Evaluation of Long-term Safety and Efficacy of ELX/TEZ/IVA in Cystic Fibrosis (CF) Participants 2 Years and Older

A Phase 3 Open-label Study Evaluating the Long-term Safety and Efficacy of Elexacaftor/Tezacaftor/Ivacaftor Triple Combination Therapy in Cystic Fibrosis Subjects 2 Years and Older

Status

Completed

Phases

Phase 3

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT05153317

Enrollment

Registered

2021-12-10

Start date

2022-01-17

Completion date

2026-01-29

Last updated

2026-03-03

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Cystic Fibrosis

Brief summary

The purpose of this study is to evaluate the long term safety, tolerability, efficacy and pharmacodynamics of elexacaftor (ELX)/tezacaftor (TEZ)/ivacaftor (IVA) in CF participants 2 years of age and older.

Interventions

DRUGELX/TEZ/IVA

Fixed-dose combination granules or tablets for oral administration.

DRUGIVA

Granules or tablets for oral administration.

Study design

Allocation

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Masking

NONE

Eligibility

Sex/Gender

ALL

Age

2 Years to No maximum

Healthy volunteers

Inclusion criteria

key Inclusion Criteria: * Completed study drug treatment in the parent study (VX20-445-111 Part B, NCT04537793), or had study drug interruption(s) in the parent study, but did not permanently discontinue study drug, and completed study visits up to the last scheduled visit of the Treatment Period of the parent study Key

Exclusion criteria

* History of study drug intolerance in the parent study * History of poor compliance with ELX/TEZ/IVA and/or procedures in the parent study Other protocol defined Inclusion/

Design outcomes

Primary

Measure	Time frame
Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)	From Day 1 up to Week 196

Secondary

Measure	Time frame
Absolute Change in Sweat Chloride (SwCl)	From Baseline up to Week 192
Absolute Change in Lung Clearance Index (LCI) 2.5	From Baseline up to Week 192

Countries

Australia, Canada, Germany, United Kingdom, United States

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Mar 4, 2026