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CF drug shows promise for toddlers in Final-Stage trial

NCT ID NCT05882357

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
Running, but no longer taking on new participants.
Completed This study
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 26, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This study tested a triple-combination drug (elexacaftor/tezacaftor/ivacaftor) in 70 toddlers aged 12 to 24 months with cystic fibrosis. The goal was to check how the drug moves through the body, its safety, and any side effects. The trial has finished, and results will help determine if this treatment is suitable for very young children.

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 (a triple-combination drug taken by mouth)
What this could lead to
If successful, this could provide a safe and effective treatment option for very young children with cystic fibrosis, potentially improving lung function and quality of life from an early age.
What could go wrong
This is a completed Phase 3 trial, but results are not yet published. The study is small (70 participants) and focused on safety and drug levels, so long-term benefits are still uncertain. Side effects are possible, as with any medication.

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

70 people

The number who actually took part.

Started

Jun 2023

Finished

Sep 2025

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 to 24 months

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: Participants who have at least 1 F508del mutation in the CF transmembrane conductance regulator (CFTR) gene or another ELX/TEZ/IVA-responsive CFTR mutation Key Exclusion Criteria: History of any illness or any clinical condition that, in the opinion of the investigator, might either confound the results of the study or pose an additional risk in administering study drug(s) to the participant 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

  • Charite Paediatric Pulmonology Department

    Berlin, Germany

  • Children and Young Adults Research Unit

    Cardiff, United Kingdom

  • Erasmus Medical Center / Sophia Children's Hospital

    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 Zuerich

    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

  • Telethon Kids Institute

    Nedlands, Australia

  • The Children's Hospital at Westmead

    Westmead, Australia

  • The Hospital for Sick Children

    Toronto, Canada

  • The Royal Children's Hospital

    Parkville, Australia

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