PTC Therapeutics has initiated a Phase 3 clinical trial of ataluren in patients with nonsense mutation cystic fibrosis (nmCF). The trial is referred to as the Ataluren Confirmatory Trial in Cystic Fibrosis, or ACT CF.

ACT CF is an international, randomized, double-blind, placebo-controlled, efficacy and safety study of ataluren in patients with nmCF not receiving chronic inhaled aminoglycosides. The primary endpoint is lung function as measured by relative change in % predicted FEV1. The study is planning to enroll 208 patients. Each participant will be randomly assigned to receive either ataluren three times per day (10-, 10-, 20-mg/kg) or placebo (a substance that looks and tastes like ataluren but does not actually contain the drug), for 48 weeks. It is anticipated that participants who have successfully completed this study will have the opportunity to receive ataluren in an extension study, except in countries where ataluren may be commercially available for the treatment of nmCF.

Requirements for Taking Part in this Study

To be eligible for this trial, potential participants must meet the following requirements:

  • Be six years of age or older
  • Have no chronic use of inhaled aminoglycosides (e.g., tobramycin) or use of inhaled aminoglycosides within 4 months prior to screening
  • Body weight ≥16 kg
  • Documentation of a sweat chloride >60 mEq/L
  • Documentation of the presence of a nonsense mutation in at least 1 allele of the CFTR gene, as determined by genotyping
  • 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
  • Resting oxygen saturation (as measured by pulse oximetry) ≥92% on room air
  • Have not participated in the previous Phase 3 clinical trial

Complete inclusion and exclusion criteria and additional details about this clinical trial are available at and will be explained in detail by the physicians conducting the study. For questions, please contact PTC Therapeutics Patient and Professional Advocacy at (866) 282-5873 or

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