Summary

Eligibility
for people ages 12 years and up (full criteria)
Location
at Sacramento, California and other locations
Dates
study started
estimated completion

Description

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.

Official Title

A Phase 3, Randomized, Double-blind, Controlled Study Evaluating the Efficacy and Safety of VX-121 Combination Therapy in Subjects With Cystic Fibrosis 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 CFTR Mutation and No F508del Mutation

Keywords

Cystic Fibrosis Fibrosis Ivacaftor Elexacaftor VX-121/TEZ/D-IVA ELX/TEZ/IVA IVA

Eligibility

You can join if…

Open to people ages 12 years and up

  • 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

You CAN'T join if...

  • 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

Locations

  • University of California Davis Medical Center accepting new patients
    Sacramento California 95817 United States
  • Children's Hospital Los Angeles accepting new patients
    Los Angeles California 90027 United States

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Vertex Pharmaceuticals Incorporated
Links
Sign up for this study
ID
NCT05076149
Phase
Phase 3 research study
Study Type
Interventional
Participants
Expecting 550 study participants
Last Updated