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    Clinical Trial Results:
    A Pilot Study of the Efficacy, Safety, and Tolerability of AX-8 for the Treatment of Refractory Chronic Cough

    Summary
    EudraCT number
    		 2017-003108-27
    Trial protocol
    		 GB  
    Global end of trial date
    11 Jun 2018

    Results information
    Results version number
    		 v1(current)
    This version publication date
    27 Jun 2019
    First version publication date
    27 Jun 2019
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    AX8-001
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 -
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Axalbion SA
    Sponsor organisation address
    EPFL-Innovation Park, Bâtiment C, Lausanne, Switzerland, CH-1015
    Public contact
    Olivier Poirot, Axalbion SA, +41 76 341 81 38, opoirot@axalbion.com
    Scientific contact
    Olivier Poirot, Axalbion SA, +41 76 341 81 38, opoirot@axalbion.com
    Paediatric regulatory details
    Is trial part of an agreed paediatric investigation plan (PIP)
    No
    Does article 45 of REGULATION (EC) No 1901/2006 apply to this trial?
    No
    Does article 46 of REGULATION (EC) No 1901/2006 apply to this trial?
    No
    Results analysis stage
    Analysis stage
    Final
    Date of interim/final analysis
    11 Jun 2018
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    11 Jun 2018
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    To assess the effectiveness of AX-8 for the treatment of refractory chronic cough (RCC) and associated upper airway symptoms after one dose of treatment in reducing awake cough frequency compared to baseline, for the purpose of planning a future randomised controlled trial.
    Protection of trial subjects
    The study was performed in accordance with the current version of the declaration of Helsinki (52nd WMA General Assembly, Edinburgh, Scotland, October 2000). The study was conducted in agreement with the International Conference on Harmonisation (ICH) guidelines on Good Clinical Practice (GCP). All subjects provided written informed consent to participate in the study prior to being screened.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    12 Dec 2017
    Long term follow-up planned
    No
    Independent data monitoring committee (IDMC) involvement?
    No
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    United Kingdom: 12
    Worldwide total number of subjects
    12
    EEA total number of subjects
    12
    Number of subjects enrolled per age group
    In utero
    0
    Preterm newborn - gestational age < 37 wk
    0
    Newborns (0-27 days)
    0
    Infants and toddlers (28 days-23 months)
    0
    Children (2-11 years)
    0
    Adolescents (12-17 years)
    0
    Adults (18-64 years)
    7
    From 65 to 84 years
    5
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 12 subjects were enrolled in the United Kingdom.

    Pre-assignment
    Screening details
    		 16 subjects were screened for the study and 12 received study drug.

    Period 1
    Period 1 title
    Screening (Visit 1)
    Is this the baseline period?
    		 No
    Allocation method
    Not applicable
    Blinding used
    		 Not blinded

    Arms
    Arm title
    		 AX-8 orally disintegrating tablet (ODT) 5 mg
    Arm description
    		 Subjects did not receive treatment during the screening period.
    Arm type
    		 Experimental

    Investigational medicinal product name
    AX-8 orally disintegrating tablet (ODT) 5 mg
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Subjects did not receive treatment during the screening period.

    Number of subjects in period 1
    		AX-8 orally disintegrating tablet (ODT) 5 mg
    Started
    12
    Completed
    12
    Period 2
    Period 2 title
    Baseline (Visit 2)
    Is this the baseline period?
    		 Yes [1]
    Allocation method
    Not applicable
    Blinding used
    		 Not blinded

    Arms
    Arm title
    		 AX-8 orally disintegrating tablet (ODT) 5 mg
    Arm description
    		 Subjects did not receive treatment during the baseline period.
    Arm type
    		 Experimental

    Investigational medicinal product name
    AX-8 orally disintegrating tablet (ODT) 5 mg
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Subjects did not receive treatment during the baseline period.

    Notes
    [1] - Period 1 is not the baseline period. It is expected that period 1 will be the baseline period.
    Justification: Period 1 was the screening period.
    Number of subjects in period 2
    		AX-8 orally disintegrating tablet (ODT) 5 mg
    Started
    12
    Completed
    12
    Period 3
    Period 3 title
    Treatment (Visits 3 and 4)
    Is this the baseline period?
    		 No
    Allocation method
    Not applicable
    Blinding used
    		 Not blinded

    Arms
    Arm title
    		 AX-8 orally disintegrating tablet (ODT) 5 mg
    Arm description
    		 Subjects received a single dose of AX-8 orally disintegrating tablet (ODT) 5 mg by mouth.
    Arm type
    		 Experimental

    Investigational medicinal product name
    AX-8 orally disintegrating tablet (ODT) 5 mg
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Subjects received a single dose of AX-8 orally disintegrating tablet (ODT) 5 mg by mouth.

    Number of subjects in period 3
    		AX-8 orally disintegrating tablet (ODT) 5 mg
    Started
    12
    Completed
    12
    Period 4
    Period 4 title
    Follow-up (Visit 5/Withdraw/Study End)
    Is this the baseline period?
    		 No
    Allocation method
    Not applicable
    Blinding used
    		 Not blinded

    Arms
    Arm title
    		 AX-8 orally disintegrating tablet (ODT) 5 mg
    Arm description
    		 Subjects did not receive treatment during the follow-up period.
    Arm type
    		 Experimental

    Investigational medicinal product name
    AX-8 orally disintegrating tablet (ODT) 5 mg
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Subjects did not receive treatment during the follow-up period.

    Number of subjects in period 4
    		AX-8 orally disintegrating tablet (ODT) 5 mg
    Started
    12
    Completed
    11
    Not completed
    1
         Adverse event, non-fatal
    1

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Baseline (Visit 2)
    Reporting group description
    		 -

    Reporting group values
    		 Baseline (Visit 2) Total
    Number of subjects
    		 12 12
    Age categorical
    Units: Subjects
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    		 63.9 ± 10.9 -
    Gender categorical
    Units: Subjects
        Female
    		 9 9
        Male
    		 3 3

    End points

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    End points reporting groups
    Reporting group title
    AX-8 orally disintegrating tablet (ODT) 5 mg
    Reporting group description
    		 Subjects did not receive treatment during the screening period.
    Reporting group title
    AX-8 orally disintegrating tablet (ODT) 5 mg
    Reporting group description
    		 Subjects did not receive treatment during the baseline period.
    Reporting group title
    AX-8 orally disintegrating tablet (ODT) 5 mg
    Reporting group description
    		 Subjects received a single dose of AX-8 orally disintegrating tablet (ODT) 5 mg by mouth.
    Reporting group title
    AX-8 orally disintegrating tablet (ODT) 5 mg
    Reporting group description
    		 Subjects did not receive treatment during the follow-up period.

    Primary: Change from Baseline in Objective Awake Cough Frequency Over 24 hours after 1 Dose of Treatment

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    End point title
    		 Change from Baseline in Objective Awake Cough Frequency Over 24 hours after 1 Dose of Treatment [1]
    End point description
    The change in awake and asleep cough rates was estimated from audio recordings and calculated by taking the total number of cough events during the monitoring period while the subject was awake and dividing by the total duration (in hours) for the monitoring period the subject was awake. Any session with a recording duration < 4 hours was considered as missing. The full analysis set included all enrolled subjects who took at least one dose of study medication and provided at least one baseline and at least one post-baseline primary endpoint observation during the treatment period.
    End point type
    Primary
    End point timeframe
    Baseline and 24 hours post-dose
    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: Descriptive statistics are presented for this single-arm analysis.
    End point values
    		 AX-8 orally disintegrating tablet (ODT) 5 mg
    Number of subjects analysed
    12
    Units: cough per hour
    median (inter-quartile range (Q1-Q3))
        Baseline
    64.1 (27.4 to 94.4)
        Post-treatment
    54.8 (16.4 to 79.5)
    No statistical analyses for this end point

    Secondary: Change from Baseline in Hourly Objective Cough Frequency Over 24 Hours After 1 Dose of Treatment

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    End point title
    		 Change from Baseline in Hourly Objective Cough Frequency Over 24 Hours After 1 Dose of Treatment
    End point description
    The change in hourly cough rates was estimated from audio recordings and calculated by taking the total number of cough events during the monitoring period (24 hours) and dividing by the total duration (in hours) for the monitoring period (24). Any session with a recording duration < 4 hours was considered as missing. The full analysis set included all enrolled subjects who took at least one dose of study medication and provided at least one baseline and at least one post-baseline primary endpoint observation during the treatment period.
    End point type
    Secondary
    End point timeframe
    Comparison of periods of 4, 8, and 24 hours after the installation of the cough monitor (baseline visit) with periods of 4, 8, and 24 hours after dosing (treatment visit)
    End point values
    		 AX-8 orally disintegrating tablet (ODT) 5 mg
    Number of subjects analysed
    12
    Units: percent change
    number (not applicable)
        Changes over 4 hours post-dose
    -42.0
        Changes over 8 hours post-dose
    -36.9
        Changes over 24 hours post-dose
    -15.3
    No statistical analyses for this end point

    Secondary: Percentage of Subjects with ≥ 30% Reduction in Awake Cough Frequency per Hour

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    End point title
    		 Percentage of Subjects with ≥ 30% Reduction in Awake Cough Frequency per Hour
    End point description
    The percentage of subjects with ≥ 30% of reduction from baseline in awake cough frequency is the number of subjects with ≤ -30% change in awake cough frequency divided by the total number of subjects with available data. The full analysis set included all enrolled subjects who took at least one dose of study medication and provided at least one baseline and at least one post-baseline primary endpoint observation during the treatment period.
    End point type
    Secondary
    End point timeframe
    24 hours post-dose
    End point values
    		 AX-8 orally disintegrating tablet (ODT) 5 mg
    Number of subjects analysed
    12
    Units: percentage of subjects
        number (not applicable)
    33.3
    No statistical analyses for this end point

    Secondary: Percentage of Subjects with ≥ 30% Reduction in Cough Frequency per Hour

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    End point title
    		 Percentage of Subjects with ≥ 30% Reduction in Cough Frequency per Hour
    End point description
    The percentage of subjects with ≥ 30% reduction from baseline in 24-cough frequency is the number of subjects with ≤ -30% change in cough frequency divided by the total number of subjects with available data. The full analysis set included all enrolled subjects who took at least one dose of study medication and provided at least one baseline and at least one post-baseline primary endpoint observation during the treatment period.
    End point type
    Secondary
    End point timeframe
    Periods of 4, 8, and 24 hours post-dose
    End point values
    		 AX-8 orally disintegrating tablet (ODT) 5 mg
    Number of subjects analysed
    12
    Units: percentage of subjects
    number (not applicable)
        Changes over 4 hours post-dose
    41.7
        Changes over 8 hours post-dose
    58.3
        Changes over 24 hours post-dose
    33.3
    No statistical analyses for this end point

    Secondary: Change from Baseline in Cough Severity Visual Analogue Scale (VAS)

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    End point title
    		 Change from Baseline in Cough Severity Visual Analogue Scale (VAS)
    End point description
    Subjects rated cough severity 30 mins before cough monitor installation or dosing, on baseline and treatment days, respectively, and then hourly for 6 hours using a 100-mm visual analogue scale (VAS). The VAS for the first 4 hours were recorded in the clinic and the last 2 hours were recorded at home using a patient diary. The subject's impression at a time point during the baseline visit was compared to the overall impression during more than a 24-hour period. Therefore, the value >24h is an overall impression at a specific time point. The full analysis set included all enrolled subjects who took at least one dose of study medication and provided at least one baseline and at least one post-baseline primary endpoint observation during the treatment period.
    End point type
    Secondary
    End point timeframe
    Baseline (Day 0) and up to ≥24 hours post-dose (Day 1)
    End point values
    		 AX-8 orally disintegrating tablet (ODT) 5 mg
    Number of subjects analysed
    12
    Units: mm
    median (full range (min-max))
        30 min before cough monitor installation (n=12)
    65.0 (53 to 95)
        1 hr after cough monitor installation (n=12)
    65.5 (14 to 86)
        2 hr after cough monitor installation (n=12)
    65.0 (8 to 91)
        3 hr after cough monitor installation (n=12)
    64.0 (5 to 85)
        4 hr after cough monitor installation (n=12)
    62.0 (2 to 82)
        5 hr after cough monitor installation (n=12)
    59.5 (13 to 88)
        6 hr after cough monitor installation (n=12)
    60.0 (11 to 92)
        30 min pre-dose (n=12)
    66.0 (18 to 87)
        1 hr post-dose (n=12)
    48.0 (2 to 81)
        2 hr post-dose (n=12)
    49.0 (2 to 86)
        3 hr post-dose (n=12)
    44.5 (2 to 85)
        4 hr post-dose (n=12)
    42.5 (2 to 91)
        5 hr post-dose (n=11)
    47.0 (2 to 88)
        6 hr post-dose (n=11)
    47.0 (1 to 86)
        Overall period ≥24 hr post-dose (n=12)
    47.0 (6 to 96)
    No statistical analyses for this end point

    Secondary: Change from Baseline in Urge-to-Cough VAS

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    End point title
    		 Change from Baseline in Urge-to-Cough VAS
    End point description
    Subjects rated urge-to-cough 30 mins before cough monitor installation or dosing, on baseline and treatment days, respectively, and then hourly for 6 hours using a 100-mm visual analogue scale (VAS). The VAS for the first 4 hours were recorded in the clinic and the last 2 hours were recorded at home using a patient diary. The subject's impression at a time point during the baseline visit was compared to the overall impression during more than a 24-hour period. Therefore, the value >24h is an overall impression at a specific time point. The full analysis set included all enrolled subjects who took at least one dose of study medication and provided at least one baseline and at least one post-baseline primary endpoint observation during the treatment period.
    End point type
    Secondary
    End point timeframe
    Baseline (Day 0) and up to ≥24 hours post-dose (Day 1)
    End point values
    		 AX-8 orally disintegrating tablet (ODT) 5 mg
    Number of subjects analysed
    12
    Units: mm
    median (full range (min-max))
        30 min before cough monitor installation (n=12)
    62.0 (14 to 93)
        1 hr after cough monitor installation (n=12)
    57.0 (11 to 88)
        2 hr after cough monitor installation (n=12)
    51.0 (3 to 92)
        3 hr after cough monitor installation (n=12)
    49.0 (4 to 86)
        4 hr after cough monitor installation (n=12)
    52.5 (2 to 90)
        5 hr after cough monitor installation (n=12)
    49.0 (2 to 83)
        6 hr after cough monitor installation (n=12)
    55.5 (4 to 90)
        30 min pre-dose (n=12)
    62.5 (7 to 90)
        1 hr post-dose (n=12)
    48.0 (1 to 92)
        2 hr post-dose (n=12)
    43.0 (2 to 87)
        3 hr post-dose (n=12)
    26.0 (2 to 85)
        4 hr post-dose (n=12)
    34.0 (1 to 92)
        5 hr post-dose (n=11)
    36.0 (1 to 83)
        6 hr post-dose (n=11)
    41.0 (1 to 84)
        Overall period ≥24 hr post-dose (n=12)
    46.0 (3 to 95)
    No statistical analyses for this end point

    Secondary: Change from Baseline in Throat Irritation VAS

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    End point title
    		 Change from Baseline in Throat Irritation VAS
    End point description
    Subjects rated cough severity 30 mins before cough monitor installation or dosing, on baseline and treatment days, respectively, and then hourly for 6 hours using a 100-mm visual analogue scale (VAS). The VAS for the first 4 hours were recorded in the clinic and the last 2 hours were recorded at home using a patient diary. The subject's impression at a time point during the baseline visit was compared to the overall impression during more than a 24-hour period. Therefore, the value >24h is an overall impression at a specific time point. The full analysis set included all enrolled subjects who took at least one dose of study medication and provided at least one baseline and at least one post-baseline primary endpoint observation during the treatment period.
    End point type
    Secondary
    End point timeframe
    Baseline (Day 0) and up to ≥24 hours post-dose (Day 1)
    End point values
    		 AX-8 orally disintegrating tablet (ODT) 5 mg
    Number of subjects analysed
    12
    Units: mm
    median (full range (min-max))
        30 min before cough monitor installation (n=12)
    56.5 (1 to 92)
        1 hr after cough monitor installation (n=12)
    42.0 (1 to 86)
        2 hr after cough monitor installation (n=12)
    39.0 (0 to 91)
        3 hr after cough monitor installation (n=12)
    32.5 (0 to 90)
        4 hr after cough monitor installation (n=12)
    36.0 (0 to 89)
        5 hr after cough monitor installation (n=12)
    30.0 (0 to 90)
        6 hr after cough monitor installation (n=12)
    35.0 (0 to 91)
        30 min pre-dose (n=12)
    40.5 (0 to 83)
        1 hr post-dose (n=12)
    22.5 (1 to 86)
        2 hr post-dose (n=12)
    7.5 (0 to 71)
        3 hr post-dose (n=12)
    5.0 (0 to 78)
        4 hr post-dose (n=12)
    5.5 (0 to 77)
        5 hr post-dose (n=11)
    7.0 (2 to 76)
        6 hr post-dose (n=11)
    6.0 (1 to 79)
        Overall period ≥24 hr post-dose (n=12)
    9.0 (1 to 97)
    No statistical analyses for this end point

    Secondary: Throat Cooling VAS

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    End point title
    		 Throat Cooling VAS
    End point description
    Subjects rated cough severity 30 mins before cough monitor installation or dosing, on baseline and treatment days, respectively, and then hourly for 6 hours using a 100-mm visual analogue scale (VAS). The VAS for the first 4 hours were recorded in the clinic and the last 2 hours were recorded at home using a patient diary. The subject's impression at a time point during the baseline visit was compared to the overall impression during more than a 24-hour period. Therefore, the value >24h is an overall impression at a specific time point. The full analysis set included all enrolled subjects who took at least one dose of study medication and provided at least one baseline and at least one post-baseline primary endpoint observation during the treatment period.
    End point type
    Secondary
    End point timeframe
    Up to ≥24 hours post-dose (Day 1)
    End point values
    		 AX-8 orally disintegrating tablet (ODT) 5 mg
    Number of subjects analysed
    12
    Units: mm
    median (full range (min-max))
        30 min pre-dose (n=10)
    1.5 (0 to 20)
        30 min post-dose (n=12)
    19.0 (2 to 80)
        1 hr post-dose (n=12)
    3.5 (1 to 67)
        1.5 hr post-dose (n=12)
    2.0 (0 to 63)
        2 hr post-dose (n=12)
    2.0 (0 to 56)
        2.5 hr post-dose (n=12)
    3.5 (0 to 65)
        3 hr post-dose (n=12)
    2.0 (0 to 42)
        4 hr post-dose (n=12)
    1.5 (0 to 14)
        5 hr post-dose (n=11)
    2.0 (0 to 45)
        6 hr post-dose (n=11)
    2.0 (0 to 48)
        Overall period ≥24 hr post-dose (n=12)
    3.0 (0 to 20)
    No statistical analyses for this end point

    Secondary: Global Rating of Change Scale (GRCS) - Cough Frequency

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    End point title
    		 Global Rating of Change Scale (GRCS) - Cough Frequency
    End point description
    Subjects assessed overall status of cough frequency since dosing using the GRCS instrument, with 3 categories and a 14-point scale range: better (1-7), about the same, and worse (8-14). GRCS were measured 4h post-dose on Treatment Day 1 and 24h post-dose on Treatment Day 2. The full analysis set included all enrolled subjects who took at least one dose of study medication and provided at least one baseline and at least one post-baseline primary endpoint observation during the treatment period. pd=post-dose.
    End point type
    Secondary
    End point timeframe
    4h post-dose on Treatment Day 1 and 24h post-dose on Treatment Day 2
    End point values
    		 AX-8 orally disintegrating tablet (ODT) 5 mg
    Number of subjects analysed
    12
    Units: Subjects
        4h pd: better (n=12)
    4
        4h pd: about the same (n=12)
    7
        4h pd: worse (n=12)
    1
        24h pd: better (n=11)
    4
        24h pd: about the same (n=11)
    6
        24h pd: worse (n=11)
    1
    No statistical analyses for this end point

    Secondary: Global Rating of Change Scale (GRCS) - Cough Severity

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    End point title
    		 Global Rating of Change Scale (GRCS) - Cough Severity
    End point description
    Subjects assessed overall status of cough severity since dosing using the GRCS instrument, with 3 categories and a 14-point scale range: better (1-7), about the same, and worse (8-14). GRCS were measured 4h post-dose on Treatment Day 1 and 24h post-dose on Treatment Day 2. The full analysis set included all enrolled subjects who took at least one dose of study medication and provided at least one baseline and at least one post-baseline primary endpoint observation during the treatment period. pd=post-dose.
    End point type
    Secondary
    End point timeframe
    4h post-dose on Treatment Day 1 and 24h post-dose on Treatment Day 2
    End point values
    		 AX-8 orally disintegrating tablet (ODT) 5 mg
    Number of subjects analysed
    12
    Units: Subjects
        4h pd: better (n=12)
    5
        4h pd: about the same (n=12)
    6
        4h pd: worse (n=12)
    1
        24h pd: better (n=11)
    4
        24h pd: about the same (n=11)
    6
        24h pd: worse (n=11)
    1
    No statistical analyses for this end point

    Secondary: Percentage of Subjects with Treatment-Emergent Adverse Events (TEAEs)

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    End point title
    		 Percentage of Subjects with Treatment-Emergent Adverse Events (TEAEs)
    End point description
    TEAEs were defined as AEs with a date of onset on or after first study medication intake. The incidence of TEAEs was classified by system organ class and preferred term using the Medical Dictionary for Regulatory Activities (MedDRA). For incidence reporting, if a subject reported more than one AE that was coded to the same system organ class or preferred term, the subject was counted only once for that specific system organ class or preferred term. The safety analysis set consisted of all enrolled subjects who received at least one dose of study medication.
    End point type
    Secondary
    End point timeframe
    Approximately 30 days
    End point values
    		 AX-8 orally disintegrating tablet (ODT) 5 mg
    Number of subjects analysed
    12
    Units: percentage of subjects
        number (not applicable)
    58.33
    No statistical analyses for this end point

    Secondary: Percentage of Subjects with Serious Adverse Events (SAEs)

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    End point title
    		 Percentage of Subjects with Serious Adverse Events (SAEs)
    End point description
    SAEs were defined as AEs that resulted in death, threat to life, hospitalization, persistent or significant incapacity, congenital anomaly/birth defect, or important medical event that was considered serious by the investigator or Sponsor or would require medical/surgical intervention to prevent any of the prior outcomes. The safety analysis set consisted of all enrolled subjects who received at least one dose of study medication.
    End point type
    Secondary
    End point timeframe
    Approximately 30 days
    End point values
    		 AX-8 orally disintegrating tablet (ODT) 5 mg
    Number of subjects analysed
    12
    Units: percentage of subjects
        number (not applicable)
    0
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Approximately 30 days
    Adverse event reporting additional description
    The safety analysis set consisted of all enrolled subjects who received at least one dose of study medication.
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    20.0
    Reporting groups
    Reporting group title
    AX-8
    Reporting group description
    		 Subjects received a single dose of AX-8 orally disintegrating tablet (ODT) 5 mg by mouth.

    Serious adverse events
    		 AX-8
    Total subjects affected by serious adverse events
         subjects affected / exposed
    0 / 12 (0.00%)
         number of deaths (all causes)
    0
         number of deaths resulting from adverse events
    0
    Frequency threshold for reporting non-serious adverse events: 0%
    Non-serious adverse events
    		 AX-8
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    7 / 12 (58.33%)
    Injury, poisoning and procedural complications
    Bruising
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    1
    Nervous system disorders
    Headache
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    1
    Twitching eyelid
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    1
    General disorders and administration site conditions
    Fatigue
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    1
    Tiredness
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    1
    Gastrointestinal disorders
    Worsening acid reflux
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    1
    Nausea
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    1
    Paraesthesia oral
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    1
    Taste disturbance
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    1
    Respiratory, thoracic and mediastinal disorders
    Sore throat
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    1
    Musculoskeletal and connective tissue disorders
    Chronic back pain
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    1
    Infections and infestations
    Lower respiratory tract infection
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    1
    Upper respiratory tract infection
         subjects affected / exposed
    2 / 12 (16.67%)
         occurrences all number
    2
    Aches and fever
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    1

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    26 Jan 2018
    The following substantial changes have been made to the protocol: Blood sampling for pharmacokinetic (PK) analysis was added (+15, +30 mins, +1, +1.25, +1.5, +1.75, +2, +2.5, +3, +3.5 hours). PK sample will be taken anytime pre-dose on Day 1, post-dose + 15, +30, +45 minutes, +1, +1.25, +1.5, +1.75, +2, +2.5, +3, +3.5, +4 hours (i.e., 1hr, 1hr15 mins, 1hr30mins, 1hr45mins, 2hr, 2hr30mins, 3hr, 3hr30mins, 4hr). PK analysis changed from being a secondary endpoint to being an exploratory endpoint. Synopsys updated: Study Objectives completed and section “Exploratory Endpoint” added. Section 2 – Study Objectives. New paragraph “Exploratory Objectives” added. New section 3.3.4 – Exploratory Endpoint. The patient information sheet (PIS) has been updated in line with these protocol changes.

    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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