± 10.33
        Change from baseline at Week 3 (n=6,8,9,23)
    12.0 ± 15.09
    4.6 ± 13.28
    -1.5 ± 14.01
    5.0 ± 9.60
        Change from baseline at Week 4 (n=6,8,8,22)
    3.8 ± 19.27
    2.1 ± 12.12
    1.8 ± 9.97
    1.3 ± 8.53
        Change from baseline at Week 6 (n=6,8,8,22)
    10.3 ± 10.91
    5.0 ± 13.77
    -0.5 ± 17.87
    6.5 ± 10.14
        Change from baseline at Week 8 (n=6,7,9,22)
    10.5 ± 14.57
    6.2 ± 14.18
    3.0 ± 17.68
    5.9 ± 11.72
        Change from baseline at Week 10 (n=6,8,9,23)
    6.7 ± 15.55
    2.9 ± 12.01
    -2.0 ± 8.88
    4.8 ± 11.74
        Change from baseline at Week 13 (n=6,8,9,23)
    9.7 ± 15.37
    2.7 ± 13.20
    0.4 ± 13.54
    0.1 ± 11.03
        Change from baseline at Week 26
    -0.7 ± 9.05
    -3.7 ± 9.41
    -9.1 ± 10.26
    -1.2 ± 7.55
        Change from baseline at Week 39 (n=7,7,9,23)
    2.4 ± 15.73
    -0.8 ± 12.44
    -4.4 ± 13.01
    -0.4 ± 9.62
        Change from baseline at Week 52
    0.3 ± 11.74
    0.0 ± 9.60
    -2.0 ± 11.89
    1.7 ± 5.52
    No statistical analyses for this end point

    Primary: Change from baseline in vital signs measurements - Respiratory Rate (breaths/min)

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    End point title
    		 Change from baseline in vital signs measurements - Respiratory Rate (breaths/min) [9] [10]
    End point description
    Change from baseline in vital signs measurements - Respiratory Rate (breaths/min)
    End point type
    Primary
    End point timeframe
    Baseline, Days 2 and 3, Weeks 1, 2, 3, 4, 6, 8, 10, 13, 26, 39 and 52
    Notes
    [9] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: Not applicable for a single arm study with a single open label dose, even if sorted by weight groups.
    [10] - 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 applicable for a single arm study with a single open label dose, even if sorted by weight groups.
    End point values
    		 OAV101 1.1e14 vg/kg 8.5-13 kg OAV101 1.1e14 vg/kg Overall OAV101 1.1e14 vg/kg >13-17kg OAV101 1.1e14 vg/kg >17-21kg
    Number of subjects analysed
    7
    24
    8
    9
    Units: breaths/min)
    arithmetic mean (standard deviation)
        Change from baseline at Day 2
    -1.1 ± 3.08
    -0.6 ± 3.66
    -1.6 ± 3.74
    0.8 ± 3.96
        Change from baseline at Day 3
    -0.1 ± 5.87
    0.1 ± 4.66
    -0.1 ± 4.52
    0.4 ± 4.30
        Change from baseline at Week 1
    2.7 ± 6.78
    1.3 ± 5.45
    -0.1 ± 4.22
    1.6 ± 5.61
        Change from baseline at Week 2
    2.4 ± 5.44
    1.0 ± 4.81
    -0.8 ± 5.01
    1.4 ± 4.16
        Change from baseline at Week 3 (n=6,8,9,23)
    5.5 ± 8.96
    1.5 ± 6.63
    -1.8 ± 5.09
    1.8 ± 5.02
        Change from baseline at Week 4 (n=7,8,8,23)
    2.6 ± 4.54
    1.7 ± 5.31
    1.4 ± 7.35
    1.4 ± 4.00
        Change from baseline at Week 6 (n=7,8,8,23)
    5.1 ± 9.56
    1.4 ± 7.19
    -0.6 ± 4.75
    0.3 ± 6.43
        Change from baseline at Week 8 (n=7,7,9,23)
    3.3 ± 7.45
    0.8 ± 5.52
    -0.6 ± 4.47
    0.0 ± 4.42
        Change from baseline at Week 10
    3.3 ± 6.58
    0.5 ± 4.99
    -1.0 ± 4.31
    -0.3 ± 3.61
        Change from baseline at Week 13
    1.7 ± 6.75
    0.1 ± 4.44
    -0.5 ± 3.51
    -0.6 ± 2.92
        Change from baseline at Week 26
    4.1 ± 8.69
    1.7 ± 5.71
    0.8 ± 3.69
    0.7 ± 4.24
        Change from baseline at Week 39 (n=7,7,9,23)
    1.4 ± 5.03
    -0.3 ± 4.61
    -1.7 ± 4.35
    -0.6 ± 4.56
        Change from baseline at Week 52
    1.7 ± 7.34
    -0.5 ± 5.18
    -0.5 ± 4.66
    -2.2 ± 3.19
    No statistical analyses for this end point

    Primary: Change from baseline in vital signs measurements - Pulse Rate (beats/min)

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    End point title
    		 Change from baseline in vital signs measurements - Pulse Rate (beats/min) [11] [12]
    End point description
    Change from baseline in vital signs measurements - Pulse Rate (beats/min)
    End point type
    Primary
    End point timeframe
    Baseline, Days 2 and 3, Weeks 1, 2, 3, 4, 6, 8, 10, 13, 26, 39 and 52
    Notes
    [11] - 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 applicable for a single arm study with a single open label dose, even if sorted by weight groups.
    [12] - 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 applicable for a single arm study with a single open label dose, even if sorted by weight groups.
    End point values
    		 OAV101 1.1e14 vg/kg 8.5-13 kg OAV101 1.1e14 vg/kg Overall OAV101 1.1e14 vg/kg >13-17kg OAV101 1.1e14 vg/kg >17-21kg
    Number of subjects analysed
    7
    24
    8
    9
    Units: beats/min
    arithmetic mean (standard deviation)
        Change from baseline at Day 2
    3.3 ± 18.54
    9.2 ± 19.35
    6.3 ± 4.43
    16.3 ± 26.54
        Change from baseline at Day 3
    -1.4 ± 19.29
    2.4 ± 16.36
    -3.1 ± 14.21
    10.2 ± 14.19
        Change from baseline at Week 1
    1.4 ± 14.27
    -4.3 ± 17.19
    -9.6 ± 18.04
    -3.9 ± 18.84
        Change from baseline at Week 2
    -13.4 ± 21.25
    -5.1 ± 17.39
    -8.8 ± 15.21
    4.7 ± 12.12
        Change from baseline at Week 3 (n=6,8,9,23)
    21.0 ± 21.83
    9.3 ± 18.60
    -3.8 ± 12.46
    13.1 ± 15.12
        Change from baseline at Week 4 (n=7,8,8,23)
    -4.7 ± 17.27
    1.8 ± 19.95
    -3.5 ± 22.82
    12.8 ± 16.23
        Change from baseline at Week 6 (n=7,8,8,23)
    12.9 ± 20.70
    7.0 ± 16.58
    0.0 ± 9.59
    8.8 ± 17.64
        Change from baseline at Week 8 (n=7,7,9,23)
    -3.6 ± 15.75
    -0.5 ± 18.83
    -6.3 ± 22.69
    6.4 ± 17.63
        Change from baseline at Week 10
    -3.9 ± 25.14
    1.1 ± 19.08
    -4.3 ± 16.57
    9.7 ± 14.13
        Change from baseline at Week 13
    -6.4 ± 12.23
    2.2 ± 16.28
    2.6 ± 15.65
    8.4 ± 18.06
        Change from baseline at Week 26
    -6.9 ± 12.85
    -2.7 ± 15.12
    -6.3 ± 17.00
    3.8 ± 14.34
        Change from baseline at Week 39 (n=7,7,9,23)
    -4.7 ± 21.85
    -2.6 ± 17.54
    -7.7 ± 13.23
    3.1 ± 17.12
        Change from baseline at Week 52
    -8.9 ± 10.32
    -6.4 ± 16.35
    -16.4 ± 14.38
    4.4 ± 16.46
    No statistical analyses for this end point

    Primary: Summary of participants meeting criteria for potentially clinically significant vital sign values by weight bracket - temperature

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    End point title
    		 Summary of participants meeting criteria for potentially clinically significant vital sign values by weight bracket - temperature [13] [14]
    End point description
    Change from baseline in vital signs measurements - temperature (degrees Celsius) Temperature-Low:<=35ºC(Any Age),High:>=38.4ºC(<18 yrs).
    End point type
    Primary
    End point timeframe
    12 months
    Notes
    [13] - 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 applicable for a single arm study with a single open label dose, even if sorted by weight groups.
    [14] - 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 applicable for a single arm study with a single open label dose, even if sorted by weight groups.
    End point values
    		 OAV101 1.1e14 vg/kg 8.5-13 kg OAV101 1.1e14 vg/kg Overall OAV101 1.1e14 vg/kg >13-17kg OAV101 1.1e14 vg/kg >17-21kg
    Number of subjects analysed
    7
    24
    8
    9
    Units: Participants
        Temperature (ºC) Low
    0
    0
    0
    0
        Temperature (ºC) High
    1
    2
    0
    1
    No statistical analyses for this end point

    Primary: Change from baseline in vital signs measurements - Temperature (Degrees Celsius)

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    End point title
    		 Change from baseline in vital signs measurements - Temperature (Degrees Celsius) [15] [16]
    End point description
    End point type
    Primary
    End point timeframe
    Baseline, Days 2 and 3, Weeks 1, 2, 3, 4, 6, 8, 10, 13, 26, 39 and 52
    Notes
    [15] - 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 applicable for a single arm study with a single open label dose, even if sorted by weight groups.
    [16] - 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 applicable for a single arm study with a single open label dose, even if sorted by weight groups.
    End point values
    		 OAV101 1.1e14 vg/kg 8.5-13 kg OAV101 1.1e14 vg/kg Overall OAV101 1.1e14 vg/kg >13-17kg OAV101 1.1e14 vg/kg >17-21kg
    Number of subjects analysed
    7
    24
    8
    9
    Units: Degrees Celsius
    arithmetic mean (standard deviation)
        Change from baseline at Day 2
    0.07 ± 0.502
    0.01 ± 0.722
    -0.11 ± 0.714
    0.07 ± 0.918
        Change from baseline at Day 3
    0.11 ± 0.261
    -0.08 ± 0.541
    -0.48 ± 0.719
    0.12 ± 0.319
        Change from baseline at Week 1
    -0.07 ± 0.386
    -0.17 ± 0.528
    -0.44 ± 0.563
    0.00 ± 0.548
        Change from baseline at Week 2
    0.30 ± 0.592
    -0.08 ± 0.618
    -0.41 ± 0.649
    -0.09 ± 0.478
        Change from baseline at Week 3 (n=6,8,9,23)
    0.32 ± 0.556
    0.01 ± 0.474
    -0.24 ± 0.403
    0.02 ± 0.387
        Change from baseline at Week 4 (n=7,8,8,23)
    0.16 ± 0.673
    -0.04 ± 0.645
    -0.31 ± 0.775
    0.05 ± 0.431
        Change from baseline at Week 6 (n=7,8,8,23)
    0.26 ± 0.458
    -0.01 ± 0.535
    -0.18 ± 0.599
    -0.09 ± 0.500
        Change from baseline at Week 8 (n=7,7,9,23)
    0.13 ± 0.547
    -0.02 ± 0.521
    -0.21 ± 0.654
    0.01 ± 0.386
        Change from baseline at Week 10
    0.34 ± 0.351
    -0.05 ± 0.635
    -0.45 ± 0.727
    0.01 ± 0.553
        Change from baseline at Week 13
    0.13 ± 0.399
    0.05 ± 0.525
    -0.06 ± 0.701
    0.09 ± 0.473
        Change from baseline at Week 26
    0.31 ± 0.453
    0.05 ± 0.460
    -0.15 ± 0.532
    0.03 ± 0.324
        Change from baseline at Week 39 (n=7,7,9,23)
    0.33 ± 0.350
    0.03 ± 0.518
    -0.10 ± 0.622
    -0.11 ± 0.491
        Change from baseline at Week 52
    0.63 ± 0.836
    -0.03 ± 0.814
    -0.53 ± 0.819
    -0.11 ± 0.401
    No statistical analyses for this end point

    Primary: Change from baseline in vital signs measurements - oxygen saturation level

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    End point title
    		 Change from baseline in vital signs measurements - oxygen saturation level [17] [18]
    End point description
    Change from baseline in vital signs measurements - oxygen saturation level (%). Oxygen saturation is the fraction of oxygen-saturated hemoglobin relative to total hemoglobin (unsaturated+saturated) in the blood and then multiplied by 100.
    End point type
    Primary
    End point timeframe
    Baseline, Days 2 and 3, Weeks 1, 2, 3, 4, 6, 8, 10, 13, 26, 39 and 52
    Notes
    [17] - 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 applicable for a single arm study with a single open label dose, even if sorted by weight groups.
    [18] - 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 applicable for a single arm study with a single open label dose, even if sorted by weight groups.
    End point values
    		 OAV101 1.1e14 vg/kg 8.5-13 kg OAV101 1.1e14 vg/kg Overall OAV101 1.1e14 vg/kg >13-17kg OAV101 1.1e14 vg/kg >17-21kg
    Number of subjects analysed
    7
    24
    8
    9
    Units: % Oxygen Saturated
    arithmetic mean (standard deviation)
        Change from baseline at Day 2
    0.0 ± 0.82
    -0.3 ± 0.99
    -0.3 ± 1.04
    -0.4 ± 1.13
        Change from baseline at Day 3
    0.3 ± 1.80
    -0.5 ± 1.41
    -0.1 ± 0.83
    -1.3 ± 1.12
        Change from baseline at Week 1
    0.1 ± 1.07
    -0.4 ± 1.47
    -0.4 ± 2.00
    -0.8 ± 1.20
        Change from baseline at Week 2
    -0.7 ± 1.89
    -0.8 ± 1.79
    -1.0 ± 1.51
    -0.8 ± 2.11
        Change from baseline at Week 3 (n=6,8,9,23)
    -1.0 ± 1.90
    -0.8 ± 1.38
    -0.3 ± 1.28
    -1.1 ± 1.05
        Change from baseline at Week 4 (n=7,8,8,23)
    0.0 ± 1.91
    -0.4 ± 1.38
    -0.5 ± 1.31
    -0.8 ± 0.89
        Change from baseline at Week 6 (n=7,8,8,23)
    -0.1 ± 1.21
    -0.6 ± 1.31
    -1.0 ± 1.51
    -0.6 ± 1.19
        Change from baseline at Week 8 (n=7,7,9,23)
    -0.7 ± 1.89
    -0.4 ± 1.31
    0.0 ± 1.15
    -0.4 ± 0.88
        Change from baseline at Week 10
    0.3 ± 1.89
    -0.5 ± 1.72
    -1.0 ± 1.93
    -0.7 ± 1.32
        Change from baseline at Week 13
    0.1 ± 1.57
    -0.3 ± 1.43
    -0.5 ± 1.07
    -0.4 ± 1.67
        Change from baseline at Week 26
    -0.6 ± 1.51
    -0.3 ± 1.60
    -0.1 ± 1.36
    -0.2 ± 1.99
        Change from baseline at Week 39 (n=7,7,8,22)
    -1.0 ± 1.41
    -0.4 ± 1.22
    0.1 ± 0.90
    -0.4 ± 1.19
        Change from baseline at Week 52
    -1.0 ± 2.00
    -0.5 ± 1.64
    -0.6 ± 1.51
    -0.1 ± 1.54
    No statistical analyses for this end point

    Secondary: Achievement of development motor milestones according to the modified and combined WHO-MGRS and Bayley scale of Infant and Toddler Development.

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    End point title
    		 Achievement of development motor milestones according to the modified and combined WHO-MGRS and Bayley scale of Infant and Toddler Development.
    End point description
    The World Health Organization-Multicentre Growth Reference Study (WHO-MGRS) and Bayley scale of Infant and Toddler Development was modified and combined into a single scale expressly for this study, to measure developmental motor milestones. These were assessed via the milestone checklist, formed of 10 yes/no questions with optional video documentation. The developmental milestones are: head control, sitting with support, sitting without support, sitting without support for 30 seconds, hands-and-knees crawling, pulls to stand, standing with assistance, walking with assistance, standing alone and walking alone. A yes response indicates that the patient reached a particular development milestone. wk = Week w/out = without
    End point type
    Secondary
    End point timeframe
    Baseline, Week 26 and Week 52
    End point values
    		 OAV101 1.1e14 vg/kg 8.5-13 kg OAV101 1.1e14 vg/kg >13-17 kg OAV101 1.1e14 vg/kg >17-21 kg OAV101 1.1e14 vg/kg Overall
    Number of subjects analysed
    7
    8
    9
    24
    Units: Participants
        Baseline Head control (Bayley GM #4)
    7
    8
    9
    24
        Baseline Sits with support (Bayley GM #19)
    7
    8
    9
    24
        Baseline Sitting without support (WHO MGRS)
    6
    7
    8
    21
        Baseline Sits w/out support 30 s Bayley GM #26
    6
    7
    8
    21
        Baseline Hands-and-knees crawling (WHO MGRS)
    1
    3
    6
    10
        Baseline Pulls to stand (Bayley GM #35)
    1
    3
    5
    9
        Baseline Standing with assistance (WHO MGRS)
    0
    2
    5
    7
        Baseline Walking with assistance (WHO MGRS)
    0
    2
    5
    7
        Baseline Standing alone (WHO MGRS)
    0
    2
    4
    6
        Baseline Walking alone (WHO MGRS)
    0
    2
    4
    6
        Week 26 Head control (Bayley GM #4)
    7
    8
    9
    24
        Week 26 Sits with support (Bayley GM #19)
    7
    8
    9
    24
        Week 26 Sitting without support (WHO MGRS)
    7
    5
    9
    21
        Week 26 Sits without support 30 s Bayley GM #26
    7
    5
    8
    20
        Week 26 Hands-and-knees crawling (WHO MGRS)
    2
    3
    6
    11
        Week 26 Pulls to stand (Bayley GM #35)
    2
    3
    5
    10
        Week 26 Standing with assistance (WHO MGRS)
    1
    3
    5
    9
        Week 26 Walking with assistance (WHO MGRS)
    1
    3
    4
    8
        Week 26 Standing alone (WHO MGRS)
    0
    3
    4
    7
        Week 26 Walking alone (WHO MGRS)
    0
    2
    4
    6
        Week 52 Head control (Bayley GM #4)
    7
    8
    9
    24
        Week 52 Sits with support (Bayley GM #19)
    7
    7
    9
    23
        Week 52 Sitting without support (WHO MGRS)
    7
    7
    8
    22
        Week 52 Sits without support 30 s Bayley GM #26
    7
    6
    8
    21
        Week 52 Hands-and-knees crawling (WHO MGRS)
    2
    3
    6
    11
        Wk 52 Pulls to stand (Bayley GM #35) (n=7,8,8,23)
    2
    3
    5
    10
        Week 52 Standing with assistance (WHO MGRS)
    2
    3
    5
    10
        Week 52 Walking with assistance (WHO MGRS)
    1
    3
    4
    8
        Week 52 Standing alone (WHO MGRS)
    0
    3
    4
    7
        Week 52 Walking alone (WHO MGRS)
    0
    2
    4
    6
    No statistical analyses for this end point

    Secondary: Change from baseline in Hammersmith Functional Motor Scale - Expanded (HFMSE), as appropriate according to participant age

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    End point title
    		 Change from baseline in Hammersmith Functional Motor Scale - Expanded (HFMSE), as appropriate according to participant age
    End point description
    The HFMSE was devised for use in children with SMA to give objective information on motor ability and clinical progression. The HFMSE is formed of 33 assessments. Each motor skill item is scored on a 3 point Likert scale from 0 (no response) to 2 (full response), with a total score range of 0 to 66. A higher score indicates a higher level of ability.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 4, Week 13, Week 26, Week 39 and Week 52
    End point values
    		 OAV101 1.1e14 vg/kg 8.5-13 kg OAV101 1.1e14 vg/kg >13-17 kg OAV101 1.1e14 vg/kg >17-21 kg OAV101 1.1e14 vg/kg Overall
    Number of subjects analysed
    5
    6
    9
    20
    Units: HFMSE total score on a scale
    arithmetic mean (standard deviation)
        Change from baseline at Week 4 (n=4,6,9,19)
    3.8 ± 2.99
    2.3 ± 2.66
    3.1 ± 3.10
    3.0 ± 2.83
        Change from baseline at Week 13 (n=4,6,9,19)
    1.3 ± 7.89
    4.5 ± 3.99
    3.9 ± 3.98
    3.5 ± 4.83
        Change from baseline at Week 26 (n=5,6,9,20)
    3.4 ± 5.18
    4.3 ± 5.05
    3.1 ± 4.14
    3.6 ± 4.45
        Change from baseline at Week 39 (n=5,5,9,19)
    2.6 ± 6.11
    -0.6 ± 4.72
    4.3 ± 3.71
    2.6 ± 4.87
        Change from baseline at Week 52 (n=5,6,7,18)
    3.0 ± 5.24
    3.7 ± 5.75
    4.3 ± 4.07
    3.7 ± 4.73
    No statistical analyses for this end point

    Secondary: Change from baseline in Revised Upper Limb Module (RULM), as appropriate according to participant age.

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    End point title
    		 Change from baseline in Revised Upper Limb Module (RULM), as appropriate according to participant age.
    End point description
    The RULM assesses motor performance in the upper limbs from childhood through adulthood in ambulatory and non-ambulatory individuals with SMA. ‘The scale consists of an entry item to establish functional levels and 19 items covering distal to proximal movements. The entry item is a modified version of the Brooke scale, including activities ranging from no functional use of hands (score 0) to full bilateral shoulder abduction (score 6). The entry item does not contribute to the total score but serves as a functional classification of overall upper limb functional ability. Of the remaining 19 items, 18 are scored on a 3 point scoring system and 1 item is scored on a 2 point scoring system. The test is performed unilaterally using the limb preferred by the participant. The total score ranges from 0, if all the items cannot be performed, to 37, if all the activities are achieved fully without any compensation. ‘ Higher scores indicate higher levels of motor ability.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 4, Week 13, Week 26, Week 39 and Week 52
    End point values
    		 OAV101 1.1e14 vg/kg 8.5-13 kg OAV101 1.1e14 vg/kg >13-17 kg OAV101 1.1e14 vg/kg >17-21 kg OAV101 1.1e14 vg/kg 8.5-13 kg Overall
    Number of subjects analysed
    4
    6
    8
    18
    Units: RULM total score on a scale
    arithmetic mean (standard deviation)
        Change from baseline at Week 4
    2.8 ± 2.36
    1.2 ± 1.33
    1.0 ± 1.93
    1.4 ± 1.89
        Change from baseline at Week 13
    4.3 ± 1.50
    0.8 ± 2.32
    2.0 ± 2.56
    2.1 ± 2.52
        Change from baseline at Week 26
    5.3 ± 2.50
    1.3 ± 1.21
    1.6 ± 2.88
    2.3 ± 2.74
        Change from baseline at Week 39 (n=4,5,8,17)
    7.3 ± 2.22
    -0.6 ± 1.34
    2.0 ± 4.34
    2.5 ± 4.29
        Change from baseline at Week 52 (n=3,6,8,17)
    6.0 ± 3.46
    0.3 ± 1.63
    1.8 ± 4.71
    2.0 ± 4.02
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Adverse events are reported from the single dose of study treatment until end of study, up to maximum duration of 12 months
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    26.0
    Reporting groups
    Reporting group title
    OAV101 1.1e14 vg/kg 8.5 to 13 kg
    Reporting group description
    		 8.5 to 13 kg

    Reporting group title
    OAV101 1.1e14 vg/kg Greater than 13 to 17 kg
    Reporting group description
    		 Greater than 13 to 17 kg

    Reporting group title
    OAV101 1.1e14 vg/kg Greater than 17 to 21 kg
    Reporting group description
    		 Greater than 17 to 21 kg

    Reporting group title
    OAV101 1.1e14 vg/kg Overall
    Reporting group description
    		 Overall

    Serious adverse events
    		 OAV101 1.1e14 vg/kg 8.5 to 13 kg OAV101 1.1e14 vg/kg Greater than 13 to 17 kg OAV101 1.1e14 vg/kg Greater than 17 to 21 kg OAV101 1.1e14 vg/kg Overall
    Total subjects affected by serious adverse events
         subjects affected / exposed
    3 / 7 (42.86%)
    7 / 8 (87.50%)
    5 / 9 (55.56%)
    15 / 24 (62.50%)
         number of deaths (all causes)
    0
    0
    0
    0
         number of deaths resulting from adverse events
    0
    0
    0
    0
    Investigations
    Liver function test increased
         subjects affected / exposed
    1 / 7 (14.29%)
    0 / 8 (0.00%)
    0 / 9 (0.00%)
    1 / 24 (4.17%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 0
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Liver function test abnormal
         subjects affected / exposed
    0 / 7 (0.00%)
    1 / 8 (12.50%)
    0 / 9 (0.00%)
    1 / 24 (4.17%)
         occurrences causally related to treatment / all
    0 / 0
    1 / 1
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Injury, poisoning and procedural complications
    Radius fracture
         subjects affected / exposed
    0 / 7 (0.00%)
    0 / 8 (0.00%)
    1 / 9 (11.11%)
    1 / 24 (4.17%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Femur fracture
         subjects affected / exposed
    0 / 7 (0.00%)
    1 / 8 (12.50%)
    0 / 9 (0.00%)
    1 / 24 (4.17%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Blood and lymphatic system disorders
    Anaemia
         subjects affected / exposed
    0 / 7 (0.00%)
    1 / 8 (12.50%)
    0 / 9 (0.00%)
    1 / 24 (4.17%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Bicytopenia
         subjects affected / exposed
    0 / 7 (0.00%)
    1 / 8 (12.50%)
    0 / 9 (0.00%)
    1 / 24 (4.17%)
         occurrences causally related to treatment / all
    0 / 0
    1 / 1
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Thrombocytopenia
         subjects affected / exposed
    0 / 7 (0.00%)
    2 / 8 (25.00%)
    1 / 9 (11.11%)
    3 / 24 (12.50%)
         occurrences causally related to treatment / all
    0 / 0
    3 / 3
    1 / 1
    4 / 4
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    General disorders and administration site conditions
    Asthenia
         subjects affected / exposed
    0 / 7 (0.00%)
    0 / 8 (0.00%)
    1 / 9 (11.11%)
    1 / 24 (4.17%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Pyrexia
         subjects affected / exposed
    0 / 7 (0.00%)
    0 / 8 (0.00%)
    1 / 9 (11.11%)
    1 / 24 (4.17%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    1 / 1
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Gastrointestinal disorders
    Constipation
         subjects affected / exposed
    0 / 7 (0.00%)
    1 / 8 (12.50%)
    0 / 9 (0.00%)
    1 / 24 (4.17%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Vomiting
         subjects affected / exposed
    0 / 7 (0.00%)
    2 / 8 (25.00%)
    1 / 9 (11.11%)
    3 / 24 (12.50%)
         occurrences causally related to treatment / all
    0 / 0
    1 / 2
    1 / 2
    2 / 4
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Hepatobiliary disorders
    Hepatic cytolysis
         subjects affected / exposed
    0 / 7 (0.00%)
    1 / 8 (12.50%)
    0 / 9 (0.00%)
    1 / 24 (4.17%)
         occurrences causally related to treatment / all
    0 / 0
    1 / 1
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Hepatotoxicity
         subjects affected / exposed
    0 / 7 (0.00%)
    0 / 8 (0.00%)
    1 / 9 (11.11%)
    1 / 24 (4.17%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    1 / 1
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Hypertransaminasaemia
         subjects affected / exposed
    0 / 7 (0.00%)
    0 / 8 (0.00%)
    1 / 9 (11.11%)
    1 / 24 (4.17%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    1 / 2
    1 / 2
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Renal and urinary disorders
    Urinary retention
         subjects affected / exposed
    0 / 7 (0.00%)
    1 / 8 (12.50%)
    0 / 9 (0.00%)
    1 / 24 (4.17%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Musculoskeletal and connective tissue disorders
    Hip deformity
         subjects affected / exposed
    1 / 7 (14.29%)
    0 / 8 (0.00%)
    0 / 9 (0.00%)
    1 / 24 (4.17%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Infections and infestations
    COVID-19
         subjects affected / exposed
    0 / 7 (0.00%)
    0 / 8 (0.00%)
    2 / 9 (22.22%)
    2 / 24 (8.33%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 2
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    COVID-19 pneumonia
         subjects affected / exposed
    0 / 7 (0.00%)
    1 / 8 (12.50%)
    0 / 9 (0.00%)
    1 / 24 (4.17%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Gastroenteritis viral
         subjects affected / exposed
    1 / 7 (14.29%)
    1 / 8 (12.50%)
    0 / 9 (0.00%)
    2 / 24 (8.33%)
         occurrences causally related to treatment / all
    0 / 2
    0 / 1
    0 / 0
    0 / 3
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Pneumonia
         subjects affected / exposed
    0 / 7 (0.00%)
    0 / 8 (0.00%)
    2 / 9 (22.22%)
    2 / 24 (8.33%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 2
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Respiratory tract infection
         subjects affected / exposed
    1 / 7 (14.29%)
    0 / 8 (0.00%)
    0 / 9 (0.00%)
    1 / 24 (4.17%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Subglottic laryngitis
         subjects affected / exposed
    1 / 7 (14.29%)
    0 / 8 (0.00%)
    0 / 9 (0.00%)
    1 / 24 (4.17%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Varicella
         subjects affected / exposed
    0 / 7 (0.00%)
    1 / 8 (12.50%)
    0 / 9 (0.00%)
    1 / 24 (4.17%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Metabolism and nutrition disorders
    Dehydration
         subjects affected / exposed
    0 / 7 (0.00%)
    1 / 8 (12.50%)
    0 / 9 (0.00%)
    1 / 24 (4.17%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Hypoglycaemia
         subjects affected / exposed
    0 / 7 (0.00%)
    1 / 8 (12.50%)
    0 / 9 (0.00%)
    1 / 24 (4.17%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 2
    0 / 0
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 0%
    Non-serious adverse events
    		 OAV101 1.1e14 vg/kg 8.5 to 13 kg OAV101 1.1e14 vg/kg Greater than 13 to 17 kg OAV101 1.1e14 vg/kg Greater than 17 to 21 kg OAV101 1.1e14 vg/kg Overall
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    7 / 7 (100.00%)
    8 / 8 (100.00%)
    9 / 9 (100.00%)
    24 / 24 (100.00%)
    General disorders and administration site conditions
    Pyrexia
         subjects affected / exposed
    4 / 7 (57.14%)
    1 / 8 (12.50%)
    5 / 9 (55.56%)
    10 / 24 (41.67%)
         occurrences all number
    10
    3
    5
    18
    Malaise
         subjects affected / exposed
    0 / 7 (0.00%)
    0 / 8 (0.00%)
    1 / 9 (11.11%)
    1 / 24 (4.17%)
         occurrences all number
    0
    0
    1
    1
    Immune system disorders
    Hypersensitivity
         subjects affected / exposed
    1 / 7 (14.29%)
    0 / 8 (0.00%)
    0 / 9 (0.00%)
    1 / 24 (4.17%)
         occurrences all number
    1
    0
    0
    1
    Respiratory, thoracic and mediastinal disorders
    Cough
         subjects affected / exposed
    3 / 7 (42.86%)
    0 / 8 (0.00%)
    0 / 9 (0.00%)
    3 / 24 (12.50%)
         occurrences all number
    5
    0
    0
    5
    Epistaxis
         subjects affected / exposed
    0 / 7 (0.00%)
    1 / 8 (12.50%)
    0 / 9 (0.00%)
    1 / 24 (4.17%)
         occurrences all number
    0
    2
    0
    2
    Nasal congestion
         subjects affected / exposed
    0 / 7 (0.00%)
    1 / 8 (12.50%)
    0 / 9 (0.00%)
    1 / 24 (4.17%)
         occurrences all number
    0
    1
    0
    1
    Nocturnal dyspnoea
         subjects affected / exposed
    0 / 7 (0.00%)
    1 / 8 (12.50%)
    0 / 9 (0.00%)
    1 / 24 (4.17%)
         occurrences all number
    0
    1
    0
    1
    Oropharyngeal pain
         subjects affected / exposed
    1 / 7 (14.29%)
    0 / 8 (0.00%)
    0 / 9 (0.00%)
    1 / 24 (4.17%)
         occurrences all number
    1
    0
    0
    1
    Respiratory tract congestion
         subjects affected / exposed
    0 / 7 (0.00%)
    1 / 8 (12.50%)
    0 / 9 (0.00%)
    1 / 24 (4.17%)
         occurrences all number
    0
    1
    0
    1
    Sneezing
         subjects affected / exposed
    0 / 7 (0.00%)
    2 / 8 (25.00%)
    0 / 9 (0.00%)
    2 / 24 (8.33%)
         occurrences all number
    0
    2
    0
    2
    Upper respiratory tract congestion
         subjects affected / exposed
    0 / 7 (0.00%)
    0 / 8 (0.00%)
    1 / 9 (11.11%)
    1 / 24 (4.17%)
         occurrences all number
    0
    0
    5
    5
    Psychiatric disorders
    Hallucination
         subjects affected / exposed
    1 / 7 (14.29%)
    0 / 8 (0.00%)
    0 / 9 (0.00%)
    1 / 24 (4.17%)
         occurrences all number
    1
    0
    0
    1
    Insomnia
         subjects affected / exposed
    0 / 7 (0.00%)
    0 / 8 (0.00%)
    1 / 9 (11.11%)
    1 / 24 (4.17%)
         occurrences all number
    0
    0
    1
    1
    Irritability
         subjects affected / exposed
    0 / 7 (0.00%)
    0 / 8 (0.00%)
    2 / 9 (22.22%)
    2 / 24 (8.33%)
         occurrences all number
    0
    0
    2
    2
    Investigations
    Liver function test abnormal
         subjects affected / exposed
    0 / 7 (0.00%)
    1 / 8 (12.50%)
    0 / 9 (0.00%)
    1 / 24 (4.17%)
         occurrences all number
    0
    1
    0
    1
    Hepatic enzyme increased
         subjects affected / exposed
    0 / 7 (0.00%)
    2 / 8 (25.00%)
    0 / 9 (0.00%)
    2 / 24 (8.33%)
         occurrences all number
    0
    2
    0
    2
    Haemoglobin decreased
         subjects affected / exposed
    0 / 7 (0.00%)
    1 / 8 (12.50%)
    0 / 9 (0.00%)
    1 / 24 (4.17%)
         occurrences all number
    0
    1
    0
    1
    Electrocardiogram T wave inversion
         subjects affected / exposed
    0 / 7 (0.00%)
    1 / 8 (12.50%)
    0 / 9 (0.00%)
    1 / 24 (4.17%)
         occurrences all number
    0
    1
    0
    1
    Blood urea increased
         subjects affected / exposed
    1 / 7 (14.29%)
    0 / 8 (0.00%)
    0 / 9 (0.00%)
    1 / 24 (4.17%)
         occurrences all number
    1
    0
    0
    1
    Blood potassium abnormal
         subjects affected / exposed
    0 / 7 (0.00%)
    1 / 8 (12.50%)
    0 / 9 (0.00%)
    1 / 24 (4.17%)
         occurrences all number
    0
    1
    0
    1
    Blood lactate dehydrogenase increased
         subjects affected / exposed
    1 / 7 (14.29%)
    0 / 8 (0.00%)
    0 / 9 (0.00%)
    1 / 24 (4.17%)
         occurrences all number
    1
    0
    0
    1
    Aspartate aminotransferase increased
         subjects affected / exposed
    1 / 7 (14.29%)
    0 / 8 (0.00%)
    1 / 9 (11.11%)
    2 / 24 (8.33%)
         occurrences all number
    2
    0
    1
    3
    Alanine aminotransferase increased
         subjects affected / exposed
    1 / 7 (14.29%)
    0 / 8 (0.00%)
    1 / 9 (11.11%)
    2 / 24 (8.33%)
         occurrences all number
    2
    0
    1
    3
    Liver function test increased
         subjects affected / exposed
    1 / 7 (14.29%)
    0 / 8 (0.00%)
    1 / 9 (11.11%)
    2 / 24 (8.33%)
         occurrences all number
    1
    0
    1
    2
    Platelet count abnormal
         subjects affected / exposed
    0 / 7 (0.00%)
    0 / 8 (0.00%)
    1 / 9 (11.11%)
    1 / 24 (4.17%)
         occurrences all number
    0
    0
    1
    1
    Platelet count decreased
         subjects affected / exposed
    3 / 7 (42.86%)
    2 / 8 (25.00%)
    2 / 9 (22.22%)
    7 / 24 (29.17%)
         occurrences all number
    3
    2
    2
    7
    Prothrombin time prolonged
         subjects affected / exposed
    0 / 7 (0.00%)
    0 / 8 (0.00%)
    1 / 9 (11.11%)
    1 / 24 (4.17%)
         occurrences all number
    0
    0
    1
    1
    Transaminases increased
         subjects affected / exposed
    3 / 7 (42.86%)
    2 / 8 (25.00%)
    1 / 9 (11.11%)
    6 / 24 (25.00%)
         occurrences all number
    3
    3
    1
    7
    Weight increased
         subjects affected / exposed
    0 / 7 (0.00%)
    1 / 8 (12.50%)
    1 / 9 (11.11%)
    2 / 24 (8.33%)
         occurrences all number
    0
    1
    1
    2
    Injury, poisoning and procedural complications
    Fall
         subjects affected / exposed
    0 / 7 (0.00%)
    0 / 8 (0.00%)
    1 / 9 (11.11%)
    1 / 24 (4.17%)
         occurrences all number
    0
    0
    1
    1
    Joint dislocation
         subjects affected / exposed
    1 / 7 (14.29%)
    0 / 8 (0.00%)
    0 / 9 (0.00%)
    1 / 24 (4.17%)
         occurrences all number
    1
    0
    0
    1
    Procedural nausea
         subjects affected / exposed
    0 / 7 (0.00%)
    0 / 8 (0.00%)
    1 / 9 (11.11%)
    1 / 24 (4.17%)
         occurrences all number
    0
    0
    1
    1
    Procedural pain
         subjects affected / exposed
    0 / 7 (0.00%)
    1 / 8 (12.50%)
    0 / 9 (0.00%)
    1 / 24 (4.17%)
         occurrences all number
    0
    1
    0
    1
    Stoma site erythema
         subjects affected / exposed
    1 / 7 (14.29%)
    0 / 8 (0.00%)
    0 / 9 (0.00%)
    1 / 24 (4.17%)
         occurrences all number
    1
    0
    0
    1
    Torus fracture
         subjects affected / exposed
    1 / 7 (14.29%)
    0 / 8 (0.00%)
    0 / 9 (0.00%)
    1 / 24 (4.17%)
         occurrences all number
    1
    0
    0
    1
    Cardiac disorders
    Bradycardia
         subjects affected / exposed
    0 / 7 (0.00%)
    1 / 8 (12.50%)
    0 / 9 (0.00%)
    1 / 24 (4.17%)
         occurrences all number
    0
    1
    0
    1
    Pericardial effusion
         subjects affected / exposed
    1 / 7 (14.29%)
    0 / 8 (0.00%)
    0 / 9 (0.00%)
    1 / 24 (4.17%)
         occurrences all number
    1
    0
    0
    1
    Tachycardia
         subjects affected / exposed
    0 / 7 (0.00%)
    0 / 8 (0.00%)
    1 / 9 (11.11%)
    1 / 24 (4.17%)
         occurrences all number
    0
    0
    1
    1
    Nervous system disorders
    Dizziness
         subjects affected / exposed
    0 / 7 (0.00%)
    0 / 8 (0.00%)
    1 / 9 (11.11%)
    1 / 24 (4.17%)
         occurrences all number
    0
    0
    1
    1
    Headache
         subjects affected / exposed
    0 / 7 (0.00%)
    0 / 8 (0.00%)
    1 / 9 (11.11%)
    1 / 24 (4.17%)
         occurrences all number
    0
    0
    1
    1
    Lethargy
         subjects affected / exposed
    1 / 7 (14.29%)
    0 / 8 (0.00%)
    0 / 9 (0.00%)
    1 / 24 (4.17%)
         occurrences all number
    1
    0
    0
    1
    Tremor
         subjects affected / exposed
    0 / 7 (0.00%)
    2 / 8 (25.00%)
    0 / 9 (0.00%)
    2 / 24 (8.33%)
         occurrences all number
    0
    2
    0
    2
    Blood and lymphatic system disorders
    Anaemia
         subjects affected / exposed
    0 / 7 (0.00%)
    1 / 8 (12.50%)
    0 / 9 (0.00%)
    1 / 24 (4.17%)
         occurrences all number
    0
    1
    0
    1
    Thrombocytopenia
         subjects affected / exposed
    1 / 7 (14.29%)
    2 / 8 (25.00%)
    2 / 9 (22.22%)
    5 / 24 (20.83%)
         occurrences all number
    1
    2
    2
    5
    Eye disorders
    Periorbital swelling
         subjects affected / exposed
    1 / 7 (14.29%)
    0 / 8 (0.00%)
    0 / 9 (0.00%)
    1 / 24 (4.17%)
         occurrences all number
    1
    0
    0
    1
    Eye irritation
         subjects affected / exposed
    0 / 7 (0.00%)
    1 / 8 (12.50%)
    0 / 9 (0.00%)
    1 / 24 (4.17%)
         occurrences all number
    0
    1
    0
    1
    Blepharitis
         subjects affected / exposed
    0 / 7 (0.00%)
    0 / 8 (0.00%)
    1 / 9 (11.11%)
    1 / 24 (4.17%)
         occurrences all number
    0
    0
    1
    1
    Gastrointestinal disorders
    Leukoplakia oral
         subjects affected / exposed
    0 / 7 (0.00%)
    1 / 8 (12.50%)
    0 / 9 (0.00%)
    1 / 24 (4.17%)
         occurrences all number
    0
    1
    0
    1
    Gastrooesophageal reflux disease
         subjects affected / exposed
    0 / 7 (0.00%)
    1 / 8 (12.50%)
    0 / 9 (0.00%)
    1 / 24 (4.17%)
         occurrences all number
    0
    1
    0
    1
    Gastritis
         subjects affected / exposed
    1 / 7 (14.29%)
    0 / 8 (0.00%)
    0 / 9 (0.00%)
    1 / 24 (4.17%)
         occurrences all number
    1
    0
    0
    1
    Faecaloma
         subjects affected / exposed
    0 / 7 (0.00%)
    1 / 8 (12.50%)
    0 / 9 (0.00%)
    1 / 24 (4.17%)
         occurrences all number
    0
    1
    0
    1
    Nausea
         subjects affected / exposed
    3 / 7 (42.86%)
    2 / 8 (25.00%)
    3 / 9 (33.33%)
    8 / 24 (33.33%)
         occurrences all number
    3
    2
    3
    8
    Constipation
         subjects affected / exposed
    1 / 7 (14.29%)
    2 / 8 (25.00%)
    0 / 9 (0.00%)
    3 / 24 (12.50%)
         occurrences all number
    1
    4
    0
    5
    Abdominal pain upper
         subjects affected / exposed
    0 / 7 (0.00%)
    2 / 8 (25.00%)
    2 / 9 (22.22%)
    4 / 24 (16.67%)
         occurrences all number
    0
    3
    2
    5
    Abdominal pain
         subjects affected / exposed
    2 / 7 (28.57%)
    0 / 8 (0.00%)
    0 / 9 (0.00%)
    2 / 24 (8.33%)
         occurrences all number
    2
    0
    0
    2
    Diarrhoea
         subjects affected / exposed
    2 / 7 (28.57%)
    1 / 8 (12.50%)
    0 / 9 (0.00%)
    3 / 24 (12.50%)
         occurrences all number
    3
    1
    0
    4
    Vomiting
         subjects affected / exposed
    5 / 7 (71.43%)
    5 / 8 (62.50%)
    7 / 9 (77.78%)
    17 / 24 (70.83%)
         occurrences all number
    6
    9
    10
    25
    Upper gastrointestinal haemorrhage
         subjects affected / exposed
    1 / 7 (14.29%)
    0 / 8 (0.00%)
    0 / 9 (0.00%)
    1 / 24 (4.17%)
         occurrences all number
    1
    0
    0
    1
    Hepatobiliary disorders
    Hypertransaminasaemia
         subjects affected / exposed
    1 / 7 (14.29%)
    1 / 8 (12.50%)
    5 / 9 (55.56%)
    7 / 24 (29.17%)
         occurrences all number
    1
    3
    7
    11
    Skin and subcutaneous tissue disorders
    Dermatitis
         subjects affected / exposed
    0 / 7 (0.00%)
    0 / 8 (0.00%)
    1 / 9 (11.11%)
    1 / 24 (4.17%)
         occurrences all number
    0
    0
    1
    1
    Eczema infantile
         subjects affected / exposed
    1 / 7 (14.29%)
    0 / 8 (0.00%)
    0 / 9 (0.00%)
    1 / 24 (4.17%)
         occurrences all number
    1
    0
    0
    1
    Hypertrichosis
         subjects affected / exposed
    0 / 7 (0.00%)
    1 / 8 (12.50%)
    0 / 9 (0.00%)
    1 / 24 (4.17%)
         occurrences all number
    0
    1
    0
    1
    Rash
         subjects affected / exposed
    1 / 7 (14.29%)
    1 / 8 (12.50%)
    0 / 9 (0.00%)
    2 / 24 (8.33%)
         occurrences all number
    1
    1
    0
    2
    Rash erythematous
         subjects affected / exposed
    0 / 7 (0.00%)
    0 / 8 (0.00%)
    1 / 9 (11.11%)
    1 / 24 (4.17%)
         occurrences all number
    0
    0
    1
    1
    Renal and urinary disorders
    Pollakiuria
         subjects affected / exposed
    0 / 7 (0.00%)
    1 / 8 (12.50%)
    0 / 9 (0.00%)
    1 / 24 (4.17%)
         occurrences all number
    0
    1
    0
    1
    Endocrine disorders
    Cushingoid
         subjects affected / exposed
    0 / 7 (0.00%)
    1 / 8 (12.50%)
    1 / 9 (11.11%)
    2 / 24 (8.33%)
         occurrences all number
    0
    1
    1
    2
    Cushing's syndrome
         subjects affected / exposed
    1 / 7 (14.29%)
    0 / 8 (0.00%)
    2 / 9 (22.22%)
    3 / 24 (12.50%)
         occurrences all number
    1
    0
    2
    3
    Musculoskeletal and connective tissue disorders
    Arthralgia
         subjects affected / exposed
    0 / 7 (0.00%)
    0 / 8 (0.00%)
    3 / 9 (33.33%)
    3 / 24 (12.50%)
         occurrences all number
    0
    0
    3
    3
    Back pain
         subjects affected / exposed
    0 / 7 (0.00%)
    1 / 8 (12.50%)
    1 / 9 (11.11%)
    2 / 24 (8.33%)
         occurrences all number
    0
    1
    1
    2
    Muscular weakness
         subjects affected / exposed
    0 / 7 (0.00%)
    1 / 8 (12.50%)
    0 / 9 (0.00%)
    1 / 24 (4.17%)
         occurrences all number
    0
    2
    0
    2
    Osteopenia
         subjects affected / exposed
    1 / 7 (14.29%)
    0 / 8 (0.00%)
    0 / 9 (0.00%)
    1 / 24 (4.17%)
         occurrences all number
    1
    0
    0
    1
    Pain in extremity
         subjects affected / exposed
    0 / 7 (0.00%)
    0 / 8 (0.00%)
    3 / 9 (33.33%)
    3 / 24 (12.50%)
         occurrences all number
    0
    0
    4
    4
    Infections and infestations
    COVID-19
         subjects affected / exposed
    1 / 7 (14.29%)
    4 / 8 (50.00%)
    2 / 9 (22.22%)
    7 / 24 (29.17%)
         occurrences all number
    1
    4
    2
    7
    Conjunctivitis
         subjects affected / exposed
    1 / 7 (14.29%)
    1 / 8 (12.50%)
    0 / 9 (0.00%)
    2 / 24 (8.33%)
         occurrences all number
    1
    1
    0
    2
    Upper respiratory tract infection
         subjects affected / exposed
    4 / 7 (57.14%)
    2 / 8 (25.00%)
    2 / 9 (22.22%)
    8 / 24 (33.33%)
         occurrences all number
    5
    2
    4
    11
    Rhinitis
         subjects affected / exposed
    0 / 7 (0.00%)
    1 / 8 (12.50%)
    0 / 9 (0.00%)
    1 / 24 (4.17%)
         occurrences all number
    0
    1
    0
    1
    Respiratory tract infection
         subjects affected / exposed
    0 / 7 (0.00%)
    0 / 8 (0.00%)
    1 / 9 (11.11%)
    1 / 24 (4.17%)
         occurrences all number
    0
    0
    1
    1
    Pneumonia
         subjects affected / exposed
    0 / 7 (0.00%)
    1 / 8 (12.50%)
    0 / 9 (0.00%)
    1 / 24 (4.17%)
         occurrences all number
    0
    1
    0
    1
    Otitis media
         subjects affected / exposed
    0 / 7 (0.00%)
    0 / 8 (0.00%)
    1 / 9 (11.11%)
    1 / 24 (4.17%)
         occurrences all number
    0
    0
    1
    1
    Nasopharyngitis
         subjects affected / exposed
    2 / 7 (28.57%)
    2 / 8 (25.00%)
    1 / 9 (11.11%)
    5 / 24 (20.83%)
         occurrences all number
    2
    2
    1
    5
    Influenza
         subjects affected / exposed
    0 / 7 (0.00%)
    1 / 8 (12.50%)
    0 / 9 (0.00%)
    1 / 24 (4.17%)
         occurrences all number
    0
    1
    0
    1
    Infectious mononucleosis
         subjects affected / exposed
    0 / 7 (0.00%)
    1 / 8 (12.50%)
    0 / 9 (0.00%)
    1 / 24 (4.17%)
         occurrences all number
    0
    1
    0
    1
    Gastroenteritis
         subjects affected / exposed
    1 / 7 (14.29%)
    0 / 8 (0.00%)
    2 / 9 (22.22%)
    3 / 24 (12.50%)
         occurrences all number
    1
    0
    4
    5
    Urinary tract infection
         subjects affected / exposed
    1 / 7 (14.29%)
    0 / 8 (0.00%)
    0 / 9 (0.00%)
    1 / 24 (4.17%)
         occurrences all number
    1
    0
    0
    1
    Viral skin infection
         subjects affected / exposed
    1 / 7 (14.29%)
    0 / 8 (0.00%)
    0 / 9 (0.00%)
    1 / 24 (4.17%)
         occurrences all number
    1
    0
    0
    1
    Metabolism and nutrition disorders
    Dehydration
         subjects affected / exposed
    1 / 7 (14.29%)
    0 / 8 (0.00%)
    0 / 9 (0.00%)
    1 / 24 (4.17%)
         occurrences all number
    1
    0
    0
    1
    Decreased appetite
         subjects affected / exposed
    1 / 7 (14.29%)
    0 / 8 (0.00%)
    1 / 9 (11.11%)
    2 / 24 (8.33%)
         occurrences all number
    1
    0
    1
    2
    Vitamin D deficiency
         subjects affected / exposed
    0 / 7 (0.00%)
    1 / 8 (12.50%)
    0 / 9 (0.00%)
    1 / 24 (4.17%)
         occurrences all number
    0
    1
    0
    1

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    28 Feb 2022
    The main purpose of the amendment was to remove virus serology form time points past screening in the Schedule of Assessments. Other changes included adding complement panel, optional pharmacogenetics and ACEND assessment as exploratory endpoints. In addition, protocol was edited for consistency and clarification purposes.

    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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