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    Clinical Trial Results:
    An Open-Label Safety, Tolerability, and Pharmacokinetics Study of Eteplirsen in Young Patients with Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping

    Summary
    EudraCT number
    		 2016-000951-29
    Trial protocol
    		 GB   BE   DE   FR   IT  
    Global end of trial date
    10 Mar 2021

    Results information
    Results version number
    		 v1(current)
    This version publication date
    25 Sep 2021
    First version publication date
    25 Sep 2021
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    4658-102
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT03218995
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Sarepta Therapeutics, Inc.
    Sponsor organisation address
    215 First Street, Cambridge, United States, 02142
    Public contact
    Medical Director, Sarepta Therapeutics, Inc., 1 800-690-2003, clinicaltrials@sarepta.com
    Scientific contact
    Medical Director, Sarepta Therapeutics, Inc., 1 800-690-2003, 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?
    Yes
    Results analysis stage
    Analysis stage
    Final
    Date of interim/final analysis
    20 May 2021
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    10 Mar 2021
    Global end of trial reached?
    Yes
    Global end of trial date
    10 Mar 2021
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    The overall purpose of the study was to evaluate the safety and tolerability of eteplirsen in participants with Duchenne muscular dystrophy (DMD) aged 6 to 48 months.
    Protection of trial subjects
    This study was conducted in accordance with the final study protocol and its amendments, Sponsor procedures, which comply with the ethical principles of Good Clinical Practice (GCP), and the Declaration of Helsinki.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    16 Aug 2017
    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
    United Kingdom: 6
    Country: Number of subjects enrolled
    France: 5
    Country: Number of subjects enrolled
    Italy: 2
    Country: Number of subjects enrolled
    Belgium: 2
    Worldwide total number of subjects
    15
    EEA total number of subjects
    9
    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)
    6
    Children (2-11 years)
    9
    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
    		 -

    Pre-assignment
    Screening details
    		 Participants with genotypically confirmed Duchenne muscular dystrophy (DMD) featuring a deletion mutation amenable to exon 51 skipping were enrolled into 2 cohorts based on their age.

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

    Arms
    Arm title
    		 Eteplirsen
    Arm description
    		 Eteplirsen was administered once every 7 days by intravenous (IV) infusion starting on Day 1 for up to 96 weeks. The starting dose was 2 milligrams/kilogram (mg/kg) eteplirsen, with escalation to 4, 10, 20, and 30 mg/kg for 10 weeks, and then participants continued to receive eteplirsen at 30 mg/kg for the duration of the study.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Eteplirsen
    Investigational medicinal product code
    Other name
    AVI-4658; EXONDYS 51®
    Pharmaceutical forms
    Infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    Eteplirsen was administered per schedule specified in the arm description.

    Number of subjects in period 1
    		Eteplirsen
    Started
    15
    Received at least 1 dose of study drug
    15
    Completed
    15

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Overall Study
    Reporting group description
    		 Eteplirsen was administered once every 7 days by IV infusion starting on Day 1 for up to 96 weeks. The starting dose was 2 mg/kg eteplirsen, with escalation to 4, 10, 20, and 30 mg/kg for 10 weeks, and then participants continued to receive eteplirsen at 30 mg/kg for the duration of the study.

    Reporting group values
    		 Overall Study Total
    Number of subjects
    		 15 15
    Age Categorical
    Units: Subjects
        In utero
    		 0 0
        Preterm newborn infants (gestational age < 37 wks)
    		 0 0
        Newborns (0-27 days)
    		 0 0
        Infants and toddlers (28 days-23 months)
    		 6 6
        Children (2-11 years)
    		 9 9
        Adolescents (12-17 years)
    		 0 0
        Adults (18-64 years)
    		 0 0
        From 65-84 years
    		 0 0
        85 years and over
    		 0 0
    Age Continuous
    Units: months
        arithmetic mean (standard deviation)
    		 28.5 ± 12.94 -
    Gender Categorical
    Units: Subjects
        Female
    		 0 0
        Male
    		 15 15
    Race
    Race data are reported as "Missing" for participants from France because French sites and the French Regulations prohibit the entry of race and ethnicity.
    Units: Subjects
        White
    		 9 9
        Other
    		 1 1
        Missing
    		 5 5
    Ethnicity
    Ethnicity data are reported as "Missing" for participants from France because French sites and the French Regulations prohibit the entry of race and ethnicity.
    Units: Subjects
        Not Hispanic or Latino
    		 7 7
        Not reported
    		 2 2
        Unknown
    		 1 1
        Missing
    		 5 5
    Subject analysis sets

    Subject analysis set title
    Cohort 1: Age 24 to 48 Months
    Subject analysis set type
    		 Sub-group analysis
    Subject analysis set description
    Participants aged 24 to 48 months old were administered eteplirsen once every 7 days by IV infusion starting on Day 1 for up to 96 weeks. The starting dose was 2 mg/kg eteplirsen, with escalation to 4, 10, 20, and 30 mg/kg for 10 weeks, and then participants continued to receive eteplirsen at 30 mg/kg for the duration of the study.

    Subject analysis set title
    Cohort 2: Age 6 to <24 Months
    Subject analysis set type
    		 Sub-group analysis
    Subject analysis set description
    Participants aged 6 to <24 months old were administered eteplirsen once every 7 days by IV infusion starting on Day 1 for up to 96 weeks. The starting dose was 2 mg/kg eteplirsen, with escalation to 4, 10, 20, and 30 mg/kg for 10 weeks, and then participants continued to receive eteplirsen at 30 mg/kg for the duration of the study.

    Subject analysis sets values
    		 Cohort 1: Age 24 to 48 Months Cohort 2: Age 6 to <24 Months
    Number of subjects
    9
    6
    Age Categorical
    Units: Subjects
        In utero
    0
    0
        Preterm newborn infants (gestational age < 37 wks)
    0
    0
        Newborns (0-27 days)
    0
    0
        Infants and toddlers (28 days-23 months)
    0
    6
        Children (2-11 years)
    9
    0
        Adolescents (12-17 years)
    0
    0
        Adults (18-64 years)
    0
    0
        From 65-84 years
    0
    0
        85 years and over
    0
    0
    Age Continuous
    Units: months
        arithmetic mean (standard deviation)
    36.8 ± 8.21
    16.0 ± 7.07
    Gender Categorical
    Units: Subjects
        Female
    0
    0
        Male
    9
    6
    Race
    Race data are reported as "Missing" for participants from France because French sites and the French Regulations prohibit the entry of race and ethnicity.
    Units: Subjects
        White
    6
    3
        Other
    0
    1
        Missing
    3
    2
    Ethnicity
    Ethnicity data are reported as "Missing" for participants from France because French sites and the French Regulations prohibit the entry of race and ethnicity.
    Units: Subjects
        Not Hispanic or Latino
    4
    3
        Not reported
    2
    0
        Unknown
    0
    1
        Missing
    3
    2

    End points

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    End points reporting groups
    Reporting group title
    Eteplirsen
    Reporting group description
    		 Eteplirsen was administered once every 7 days by intravenous (IV) infusion starting on Day 1 for up to 96 weeks. The starting dose was 2 milligrams/kilogram (mg/kg) eteplirsen, with escalation to 4, 10, 20, and 30 mg/kg for 10 weeks, and then participants continued to receive eteplirsen at 30 mg/kg for the duration of the study.

    Subject analysis set title
    Cohort 1: Age 24 to 48 Months
    Subject analysis set type
    		 Sub-group analysis
    Subject analysis set description
    Participants aged 24 to 48 months old were administered eteplirsen once every 7 days by IV infusion starting on Day 1 for up to 96 weeks. The starting dose was 2 mg/kg eteplirsen, with escalation to 4, 10, 20, and 30 mg/kg for 10 weeks, and then participants continued to receive eteplirsen at 30 mg/kg for the duration of the study.

    Subject analysis set title
    Cohort 2: Age 6 to <24 Months
    Subject analysis set type
    		 Sub-group analysis
    Subject analysis set description
    Participants aged 6 to <24 months old were administered eteplirsen once every 7 days by IV infusion starting on Day 1 for up to 96 weeks. The starting dose was 2 mg/kg eteplirsen, with escalation to 4, 10, 20, and 30 mg/kg for 10 weeks, and then participants continued to receive eteplirsen at 30 mg/kg for the duration of the study.

    Primary: Number of Participants with Treatment Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), and TEAEs Leading to Discontinuation from Study Drug

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    End point title
    		 Number of Participants with Treatment Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), and TEAEs Leading to Discontinuation from Study Drug [1]
    End point description
    TEAEs were defined as adverse events (AEs) with an onset following administration of the first dose of study drug. An AE was any untoward medical occurrence in a participant, which does not necessarily have a causal relationship with the study drug. Abnormalities presented at Baseline were considered AEs if they reoccurred after resolution or worsen during the AE collection period. An SAE was defined as any AE that, in the view of either the Investigator or Sponsor, resulted in any of the following outcomes as fatal, life-threatening, required hospitalization or prolongation of existing hospitalization, persistent or significant disability/incapacity, a congenital anomaly/birth defect, or an important medical event. A summary of all SAEs and Other AEs (nonserious) regardless of causality is located in the ‘Adverse Events’ Section. The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
    End point type
    Primary
    End point timeframe
    Baseline up to Week 100
    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: Analysis were descriptive in nature.
    End point values
    		 Cohort 1: Age 24 to 48 Months Cohort 2: Age 6 to <24 Months
    Number of subjects analysed
    9
    6
    Units: participants
        TEAE
    9
    6
        SAE
    0
    1
        AE leading to discontinuation from study drug
    0
    0
    No statistical analyses for this end point

    Primary: Number of Participants with at Least 1 Potentially Clinically Significant Clinical Safety Laboratory Abnormality

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    End point title
    		 Number of Participants with at Least 1 Potentially Clinically Significant Clinical Safety Laboratory Abnormality [2]
    End point description
    Clinical laboratory parameters that were evaluated included •Any Grade ≥2 (moderate) or serious event without an alternative etiology that the Investigator deemed was related to study drug •Two consecutive drug-related serum creatinine levels ≥2*upper limit of normal (ULN) without an alternative etiology •Creatine kinase (CK) levels >50,000 units/liter (U/L) •A confirmed, unexplained, increase in gamma glutamyl transferase (GGT) >3*ULN and either an increase in bilirubin >2*ULN or nascent prothrombin time >2*ULN concurrently, without an alternative etiology
    End point type
    Primary
    End point timeframe
    Baseline up to Week 100
    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: Analysis were descriptive in nature.
    End point values
    		 Cohort 1: Age 24 to 48 Months Cohort 2: Age 6 to <24 Months
    Number of subjects analysed
    9
    6
    Units: participants
    9
    6
    No statistical analyses for this end point

    Primary: Abnormal Changes from Baseline or Worsening of Physical Examination Findings

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    End point title
    		 Abnormal Changes from Baseline or Worsening of Physical Examination Findings [3]
    End point description
    Data not collected during the study for this Outcome Measure. A summary of all SAEs and Other AEs (nonserious) regardless of causality is located in the ‘Adverse Events’ Section.
    End point type
    Primary
    End point timeframe
    Baseline up to Week 100
    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: Analysis were descriptive in nature.
    End point values
    		 Cohort 1: Age 24 to 48 Months Cohort 2: Age 6 to <24 Months
    Number of subjects analysed
    0 [4]
    0 [5]
    Units: Not applicable
        number (not applicable)
    Notes
    [4] - Data not collected.
    [5] - Data not collected.
    No statistical analyses for this end point

    Primary: Number of Participants with at Least 1 Markedly Abnormal Vital Sign

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    End point title
    		 Number of Participants with at Least 1 Markedly Abnormal Vital Sign [6]
    End point description
    The vital sign parameters that were evaluated included blood pressure, heart rate, respiration, and temperature. A summary of all SAEs and Other AEs (nonserious) regardless of causality is located in the ‘Adverse Events’ Section. The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
    End point type
    Primary
    End point timeframe
    Baseline up to Week 100
    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: Analysis were descriptive in nature.
    End point values
    		 Cohort 1: Age 24 to 48 Months Cohort 2: Age 6 to <24 Months
    Number of subjects analysed
    9
    6
    Units: participants
    9
    6
    No statistical analyses for this end point

    Primary: Number of Participants with at Least 1 Markedly Abnormal Electrocardiogram (ECG) and Echocardiogram (ECHO)

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    End point title
    		 Number of Participants with at Least 1 Markedly Abnormal Electrocardiogram (ECG) and Echocardiogram (ECHO) [7]
    End point description
    The ECG was manually reviewed and interpreted by medically qualified personnel using a central vendor according to pre-specified criteria. The Investigator determined if the findings in the centrally read ECG report were clinically significant. Clinical significance was defined as any variation in ECG findings that had medical relevance resulting in an alteration in medical care. The ECHO was reviewed and interpreted by medically qualified personnel using a central vendor according to pre-specified criteria. The Investigator determined if the findings in the ECHO report were clinically significant. Clinical significance was defined as any variation in ECHO findings that had medical relevance resulting in an alteration in medical care. A summary of all SAEs and Other AEs (nonserious) regardless of causality is located in the ‘Adverse Events’ Section. The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
    End point type
    Primary
    End point timeframe
    Weeks 8, 12, 24, 36, 48, 60, 72, 84, 96
    Notes
    [7] - 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: Analysis were descriptive in nature.
    End point values
    		 Cohort 1: Age 24 to 48 Months Cohort 2: Age 6 to <24 Months
    Number of subjects analysed
    9
    6
    Units: participants
    4
    5
    No statistical analyses for this end point

    Secondary: Maximum Plasma Concentration (Cmax) of Eteplirsen

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    End point title
    		 Maximum Plasma Concentration (Cmax) of Eteplirsen
    End point description
    The Safety Set was used for pharmacokinetic(s) (PK) analysis and included all participants who were enrolled and received at least 1 dose of eteplirsen during the study. PK data were not collected for participants who received 4 mg of eteplirsen. Here, ‘n’ signifies number of participants evaluable at the specified timepoint.
    End point type
    Secondary
    End point timeframe
    Pre-infusion, immediately prior to end of infusion, and approximately 1-3 hours and 6-8 hours after completion of infusion during Weeks 2 (2 mg/kg dose level), 6 (10 mg/kg dose level), 8 (20 mg/kg dose level), and 10 and 24 (30 mg/kg dose level)
    End point values
    		 Cohort 1: Age 24 to 48 Months Cohort 2: Age 6 to <24 Months
    Number of subjects analysed
    9
    6
    Units: microgram per milliliter (μg/mL)
    geometric mean (geometric coefficient of variation)
        Week 2 (2 mg/kg [n=8, 5])
    9.67 ± 75.9
    4.22 ± 120
        Week 6 (10 mg/kg)
    46.5 ± 72.3
    17.2 ± 192
        Week 8 (20 mg/kg)
    63.3 ± 123
    85.0 ± 67.6
        Week 10 (30 mg/kg)
    93.7 ± 55.5
    63.8 ± 124
        Week 24 (30 mg/kg [n=8, 6])
    78.2 ± 92.2
    59.7 ± 82.7
    No statistical analyses for this end point

    Secondary: Time to Reach Maximum Plasma Concentration (Tmax) of Eteplirsen

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    End point title
    		 Time to Reach Maximum Plasma Concentration (Tmax) of Eteplirsen
    End point description
    The Safety Set was used for PK analysis and included all participants who were enrolled and received at least 1 dose of eteplirsen during the study. PK data were not collected for participants who received 4 mg of eteplirsen. Here, ‘n’ signifies number of participants evaluable at the specified timepoint.
    End point type
    Secondary
    End point timeframe
    Pre-infusion, immediately prior to end of infusion, and approximately 1-3 hours and 6-8 hours after completion of infusion during Weeks 2 (2 mg/kg dose level), 6 (10 mg/kg dose level), 8 (20 mg/kg dose level), and 10 and 24 (30 mg/kg dose level)
    End point values
    		 Cohort 1: Age 24 to 48 Months Cohort 2: Age 6 to <24 Months
    Number of subjects analysed
    9
    6
    Units: hours (hr)
    median (full range (min-max))
        2 mg/kg (Week 2 [n= 8, 5])
    0.58 (0.17 to 2.67)
    0.58 (0.42 to 0.67)
        10 mg/kg (Week 6)
    0.58 (0.47 to 4.25)
    0.72 (0.58 to 3.32)
        20 mg/kg (Week 8)
    0.78 (0.50 to 2.75)
    0.73 (0.53 to 1.17)
        30 mg/kg (Week 10)
    0.58 (0.50 to 1.48)
    0.92 (0.50 to 2.75)
        30 mg/kg (Week 24 [n=8, 6])
    0.63 (0.42 to 6.83)
    0.72 (0.58 to 1.83)
    No statistical analyses for this end point

    Secondary: Area Under Concentration-Time Curve From Time 0 to the Last Quantifiable Concentration (AUClast) of Eteplirsen in Plasma

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    End point title
    		 Area Under Concentration-Time Curve From Time 0 to the Last Quantifiable Concentration (AUClast) of Eteplirsen in Plasma
    End point description
    The Safety Set was used for PK analysis and included all participants who were enrolled and received at least 1 dose of eteplirsen during the study. PK data were not collected for participants who received 4 mg of eteplirsen. Here, ‘n’ signifies number of participants evaluable at the specified timepoint.
    End point type
    Secondary
    End point timeframe
    Pre-infusion, immediately prior to end of infusion, and approximately 1-3 hours and 6-8 hours after completion of infusion during Weeks 2 (2 mg/kg dose level), 6 (10 mg/kg dose level), 8 (20 mg/kg dose level), and 10 and 24 (30 mg/kg dose level)
    End point values
    		 Cohort 1: Age 24 to 48 Months Cohort 2: Age 6 to <24 Months
    Number of subjects analysed
    9
    6
    Units: μg*hr/mL
    geometric mean (geometric coefficient of variation)
        2 mg/kg (Week 2 [n= 8, 5])
    13.8 ± 118
    6.13 ± 73.1
        10 mg/kg (Week 6)
    56.1 ± 57.2
    27.8 ± 113
        20 mg/kg (Week 8)
    92.1 ± 94.7
    81.4 ± 89.6
        30 mg/kg (Week 10)
    119 ± 30.8
    85.0 ± 114
        30 mg/kg (Week 24 [n=8, 6])
    100 ± 42.5
    89.6 ± 43.8
    No statistical analyses for this end point

    Secondary: Amount of Drug Eliminated in Urine

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    End point title
    		 Amount of Drug Eliminated in Urine
    End point description
    Amount of unchanged drug excreted in urine from time 0 to 4 hours after completion of dosing is reported. The Safety Set was used for PK analysis and included all participants who were enrolled and received at least 1 dose of eteplirsen during the study. PK data were not collected for participants who received 4 mg of eteplirsen. Here, ‘Number of Subjects Analyzed’ (N) signifies number of participants evaluable for this outcome measure and ‘n’ signifies number of participants evaluable at the specified timepoint.
    End point type
    Secondary
    End point timeframe
    Pre-infusion, immediately prior to end of infusion, and approximately 1-3 hours and 6-8 hours after completion of infusion during Weeks 2 (2 mg/kg dose level), 6 (10 mg/kg dose level), 8 (20 mg/kg dose level), and 10 and 24 (30 mg/kg dose level)
    End point values
    		 Cohort 1: Age 24 to 48 Months Cohort 2: Age 6 to <24 Months
    Number of subjects analysed
    8
    6
    Units: μg
    arithmetic mean (standard deviation)
        2 mg/kg dose (Week 2 [n= 3, 3])
    7720 ± 9060
    1430 ± 1390
        10 mg/kg (Week 6 [n=7, 6])
    56000 ± 73300
    28700 ± 24100
        20 mg/kg (Week 8 [n=6, 5])
    102000 ± 108000
    65600 ± 47900
        20 mg/kg (Week 8 [n=8, 6)
    263000 ± 209000
    94700 ± 68500
        30 mg/kg (Week 24 [n=7, 4])
    239000 ± 140000
    147000 ± 132000
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Baseline up to Week 100
    Adverse event reporting additional description
    The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    22.1
    Reporting groups
    Reporting group title
    Cohort 2: Age 6 to <24 Months
    Reporting group description
    		 Participants between 6 to <24 months old were administered eteplirsen once every 7 days by IV infusion starting on Day 1 for up to 96 weeks. The starting dose was 2 mg/kg eteplirsen, with escalation to 4, 10, 20, and 30 mg/kg for 10 weeks and then participants continued to receive eteplirsen at 30 mg/kg for the duration of the study.

    Reporting group title
    Cohort 1: Age 24 to 48 Months
    Reporting group description
    		 Participants between 24 to 48 months old were administered eteplirsen once every 7 days by IV infusion starting on Day 1 for up to 96 weeks. The starting dose was 2 mg/kg eteplirsen, with escalation to 4, 10, 20, and 30 mg/kg for 10 weeks and then participants continued to receive eteplirsen at 30 mg/kg for the duration of the study.

    Serious adverse events
    		 Cohort 2: Age 6 to <24 Months Cohort 1: Age 24 to 48 Months
    Total subjects affected by serious adverse events
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 9 (0.00%)
         number of deaths (all causes)
    0
    0
         number of deaths resulting from adverse events
    0
    0
    Infections and infestations
    Bronchiolitis
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 9 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 0%
    Non-serious adverse events
    		 Cohort 2: Age 6 to <24 Months Cohort 1: Age 24 to 48 Months
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    6 / 6 (100.00%)
    9 / 9 (100.00%)
    Vascular disorders
    Flushing
         subjects affected / exposed
    0 / 6 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    1
    Haematoma
         subjects affected / exposed
    0 / 6 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    1
    Hypertension
         subjects affected / exposed
    0 / 6 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    1
    General disorders and administration site conditions
    Pyrexia
         subjects affected / exposed
    6 / 6 (100.00%)
    7 / 9 (77.78%)
         occurrences all number
    21
    21
    Catheter site swelling
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 9 (0.00%)
         occurrences all number
    1
    0
    Catheter site eczema
         subjects affected / exposed
    0 / 6 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    1
    Fatigue
         subjects affected / exposed
    0 / 6 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    1
    Discomfort
         subjects affected / exposed
    0 / 6 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    1
    Hyperpyrexia
         subjects affected / exposed
    0 / 6 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    1
    Infusion site extravasation
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 9 (0.00%)
         occurrences all number
    1
    0
    Localised oedema
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 9 (0.00%)
         occurrences all number
    3
    0
    Immune system disorders
    Allergy to chemicals
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 9 (0.00%)
         occurrences all number
    1
    0
    Reproductive system and breast disorders
    Genital cyst
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 9 (0.00%)
         occurrences all number
    1
    0
    Respiratory, thoracic and mediastinal disorders
    Cough
         subjects affected / exposed
    5 / 6 (83.33%)
    7 / 9 (77.78%)
         occurrences all number
    14
    25
    Rhinorrhoea
         subjects affected / exposed
    3 / 6 (50.00%)
    4 / 9 (44.44%)
         occurrences all number
    6
    7
    Epistaxis
         subjects affected / exposed
    0 / 6 (0.00%)
    2 / 9 (22.22%)
         occurrences all number
    0
    9
    Productive cough
         subjects affected / exposed
    0 / 6 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    1
    Psychiatric disorders
    Abnormal behaviour
         subjects affected / exposed
    0 / 6 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    1
    Anger
         subjects affected / exposed
    0 / 6 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    1
    Personality change
         subjects affected / exposed
    0 / 6 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    1
    Irritability
         subjects affected / exposed
    0 / 6 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    2
    Initial insomnia
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 9 (0.00%)
         occurrences all number
    1
    0
    Investigations
    Body temperature
         subjects affected / exposed
    1 / 6 (16.67%)
    1 / 9 (11.11%)
         occurrences all number
    1
    3
    Blood iron decreased
         subjects affected / exposed
    0 / 6 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    1
    Amylase increased
         subjects affected / exposed
    0 / 6 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    1
    Body temperature increased
         subjects affected / exposed
    1 / 6 (16.67%)
    1 / 9 (11.11%)
         occurrences all number
    3
    2
    Haemophilus test positive
         subjects affected / exposed
    0 / 6 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    1
    Streptococcus test positive
         subjects affected / exposed
    0 / 6 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    1
    Injury, poisoning and procedural complications
    Contusion
         subjects affected / exposed
    0 / 6 (0.00%)
    2 / 9 (22.22%)
         occurrences all number
    0
    2
    Head injury
         subjects affected / exposed
    2 / 6 (33.33%)
    5 / 9 (55.56%)
         occurrences all number
    3
    7
    Fall
         subjects affected / exposed
    2 / 6 (33.33%)
    5 / 9 (55.56%)
         occurrences all number
    5
    5
    Laceration
         subjects affected / exposed
    1 / 6 (16.67%)
    1 / 9 (11.11%)
         occurrences all number
    1
    1
    Procedural pain
         subjects affected / exposed
    1 / 6 (16.67%)
    1 / 9 (11.11%)
         occurrences all number
    1
    1
    Post-traumatic pain
         subjects affected / exposed
    1 / 6 (16.67%)
    1 / 9 (11.11%)
         occurrences all number
    1
    1
    Arthropod bite
         subjects affected / exposed
    0 / 6 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    2
    Joint injury
         subjects affected / exposed
    0 / 6 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    1
    Excoriation
         subjects affected / exposed
    0 / 6 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    1
    Lip injury
         subjects affected / exposed
    0 / 6 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    1
    Penis injury
         subjects affected / exposed
    0 / 6 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    1
    Tracheal obstruction
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 9 (0.00%)
         occurrences all number
    1
    0
    Tongue injury
         subjects affected / exposed
    0 / 6 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    1
    Spinal fracture
         subjects affected / exposed
    0 / 6 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    1
    Vaccination complication
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 9 (0.00%)
         occurrences all number
    2
    0
    Congenital, familial and genetic disorders
    Haemoglobinopathy
         subjects affected / exposed
    0 / 6 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    1
    Nervous system disorders
    Lethargy
         subjects affected / exposed
    2 / 6 (33.33%)
    2 / 9 (22.22%)
         occurrences all number
    3
    2
    Headache
         subjects affected / exposed
    0 / 6 (0.00%)
    2 / 9 (22.22%)
         occurrences all number
    0
    2
    Psychomotor hyperactivity
         subjects affected / exposed
    0 / 6 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    1
    Blood and lymphatic system disorders
    Autoimmune neutropenia
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 9 (0.00%)
         occurrences all number
    1
    0
    Hypochromic anaemia
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 9 (0.00%)
         occurrences all number
    1
    0
    Iron deficiency anaemia
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 9 (0.00%)
         occurrences all number
    1
    0
    Ear and labyrinth disorders
    Ear pain
         subjects affected / exposed
    2 / 6 (33.33%)
    1 / 9 (11.11%)
         occurrences all number
    2
    1
    Cerumen impaction
         subjects affected / exposed
    1 / 6 (16.67%)
    1 / 9 (11.11%)
         occurrences all number
    1
    1
    External ear inflammation
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 9 (0.00%)
         occurrences all number
    1
    0
    Inner ear inflammation
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 9 (0.00%)
         occurrences all number
    1
    0
    Tympanic membrane hyperaemia
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 9 (0.00%)
         occurrences all number
    1
    0
    Eye disorders
    Chalazion
         subjects affected / exposed
    1 / 6 (16.67%)
    1 / 9 (11.11%)
         occurrences all number
    2
    3
    Eye irritation
         subjects affected / exposed
    0 / 6 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    1
    Visual acuity reduced
         subjects affected / exposed
    0 / 6 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    1
    Eye pruritus
         subjects affected / exposed
    0 / 6 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    1
    Gastrointestinal disorders
    Diarrhoea
         subjects affected / exposed
    3 / 6 (50.00%)
    5 / 9 (55.56%)
         occurrences all number
    9
    6
    Vomiting
         subjects affected / exposed
    4 / 6 (66.67%)
    8 / 9 (88.89%)
         occurrences all number
    4
    15
    Abdominal pain
         subjects affected / exposed
    1 / 6 (16.67%)
    1 / 9 (11.11%)
         occurrences all number
    1
    1
    Teething
         subjects affected / exposed
    2 / 6 (33.33%)
    1 / 9 (11.11%)
         occurrences all number
    3
    1
    Constipation
         subjects affected / exposed
    1 / 6 (16.67%)
    2 / 9 (22.22%)
         occurrences all number
    1
    8
    Abdominal pain upper
         subjects affected / exposed
    0 / 6 (0.00%)
    2 / 9 (22.22%)
         occurrences all number
    0
    2
    Dental caries
         subjects affected / exposed
    0 / 6 (0.00%)
    2 / 9 (22.22%)
         occurrences all number
    0
    2
    Dental discomfort
         subjects affected / exposed
    0 / 6 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    2
    Faeces discoloured
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 9 (0.00%)
         occurrences all number
    1
    0
    Toothache
         subjects affected / exposed
    0 / 6 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    1
    Skin and subcutaneous tissue disorders
    Eczema
         subjects affected / exposed
    1 / 6 (16.67%)
    2 / 9 (22.22%)
         occurrences all number
    1
    2
    Rash
         subjects affected / exposed
    1 / 6 (16.67%)
    2 / 9 (22.22%)
         occurrences all number
    4
    2
    Rash maculo-papular
         subjects affected / exposed
    3 / 6 (50.00%)
    0 / 9 (0.00%)
         occurrences all number
    8
    0
    Dermatitis contact
         subjects affected / exposed
    1 / 6 (16.67%)
    1 / 9 (11.11%)
         occurrences all number
    2
    1
    Dermatitis diaper
         subjects affected / exposed
    1 / 6 (16.67%)
    1 / 9 (11.11%)
         occurrences all number
    1
    1
    Rash generalised
         subjects affected / exposed
    1 / 6 (16.67%)
    1 / 9 (11.11%)
         occurrences all number
    3
    1
    Eczema nummular
         subjects affected / exposed
    0 / 6 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    1
    Urticaria
         subjects affected / exposed
    0 / 6 (0.00%)
    2 / 9 (22.22%)
         occurrences all number
    0
    2
    Miliaria
         subjects affected / exposed
    0 / 6 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    1
    Erythema
         subjects affected / exposed
    0 / 6 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    1
    Pruritus generalised
         subjects affected / exposed
    0 / 6 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    1
    Rash macular
         subjects affected / exposed
    0 / 6 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    1
    Rash papular
         subjects affected / exposed
    0 / 6 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    1
    Renal and urinary disorders
    Dysuria
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 9 (0.00%)
         occurrences all number
    1
    0
    Musculoskeletal and connective tissue disorders
    Pain in extremity
         subjects affected / exposed
    0 / 6 (0.00%)
    3 / 9 (33.33%)
         occurrences all number
    0
    8
    Arthralgia
         subjects affected / exposed
    0 / 6 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    1
    Infections and infestations
    Nasopharyngitis
         subjects affected / exposed
    5 / 6 (83.33%)
    7 / 9 (77.78%)
         occurrences all number
    19
    27
    Rhinitis
         subjects affected / exposed
    4 / 6 (66.67%)
    4 / 9 (44.44%)
         occurrences all number
    9
    4
    Ear infection
         subjects affected / exposed
    3 / 6 (50.00%)
    3 / 9 (33.33%)
         occurrences all number
    3
    6
    Gastroenteritis
         subjects affected / exposed
    3 / 6 (50.00%)
    3 / 9 (33.33%)
         occurrences all number
    3
    6
    Bronchitis
         subjects affected / exposed
    2 / 6 (33.33%)
    2 / 9 (22.22%)
         occurrences all number
    2
    2
    Influenza
         subjects affected / exposed
    2 / 6 (33.33%)
    1 / 9 (11.11%)
         occurrences all number
    2
    1
    Pharyngitis
         subjects affected / exposed
    1 / 6 (16.67%)
    1 / 9 (11.11%)
         occurrences all number
    1
    2
    Conjunctivitis
         subjects affected / exposed
    1 / 6 (16.67%)
    1 / 9 (11.11%)
         occurrences all number
    1
    1
    Eye infection
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 9 (0.00%)
         occurrences all number
    2
    0
    Upper respiratory tract infection
         subjects affected / exposed
    2 / 6 (33.33%)
    0 / 9 (0.00%)
         occurrences all number
    4
    0
    Gastrointestinal viral infection
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 9 (0.00%)
         occurrences all number
    1
    0
    Hand-foot-and-mouth disease
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 9 (0.00%)
         occurrences all number
    1
    0
    Hordeolum
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 9 (0.00%)
         occurrences all number
    1
    0
    Lower respiratory tract infection
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 9 (0.00%)
         occurrences all number
    1
    0
    Molluscum contagiosum
         subjects affected / exposed
    0 / 6 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    1
    Otitis media acute
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 9 (0.00%)
         occurrences all number
    2
    0
    Roseola
         subjects affected / exposed
    0 / 6 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    1
    Varicella
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 9 (0.00%)
         occurrences all number
    1
    0
    Viral upper respiratory tract infection
         subjects affected / exposed
    0 / 6 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    1
    Metabolism and nutrition disorders
    Iron deficiency
         subjects affected / exposed
    2 / 6 (33.33%)
    1 / 9 (11.11%)
         occurrences all number
    2
    1
    Increased appetite
         subjects affected / exposed
    0 / 6 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    1
    Hyposideraemia
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 9 (0.00%)
         occurrences all number
    1
    0

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    07 Feb 2020
    To reflect updates made to recently approved language from other recent Sarepta protocol amendments.

    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
    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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