VX-121 Combination Therapy in Participants With Cystic Fibrosis (CF) Who Are Homozygous for F508del, Heterozygous for F508del and a Gating (F/G) or Residual Function (F/RF) Mutation, or Have At Least 1 Other Triple Combination Responsive (TCR) CFTR Mutation and No F508del Mutation

 Cystic Fibrosis / Posted 2 months ago

Brief Summary
The purpose of this study is to evaluate the efficacy and safety of VX-121/tezacaftor/deutivacaftor (VX-121/TEZ/D-IVA) in CF participants who are homozygous for F508del, heterozygous for F508del and a gating (F/G) or residual function (F/RF) mutation, or have at least 1 other TCR CF transmembrane conductance regulator (CFTR) gene mutation and no F508del mutation.
  • Inclusion Criteria :
    • Ages Eligible for Study: 12 Years and older
    • Sexes Eligible for Study: All
    • Accepts Healthy Volunteers: No
    • Participant has one of the following genotypes:
    • Homozygous for F508del;
    • Heterozygous for F508del and a gating (F/G) mutation;
    • Heterozygous for F508del and a residual function (F/RF) mutation;
    • At least 1 other TCR CFTR gene mutation identified as responsive to ELX/TEZ/IVA and no F508del mutation
    • Forced expiratory volume in 1 second (FEV1) value between 40% and 90% of predicted mean for age, sex, and height for participants currently receiving CFTR protein modulator therapy; FEV1 between 40% and 80% for participants not currently receiving CFTR protein modulator therapy
  • Exclusion Criteria :
    • History of solid organ or hematological transplantation
    • Hepatic cirrhosis with portal hypertension, moderate hepatic impairment (Child Pugh Score 7 to 9), or severe hepatic impairment (Child Pugh Score 10 to 15)
    • Lung infection with organisms associated with a more rapid decline in pulmonary status
    • Pregnant or breast-feeding females
    • Other protocol defined Inclusion/Exclusion criteria may apply
  • Study end date : February 2024
Contact details

All Wales Adult Cystic Fibrosis CentreUniversity Hospital Llandough,Cardiff,CF64 2XX medicalinfo@vrtx.com

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