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  • A Study of 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

A Study of 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

Principal Investigator

Maria Tupayachi Ortiz

Enrollment Status

Closed

Clinical Trial ID

Institutional Protocol # 20210495
National Clinical Trials Identifier NCT05076149

Clinical Trial 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.


Phase

Phase 3


Funding Agency/Sponsor

Industry


Disease

Cystic Fibrosis (CF)


Enrollment Eligibility

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


Contact Information

Study Contact Patricia Graham
Phone Number +1 (305) 2435119
Email pgraham1@miami.edu
Get detailed information on ClinicalTrials.Gov

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