Elexacaftor/Tezacaftor/Ivacaftor for Cystic Fibrosis

Recruiting in Palo Alto (17 mi)

+16 other locations

Age: Any Age

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 3

Waitlist Available

Sponsor: Vertex Pharmaceuticals Incorporated

No Placebo Group

Pivotal Trial (Near Approval)

Prior Safety Data

Approved in 4 Jurisdictions

Trial Summary

What is the purpose of this trial?The purpose of the study is to evaluate the long-term safety, tolerability, efficacy, and pharmacodynamics (PD) of elexacaftor (ELX)/tezacaftor (TEZ)/ivacaftor (IVA).

Eligibility Criteria

This trial is for individuals aged 12 or older with cystic fibrosis who completed treatment in a prior study (VX22-445-122 Part B) or had interruptions but didn't stop the drug and finished all visits up to the last scheduled one.

Inclusion Criteria

I finished or partially completed the VX22-445-122 Part B study without quitting the study drug.

Key

Participant Groups

The study aims to assess long-term safety, tolerability, effectiveness, and pharmacodynamics of a combination medication (ELX/TEZ/IVA) compared to ivacaftor alone in treating cystic fibrosis.

1Treatment groups

Experimental Treatment

Group I: ELX/TEZ/IVAExperimental Treatment2 Interventions

Participants will receive ELX/TEZ/IVA in the morning and IVA in the evening.

ELX/TEZ/IVA is already approved in United States, European Union, Canada, Australia for the following indications:

🇺🇸 Approved in United States as Trikafta for:

Cystic fibrosis in patients aged 2 years and older with at least one copy of the F508del mutation in the CFTR gene or another mutation responsive to treatment

🇪🇺 Approved in European Union as Kaftrio for:

Cystic fibrosis in patients aged 2 years and older with at least one copy of the F508del mutation in the CFTR gene or another mutation responsive to treatment

🇨🇦 Approved in Canada as Trikafta for:

Cystic fibrosis in patients aged 2 years and older with at least one copy of the F508del mutation in the CFTR gene or another mutation responsive to treatment

🇦🇺 Approved in Australia as Kaftrio for:

Cystic fibrosis in patients aged 2 years and older with at least one copy of the F508del mutation in the CFTR gene or another mutation responsive to treatment