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    Clinical Trial Results:
    A Phase 2 Study of the Safety, Pharmacokinetics, and Pharmacodynamics of Ataluren (PTC124®) in Patients Aged ≥2 to <5 Years Old with Nonsense Mutation Dystrophinopathy

    Summary
    EudraCT number
    		 2016-001764-11
    Trial protocol
    		 Outside EU/EEA  
    Global end of trial date
    09 Feb 2018

    Results information
    Results version number
    		 v1(current)
    This version publication date
    03 Oct 2020
    First version publication date
    03 Oct 2020
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    PTC124-GD-030-DMD
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT02819557
    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)
    Yes
    EMA paediatric investigation plan number(s)
    		 EMEA-000115-PIP01-07
    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
    29 Mar 2018
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    09 Feb 2018
    Global end of trial reached?
    Yes
    Global end of trial date
    09 Feb 2018
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    Evaluate the safety of ataluren as measured by type, frequency, severity, timing, and relationship to study drug of treatment emergent adverse events (TEAEs), laboratory abnormalities, and electrocardiogram (ECGs).
    Protection of trial subjects
    This study was conducted in accordance with International Conference on Harmonisation (ICH) Good Clinical Practice, and the principles of the Declaration of Helsinki, in addition to following the laws and regulations of the country or countries in which a study is conducted.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    09 Jun 2016
    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 States: 14
    Worldwide total number of subjects
    14
    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)
    14
    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
    		 Participants aged ≥2 to <5 years old with Duchenne muscular dystrophy (DMD) caused by a nonsense mutation in the dystrophin gene were recruited for this study.

    Pre-assignment
    Screening details
    		 Participants in the Evaluable Population included all participants who received at least 1 dose of ataluren and had a baseline and at least 1 postbaseline measurement for Timed Function Test (TFT), North Star Ambulatory Assessment (NSAA), or response to the palatability of ataluren questions.

    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 were administered ataluren orally at a dose of 10 milligrams/kilograms (mg/kg) in the morning, 10 mg/kg at midday, and 20 mg/kg in the evening (for a total of 40 mg/kg/day) for up to 52 weeks. Dose was provided based upon the weight of each participant, which was assessed every 12 weeks.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Ataluren
    Investigational medicinal product code
    PTC124
    Other name
    Translarna
    Pharmaceutical forms
    Powder for oral suspension
    Routes of administration
    Oral use
    Dosage and administration details
    White to off-white powder for oral suspension.

    Number of subjects in period 1
    		Ataluren
    Started
    14
    Safety Population
    14
    Pharmacokinetics (PK) Population
    14
    Evaluable Population
    14
    Completed
    14

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Ataluren
    Reporting group description
    		 Participants were administered ataluren orally at a dose of 10 milligrams/kilograms (mg/kg) in the morning, 10 mg/kg at midday, and 20 mg/kg in the evening (for a total of 40 mg/kg/day) for up to 52 weeks. Dose was provided based upon the weight of each participant, which was assessed every 12 weeks.

    Reporting group values
    		 Ataluren Total
    Number of subjects
    		 14 14
    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)
    		 14 14
        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: years
        arithmetic mean (standard deviation)
    		 3.4 ± 0.76 -
    Sex: Female, Male
    Units: participants
        Female
    		 0 0
        Male
    		 14 14
    Race (NIH/OMB)
    Units: Subjects
        American Indian or Alaska Native
    		 0 0
        Asian
    		 3 3
        Native Hawaiian or Other Pacific Islander
    		 0 0
        Black or African American
    		 0 0
        White
    		 11 11
        More than one race
    		 0 0
        Unknown or Not Reported
    		 0 0
    Ethnicity (NIH/OMB)
    Units: Subjects
        Hispanic or Latino
    		 3 3
        Not Hispanic or Latino
    		 11 11
        Unknown or Not Reported
    		 0 0

    End points

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    End points reporting groups
    Reporting group title
    Ataluren
    Reporting group description
    		 Participants were administered ataluren orally at a dose of 10 milligrams/kilograms (mg/kg) in the morning, 10 mg/kg at midday, and 20 mg/kg in the evening (for a total of 40 mg/kg/day) for up to 52 weeks. Dose was provided based upon the weight of each participant, which was assessed every 12 weeks.

    Primary: Number of Participants With Treatment Emergent Adverse Events (TEAEs), TEAEs Leading to Discontinuation, and Serious Adverse Events (SAEs)

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    End point title
    		 Number of Participants With Treatment Emergent Adverse Events (TEAEs), TEAEs Leading to Discontinuation, and Serious Adverse Events (SAEs) [1]
    End point description
    A TEAE was any untoward medical occurrence or undesirable event that begins or worsens following administration of study drug, whether or not considered related to study drug by Investigator. An SAE was an adverse event (AE) resulting in any of the following outcomes or deemed significant for any other reason, death, initial or prolonged inpatient hospitalization, life-threatening experience (immediate risk of dying) or persistent or significant disability/incapacity not related to dystrophinopathy. An event was not reported as an SAE, if event was exclusively a relapse or expected change or progression of baseline dystrophinopathy. AEs included both SAEs and nonserious AEs. AEs classified according to NCI CTCAE v4.0 and coded using MedDRA. Population included all participants who received at least 1 dose of ataluren (Safety Population). A summary of SAEs and all non-serious AEs, regardless of causality, is located in the Reported Adverse Events section.
    End point type
    Primary
    End point timeframe
    Baseline up to Week 56
    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 analyses not applicable for this endpoint.
    End point values
    		 Ataluren
    Number of subjects analysed
    14
    Units: participants
        At least 1 TEAE
    14
        Mild TEAE
    5
        Moderate TEAE
    8
        Severe TEAE
    1
        TEAE Related to Study Drug
    5
        TEAE Leading to Participant Study Discontinuation
    0
        Serious TEAE
    0
    No statistical analyses for this end point

    Primary: Number of Participants With a Clinically Meaningful Abnormal Clinical Laboratory (Biochemistry, Hematology, and Urinalysis) Parameter

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    End point title
    		 Number of Participants With a Clinically Meaningful Abnormal Clinical Laboratory (Biochemistry, Hematology, and Urinalysis) Parameter [2]
    End point description
    Clinical laboratory results that were considered clinically meaningful were determined by the Investigator and Sponsor. Biochemistry parameters: sodium, potassium, chloride, bicarbonate, blood urea nitrogen, creatinine, magnesium, calcium, phosphorus, uric acid, glucose, total protein, albumin, bilirubin (total, direct, and indirect), AST, ALT, gamma-glutamyl transferase, creatine kinase, lactate dehydrogenase, ALP, total cholesterol, high-density lipoprotein, low-density lipoprotein, triglycerides, and cystatin C. Hematology parameters: WBC count with differential, hemoglobin, hematocrit, other red cell parameters, and platelet count. Urinalysis parameters: pH, specific gravity, glucose, ketones, blood, protein, urobilinogen, bilirubin, nitrite, and leukocyte esterase. Population included all participants who received at least 1 dose of ataluren (Safety Population). A summary of other non-serious AEs and all serious AEs, regardless of causality is located in Reported AE section.
    End point type
    Primary
    End point timeframe
    Baseline up to Week 56
    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: Statistical analyses not applicable for this endpoint.
    End point values
    		 Ataluren
    Number of subjects analysed
    14
    Units: participants
    0
    No statistical analyses for this end point

    Primary: Number of Participants With a Clinically Meaningful Abnormal ECG Test Results

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    End point title
    		 Number of Participants With a Clinically Meaningful Abnormal ECG Test Results [3]
    End point description
    ECG results that were considered clinically meaningful were to be determined by the Investigator. Population included all participants who received at least 1 dose of ataluren (Safety Population). A summary of other non-serious AEs and all serious AEs, regardless of causality is located in Reported AE section.
    End point type
    Primary
    End point timeframe
    Baseline up to Week 56
    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: Statistical analyses not applicable for this endpoint.
    End point values
    		 Ataluren
    Number of subjects analysed
    14
    Units: participants
    0
    No statistical analyses for this end point

    Primary: Number of Participants with a Dose-Limiting Toxicity as Measured by Hepatic and Renal Toxicity

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    End point title
    		 Number of Participants with a Dose-Limiting Toxicity as Measured by Hepatic and Renal Toxicity [4]
    End point description
    Dose-limiting toxicity was measured through clinical evaluations for potential hepatic and renal toxicities. The clinical evaluations included the following: • Hepatic: The participant’s medical history, hepatitis screening results, all clinical blood values (particularly serum bilirubin, gamma-glutamyl transferase [GGT], aspartate aminotransferase [AST], and alanine aminotransferase [ALT] values), and all concomitant medications were reviewed. • Renal: The participant’s medical history, all clinical blood and urine renal values, serum electrolytes, medications, and potential pre- or post-renal conditions were reviewed. Population included all participants who received at least 1 dose of ataluren (Safety Population).
    End point type
    Primary
    End point timeframe
    Baseline up to Week 56
    Notes
    [4] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: Statistical analyses not applicable for this endpoint.
    End point values
    		 Ataluren
    Number of subjects analysed
    14
    Units: participants
    0
    No statistical analyses for this end point

    Secondary: Pharmacokinetics: Maximum Observed Plasma Concentration From Time Zero up to 6 Hours After the Morning Dose (Cmax0-6hr)

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    End point title
    		 Pharmacokinetics: Maximum Observed Plasma Concentration From Time Zero up to 6 Hours After the Morning Dose (Cmax0-6hr)
    End point description
    Ataluren concentrations in plasma were analyzed using a validated high performance liquid chromatography-tandem mass spectrometry (HPLC-MS/MS) method. Population included all participants who received at least 1 dose of ataluren and had at least 1 PK concentration datum (PK Population).
    End point type
    Secondary
    End point timeframe
    0 (predose), 1, 2, 4, and 6 (postdose) hours on Days 1 and 28
    End point values
    		 Ataluren
    Number of subjects analysed
    14
    Units: microgram/milliliter (µg/mL)
    arithmetic mean (standard deviation)
        Day 1
    15.95 ± 9.51
        Day 28
    12.54 ± 4.43
    No statistical analyses for this end point

    Secondary: Pharmacokinetics: Time to Reach Maximum Observed Plasma Concentration From Time Zero up to 6 Hours After the Morning Dose (Tmax0-6hr)

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    End point title
    		 Pharmacokinetics: Time to Reach Maximum Observed Plasma Concentration From Time Zero up to 6 Hours After the Morning Dose (Tmax0-6hr)
    End point description
    Ataluren concentrations in plasma were analyzed using a validated HPLC-MS/MS method. Population included all participants who received at least 1 dose of ataluren and had at least 1 PK concentration datum (PK Population).
    End point type
    Secondary
    End point timeframe
    0 (predose), 1, 2, 4, and 6 (postdose) hours on Days 1 and 28
    End point values
    		 Ataluren
    Number of subjects analysed
    14
    Units: hours
    arithmetic mean (standard deviation)
        Day 1
    3.84 ± 1.82
        Day 28
    2.72 ± 1.98
    No statistical analyses for this end point

    Secondary: Area Under the Plasma Concentration Time Curve From Time Zero up to 10 Hours After the Morning Dose (AUC0-10hr)

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    End point title
    		 Area Under the Plasma Concentration Time Curve From Time Zero up to 10 Hours After the Morning Dose (AUC0-10hr)
    End point description
    Ataluren concentrations in plasma were analyzed using a validated HPLC-MS/MS method. AUC0-10hr was measured using the linear trapezoidal rule during the ascending portion of the curve and the log-trapezoidal rule during the descending portion of the curve. Population included all participants who received at least 1 dose of ataluren and had at least 1 PK concentration datum (PK Population).
    End point type
    Secondary
    End point timeframe
    0 (predose), 1, 2, 4, 6, 8, and 10 (postdose) hours on Days 1 and 28
    End point values
    		 Ataluren
    Number of subjects analysed
    14
    Units: hour*μg/mL
    arithmetic mean (standard deviation)
        Day 1
    101.64 ± 58.52
        Day 28
    82.13 ± 27.43
    No statistical analyses for this end point

    Secondary: Pharmacokinetics: Concentration at the End of the First (Morning) Dose Interval (Ctrough6hr)

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    End point title
    		 Pharmacokinetics: Concentration at the End of the First (Morning) Dose Interval (Ctrough6hr)
    End point description
    Ataluren concentrations in plasma were analyzed using a validated HPLC-MS/MS method. Population included all participants who received at least 1 dose of ataluren and had at least 1 PK concentration datum (PK Population).
    End point type
    Secondary
    End point timeframe
    0 (predose), 1, 2, 4, and 6 (postdose) hours on Days 1 and 28
    End point values
    		 Ataluren
    Number of subjects analysed
    14
    Units: microgram per milliliter (µg/ml)
    arithmetic mean (standard deviation)
        Day 1
    9.12 ± 8.88
        Day 28
    5.43 ± 3.15
    No statistical analyses for this end point

    Secondary: Change From Baseline in Proximal Muscle Function as Assessed by Speed During TFTs

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    End point title
    		 Change From Baseline in Proximal Muscle Function as Assessed by Speed During TFTs
    End point description
    TFTs included time to stand from supine position (rise to standing), time to run/walk 10 meters (m), and time to ascend/descend 4 stairs. A decrease from baseline reflects faster completion of the functional task and, thus, better muscle function. If the time taken to perform a test exceeded 30 seconds or if a participant could not perform the test due to disease progression (PD), a value of 30 seconds was used. Population included all participants who received at least 1 dose of ataluren and had a baseline and at least 1 postbaseline measurement for TFT, NSAA, or response to the palatability of ataluren questions (Evaluable Population) and had evaluable data for the applicable timed function test.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 28 (W28) and Week 52 (W52)
    End point values
    		 Ataluren
    Number of subjects analysed
    14
    Units: seconds
    arithmetic mean (standard deviation)
        Rise to Standing, Baseline
    7.2 ± 7.21
        Rise to Standing Change from Baseline at W28, n=13
    -3.1 ± 6.47
        Rise to Standing, Change from Baseline at Week 52
    -3.1 ± 6.50
        Walk/Run 10 m, Baseline
    6.6 ± 2.37
        Walk/Run 10 m, Change from Baseline at W28, n=13
    -0.8 ± 1.54
        Walk/Run 10 m, Change from Baseline at Week 52
    -1.1 ± 1.35
        Ascend 4 Stairs, Baseline
    7.1 ± 6.95
        Ascend 4 Stairs, Change from Baseline at W28, n=13
    -1.8 ± 4.85
        Ascend 4 Stairs, Change from Baseline at Week 52
    -2.6 ± 5.00
        Descend 4 Stairs, Baseline, n=13
    7.5 ± 3.95
        Descend 4 Stairs Change from Baseline at W28, n=12
    -0.6 ± 1.93
        Descend 4 Stairs Change from Baseline at W52, n=13
    -2.2 ± 2.58
    No statistical analyses for this end point

    Secondary: Change From Baseline in Physical Function as Measured by the NSAA

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    End point title
    		 Change From Baseline in Physical Function as Measured by the NSAA
    End point description
    NSAA: 17 activities including items assessing abilities necessary to remain functionally ambulant (ie, ability to rise from floor, to go from lying to sitting/sitting to standing, and that are known to progressively deteriorate); items that can be partly present in DMD early stages (ie, assessing head raise and standing on heels); and a number of activities such as hopping, jumping and running. Since the boys were <5 years old, revised 16, 8, and 3-point (pt), scales were used over the 17 pt scale. Scores for evaluations=0 (Unable to achieve independently), 1 (Modified method but achieved goal independent of physical assistance) or 2 (Normal, no obvious modification of activity). Maximum total score for the 16-pt scale=32, 8-pt scale=16 and 3-pt scale=6. If an activity couldn’t be performed due to PD/loss of ambulation, a score of 0 was assigned. Change from Baseline=subtracting Baseline value from Week 28 and Week 52 values. Population=Evaluable Population with evaluable NSAA data.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 28 and Week 52
    End point values
    		 Ataluren
    Number of subjects analysed
    14
    Units: units on a scale
    arithmetic mean (standard deviation)
        16-Point Scale, Baseline
    16.0 ± 4.66
        16-Point Scale Change from Baseline at Wk 28, n=13
    3.5 ± 3.43
        16-Point Scale, Change from Baseline at Week 52
    5.5 ± 4.43
        8-Point Scale, Baseline
    10.5 ± 2.56
        8-Point Scale, Change from Baseline at Wk 28, n=13
    1.5 ± 1.39
        8-Point Scale, Change from Baseline at Week 52
    2.3 ± 2.13
        3-Point Scale, Baseline
    5.4 ± 0.63
        3-Point Scale, Change from Baseline at Wk 28, n=13
    0.5 ± 0.78
        3-Point Scale, Change from Baseline at Week 52
    0.3 ± 0.73
    No statistical analyses for this end point

    Secondary: Change From Baseline in Height of Participants at Weeks 4, 16, 28, 40, 52, and 56

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    End point title
    		 Change From Baseline in Height of Participants at Weeks 4, 16, 28, 40, 52, and 56
    End point description
    Population included all participants who received at least 1 dose of ataluren (Safety Population) and had evaluable height data.
    End point type
    Secondary
    End point timeframe
    Baseline, Weeks 4, 16, 28, 40, 52, and 56
    End point values
    		 Ataluren
    Number of subjects analysed
    14
    Units: centimeters
    arithmetic mean (standard deviation)
        Baseline
    99.43 ± 5.278
        Change at Week 4
    0.83 ± 1.595
        Change at Week 16
    1.84 ± 1.466
        Change at Week 28
    3.11 ± 1.386
        Change at Week 40, n=13
    3.82 ± 1.506
        Change at Week 52
    5.95 ± 2.096
        Change at Week 56
    6.04 ± 2.075
    No statistical analyses for this end point

    Secondary: Change From Baseline in Weight of Participants at Weeks 4, 16, 28, 40, 52, and 56

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    End point title
    		 Change From Baseline in Weight of Participants at Weeks 4, 16, 28, 40, 52, and 56
    End point description
    Population included all participants who received at least 1 dose of ataluren (Safety Population) and had evaluable weight data.
    End point type
    Secondary
    End point timeframe
    Baseline, Weeks 4, 16, 28, 40, 52, and 56
    End point values
    		 Ataluren
    Number of subjects analysed
    14
    Units: kg
    arithmetic mean (standard deviation)
        Baseline
    16.99 ± 3.257
        Change at Week 4
    -0.04 ± 0.502
        Change at Week 16
    0.73 ± 0.974
        Change at Week 28
    1.16 ± 0.940
        Change at Week 40, n=13
    1.39 ± 0.882
        Change at Week 52
    1.90 ± 1.259
        Change at Week 56
    2.13 ± 1.340
    No statistical analyses for this end point

    Secondary: Change From Baseline in Body Mass Index of Participants at Weeks 4, 16, 28, 40, 52, and 56

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    End point title
    		 Change From Baseline in Body Mass Index of Participants at Weeks 4, 16, 28, 40, 52, and 56
    End point description
    Body mass index is an estimate of body fat based on body weight divided by height squared. Population included all participants who received at least 1 dose of ataluren (Safety Population) and had evaluable body mass index data.
    End point type
    Secondary
    End point timeframe
    Baseline, Weeks 4, 16, 28, 40, 52, and 56
    End point values
    		 Ataluren
    Number of subjects analysed
    14
    Units: kilograms per square meter (kg/m^2)
    arithmetic mean (standard deviation)
        Baseline
    17.094 ± 2.2196
        Change at Week 4
    -0.346 ± 0.6804
        Change at Week 16
    0.026 ± 0.3822
        Change at Week 28
    0.030 ± 0.5567
        Change at Week 40, n=13
    0.008 ± 0.6399
        Change at Week 52
    -0.186 ± 0.9237
        Change at Week 56
    0.015 ± 1.2161
    No statistical analyses for this end point

    Secondary: Ataluren Palatability Characteristics as Determined by a Parent/Caregiver Questionnaire

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    End point title
    		 Ataluren Palatability Characteristics as Determined by a Parent/Caregiver Questionnaire
    End point description
    To assess palatability characteristics, participants/parents or guardians were asked to provide a response of "Strongly disagree", "Disagree", "Neither agree or disagree", "Agree", or "Strongly Agree" to the following 3 questions: Question 1. "Is the medicine palatable?" Question 2. "On the basis of reaction / facial expression of your child, do you think that the medication is pleasant?" Question 3."You sometimes have problems in giving the medication to your child because he/she refuses to take it or throws it up?" Population included all participants who received at least 1 dose of ataluren and had a baseline and at least 1 postbaseline measurement for TFT, NSAA, or response to the palatability of ataluren questions (Evaluable Population).
    End point type
    Secondary
    End point timeframe
    Baseline up to Week 28
    End point values
    		 Ataluren
    Number of subjects analysed
    14
    Units: Count of participants
        Question 1, Strongly disagree
    0
        Question 1, Disagree
    0
        Question 1, Neither Agree nor Disagree
    2
        Question 1, Agree
    0
        Question 1, Strongly agree
    0
        Question 1, No Response
    12
        Question 2, Strongly disagree
    0
        Question 2, Disagree
    2
        Question 2, Neither Agree nor Disagree
    2
        Question 2, Agree
    6
        Question 2, Strongly Agree
    4
        Question 2, No response
    0
        Question 3, Strongly Disagree
    5
        Question 3, Disagree
    7
        Question 3, Neither agree or disagree
    0
        Question 3, Agree
    2
        Question 3, Strongly Agree
    0
        Question 3, No response
    0
    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 56
    Adverse event reporting additional description
    Adverse events were collected from all participants who received at least 1 dose of ataluren (Safety Population).
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    19.0
    Reporting groups
    Reporting group title
    Ataluren
    Reporting group description
    		 Participants were administered ataluren orally at a dose of 10 mg/kg in the morning, 10 mg/kg at midday, and 20 mg/kg in the evening (for a total of 40 mg/kg/day) for up to 52 weeks. Dose was provided based upon the weight of each participant, which was assessed every 12 weeks.

    Serious adverse events
    		 Ataluren
    Total subjects affected by serious adverse events
         subjects affected / exposed
    0 / 14 (0.00%)
         number of deaths (all causes)
    0
         number of deaths resulting from adverse events
    Frequency threshold for reporting non-serious adverse events: 0%
    Non-serious adverse events
    		 Ataluren
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    14 / 14 (100.00%)
    Investigations
    Hepatic Enzyme Increased
         subjects affected / exposed
    1 / 14 (7.14%)
         occurrences all number
    1
    Injury, poisoning and procedural complications
    Fall
         subjects affected / exposed
    1 / 14 (7.14%)
         occurrences all number
    3
    Contusion
         subjects affected / exposed
    1 / 14 (7.14%)
         occurrences all number
    1
    Arthropod Bite
         subjects affected / exposed
    2 / 14 (14.29%)
         occurrences all number
    2
    Tibia Fracture
         subjects affected / exposed
    1 / 14 (7.14%)
         occurrences all number
    1
    Nervous system disorders
    Headache
         subjects affected / exposed
    1 / 14 (7.14%)
         occurrences all number
    1
    General disorders and administration site conditions
    Fatigue
         subjects affected / exposed
    1 / 14 (7.14%)
         occurrences all number
    1
    Gait Disturbance
         subjects affected / exposed
    1 / 14 (7.14%)
         occurrences all number
    1
    Pyrexia
         subjects affected / exposed
    6 / 14 (42.86%)
         occurrences all number
    14
    Gastrointestinal disorders
    Constipation
         subjects affected / exposed
    2 / 14 (14.29%)
         occurrences all number
    2
    Abdominal Pain Upper
         subjects affected / exposed
    2 / 14 (14.29%)
         occurrences all number
    3
    Flatulence
         subjects affected / exposed
    2 / 14 (14.29%)
         occurrences all number
    2
    Nausea
         subjects affected / exposed
    1 / 14 (7.14%)
         occurrences all number
    1
    Stomatitis
         subjects affected / exposed
    1 / 14 (7.14%)
         occurrences all number
    1
    Vomiting
         subjects affected / exposed
    3 / 14 (21.43%)
         occurrences all number
    6
    Respiratory, thoracic and mediastinal disorders
    Cough
         subjects affected / exposed
    3 / 14 (21.43%)
         occurrences all number
    6
    Epistaxis
         subjects affected / exposed
    1 / 14 (7.14%)
         occurrences all number
    1
    Rhinorrhoea
         subjects affected / exposed
    1 / 14 (7.14%)
         occurrences all number
    1
    Tonsillar Hypertrophy
         subjects affected / exposed
    1 / 14 (7.14%)
         occurrences all number
    1
    Skin and subcutaneous tissue disorders
    Hypertrichosis
         subjects affected / exposed
    1 / 14 (7.14%)
         occurrences all number
    1
    Rash
         subjects affected / exposed
    3 / 14 (21.43%)
         occurrences all number
    3
    Rash Generalised
         subjects affected / exposed
    1 / 14 (7.14%)
         occurrences all number
    1
    Urticaria
         subjects affected / exposed
    1 / 14 (7.14%)
         occurrences all number
    1
    Psychiatric disorders
    Abnormal Behaviour
         subjects affected / exposed
    1 / 14 (7.14%)
         occurrences all number
    1
    Enuresis
         subjects affected / exposed
    1 / 14 (7.14%)
         occurrences all number
    1
    Insomnia
         subjects affected / exposed
    1 / 14 (7.14%)
         occurrences all number
    1
    Renal and urinary disorders
    Chromaturia
         subjects affected / exposed
    1 / 14 (7.14%)
         occurrences all number
    2
    Musculoskeletal and connective tissue disorders
    Arthralgia
         subjects affected / exposed
    1 / 14 (7.14%)
         occurrences all number
    1
    Pain In Extremity
         subjects affected / exposed
    1 / 14 (7.14%)
         occurrences all number
    3
    Infections and infestations
    Bronchitis Viral
         subjects affected / exposed
    1 / 14 (7.14%)
         occurrences all number
    1
    Conjunctivitis
         subjects affected / exposed
    1 / 14 (7.14%)
         occurrences all number
    1
    Croup Infectious
         subjects affected / exposed
    1 / 14 (7.14%)
         occurrences all number
    1
    Ear Infection
         subjects affected / exposed
    5 / 14 (35.71%)
         occurrences all number
    5
    Gastroenteritis
         subjects affected / exposed
    1 / 14 (7.14%)
         occurrences all number
    1
    Gastroenteritis Norovirus
         subjects affected / exposed
    1 / 14 (7.14%)
         occurrences all number
    1
    Gingival Abscess
         subjects affected / exposed
    1 / 14 (7.14%)
         occurrences all number
    1
    Hordeolum
         subjects affected / exposed
    1 / 14 (7.14%)
         occurrences all number
    1
    Influenza
         subjects affected / exposed
    2 / 14 (14.29%)
         occurrences all number
    3
    Nasopharyngitis
         subjects affected / exposed
    4 / 14 (28.57%)
         occurrences all number
    8
    Pharyngitis Streptococcal
         subjects affected / exposed
    1 / 14 (7.14%)
         occurrences all number
    2
    Respiratory Tract Infection
         subjects affected / exposed
    1 / 14 (7.14%)
         occurrences all number
    1
    Rhinitis
         subjects affected / exposed
    1 / 14 (7.14%)
         occurrences all number
    1
    Upper Respiratory Tract Infection
         subjects affected / exposed
    2 / 14 (14.29%)
         occurrences all number
    2
    Viral Rash
         subjects affected / exposed
    1 / 14 (7.14%)
         occurrences all number
    1
    Metabolism and nutrition disorders
    Decreased Appetite
         subjects affected / exposed
    1 / 14 (7.14%)
         occurrences all number
    1
    Hyperlipidaemia
         subjects affected / exposed
    1 / 14 (7.14%)
         occurrences all number
    1
    Metabolic Acidosis
         subjects affected / exposed
    1 / 14 (7.14%)
         occurrences all number
    1

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? No

    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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