VX-121 Combination Therapy in Subjects With Cystic Fibrosis VX20-121-103
- Study HIC#:2000030926
- Last Updated:02/02/2024
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.
- Age12 years and older
- GenderBoth
Contact Us
For more information about this study, including how to volunteer, contact:
Marie Egan
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Trial Purpose and Description
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.
Eligibility Criteria
Key Inclusion Criteria:
- 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 >=40% and <=90% of predicted mean for age, sex, and height for participants currently receiving CFTR protein modulator therapy; FEV1 >=40% and <=80% for participants not currently receiving CFTR protein modulator therapy
Key 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