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    Clinical Trial Results:
    An Open-Label, Multi-Center Study to Evaluate the Safety, Efficacy and Tolerability of Eteplirsen in Early Stage Duchenne Muscular Dystrophy

    Summary
    EudraCT number
    		 2016-005023-92
    Trial protocol
    		 Outside EU/EEA  
    Global end of trial date
    17 Dec 2018

    Results information
    Results version number
    		 v2(current)
    This version publication date
    16 Jan 2021
    First version publication date
    22 Sep 2019
    Other versions
    		 v1
    Version creation reason
    • New data added to full data set
    we need to incorporate secondary outcome measures

    Trial information

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    Trial identification
    Sponsor protocol code
    4658-203
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT02420379
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Sarepta Therapeutics, Inc.
    Sponsor organisation address
    215 First Street, Cambridge, United States, MA, 02142
    Public contact
    Medical Director, Sarepta Therapeutics, Inc., +1 617274 4000, clinicaltrials@sarepta.com
    Scientific contact
    Medical Director, Sarepta Therapeutics, Inc., +1 617274 4000, clinicaltrials@sarepta.com
    Paediatric regulatory details
    Is trial part of an agreed paediatric investigation plan (PIP)
    Yes
    EMA paediatric investigation plan number(s)
    		 EMEA-001722-PIP01-14
    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
    30 May 2019
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    17 Dec 2018
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    To evaluate the safety and tolerability of eteplirsen in subjects with Duchenne muscular dystrophy (DMD) between 4 and 6 years of age who are amenable to exon 51 skipping.
    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.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    30 Jun 2015
    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
    United States: 33
    Worldwide total number of subjects
    33
    EEA total number of subjects
    0
    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)
    33
    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
    		 The study was conducted at 13 sites in the United States.

    Pre-assignment
    Screening details
    		 A total of 33 subjects were enrolled in the study treatment.

    Period 1
    Period 1 title
    Overall Study (overall period)
    Is this the baseline period?
    		 Yes
    Allocation method
    Non-randomised - controlled
    Blinding used
    		 Not blinded

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Eteplirsen 30 mg/kg
    Arm description
    		 Subjects with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping received eteplirsen 30 milligram per kilogram (mg/kg) intravenous (IV) infusions, once weekly, for 96 weeks.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Eteplirsen
    Investigational medicinal product code
    Other name
    EXONDYS 51™®
    Pharmaceutical forms
    Infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    Subjects received eteplirsen 30 mg/kg IV infusion once weekly.

    Arm title
    		 Control Group (Untreated) (Non-exon 51 Amenable Subjects)
    Arm description
    		 Subjects with DMD not amenable to exon 51 skipping were observed for 96 weeks.
    Arm type
    		 No intervention

    Investigational medicinal product name
    No investigational medicinal product assigned in this arm
    Number of subjects in period 1
    		Eteplirsen 30 mg/kg Control Group (Untreated) (Non-exon 51 Amenable Subjects)
    Started
    26
    7
    Completed
    25
    3
    Not completed
    1
    4
         Consent withdrawn by subject
    -
    3
         Subject was unable to complete due to schooling
    -
    1
         Subject transitioned to commercial drug
    1
    -

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Eteplirsen 30 mg/kg
    Reporting group description
    		 Subjects with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping received eteplirsen 30 milligram per kilogram (mg/kg) intravenous (IV) infusions, once weekly, for 96 weeks.

    Reporting group title
    Control Group (Untreated) (Non-exon 51 Amenable Subjects)
    Reporting group description
    		 Subjects with DMD not amenable to exon 51 skipping were observed for 96 weeks.

    Reporting group values
    		 Eteplirsen 30 mg/kg Control Group (Untreated) (Non-exon 51 Amenable Subjects) Total
    Number of subjects
    		 26 7 33
    Age categorical
    Units: Subjects
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    		 5.0 ( 0.82 ) 5.0 ( 1.00 ) -
    Gender categorical
    Units: Subjects
        Female
    		 0 0 0
        Male
    		 26 7 33
    Race
    Units: Subjects
        American Indian or Alaska Native
    		 0 0 0
        Asian
    		 2 1 3
        Black
    		 1 0 1
        Native Hawaiian or Other Pacific Islander
    		 0 0 0
        White
    		 22 6 28
        Other
    		 1 0 1
    Ethnicity
    Units: Subjects
        Hispanic or Latino
    		 5 0 5
        Not Hispanic or Latino
    		 21 7 28

    End points

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    End points reporting groups
    Reporting group title
    Eteplirsen 30 mg/kg
    Reporting group description
    		 Subjects with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping received eteplirsen 30 milligram per kilogram (mg/kg) intravenous (IV) infusions, once weekly, for 96 weeks.

    Reporting group title
    Control Group (Untreated) (Non-exon 51 Amenable Subjects)
    Reporting group description
    		 Subjects with DMD not amenable to exon 51 skipping were observed for 96 weeks.

    Primary: Number of Subjects with Treatment Emergent Adverse Events (TEAEs), Serious TEAEs and TEAEs Leading to Discontinuation

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    End point title
    		 Number of Subjects with Treatment Emergent Adverse Events (TEAEs), Serious TEAEs and TEAEs Leading to Discontinuation [1]
    End point description
    AE was any untoward medical occurrence in a subject that did not necessarily have a causal relationship with the study drug. Serious adverse event (SAE) was an AE resulting in any of the following outcomes: death; Life-threatening event; Required or prolonged inpatient hospitalization; persistent or significant disability/incapacity; congenital anomaly. Treatment-emergent adverse events were events that developed or worsened during the on-treatment period (defined as time from first dose of study drug and up to 28 days after last dose of study drug [up to 100 weeks]) that were absent before treatment or that worsened relative to pre-treatment state. AEs included both serious and non-serious adverse events. Full set includes all subjects who are enrolled in the eteplirsen group and received at least 1 dose of eteplirsen as well as all subjects who were enrolled in the untreated group and had at least 1 assessment post-enrollment assessment.
    End point type
    Primary
    End point timeframe
    Baseline up to 100 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 testing was performed for the primary endpoint.
    End point values
    		 Eteplirsen 30 mg/kg Control Group (Untreated) (Non-exon 51 Amenable Subjects)
    Number of subjects analysed
    26
    7
    Units: subjects
        Subjects with TEAEs
    26
    5
        Subjects with Serious TEAEs
    4
    0
        Subjects with TEAEs leading to discontinuation
    0
    0
    No statistical analyses for this end point

    Primary: Number of Subjects With Potentially Clinically Significant Laboratory Abnormalities Reported as TEAEs

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    End point title
    		 Number of Subjects With Potentially Clinically Significant Laboratory Abnormalities Reported as TEAEs [2]
    End point description
    Laboratory parameters included hematology, serum chemistry (SC), urinalysis and coagulation. Number of subjects with at least one potentially clinically significant abnormal findings were reported as TEAEs. The Investigator determined whether abnormal assessment results were clinically significant or not clinically significant. Clinical significance was defined as any variation in assessment results that had medical relevance resulting in an alteration in medical care. Full analysis set includes all subjects who are enrolled in the eteplirsen group and receive at least 1 dose of eteplirsen as well as all subjects who are enrolled in the untreated group who have at least 1 assessment post-enrollment assessment.
    End point type
    Primary
    End point timeframe
    Baseline up to 100 weeks
    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 testing was performed for the primary endpoint.
    End point values
    		 Eteplirsen 30 mg/kg Control Group (Untreated) (Non-exon 51 Amenable Subjects)
    Number of subjects analysed
    26
    7
    Units: Subjects
        SC: Blood creatine phosphokinase increased
    1
    0
        Hematology: Anaemia
    1
    0
        Hematology: Iron deficiency Anaemia
    1
    0
        Urinalysis: chromaturia
    3
    0
        Urinalysis: pollakiuria
    1
    0
        Coagulation: Thrombocytopenia
    0
    0
    No statistical analyses for this end point

    Primary: Number of Subjects With Potentially Clinically Significant Abnormalities in Vital Signs Reported as TEAEs

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    End point title
    		 Number of Subjects With Potentially Clinically Significant Abnormalities in Vital Signs Reported as TEAEs [3]
    End point description
    Vital sign parameters included systolic blood pressure (SBP), diastolic blood pressure (DBP), heart rate (HR), and body temperature. Number of subjects with at least one potentially clinically significant abnormal vital sign findings were reported as TEAEs. The Investigator determined whether abnormal assessment results were clinically significant or not clinically significant. Clinical significance was defined as any variation in assessment results that had medical relevance resulting in an alteration in medical care. Full analysis set includes all subjects who are enrolled in the eteplirsen group and receive at least 1 dose of eteplirsen as well as all subjects who are enrolled in the untreated group who have at least 1 assessment post-enrollment assessment.
    End point type
    Primary
    End point timeframe
    Baseline up to 100 weeks
    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 testing was performed for the primary endpoint.
    End point values
    		 Eteplirsen 30 mg/kg Control Group (Untreated) (Non-exon 51 Amenable Subjects)
    Number of subjects analysed
    26
    7
    Units: Subjects
        Pyrexia
    11
    1
        Pulse pressure increased
    1
    0
        Tachycardia
    1
    0
    No statistical analyses for this end point

    Primary: Number of Subjects With at Least One Abnormal Physical Examination Finding

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    End point title
    		 Number of Subjects With at Least One Abnormal Physical Examination Finding [4]
    End point description
    Physical examinations, full and brief, were performed by the Investigator, a physician Sub-Investigator, or a Nurse Practitioner (if licensed in the state or province to perform physical examinations). Full physical examinations included examination of general appearance; head, ears, eyes, nose, and throat; heart; lungs; chest; abdomen; skin; lymph nodes; and musculoskeletal and neurological systems. Number of subjects with at least one abnormal physical examination finding was reported. Abnormality in physical examinations was based on Investigator’s discretion. Full analysis set includes all subjects who are enrolled in the eteplirsen group and receive at least 1 dose of eteplirsen as well as all subjects who are enrolled in the untreated group who have at least 1 assessment post-enrollment assessment.
    End point type
    Primary
    End point timeframe
    Baseline up to 100 weeks
    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: No statistical testing was performed for the primary endpoint.
    End point values
    		 Eteplirsen 30 mg/kg Control Group (Untreated) (Non-exon 51 Amenable Subjects)
    Number of subjects analysed
    26
    7
    Units: Subjects
    26
    7
    No statistical analyses for this end point

    Primary: Number of Subjects With Abnormalities in Electrocardiograms (ECGs) Reported as TEAEs

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    End point title
    		 Number of Subjects With Abnormalities in Electrocardiograms (ECGs) Reported as TEAEs [5]
    End point description
    Twelve-lead ECGs and Holter ECGs were performed at a consistent time of day throughout the study. Electrocardiograms were performed only after the subject was in the supine position, resting, and quiet for a minimum of 15 minutes. The ECG was manually reviewed and interpreted by medically qualified personnel. Number of Subjects with at least one abnormalities in ECGs were reported as TEAEs. Full analysis set includes all subjects who are enrolled in the eteplirsen group and receive at least 1 dose of eteplirsen as well as all subjects who are enrolled in the untreated group who have at 1 assessment post-enrollment assessment.
    End point type
    Primary
    End point timeframe
    Baseline up to 96 weeks
    Notes
    [5] - 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 testing was performed for the primary endpoint.
    End point values
    		 Eteplirsen 30 mg/kg Control Group (Untreated) (Non-exon 51 Amenable Subjects)
    Number of subjects analysed
    26
    7
    Units: Subjects
    1
    0
    No statistical analyses for this end point

    Primary: Number of Subjects With Abnormalities in Echocardiograms (ECHO) Reported as TEAEs

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    End point title
    		 Number of Subjects With Abnormalities in Echocardiograms (ECHO) Reported as TEAEs [6]
    End point description
    Standard, 2-dimensional ECHOs were performed at a consistent time of day throughout the study. Cardiac function events included cardiomegaly, tachycardia, and dyspnoea. The ECHO was reviewed and interpreted by medically qualified personnel. Number of subjects with at least one abnormalities in ECHO were reported as TEAEs. Full analysis set includes all subjects who are enrolled in the eteplirsen group and receive at least 1 dose of eteplirsen as well as all subjects who are enrolled in the untreated group who have at least 1 assessment post-enrollment assessment.
    End point type
    Primary
    End point timeframe
    Baseline up to 96 weeks
    Notes
    [6] - 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 testing was performed for the primary endpoint.
    End point values
    		 Eteplirsen 30 mg/kg Control Group (Untreated) (Non-exon 51 Amenable Subjects)
    Number of subjects analysed
    26
    7
    Units: Subjects
    1
    0
    No statistical analyses for this end point

    Secondary: Change From Baseline in Dystrophin Protein Levels Quantified by Western Blot at Week 48 and 96

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    End point title
    		 Change From Baseline in Dystrophin Protein Levels Quantified by Western Blot at Week 48 and 96 [7]
    End point description
    Change from baseline in dystrophin protein levels (in muscle biopsy samples) were determined by Western blot at Week 48 and 96 was reported. For each time point, 2 blocks of tissues were analyzed by Western blot, each with 2 replicates of gels to determine the dystrophin level as compared to a healthy individual (Percent Normal). The block average value from 2 replicate gels was computed. The overall average was calculated as the mean of the block average values. The overall average values were used for all analyses. In case only 1 gel was available for a block, then that value was used as the block average value. Muscle Biopsy Set included all subjects who received at least 1 dose of eteplirsen and who had data from both baseline (pre-treatment) and Week 48 or 96 (on-treatment) muscle biopsy samples. Here, "number of subjects analysed" signifies number of subjects who were evaluable for this endpoint.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 48 and 96
    Notes
    [7] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: Data for this endpoint was not planned to be collected and analysed for control group (untreated) (non-exon 51 amenable subjects).
    End point values
    		 Eteplirsen 30 mg/kg
    Number of subjects analysed
    25
    Units: Percent Normal Dystrophin Protein Level
    arithmetic mean (standard deviation)
        Change at Week 48 (n=14)
    0.102 ( 0.0896 )
        Change at Week 96 (n=11)
    0.321 ( 0.4863 )
    No statistical analyses for this end point

    Secondary: Change From Baseline in Dystrophin Intensity Levels Determined by Immunohistochemistry (IHC) at Week 48 and 96

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    End point title
    		 Change From Baseline in Dystrophin Intensity Levels Determined by Immunohistochemistry (IHC) at Week 48 and 96 [8]
    End point description
    Change from baseline in dystrophin intensity levels (in muscle biopsy samples) was determined by Immunohistochemistry at Week 48 and 96 was reported. Muscle Biopsy Set included all subjects who received at least 1 dose of eteplirsen and who had data from both baseline (pre-treatment) and Week 48 or 96 (on-treatment) muscle biopsy samples. Data for this outcome was not planned to be collected and analyzed for control group (untreated) (non-exon 51 amenable subjects). Here, "number of subjects analysed" signifies number of subjects who were evaluable for this endpoint.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 48 and 96
    Notes
    [8] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: Data for this endpoint was not planned to be collected and analysed for control group (untreated) (non-exon 51 amenable subjects).
    End point values
    		 Eteplirsen 30 mg/kg
    Number of subjects analysed
    25
    Units: Percent dystrophin positive fibers
    arithmetic mean (standard deviation)
        Change at Week 48 (n=14)
    0.004 ( 0.0096 )
        Change at Week 96 (n=11)
    0.015 ( 0.0175 )
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Baseline up to 100 weeks
    Adverse event reporting additional description
    Safety population included all subjects who received at least 1 dose of eteplirsen and Control group.
    Assessment type
    		 Non-systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    17.1
    Reporting groups
    Reporting group title
    Eteplirsen 30 mg/kg
    Reporting group description
    		 Subjects received eteplirsen 30 mg/kg IV infusions, weekly, for 96 weeks.

    Reporting group title
    Control Group (Untreated) (Non-exon 51 Amenable Subjects)
    Reporting group description
    		 Subjects with Duchenne muscular dystrophy (DMD) not amenable to exon 51 skipping was observed for 96 weeks.

    Serious adverse events
    		 Eteplirsen 30 mg/kg Control Group (Untreated) (Non-exon 51 Amenable Subjects)
    Total subjects affected by serious adverse events
         subjects affected / exposed
    4 / 26 (15.38%)
    0 / 7 (0.00%)
         number of deaths (all causes)
    0
    0
         number of deaths resulting from adverse events
    0
    0
    Injury, poisoning and procedural complications
    Foreign body
         subjects affected / exposed
    1 / 26 (3.85%)
    0 / 7 (0.00%)
         occurrences causally related to treatment / all
    1 / 4
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Musculoskeletal and connective tissue disorders
    Rhabdomyolysis
         subjects affected / exposed
    1 / 26 (3.85%)
    0 / 7 (0.00%)
         occurrences causally related to treatment / all
    1 / 4
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Infections and infestations
    Coxsackie viral infection
         subjects affected / exposed
    1 / 26 (3.85%)
    0 / 7 (0.00%)
         occurrences causally related to treatment / all
    1 / 4
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Influenza
         subjects affected / exposed
    1 / 26 (3.85%)
    0 / 7 (0.00%)
         occurrences causally related to treatment / all
    1 / 4
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Rhinovirus infection
         subjects affected / exposed
    1 / 26 (3.85%)
    0 / 7 (0.00%)
         occurrences causally related to treatment / all
    1 / 4
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 Eteplirsen 30 mg/kg Control Group (Untreated) (Non-exon 51 Amenable Subjects)
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    26 / 26 (100.00%)
    5 / 7 (71.43%)
    Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    Skin papilloma
         subjects affected / exposed
    2 / 26 (7.69%)
    0 / 7 (0.00%)
         occurrences all number
    2
    0
    Surgical and medical procedures
    Adenoidectomy
         subjects affected / exposed
    0 / 26 (0.00%)
    1 / 7 (14.29%)
         occurrences all number
    0
    1
    Myringotomy
         subjects affected / exposed
    0 / 26 (0.00%)
    1 / 7 (14.29%)
         occurrences all number
    0
    1
    Orchidopexy
         subjects affected / exposed
    0 / 26 (0.00%)
    1 / 7 (14.29%)
         occurrences all number
    0
    1
    General disorders and administration site conditions
    Pyrexia
         subjects affected / exposed
    11 / 26 (42.31%)
    1 / 7 (14.29%)
         occurrences all number
    22
    1
    Catheter site pain
         subjects affected / exposed
    3 / 26 (11.54%)
    0 / 7 (0.00%)
         occurrences all number
    3
    0
    Catheter site bruise
         subjects affected / exposed
    2 / 26 (7.69%)
    0 / 7 (0.00%)
         occurrences all number
    2
    0
    Immune system disorders
    Seasonal allergy
         subjects affected / exposed
    3 / 26 (11.54%)
    0 / 7 (0.00%)
         occurrences all number
    7
    0
    Hypersensitivity
         subjects affected / exposed
    0 / 26 (0.00%)
    1 / 7 (14.29%)
         occurrences all number
    0
    1
    Respiratory, thoracic and mediastinal disorders
    Cough
         subjects affected / exposed
    23 / 26 (88.46%)
    3 / 7 (42.86%)
         occurrences all number
    57
    3
    Rhinorrhoea
         subjects affected / exposed
    13 / 26 (50.00%)
    0 / 7 (0.00%)
         occurrences all number
    18
    0
    Nasal congestion
         subjects affected / exposed
    10 / 26 (38.46%)
    0 / 7 (0.00%)
         occurrences all number
    18
    0
    Epistaxis
         subjects affected / exposed
    5 / 26 (19.23%)
    0 / 7 (0.00%)
         occurrences all number
    5
    0
    Oropharyngeal pain
         subjects affected / exposed
    4 / 26 (15.38%)
    0 / 7 (0.00%)
         occurrences all number
    5
    0
    Productive cough
         subjects affected / exposed
    2 / 26 (7.69%)
    0 / 7 (0.00%)
         occurrences all number
    3
    0
    Upper respiratory tract congestion
         subjects affected / exposed
    2 / 26 (7.69%)
    0 / 7 (0.00%)
         occurrences all number
    3
    0
    Psychiatric disorders
    Aggression
         subjects affected / exposed
    2 / 26 (7.69%)
    0 / 7 (0.00%)
         occurrences all number
    2
    0
    Investigations
    Cardiac murmur
         subjects affected / exposed
    4 / 26 (15.38%)
    0 / 7 (0.00%)
         occurrences all number
    5
    0
    Injury, poisoning and procedural complications
    Fall
         subjects affected / exposed
    9 / 26 (34.62%)
    0 / 7 (0.00%)
         occurrences all number
    37
    0
    Contusion
         subjects affected / exposed
    5 / 26 (19.23%)
    0 / 7 (0.00%)
         occurrences all number
    6
    0
    Procedural pain
         subjects affected / exposed
    4 / 26 (15.38%)
    0 / 7 (0.00%)
         occurrences all number
    5
    0
    Skin abrasion
         subjects affected / exposed
    4 / 26 (15.38%)
    0 / 7 (0.00%)
         occurrences all number
    4
    0
    Scratch
         subjects affected / exposed
    3 / 26 (11.54%)
    0 / 7 (0.00%)
         occurrences all number
    3
    0
    Head injury
         subjects affected / exposed
    2 / 26 (7.69%)
    0 / 7 (0.00%)
         occurrences all number
    3
    0
    Sunburn
         subjects affected / exposed
    2 / 26 (7.69%)
    0 / 7 (0.00%)
         occurrences all number
    2
    0
    Ligament sprain
         subjects affected / exposed
    0 / 26 (0.00%)
    1 / 7 (14.29%)
         occurrences all number
    0
    1
    Nervous system disorders
    Headache
         subjects affected / exposed
    10 / 26 (38.46%)
    0 / 7 (0.00%)
         occurrences all number
    16
    0
    Migraine
         subjects affected / exposed
    2 / 26 (7.69%)
    0 / 7 (0.00%)
         occurrences all number
    3
    0
    Ear and labyrinth disorders
    Ear pain
         subjects affected / exposed
    7 / 26 (26.92%)
    0 / 7 (0.00%)
         occurrences all number
    9
    0
    Gastrointestinal disorders
    Vomiting
         subjects affected / exposed
    11 / 26 (42.31%)
    2 / 7 (28.57%)
         occurrences all number
    18
    3
    Diarrhoea
         subjects affected / exposed
    7 / 26 (26.92%)
    1 / 7 (14.29%)
         occurrences all number
    10
    1
    Abdominal pain
         subjects affected / exposed
    5 / 26 (19.23%)
    0 / 7 (0.00%)
         occurrences all number
    5
    0
    Abdominal pain upper
         subjects affected / exposed
    5 / 26 (19.23%)
    0 / 7 (0.00%)
         occurrences all number
    12
    0
    Constipation
         subjects affected / exposed
    3 / 26 (11.54%)
    1 / 7 (14.29%)
         occurrences all number
    4
    1
    Nausea
         subjects affected / exposed
    2 / 26 (7.69%)
    1 / 7 (14.29%)
         occurrences all number
    2
    1
    Dental caries
         subjects affected / exposed
    2 / 26 (7.69%)
    0 / 7 (0.00%)
         occurrences all number
    2
    0
    Skin and subcutaneous tissue disorders
    Rash
         subjects affected / exposed
    13 / 26 (50.00%)
    0 / 7 (0.00%)
         occurrences all number
    20
    0
    Dermatitis contact
         subjects affected / exposed
    3 / 26 (11.54%)
    0 / 7 (0.00%)
         occurrences all number
    3
    0
    Keloid scar
         subjects affected / exposed
    2 / 26 (7.69%)
    0 / 7 (0.00%)
         occurrences all number
    2
    0
    Rash pruritic
         subjects affected / exposed
    2 / 26 (7.69%)
    0 / 7 (0.00%)
         occurrences all number
    2
    0
    Renal and urinary disorders
    Chromaturia
         subjects affected / exposed
    3 / 26 (11.54%)
    0 / 7 (0.00%)
         occurrences all number
    3
    0
    Musculoskeletal and connective tissue disorders
    Pain in extremity
         subjects affected / exposed
    8 / 26 (30.77%)
    0 / 7 (0.00%)
         occurrences all number
    18
    0
    Arthralgia
         subjects affected / exposed
    6 / 26 (23.08%)
    0 / 7 (0.00%)
         occurrences all number
    9
    0
    Back pain
         subjects affected / exposed
    4 / 26 (15.38%)
    0 / 7 (0.00%)
         occurrences all number
    4
    0
    Muscle spasms
         subjects affected / exposed
    4 / 26 (15.38%)
    0 / 7 (0.00%)
         occurrences all number
    5
    0
    Muscular weakness
         subjects affected / exposed
    4 / 26 (15.38%)
    0 / 7 (0.00%)
         occurrences all number
    4
    0
    Musculoskeletal pain
         subjects affected / exposed
    2 / 26 (7.69%)
    0 / 7 (0.00%)
         occurrences all number
    2
    0
    Scoliosis
         subjects affected / exposed
    0 / 26 (0.00%)
    1 / 7 (14.29%)
         occurrences all number
    0
    1
    Infections and infestations
    Nasopharyngitis
         subjects affected / exposed
    16 / 26 (61.54%)
    2 / 7 (28.57%)
         occurrences all number
    27
    2
    Upper respiratory tract infection
         subjects affected / exposed
    11 / 26 (42.31%)
    1 / 7 (14.29%)
         occurrences all number
    20
    1
    Ear infection
         subjects affected / exposed
    8 / 26 (30.77%)
    1 / 7 (14.29%)
         occurrences all number
    15
    1
    Influenza
         subjects affected / exposed
    5 / 26 (19.23%)
    0 / 7 (0.00%)
         occurrences all number
    7
    0
    Otitis media
         subjects affected / exposed
    2 / 26 (7.69%)
    1 / 7 (14.29%)
         occurrences all number
    2
    2
    Pharyngitis streptococcal
         subjects affected / exposed
    3 / 26 (11.54%)
    0 / 7 (0.00%)
         occurrences all number
    3
    0
    Rhinitis
         subjects affected / exposed
    3 / 26 (11.54%)
    0 / 7 (0.00%)
         occurrences all number
    10
    0
    Conjunctivitis
         subjects affected / exposed
    2 / 26 (7.69%)
    0 / 7 (0.00%)
         occurrences all number
    3
    0
    Gastroenteritis viral
         subjects affected / exposed
    2 / 26 (7.69%)
    0 / 7 (0.00%)
         occurrences all number
    2
    0
    Staphylococcal infection
         subjects affected / exposed
    2 / 26 (7.69%)
    0 / 7 (0.00%)
         occurrences all number
    2
    0
    Impetigo
         subjects affected / exposed
    0 / 26 (0.00%)
    1 / 7 (14.29%)
         occurrences all number
    0
    1
    Metabolism and nutrition disorders
    Decreased appetite
         subjects affected / exposed
    2 / 26 (7.69%)
    0 / 7 (0.00%)
         occurrences all number
    2
    0

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    20 Jan 2015
    Amendment 1: Added “eteplirsen-treated” to concepts and procedures relating to treated subjects versus untreated subjects; Created new schedule of events table for untreated subjects; Revised language for vital signs time points with regard to reducing the number of collections after 1 year of treatment if no infusion reaction occurred; also added language for the untreated group; Updated AE section to explain follow-up and resolution of AE; Revised overall number of subjects for trial selection.
    08 Jun 2017
    Amendment 2: Adjusted the order of endpoints and associated objectives due to recent guidance that dystrophin quantification by Western blot was preferred over assessments using immunohistochemistry; Updated the number of subjects to allow the untreated group to enroll less than 20 subjects; Added that subjects who discontinued eteplirsen dosing at Week 96 and transitioned to commercial drug did not need to complete the end-of-trial visit assessments; Clarified that the definition of analysis populations other than the safety population were provided in the Statistical Analysis Plan.

    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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