This study is in progress, not accepting new patients

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)

a study on Cystic Fibrosis Fibrosis

Summary

Eligibility
for people ages 12 years and up (full criteria)
Location
at San Francisco, 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 heterozygous for F508del and a minimal function mutation (F/MF participants).

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 (CF) Who Are Heterozygous for F508del and a Minimal Function Mutation (F/MF)

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

  • Heterozygous for F508del and a minimal function mutation (F/MF genotype)
  • Forced expiratory volume in 1 second (FEV1) value >=40% and <=90% of predicted mean for age, sex, and height for participants currently receiving ELX/TEZ/IVA therapy; FEV1 >=40% and <=80% for participants not currently receiving ELX/TEZ/IVA

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

  • UCSF, Lung Transplant Program
    San Francisco California 94143 United States
  • Kaiser Permanente
    Oakland California 94611 United States

Details

Status
in progress, not accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Vertex Pharmaceuticals Incorporated
ID
NCT05033080
Phase
Phase 3 research study
Study Type
Interventional
Participants
About 436 people participating
Last Updated