Clinical trial NCT00237380 (phase 2, status: completed)
Conditions: cystic fibrosis
ICD codes: E84.9, Z14.1, E84.0, E84.11, E84.8, E84.19, P09.4
Drugs: ataluren
SMILES: OC(=O)C1=CC=CC(=C1)C1=NOC(=N1)C1=CC=CC=C1F
Eligibility criteria:

        Inclusion Criteria:

          -  Diagnosis of CF based on documented evidence of a conclusively abnormal sweat test
             (sweat chloride >60 milliequivalents/litre [mEq/liter]).

          -  Abnormal chloride secretion as measured by TEPD (a less than -5 mV TEPD assessment of
             chloride secretion with chloride-free amiloride and isoproterenol).

          -  Presence of a nonsense mutation in one of the alleles of the CFTR gene.

          -  Age ≥18 years.

          -  Body weight ≥40 kg.

          -  Forced expiratory volume in 1 second (FEV1) ≥40% of predicted for age, gender, and
             height (Knudson standards).

          -  Oxygen saturation (as measured by pulse oximetry) ≥92% on room air.

          -  Willingness of male and female participants, if not surgically sterile, to abstain
             from sexual intercourse or employ a barrier or medical method of contraception during
             the study drug administration and follow-up periods.

          -  Negative pregnancy test (for females of childbearing potential).

          -  Willingness and ability to comply with scheduled visits, drug administration plan,
             study procedures, and study restrictions.

          -  Ability to provide written informed consent.

        Exclusion Criteria:

          -  Prior or ongoing medical condition, 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.

          -  Ongoing acute illness including acute upper or lower respiratory infections within 2
             weeks before start of study treatment.

          -  History of major complications of lung disease within 2 months prior to start of study
             treatment.

          -  Abnormalities on screening chest x-ray suggesting clinically significant active
             pulmonary disease other than CF, or new, significant abnormalities that may be
             indicative of clinically significant active pulmonary involvement secondary to CF.

          -  Positive hepatitis B surface antigen, hepatitis C antibody test, or human
             immunodeficiency virus (HIV) test.

          -  Hemoglobin <10 grams per deciliter (g/dL).

          -  Serum albumin <2.5 g/dL.

          -  Abnormal liver function (serum alanine aminotransferase [ALT], aspartate
             aminotransferase [AST], gamma-glutamyl transferase [GGT], alkaline phosphatase,
             lactate dehydrogenase [LDH], or total bilirubin > upper limit of normal).

          -  Abnormal renal function (serum creatinine >1.5 times upper limit of normal).

          -  Pregnancy or breast-feeding.

          -  History of solid organ or hematological transplantation.

          -  Exposure to another investigational drug within 14 days prior to start of study
             treatment.

          -  Ongoing participation in any other therapeutic clinical trial.

          -  Ongoing use of thiazolidinedione peroxisome proliferator-activated receptor gamma
             (PPAR γ) agonists, eg, rosiglitazone (Avandia® or equivalent) or pioglitazone (Actos®
             or equivalent)

          -  Change in intranasal medications (including use of corticosteroids, cromolyn,
             ipratropium bromide, phenylephrine, or oxymetazoline) within 14 days prior to start of
             study treatment.

          -  Change in treatment with systemic or inhaled corticosteroids within 14 days prior to
             start of study treatment.

          -  Use or requirement for inhaled gentamicin or amikacin within 14 days prior to start of
             study treatment or during study treatment.

          -  Requirement for systemic aminoglycoside antibiotics within 14 days prior to start of
             study treatment.