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    Clinical Trial Results:
    Phase 2, Non-Interventional, Clinical Study to Assess Dystrophin Levels in Subjects With Nonsense Mutation Duchenne Muscular Dystrophy who Have Been Treated With Ataluren for ≥9 Months

    Summary
    EudraCT number
    		 2019-001691-11
    Trial protocol
    		 Outside EU/EEA  
    Global end of trial date
    03 Jun 2019

    Results information
    Results version number
    		 v1(current)
    This version publication date
    20 May 2022
    First version publication date
    20 May 2022
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    PTC124-GD-046-DMD
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT03796637
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    PTC Therapeutics, Inc.
    Sponsor organisation address
    100 Corporate Court, South Plainfield, United States, NJ 07080
    Public contact
    Medical Information, PTC Therapeutics, Inc., +011 44 1-866-562-4620, medinfo@ptcbio.com
    Scientific contact
    Medical Information, PTC Therapeutics International Limited, +353 19068700, medinfo@ptcbio.com
    Paediatric regulatory details
    Is trial part of an agreed paediatric investigation plan (PIP)
    No
    Does article 45 of REGULATION (EC) No 1901/2006 apply to this trial?
    No
    Does article 46 of REGULATION (EC) No 1901/2006 apply to this trial?
    Yes
    Results analysis stage
    Analysis stage
    Final
    Date of interim/final analysis
    03 Jun 2019
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    03 Jun 2019
    Global end of trial reached?
    Yes
    Global end of trial date
    03 Jun 2019
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    The main objective of this trial was to assess the levels of dystrophin in ambulatory participants with nonsense mutation duchenne muscular dystrophy (nmDMD) currently being treated with ataluren for ≥9 months using a quantitative electrochemiluminescence (ECL) assay.
    Protection of trial subjects
    The study was conducted in accordance with the ethical principles that have their origins in the Declaration of Helsinki (revised version 2013) and in conformance with the International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use (ICH) Good Clinical Practice (GCP) guidance documents.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    11 Apr 2019
    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: 6
    Worldwide total number of subjects
    6
    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)
    4
    Adolescents (12-17 years)
    2
    Adults (18-64 years)
    0
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 -

    Pre-assignment
    Screening details
    		 Six ambulatory male participants with nmDMD who had been receiving ataluren were enrolled and treated in this study.

    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
    		 Ataluren
    Arm description
    		 Participants who had been receiving ataluren, were dosed daily 10 milligrams (mg)/kilogram (kg) in the morning, 10 mg/kg at midday, and 20 mg/kg in the evening, for ≥9 months from ongoing PTC-sponsored nmDMD clinical trials.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Ataluren
    Investigational medicinal product code
    PTC124
    Other name
    Pharmaceutical forms
    Oral suspension
    Routes of administration
    Oral use
    Dosage and administration details
    Ataluren was administered as per the dose and schedule specified in the arm.

    Number of subjects in period 1
    		Ataluren
    Started
    6
    Completed
    6

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Ataluren
    Reporting group description
    		 Participants who had been receiving ataluren, were dosed daily 10 milligrams (mg)/kilogram (kg) in the morning, 10 mg/kg at midday, and 20 mg/kg in the evening, for ≥9 months from ongoing PTC-sponsored nmDMD clinical trials.

    Reporting group values
    		 Ataluren Total
    Number of subjects
    		 6 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 0
        Children (2-11 years)
    		 4 4
        Adolescents (12-17 years)
    		 2 2
        Adults (18-64 years)
    		 0 0
        From 65-84 years
    		 0 0
        85 years and over
    		 0 0
    Age Continuous
    Units: years
        arithmetic mean (standard deviation)
    		 10.2 ± 2.04 -
    Sex: Female, Male
    Units: participants
        Female
    		 0 0
        Male
    		 6 6
    Ethnicity (NIH/OMB)
    Units: Subjects
        Hispanic or Latino
    		 0 0
        Not Hispanic or Latino
    		 6 6
        Unknown or Not Reported
    		 0 0
    Race (NIH/OMB)
    Units: Subjects
        American Indian or Alaska Native
    		 0 0
        Asian
    		 0 0
        Native Hawaiian or Other Pacific Islander
    		 0 0
        Black or African American
    		 0 0
        White
    		 6 6
        More than one race
    		 0 0
        Unknown or Not Reported
    		 0 0

    End points

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    End points reporting groups
    Reporting group title
    Ataluren
    Reporting group description
    		 Participants who had been receiving ataluren, were dosed daily 10 milligrams (mg)/kilogram (kg) in the morning, 10 mg/kg at midday, and 20 mg/kg in the evening, for ≥9 months from ongoing PTC-sponsored nmDMD clinical trials.

    Primary: Mean Dystrophin Levels as Measured by Electrochemiluminescence (ECL)

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    End point title
    		 Mean Dystrophin Levels as Measured by Electrochemiluminescence (ECL) [1]
    End point description
    The mean dystrophin protein levels were measured by ECL. Dystrophin levels are reported by muscle group (gastrocnemius, tibialis anterior, and across muscle locations). Results below the limit of quantitation were imputed as half of lower limit of quantitation (LLOQ). LLOQ = 0.5 micrograms (μg)/milliliter (mL). Intent-to-treat (ITT) population included all enrolled participants with a valid assessment of dystrophin level, as measured by ECL.
    End point type
    Primary
    End point timeframe
    Day 1 of biopsy
    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: Statistical analysis was not planned for this endpoint.
    End point values
    		 Ataluren
    Number of subjects analysed
    6
    Units: nanograms (ng)/mg
    arithmetic mean (standard deviation)
        Gastrocnemius
    0.0844 ± 0.05874
        Tibialis Anterior
    0.1002 ± 0.08060
        Across Muscle Locations
    0.1054 ± 0.08300
    No statistical analyses for this end point

    Secondary: Dystrophin Protein Levels as Determined by Immunohistochemistry

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    End point title
    		 Dystrophin Protein Levels as Determined by Immunohistochemistry
    End point description
    Dystrophin levels by IHC mean membrane stain density are reported by muscle group (gastrocnemius, tibialis anterior, and across muscle locations). ITT population included all enrolled participants with a valid assessment of dystrophin level, as measured by ECL.
    End point type
    Secondary
    End point timeframe
    Day 1 of biopsy
    End point values
    		 Ataluren
    Number of subjects analysed
    6
    Units: ng/mg
    arithmetic mean (standard deviation)
        Gastrocnemius
    0.28817 ± 0.220547
        Tibialis Anterior
    0.26578 ± 0.226510
        Across Muscle Locations
    0.30483 ± 0.218600
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Baseline (Day 1) up to Week 1
    Adverse event reporting additional description
    Safety population included all participants who received at least 1 dose of ataluren.
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    22.1
    Reporting groups
    Reporting group title
    Ataluren
    Reporting group description
    		 Participants who had been receiving ataluren, were dosed daily 10 mg/kg in the morning, 10 mg/kg at midday, and 20 mg/kg in the evening, for ≥9 months from ongoing PTC-sponsored nmDMD clinical trials.

    Serious adverse events
    		 Ataluren
    Total subjects affected by serious adverse events
         subjects affected / exposed
    0 / 6 (0.00%)
         number of deaths (all causes)
    0
         number of deaths resulting from adverse events
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 Ataluren
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    2 / 6 (33.33%)
    Injury, poisoning and procedural complications
    Procedural pain
         subjects affected / exposed
    2 / 6 (33.33%)
         occurrences all number
    2
    General disorders and administration site conditions
    Puncture site discharge
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    20 Mar 2019
    The overall reason for the amendment was to ensure that sufficient muscle tissue for analysis was obtained from the biopsy procedure.

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

    Limitations and caveats
    Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data.
    None reported
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    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
    As of 31 January 2023, all EU/EEA initial clinical trial applications must be submitted through CTIS . Updated EudraCT trials information and information on PIP/Art 46 trials conducted exclusively in third countries continues to be submitted through EudraCT and published on this website.

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