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    Clinical Trial Results:
    A Multicenter, Double-blind, Placebo-controlled, Phase 1 Study of WVE-210201 Administered Intravenously to Patients with Duchenne Muscular Dystrophy

    Summary
    EudraCT number
    		 2017-002686-21
    Trial protocol
    		 GB   FR   BE   NL   IT  
    Global end of trial date
    06 Mar 2019

    Results information
    Results version number
    		 v1(current)
    This version publication date
    26 Mar 2020
    First version publication date
    26 Mar 2020
    Other versions

    Trial information

    		 Close Top of page
    Trial identification
    Sponsor protocol code
    WVE-DMDX51-001
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT03508947
    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, +44 (617) 949-2900, info@wavelifesci.com
    Scientific contact
    Chief Medical Officer, Wave Life Sciences, +44 (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
    27 Mar 2019
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    06 Mar 2019
    Global end of trial reached?
    Yes
    Global end of trial date
    06 Mar 2019
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    To evaluate the safety and tolerability of single ascending doses of WVE-210201 in patients with DMD.
    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 will be 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. The decision regarding escalation to each subsequent dose level was made based on the recommendation of the Dose Escalation Committee (DEC). The DEC was blinded to treatment assignment throughout the study. The unblinded, independent Safety Monitoring Committee (SMC) reviewed aggregate safety data periodically and SAEs as they occurred. The SMC also reviewed any serious adverse events (SAEs) that occurred in sentinel patients in order to determine if the cohort should continue or, in conjunction with the DEC, whether an intermediate dose should be selected for the remaining patients in that cohort. In addition, if treatment-emergent adverse events (TEAEs) occurred that met the Dose Escalation Stopping Criteria, the SMC reviewed the unblinded safety data and determined whether it was safe to proceed with dosing.
    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
    Netherlands: 1
    Country: Number of subjects enrolled
    United Kingdom: 6
    Country: Number of subjects enrolled
    Belgium: 10
    Country: Number of subjects enrolled
    France: 5
    Country: Number of subjects enrolled
    Italy: 4
    Country: Number of subjects enrolled
    United States: 10
    Worldwide total number of subjects
    36
    EEA total number of subjects
    26
    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)
    34
    Adolescents (12-17 years)
    2
    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 6 countries (Belgium, France, Italy, Netherlands, United Kingdom, and United States) from 24 January 2018 to 06 March 2019.

    Pre-assignment
    Screening details
    		 A total of 42 subjects were screened and 36 subjects were enrolled and randomized to study treatment or placebo.

    Pre-assignment period milestones
    Number of subjects started
    		 36
    Number of subjects completed
    		 36

    Period 1
    Period 1 title
    Overall Study (overall period)
    Is this the baseline period?
    		 Yes
    Allocation method
    Randomised - controlled
    Blinding used
    		 Double blind
    Roles blinded
    		 Subject, Investigator, Data analyst, Carer, Assessor

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Pooled Placebo
    Arm description
    		 0.9% Sodium Chloride injection or 0.45% Sodium Chloride injection solution administered alone via IV infusion
    Arm type
    		 Placebo Comparator

    Investigational medicinal product name
    No investigational medicinal product assigned in this arm
    Arm title
    		 0.5 mg/kg WVE-210201
    Arm description
    		 0.5 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
    Powder for infusion, Solution for infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    Patients received a single IV infusion of suvodirsen or placebo.

    Arm title
    		 1 mg/kg WVE-210201
    Arm description
    		 1 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
    Powder for infusion, Solution for infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    Patients received a single IV infusion of suvodirsen or placebo.

    Arm title
    		 2 mg/kg WVE-210201
    Arm description
    		 2 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
    Powder for infusion, Solution for infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    Patients received a single IV infusion of suvodirsen or placebo.

    Arm title
    		 5 mg/kg WVE-210201
    Arm description
    		 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
    Powder for infusion, Solution for infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    Patients received a single IV infusion of suvodirsen or placebo.

    Arm title
    		 7/10 mg/kg WVE-210201
    Arm description
    		 7 or 10 mg/kg administered via IV infusion
    Arm type
    		 Experimental

    Investigational medicinal product name
    Suvodirsen
    Investigational medicinal product code
    WVE-210201
    Other name
    Pharmaceutical forms
    Powder for infusion, Solution for infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    Patients received a single IV infusion of suvodirsen or placebo.

    Number of subjects in period 1
    		Pooled Placebo 0.5 mg/kg WVE-210201 1 mg/kg WVE-210201 2 mg/kg WVE-210201 5 mg/kg WVE-210201 7/10 mg/kg WVE-210201
    Started
    10
    6
    6
    6
    6
    2
    Completed
    10
    6
    6
    6
    6
    2

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Pooled Placebo
    Reporting group description
    		 0.9% Sodium Chloride injection or 0.45% Sodium Chloride injection solution administered alone via IV infusion

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

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

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

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

    Reporting group title
    7/10 mg/kg WVE-210201
    Reporting group description
    		 7 or 10 mg/kg administered via IV infusion

    Reporting group values
    		 Pooled Placebo 0.5 mg/kg WVE-210201 1 mg/kg WVE-210201 2 mg/kg WVE-210201 5 mg/kg WVE-210201 7/10 mg/kg WVE-210201 Total
    Number of subjects
    		 10 6 6 6 6 2 36
    Age categorical
    Units: Subjects
        In utero
    		 0 0 0 0 0 0 0
        Preterm newborn infants (gestational age < 37 wks)
    		 0 0 0 0 0 0 0
        Newborns (0-27 days)
    		 0 0 0 0 0 0 0
        Infants and toddlers (28 days-23 months)
    		 0 0 0 0 0 0 0
        Children (2-11 years)
    		 10 5 6 5 6 2 34
        Adolescents (12-17 years)
    		 0 1 0 1 0 0 2
        Adults (18-64 years)
    		 0 0 0 0 0 0 0
        From 65-84 years
    		 0 0 0 0 0 0 0
        85 years and over
    		 0 0 0 0 0 0 0
    Gender categorical
    Units: Subjects
        Female
    		 0 0 0 0 0 0 0
        Male
    		 10 6 6 6 6 2 36
    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
    36
    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)
    34
        Adolescents (12-17 years)
    2
        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
    36

    End points

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    End points reporting groups
    Reporting group title
    Pooled Placebo
    Reporting group description
    		 0.9% Sodium Chloride injection or 0.45% Sodium Chloride injection solution administered alone via IV infusion

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

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

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

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

    Reporting group title
    7/10 mg/kg WVE-210201
    Reporting group description
    		 7 or 10 mg/kg 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: Number of patients with treatment-emergent adverse events (TEAEs)

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    End point title
    		 Number of patients with treatment-emergent adverse events (TEAEs) [1]
    End point description
    A TEAE was defined as any untoward medical occurrence in a patient following study drug administration, regardless of its causal relationship to study treatment.
    End point type
    Primary
    End point timeframe
    Day 1 to Day 85 (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: No statistical analysis were performed
    End point values
    		 Pooled Placebo 0.5 mg/kg WVE-210201 1 mg/kg WVE-210201 2 mg/kg WVE-210201 5 mg/kg WVE-210201 7/10 mg/kg WVE-210201
    Number of subjects analysed
    10
    6
    6
    6
    6
    2
    Units: Subjects
        number (not applicable)
    8
    3
    5
    4
    4
    2
    No statistical analyses for this end point

    Primary: Number of Patients who Experienced a Severe TEAE

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    End point title
    		 Number of Patients who Experienced a Severe TEAE [2]
    End point description
    End point type
    Primary
    End point timeframe
    Day 1 to Day 85 (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: No statistical analysis were performed
    End point values
    		 Pooled Placebo 0.5 mg/kg WVE-210201 1 mg/kg WVE-210201 2 mg/kg WVE-210201 5 mg/kg WVE-210201 7/10 mg/kg WVE-210201
    Number of subjects analysed
    10
    6
    6
    6
    6
    2
    Units: Subjects
    number (not applicable)
        Number of Patients who Experienced a Severe TEAE
    2
    0
    0
    0
    0
    2
    No statistical analyses for this end point

    Primary: Number of Patients who Experienced a Serious TEAE

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    End point title
    		 Number of Patients who Experienced a Serious TEAE [3]
    End point description
    An SAE was defined as any event that resulted in death, was immediately life threatening, required inpatient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, or was a congenital anomaly/birth defect not present at Screening. Important medical events that did not result in death, were life threatening, or required hospitalization were considered SAEs when, based upon appropriate medical judgment, they jeopardized the patient or required medical or surgical intervention to prevent one of the outcomes listed in this definition.
    End point type
    Primary
    End point timeframe
    Day 1 to Day 85 (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: No statistical analysis were performed
    End point values
    		 Pooled Placebo 0.5 mg/kg WVE-210201 1 mg/kg WVE-210201 2 mg/kg WVE-210201 5 mg/kg WVE-210201 7/10 mg/kg WVE-210201
    Number of subjects analysed
    10
    6
    6
    6
    6
    2
    Units: Subjects
    number (not applicable)
        Number of Patients who Experienced an SAE
    1
    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
    Day 1 to Day 85 (end of study)
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    20.1
    Reporting groups
    Reporting group title
    Pooled Placebo
    Reporting group description
    		 -

    Reporting group title
    0.5 mg/kg
    Reporting group description
    		 -

    Reporting group title
    1 mg/kg
    Reporting group description
    		 -

    Reporting group title
    2 mg/kg
    Reporting group description
    		 -

    Reporting group title
    5 mg/kg
    Reporting group description
    		 -

    Reporting group title
    7/10 mg/kg
    Reporting group description
    		 -

    Serious adverse events
    		 Pooled Placebo 0.5 mg/kg 1 mg/kg 2 mg/kg 5 mg/kg 7/10 mg/kg
    Total subjects affected by serious adverse events
         subjects affected / exposed
    1 / 10 (10.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 2 (0.00%)
         number of deaths (all causes)
    0
    0
    0
    0
    0
    0
         number of deaths resulting from adverse events
    0
    0
    0
    0
    0
    0
    Infections and infestations
    Corona virus infection
         subjects affected / exposed
    1 / 10 (10.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 2 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Influenza
         subjects affected / exposed
    1 / 10 (10.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 2 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
         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
    		 Pooled Placebo 0.5 mg/kg 1 mg/kg 2 mg/kg 5 mg/kg 7/10 mg/kg
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    8 / 10 (80.00%)
    5 / 6 (83.33%)
    5 / 6 (83.33%)
    4 / 6 (66.67%)
    4 / 6 (66.67%)
    2 / 2 (100.00%)
    General disorders and administration site conditions
    Catheter site pain
         subjects affected / exposed
    1 / 10 (10.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 2 (0.00%)
         occurrences all number
    1
    0
    0
    0
    0
    0
    Chills
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    1 / 2 (50.00%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    Fatigue
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    1 / 6 (16.67%)
    0 / 6 (0.00%)
    1 / 2 (50.00%)
         occurrences all number
    0
    0
    0
    1
    0
    1
    Influenza like illness
         subjects affected / exposed
    1 / 10 (10.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 2 (0.00%)
         occurrences all number
    1
    0
    0
    0
    0
    0
    Infusion site pain
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    1 / 6 (16.67%)
    0 / 6 (0.00%)
    0 / 2 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    0
    Peripheral swelling
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    1 / 6 (16.67%)
    0 / 6 (0.00%)
    0 / 2 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    0
    Pyrexia
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    4 / 6 (66.67%)
    2 / 2 (100.00%)
         occurrences all number
    0
    0
    0
    0
    4
    2
    Respiratory, thoracic and mediastinal disorders
    Cough
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 6 (0.00%)
    1 / 6 (16.67%)
    1 / 6 (16.67%)
    1 / 6 (16.67%)
    0 / 2 (0.00%)
         occurrences all number
    0
    0
    1
    2
    1
    0
    Epistaxis
         subjects affected / exposed
    1 / 10 (10.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 2 (0.00%)
         occurrences all number
    1
    0
    0
    0
    0
    0
    Nasal congestion
         subjects affected / exposed
    0 / 10 (0.00%)
    1 / 6 (16.67%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 2 (0.00%)
         occurrences all number
    0
    1
    0
    0
    0
    0
    Pharyngeal erythema
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    1 / 6 (16.67%)
    0 / 2 (0.00%)
         occurrences all number
    0
    0
    0
    0
    1
    0
    Tonsillar hypertrophy
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    1 / 2 (50.00%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    Investigations
    Body temperature increased
         subjects affected / exposed
    1 / 10 (10.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 2 (0.00%)
         occurrences all number
    1
    0
    0
    0
    0
    0
    C-reactive protein increased
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    1 / 2 (50.00%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    Neutrophil count increased
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 6 (0.00%)
    1 / 6 (16.67%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    1 / 2 (50.00%)
         occurrences all number
    0
    0
    1
    0
    0
    1
    Injury, poisoning and procedural complications
    Fall
         subjects affected / exposed
    1 / 10 (10.00%)
    1 / 6 (16.67%)
    0 / 6 (0.00%)
    1 / 6 (16.67%)
    0 / 6 (0.00%)
    0 / 2 (0.00%)
         occurrences all number
    1
    1
    0
    1
    0
    0
    Joint injury
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    1 / 6 (16.67%)
    0 / 2 (0.00%)
         occurrences all number
    0
    0
    0
    0
    1
    0
    Cardiac disorders
    Bradycardia
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 6 (0.00%)
    1 / 6 (16.67%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 2 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    0
    Sinus bradycardia
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 6 (0.00%)
    1 / 6 (16.67%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 2 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    0
    Tachycardia
         subjects affected / exposed
    1 / 10 (10.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    1 / 6 (16.67%)
    2 / 2 (100.00%)
         occurrences all number
    1
    0
    0
    0
    1
    2
    Nervous system disorders
    Headache
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 6 (0.00%)
    2 / 6 (33.33%)
    1 / 6 (16.67%)
    3 / 6 (50.00%)
    1 / 2 (50.00%)
         occurrences all number
    0
    0
    3
    1
    3
    1
    Tremor
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    1 / 6 (16.67%)
    0 / 2 (0.00%)
         occurrences all number
    0
    0
    0
    0
    1
    0
    Blood and lymphatic system disorders
    Leukocytosis
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    1 / 2 (50.00%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    Ear and labyrinth disorders
    Ear pain
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    1 / 2 (50.00%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    Tympanic membrane disorder
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    1 / 2 (50.00%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    Tympanic membrane hyperaemia
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    1 / 2 (50.00%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    Eye disorders
    Blepharospasm
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    1 / 6 (16.67%)
    0 / 6 (0.00%)
    0 / 2 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    0
    Gastrointestinal disorders
    Abdominal pain
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    1 / 6 (16.67%)
    0 / 6 (0.00%)
    0 / 2 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    0
    Abdominal pain upper
         subjects affected / exposed
    0 / 10 (0.00%)
    1 / 6 (16.67%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    1 / 6 (16.67%)
    0 / 2 (0.00%)
         occurrences all number
    0
    1
    0
    0
    1
    0
    Abnormal faeces
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    1 / 2 (50.00%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    Diarrhoea
         subjects affected / exposed
    1 / 10 (10.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 2 (0.00%)
         occurrences all number
    1
    0
    0
    0
    0
    0
    Eructation
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    1 / 6 (16.67%)
    0 / 6 (0.00%)
    0 / 2 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    0
    Gingival erythema
         subjects affected / exposed
    1 / 10 (10.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 2 (0.00%)
         occurrences all number
    1
    0
    0
    0
    0
    0
    Gingival swelling
         subjects affected / exposed
    1 / 10 (10.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 2 (0.00%)
         occurrences all number
    1
    0
    0
    0
    0
    0
    Nausea
         subjects affected / exposed
    0 / 10 (0.00%)
    1 / 6 (16.67%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    1 / 2 (50.00%)
         occurrences all number
    0
    1
    0
    0
    0
    1
    Vomiting
         subjects affected / exposed
    0 / 10 (0.00%)
    1 / 6 (16.67%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    2 / 6 (33.33%)
    1 / 2 (50.00%)
         occurrences all number
    0
    1
    0
    0
    3
    1
    Skin and subcutaneous tissue disorders
    Erythema
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 6 (0.00%)
    1 / 6 (16.67%)
    1 / 6 (16.67%)
    0 / 6 (0.00%)
    0 / 2 (0.00%)
         occurrences all number
    0
    0
    1
    1
    0
    0
    Hyperhidrosis
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    1 / 6 (16.67%)
    0 / 6 (0.00%)
    0 / 2 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    0
    Livedo reticularis
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    1 / 2 (50.00%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    Pruritus
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    1 / 6 (16.67%)
    0 / 6 (0.00%)
    0 / 2 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    0
    Renal and urinary disorders
    Dysuria
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 6 (0.00%)
    1 / 6 (16.67%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 2 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    0
    Musculoskeletal and connective tissue disorders
    Arthralgia
         subjects affected / exposed
    1 / 10 (10.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 2 (0.00%)
         occurrences all number
    1
    0
    0
    0
    0
    0
    Back pain
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 6 (0.00%)
    1 / 6 (16.67%)
    0 / 6 (0.00%)
    1 / 6 (16.67%)
    0 / 2 (0.00%)
         occurrences all number
    0
    0
    1
    0
    1
    0
    Pain in extremity
         subjects affected / exposed
    2 / 10 (20.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 2 (0.00%)
         occurrences all number
    2
    0
    0
    0
    0
    0
    Infections and infestations
    Bullous impetigo
         subjects affected / exposed
    0 / 10 (0.00%)
    1 / 6 (16.67%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 2 (0.00%)
         occurrences all number
    0
    1
    0
    0
    0
    0
    Ear infection
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 6 (0.00%)
    1 / 6 (16.67%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 2 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    0
    Gastroenteritis viral
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    1 / 6 (16.67%)
    0 / 6 (0.00%)
    0 / 2 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    0
    Nasopharyngitis
         subjects affected / exposed
    2 / 10 (20.00%)
    0 / 6 (0.00%)
    1 / 6 (16.67%)
    1 / 6 (16.67%)
    0 / 6 (0.00%)
    1 / 2 (50.00%)
         occurrences all number
    3
    0
    1
    1
    0
    1
    Otitis media
         subjects affected / exposed
    1 / 10 (10.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 2 (0.00%)
         occurrences all number
    1
    0
    0
    0
    0
    0
    Tooth abscess
         subjects affected / exposed
    1 / 10 (10.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 2 (0.00%)
         occurrences all number
    1
    0
    0
    0
    0
    0
    Upper respiratory tract infection
         subjects affected / exposed
    1 / 10 (10.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 2 (0.00%)
         occurrences all number
    1
    0
    0
    0
    0
    0
    Viral upper respiratory tract infection
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    1 / 6 (16.67%)
    0 / 2 (0.00%)
         occurrences all number
    0
    0
    0
    0
    1
    0
    Metabolism and nutrition disorders
    Decreased appetite
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 6 (0.00%)
    1 / 6 (16.67%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 2 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    0

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    31 May 2018
    - Information on the new WVE-210201 liquid formulation was provided - GGT was specified in the clinical laboratory panel for evaluation as a potential marker of changes in liver function in patients with DMD - Administrative language regarding withdrawal, discontinuations, un-blinding, randomization, and study termination was clarified - Language regarding reconsent once patients reach legal age was added

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

    Limitations and caveats
    Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data.
    None reported
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    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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