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Drug & research development » Clinical trials » NCT02139306

Study of Ataluren in Nonsense Mutation Cystic Fibrosis (ACT CF)

Official title: A Phase 3 Efficacy and Safety Study of Ataluren (PTC124®) in Patients With Nonsense Mutation Cystic Fibrosis

Clinical Trials gov number: NCT02139306

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Purpose: A Phase 3, international, multicenter, randomized, double-blind, placebo-controlled, efficacy and safety study of ataluren in patients with nonsense mutation cystic fibrosis (nmCF) not receiving chronic inhaled aminoglycosides.

Phase: 3

Type: Interventional, randomized, double-blind, parallel assignment

Study sponsor: PTC Therapeutics; Cystic Fibrosis Foundation Therapeutics-Therapeutics Development Network (CCFT-TDN); European Cystic Fibrosis Society-Clinical Trial Network (ECFS-CTN)

  • 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
  • Body weight ≥16 kg.
  • 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 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


Geographical Location
Argentina, Australia, Belgium, Brazil, Bulgaria, Canada, France, Israel, Germany, Italy, Netherlands, Poland, Spain, United Kingdom, United States

Number of Participants
101-1000 (≥6 years)

Primary Endpoint

FEV1 by spirometry (48 weeks)

Secondary Endpoint

Week 48

  • Rate of pulmonary exacerbations
  • Respiratory HRQL as assessed by the CFQ-R respiratory domain
  • Body weight and body mass index

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  • Experimental: Ataluren (PTC124®)

Oral powder for suspension taken 3 times per day (10-, 10-, and 20-mg/kg morning, midday and evening, respectively) for 48 weeks

  • Placebo

Matching placebo taken 3 times per day for 48 weeks

View Trial Results