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    Clinical Trial Results:
    A Multicenter, Open-label Extension Study to Evaluate the Safety, Pharmacodynamics, and Clinical Effects of WVE-120101 in Patients with Huntington’s Disease

    Summary
    EudraCT number
    		 2019-003637-42
    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-HDSNP1-002
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT04617847
    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-120101 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: 8
    Country: Number of subjects enrolled
    Canada: 6
    Country: Number of subjects enrolled
    Denmark: 2
    Country: Number of subjects enrolled
    France: 2
    Country: Number of subjects enrolled
    Poland: 9
    Worldwide total number of subjects
    27
    EEA total number of subjects
    13
    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)
    27
    From 65 to 84 years
    0
    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-HDSNP1-001.

    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 27 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-120101
    Arm description
    		 Enrolled at 4 milligram (mg) WVE-120101 dose level.
    Arm type
    		 Experimental

    Investigational medicinal product name
    WVE-120101
    Investigational medicinal product code
    WVE-120101
    Other name
    Pharmaceutical forms
    Powder for solution for injection
    Routes of administration
    Intrathecal use
    Dosage and administration details
    WVE-120101 4 mg was administered monthly via intrathecal dosing through Week 97.

    Arm title
    		 16 mg WVE-120101
    Arm description
    		 Enrolled at 16 mg WVE-120101 dose level.
    Arm type
    		 Experimental

    Investigational medicinal product name
    WVE-120101
    Investigational medicinal product code
    WVE-120101
    Other name
    Pharmaceutical forms
    Powder for solution for injection
    Routes of administration
    Intrathecal use
    Dosage and administration details
    WVE-120101 16 mg was administered monthly via intrathecal dosing through Week 97.

    Number of subjects in period 1
    		4 mg WVE-120101 16 mg WVE-120101
    Started
    3
    24
    Dose Modified to 16 mg WVE-120101
    3
    1
    Dose Modified to 32 mg WVE-120101
    0
    5
    Completed
    0
    0
    Not completed
    3
    24
         Consent withdrawn by subject
    1
    -
         Adverse event, non-fatal
    -
    2
         Death
    -
    1
         Termination of Study by Sponsor
    2
    21

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    4 mg WVE-120101
    Reporting group description
    		 Enrolled at 4 milligram (mg) WVE-120101 dose level.

    Reporting group title
    16 mg WVE-120101
    Reporting group description
    		 Enrolled at 16 mg WVE-120101 dose level.

    Reporting group values
    		 4 mg WVE-120101 16 mg WVE-120101 Total
    Number of subjects
    		 3 24 27
    Age categorical
    Patients age at the time of enrollment in the WVE-HDSNP1-001 study is presented.
    Units: Subjects
        In utero
    		 0 0 0
        Preterm newborn infants (gestational age < 37 wks)
    		 0 0 0
        Newborns (0-27 days)
    		 0 0 0
        Infants and toddlers (28 days-23 months)
    		 0 0 0
        Children (2-11 years)
    		 0 0 0
        Adolescents (12-17 years)
    		 0 0 0
        Adults (18-64 years)
    		 3 24 27
        From 65-84 years
    		 0 0 0
        85 years and over
    		 0 0 0
    Gender categorical
    Units: Subjects
        Female
    		 2 15 17
        Male
    		 1 9 10
    Ethnicity
    Units: Subjects
        Hispanic or Latino
    		 0 0 0
        Not Hispanic or Latino
    		 3 24 27
        Unknown or Not Reported
    		 0 0 0
    Race
    Units: Subjects
        American Indian or Alaska Native
    		 0 0 0
        Asian
    		 0 0 0
        Native Hawaiian or Other Pacific Islander
    		 0 0 0
        Black or African American
    		 0 0 0
        White
    		 3 24 27
        More than one race
    		 0 0 0
        Unknown or Not Reported
    		 0 0 0
    Region of Enrollment
    Units: Subjects
        Australia
    		 0 8 8
        Canada
    		 3 3 6
        Denmark
    		 0 2 2
        France
    		 0 2 2
        Poland
    		 0 9 9

    End points

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    End points reporting groups
    Reporting group title
    4 mg WVE-120101
    Reporting group description
    		 Enrolled at 4 milligram (mg) WVE-120101 dose level.

    Reporting group title
    16 mg WVE-120101
    Reporting group description
    		 Enrolled at 16 mg WVE-120101 dose level.

    Subject analysis set title
    4 mg WVE-120101
    Subject analysis set type
    		 Safety analysis
    Subject analysis set description
    Patients who received 4 mg WVE-120101 at any point in the study.

    Subject analysis set title
    16 mg WVE-120101
    Subject analysis set type
    		 Safety analysis
    Subject analysis set description
    Patients who received 16 mg WVE-120101 at any point in the study.

    Subject analysis set title
    32 mg WVE-120101
    Subject analysis set type
    		 Safety analysis
    Subject analysis set description
    Patients who received 32 mg WVE-120101 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-120101 16 mg WVE-120101 32 mg WVE-120101
    Number of subjects analysed
    3
    27
    5
    Units: patients
    2
    17
    2
    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-120101 16 mg WVE-120101 32 mg WVE-120101
    Number of subjects analysed
    3
    27
    5
    Units: patients
    0
    5
    0
    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-120101 16 mg WVE-120101 32 mg WVE-120101
    Number of subjects analysed
    3
    27
    5
    Units: patients
    0
    3
    1
    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-120101 16 mg WVE-120101 32 mg WVE-120101
    Number of subjects analysed
    3
    27
    5
    Units: patients
    0
    1
    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-120101.
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    8.2
    Reporting groups
    Reporting group title
    4 mg WVE-120101
    Reporting group description
    		 Patients who received 4 mg WVE-120101 at any point in the study.

    Reporting group title
    16 mg WVE-120101
    Reporting group description
    		 Patients who received 16 mg WVE-120101 at any point in the study.

    Reporting group title
    32 mg WVE-120101
    Reporting group description
    		 Patients who received 32 mg WVE-120101 at any point in the study.

    Serious adverse events
    		 4 mg WVE-120101 16 mg WVE-120101 32 mg WVE-120101
    Total subjects affected by serious adverse events
         subjects affected / exposed
    0 / 3 (0.00%)
    3 / 27 (11.11%)
    1 / 5 (20.00%)
         number of deaths (all causes)
    0
    2
    0
         number of deaths resulting from adverse events
    0
    2
    0
    Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    Lung Cancer Metastatic
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 27 (3.70%)
    0 / 5 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
    Neoplasm
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 27 (3.70%)
    0 / 5 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
    Injury, poisoning and procedural complications
    Head Injury
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 27 (3.70%)
    0 / 5 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Nervous system disorders
    Balance disorder
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 27 (0.00%)
    1 / 5 (20.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Headache
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 27 (3.70%)
    0 / 5 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 3
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Psychiatric disorders
    Completed Suicide
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 27 (3.70%)
    0 / 5 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 4 mg WVE-120101 16 mg WVE-120101 32 mg WVE-120101
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    2 / 3 (66.67%)
    16 / 27 (59.26%)
    2 / 5 (40.00%)
    Investigations
    CSF Protein Increased
         subjects affected / exposed
    1 / 3 (33.33%)
    1 / 27 (3.70%)
    0 / 5 (0.00%)
         occurrences all number
    1
    1
    0
    CSF white blood cell count increased
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 27 (0.00%)
    0 / 5 (0.00%)
         occurrences all number
    1
    0
    0
    Lymphocyte count increased
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 27 (0.00%)
    0 / 5 (0.00%)
         occurrences all number
    1
    0
    0
    Injury, poisoning and procedural complications
    Fall
         subjects affected / exposed
    0 / 3 (0.00%)
    3 / 27 (11.11%)
    0 / 5 (0.00%)
         occurrences all number
    0
    4
    0
    Procedural Pain
         subjects affected / exposed
    0 / 3 (0.00%)
    3 / 27 (11.11%)
    0 / 5 (0.00%)
         occurrences all number
    0
    6
    0
    Nervous system disorders
    Balance disorder
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 27 (0.00%)
    1 / 5 (20.00%)
         occurrences all number
    0
    0
    1
    Dizziness
         subjects affected / exposed
    0 / 3 (0.00%)
    7 / 27 (25.93%)
    0 / 5 (0.00%)
         occurrences all number
    0
    13
    0
    Dysarthria
         subjects affected / exposed
    0 / 3 (0.00%)
    2 / 27 (7.41%)
    0 / 5 (0.00%)
         occurrences all number
    0
    2
    0
    Headache
         subjects affected / exposed
    0 / 3 (0.00%)
    5 / 27 (18.52%)
    0 / 5 (0.00%)
         occurrences all number
    0
    10
    0
    Restless legs syndrome
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 27 (0.00%)
    1 / 5 (20.00%)
         occurrences all number
    0
    0
    1
    Musculoskeletal and connective tissue disorders
    Back pain
         subjects affected / exposed
    0 / 3 (0.00%)
    3 / 27 (11.11%)
    0 / 5 (0.00%)
         occurrences all number
    0
    5
    0
    Pain in extremity
         subjects affected / exposed
    0 / 3 (0.00%)
    2 / 27 (7.41%)
    0 / 5 (0.00%)
         occurrences all number
    0
    2
    0

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    31 Mar 2020
    Protocol was amended to modify dose of all patients to the 16 mg dose or higher doses, following evaluation 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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