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    Clinical Trial Results:
    A Multicenter, Open-label Extension Study to Evaluate the Safety, Pharmacodynamics, and Clinical Effects of WVE-120102 in Patients with Huntington’s Disease

    Summary
    EudraCT number
    		 2019-002178-30
    Trial protocol
    		 PL   DK   FR   DE  
    Global end of trial date
    03 May 2021

    Results information
    Results version number
    		 v1(current)
    This version publication date
    04 Feb 2022
    First version publication date
    04 Feb 2022
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    WVE-HDSNP2-002
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT04617860
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Wave Life Sciences UK Limited
    Sponsor organisation address
    1 Chamberlain Square CS, Birmingham, United Kingdom, B3 3AX
    Public contact
    Chief Medical Officer, Wave Life Sciences, +1 617-949-2900, info@wavelifesci.com
    Scientific contact
    Chief Medical Officer, Wave Life Sciences, +1 617-949-2900, info@wavelifesci.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
    03 May 2021
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    03 May 2021
    Was the trial ended prematurely?
    Yes
    General information about the trial
    Main objective of the trial
    To evaluate the safety and tolerability of long-term exposure to WVE-120102 in patients with early manifest Huntington’s disease (HD).
    Protection of trial subjects
    The study was conducted according to the study protocol and standard operating procedures that meet the guidelines provided by the International Conference on Harmonisation for Good Clinical Practice in clinical studies, and any other applicable local regulatory requirements.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    13 Apr 2020
    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
    Australia: 1
    Country: Number of subjects enrolled
    Canada: 21
    Country: Number of subjects enrolled
    Denmark: 5
    Country: Number of subjects enrolled
    Poland: 9
    Worldwide total number of subjects
    36
    EEA total number of subjects
    14
    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)
    33
    From 65 to 84 years
    3
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 This Phase 1b/2a open-label extension study was conducted in adult patients with early manifest HD and who completed their final cerebrospinal fluid (CSF) collection or next visit after the final CSF collection (i.e., Day 168 or 196 depending upon dosing cohort and requirements in a given country) of the Phase 1b/2a clinical study WVE-HDSNP2-001.

    Pre-assignment
    Screening details
    		 The study consists of screening period (4 weeks), treatment period (97 weeks), and follow-up period (4 weeks). A total of 36 patients received treatment in this study.

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

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 4 mg WVE-120102
    Arm description
    		 Enrolled at 4 milligram (mg) WVE-120102 dose level.
    Arm type
    		 Experimental

    Investigational medicinal product name
    WVE-120102
    Investigational medicinal product code
    WVE-120102
    Other name
    Pharmaceutical forms
    Powder for solution for injection
    Routes of administration
    Intrathecal use
    Dosage and administration details
    WVE-120102 4 mg was administered monthly via intrathecal dosing through Week 97.

    Arm title
    		 8 mg WVE-120102
    Arm description
    		 Enrolled at 8 mg WVE-120102 dose level.
    Arm type
    		 Experimental

    Investigational medicinal product name
    WVE-120102
    Investigational medicinal product code
    WVE-120102
    Other name
    Pharmaceutical forms
    Powder for solution for injection
    Routes of administration
    Intrathecal use
    Dosage and administration details
    WVE-120102 8 mg was administered monthly via intrathecal dosing through Week 97.

    Arm title
    		 16 mg WVE-120102
    Arm description
    		 Enrolled at 16 mg WVE-120102 dose level.
    Arm type
    		 Experimental

    Investigational medicinal product name
    WVE-120102
    Investigational medicinal product code
    WVE-120102
    Other name
    Pharmaceutical forms
    Powder for solution for injection
    Routes of administration
    Intrathecal use
    Dosage and administration details
    WVE-120102 16 mg was administered monthly via intrathecal dosing through Week 97.

    Number of subjects in period 1
    		4 mg WVE-120102 8 mg WVE-120102 16 mg WVE-120102
    Started
    8
    10
    18
    Dose Modified to 8 mg WVE-120102
    8
    1
    0
    Dose Modified to 16 mg WVE-120102
    8
    9
    8
    Dose Modified to 32 mg WVE-120102
    2
    4
    11
    Completed
    0
    0
    0
    Not completed
    8
    10
    18
         Physician decision
    -
    1
    -
         Consent withdrawn by subject
    -
    -
    2
         Adverse event, non-fatal
    -
    2
    2
         Termination of Study by Sponsor
    8
    4
    13
         Sponsor decision
    -
    3
    1

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    4 mg WVE-120102
    Reporting group description
    		 Enrolled at 4 milligram (mg) WVE-120102 dose level.

    Reporting group title
    8 mg WVE-120102
    Reporting group description
    		 Enrolled at 8 mg WVE-120102 dose level.

    Reporting group title
    16 mg WVE-120102
    Reporting group description
    		 Enrolled at 16 mg WVE-120102 dose level.

    Reporting group values
    		 4 mg WVE-120102 8 mg WVE-120102 16 mg WVE-120102 Total
    Number of subjects
    		 8 10 18 36
    Age categorical
    Units: Subjects
        In utero
    		 0 0 0 0
        Preterm newborn infants (gestational age < 37 wks)
    		 0 0 0 0
        Newborns (0-27 days)
    		 0 0 0 0
        Infants and toddlers (28 days-23 months)
    		 0 0 0 0
        Children (2-11 years)
    		 0 0 0 0
        Adolescents (12-17 years)
    		 0 0 0 0
        Adults (18-64 years)
    		 7 10 16 33
        From 65-84 years
    		 1 0 2 3
        85 years and over
    		 0 0 0 0
    Gender categorical
    Units: Subjects
        Female
    		 3 6 7 16
        Male
    		 5 4 11 20
    Ethnicity
    Units: Subjects
        Hispanic or Latino
    		 0 0 0 0
        Not Hispanic or Latino
    		 8 10 18 36
        Unknown or Not Reported
    		 0 0 0 0
    Race
    Units: Subjects
        American Indian or Alaska Native
    		 0 0 0 0
        Asian
    		 0 0 0 0
        Native Hawaiian or Other Pacific Islander
    		 0 0 0 0
        Black or African American
    		 0 0 0 0
        White
    		 8 10 18 36
        More than one race
    		 0 0 0 0
        Unknown or Not Reported
    		 0 0 0 0
    Region of Enrollment
    Units: Subjects
        Australia
    		 0 0 1 1
        Canada
    		 8 1 12 21
        Denmark
    		 0 5 0 5
        Poland
    		 0 4 5 9

    End points

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    End points reporting groups
    Reporting group title
    4 mg WVE-120102
    Reporting group description
    		 Enrolled at 4 milligram (mg) WVE-120102 dose level.

    Reporting group title
    8 mg WVE-120102
    Reporting group description
    		 Enrolled at 8 mg WVE-120102 dose level.

    Reporting group title
    16 mg WVE-120102
    Reporting group description
    		 Enrolled at 16 mg WVE-120102 dose level.

    Subject analysis set title
    4 mg WVE-120102
    Subject analysis set type
    		 Safety analysis
    Subject analysis set description
    Patients who received 4 mg WVE-120102 at any point in the study.

    Subject analysis set title
    8 mg WVE-120102
    Subject analysis set type
    		 Safety analysis
    Subject analysis set description
    Patients who received 8 mg WVE-120102 at any point in the study.

    Subject analysis set title
    16 mg WVE-120102
    Subject analysis set type
    		 Safety analysis
    Subject analysis set description
    Patients who received 16 mg WVE-120102 at any point in the study.

    Subject analysis set title
    32 mg WVE-120102
    Subject analysis set type
    		 Safety analysis
    Subject analysis set description
    Patients who received 32 mg WVE-120102 at any point in the study.

    Primary: Safety: Number of Patients With Treatment-Emergent Adverse Events (TEAEs)

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    End point title
    		 Safety: Number of Patients With Treatment-Emergent Adverse Events (TEAEs) [1]
    End point description
    Patients treated at more than one dose level (e.g., initial dose and after dose modification) are included in each applicable dose group. Adverse events (AEs) are counted in the dose the patient was receiving at the time of onset. A summary of serious and all other non-serious AEs, regardless of causality, is located in the reported AEs module.
    End point type
    Primary
    End point timeframe
    Day 1 to Week 101/end of study
    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: Only descriptive statistical analysis was performed for the primary end point.
    End point values
    		 4 mg WVE-120102 8 mg WVE-120102 16 mg WVE-120102 32 mg WVE-120102
    Number of subjects analysed
    8
    18
    35
    17
    Units: patients
    7
    14
    34
    17
    No statistical analyses for this end point

    Primary: Safety: Number of Patients With a Severe TEAE

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    End point title
    		 Safety: Number of Patients With a Severe TEAE [2]
    End point description
    Patients treated at more than one dose level (e.g., initial dose and after dose modification) are included in each applicable dose group. AEs are counted in the dose the patient was receiving at the time of onset. A summary of serious and all other non-serious AEs, regardless of causality, is located in the reported AEs module.
    End point type
    Primary
    End point timeframe
    Day 1 to Week 101/end of study
    Notes
    [2] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: Only descriptive statistical analysis was performed for the primary end point.
    End point values
    		 4 mg WVE-120102 8 mg WVE-120102 16 mg WVE-120102 32 mg WVE-120102
    Number of subjects analysed
    8
    18
    35
    17
    Units: patients
    0
    0
    2
    5
    No statistical analyses for this end point

    Primary: Safety: Number of Patients With Serious TEAEs

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    End point title
    		 Safety: Number of Patients With Serious TEAEs [3]
    End point description
    Patients treated at more than one dose level (e.g., initial dose and after dose modification) are included in each applicable dose group. AEs are counted in the dose the patient was receiving at the time of onset. A summary of serious and all other non-serious AEs, regardless of causality, is located in the reported AEs module.
    End point type
    Primary
    End point timeframe
    Day 1 to Week 101/end of study
    Notes
    [3] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: Only descriptive statistical analysis was performed for the primary end point.
    End point values
    		 4 mg WVE-120102 8 mg WVE-120102 16 mg WVE-120102 32 mg WVE-120102
    Number of subjects analysed
    8
    18
    35
    17
    Units: patients
    0
    1
    5
    3
    No statistical analyses for this end point

    Primary: Safety and Tolerability: Number of Patients Who Withdraw Due to TEAEs

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    End point title
    		 Safety and Tolerability: Number of Patients Who Withdraw Due to TEAEs [4]
    End point description
    Patients treated at more than one dose level (e.g., initial dose and after dose modification) are included in each applicable dose group. AEs are counted in the dose the patient was receiving at the time of onset. A summary of serious and all other non-serious AEs, regardless of causality, is located in the reported AEs module.
    End point type
    Primary
    End point timeframe
    Day 1 to Week 101/end of study
    Notes
    [4] - 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: Only descriptive statistical analysis was performed for the primary end point.
    End point values
    		 4 mg WVE-120102 8 mg WVE-120102 16 mg WVE-120102 32 mg WVE-120102
    Number of subjects analysed
    8
    18
    35
    17
    Units: patients
    0
    1
    3
    0
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    First dose received (Day 1) through the Study Termination visit (maximum of 45 weeks of treatment).
    Adverse event reporting additional description
    Safety population included all patients who received at least 1 dose of WVE-120102.
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    8.2
    Reporting groups
    Reporting group title
    4 mg WVE-120102
    Reporting group description
    		 Patients who received 4 mg WVE-120102 at any point in the study.

    Reporting group title
    8 mg WVE-120102
    Reporting group description
    		 Patients who received 8 mg WVE-120102 at any point in the study.

    Reporting group title
    16 mg WVE-120102
    Reporting group description
    		 Patients who received 16 mg WVE-120102 at any point in the study.

    Reporting group title
    32 mg WVE-120102
    Reporting group description
    		 Patients who received 32 mg WVE-120102 at any point in the study.

    Serious adverse events
    		 4 mg WVE-120102 8 mg WVE-120102 16 mg WVE-120102 32 mg WVE-120102
    Total subjects affected by serious adverse events
         subjects affected / exposed
    0 / 8 (0.00%)
    1 / 18 (5.56%)
    5 / 35 (14.29%)
    3 / 17 (17.65%)
         number of deaths (all causes)
    0
    0
    0
    0
         number of deaths resulting from adverse events
    0
    0
    0
    0
    Nervous system disorders
    Amnesia
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 18 (0.00%)
    0 / 35 (0.00%)
    2 / 17 (11.76%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    2 / 2
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Nervous system disorder
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 18 (0.00%)
    0 / 35 (0.00%)
    1 / 17 (5.88%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    General disorders and administration site conditions
    Pyrexia
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 18 (0.00%)
    1 / 35 (2.86%)
    0 / 17 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Psychiatric disorders
    Aggression
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 18 (0.00%)
    1 / 35 (2.86%)
    0 / 17 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Agitation
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 18 (0.00%)
    0 / 35 (0.00%)
    1 / 17 (5.88%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Confusional state
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 18 (0.00%)
    0 / 35 (0.00%)
    2 / 17 (11.76%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    2 / 2
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Infections and infestations
    Meningitis
         subjects affected / exposed
    0 / 8 (0.00%)
    1 / 18 (5.56%)
    1 / 35 (2.86%)
    0 / 17 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    1 / 1
    1 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Meningitis aseptic
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 18 (0.00%)
    2 / 35 (5.71%)
    0 / 17 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    3 / 3
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 4 mg WVE-120102 8 mg WVE-120102 16 mg WVE-120102 32 mg WVE-120102
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    7 / 8 (87.50%)
    14 / 18 (77.78%)
    34 / 35 (97.14%)
    17 / 17 (100.00%)
    Vascular disorders
    Hypotension
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 18 (0.00%)
    0 / 35 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    0
    0
    0
    1
    General disorders and administration site conditions
    Fatigue
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 18 (0.00%)
    6 / 35 (17.14%)
    4 / 17 (23.53%)
         occurrences all number
    0
    0
    11
    5
    Gait disturbance
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 18 (0.00%)
    2 / 35 (5.71%)
    1 / 17 (5.88%)
         occurrences all number
    0
    0
    2
    1
    Injection site erythema
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 18 (0.00%)
    0 / 35 (0.00%)
    0 / 17 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Injection site hypersensitivity
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 18 (0.00%)
    0 / 35 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    0
    0
    0
    1
    Pain
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 18 (0.00%)
    3 / 35 (8.57%)
    1 / 17 (5.88%)
         occurrences all number
    1
    0
    3
    1
    Pyrexia
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 18 (0.00%)
    6 / 35 (17.14%)
    2 / 17 (11.76%)
         occurrences all number
    0
    0
    9
    2
    Reproductive system and breast disorders
    Breast tenderness
         subjects affected / exposed
    0 / 8 (0.00%)
    1 / 18 (5.56%)
    0 / 35 (0.00%)
    0 / 17 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Respiratory, thoracic and mediastinal disorders
    Cough
         subjects affected / exposed
    0 / 8 (0.00%)
    1 / 18 (5.56%)
    0 / 35 (0.00%)
    0 / 17 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Dyspnoea
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 18 (0.00%)
    0 / 35 (0.00%)
    0 / 17 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Oropharyngeal pain
         subjects affected / exposed
    0 / 8 (0.00%)
    1 / 18 (5.56%)
    3 / 35 (8.57%)
    0 / 17 (0.00%)
         occurrences all number
    0
    1
    4
    0
    Psychiatric disorders
    Apathy
         subjects affected / exposed
    0 / 8 (0.00%)
    1 / 18 (5.56%)
    1 / 35 (2.86%)
    0 / 17 (0.00%)
         occurrences all number
    0
    1
    1
    0
    Behaviour disorder
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 18 (0.00%)
    2 / 35 (5.71%)
    0 / 17 (0.00%)
         occurrences all number
    0
    0
    2
    0
    Confusional state
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 18 (0.00%)
    3 / 35 (8.57%)
    5 / 17 (29.41%)
         occurrences all number
    0
    0
    4
    7
    Hallucination, visual
         subjects affected / exposed
    0 / 8 (0.00%)
    1 / 18 (5.56%)
    0 / 35 (0.00%)
    0 / 17 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Insomnia
         subjects affected / exposed
    0 / 8 (0.00%)
    1 / 18 (5.56%)
    1 / 35 (2.86%)
    0 / 17 (0.00%)
         occurrences all number
    0
    1
    1
    0
    Irritability
         subjects affected / exposed
    0 / 8 (0.00%)
    2 / 18 (11.11%)
    0 / 35 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    0
    2
    0
    1
    Paranoia
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 18 (0.00%)
    0 / 35 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    0
    0
    0
    1
    Persecutory delusion
         subjects affected / exposed
    0 / 8 (0.00%)
    1 / 18 (5.56%)
    0 / 35 (0.00%)
    0 / 17 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Restlessness
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 18 (0.00%)
    2 / 35 (5.71%)
    0 / 17 (0.00%)
         occurrences all number
    0
    0
    3
    0
    Investigations
    Blood bilirubin increased
         subjects affected / exposed
    0 / 8 (0.00%)
    1 / 18 (5.56%)
    0 / 35 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    0
    1
    0
    1
    CSF lymphocyte count increased
         subjects affected / exposed
    0 / 8 (0.00%)
    2 / 18 (11.11%)
    5 / 35 (14.29%)
    0 / 17 (0.00%)
         occurrences all number
    0
    3
    6
    0
    CSF protein increased
         subjects affected / exposed
    0 / 8 (0.00%)
    3 / 18 (16.67%)
    7 / 35 (20.00%)
    4 / 17 (23.53%)
         occurrences all number
    0
    3
    9
    4
    CSF test abnormal
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 18 (0.00%)
    0 / 35 (0.00%)
    3 / 17 (17.65%)
         occurrences all number
    0
    0
    0
    3
    CSF white blood cell count
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 18 (0.00%)
    2 / 35 (5.71%)
    0 / 17 (0.00%)
         occurrences all number
    0
    0
    2
    0
    Complement factor increased
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 18 (0.00%)
    0 / 35 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    0
    0
    0
    1
    Dermatologic examination abnormal
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 18 (0.00%)
    0 / 35 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    0
    0
    0
    1
    Lymphocyte count increased
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 18 (0.00%)
    2 / 35 (5.71%)
    0 / 17 (0.00%)
         occurrences all number
    0
    0
    2
    0
    Neurological examination abnormal
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 18 (0.00%)
    0 / 35 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    0
    0
    0
    1
    Injury, poisoning and procedural complications
    Contusion
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 18 (0.00%)
    0 / 35 (0.00%)
    0 / 17 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Fall
         subjects affected / exposed
    2 / 8 (25.00%)
    3 / 18 (16.67%)
    5 / 35 (14.29%)
    2 / 17 (11.76%)
         occurrences all number
    2
    3
    5
    3
    Post procedural discomfort
         subjects affected / exposed
    0 / 8 (0.00%)
    1 / 18 (5.56%)
    0 / 35 (0.00%)
    0 / 17 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Procedural dizziness
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 18 (0.00%)
    0 / 35 (0.00%)
    0 / 17 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Procedural headache
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 18 (0.00%)
    2 / 35 (5.71%)
    0 / 17 (0.00%)
         occurrences all number
    1
    0
    2
    0
    Procedural pain
         subjects affected / exposed
    2 / 8 (25.00%)
    0 / 18 (0.00%)
    4 / 35 (11.43%)
    2 / 17 (11.76%)
         occurrences all number
    2
    0
    8
    3
    Scapula fracture
         subjects affected / exposed
    0 / 8 (0.00%)
    1 / 18 (5.56%)
    0 / 35 (0.00%)
    0 / 17 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Skin laceration
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 18 (0.00%)
    1 / 35 (2.86%)
    0 / 17 (0.00%)
         occurrences all number
    1
    0
    1
    0
    Nervous system disorders
    Amnesia
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 18 (0.00%)
    0 / 35 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    0
    0
    0
    1
    Ataxia
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 18 (0.00%)
    0 / 35 (0.00%)
    3 / 17 (17.65%)
         occurrences all number
    0
    0
    0
    3
    Balance disorder
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 18 (0.00%)
    0 / 35 (0.00%)
    2 / 17 (11.76%)
         occurrences all number
    0
    0
    0
    2
    Disturbance in attention
         subjects affected / exposed
    0 / 8 (0.00%)
    1 / 18 (5.56%)
    0 / 35 (0.00%)
    0 / 17 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Dizziness
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 18 (0.00%)
    4 / 35 (11.43%)
    3 / 17 (17.65%)
         occurrences all number
    0
    0
    7
    4
    Dysarthria
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 18 (0.00%)
    0 / 35 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    0
    0
    0
    2
    Dyskinesia
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 18 (0.00%)
    1 / 35 (2.86%)
    0 / 17 (0.00%)
         occurrences all number
    1
    0
    1
    0
    Headache
         subjects affected / exposed
    2 / 8 (25.00%)
    6 / 18 (33.33%)
    14 / 35 (40.00%)
    6 / 17 (35.29%)
         occurrences all number
    6
    15
    40
    8
    Hypotonia
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 18 (0.00%)
    0 / 35 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    0
    0
    0
    1
    Memory impairment
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 18 (0.00%)
    2 / 35 (5.71%)
    0 / 17 (0.00%)
         occurrences all number
    0
    0
    2
    0
    Migraine
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 18 (0.00%)
    0 / 35 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    0
    0
    0
    2
    Presyncope
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 18 (0.00%)
    0 / 35 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    2
    0
    0
    1
    Somnolence
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 18 (0.00%)
    0 / 35 (0.00%)
    2 / 17 (11.76%)
         occurrences all number
    0
    0
    0
    5
    Ear and labyrinth disorders
    Tinnitus
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 18 (0.00%)
    0 / 35 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    0
    0
    0
    1
    Gastrointestinal disorders
    Dysphagia
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 18 (0.00%)
    0 / 35 (0.00%)
    0 / 17 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Nausea
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 18 (0.00%)
    3 / 35 (8.57%)
    2 / 17 (11.76%)
         occurrences all number
    0
    0
    4
    2
    Paraesthesia oral
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 18 (0.00%)
    0 / 35 (0.00%)
    0 / 17 (0.00%)
         occurrences all number
    3
    0
    0
    0
    Vomiting
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 18 (0.00%)
    3 / 35 (8.57%)
    0 / 17 (0.00%)
         occurrences all number
    0
    0
    3
    0
    Skin and subcutaneous tissue disorders
    Erythema
         subjects affected / exposed
    0 / 8 (0.00%)
    1 / 18 (5.56%)
    0 / 35 (0.00%)
    0 / 17 (0.00%)
         occurrences all number
    0
    2
    0
    0
    Renal and urinary disorders
    Urinary retention
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 18 (0.00%)
    0 / 35 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    0
    0
    0
    1
    Musculoskeletal and connective tissue disorders
    Back pain
         subjects affected / exposed
    1 / 8 (12.50%)
    1 / 18 (5.56%)
    6 / 35 (17.14%)
    5 / 17 (29.41%)
         occurrences all number
    1
    1
    12
    6
    Muscular weakness
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 18 (0.00%)
    2 / 35 (5.71%)
    3 / 17 (17.65%)
         occurrences all number
    0
    0
    2
    4
    Myalgia
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 18 (0.00%)
    2 / 35 (5.71%)
    1 / 17 (5.88%)
         occurrences all number
    0
    0
    2
    1
    Neck pain
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 18 (0.00%)
    1 / 35 (2.86%)
    2 / 17 (11.76%)
         occurrences all number
    0
    0
    1
    2
    Pain in extremity
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 18 (0.00%)
    5 / 35 (14.29%)
    2 / 17 (11.76%)
         occurrences all number
    0
    0
    10
    2
    Infections and infestations
    Cystitis
         subjects affected / exposed
    0 / 8 (0.00%)
    1 / 18 (5.56%)
    0 / 35 (0.00%)
    0 / 17 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Nasopharyngitis
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 18 (0.00%)
    2 / 35 (5.71%)
    0 / 17 (0.00%)
         occurrences all number
    0
    0
    2
    0
    Oral candidiasis
         subjects affected / exposed
    0 / 8 (0.00%)
    1 / 18 (5.56%)
    0 / 35 (0.00%)
    0 / 17 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Upper respiratory tract infection
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 18 (0.00%)
    0 / 35 (0.00%)
    0 / 17 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Urinary tract infection
         subjects affected / exposed
    0 / 8 (0.00%)
    1 / 18 (5.56%)
    0 / 35 (0.00%)
    0 / 17 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Metabolism and nutrition disorders
    Decreased appetite
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 18 (0.00%)
    1 / 35 (2.86%)
    1 / 17 (5.88%)
         occurrences all number
    0
    0
    1
    1
    Increased appetite
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 18 (0.00%)
    0 / 35 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    0
    0
    0
    1
    Polydipsia
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 18 (0.00%)
    0 / 35 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    0
    0
    0
    1

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    11 Nov 2019
    Included United Kingdom and Australia changes into one protocol. Clarified the schedule of assessments.
    19 Mar 2020
    Updated to move all patients to the 16 mg dose and to include language allowing patients to receive higher doses as long as they were tested in the Phase 1b/2a study.

    Interruptions (globally)
    Were there any global interruptions to the trial? No

    Limitations and caveats
    Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data.
    Based on the efficacy findings in this study at the time of the interim analysis, the Sponsor decided to terminate the study as the benefit/risk analysis did not warrant continued dose escalation.
    For support, Contact us.
    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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