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Trikafta's long-term safety tested in toddlers with cystic fibrosis

NCT ID NCT06460506

Disease control Sponsor: Vertex Pharmaceuticals Incorporated Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing This study
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This phase 3 study is testing the long-term safety and effectiveness of the drug combination elexacaftor/tezacaftor/ivacaftor (known as Trikafta) in 50 people with cystic fibrosis who are at least 12 months old. Participants previously completed a related study and will continue taking the drug to see how well it works over time. The main goal is to track side effects and changes in sweat chloride levels, a key measure of cystic fibrosis severity.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
elexacaftor/tezacaftor/ivacaftor (Trikafta)
What this could lead to
If successful, this study could confirm that Trikafta is safe and effective for long-term use in young children with cystic fibrosis, helping them breathe easier and have fewer lung infections.
What could go wrong
This is an open-label extension study with no placebo group, so results may be less reliable. Side effects like liver problems or drug interactions are possible, and the small size (50 participants) limits how broadly the findings can be applied.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

50 people

The number who actually took part.

Started

Nov 2024

Expected to finish

Sep 2027

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

12 months and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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.

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Conditions

The condition(s) this trial relates to.

cystic fibrosis

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Cystic fibrosis

Contacts and locations

Locations

  • Alder Hey Children's NHS Foundation Trust

    Liverpool, United Kingdom

  • British Columbia Children's Hospital

    Vancouver, Canada

  • Charité Universitätsmedizin BerlinX

    Berlin, Germany

  • Children and Young Adults Research Unit

    Cardiff, United Kingdom

  • Erasmus Medical Center

    Rotterdam, Netherlands

  • Great Ormond Street Hospital for Children

    London, United Kingdom

  • Inselspital - Universitaetsspital Bern

    Bern, Switzerland

  • Juliane Marie Center, Rigshospitalet

    Copenhagen, Denmark

  • Kinderklinik III, Abt. fur Pneumologie

    Essen, Germany

  • Kinderspital Zurich

    Zurich, Switzerland

  • Leeds General Infirmary

    Leeds, United Kingdom

  • Medizinische Hochschule Hannover

    Hanover, Germany

  • Queensland Children's Hospital

    South Brisbane, Australia

  • Royal Brompton Hospital

    London, United Kingdom

  • Southampton General Hospital

    Southampton, United Kingdom

  • The Hospital for Sick Children

    Toronto, Canada

  • The Kids Research Institute Australia

    Nedlands, Australia

  • The Royal Children's Hospital

    Parkville, Australia

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