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    Clinical Trial Results:
    A Phase 1/2 Open-label, Multiple-dose, Dose-escalating Clinical Trial of the Safety and Tolerability of GTX-102 in Pediatric Patients With Angelman Syndrome (AS)

    Summary
    EudraCT number
    		 2021-001793-36
    Trial protocol
    		 DE   ES   FR  
    Global end of trial date
    08 Jan 2025

    Results information
    Results version number
    		 v1(current)
    This version publication date
    23 Jan 2026
    First version publication date
    23 Jan 2026
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    GTX-102-001
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT04259281
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Ultragenyx Pharmaceutical Inc.
    Sponsor organisation address
    60 Leveroni Court, Novato, United States, 94949
    Public contact
    Trial Recruitment, Ultragenyx Pharmaceutical Inc., +1 8887568657, trialrecruitment@ultragenyx.com
    Scientific contact
    Medical Information, Ultragenyx Pharmaceutical Inc., +1 8887568657, medinfo@ultragenyx.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
    08 Jan 2025
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    08 Jan 2025
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    To evaluate the safety and tolerability of multiple-ascending doses of GTX-102 administered by intrathecal (IT) injection to patients with AS.
    Protection of trial subjects
    All participants, parents and/or legal guardians of the participant will be provided with an Information Sheet and/or Consent Form describing this study and providing sufficient information to make an informed decision about the participation of their represented participant in this study. Where required, consent will be sought from the parents and/or legal guardian of the participant. The Informed Consent Forms (ICFs) and Patient Informed Consent Forms (PICFs) will include all elements required by ICH, Good Clinical Practice (GCP) and applicable regulatory requirements. The Information Sheet and/or Consent Form will be submitted with the protocol for review and approval by the relevant Ethical Review Board for the study in each country. The formal consent of a participant, using the Informed Consent Form approved by the relevant Ethical Review Board, must be obtained before that participant undergoes any study procedure. The consent form must be signed by the participant’s parent/legal guardian, and the Investigator-designated research professional obtaining the consent. Where required by the relevant Ethical Review Board, a witness may sign the consent form.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    24 Feb 2020
    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
    Spain: 5
    Country: Number of subjects enrolled
    France: 7
    Country: Number of subjects enrolled
    Germany: 4
    Country: Number of subjects enrolled
    Australia: 5
    Country: Number of subjects enrolled
    Canada: 20
    Country: Number of subjects enrolled
    United Kingdom: 7
    Country: Number of subjects enrolled
    Israel: 2
    Country: Number of subjects enrolled
    United States: 24
    Worldwide total number of subjects
    74
    EEA total number of subjects
    16
    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)
    60
    Adolescents (12-17 years)
    14
    Adults (18-64 years)
    0
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

    		 Close Top of page
    Recruitment
    Recruitment details
    		 -

    Pre-assignment
    Screening details
    		 A total of 74 participants were enrolled sequentially into 13 cohorts. After a dosing pause, participants enrolled in Cohorts 1 & 2 restarted in a redosing cohort. The Cohort 3 participant continued to be followed in an expanded access trial.

    Period 1
    Period 1 title
    Original Dosing Period
    Is this the baseline period?
    		 No
    Allocation method
    Non-randomised - controlled
    Blinding used
    		 Not blinded

    Arms
    Arm title
    		 GTX-102 Cohorts 1-3
    Arm description
    		 Participants received doses ranging from 3.3 to 20 mg followed by dose escalation up to a maximum of 36 mg.
    Arm type
    		 Experimental

    Investigational medicinal product name
    GTX-102
    Investigational medicinal product code
    GTX-102
    Other name
    apazunursen
    Pharmaceutical forms
    Solution for injection
    Routes of administration
    Intrathecal use
    Dosage and administration details
    GTX-102 is given through an intrathecal injection via lumbar puncture.

    Number of subjects in period 1
    		GTX-102 Cohorts 1-3
    Started
    5
    Completed
    0
    Not completed
    5
         Adverse event, non-fatal
    5
    Period 2
    Period 2 title
    Amended Dosing Period
    Is this the baseline period?
    		 Yes [1]
    Allocation method
    Non-randomised - controlled
    Blinding used
    		 Not blinded

    Arms
    Are arms mutually exclusive
    No

    Arm title
    		 GTX-102 US Redosing Cohorts 1-2
    Arm description
    		 Participants from Cohorts 1-3 were restarted at doses ranging from 5 to 10 mg followed by dose escalation up to a maximum dose of 14 mg Q3M.
    Arm type
    		 Experimental

    Investigational medicinal product name
    GTX-102
    Investigational medicinal product code
    GTX-102
    Other name
    apazunursen
    Pharmaceutical forms
    Solution for injection
    Routes of administration
    Intrathecal use
    Dosage and administration details
    GTX-102 is given through an intrathecal injection via lumbar puncture.

    Arm title
    		 GTX-102 Cohorts 4-7
    Arm description
    		 Participants received up to 4 loading doses ranging from 3.3 to 10 mg followed by dose escalation up to a maximum dose of 14 mg Q3M.
    Arm type
    		 Experimental

    Investigational medicinal product name
    GTX-102
    Investigational medicinal product code
    GTX-102
    Other name
    apazunursen
    Pharmaceutical forms
    Solution for injection
    Routes of administration
    Intrathecal use
    Dosage and administration details
    GTX-102 is given through an intrathecal injection via lumbar puncture.

    Arm title
    		 GTX-102 Cohorts A&B
    Arm description
    		 Participants received up to 4 loading doses of 7.5 mg followed by dose escalation up to a maximum dose of 14 mg Q3M.
    Arm type
    		 Experimental

    Investigational medicinal product name
    GTX-102
    Investigational medicinal product code
    GTX-102
    Other name
    apazunursen
    Pharmaceutical forms
    Solution for injection
    Routes of administration
    Intrathecal use
    Dosage and administration details
    GTX-102 is given through an intrathecal injection via lumbar puncture.

    Arm title
    		 GTX-102 Cohorts C&D
    Arm description
    		 Participants received up to 4 loading doses ranging from 5 to 7.5 mg followed by dose escalation up to a maximum dose of 10 mg Q3M.
    Arm type
    		 Experimental

    Investigational medicinal product name
    GTX-102
    Investigational medicinal product code
    GTX-102
    Other name
    apazunursen
    Pharmaceutical forms
    Solution for injection
    Routes of administration
    Intrathecal use
    Dosage and administration details
    GTX-102 is given through an intrathecal injection via lumbar puncture.

    Arm title
    		 GTX-102 US 2 mg/Cohort E
    Arm description
    		 Participants received either no treatment or GTX-102. Participants treated with GTX-102 received 4 monthly loading doses of 2 mg, followed by 2 mg Q3M. Participants who received no treatment during the initial period then received 4 monthly doses of 2 mg, followed by 2 mg Q3M. All participants then transitioned to Cohort E regardless of prior GTX-102 treatment duration and received up to 4 loading doses of 5 to 7.5 mg, followed by 7.5 mg every 2 months (Q2M).
    Arm type
    		 Experimental

    Investigational medicinal product name
    GTX-102
    Investigational medicinal product code
    GTX-102
    Other name
    apazunursen
    Pharmaceutical forms
    Solution for injection
    Routes of administration
    Intrathecal use
    Dosage and administration details
    GTX-102 is given through an intrathecal injection via lumbar puncture.

    Notes
    [1] - Period 1 is not the baseline period. It is expected that period 1 will be the baseline period.
    Justification: Period 2 is defined as the baseline.
    Number of subjects in period 2
    		GTX-102 US Redosing Cohorts 1-2 GTX-102 Cohorts 4-7 GTX-102 Cohorts A&B GTX-102 Cohorts C&D GTX-102 US 2 mg/Cohort E
    Started
    4
    15
    34
    12
    8
    Completed
    4
    13
    31
    12
    7
    Not completed
    0
    2
    3
    0
    1
         Consent withdrawn by subject
    -
    1
    1
    -
    1
         Adverse event, non-fatal
    -
    1
    1
    -
    -
         Parental Decision
    -
    -
    1
    -
    -

    Baseline characteristics

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    Baseline characteristics reporting groups [1]
    Reporting group title
    GTX-102 US Redosing Cohorts 1-2
    Reporting group description
    		 Participants from Cohorts 1-3 were restarted at doses ranging from 5 to 10 mg followed by dose escalation up to a maximum dose of 14 mg Q3M.

    Reporting group title
    GTX-102 Cohorts 4-7
    Reporting group description
    		 Participants received up to 4 loading doses ranging from 3.3 to 10 mg followed by dose escalation up to a maximum dose of 14 mg Q3M.

    Reporting group title
    GTX-102 Cohorts A&B
    Reporting group description
    		 Participants received up to 4 loading doses of 7.5 mg followed by dose escalation up to a maximum dose of 14 mg Q3M.

    Reporting group title
    GTX-102 Cohorts C&D
    Reporting group description
    		 Participants received up to 4 loading doses ranging from 5 to 7.5 mg followed by dose escalation up to a maximum dose of 10 mg Q3M.

    Reporting group title
    GTX-102 US 2 mg/Cohort E
    Reporting group description
    		 Participants received either no treatment or GTX-102. Participants treated with GTX-102 received 4 monthly loading doses of 2 mg, followed by 2 mg Q3M. Participants who received no treatment during the initial period then received 4 monthly doses of 2 mg, followed by 2 mg Q3M. All participants then transitioned to Cohort E regardless of prior GTX-102 treatment duration and received up to 4 loading doses of 5 to 7.5 mg, followed by 7.5 mg every 2 months (Q2M).

    Notes
    [1] - The number of subjects reported to be in the baseline period is not equal to the worldwide number of subjects enrolled in the trial. It is expected that these numbers will be the same.
    Justification: A total of 74 subjects were enrolled worldwide across 2 periods.
    Reporting group values
    		 GTX-102 US Redosing Cohorts 1-2 GTX-102 Cohorts 4-7 GTX-102 Cohorts A&B GTX-102 Cohorts C&D GTX-102 US 2 mg/Cohort E Total
    Number of subjects
    		 4 15 34 12 8 73
    Age categorical
    Units: Subjects
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    		 13.98 ( 4.4 ) 8.4 ( 3.9 ) 7.8 ( 3.8 ) 8.8 ( 4.3 ) 5.9 ( 1.2 ) -
    Gender categorical
    Units: Subjects
        Female
    		 2 8 21 9 5 45
        Male
    		 2 7 13 3 3 28
    Race
    Units: Subjects
        Not Allowed per Local Regulation
    		 0 0 7 0 0 7
        American Indian or Alaska Native
    		 0 0 0 0 0 0
        Asian
    		 0 2 1 1 1 5
        Black or African American
    		 0 0 1 0 0 1
        Native Hawaiian or Other Pacific Islander
    		 0 0 1 0 0 1
        White
    		 4 7 23 11 7 48
        Other
    		 0 6 1 0 0 7
    Subject analysis sets

    Subject analysis set title
    GTX-102 Cohorts 1-3
    Subject analysis set type
    		 Per protocol
    Subject analysis set description
    Participants received doses of 3.3 mg to 20 mg of GTX-102 which were escalated up to a maximum maintenance dose of 36 mg.

    Subject analysis sets values
    		 GTX-102 Cohorts 1-3
    Number of subjects
    5
    Age categorical
    Units: Subjects
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    9.4 ( 4.4 )
    Gender categorical
    Units: Subjects
        Female
    2
        Male
    3
    Race
    Units: Subjects
        Not Allowed per Local Regulation
    0
        American Indian or Alaska Native
    0
        Asian
    0
        Black or African American
    0
        Native Hawaiian or Other Pacific Islander
    0
        White
    5
        Other
    0

    End points

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    End points reporting groups
    Reporting group title
    GTX-102 Cohorts 1-3
    Reporting group description
    		 Participants received doses ranging from 3.3 to 20 mg followed by dose escalation up to a maximum of 36 mg.
    Reporting group title
    GTX-102 US Redosing Cohorts 1-2
    Reporting group description
    		 Participants from Cohorts 1-3 were restarted at doses ranging from 5 to 10 mg followed by dose escalation up to a maximum dose of 14 mg Q3M.

    Reporting group title
    GTX-102 Cohorts 4-7
    Reporting group description
    		 Participants received up to 4 loading doses ranging from 3.3 to 10 mg followed by dose escalation up to a maximum dose of 14 mg Q3M.

    Reporting group title
    GTX-102 Cohorts A&B
    Reporting group description
    		 Participants received up to 4 loading doses of 7.5 mg followed by dose escalation up to a maximum dose of 14 mg Q3M.

    Reporting group title
    GTX-102 Cohorts C&D
    Reporting group description
    		 Participants received up to 4 loading doses ranging from 5 to 7.5 mg followed by dose escalation up to a maximum dose of 10 mg Q3M.

    Reporting group title
    GTX-102 US 2 mg/Cohort E
    Reporting group description
    		 Participants received either no treatment or GTX-102. Participants treated with GTX-102 received 4 monthly loading doses of 2 mg, followed by 2 mg Q3M. Participants who received no treatment during the initial period then received 4 monthly doses of 2 mg, followed by 2 mg Q3M. All participants then transitioned to Cohort E regardless of prior GTX-102 treatment duration and received up to 4 loading doses of 5 to 7.5 mg, followed by 7.5 mg every 2 months (Q2M).

    Subject analysis set title
    GTX-102 Cohorts 1-3
    Subject analysis set type
    		 Per protocol
    Subject analysis set description
    Participants received doses of 3.3 mg to 20 mg of GTX-102 which were escalated up to a maximum maintenance dose of 36 mg.

    Primary: Number of Participants with Treatment-emergent Adverse Events (TEAEs), Serious TEAEs (SAEs), Adverse Events of Special Interest (AESIs), TEAEs Leading to Discontinuation and TEAEs by Severity

    		 Close Top of page
    End point title
    		 Number of Participants with Treatment-emergent Adverse Events (TEAEs), Serious TEAEs (SAEs), Adverse Events of Special Interest (AESIs), TEAEs Leading to Discontinuation and TEAEs by Severity [1]
    End point description
    An AE is defined as any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug related. A TEAE is defined as any AE not present prior to the initiation of the drug treatment or any AE already present that worsens in either intensity or frequency following exposure to the drug treatment. An SAE is an AE that meets any of the following criteria in the view of either the Investigator or Ultragenyx: death; life-threatening; inpatient hospitalization or prolongation of existing hospitalization; disability/Incapacity; congenital anomaly/birth defect not present at screening; other important medical events. Severity of events were graded as mild (grade1), moderate (grade 2), severe (grade 3), life-threatening (grade 4), or death (grade 5). An AESI is an event of scientific or medical interest specific to the drug treatment requiring further investigation and close monitoring to further characterize them.
    End point type
    Primary
    End point timeframe
    From the first dose of study drug through the last dose of study drug plus 42 (±14) days, for an overall mean study duration of 604.1 days.
    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 per protocol.
    End point values
    		 GTX-102 Cohorts 1-3 GTX-102 US Redosing Cohorts 1-2 GTX-102 Cohorts 4-7 GTX-102 Cohorts A&B GTX-102 Cohorts C&D GTX-102 US 2 mg/Cohort E
    Number of subjects analysed
    5
    4
    15
    34
    12
    8
    Units: number of participants
        Treatment-Emergent Adverse Event (TEAE)
    5
    2
    15
    34
    12
    8
        Related TEAE
    5
    0
    4
    17
    4
    1
        Serious TEAE
    5
    0
    6
    10
    4
    5
        Serious Related TEAE
    5
    0
    1
    2
    0
    0
        Grade 3 or 4 Related TEAE
    1
    0
    1
    2
    0
    0
        TEAE with Maximum Severity Grade 5
    0
    0
    0
    0
    0
    0
        TEAE with Maximum Severity Grade 4
    0
    0
    0
    1
    0
    0
        TEAE with Maximum Severity Grade 3
    1
    0
    3
    3
    1
    1
        TEAE with Maximum Severity Grade 2
    4
    1
    9
    24
    8
    6
        TEAE with Maximum Severity Grade 1
    0
    1
    3
    6
    3
    1
        Adverse Event of Special Interest
    5
    0
    1
    2
    0
    0
        TEAE Leading to Dose Reduced
    0
    0
    0
    4
    0
    0
        TEAE Leading to Dose Interruption
    1
    0
    3
    2
    2
    1
        TEAE Leading to Dose Withdrawn
    0
    0
    1
    2
    0
    0
        TEAE Leading to Study Discontinuation
    0
    0
    1
    1
    0
    0
        TEAE Leading to Death
    0
    0
    0
    0
    0
    0
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    From signing of informed consent (SAEs) or first dose of study drug (non-serious AEs) through the last dose of study drug plus 42 (±14) days, for an overall mean study duration of 604.1 days.
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    27.1
    Reporting groups
    Reporting group title
    GTX-102 Cohorts 1 - 3
    Reporting group description
    		 Participants received doses ranging from 3.3 to 20 mg followed by dose escalation up to a maximum of 36 mg.

    Reporting group title
    GTX-102 US Redosing Cohorts 1 - 2
    Reporting group description
    		 Participants from Cohorts 1-3 were restarted at doses ranging from 5 to 10 mg followed by dose escalation up to a maximum dose of 14 mg Q3M.

    Reporting group title
    GTX-102 US 2mg/Cohort E
    Reporting group description
    		 Participants received either no treatment or GTX-102. Participants treated with GTX-102 received 4 monthly loading doses of 2 mg, followed by 2 mg Q3M. Participants who received no treatment during the initial period then received 4 monthly doses of 2 mg, followed by 2 mg Q3M. All participants then transitioned to Cohort E regardless of prior GTX-102 treatment duration and received up to 4 loading doses of 5 to 7.5 mg, followed by 7.5 mg every 2 months (Q2M).

    Reporting group title
    GTX-102 Cohorts A & B
    Reporting group description
    		 Participants received up to 4 loading doses of 7.5 mg followed by dose escalation up to a maximum dose of 14 mg Q3M.

    Reporting group title
    GTX-102 Cohorts C & D
    Reporting group description
    		 Participants received up to 4 loading doses ranging from 5 to 7.5 mg followed by dose escalation up to a maximum dose of 10 mg Q3M.

    Reporting group title
    GTX-102 Cohorts 4 - 7
    Reporting group description
    		 Participants received up to 4 loading doses ranging from 3.3 to 10 mg followed by dose escalation up to a maximum dose of 14 mg Q3M.

    Serious adverse events
    		 GTX-102 Cohorts 1 - 3 GTX-102 US Redosing Cohorts 1 - 2 GTX-102 US 2mg/Cohort E GTX-102 Cohorts A & B GTX-102 Cohorts C & D GTX-102 Cohorts 4 - 7
    Total subjects affected by serious adverse events
         subjects affected / exposed
    5 / 5 (100.00%)
    0 / 4 (0.00%)
    5 / 8 (62.50%)
    10 / 34 (29.41%)
    4 / 12 (33.33%)
    6 / 15 (40.00%)
         number of deaths (all causes)
    0
    0
    0
    0
    0
    0
         number of deaths resulting from adverse events
    0
    0
    0
    0
    0
    0
    Injury, poisoning and procedural complications
    Humerus Fracture
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    1 / 34 (2.94%)
    0 / 12 (0.00%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Post Lumbar Puncture Syndrome
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    1 / 8 (12.50%)
    3 / 34 (8.82%)
    0 / 12 (0.00%)
    2 / 15 (13.33%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
    0 / 3
    0 / 0
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Skin Laceration
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    1 / 34 (2.94%)
    0 / 12 (0.00%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Wound Decomposition
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    0 / 34 (0.00%)
    0 / 12 (0.00%)
    1 / 15 (6.67%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Nervous system disorders
    Epilepsy
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    1 / 34 (2.94%)
    0 / 12 (0.00%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Febrile Convulsion
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    1 / 8 (12.50%)
    0 / 34 (0.00%)
    0 / 12 (0.00%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Radiculopathy
         subjects affected / exposed
    5 / 5 (100.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    1 / 34 (2.94%)
    0 / 12 (0.00%)
    1 / 15 (6.67%)
         occurrences causally related to treatment / all
    5 / 5
    0 / 0
    0 / 0
    1 / 1
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Seizure
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    1 / 8 (12.50%)
    1 / 34 (2.94%)
    0 / 12 (0.00%)
    1 / 15 (6.67%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
    0 / 1
    0 / 0
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Seizure Cluster
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    1 / 8 (12.50%)
    0 / 34 (0.00%)
    0 / 12 (0.00%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Status Epilepticus
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    1 / 8 (12.50%)
    0 / 34 (0.00%)
    0 / 12 (0.00%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Gastrointestinal disorders
    Vomiting
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    0 / 34 (0.00%)
    1 / 12 (8.33%)
    1 / 15 (6.67%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 1
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Pancreatitis Acute
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    0 / 34 (0.00%)
    1 / 12 (8.33%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Pancreatitis
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    0 / 34 (0.00%)
    1 / 12 (8.33%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Intestinal Obstruction
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    0 / 34 (0.00%)
    0 / 12 (0.00%)
    1 / 15 (6.67%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Faecaloma
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    0 / 34 (0.00%)
    1 / 12 (8.33%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Constipation
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    0 / 34 (0.00%)
    1 / 12 (8.33%)
    1 / 15 (6.67%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 1
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Respiratory, thoracic and mediastinal disorders
    Acute Respiratory Failure
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    1 / 8 (12.50%)
    0 / 34 (0.00%)
    0 / 12 (0.00%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Choking
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    1 / 34 (2.94%)
    0 / 12 (0.00%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Musculoskeletal and connective tissue disorders
    Muscular Weakness
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    1 / 34 (2.94%)
    0 / 12 (0.00%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    1 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Infections and infestations
    Streptococcal Infection
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    1 / 8 (12.50%)
    0 / 34 (0.00%)
    0 / 12 (0.00%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Pneumonia
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    0 / 34 (0.00%)
    0 / 12 (0.00%)
    2 / 15 (13.33%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Medical Device Site Infection
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    0 / 34 (0.00%)
    0 / 12 (0.00%)
    1 / 15 (6.67%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Herpes Zoster Meningitis
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    0 / 34 (0.00%)
    0 / 12 (0.00%)
    1 / 15 (6.67%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Gastroenteritis Viral
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    0 / 34 (0.00%)
    0 / 12 (0.00%)
    1 / 15 (6.67%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Metabolism and nutrition disorders
    Hypophagia
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    0 / 34 (0.00%)
    0 / 12 (0.00%)
    1 / 15 (6.67%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 GTX-102 Cohorts 1 - 3 GTX-102 US Redosing Cohorts 1 - 2 GTX-102 US 2mg/Cohort E GTX-102 Cohorts A & B GTX-102 Cohorts C & D GTX-102 Cohorts 4 - 7
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    5 / 5 (100.00%)
    2 / 4 (50.00%)
    8 / 8 (100.00%)
    33 / 34 (97.06%)
    12 / 12 (100.00%)
    15 / 15 (100.00%)
    Vascular disorders
    Hypotension
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    3 / 34 (8.82%)
    0 / 12 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    0
    5
    0
    2
    General disorders and administration site conditions
    Asthenia
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    4 / 34 (11.76%)
    1 / 12 (8.33%)
    0 / 15 (0.00%)
         occurrences all number
    0
    0
    0
    5
    2
    0
    Decreased Activity
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    0 / 34 (0.00%)
    0 / 12 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    Decreased Gait Velocity
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    0 / 34 (0.00%)
    0 / 12 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    Fatigue
         subjects affected / exposed
    2 / 5 (40.00%)
    1 / 4 (25.00%)
    1 / 8 (12.50%)
    5 / 34 (14.71%)
    1 / 12 (8.33%)
    3 / 15 (20.00%)
         occurrences all number
    3
    1
    1
    6
    1
    6
    Gait Disturbance
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    2 / 8 (25.00%)
    1 / 34 (2.94%)
    1 / 12 (8.33%)
    0 / 15 (0.00%)
         occurrences all number
    0
    0
    2
    2
    1
    0
    Influenza Like Illness
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    3 / 34 (8.82%)
    0 / 12 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    0
    5
    0
    1
    Pain
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    0 / 34 (0.00%)
    0 / 12 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    Pyrexia
         subjects affected / exposed
    1 / 5 (20.00%)
    1 / 4 (25.00%)
    3 / 8 (37.50%)
    7 / 34 (20.59%)
    1 / 12 (8.33%)
    4 / 15 (26.67%)
         occurrences all number
    1
    1
    3
    10
    1
    6
    Malaise
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    0 / 34 (0.00%)
    0 / 12 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    0
    0
    0
    2
    Immune system disorders
    Hypersensitivity
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    2 / 34 (5.88%)
    0 / 12 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    0
    0
    0
    3
    0
    0
    Reproductive system and breast disorders
    Bartholin's Cyst
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    0 / 34 (0.00%)
    0 / 12 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    Dysmenorrhoea
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    1 / 34 (2.94%)
    0 / 12 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    0
    1
    0
    1
    Respiratory, thoracic and mediastinal disorders
    Adenoidal Hypertrophy
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    0 / 34 (0.00%)
    0 / 12 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    Dyspnoea
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    1 / 34 (2.94%)
    0 / 12 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    0
    1
    0
    1
    Epistaxis
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    0 / 34 (0.00%)
    0 / 12 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    0
    0
    0
    2
    Nasal Congestion
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 4 (25.00%)
    0 / 8 (0.00%)
    0 / 34 (0.00%)
    0 / 12 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    1
    0
    0
    0
    1
    Rhinorrhoea
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    2 / 34 (5.88%)
    0 / 12 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    0
    0
    0
    2
    0
    0
    Tonsillar Hypertrophy
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    1 / 34 (2.94%)
    0 / 12 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    0
    1
    0
    1
    Cough
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    2 / 34 (5.88%)
    0 / 12 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    0
    2
    0
    1
    Psychiatric disorders
    Agitation
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    0 / 34 (0.00%)
    0 / 12 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    0
    0
    0
    2
    Abnormal Behaviour
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    1 / 34 (2.94%)
    2 / 12 (16.67%)
    2 / 15 (13.33%)
         occurrences all number
    0
    0
    0
    1
    2
    3
    Aggression
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    1 / 34 (2.94%)
    1 / 12 (8.33%)
    0 / 15 (0.00%)
         occurrences all number
    0
    0
    0
    1
    1
    0
    Insomnia
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    1 / 8 (12.50%)
    2 / 34 (5.88%)
    0 / 12 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    0
    0
    1
    2
    0
    0
    Irritability
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    1 / 34 (2.94%)
    1 / 12 (8.33%)
    0 / 15 (0.00%)
         occurrences all number
    0
    0
    0
    1
    1
    0
    Investigations
    Blood Alkaline Phosphatase Increased
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    0 / 34 (0.00%)
    0 / 12 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    Blood Pressure Diastolic Decreased
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    0 / 34 (0.00%)
    0 / 12 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    Body Temperature Increased
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    3 / 34 (8.82%)
    0 / 12 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    0
    0
    0
    5
    0
    0
    Csf Immunoglobulin Increased
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    2 / 34 (5.88%)
    0 / 12 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    0
    0
    0
    3
    0
    0
    Csf Pressure Increased
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    1 / 8 (12.50%)
    0 / 34 (0.00%)
    0 / 12 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    0
    Csf Protein Increased
         subjects affected / exposed
    3 / 5 (60.00%)
    0 / 4 (0.00%)
    2 / 8 (25.00%)
    6 / 34 (17.65%)
    2 / 12 (16.67%)
    4 / 15 (26.67%)
         occurrences all number
    3
    0
    2
    7
    2
    5
    Monocyte Count Increased
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    0 / 34 (0.00%)
    0 / 12 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    Csf White Blood Cell Count Increased
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    1 / 34 (2.94%)
    1 / 12 (8.33%)
    0 / 15 (0.00%)
         occurrences all number
    0
    0
    0
    1
    1
    0
    Culture Urine Positive
         subjects affected / exposed
    1 / 5 (20.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    0 / 34 (0.00%)
    0 / 12 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    1
    0
    0
    0
    0
    0
    Glucose Urine Present
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    0 / 34 (0.00%)
    1 / 12 (8.33%)
    0 / 15 (0.00%)
         occurrences all number
    0
    0
    0
    0
    1
    0
    International Normalised Ratio Increased
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    0 / 34 (0.00%)
    0 / 12 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    Csf Red Blood Cell Count Positive
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    0 / 34 (0.00%)
    1 / 12 (8.33%)
    0 / 15 (0.00%)
         occurrences all number
    0
    0
    0
    0
    1
    0
    Neutrophil Count Decreased
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    0 / 34 (0.00%)
    0 / 12 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    Vitamin D Decreased
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    0 / 34 (0.00%)
    0 / 12 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    Weight Decreased
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    0 / 34 (0.00%)
    0 / 12 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    Weight Increased
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    2 / 34 (5.88%)
    0 / 12 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    0
    0
    0
    2
    0
    0
    White Blood Cell Count Increased
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    0 / 34 (0.00%)
    0 / 12 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    Injury, poisoning and procedural complications
    Head Injury
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    0 / 34 (0.00%)
    0 / 12 (0.00%)
    2 / 15 (13.33%)
         occurrences all number
    0
    0
    0
    0
    0
    2
    Accidental Exposure To Product
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    1 / 8 (12.50%)
    0 / 34 (0.00%)
    0 / 12 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    0
    Arthropod Bite
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    2 / 8 (25.00%)
    0 / 34 (0.00%)
    0 / 12 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    0
    0
    2
    0
    0
    0
    Contusion
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    2 / 34 (5.88%)
    1 / 12 (8.33%)
    0 / 15 (0.00%)
         occurrences all number
    0
    0
    0
    3
    1
    0
    Eye Contusion
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    0 / 34 (0.00%)
    1 / 12 (8.33%)
    0 / 15 (0.00%)
         occurrences all number
    0
    0
    0
    0
    1
    0
    Fall
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    1 / 8 (12.50%)
    0 / 34 (0.00%)
    0 / 12 (0.00%)
    4 / 15 (26.67%)
         occurrences all number
    0
    0
    1
    0
    0
    6
    Foreign Body In Respiratory Tract
         subjects affected / exposed
    1 / 5 (20.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    0 / 34 (0.00%)
    0 / 12 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    1
    0
    0
    0
    0
    0
    Skin Laceration
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    1 / 34 (2.94%)
    1 / 12 (8.33%)
    0 / 15 (0.00%)
         occurrences all number
    0
    0
    0
    1
    1
    0
    Soft Tissue Injury
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    1 / 34 (2.94%)
    0 / 12 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    0
    1
    0
    1
    Thermal Burn
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    0 / 34 (0.00%)
    0 / 12 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    Tooth Fracture
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    0 / 34 (0.00%)
    0 / 12 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    Wrist Fracture
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    0 / 34 (0.00%)
    1 / 12 (8.33%)
    0 / 15 (0.00%)
         occurrences all number
    0
    0
    0
    0
    1
    0
    Joint Injury
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    1 / 34 (2.94%)
    0 / 12 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    0
    1
    0
    2
    Ligament Sprain
         subjects affected / exposed
    1 / 5 (20.00%)
    1 / 4 (25.00%)
    0 / 8 (0.00%)
    1 / 34 (2.94%)
    0 / 12 (0.00%)
    3 / 15 (20.00%)
         occurrences all number
    1
    1
    0
    1
    0
    3
    Limb Injury
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    1 / 8 (12.50%)
    0 / 34 (0.00%)
    0 / 12 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    1
    0
    0
    1
    Lip Injury
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    1 / 34 (2.94%)
    1 / 12 (8.33%)
    0 / 15 (0.00%)
         occurrences all number
    0
    0
    0
    1
    1
    0
    Post Lumbar Puncture Syndrome
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    7 / 34 (20.59%)
    2 / 12 (16.67%)
    2 / 15 (13.33%)
         occurrences all number
    0
    0
    0
    7
    2
    3
    Procedural Pain
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    1 / 34 (2.94%)
    0 / 12 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    0
    2
    0
    1
    Cardiac disorders
    Tachycardia
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    2 / 34 (5.88%)
    0 / 12 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    0
    0
    0
    4
    0
    0
    Nervous system disorders
    Ataxia
         subjects affected / exposed
    5 / 5 (100.00%)
    0 / 4 (0.00%)
    3 / 8 (37.50%)
    4 / 34 (11.76%)
    0 / 12 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    11
    0
    4
    4
    0
    0
    Atonic Seizures
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    6 / 34 (17.65%)
    0 / 12 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    0
    0
    0
    7
    0
    0
    Balance Disorder
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    1 / 34 (2.94%)
    1 / 12 (8.33%)
    0 / 15 (0.00%)
         occurrences all number
    0
    0
    0
    1
    1
    0
    Migraine
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    0 / 34 (0.00%)
    1 / 12 (8.33%)
    0 / 15 (0.00%)
         occurrences all number
    0
    0
    0
    0
    1
    0
    Drooling
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    1 / 34 (2.94%)
    0 / 12 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    0
    1
    0
    1
    Epilepsy
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    4 / 34 (11.76%)
    0 / 12 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    0
    6
    0
    1
    Headache
         subjects affected / exposed
    2 / 5 (40.00%)
    1 / 4 (25.00%)
    1 / 8 (12.50%)
    8 / 34 (23.53%)
    1 / 12 (8.33%)
    3 / 15 (20.00%)
         occurrences all number
    3
    1
    1
    11
    1
    9
    Hyperreflexia
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    1 / 8 (12.50%)
    0 / 34 (0.00%)
    0 / 12 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    0
    0
    2
    0
    0
    0
    Hypotonia
         subjects affected / exposed
    1 / 5 (20.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    0 / 34 (0.00%)
    0 / 12 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    1
    0
    0
    0
    0
    0
    Lethargy
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    0 / 34 (0.00%)
    1 / 12 (8.33%)
    0 / 15 (0.00%)
         occurrences all number
    0
    0
    0
    0
    1
    0
    Disturbance In Attention
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    2 / 34 (5.88%)
    0 / 12 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    0
    0
    0
    2
    0
    0
    Myoclonus
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    3 / 34 (8.82%)
    0 / 12 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    0
    0
    0
    7
    0
    0
    Petit Mal Epilepsy
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    3 / 34 (8.82%)
    0 / 12 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    0
    0
    0
    6
    0
    0
    Post-Traumatic Epilepsy
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    0 / 34 (0.00%)
    0 / 12 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    Radicular Pain
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    0 / 34 (0.00%)
    0 / 12 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    Radiculopathy
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    2 / 34 (5.88%)
    0 / 12 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    0
    0
    0
    2
    0
    0
    Seizure
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    2 / 8 (25.00%)
    12 / 34 (35.29%)
    1 / 12 (8.33%)
    2 / 15 (13.33%)
         occurrences all number
    0
    0
    2
    15
    1
    5
    Sensory Disturbance
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    0 / 34 (0.00%)
    0 / 12 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    Somnolence
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    3 / 34 (8.82%)
    1 / 12 (8.33%)
    0 / 15 (0.00%)
         occurrences all number
    0
    0
    0
    3
    1
    0
    Syncope
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    0 / 34 (0.00%)
    0 / 12 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    Seizure Like Phenomena
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    1 / 8 (12.50%)
    0 / 34 (0.00%)
    0 / 12 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    0
    Blood and lymphatic system disorders
    Anaemia
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    0 / 34 (0.00%)
    0 / 12 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    Lymphocytosis
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    0 / 34 (0.00%)
    0 / 12 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    Ear and labyrinth disorders
    Ear Discomfort
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    0 / 34 (0.00%)
    0 / 12 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    Ear Haemorrhage
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    0 / 34 (0.00%)
    0 / 12 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    Middle Ear Effusion
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 4 (25.00%)
    0 / 8 (0.00%)
    0 / 34 (0.00%)
    0 / 12 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    0
    1
    0
    0
    0
    0
    Ear Pain
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    0 / 34 (0.00%)
    0 / 12 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    0
    0
    0
    2
    Eye disorders
    Eyelid Myoclonus
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    2 / 34 (5.88%)
    0 / 12 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    0
    0
    0
    2
    0
    0
    Ocular Hyperaemia
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    0 / 34 (0.00%)
    0 / 12 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    Gastrointestinal disorders
    Angular Cheilitis
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    0 / 34 (0.00%)
    0 / 12 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    Abdominal Pain
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    0 / 34 (0.00%)
    0 / 12 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    Aphthous Ulcer
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    2 / 34 (5.88%)
    0 / 12 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    0
    0
    0
    2
    0
    0
    Constipation
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    4 / 34 (11.76%)
    1 / 12 (8.33%)
    3 / 15 (20.00%)
         occurrences all number
    0
    0
    0
    4
    1
    8
    Dental Discomfort
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 4 (25.00%)
    0 / 8 (0.00%)
    0 / 34 (0.00%)
    0 / 12 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    0
    3
    0
    0
    0
    0
    Diarrhoea
         subjects affected / exposed
    1 / 5 (20.00%)
    0 / 4 (0.00%)
    1 / 8 (12.50%)
    4 / 34 (11.76%)
    0 / 12 (0.00%)
    2 / 15 (13.33%)
         occurrences all number
    1
    0
    1
    6
    0
    4
    Food Poisoning
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    0 / 34 (0.00%)
    0 / 12 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    Gastritis
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    0 / 34 (0.00%)
    0 / 12 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    Vomiting
         subjects affected / exposed
    1 / 5 (20.00%)
    0 / 4 (0.00%)
    4 / 8 (50.00%)
    15 / 34 (44.12%)
    6 / 12 (50.00%)
    7 / 15 (46.67%)
         occurrences all number
    1
    0
    8
    20
    9
    10
    Lower Gastrointestinal Haemorrhage
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    0 / 34 (0.00%)
    0 / 12 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    Nausea
         subjects affected / exposed
    1 / 5 (20.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    2 / 34 (5.88%)
    1 / 12 (8.33%)
    0 / 15 (0.00%)
         occurrences all number
    1
    0
    0
    4
    1
    0
    Retching
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    1 / 34 (2.94%)
    0 / 12 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    0
    1
    0
    1
    Salivary Hypersecretion
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    0 / 34 (0.00%)
    0 / 12 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    Stomatitis
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    2 / 34 (5.88%)
    0 / 12 (0.00%)
    2 / 15 (13.33%)
         occurrences all number
    0
    0
    0
    4
    0
    2
    Toothache
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    0 / 34 (0.00%)
    0 / 12 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    Gastrooesophageal Reflux Disease
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    3 / 34 (8.82%)
    1 / 12 (8.33%)
    0 / 15 (0.00%)
         occurrences all number
    0
    0
    0
    3
    1
    0
    Hepatobiliary disorders
    Hepatic Steatosis
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    0 / 34 (0.00%)
    0 / 12 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    Skin and subcutaneous tissue disorders
    Dermatitis Atopic
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    1 / 8 (12.50%)
    0 / 34 (0.00%)
    0 / 12 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    0
    Dermatitis
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    0 / 34 (0.00%)
    1 / 12 (8.33%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    0
    0
    1
    1
    Macule
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    0 / 34 (0.00%)
    0 / 12 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    Perioral Dermatitis
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    1 / 8 (12.50%)
    0 / 34 (0.00%)
    0 / 12 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    0
    Rash
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    1 / 8 (12.50%)
    2 / 34 (5.88%)
    0 / 12 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    1
    2
    0
    1
    Skin Irritation
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    0 / 34 (0.00%)
    1 / 12 (8.33%)
    0 / 15 (0.00%)
         occurrences all number
    0
    0
    0
    0
    1
    0
    Urticaria
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    1 / 34 (2.94%)
    0 / 12 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    0
    1
    0
    1
    Skin Hyperpigmentation
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    0 / 34 (0.00%)
    0 / 12 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    Renal and urinary disorders
    Glycosuria
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    0 / 34 (0.00%)
    1 / 12 (8.33%)
    0 / 15 (0.00%)
         occurrences all number
    0
    0
    0
    0
    1
    0
    Musculoskeletal and connective tissue disorders
    Mobility Decreased
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    0 / 34 (0.00%)
    0 / 12 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    0
    0
    0
    3
    Back Pain
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    3 / 34 (8.82%)
    1 / 12 (8.33%)
    3 / 15 (20.00%)
         occurrences all number
    0
    0
    0
    3
    1
    5
    Jaw Disorder
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    0 / 34 (0.00%)
    0 / 12 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    Limb Discomfort
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    0 / 34 (0.00%)
    0 / 12 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    Muscular Weakness
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    2 / 34 (5.88%)
    0 / 12 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    0
    0
    0
    3
    0
    0
    Neck Pain
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    0 / 34 (0.00%)
    0 / 12 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    Pain In Extremity
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    2 / 34 (5.88%)
    0 / 12 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    0
    0
    0
    2
    0
    0
    Infections and infestations
    Covid-19
         subjects affected / exposed
    1 / 5 (20.00%)
    0 / 4 (0.00%)
    1 / 8 (12.50%)
    4 / 34 (11.76%)
    0 / 12 (0.00%)
    5 / 15 (33.33%)
         occurrences all number
    1
    0
    1
    4
    0
    5
    Bronchitis
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    2 / 34 (5.88%)
    0 / 12 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    0
    0
    0
    2
    0
    0
    Croup Infectious
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    1 / 8 (12.50%)
    1 / 34 (2.94%)
    0 / 12 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    0
    0
    1
    1
    0
    0
    Ear Infection
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    2 / 8 (25.00%)
    3 / 34 (8.82%)
    2 / 12 (16.67%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    2
    4
    3
    1
    Enterobiasis
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    2 / 34 (5.88%)
    0 / 12 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    0
    0
    0
    2
    0
    0
    Eye Infection
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    1 / 8 (12.50%)
    1 / 34 (2.94%)
    0 / 12 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    0
    0
    1
    1
    0
    0
    Fungal Skin Infection
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    0 / 34 (0.00%)
    1 / 12 (8.33%)
    0 / 15 (0.00%)
         occurrences all number
    0
    0
    0
    0
    3
    0
    Gastroenteritis
         subjects affected / exposed
    1 / 5 (20.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    5 / 34 (14.71%)
    2 / 12 (16.67%)
    4 / 15 (26.67%)
         occurrences all number
    1
    0
    0
    7
    3
    4
    Lower Respiratory Tract Infection
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    0 / 34 (0.00%)
    0 / 12 (0.00%)
    2 / 15 (13.33%)
         occurrences all number
    0
    0
    0
    0
    0
    3
    Hand-Foot-And-Mouth Disease
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    1 / 8 (12.50%)
    2 / 34 (5.88%)
    0 / 12 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    0
    0
    1
    3
    0
    0
    Hepatitis Infectious Mononucleosis
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    1 / 8 (12.50%)
    0 / 34 (0.00%)
    0 / 12 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    0
    Impetigo
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    1 / 34 (2.94%)
    0 / 12 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    0
    1
    0
    2
    Influenza
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    1 / 8 (12.50%)
    5 / 34 (14.71%)
    3 / 12 (25.00%)
    0 / 15 (0.00%)
         occurrences all number
    0
    0
    1
    8
    3
    0
    Laryngitis
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    2 / 34 (5.88%)
    0 / 12 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    0
    0
    0
    3
    0
    0
    Lice Infestation
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    0 / 34 (0.00%)
    0 / 12 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    0
    0
    0
    2
    Gastroenteritis Viral
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    1 / 8 (12.50%)
    1 / 34 (2.94%)
    0 / 12 (0.00%)
    2 / 15 (13.33%)
         occurrences all number
    0
    0
    1
    1
    0
    2
    Medical Device Site Infection
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    0 / 34 (0.00%)
    0 / 12 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    0
    0
    0
    2
    Myringitis
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    1 / 8 (12.50%)
    0 / 34 (0.00%)
    0 / 12 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    0
    Nasopharyngitis
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 4 (25.00%)
    1 / 8 (12.50%)
    8 / 34 (23.53%)
    5 / 12 (41.67%)
    6 / 15 (40.00%)
         occurrences all number
    0
    1
    1
    12
    6
    14
    Oral Candidiasis
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    1 / 34 (2.94%)
    0 / 12 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    0
    1
    0
    1
    Otitis Externa
         subjects affected / exposed
    1 / 5 (20.00%)
    1 / 4 (25.00%)
    0 / 8 (0.00%)
    0 / 34 (0.00%)
    0 / 12 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    1
    1
    0
    0
    0
    0
    Otitis Media
         subjects affected / exposed
    1 / 5 (20.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    2 / 34 (5.88%)
    0 / 12 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    1
    0
    0
    3
    0
    0
    Pneumonia
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    2 / 34 (5.88%)
    1 / 12 (8.33%)
    2 / 15 (13.33%)
         occurrences all number
    0
    0
    0
    2
    1
    2
    Pantoea Agglomerans Infection
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    0 / 34 (0.00%)
    1 / 12 (8.33%)
    0 / 15 (0.00%)
         occurrences all number
    0
    0
    0
    0
    1
    0
    Parvovirus B19 Infection
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    2 / 34 (5.88%)
    0 / 12 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    0
    0
    0
    2
    0
    0
    Pertussis
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    0 / 34 (0.00%)
    0 / 12 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    Pharyngitis
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    3 / 34 (8.82%)
    0 / 12 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    0
    0
    0
    3
    0
    0
    Pharyngitis Streptococcal
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    1 / 8 (12.50%)
    1 / 34 (2.94%)
    4 / 12 (33.33%)
    0 / 15 (0.00%)
         occurrences all number
    0
    0
    2
    3
    6
    0
    Otitis Media Acute
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    1 / 8 (12.50%)
    2 / 34 (5.88%)
    0 / 12 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    0
    0
    1
    2
    0
    0
    Rhinitis
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    2 / 34 (5.88%)
    0 / 12 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    0
    0
    0
    2
    0
    0
    Rhinovirus Infection
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    0 / 34 (0.00%)
    1 / 12 (8.33%)
    0 / 15 (0.00%)
         occurrences all number
    0
    0
    0
    0
    1
    0
    Sinusitis
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    1 / 34 (2.94%)
    2 / 12 (16.67%)
    0 / 15 (0.00%)
         occurrences all number
    0
    0
    0
    1
    2
    0
    Tonsillitis
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    2 / 34 (5.88%)
    0 / 12 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    0
    0
    0
    3
    0
    0
    Upper Respiratory Tract Infection
         subjects affected / exposed
    1 / 5 (20.00%)
    1 / 4 (25.00%)
    4 / 8 (50.00%)
    6 / 34 (17.65%)
    6 / 12 (50.00%)
    5 / 15 (33.33%)
         occurrences all number
    1
    1
    5
    10
    8
    15
    Urinary Tract Infection
         subjects affected / exposed
    2 / 5 (40.00%)
    0 / 4 (0.00%)
    1 / 8 (12.50%)
    2 / 34 (5.88%)
    0 / 12 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    3
    0
    1
    2
    0
    4
    Viral Infection
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    1 / 8 (12.50%)
    10 / 34 (29.41%)
    0 / 12 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    1
    12
    0
    1
    Metabolism and nutrition disorders
    Dehydration
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    0 / 34 (0.00%)
    0 / 12 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    Hypoglycaemia
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    0 / 34 (0.00%)
    0 / 12 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    Decreased Appetite
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 8 (0.00%)
    0 / 34 (0.00%)
    2 / 12 (16.67%)
    2 / 15 (13.33%)
         occurrences all number
    0
    0
    0
    0
    2
    3

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    17 Dec 2019
    *Changed number of patients treated by cohort *Medical monitor review of safety data prior to dose escalation within dose cohort *Change to DSMB requirements *Updated number of patients per cohort *Added new exclusion criteria *Open label extension will be conducted under a separate protocol, clarification that patients may remain on study without treatment until initiation of OLE *Added details on anesthesia use *Added details related to LP procedure *Eliminated obtaining weights every 2 weeks *Changed inflammatory marker to be analyzed.
    01 May 2020
    *LPs allowed without anesthesia *Added inclusion criteria *Given COVID-19 pandemic; allow EEG to be obtained outside in-patient hospital setting, allow safety visits via video conference/telemedicine and home visits from visiting nurses or health care provider *Added blood and urine collection for other biomarkers *Changed time of ECG for patient convenience *Changed time of neurological exam *Specified time limit on duration of AEs that require discontinuation from study *Updated management of thrombocytopenia and proteinuria.
    29 Jul 2022
    *Change of sponsor from GeneTx to Ultragenyx *Removed specificity of UK and Canada *Adjusted efficacy measures
    13 Mar 2023
    *Removed Dose-selection Cohorts 8-12 *Expansion Cohorts A and B updates to loading and maintenance period *Dose-selection Cohorts 4-7 maintenance dosing modified *Dexamethasone premedication required for loading doses *Radiculopathy defined as AESI *Contraception guidance added.

    Interruptions (globally)
    Were there any global interruptions to the trial? Yes
    Date
    Interruption
    Restart date
    20 Jul 2020
    The Sponsor suspended dosing and enrollment into the study according to the protocol-defined dose stopping criteria after the first patient experienced the related SAE of radiculopathy. On 03 November 2020, the FDA placed the study on a Full Clinical Hold. The Sponsor engaged with the Agency to review data and discuss changes to the study design.
    24 Sep 2021

    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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