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: 140
Group description: Participants received ataluren as oral powder for suspension at the dosages of 10, 10, and 20-mg/kg at morning, midday and evening, respectively for 48 weeks of treatment duration or until treatment discontinuation.
Eligibility criteria:
Inclusion Criteria:

Evidence of signed and dated informed consent/assent document(s) indicating that the subject (and/or his parent/legal guardian) has been informed of all pertinent aspects of the trial
Age >=6 years.
Body weight >=16 kg.
Sweat chloride >60 milliequivalent per liter (mEq/L)
Documentation of the presence of a nonsense mutation in at least 1 allele of the cystic fibrosis transmembrane conductance regulator (CFTR) gene, as determined by genotyping performed at a laboratory certified by the College of American Pathologists (CAP), or under the Clinical Laboratory Improvement Act/Amendment (CLIA), or by an equivalent organization
Verification that a blood sample has been drawn for sequencing of 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
Demonstration at Visit 2 of a valid %-predicted FEV1 within 15% of the Screening % predicted FEV1 value
Resting oxygen saturation (as measured by pulse oximetry) >=92% on room air.
Confirmed screening laboratory values within pre-specified ranges
In subjects 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 60-day follow-up period
Willingness and ability to comply with all study procedures and assessments, including 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
Previous participation in the Phase 3 trial of ataluren (PTC124-GD-009-CF).
Any change (initiation, change in type of drug, dose modification, schedule modification, interruption, discontinuation, or re-initiation) in a chronic treatment/prophylaxis regimen for Cystic Fibrosis (CF) or for CF-related conditions within 4 weeks prior to screening
Chronic use of inhaled aminoglycosides (eg, tobramycin) or use of inhaled aminoglycosides within 4 weeks prior to screening.
Exposure to another investigational drug within 4 weeks prior to screening
Ongoing participation in any other therapeutic clinical trial
Evidence of pulmonary exacerbation or acute upper or lower respiratory tract infection (including viral illnesses) within 3 weeks prior to screening
Treatment with intravenous antibiotics within 3 weeks prior to screening
Ongoing immunosuppressive therapy (other than corticosteroids)
Ongoing warfarin, phenytoin, or tolbutamide therapy
History of solid organ or hematological transplantation
Major complications of lung disease (including massive hemoptysis, pneumothorax, or pleural effusion) within 8 weeks prior to screening
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 x number of years smoked).
Prior or ongoing medical condition (eg, concomitant illness, alcoholism, drug abuse, psychiatric condition), medical history, physical findings, electrocardiogram (ECG) findings, or laboratory abnormality that, in the investigator's opinion, could adversely affect the safety of the subject, makes it unlikely that the course of treatment or follow-up would be completed, or could impair the assessment of study results