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New triple therapy for kids with cystic fibrosis shows promise in phase 3 trial

NCT ID NCT05422222

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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 27, 2026 · Last updated Jun 27, 2026

Summary

This study tests a triple-combination drug (VX-121/tezacaftor/deutivacaftor) in children aged 1 to 11 with cystic fibrosis who have a specific genetic mutation. The goal is to check how the medicine works in the body, its safety, and how well children tolerate it. Around 210 children will take part, and the study also looks at changes in sweat chloride levels, a key marker of CF.

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

About 210 people

The number the study aims to enrol. It can still change while the study runs.

Started

Jun 2022

Expected to finish

Jun 2030

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

1 year to 11 years

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 with stable CF and at least 1 TCR mutation (including F508del) in the CFTR gene Key Exclusion Criteria: * History of solid organ, hematological transplantation, or cancer * Hepatic cirrhosis with portal hypertension, moderate hepatic impairment (Child Pugh Score 7 to 9), or severe hepatic impairment (Child Pugh Score 10 to 15) * Lung infection with organisms associated with a more rapid decline in pulmonary status 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

  • American Family Childrens Hospital

    Madison, Wisconsin, 53792, United States

  • Ann & Robert H. Lurie Children's Hospital of Chicago

    Chicago, Illinois, 60611, United States

  • Boston Children's Hospital

    Boston, Massachusetts, 02115, United States

  • British Columbia Children's Hospital

    Vancouver, Canada

  • CHU Lyon - Hopital Femme Mere-Enfant

    Bron, France

  • Charité - Paediatric Pulmonology Department

    Berlin, Germany

  • Children and Young Adults Research Unit

    Cardiff, United Kingdom

  • Children's Hospital of Colorado

    Aurora, Colorado, 80045, United States

  • Children's Hospital of Orange County

    Orange, California, 92868, United States

  • Children's Respiratory and Critical Care Specialists, P.A., Children's Hospitals and Clinics of Minnesota

    Minneapolis, Minnesota, 55404, United States

  • Cincinnati Children's Hospital Medical Center

    Cincinnati, Ohio, 45229, United States

  • Cohen Children's Medical Center

    Lake Success, New York, 11042, United States

  • Erasmus Medical Center / Sophia Children's Hospital

    Rotterdam, Netherlands

  • Great Ormond Street Hospital for Children

    London, United Kingdom

  • Hopital Necker, Enfants Malades

    Paris, France

  • Inselspital - Universitaetsspital Bern

    Bern, Switzerland

  • Karolinska University Hospital - Pulmonology

    Stockholm, Sweden

  • Kinderspital Zurich

    Zurich, Switzerland

  • Medizinische Hochschule Hannover

    Hanover, Germany

  • Nationwide Children's Hospital

    Columbus, Ohio, 43205, United States

  • Oregon Health & Science University

    Portland, Oregon, 97239, United States

  • Queensland Children's Hospital

    South Brisbane, Australia

  • Rainbow Babies and Children's Hospital/University Hospitals Cleveland Medical Center

    Cleveland, Ohio, 44106, United States

  • Riley Hospital for Children at Indiana University Health

    Indianapolis, Indiana, 46202, United States

  • Sahlgrenska Universitetssjukhuset

    Gothenburg, Sweden

  • Stanford University Clinical and Translational Research Unit

    Palo Alto, California, 94304, United States

  • Starship Children's Hospital

    Grafton, New Zealand

  • Texas Children's Hospital - Wallace Tower

    Houston, Texas, 77030, United States

  • The Children's Mercy Hospital

    Kansas City, Missouri, 64108, United States

  • The Emory Clinic / Children's Healthcare of Atlanta at Egleston

    Atlanta, Georgia, 30322, United States

  • The Hospital for Sick Children

    Toronto, Canada

  • The Kids Research Institute Australia

    Nedlands, Australia

  • The Royal Children's Hospital

    Parkville, Australia

  • UPMC Children's Hospital of Pittsburgh

    Pittsburgh, Pennsylvania, 15224, United States

  • Universitatsklinikum Essen

    Essen, Germany

  • Vermont Lung Center

    Colchester, Vermont, 05446, United States

  • Washington University School of Medicine / St. Louis Children's Hospital

    St Louis, Missouri, 63110, United States

  • Women's and Children's Hospital

    North Adelaide, Australia

More trials for these conditions

Other studies related to the condition(s) this trial covers.