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    Clinical Trial Results:
    Dose-Ranging Study of AVI-4658 to Induce Dystrophin Expression in Selected Duchenne Muscular Dystrophy (DMD) Patients

    Summary
    EudraCT number
    		 2007-004695-39
    Trial protocol
    		 GB  
    Global end of trial date
    08 Jun 2010

    Results information
    Results version number
    		 v1(current)
    This version publication date
    09 Jun 2016
    First version publication date
    09 Jun 2016
    Other versions

    Trial information

    		 Close Top of page
    Trial identification
    Sponsor protocol code
    AVI-4658-28
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT00844597
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Sarepta Therapeutics
    Sponsor organisation address
    215 First Street, Cambridge, United States, 02142
    Public contact
    Shamim Ruff, Sarepta Therapeutics, 1 6172744009, SRuff@Sarepta.com
    Scientific contact
    Shamim Ruff, Sarepta Therapeutics, 1 6172744009, SRuff@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
    08 Jun 2010
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    08 Jun 2010
    Global end of trial reached?
    Yes
    Global end of trial date
    08 Jun 2010
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    The primary objective of this Phase 1b study is to assess the safety of escalating doses of AVI-4658 when administered by 12 weekly doses in boys with Duchenne muscular dystrophy (DMD).
    Protection of trial subjects
    In addition to the informed consent process required of the patient’s parent(s) or legal guardian(s), all possible steps to ensure the protection of this vulnerable class of trial patients, i.e., a pediatric population with Duchenne muscular dystrophy (DMD) were taken including the requirement of assent by each patient, where appropriate. The study site was responsible for producing the patient informed assent form and the parent(s)/legal guardian(s) informed consent form. Before initiation of the study, the assent and consent forms were submitted for approval to both the Sponsor and to the EC. Prior to the conduct of any pre-entry tests not performed routinely in the treatment of the patient, the Investigator fully explained the study to the patient and the patient’s parent(s) or legal guardian(s). The explanation was sufficiently detailed to allow for an informed decision to participate made by the patient and the patient’s parent(s) or legal guardian(s). If the patient (with permission from the patient’s parent[s] or legal guardian[s]) was willing to participate in the study, he was requested to give written informed assent, where appropriate, and the patient’s parent(s) or legal guardian(s) were requested to give written informed consent. In accordance with institutional and applicable local regulations, assent and written informed consent were obtained prior to performing any study-related procedures, including (non-routine) Screening assessments and administration of study drug. Administration via the IV route over 60 minutes was selected for patient comfort and because IV access was needed for safety laboratory and PK sample collection. Anesthetic topical cream was used prior to IV placement.
    Background therapy
    		 Patients continued on their standard of care treatment for DMD. 18 of the 19 patients were on some kind of Corticosteroid Treatment including Prednisoline, Hydrocortisone and Deflazacort. 1 patient - no corticosteroid treatments 5 patients - Prednisolone regimen 9 patients - Prednisoline regimen and 1 or more hydrocortisone infusions 1 patient - Deflazacort regimen 2 patients - Prednosoline, Hydrocortisone and Deflazacort treatments
    Evidence for comparator
    		 -
    Actual start date of recruitment
    07 Jan 2009
    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: 19
    Worldwide total number of subjects
    19
    EEA total number of subjects
    19
    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)
    17
    Adolescents (12-17 years)
    2
    Adults (18-64 years)
    0
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

    		 Close Top of page
    Recruitment
    Recruitment details
    		 Patients were recruited at two sites in the United Kingdom in London and Newcastle Upon Tyne. The first subject was enrolled in January 2009 and received their first study drug dose February 18, 2009. The last subject was enrolled November 27, 2009 and received their first study drug dose on December 14, 2009.

    Pre-assignment
    Screening details
    		 The first patient was screened February 11, 2009 and the last was screened December 6, 2009. There were 20 patients screened and 19 of these patients were enrolled. The patient who screened failed did not have the correct mutation to be included in the trial.

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

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Cohort 1 - 0.5mg/kg
    Arm description
    		 Subjects in this group received a 0.5 mg/kg/wk dose of AVI-4658 over 12 weekly IV infusions in 50 mL of normal saline solution over a 60-minute period
    Arm type
    		 Experimental

    Investigational medicinal product name
    Eteplirsen
    Investigational medicinal product code
    AVI-4658
    Other name
    Pharmaceutical forms
    Concentrate for solution for infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    Eteplirsen, formulated in phosphate buffered solution, was supplied in single-use vials at a concentration of 100 mg/mL. Six cohorts of patients were sequentially allocated to receive 0.5, 1.0, 2.0, 4.0, 10.0, or 20.0 mg/kg/wk IV infusions of eteplirsen for 12 weeks. Eteplirsen was diluted up to 50 mL with normal saline solution into a syringe and administered IV over a 60 minute period. Lot numbers of eteplirsen used in this study: 44GD-DE01 and 60GD-DE01

    Arm title
    		 Cohort 2 - 1.0 mg/kg/wk
    Arm description
    		 Subjects in this group will receive a 1.0 mg/kg/wk dose of AVI-4658 over 12 weekly IV infusions in 50 mL of normal saline solution over a 60-minute period
    Arm type
    		 Experimental

    Investigational medicinal product name
    Eteplirsen
    Investigational medicinal product code
    AVI-4658
    Other name
    SRP-4658
    Pharmaceutical forms
    Concentrate for solution for infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    Eteplirsen, formulated in phosphate buffered solution, was supplied in single-use vials at a concentration of 100 mg/mL. Six cohorts of patients were sequentially allocated to receive 0.5, 1.0, 2.0, 4.0, 10.0, or 20.0 mg/kg/wk IV infusions of eteplirsen for 12 weeks. Eteplirsen was diluted up to 50 mL with normal saline solution into a syringe and administered IV over a 60 minute period. Lot numbers of eteplirsen used in this study: 44GD-DE01 and 60GD-DE01

    Arm title
    		 Cohort 3 - 2.0 mg/kg/wk
    Arm description
    		 Subjects in this group will receive a 2.0 mg/kg/wk dose of AVI-4658 over 12 weekly IV infusions in 50 mL of normal saline solution over a 60-minute period
    Arm type
    		 Experimental

    Investigational medicinal product name
    Eteplirsen
    Investigational medicinal product code
    AVI-4658
    Other name
    SRP-4658
    Pharmaceutical forms
    Concentrate for solution for infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    Eteplirsen, formulated in phosphate buffered solution, was supplied in single-use vials at a concentration of 100 mg/mL. Six cohorts of patients were sequentially allocated to receive 0.5, 1.0, 2.0, 4.0, 10.0, or 20.0 mg/kg/wk IV infusions of eteplirsen for 12 weeks. Eteplirsen was diluted up to 50 mL with normal saline solution into a syringe and administered IV over a 60 minute period. Lot numbers of eteplirsen used in this study: 44GD-DE01 and 60GD-DE01

    Arm title
    		 Cohort 4 - 4.0 mg/kg/wk
    Arm description
    		 Subjects in this group will receive a 4.0 mg/kg/wk dose of AVI-4658 over 12 weekly IV infusions in 50 mL of normal saline solution over a 60-minute period
    Arm type
    		 Experimental

    Investigational medicinal product name
    Eteplirsen
    Investigational medicinal product code
    AVI-4658
    Other name
    SRP-4658
    Pharmaceutical forms
    Concentrate for solution for infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    Eteplirsen, formulated in phosphate buffered solution, was supplied in single-use vials at a concentration of 100 mg/mL. Six cohorts of patients were sequentially allocated to receive 0.5, 1.0, 2.0, 4.0, 10.0, or 20.0 mg/kg/wk IV infusions of eteplirsen for 12 weeks. Eteplirsen was diluted up to 50 mL with normal saline solution into a syringe and administered IV over a 60 minute period. Lot numbers of eteplirsen used in this study: 44GD-DE01 and 60GD-DE01

    Arm title
    		 Cohort 5 - 10 mg/kg/wk
    Arm description
    		 Subjects in this group will receive a 10.0 mg/kg/wk dose of AVI-4658 over 12 weekly IV infusions in 50 mL of normal saline solution over a 60-minute period
    Arm type
    		 Experimental

    Investigational medicinal product name
    Eteplirsen
    Investigational medicinal product code
    AVI-4658
    Other name
    SRP-4658
    Pharmaceutical forms
    Concentrate for solution for infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    Eteplirsen, formulated in phosphate buffered solution, was supplied in single-use vials at a concentration of 100 mg/mL. Six cohorts of patients were sequentially allocated to receive 0.5, 1.0, 2.0, 4.0, 10.0, or 20.0 mg/kg/wk IV infusions of eteplirsen for 12 weeks. Eteplirsen was diluted up to 50 mL with normal saline solution into a syringe and administered IV over a 60 minute period. Lot numbers of eteplirsen used in this study: 44GD-DE01 and 60GD-DE01

    Arm title
    		 Cohort 6 - 20mg/kg/wk
    Arm description
    		 Subjects in this group will receive a 20.0 mg/kg/wk dose of AVI-4658 over 12 weekly IV infusions in 50 mL of normal saline solution over a 60-minute period
    Arm type
    		 Experimental

    Investigational medicinal product name
    Eteplirsen
    Investigational medicinal product code
    AVI-4658
    Other name
    SRP-4658
    Pharmaceutical forms
    Concentrate for solution for infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    Eteplirsen, formulated in phosphate buffered solution, was supplied in single-use vials at a concentration of 100 mg/mL. Six cohorts of patients were sequentially allocated to receive 0.5, 1.0, 2.0, 4.0, 10.0, or 20.0 mg/kg/wk IV infusions of eteplirsen for 12 weeks. Eteplirsen was diluted up to 50 mL with normal saline solution into a syringe and administered IV over a 60 minute period. Lot numbers of eteplirsen used in this study: 44GD-DE01 and 60GD-DE01

    Number of subjects in period 1
    		Cohort 1 - 0.5mg/kg Cohort 2 - 1.0 mg/kg/wk Cohort 3 - 2.0 mg/kg/wk Cohort 4 - 4.0 mg/kg/wk Cohort 5 - 10 mg/kg/wk Cohort 6 - 20mg/kg/wk
    Started
    4
    2
    2
    3
    4
    4
    Completed
    4
    2
    2
    2
    4
    4
    Not completed
    0
    0
    0
    1
    0
    0
         Adverse event, non-fatal
    -
    -
    -
    1
    -
    -

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Cohort 1 - 0.5mg/kg
    Reporting group description
    		 Subjects in this group received a 0.5 mg/kg/wk dose of AVI-4658 over 12 weekly IV infusions in 50 mL of normal saline solution over a 60-minute period

    Reporting group title
    Cohort 2 - 1.0 mg/kg/wk
    Reporting group description
    		 Subjects in this group will receive a 1.0 mg/kg/wk dose of AVI-4658 over 12 weekly IV infusions in 50 mL of normal saline solution over a 60-minute period

    Reporting group title
    Cohort 3 - 2.0 mg/kg/wk
    Reporting group description
    		 Subjects in this group will receive a 2.0 mg/kg/wk dose of AVI-4658 over 12 weekly IV infusions in 50 mL of normal saline solution over a 60-minute period

    Reporting group title
    Cohort 4 - 4.0 mg/kg/wk
    Reporting group description
    		 Subjects in this group will receive a 4.0 mg/kg/wk dose of AVI-4658 over 12 weekly IV infusions in 50 mL of normal saline solution over a 60-minute period

    Reporting group title
    Cohort 5 - 10 mg/kg/wk
    Reporting group description
    		 Subjects in this group will receive a 10.0 mg/kg/wk dose of AVI-4658 over 12 weekly IV infusions in 50 mL of normal saline solution over a 60-minute period

    Reporting group title
    Cohort 6 - 20mg/kg/wk
    Reporting group description
    		 Subjects in this group will receive a 20.0 mg/kg/wk dose of AVI-4658 over 12 weekly IV infusions in 50 mL of normal saline solution over a 60-minute period

    Reporting group values
    		 Cohort 1 - 0.5mg/kg Cohort 2 - 1.0 mg/kg/wk Cohort 3 - 2.0 mg/kg/wk Cohort 4 - 4.0 mg/kg/wk Cohort 5 - 10 mg/kg/wk Cohort 6 - 20mg/kg/wk Total
    Number of subjects
    		 4 2 2 3 4 4 19
    Age categorical
    Units: Subjects
        Children (2-11 years)
    		 4 2 1 3 3 4 17
        Adolescents (12-17 years)
    		 0 0 1 0 1 0 2
    Age continuous
    Units: years
        least squares mean (standard deviation)
    		 8.3 ( 0.5 ) 6 ( 0 ) 11 ( 2.83 ) 9.7 ( 0.58 ) 8.8 ( 2.75 ) 8.8 ( 1.26 ) -
    Gender categorical
    Units: Subjects
        Female
    		 0 0 0 0 0 0 0
        Male
    		 4 2 2 3 4 4 19
    Subject analysis sets

    Subject analysis set title
    Safety Population
    Subject analysis set type
    		 Safety analysis
    Subject analysis set description
    Included all patients who were enrolled in the study and received at least 1 dose of study treatment.

    Subject analysis set title
    Per Protocol Population
    Subject analysis set type
    		 Per protocol
    Subject analysis set description
    Included all patients who received all 12 doses of study treatment.

    Subject analysis set title
    PK Evaluable Population
    Subject analysis set type
    		 Sub-group analysis
    Subject analysis set description
    Included all patients who provided at least 1 PK sample. The reportable PK population included those patients with at least Cmax, Tmax, and AUC0-24 computed from 1 or more of the 3 sampling days (1st, 6th, 12th dose [Weeks 1, 6, and 12]).

    Subject analysis sets values
    		 Safety Population Per Protocol Population PK Evaluable Population
    Number of subjects
    19
    18
    19
    Age categorical
    Units: Subjects
        Children (2-11 years)
    17
    16
    17
        Adolescents (12-17 years)
    2
    2
    2
    Age continuous
    Units: years
        least squares mean (standard deviation)
    8.7 ( 1.91 )
    8.6 ( 1.94 )
    8.7 ( 1.91 )
    Gender categorical
    Units: Subjects
        Female
        Male

    End points

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    End points reporting groups
    Reporting group title
    Cohort 1 - 0.5mg/kg
    Reporting group description
    		 Subjects in this group received a 0.5 mg/kg/wk dose of AVI-4658 over 12 weekly IV infusions in 50 mL of normal saline solution over a 60-minute period

    Reporting group title
    Cohort 2 - 1.0 mg/kg/wk
    Reporting group description
    		 Subjects in this group will receive a 1.0 mg/kg/wk dose of AVI-4658 over 12 weekly IV infusions in 50 mL of normal saline solution over a 60-minute period

    Reporting group title
    Cohort 3 - 2.0 mg/kg/wk
    Reporting group description
    		 Subjects in this group will receive a 2.0 mg/kg/wk dose of AVI-4658 over 12 weekly IV infusions in 50 mL of normal saline solution over a 60-minute period

    Reporting group title
    Cohort 4 - 4.0 mg/kg/wk
    Reporting group description
    		 Subjects in this group will receive a 4.0 mg/kg/wk dose of AVI-4658 over 12 weekly IV infusions in 50 mL of normal saline solution over a 60-minute period

    Reporting group title
    Cohort 5 - 10 mg/kg/wk
    Reporting group description
    		 Subjects in this group will receive a 10.0 mg/kg/wk dose of AVI-4658 over 12 weekly IV infusions in 50 mL of normal saline solution over a 60-minute period

    Reporting group title
    Cohort 6 - 20mg/kg/wk
    Reporting group description
    		 Subjects in this group will receive a 20.0 mg/kg/wk dose of AVI-4658 over 12 weekly IV infusions in 50 mL of normal saline solution over a 60-minute period

    Subject analysis set title
    Safety Population
    Subject analysis set type
    		 Safety analysis
    Subject analysis set description
    Included all patients who were enrolled in the study and received at least 1 dose of study treatment.

    Subject analysis set title
    Per Protocol Population
    Subject analysis set type
    		 Per protocol
    Subject analysis set description
    Included all patients who received all 12 doses of study treatment.

    Subject analysis set title
    PK Evaluable Population
    Subject analysis set type
    		 Sub-group analysis
    Subject analysis set description
    Included all patients who provided at least 1 PK sample. The reportable PK population included those patients with at least Cmax, Tmax, and AUC0-24 computed from 1 or more of the 3 sampling days (1st, 6th, 12th dose [Weeks 1, 6, and 12]).

    Primary: Safety and Tolerability

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    End point title
    		 Safety and Tolerability [1]
    End point description
    Number of subjects with 1 or more Treatment Emergent Adverse Event that are possibly related to the investigational drug
    End point type
    Primary
    End point timeframe
    Baseline to 6 months
    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: This study was primarily descriptive in nature; therefore, there were no formal statistical hypothesis tests planned.
    End point values
    		 Safety Population
    Number of subjects analysed
    19
    Units: Subjects
    14
    No statistical analyses for this end point

    Primary: Treatment Emergent Adverse Events

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    End point title
    		 Treatment Emergent Adverse Events [2]
    End point description
    Number of Patients with Treatment Emergent Adverse Events
    End point type
    Primary
    End point timeframe
    from Baseline to Follow-up (27 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: This study was primarily descriptive in nature; therefore, there were no formal statistical hypothesis tests planned.
    End point values
    		 Safety Population
    Number of subjects analysed
    19
    Units: Patients
    19
    No statistical analyses for this end point

    Secondary: Pharmacokinetics - Mean Peak Plasma Concentration of AVI-4658 after Administration

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    End point title
    		 Pharmacokinetics - Mean Peak Plasma Concentration of AVI-4658 after Administration
    End point description
    Standard Pharmacokinetic parameters estimated using non-compartmental modeling of plasma concentration data.
    End point type
    Secondary
    End point timeframe
    Samples were taken: 30 minutes pre dose; and at 5 (±1), 15 (±2), 30 (±5), 60 (±5), and 90 (±5) minutes; and 2, 4, 6, 8, 12, and 24 hours (all ± 15 minutes) post dose at Weeks 1, 6, and 12
    End point values
    		 PK Evaluable Population
    Number of subjects analysed
    19
    Units: ng/mL
        least squares mean (standard deviation)
    39000 ( 16900 )
    No statistical analyses for this end point

    Secondary: Efficacy of Eteplirsen Over 12 Weeks of Dosing

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    End point title
    		 Efficacy of Eteplirsen Over 12 Weeks of Dosing
    End point description
    Efficacy was defined as an estimated change in the percentage of dystrophin positive fibers (assessed by IHC) at Week 14 from Baseline after 12 weekly doses of eterplirsen. This outcome measure represents the number of patients to show an increase in the percentage of dystrophin-positive fibers.
    End point type
    Secondary
    End point timeframe
    Biopsies were taken at Baseline and Week 14
    End point values
    		 Per Protocol Population
    Number of subjects analysed
    17
    Units: patients
    11
    No statistical analyses for this end point

    Post-hoc: Adverse Events >15%

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    End point title
    		 Adverse Events >15%
    End point description
    Adverse events that occurred in >15% of overall patient population across dose level arms.
    End point type
    Post-hoc
    End point timeframe
    27 Weeks
    End point values
    		 Cohort 1 - 0.5mg/kg Cohort 2 - 1.0 mg/kg/wk Cohort 3 - 2.0 mg/kg/wk Cohort 4 - 4.0 mg/kg/wk Cohort 5 - 10 mg/kg/wk Cohort 6 - 20mg/kg/wk Safety Population
    Number of subjects analysed
    4
    2
    2
    3
    4
    4
    19
    Units: Occurances
        Cardiomyopathy
    0
    0
    0
    1
    0
    2
    3
        Tachycardia
    0
    0
    0
    1
    0
    2
    3
        Abdominal Pain
    0
    1
    0
    1
    1
    0
    3
        Nausea
    0
    0
    1
    1
    0
    1
    3
        Vomiting
    0
    0
    1
    1
    1
    0
    3
        Influenza like Illness
    0
    0
    2
    0
    1
    0
    3
        Rhinitis
    1
    0
    0
    1
    4
    1
    7
        Upper respiratory tract infection
    2
    1
    0
    1
    2
    2
    8
        Fall
    2
    0
    0
    2
    0
    1
    5
        Arthralgia
    1
    0
    1
    1
    0
    0
    3
        Back Pain
    1
    0
    1
    2
    2
    1
    7
        Myalgia
    1
    1
    1
    0
    1
    0
    4
        Dizziness
    0
    0
    1
    1
    0
    1
    3
        Headache
    2
    1
    2
    0
    2
    1
    8
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Adverse Event data for this study was collected from Baseline to Follow-up (27 Weeks)
    Assessment type
    		 Non-systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    12.0
    Reporting groups
    Reporting group title
    Cohort 1 - 0.05mg/kg
    Reporting group description
    		 Subjects in this group received a 0.5 mg/kg/wk dose of AVI-4658 over 12 weekly IV infusions in 50 mL of normal saline solution over a 60-minute period

    Reporting group title
    Cohort 2 - 1.0 mg/kg/wk
    Reporting group description
    		 Subjects in this group will receive a 1.0 mg/kg/wk dose of AVI-4658 over 12 weekly IV infusions in 50 mL of normal saline solution over a 60-minute period

    Reporting group title
    Cohort 3 - 2.0 mg/kg/wk
    Reporting group description
    		 Subjects in this group will receive a 2.0 mg/kg/wk dose of AVI-4658 over 12 weekly IV infusions in 50 mL of normal saline solution over a 60-minute period

    Reporting group title
    Cohort 4 - 4.0 mg/kg/wk
    Reporting group description
    		 Subjects in this group will receive a 4.0 mg/kg/wk dose of AVI-4658 over 12 weekly IV infusions in 50 mL of normal saline solution over a 60-minute period

    Reporting group title
    Cohort 5 - 10 mg/kg/wk
    Reporting group description
    		 Subjects in this group will receive a 10.0 mg/kg/wk dose of AVI-4658 over 12 weekly IV infusions in 50 mL of normal saline solution over a 60-minute period

    Reporting group title
    Cohort 6 - 20mg/kg/wk
    Reporting group description
    		 Subjects in this group will receive a 20.0 mg/kg/wk dose of AVI-4658 over 12 weekly IV infusions in 50 mL of normal saline solution over a 60-minute period

    Serious adverse events
    		 Cohort 1 - 0.05mg/kg Cohort 2 - 1.0 mg/kg/wk Cohort 3 - 2.0 mg/kg/wk Cohort 4 - 4.0 mg/kg/wk Cohort 5 - 10 mg/kg/wk Cohort 6 - 20mg/kg/wk
    Total subjects affected by serious adverse events
         subjects affected / exposed
    0 / 4 (0.00%)
    0 / 2 (0.00%)
    1 / 2 (50.00%)
    1 / 3 (33.33%)
    0 / 4 (0.00%)
    0 / 4 (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
    Gastrointestinal disorders
    Post-Operative Nausea and Vomiting
         subjects affected / exposed
    0 / 4 (0.00%)
    0 / 2 (0.00%)
    1 / 2 (50.00%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    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
    Musculoskeletal and connective tissue disorders
    Fracture of Left Medial Malleolus
         subjects affected / exposed
    0 / 4 (0.00%)
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    1 / 3 (33.33%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    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
    		 Cohort 1 - 0.05mg/kg Cohort 2 - 1.0 mg/kg/wk Cohort 3 - 2.0 mg/kg/wk Cohort 4 - 4.0 mg/kg/wk Cohort 5 - 10 mg/kg/wk Cohort 6 - 20mg/kg/wk
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    4 / 4 (100.00%)
    2 / 2 (100.00%)
    2 / 2 (100.00%)
    3 / 3 (100.00%)
    4 / 4 (100.00%)
    4 / 4 (100.00%)
    Vascular disorders
    Haematoma
         subjects affected / exposed
    1 / 4 (25.00%)
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
    1 / 4 (25.00%)
         occurrences all number
    1
    0
    0
    0
    0
    1
    Pallor
         subjects affected / exposed
    0 / 4 (0.00%)
    0 / 2 (0.00%)
    1 / 2 (50.00%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    0
    General disorders and administration site conditions
    Influenza like illness
         subjects affected / exposed
    0 / 4 (0.00%)
    0 / 2 (0.00%)
    2 / 2 (100.00%)
    0 / 3 (0.00%)
    1 / 4 (25.00%)
    0 / 4 (0.00%)
         occurrences all number
    0
    0
    2
    0
    1
    0
    Abasia
         subjects affected / exposed
    0 / 4 (0.00%)
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    1 / 4 (25.00%)
    0 / 4 (0.00%)
         occurrences all number
    0
    0
    0
    0
    0
    0
    Application site rash
         subjects affected / exposed
    0 / 4 (0.00%)
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
    1 / 4 (25.00%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    Catheter Site Pain
         subjects affected / exposed
    0 / 4 (0.00%)
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    1 / 4 (25.00%)
    0 / 4 (0.00%)
         occurrences all number
    0
    0
    0
    0
    1
    0
    Disease Progression
         subjects affected / exposed
    0 / 4 (0.00%)
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    1 / 4 (25.00%)
    1 / 4 (25.00%)
         occurrences all number
    0
    0
    0
    0
    1
    1
    Fatigue
         subjects affected / exposed
    0 / 4 (0.00%)
    0 / 2 (0.00%)
    1 / 2 (50.00%)
    0 / 3 (0.00%)
    1 / 4 (25.00%)
    0 / 4 (0.00%)
         occurrences all number
    0
    0
    1
    0
    1
    0
    Infusion related reaction
         subjects affected / exposed
    0 / 4 (0.00%)
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    1 / 4 (25.00%)
    0 / 4 (0.00%)
         occurrences all number
    0
    0
    0
    0
    1
    0
    Pyrexia
         subjects affected / exposed
    0 / 4 (0.00%)
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    1 / 3 (33.33%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    0
    Vaccination site pain
         subjects affected / exposed
    0 / 4 (0.00%)
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
    1 / 4 (25.00%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    Vessel puncture site haematoma
         subjects affected / exposed
    0 / 4 (0.00%)
    0 / 2 (0.00%)
    1 / 2 (50.00%)
    1 / 3 (33.33%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    0
    0
    1
    1
    0
    0
    Respiratory, thoracic and mediastinal disorders
    Asthma
         subjects affected / exposed
    0 / 4 (0.00%)
    1 / 2 (50.00%)
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    0
    1
    0
    0
    0
    0
    Cough
         subjects affected / exposed
    2 / 4 (50.00%)
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    2
    0
    0
    0
    0
    0
    Epistaxis
         subjects affected / exposed
    0 / 4 (0.00%)
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
    1 / 4 (25.00%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    Productive cough
         subjects affected / exposed
    0 / 4 (0.00%)
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
    1 / 4 (25.00%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    Injury, poisoning and procedural complications
    Fall
         subjects affected / exposed
    2 / 4 (50.00%)
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    2 / 3 (66.67%)
    0 / 4 (0.00%)
    1 / 4 (25.00%)
         occurrences all number
    2
    0
    0
    2
    0
    1
    Ankle Fracture
         subjects affected / exposed
    0 / 4 (0.00%)
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    1 / 3 (33.33%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    0
    Arthropod bite
         subjects affected / exposed
    0 / 4 (0.00%)
    0 / 2 (0.00%)
    1 / 2 (50.00%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    0
    Arthropod sting
         subjects affected / exposed
    1 / 4 (25.00%)
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    0
    0
    0
    0
    Contusion
         subjects affected / exposed
    0 / 4 (0.00%)
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    1 / 3 (33.33%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    0
    Excoriation
         subjects affected / exposed
    0 / 4 (0.00%)
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
    1 / 4 (25.00%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    Head injury
         subjects affected / exposed
    0 / 4 (0.00%)
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    1 / 3 (33.33%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    0
    Heat stroke
         subjects affected / exposed
    0 / 4 (0.00%)
    0 / 2 (0.00%)
    1 / 2 (50.00%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    0
    Lumbar vertebral fracture
         subjects affected / exposed
    0 / 4 (0.00%)
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    1 / 3 (33.33%)
    0 / 4 (0.00%)
    1 / 4 (25.00%)
         occurrences all number
    0
    0
    0
    1
    0
    1
    Post procedural haematoma
         subjects affected / exposed
    0 / 4 (0.00%)
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    1 / 3 (33.33%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    0
    Procedural Pain
         subjects affected / exposed
    2 / 4 (50.00%)
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    2
    0
    0
    0
    0
    0
    Vaccination complication
         subjects affected / exposed
    0 / 4 (0.00%)
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    1 / 3 (33.33%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    0
    Cardiac disorders
    Tachycardia
         subjects affected / exposed
    0 / 4 (0.00%)
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    1 / 3 (33.33%)
    0 / 4 (0.00%)
    2 / 4 (50.00%)
         occurrences all number
    0
    0
    0
    1
    0
    2
    Cardiomyopathy
         subjects affected / exposed
    0 / 4 (0.00%)
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    1 / 3 (33.33%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    0
    Sinus tachycardia
         subjects affected / exposed
    0 / 4 (0.00%)
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    1 / 3 (33.33%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    0
    Nervous system disorders
    Headache
         subjects affected / exposed
    2 / 4 (50.00%)
    1 / 2 (50.00%)
    2 / 2 (100.00%)
    0 / 3 (0.00%)
    2 / 4 (50.00%)
    1 / 4 (25.00%)
         occurrences all number
    2
    1
    2
    0
    2
    1
    Dizziness
         subjects affected / exposed
    0 / 4 (0.00%)
    0 / 2 (0.00%)
    1 / 2 (50.00%)
    1 / 3 (33.33%)
    0 / 4 (0.00%)
    1 / 4 (25.00%)
         occurrences all number
    0
    0
    1
    1
    0
    1
    Blood and lymphatic system disorders
    Platelet anisocytosis
         subjects affected / exposed
    0 / 4 (0.00%)
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    1 / 3 (33.33%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    0
    0
    0
    0
    0
    0
    Ear and labyrinth disorders
    Ear Pain
         subjects affected / exposed
    1 / 4 (25.00%)
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    0
    0
    0
    0
    Eye disorders
    Enteritis
         subjects affected / exposed
    0 / 4 (0.00%)
    0 / 2 (0.00%)
    1 / 2 (50.00%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    0
    Gastrointestinal disorders
    Nausea
         subjects affected / exposed
    0 / 4 (0.00%)
    0 / 2 (0.00%)
    1 / 2 (50.00%)
    1 / 3 (33.33%)
    0 / 4 (0.00%)
    1 / 4 (25.00%)
         occurrences all number
    0
    0
    1
    1
    0
    1
    Vomiting
         subjects affected / exposed
    0 / 4 (0.00%)
    0 / 2 (0.00%)
    1 / 2 (50.00%)
    1 / 3 (33.33%)
    1 / 4 (25.00%)
    0 / 4 (0.00%)
         occurrences all number
    0
    0
    1
    1
    1
    0
    Abdominal pain
         subjects affected / exposed
    0 / 4 (0.00%)
    1 / 2 (50.00%)
    0 / 2 (0.00%)
    1 / 3 (33.33%)
    1 / 4 (25.00%)
    0 / 4 (0.00%)
         occurrences all number
    0
    1
    0
    1
    1
    0
    Abdominal Pain - Upper
         subjects affected / exposed
    1 / 4 (25.00%)
    0 / 2 (0.00%)
    1 / 2 (50.00%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    1
    0
    0
    0
    Constipation
         subjects affected / exposed
    1 / 4 (25.00%)
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    0
    0
    0
    0
    Diarrhoea
         subjects affected / exposed
    0 / 4 (0.00%)
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    1 / 3 (33.33%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    0
    Lip Dry
         subjects affected / exposed
    0 / 4 (0.00%)
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
    1 / 4 (25.00%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    Skin and subcutaneous tissue disorders
    Drug eruption
         subjects affected / exposed
    0 / 4 (0.00%)
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
    1 / 4 (25.00%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    Skin irritation
         subjects affected / exposed
    1 / 4 (25.00%)
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    0
    0
    0
    0
    Renal and urinary disorders
    Enuresis
         subjects affected / exposed
    0 / 4 (0.00%)
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
    1 / 4 (25.00%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    Musculoskeletal and connective tissue disorders
    Back Pain
         subjects affected / exposed
    1 / 4 (25.00%)
    0 / 2 (0.00%)
    1 / 2 (50.00%)
    2 / 3 (66.67%)
    2 / 4 (50.00%)
    1 / 4 (25.00%)
         occurrences all number
    1
    0
    1
    2
    2
    1
    Myalgia
         subjects affected / exposed
    1 / 4 (25.00%)
    1 / 2 (50.00%)
    1 / 2 (50.00%)
    0 / 3 (0.00%)
    1 / 4 (25.00%)
    0 / 4 (0.00%)
         occurrences all number
    1
    1
    1
    0
    1
    0
    Arthralgia
         subjects affected / exposed
    1 / 4 (25.00%)
    0 / 2 (0.00%)
    1 / 2 (50.00%)
    1 / 3 (33.33%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    1
    1
    0
    0
    Coccydynia
         subjects affected / exposed
    0 / 4 (0.00%)
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    1 / 3 (33.33%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    0
    Musculoskeletal pain
         subjects affected / exposed
    1 / 4 (25.00%)
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    0
    0
    0
    0
    Osteopenia
         subjects affected / exposed
    1 / 4 (25.00%)
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    0
    0
    0
    0
    Pain in extremity
         subjects affected / exposed
    1 / 4 (25.00%)
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    1 / 4 (25.00%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    0
    0
    1
    0
    Infections and infestations
    Rhinitis
         subjects affected / exposed
    1 / 4 (25.00%)
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    1 / 3 (33.33%)
    4 / 4 (100.00%)
    1 / 4 (25.00%)
         occurrences all number
    1
    0
    0
    1
    4
    1
    Upper Respiratory Tract Infection
         subjects affected / exposed
    2 / 4 (50.00%)
    1 / 2 (50.00%)
    0 / 2 (0.00%)
    1 / 3 (33.33%)
    2 / 4 (50.00%)
    2 / 4 (50.00%)
         occurrences all number
    2
    1
    0
    1
    2
    2
    Bronchitis
         subjects affected / exposed
    0 / 4 (0.00%)
    1 / 2 (50.00%)
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    1 / 4 (25.00%)
    0 / 4 (0.00%)
         occurrences all number
    0
    1
    0
    0
    1
    0
    Hordeolum
         subjects affected / exposed
    0 / 4 (0.00%)
    1 / 2 (50.00%)
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    0
    1
    0
    0
    0
    0
    Incision site infection
         subjects affected / exposed
    0 / 4 (0.00%)
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    1 / 4 (25.00%)
    0 / 4 (0.00%)
         occurrences all number
    0
    0
    0
    0
    1
    0
    Nasopharyngitis
         subjects affected / exposed
    0 / 4 (0.00%)
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    1 / 4 (25.00%)
    0 / 4 (0.00%)
         occurrences all number
    0
    0
    0
    0
    1
    0
    Respiratory tract infection viral
         subjects affected / exposed
    0 / 4 (0.00%)
    0 / 2 (0.00%)
    1 / 2 (50.00%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    0
    Tinea infection
         subjects affected / exposed
    0 / 4 (0.00%)
    1 / 2 (50.00%)
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    0
    1
    0
    0
    0
    0
    Viral Infection
         subjects affected / exposed
    0 / 4 (0.00%)
    0 / 2 (0.00%)
    1 / 2 (50.00%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
    1 / 4 (25.00%)
         occurrences all number
    0
    0
    1
    0
    0
    1
    Metabolism and nutrition disorders
    Decreased appetite
         subjects affected / exposed
    0 / 4 (0.00%)
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    1 / 3 (33.33%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    0

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    18 Mar 2009
    Decreased duration of follow-up period from 40 to 14 weeks, decreased number of patients per each dose cohort, updated DSMB responsibilities, modified inclusion and exclusion criteria to expand patient qualification, modified study termination criteria, eliminated most overnight stays after dosing visits. Altered various data collection requirements in the Schedule of Assessments, including: frequency of ECG and PFT assessments, ECHO procedures, lab assessments, muscle function tests, blood and urine collection. Added 6MWT and changed drug diluent to normal saline.
    23 Jun 2009
    Added 2 additional dose cohorts with dosage of 10.0 and 20.0 mg/kg/wk. Added 10 day window to obtain Baseline StepWatch data, lab assessments, physical exam and vitals Added lab assessments at Week 3
    02 Mar 2010
    Added exon skipping as a measurement of exploratory efficacy Removed reference to full passive range of ankle dorsiflexion bilaterally throughout the protocol Specified that assays relevant to muscle biopsy were to be performed at GOSH

    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.
    Due to the small number of study participants, a single adverse event (AE) in 1 patient exceeds the reporting threshold of 5%. Refer to the "Post-Hoc Outcome Measures" for a summary of frequent and related AEs.

    Online references
    http://www.ncbi.nlm.nih.gov/pubmed/21784508
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