Key inclusion & exclusion criteria
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Inclusion Criteria:
- Completion of blinded study drug treatment in the previous Phase 3 study
(PTC124-GD-009-CF).
- Ability to provide written informed consent (parental/guardian consent if
applicable)/assent (if <18 years of age).
- In participants who are sexually active, willingness to abstain from sexual
intercourse or employ a barrier or medical method of contraception during ataluren
administration and the 4-week follow up period.
- Willingness and ability to comply with scheduled visits, drug administration plan,
study procedures, laboratory tests, and study restrictions.
Exclusion Criteria:
- Known hypersensitivity to any of the ingredients or excipients of the study drug (list
provided at study sites).
- Current pregnancy or lactating, or pregnancy or lactating during the previous Phase 3
study.
- Ongoing participation in any other therapeutic clinical trial.
- Prior or ongoing medical condition (for example, concomitant illness, psychiatric
condition, behavioral disorder, alcoholism, drug abuse), medical history, physical
findings, ECG findings, or laboratory abnormality that, in the Investigator's opinion,
could adversely affect the safety of the participant, makes it unlikely that the
course of treatment or follow up would be completed, or could impair the assessment of
study results.
Age minimum:
6 Years
Age maximum:
N/A
Gender:
All
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Secondary Outcome(s)
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Duration of Pulmonary Exacerbations as Defined by Modified Fuch's Criteria
[Time Frame: Weeks 43 up to 48 and Weeks 91 up to 96 (Total Study Weeks 91 up to 96 and Weeks 139 up to 144)]
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Duration of Disruptions in Activities of Daily Living Because of Pulmonary Symptoms
[Time Frame: Baseline (Week 1 [Total Study Week 48]) up to EOT (Week 96 [Total Study Week 144])]
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Percentage Change From Baseline in Percent-Predicted of FEF25-75 at Weeks 48 and 96
[Time Frame: Week 48 (Total Study Week 96), EOT (Week 96 [Total Study Week 144])]
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Percentage Change From Baseline in Percent-Predicted of FVC at Weeks 48 and 96
[Time Frame: Week 48 (Total Study Week 96), EOT (Week 96 [Total Study Week 144])]
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Rate of Study Drug Compliance
[Time Frame: Baseline (Week 1 [Total Study Week 48]) up to EOT (Week 96 [Total Study Week 144])]
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Change From Baseline for the Respiratory Domain Score of the Cystic Fibrosis (CF) Questionnaire-Revised (CFQ-R) at Weeks 48 and 96
[Time Frame: Baseline (Week 1 [Total Study Week 48]), Week 48 (Total Study Week 96), EOT (Week 96 [Total Study Week 144])]
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Predose Concentration of Ataluren
[Time Frame: Predose at Weeks 1, 16, 32, 48, 64, 80 and EOT (Week 96) (Total Study Weeks 48, 64, 80 96, 112, 128, and 144, respectively)]
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Rate of Pulmonary Exacerbations as Defined by Modified Fuch's Criteria Over 48 Weeks
[Time Frame: Baseline (Week 1 [Total Study Week 48]) up to Week 48 (Total Study Week 96)]
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Change From Baseline in Body Mass Index (BMI) at Weeks 48 and 96
[Time Frame: Baseline (Week 1 [Total Study Week 48]), Week 48 (Total Study Week 96), EOT (Week 96 [Total Study Week 144])]
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Number of Participants Who Required Interventions for Pulmonary Symptoms
[Time Frame: Baseline (Week 1 [Total Study Week 48]) up to EOT (Week 96 [Total Study Week 144])]
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Number of Participants With Disruptions in Activities of Daily Living Because of Pulmonary Symptoms
[Time Frame: Baseline (Week 1 [Total Study Week 48]) up to EOT (Week 96 [Total Study Week 144])]
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Percent-Predicted of Forced Expiratory Flow Between 25% and 75% of Expiration (FEF25-75) at Baseline
[Time Frame: Baseline (Week 1 [Total Study Week 48])]
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Change From Baseline in the Nasal Transepithelial Potential Difference (TEPD) at Week 48
[Time Frame: Baseline (Week 1 [Total Study Week 48]) and Week 48 (Total Study Week 96)]
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Number of Participants With Severe Pulmonary Exacerbations as Defined by Modified Fuch's Criteria
[Time Frame: Weeks 43 up to 48 and Weeks 91 up to 96 (Total Study Weeks 91 up to 96 and Weeks 139 up to 144)]
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Change From Baseline in the Concentration of Sweat Chloride at Week 48
[Time Frame: Baseline (Week 1 [Total Study Week 48]), Week 48 (Total Study Week 96)]
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Total Lung Computed Tomography (CT) Score at Weeks 48 and 96
[Time Frame: Week 48 (Total Study Week 96) and EOT (Week 96 [Total Study Week 144])]
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Number of Participants With Pulmonary Exacerbations as Defined by Modified Fuch's Criteria
[Time Frame: Baseline (Week 1 [Total Study Week 48]) up to Week 48 and EOT (Week 96) (Total Study Weeks 96 and 144)]
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Percent-Predicted of Forced Vital Capacity (FVC) at Baseline
[Time Frame: Baseline (Week 1 [Total Study Week 48])]
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Percentage Change From Baseline in Percent-Predicted of FEV1 at Weeks 48 and 96
[Time Frame: Week 48 (Total Study Week 96), End of Treatment (EOT) (Week 96 [Total Study Week 144])]
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Percentage of Predicted Function (Percent-Predicted) of Forced Expiratory Volume in 1 Second (FEV1) at Baseline
[Time Frame: Baseline (Week 1 [Total Study Week 48])]
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Change From Baseline in Body Weight at Weeks 48 and 96
[Time Frame: Baseline (Week 1 [Total Study Week 48]), Week 48 (Total Study Week 96), EOT (Week 96 [Total Study Week 144])]
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