Sermet‐Gaudelus 2010.

Methods	Randomised controlled trial. Cross‐over design. Duration: 2x 28‐day cycles of 14‐day on and 14‐day off. Location: multicentre (3 centres ‐ 1 in France and 2 in Belgium).
Participants	Children (at least 6 years old and no older than 18 years) with a nonsense mutation of CF. 30 participants (16 children aged 6 ‐ 12 years and 14 adolescents aged 13 ‐ 18 years) enrolled, no drop outs. Age (median (range)): 12 (6 ‐ 18) years. Gender: 16 males, 14 females.
Interventions	Ataluren in the form of vanilla flavoured granules (in aluminium foil sachets) to mix with either water, apple juice or milk to make a suspension for oral administration. Treatment taken 3x daily with dosage based on weight, but cycles had different dosing levels. Order of dose levels randomised. Cycle 1 (lower dosing regimen): 4 mg/kg in morning, 4 mg/kg at midday, 8 mg/kg in evening. Cycle 2 (higher dosing regimen): 10 mg/kg in morning, 10 mg/kg at midday, 20 mg/kg in evening.
Outcomes	Primary outcome: CFTR chloride transport (nasal TEPD). Secondary outcomes: ion channel activity, proportion of cells showing atypical CFTR protein expression, disease‐related clinical parameters, safety, compliance, pharmacokinetics, FEV₁, FVC, body weight, adverse events.
Notes	Trial drug provided by PTC Therapeutics. Powered to detect change in total chloride transport.

CF: cystic fibrosis
 CFTR: cystic fibrosis transmembrane conductance regulator
 FEV₁; forced expiratory volume in one second
 FVC: forced vital capacity
 TEPD: transepithelial potential difference