• Restore CFTR ProteinCompleted with Results

  Phase 2 study of PTI-428 drug in people with CF ages 18 and older who have two copies of the F508del CFTR mutation , protocol number Proteostasis PTI-428-06

  This study evaluated the safety and tolerability of the drug PTI-428. This study was for people with CF ages 18 and older who have two copies of the F508del CFTR mutation and were already taking tezacaftor/ivacaftor (Symdeko®). A key goal of the study was to determine if PTI-428 could increase the amount of CFTR protein produced in people who were already taking tezacaftor/ivacaftor (Symdeko®).

  • Age:

    18 Years and Older

  • Mutation(s):

    Two Copies F508del

  • FEV1% Predicted:

    40 to 90%

  • Number of Visits:

    10

  • Length of Participation:

    74 days

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.