Evaluation of Long-term Safety and Efficacy of ELX/TEZ/IVA in Cystic Fibrosis Participants 12 Months of Age and Older

June 3, 2026 updated by: Vertex Pharmaceuticals Incorporated

A Phase 3, Open-label Study Evaluating the Long-term Safety and Efficacy of Elexacaftor/Tezacaftor/Ivacaftor in Cystic Fibrosis Subjects 12 Months of Age and Older

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

Study Overview

Status

Active, not recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

50

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Australia
      • Nedlands, Australia
        • The Kids Research Institute Australia
      • Parkville, Australia
        • The Royal Children's Hospital
      • South Brisbane, Australia
        • Queensland Children's Hospital
  • Canada
      • Toronto, Canada
        • The Hospital for Sick Children
      • Vancouver, Canada
        • British Columbia Children's Hospital
  • Denmark
      • Copenhagen, Denmark
        • Juliane Marie Center, Rigshospitalet
  • Germany
      • Berlin, Germany
        • Charité Universitätsmedizin BerlinX
      • Essen, Germany
        • Kinderklinik III, Abt. fur Pneumologie
      • Hanover, Germany
        • Medizinische Hochschule Hannover
  • Netherlands
      • Rotterdam, Netherlands
        • Erasmus Medical Center
  • Switzerland
      • Bern, Switzerland
        • Inselspital - Universitaetsspital Bern
      • Zurich, Switzerland
        • Kinderspital Zürich
  • United Kingdom
      • Cardiff, United Kingdom
        • Children and Young Adults Research Unit
      • Leeds, United Kingdom
        • Leeds General Infirmary
      • Liverpool, United Kingdom
        • Alder Hey Children's NHS Foundation Trust
      • London, United Kingdom
        • Royal Brompton Hospital
      • London, United Kingdom
        • Great Ormond Street Hospital for Children
      • Southampton, United Kingdom
        • Southampton General Hospital

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

Key Inclusion Criteria:

  • Completed study drug treatment in the parent study VX22-445-122 Part B (NCT05882357) 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 drug intolerance in the parent study that would pose an additional risk to the participant
  • Current participation in an investigational drug trial other than the parent study

Other protocol defined Inclusion/Exclusion criteria may apply.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: ELX/TEZ/IVA
Participants will receive ELX/TEZ/IVA in the morning and IVA in the evening.
Drug: ELX/TEZ/IVA
Fixed-dose combination granules for oral administration.
Other Names:
  • VX-445/VX-661/VX-770
  • elexacaftor/tezacaftor/ivacaftor
Drug: IVA
Granules for oral administration
Other Names:
  • VX-770
  • ivacaftor

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Safety and Tolerability as Assessed by Number of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs)
Time Frame: Day 1 up to Week 100
Day 1 up to Week 100

Secondary Outcome Measures

Outcome Measure
Time Frame
Absolute Change in Sweat Chloride (SwCl)
Time Frame: From Baseline through Week 96
From Baseline through Week 96

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Vertex Pharmaceuticals Incorporated

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

November 21, 2024

Primary Completion (Estimated)

September 30, 2027

Study Completion (Estimated)

September 30, 2027

Study Registration Dates

First Submitted

June 10, 2024

First Submitted That Met QC Criteria

June 10, 2024

First Posted (Actual)

June 14, 2024

Study Record Updates

Last Update Posted (Actual)

June 5, 2026

Last Update Submitted That Met QC Criteria

June 3, 2026

Last Verified

June 1, 2026

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • VX22-445-123
  • 2023-509563-24-00 (Other Identifier: EU CT Number)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

IPD Plan Description

Details on Vertex data sharing criteria and process for requesting access can be found at: https://www.vrtx.com/our-science/clinical-trials-data-sharing/

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

Clinical Trials on Cystic Fibrosis

Clinical Trials on ELX/TEZ/IVA

Search Similar Trials

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Cambodia

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations

Subscribe