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

This study is ongoing, but not recruiting participants.
Cystic Fibrosis Foundation Therapeutics
European Cystic Fibrosis Society-Clinical Trial Network (ECFS-CTN)
Information provided by (Responsible Party):
PTC Therapeutics Identifier:
First received: May 13, 2014
Last updated: November 25, 2015
Last verified: November 2015
This is 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.

Condition Intervention Phase
Cystic Fibrosis Drug: Ataluren (PTC124®) Drug: Placebo Phase 3

Study Type: Interventional
Study Design: Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Phase 3 Efficacy and Safety Study of Ataluren (PTC124®) in Patients With Nonsense Mutation Cystic Fibrosis

Resource links provided by NLM:

Further study details as provided by PTC Therapeutics:

Primary Outcome Measures:
  • FEV1 by spirometry [ Time Frame: 48 weeks ]

Secondary Outcome Measures:
  • Rate of pulmonary exacerbations (modified Fuchs criteria) [ Time Frame: 48 weeks ]
  • Respiratory HRQL as assessed by the CFQ-R respiratory domain [ Time Frame: 48 weeks ]
  • Body weight and BMI [ Time Frame: 48 weeks ]

Other Outcome Measures:
  • FVC and FEF25-75 by spirometry [ Time Frame: 48 weeks ]
  • Incidence and duration of pulmonary exacerbations [ Time Frame: 48 weeks ]
  • Other HRQL domains as assessed by the CFQ-R [ Time Frame: 48 weeks ]
  • Concentrations of liver enzyme tests (AST, ALT, GGT) [ Time Frame: 48 weeks ]
  • Fecal calprotectin level [ Time Frame: 48 weeks ]
  • New Pseudomonas aeruginosa lung infection [ Time Frame: 48 weeks ]
  • Safety profile characterized by type, frequency, severity, timing, and relationship to study drug of treatment-emergent adverse events, laboratory abnormalities, and ECG abnormalities [ Time Frame: 48 weeks ]
  • Study drug compliance as assessed by quantification of unused study drug [ Time Frame: 48 weeks ]
  • Trough ataluren plasma concentrations [ Time Frame: 48 weeks ]

Estimated Enrollment: 208
Study Start Date: June 2014
Estimated Study Completion Date: November 2016
Estimated Primary Completion Date: November 2016 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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
Drug: Ataluren (PTC124®)
Placebo Comparator: Placebo
Matching placebo taken 3 times per day for 48 weeks
Drug: Placebo


Ages Eligible for Study:   6 Years and older   (Child, Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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 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

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 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 months 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, 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
  Contacts and Locations
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Please refer to this study by its identifier: NCT02139306

  Show 88 Study Locations
Sponsors and Collaborators
PTC Therapeutics
Cystic Fibrosis Foundation Therapeutics
European Cystic Fibrosis Society-Clinical Trial Network (ECFS-CTN)
Study Director: Joseph McIntosh, MD PTC Therapeutics
  More Information

Additional Information:
Responsible Party: PTC Therapeutics Identifier: NCT02139306     History of Changes
Other Study ID Numbers: PTC124-GD-021-CF
2013-004581-34 ( EudraCT Number )
Study First Received: May 13, 2014
Last Updated: November 25, 2015

Additional relevant MeSH terms:
Cystic Fibrosis
Pathologic Processes
Pancreatic Diseases
Digestive System Diseases
Lung Diseases
Respiratory Tract Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases processed this record on August 17, 2017