Intervention: Ataluren
Trial phase: Phase 3
ATC code: M09AX03
SMILES: O=C(O)c1cccc(-c2noc(-c3ccccc3F)n2)c1
Sex: All
Age groups: Child | Adult | Older Adult
Healthy volunteers: No
Participants at risk: 120
Group description: Participants received ataluren TID: 10 mg/kg of body weight with breakfast, 10 mg/kg with lunch, and 20 mg/kg with dinner (total daily dose 40 mg/kg). Treatment continued for 48 weeks, after which participants were followed for 4 weeks.
Eligibility criteria:
Inclusion Criteria:

Ability to provide written informed consent (parental/guardian consent and participant assent if <18 years of age)
Age ≥6 years
Body weight ≥16 kg
Abnormal nasal transepithelial potential difference (TEPD) total chloride conductance (a less electrically negative value than -5 millivolts (mV) for total chloride conductance [Δchloride-free+isoproterenol])
Sweat chloride >40 milliequivalents/liter (mEq/L)
Documentation of the simultaneous presence of a nonsense mutation in at least 1 allele of the CFTR gene and a CF-causing mutation in the other CFTR allele, as determined by gene sequencing from a laboratory certified by the College of American Pathologists (CAP), under the Clinical Laboratory Improvement Act/Amendment (CLIA), or by an equivalent organization
Verification that a blood sample has been drawn for confirmation of the presence of a nonsense mutation in the CFTR gene
Ability to perform a valid, reproducible spirometry test using the study-specific spirometer with demonstration of an FEV1 ≥40% and ≤90% of predicted for age, gender, and height
Resting oxygen saturation (as measured by pulse oximetry) ≥92% on room air
Documentation by VivoMetrics that the participant has satisfactorily completed a 24-hour LifeShirt® cough frequency assessment
Confirmed screening laboratory values within the central laboratory ranges (hepatic, adrenal, renal, serum electrolytes, and reproduction [women only] parameters)
In participants who are sexually active, willingness to abstain from sexual intercourse or employ a barrier or medical method of contraception during the study drug administration and 4-week follow-up period
Willingness and ability to comply with scheduled visits, drug administration plan, study restrictions, and study procedures

Exclusion Criteria:

Known hypersensitivity to any of the ingredients or excipients of the study drug
Any change (initiation, change in type of drug, dose modification, schedule modification, interruption, discontinuation, or reinitiation) in a chronic treatment/prophylaxis regimen for CF or for CF-related conditions within 4 weeks prior to start of study treatment
Exposure to another investigational drug within 4 weeks prior to start of study treatment
Treatment with systemic aminoglycoside antibiotics at the time of the Baseline TEPD assessment
Treatment with intravenous antibiotics within 3 weeks prior to start of study treatment
History of solid organ or hematological transplantation
Ongoing immunosuppressive therapy (other than corticosteroids)
Ongoing warfarin, phenytoin, or tolbutamide therapy
Ongoing participation in any other therapeutic clinical trial
Major complications of lung disease (including massive hemoptysis, pneumothorax, or pleural effusion) within 8 weeks prior to start of study treatment
Evidence of pulmonary exacerbation or acute upper or lower respiratory tract infection (including viral illnesses) within 3 weeks prior to randomization
Known portal hypertension
Positive hepatitis B surface antigen, hepatitis C antibody test, or human immunodeficiency virus (HIV) test
Pregnancy or breast-feeding
Current smoker or a smoking history of ≥10 pack-years (number of cigarette packs/day * number of years smoked)
Prior or ongoing medical condition (for example, concomitant illness, alcoholism, drug abuse, psychiatric condition), medical history, physical findings, electrocardiography 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.