A Phase 3 Study of VX-121 Combination Therapy in Participants With Cystic Fibrosis (CF) Heterozygous for F508del and a Minimal Function Mutation (F/MF)
Cystic Fibrosis / Posted 1 year 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 heterozygous for F508del and a minimal function mutation (F/MF participants).
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Inclusion Criteria :
- Ages Eligible for Study: 12 Years and older (Child, Adult, Older Adult)
- Sexes Eligible for Study: All
- Accepts Healthy Volunteers: No
- Heterozygous for F508del and a minimal function mutation (F/MF genotype)
- Forced expiratory volume in 1 second (FEV1) value between 40% and 90% of predicted mean for age, sex, and height for participants currently receiving ELX/TEZ/IVA therapy; FEV1 between 40% and 80% for participants not currently receiving ELX/TEZ/IVA
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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 : January 2024
- Wales-Based Study Contact : Please speak to your clinician
- Principal Investigator : Vertex Pharmaceuticals Incorporated