• Restore CFTR ProteinCompleted with Results

  Phase 3 study of Ataluren in people with CF who have a nonsense mutation and are not taking aminoglycosides , protocol number PTC124-GD-021-CF

  This study evaluated the safety and effectiveness of the drug Ataluren (PTC124®) compared with placebo. This study was for people with CF who have a nonsense mutation and were not receiving chronic inhaled aminoglycosides.  

  • Age:

    6 Years and Older

  • Mutation(s):

    One Copy F508del or No Copies F508del

  • FEV1% Predicted:

    60 to 90%

  • Number of Visits:

    10

  • Length of Participation:

    56 weeks

• Restore CFTR ProteinCompleted with Results

  Ataluren (PTC 124) in Cystic Fibrosis , protocol number PTC124-GD-009-CF

  This trial looked at the effectiveness and safety of ataluren in people with CF who have a nonsense mutation of cystic fibrosis.

  • Age:

    6 Years and Older

  • Mutation(s):

    One Copy F508del or No Copies F508del

  • FEV1% Predicted:

    40 to 90%

  • Number of Visits:

    15

  • Length of Participation:

    48 weeks

Studies in this tool are multi-center studies facilitated by the Cystic Fibrosis Therapeutics Development Network. For a complete list of cystic fibrosis related studies, visit www.clinicaltrials.gov.