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Study Name: Ataluren (PTC 124) Open Label Extension Study in Cystic Fibrosis
Study Type: Interventional
Intervention Category: CFTR Modulation
Study Sponsor: PTC
Study Phase: 3
Recruitment Status: Recruiting Completed
Study Drug(s): PTC124
Number of Participants Being Recruited: 190
Single / Multi-Center: Multi-Center
Cystic fibrosis (CF) is a genetic disorder caused by a mutation in the gene that makes the cystic fibrosis transmembrane conductance regulator (CFTR) protein. A specific type of mutation called a nonsense (premature stop codon) mutation is the cause of CF in approximately 10% of subjects with the disease. Ataluren (PTC124) is an orally delivered investigational drug that has the potential to overcome the effects of the nonsense mutation. This study is a Phase 3 extension trial that will evaluate the long-term safety of ataluren (PTC124) in adult and pediatric patients with nonsense mutation CF, as determined by adverse events and laboratory abnormalities. The study will also assess changes in pulmonary function, CF pulmonary exacerbations, health-related quality of life, antibiotic use for CF-related infections, CF-related disruptions to daily living, body weight, and CF pathophysiology.
Age: >= 6 Years
FEV1: 40 - 90 Percent Predicted
P. aeruginosa status: Not applicable
B. cepacia status: Not applicable
Other Primary Eligibility Requirements:

To be included in this study, subjects must have completed the placebo controlled phase 3 study of PTC (PTC124-009) and as noted for that study must have a documented nonsense mutation in at least 1 allele of the CFTR gene and a CF-causing mutation in the other CFTR allele, as determined by gene sequencing.
Note: Detailed eligibility criteria information may be available on If a specific trial listing for this trial is available, a link to the specific listing will be present in the "More Information" section below.
Sponsor Contact Information: Goetz, Diane
(908) 912-9256
Trial Specific Link on
Clinical Research Terms Glossary: Click here
The sites listed below are currently recruiting subjects. Please note that this list generally lists either the pediatric or adult program but not both even though both programs may be recruiting subjects. Also, this list does not include sites that may be participating in the study but are not yet open for recruitment. If you are interested in this study please contact your center for more information.
  • The Children's Hospital/University of Alabama at Birmingham (Pediatric), Birmingham, AL, 35233
    Contact Information:
        Hathorne, Heather
        Phone: (205) 638-9568
  • Miller Children's Hospital (Pediatric), Long Beach, CA, 90801
    Contact Information:
        O'Donnell, Nan
        Phone: (562) 933-5607
  • Stanford University Medical Center (Pediatric), Palo Alto, CA, 94304
    Contact Information:
        Dunn, Colleen
        Phone: (650) 736-0388
  • Children's Hospital Colorado (Pediatric), Aurora, CO, 80045
    Contact Information:
        Pardee, Churee
        Phone: (720) 777-6162
  • Emory University (Pediatric), Atlanta, GA, 30322
    Contact Information:
        Hunter, Eric
        Phone: (404) 785-3102
  • Ann and Robert H. Lurie Children's Hospital of Chicago (Pediatric), Chicago, IL, 60611
    Contact Information:
        Singh, Noopur
        Phone: (312) 227-6861
  • Children's Hospital Boston (Pediatric), Boston, MA, 02115
    Contact Information:
        Fowler, Robert
        Phone: (617) 355-1834
  • Johns Hopkins University (Pediatric), Baltimore, MD, 21287
    Contact Information:
        Callahan, Karen
        Phone: (443) 287-8983
  • St. Louis Children's Hospital Washington University (Pediatric), St. Louis, MO, 63110
    Contact Information:
        Burks, Patricia
        Phone: (314) 454-5175
  • University of North Carolina at Chapel Hill (Pediatric), Chapel Hill, NC, 27599
    Contact Information:
        Barlow, Carol
        Phone: (919) 966-9146
  • Beth Israel Medical Center (Pediatric), New York, NY, 10003
    Contact Information:
        DiBenedetto, Donna
        Phone: (212) 420-4100
  • Beth Israel Medical Center (Pediatric), New York, NY, 10003
    Contact Information:
        Grece, Carroll Anne
        Phone: (212) 420-4196
  • University Hospitals Case Medical Center/Rainbow Babies and Children's Hospital (Pediatric), Cleveland, OH, 44106
    Contact Information:
        Ksenich, Roberta (Bobbi)
        Phone: (216) 844-5733
  • University of Pittsburgh School of Medicine (Adult), Pittsburgh, PA, 15213
    Contact Information:
        Hartigan, Elizabeth
        Phone: (412) 692-7060
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