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    Clinical Trial Results:
    Phase 3 Extension Study of Ataluren (PTC124) in Patients with Nonsense Mutation Cystic Fibrosis

    Summary
    EudraCT number
    		 2014-005355-83
    Trial protocol
    		 BE   IT   ES   NL   FR   BG   GR   GB  
    Global end of trial date
    02 Jun 2017

    Results information
    Results version number
    		 v1(current)
    This version publication date
    30 Apr 2020
    First version publication date
    30 Apr 2020
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    PTC124-GD-021e-CF
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT02456103
    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, Inc., +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-PIP02-09
    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
    28 Jul 2017
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    02 Jun 2017
    Global end of trial reached?
    Yes
    Global end of trial date
    02 Jun 2017
    Was the trial ended prematurely?
    Yes
    General information about the trial
    Main objective of the trial
    The primary objective of this Phase 3 extension study was to obtain long-term safety data to augment the overall safety database. The secondary objectives was to augment the efficacy data collected in the double-blind study (PTC124-GD-021-CF; NCT02139306).
    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 of Edinburgh, Scotland, 2000) and in conformance with the International Council for Harmonisation (ICH) Good Clinical Practice (GCP) guidance documents.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    31 Aug 2015
    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
    Argentina: 6
    Country: Number of subjects enrolled
    Australia: 14
    Country: Number of subjects enrolled
    Belgium: 6
    Country: Number of subjects enrolled
    Brazil: 1
    Country: Number of subjects enrolled
    Bulgaria: 6
    Country: Number of subjects enrolled
    Canada: 6
    Country: Number of subjects enrolled
    France: 19
    Country: Number of subjects enrolled
    Germany: 19
    Country: Number of subjects enrolled
    Greece: 5
    Country: Number of subjects enrolled
    Israel: 16
    Country: Number of subjects enrolled
    Italy: 33
    Country: Number of subjects enrolled
    Netherlands: 3
    Country: Number of subjects enrolled
    Poland: 14
    Country: Number of subjects enrolled
    Spain: 23
    Country: Number of subjects enrolled
    United Kingdom: 2
    Country: Number of subjects enrolled
    United States: 73
    Worldwide total number of subjects
    246
    EEA total number of subjects
    130
    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)
    28
    Adolescents (12-17 years)
    68
    Adults (18-64 years)
    150
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 -

    Pre-assignment
    Screening details
    		 All eligible participants, including those who received placebo in the double-blind study (PTC124-GD-021-CF; NCT02139306), were enrolled to this open-label extension study. To avoid interruption in treatment, when possible, Screening/Baseline for this extension study was to occur on the same day as the End-of-Study visit for the double-blind 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 were administered ataluren orally at a dose of 10 milligrams/grams (mg/kg) in the morning, 10 mg/kg at midday, and 20 mg/kg in the evening for up to 96 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
    Ataluren will be provided as a vanilla-flavored powder to be mixed with water or milk.

    Number of subjects in period 1
    		Ataluren
    Started
    246
    As-Treated Population
    245
    Intent-to-treat (ITT) Population
    244
    Completed
    0
    Not completed
    246
         Adverse event, serious fatal
    1
         Physician decision
    1
         Consent withdrawn by subject
    24
         Study Closure
    207
         Adverse event, non-fatal
    4
         Abnormal Laboratory Value
    2
         Required prohibited medications
    5
         Lost to follow-up
    1
         Protocol deviation
    1

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Overall Study
    Reporting group description
    		 -

    Reporting group values
    		 Overall Study Total
    Number of subjects
    		 246 246
    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)
    		 28 28
        Adolescents (12-17 years)
    		 68 68
        Adults (18-64 years)
    		 150 150
        From 65-84 years
    		 0 0
        85 years and over
    		 0 0
    Age Continuous
    Units: years
        arithmetic mean (standard deviation)
    		 23.12 ( 10.86 ) -
    Sex: Female, Male
    Units: participants
        Female
    		 113 113
        Male
    		 133 133
    Race/Ethnicity, Customized
    Units: Subjects
        Asian
    		 4 4
        White
    		 236 236
        White-Arabic/North African Heritage
    		 1 1
        Non-White
    		 5 5
    Subject analysis sets

    Subject analysis set title
    Ataluren (As-Treated Population)
    Subject analysis set type
    		 Safety analysis
    Subject analysis set description
    Participants who received at least 1 dose of ataluren.

    Subject analysis set title
    Ataluren (ITT Population)
    Subject analysis set type
    		 Intention-to-treat
    Subject analysis set description
    Participants who received at least 1 dose of ataluren and have at least 1 postbaseline efficacy assessment.

    Subject analysis sets values
    		 Ataluren (As-Treated Population) Ataluren (ITT Population)
    Number of subjects
    245
    244
    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)
    28
    28
        Adolescents (12-17 years)
    67
    67
        Adults (18-64 years)
    150
    149
        From 65-84 years
    0
    0
        85 years and over
    0
    0
    Age Continuous
    Units: years
        arithmetic mean (standard deviation)
    23.1 ( 10.88 )
    23.2 ( 10.9 )
    Sex: Female, Male
    Units: participants
        Female
    113
    112
        Male
    132
    132
    Race/Ethnicity, Customized
    Units: Subjects
        Asian
    4
    4
        White
    235
    234
        White-Arabic/North African Heritage
    1
    1
        Non-White
    5
    5

    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/grams (mg/kg) in the morning, 10 mg/kg at midday, and 20 mg/kg in the evening for up to 96 weeks.

    Subject analysis set title
    Ataluren (As-Treated Population)
    Subject analysis set type
    		 Safety analysis
    Subject analysis set description
    Participants who received at least 1 dose of ataluren.

    Subject analysis set title
    Ataluren (ITT Population)
    Subject analysis set type
    		 Intention-to-treat
    Subject analysis set description
    Participants who received at least 1 dose of ataluren and have at least 1 postbaseline efficacy assessment.

    Primary: Number of Participants With Treatment Emergent Adverse Events (TEAEs)

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    End point title
    		 Number of Participants With Treatment Emergent Adverse Events (TEAEs) [1]
    End point description
    TEAE: 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. Serious adverse event (SAE): an adverse event (AE) resulting in any of 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. Except for cystic fibrosis (CF) pulmonary exacerbations, an event wasn’t reported as an SAE, if event was exclusively a relapse or an expected change or progression of baseline CF. AEs included both SAEs and nonserious AEs. AEs classified according to NCI CTCAE v3.0 and coded using MedDRA. Population included participants who received at least 1 dose of ataluren (As-Treated Population). A summary of SAEs and all nonserious AEs, regardless of causality, is located in the Reported Adverse Events section.
    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: Statistical analyses not applicable for this endpoint.
    End point values
    		 Ataluren (As-Treated Population)
    Number of subjects analysed
    245
    Units: participants
        At least 1 TEAE
    222
        Mild TEAE
    28
        Moderate TEAE
    147
        Severe TEAE
    46
        Life-Threatening TEAE
    1
        Fatal TEAE
    0
        Serious TEAE
    89
        TEAE Unrelated to Study Drug
    140
        TEAE Unlikely Related to Study Drug
    55
        TEAE Possibly Related to Study Drug
    23
        TEAE Probably Related to Study Drug
    4
    No statistical analyses for this end point

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

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    End point title
    		 Number of Participants With a Clinically Meaningful Abnormal Clinical Laboratory (Serum Biochemistry, Hematology, and Urinalysis) Parameter [2]
    End point description
    Clinical laboratory results considered clinically meaningful were determined by Investigator. Serum biochemistry parameters: sodium, potassium, chloride, bicarbonate, blood urea nitrogen, creatinine, magnesium, calcium, phosphorus, uric acid, glucose, total protein, albumin, globulin, bilirubin, creatine kinase, lactate dehydrogenase, ALT, AST, gamma glutamyl transferase, ALP, total cholesterol, high-density lipoprotein, low-density lipoprotein, triglycerides, cystatin C. Hematology parameters: WBC count, hemoglobin, hematocrit, mean corpuscular volume, mean corpuscular hemoglobin, red cell count with morphology, platelet count. Urinalysis parameters: pH, specific gravity, glucose, ketones, blood, protein, creatinine, urobilinogen, bilirubin, nitrite, leukocyte esterase. Population included participants who received at least 1 dose of ataluren (As-Treated Population). A summary of all SAEs/nonserious AEs, regardless of causality, is located in the Reported Adverse Events section.
    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: Statistical analyses not applicable for this endpoint.
    End point values
    		 Ataluren (As-Treated Population)
    Number of subjects analysed
    245
    Units: participants
    0
    No statistical analyses for this end point

    Secondary: Change From Baseline in Percent-Predicted Forced Expiratory Volume in 1 Second (FEV1) as Measured by Spirometry at Week 24

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    End point title
    		 Change From Baseline in Percent-Predicted Forced Expiratory Volume in 1 Second (FEV1) as Measured by Spirometry at Week 24
    End point description
    Pulmonary function of percent-predicted FEV1 was measured using a spirometer. FEV1 is the volume of air that can forcibly be blown out in 1 second. Each percent-predicted FEV1 was based gender, age, and the height value obtained at the same study visit. The percentage of change in percent-predicted of FEV1 was calculated as follows: (percent-predicted FEV1 - Baseline percent-predicted FEV1/Baseline percent-predicted FEV1)*100. Population included participants who received at least 1 dose of ataluren and have at least 1 postbaseline efficacy assessment (ITT Population) and had evaluable FEV1 data.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 24
    End point values
    		 Ataluren (ITT Population)
    Number of subjects analysed
    233
    Units: percentage of predicted FEV1
    arithmetic mean (standard deviation)
        Baseline (n=233)
    60.219 ( 17.6163 )
        Change from Baseline at Week 24 (n=205)
    0.015 ( 6.7180 )
    No statistical analyses for this end point

    Secondary: Change From Baseline in Percent-Predicted of Forced Vital Capacity (FVC) as Measured by Spirometry at Week 24

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    End point title
    		 Change From Baseline in Percent-Predicted of Forced Vital Capacity (FVC) as Measured by Spirometry at Week 24
    End point description
    Pulmonary function of FVC was measured using a spirometer. FVC is the volume of air that can forcibly be blown out. Each percent-predicted FVC was based gender, age, and the height value obtained at the same study visit. The percentage of change in percent-predicted of FVC was calculated as follows: (percent-predicted FVC - Baseline percent-predicted FVC/Baseline percent-predicted FVC)*100. Population included participants who received at least 1 dose of ataluren and have at least 1 postbaseline efficacy assessment (ITT Population) and had evaluable FVC data.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 24
    End point values
    		 Ataluren (ITT Population)
    Number of subjects analysed
    233
    Units: percentage of predicted FVC
    arithmetic mean (standard deviation)
        Baseline (n=233)
    75.249 ( 15.8508 )
        Change from Baseline at Week 24 (n=205)
    0.166 ( 6.5276 )
    No statistical analyses for this end point

    Secondary: Change From Baseline in Forced Expiratory Flow Between 25% and 75% of Expiration (FEF25-75) as Measured by Spirometry at Week 24

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    End point title
    		 Change From Baseline in Forced Expiratory Flow Between 25% and 75% of Expiration (FEF25-75) as Measured by Spirometry at Week 24
    End point description
    Pulmonary function of FEF25-75 was measured using a spirometer. FEF25-75 is the forced expiratory flow between 25% and 75% of vital capacity. Each percent-predicted FEF25-75 was based gender, age, and the height value obtained at the same study visit. The percentage of change in percent-predicted of FEF25-75 was calculated as follows: (percent-predicted FEF25-75 - Baseline percent-predicted FEF25-75/Baseline percent-predicted FEF25-75)*100. Participants who received at least 1 dose of ataluren and have at least 1 postbaseline efficacy assessment (ITT Population) and had evaluable FEF25-75 data.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 24
    End point values
    		 Ataluren (ITT Population)
    Number of subjects analysed
    233
    Units: percentage of FEF25-75
    arithmetic mean (standard deviation)
        Baseline (n=233)
    38.099 ( 22.0980 )
        Change from Baseline at Week 24 (n=205)
    0.698 ( 13.1241 )
    No statistical analyses for this end point

    Secondary: Rate of Pulmonary Exacerbations as Defined by Modified Fuch's Criteria Over 48 Weeks

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    End point title
    		 Rate of Pulmonary Exacerbations as Defined by Modified Fuch's Criteria Over 48 Weeks
    End point description
    A modified Fuchs’ exacerbation was defined as an event requiring treatment with or without intravenous antibiotics for any 4 of the following 12 symptoms: change in sputum; new or increased hemoptysis; increased cough; increased dyspnea; fatigue; temperature >38°C; anorexia; sinus pain; change in sinus discharge; change in physical examination of the chest; decrease in pulmonary function by 10 percent or more from a previously recorded value; or radiographic changes indicative of pulmonary function. The 48-week rate = (the total number of events/ treatment duration by week)*48. Population included participants who received at least 1 dose of ataluren and have at least 1 postbaseline efficacy assessment (ITT Population).
    End point type
    Secondary
    End point timeframe
    Baseline up to Week 48
    End point values
    		 Ataluren (ITT Population)
    Number of subjects analysed
    Units: exacerbations
        arithmetic mean (standard deviation)
    1.051 ( 2.1654 )
    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
    Adverse events collected from participants who received at least 1 dose of ataluren (As-Treated Population).
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 17.0
    Dictionary version
    17.0
    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 up to 96 weeks.

    Serious adverse events
    		 Ataluren
    Total subjects affected by serious adverse events
         subjects affected / exposed
    89 / 245 (36.33%)
         number of deaths (all causes)
    1
         number of deaths resulting from adverse events
    1
    Investigations
    Forced expiratory volume decreased
         subjects affected / exposed
    2 / 245 (0.82%)
         occurrences causally related to treatment / all
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    Protein urine present
         subjects affected / exposed
    1 / 245 (0.41%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Pseudomonas test positive
         subjects affected / exposed
    1 / 245 (0.41%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Pulmonary function test decreased
         subjects affected / exposed
    1 / 245 (0.41%)
         occurrences causally related to treatment / all
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    Squamous cell carcinoma of the cervix
    Additional description: This is a sex-specific adverse event that only affects female participants.
         subjects affected / exposed [1]
    1 / 113 (0.88%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Congenital, familial and genetic disorders
    Cystic fibrosis lung
         subjects affected / exposed
    1 / 245 (0.41%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Nervous system disorders
    Depressed level of consciousness
         subjects affected / exposed
    1 / 245 (0.41%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Pregnancy, puerperium and perinatal conditions
    Pregnancy
    Additional description: This is a sex-specific adverse event that only affects female participants.
         subjects affected / exposed [2]
    1 / 113 (0.88%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Gastrointestinal disorders
    Abdominal pain lower
         subjects affected / exposed
    1 / 245 (0.41%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    Distal intestinal obstruction syndrome
         subjects affected / exposed
    1 / 245 (0.41%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Duodenal ulcer
         subjects affected / exposed
    1 / 245 (0.41%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Enteritis
         subjects affected / exposed
    1 / 245 (0.41%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Intestinal obstruction
         subjects affected / exposed
    3 / 245 (1.22%)
         occurrences causally related to treatment / all
    0 / 3
         deaths causally related to treatment / all
    0 / 0
    Pancreatitis
         subjects affected / exposed
    1 / 245 (0.41%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    Respiratory, thoracic and mediastinal disorders
    Acute respiratory failure
         subjects affected / exposed
    1 / 245 (0.41%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 1
    Aspiration
         subjects affected / exposed
    1 / 245 (0.41%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Asthma
         subjects affected / exposed
    1 / 245 (0.41%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Dyspnoea exertional
         subjects affected / exposed
    1 / 245 (0.41%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Haemoptysis
         subjects affected / exposed
    4 / 245 (1.63%)
         occurrences causally related to treatment / all
    0 / 4
         deaths causally related to treatment / all
    0 / 0
    Renal and urinary disorders
    Nephrolithiasis
         subjects affected / exposed
    2 / 245 (0.82%)
         occurrences causally related to treatment / all
    1 / 2
         deaths causally related to treatment / all
    0 / 0
    Renal colic
         subjects affected / exposed
    2 / 245 (0.82%)
         occurrences causally related to treatment / all
    2 / 2
         deaths causally related to treatment / all
    0 / 0
    Renal failure acute
         subjects affected / exposed
    1 / 245 (0.41%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Infections and infestations
    Bronchopulmonary aspergillosis allergic
         subjects affected / exposed
    1 / 245 (0.41%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Device related infection
         subjects affected / exposed
    1 / 245 (0.41%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Gastroenteritis viral
         subjects affected / exposed
    1 / 245 (0.41%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Infective pulmonary exacerbation of cystic fibrosis
         subjects affected / exposed
    63 / 245 (25.71%)
         occurrences causally related to treatment / all
    0 / 85
         deaths causally related to treatment / all
    0 / 0
    Mycobacterium abscessus infection
         subjects affected / exposed
    1 / 245 (0.41%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Peritonitis
         subjects affected / exposed
    1 / 245 (0.41%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Pneumonia
         subjects affected / exposed
    1 / 245 (0.41%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Pseudomonas infection
         subjects affected / exposed
    1 / 245 (0.41%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Respiratory syncytial virus infection
         subjects affected / exposed
    1 / 245 (0.41%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Sinusitis
         subjects affected / exposed
    1 / 245 (0.41%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Viral pericarditis
         subjects affected / exposed
    1 / 245 (0.41%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Appendicitis
         subjects affected / exposed
    1 / 245 (0.41%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Metabolism and nutrition disorders
    Dehydration
         subjects affected / exposed
    1 / 245 (0.41%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Notes
    [1] - The number of subjects exposed to this adverse event is less than the total number of subjects exposed to this adverse event. These numbers are expected to be equal.
    Justification: This is a sex-specific adverse event that only affects female participants.
    [2] - The number of subjects exposed to this adverse event is less than the total number of subjects exposed to this adverse event. These numbers are expected to be equal.
    Justification: This is a sex-specific adverse event that only affects female participants.
    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
    191 / 245 (77.96%)
    Investigations
    Forced expiratory volume decreased
         subjects affected / exposed
    16 / 245 (6.53%)
         occurrences all number
    18
    Nervous system disorders
    Headache
         subjects affected / exposed
    16 / 245 (6.53%)
         occurrences all number
    28
    General disorders and administration site conditions
    Pyrexia
         subjects affected / exposed
    13 / 245 (5.31%)
         occurrences all number
    14
    Gastrointestinal disorders
    Nausea
         subjects affected / exposed
    14 / 245 (5.71%)
         occurrences all number
    18
    Respiratory, thoracic and mediastinal disorders
    Cough
         subjects affected / exposed
    32 / 245 (13.06%)
         occurrences all number
    47
    Haemoptysis
         subjects affected / exposed
    14 / 245 (5.71%)
         occurrences all number
    35
    Infections and infestations
    Infective pulmonary exacerbation of cystic fibrosis
         subjects affected / exposed
    136 / 245 (55.51%)
         occurrences all number
    224
    Influenza
         subjects affected / exposed
    13 / 245 (5.31%)
         occurrences all number
    16
    Sinusitis
         subjects affected / exposed
    25 / 245 (10.20%)
         occurrences all number
    29
    Upper respiratory tract infection
         subjects affected / exposed
    21 / 245 (8.57%)
         occurrences all number
    26
    Viral upper respiratory tract infection
         subjects affected / exposed
    30 / 245 (12.24%)
         occurrences all number
    40

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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.
    This study was terminated early because the CF data from the double-blind CF Study PTC124-GD-021-CF (NCT02139306) did not meet endpoints.
    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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