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    Clinical Trial Results:
    A Randomized, Double-blind, Placebo-controlled, Efficacy and Safety Study of WVE-210201 with Open-label Extension in Ambulatory Patients with Duchenne Muscular Dystrophy (DYSTANCE 51)

    Summary
    EudraCT number
    		 2018-004009-22
    Trial protocol
    		 FR   GB   SE   NL   BE   PL   CZ   DE   IT  
    Global end of trial date
    09 Jan 2020

    Results information
    Results version number
    		 v1(current)
    This version publication date
    13 Sep 2020
    First version publication date
    13 Sep 2020
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    WVE-DMDX51-003
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT03907072
    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, +617 949-2900, info@wavelifesci.com
    Scientific contact
    Chief Medical Officer, Wave Life Sciences, +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
    11 Feb 2020
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    09 Jan 2020
    Was the trial ended prematurely?
    Yes
    General information about the trial
    Main objective of the trial
    To evaluate the efficacy of WVE-210201 by assessing changes in motor function by North Star Ambulatory Assessment (NSAA) (EU/Japan).
    Protection of trial subjects
    Written informed consent from each patient or patient’s parent(s) or legal guardian(s), if applicable, and written assent from each patient, if applicable, were obtained before any study-specific screening or baseline period evaluations were performed. The anonymity of participating patients was maintained to the extent required by applicable laws and in accordance with current HIPAA standards. This study was designed and monitored in accordance with Sponsor procedures, which complied with the ethical principles of Good Clinical Practice (GCP) as required by the major regulatory authorities, and in accordance with the Declaration of Helsinki. A Data Monitoring Committee (DMC) was to review unblinded safety data periodically and on an ad hoc basis at least until completion of the Double-blind period. In addition, the DMC was to review the results from the planned interim analyses.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    24 Jan 2018
    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
    Sweden: 1
    Country: Number of subjects enrolled
    Belgium: 1
    Country: Number of subjects enrolled
    France: 2
    Country: Number of subjects enrolled
    Italy: 2
    Worldwide total number of subjects
    6
    EEA total number of subjects
    6
    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)
    6
    Adolescents (12-17 years)
    0
    Adults (18-64 years)
    0
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 This study was conducted in 4 countries (Italy, France, Belgium, and Sweden from 04 September 2019 to 09 January 2020).

    Pre-assignment
    Screening details
    		 Screening evaluations were completed within 6 weeks of signing the informed consent form. A total of 6 subjects were screened and enrolled in study treatment.

    Period 1
    Period 1 title
    Period 1 (overall period)
    Is this the baseline period?
    		 Yes
    Allocation method
    Randomised - controlled
    Blinding used
    		 Double blind
    Roles blinded
    		 Subject, Investigator, Monitor, Data analyst, Carer, Assessor
    Blinding implementation details
    Study was conducted as randomized, placebo-controlled, and double-blind. Results for the OLE portion of the study were not reported due to early study termination.

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Placebo
    Arm description
    		 Matched saline solution administered alone via IV infusion
    Arm type
    		 Placebo

    Investigational medicinal product name
    Placebo
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Solution for infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    Matched saline solution administered alone via IV infusion

    Arm title
    		 3 mg/kg WVE-210201
    Arm description
    		 3 mg/kg WVE-210201 administered via IV infusion
    Arm type
    		 Experimental

    Investigational medicinal product name
    Suvodirsen
    Investigational medicinal product code
    WVE-210201
    Other name
    Pharmaceutical forms
    Solution for infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    Patients received weekly IV infusions of suvodirsen or placebo. WVE-210201 was provided as an isotonic solution for dilution for infusion.

    Arm title
    		 4.5 mg/kg WVE-210201
    Arm description
    		 4.5 mg/kg WVE-210201 administered via IV infusion
    Arm type
    		 Experimental

    Investigational medicinal product name
    Suvodirsen
    Investigational medicinal product code
    WVE-210201
    Other name
    Pharmaceutical forms
    Solution for infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    Patients received weekly IV infusions of suvodirsen or placebo. WVE-210201 was provided as an isotonic solution for dilution for infusion.

    Number of subjects in period 1
    		Placebo 3 mg/kg WVE-210201 4.5 mg/kg WVE-210201
    Started
    2
    2
    2
    Completed
    0
    0
    0
    Not completed
    2
    2
    2
         Study Terminated by Sponsor
    2
    2
    2

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Placebo
    Reporting group description
    		 Matched saline solution administered alone via IV infusion

    Reporting group title
    3 mg/kg WVE-210201
    Reporting group description
    		 3 mg/kg WVE-210201 administered via IV infusion

    Reporting group title
    4.5 mg/kg WVE-210201
    Reporting group description
    		 4.5 mg/kg WVE-210201 administered via IV infusion

    Reporting group values
    		 Placebo 3 mg/kg WVE-210201 4.5 mg/kg WVE-210201 Total
    Number of subjects
    		 2 2 2 6
    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)
    		 2 2 2 6
        Adolescents (12-17 years)
    		 0 0 0 0
        Adults (18-64 years)
    		 0 0 0 0
        From 65-84 years
    		 0 0 0 0
        85 years and over
    		 0 0 0 0
    Gender categorical
    Units: Subjects
        Female
    		 0 0 0 0
        Male
    		 2 2 2 6
    Subject analysis sets

    Subject analysis set title
    Safety population
    Subject analysis set type
    		 Safety analysis
    Subject analysis set description
    All randomly assigned patients who received at least 1 dose of WVE-210201 or placebo.

    Subject analysis sets values
    		 Safety population
    Number of subjects
    6
    Age categorical
    Units: Subjects
        In utero
    0
        Preterm newborn infants (gestational age < 37 wks)
    0
        Newborns (0-27 days)
    0
        Infants and toddlers (28 days-23 months)
    0
        Children (2-11 years)
    6
        Adolescents (12-17 years)
    0
        Adults (18-64 years)
    0
        From 65-84 years
    0
        85 years and over
    0
    Age continuous
    Units:
        
    ( )
    Gender categorical
    Units: Subjects
        Female
    0
        Male
    6

    End points

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    End points reporting groups
    Reporting group title
    Placebo
    Reporting group description
    		 Matched saline solution administered alone via IV infusion

    Reporting group title
    3 mg/kg WVE-210201
    Reporting group description
    		 3 mg/kg WVE-210201 administered via IV infusion

    Reporting group title
    4.5 mg/kg WVE-210201
    Reporting group description
    		 4.5 mg/kg WVE-210201 administered via IV infusion

    Subject analysis set title
    Safety population
    Subject analysis set type
    		 Safety analysis
    Subject analysis set description
    All randomly assigned patients who received at least 1 dose of WVE-210201 or placebo.

    Primary: Change from baseline in NSAA through 48 weeks (EU/Japan)

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    End point title
    		 Change from baseline in NSAA through 48 weeks (EU/Japan) [1]
    End point description
    The NSAA is a validated unidimensional scale, designed for measuring motor function in ambulatory boys with DMD. There are no primary endpoint results due to patients early termination.
    End point type
    Primary
    End point timeframe
    From baseline over 48 weeks
    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: No statistical analysis has been performed due to early study termination.
    End point values
    		 Placebo 3 mg/kg WVE-210201 4.5 mg/kg WVE-210201
    Number of subjects analysed
    2
    2
    2
    Units: Subjects
        number (not applicable)
    2
    2
    2
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Day 1 to Early Termination
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    22.1
    Reporting groups
    Reporting group title
    Placebo
    Reporting group description
    		 Matched saline solution administered alone via IV infusion

    Reporting group title
    3 mg/kg WVE-210201
    Reporting group description
    		 3 mg/kg WVE-210201 administered via IV infusion

    Reporting group title
    4.5 mg/kg WVE-210201
    Reporting group description
    		 4.5 mg/kg WVE-210201 administered via IV infusion

    Serious adverse events
    		 Placebo 3 mg/kg WVE-210201 4.5 mg/kg WVE-210201
    Total subjects affected by serious adverse events
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    1 / 2 (50.00%)
         number of deaths (all causes)
    0
    0
    0
         number of deaths resulting from adverse events
    0
    0
    0
    Cardiac disorders
    Cardiac disorder
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    1 / 2 (50.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 Placebo 3 mg/kg WVE-210201 4.5 mg/kg WVE-210201
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    2 / 2 (100.00%)
    2 / 2 (100.00%)
    2 / 2 (100.00%)
    Investigations
    C-reactive protein increased
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    1 / 2 (50.00%)
         occurrences all number
    0
    0
    1
    Injury, poisoning and procedural complications
    Bone contusion
         subjects affected / exposed
    1 / 2 (50.00%)
    0 / 2 (0.00%)
    0 / 2 (0.00%)
         occurrences all number
    1
    0
    0
    Nervous system disorders
    Headache
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    1 / 2 (50.00%)
         occurrences all number
    0
    0
    4
    General disorders and administration site conditions
    Pyrexia
         subjects affected / exposed
    0 / 2 (0.00%)
    1 / 2 (50.00%)
    2 / 2 (100.00%)
         occurrences all number
    0
    3
    4
    Gastrointestinal disorders
    Diarrhoea
         subjects affected / exposed
    0 / 2 (0.00%)
    1 / 2 (50.00%)
    1 / 2 (50.00%)
         occurrences all number
    0
    1
    1
    Vomiting
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    1 / 2 (50.00%)
         occurrences all number
    0
    0
    4
    Infections and infestations
    Gastroenteritis
         subjects affected / exposed
    1 / 2 (50.00%)
    0 / 2 (0.00%)
    0 / 2 (0.00%)
         occurrences all number
    1
    0
    0
    Nasopharyngitis
         subjects affected / exposed
    0 / 2 (0.00%)
    1 / 2 (50.00%)
    1 / 2 (50.00%)
         occurrences all number
    0
    1
    1
    Oral herpes
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    1 / 2 (50.00%)
         occurrences all number
    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
    09 Jul 2019
    Amendment 2.0, 48-week open-label extension period added to the 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.
    Not applicable
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