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New triple therapy aims to control cystic fibrosis Long-Term

NCT ID NCT05844449

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 This study
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
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 phase 3 study tests the long-term safety and effectiveness of a triple combination drug (vanzacaftor/tezacaftor/deutivacaftor) in 174 people with cystic fibrosis aged 1 and older. Participants who completed a prior study will receive the drug to see if it helps control symptoms and improve lung function over time.

What this could mean

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

Active substance
Vanzacaftor/tezacaftor/deutivacaftor (triple combination therapy)
What this could lead to
If successful, this could provide a long-term treatment option to help control cystic fibrosis symptoms and improve lung function.
What could go wrong
This is an open-label extension study, so results may be less reliable than blinded trials. Side effects or lack of sustained benefit are possible.

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 174 people

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

Started

Aug 2023

Expected to finish

Jul 2029

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 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: Parts A and B: • Participants who have completed study drug treatment in the parent study (VX21-121-105; NCT Number: NCT05422222) Part B: -Meets at least 1 of the following criteria: * Completed study drug treatment in Part A * Had study drug interruption(s) in Part A, but did not permanently discontinue study drug and completed study visits up to the last scheduled visit of the treatment period of Part A Key Exclusion Criteria: * Hepatic cirrhosis with portal hypertension, moderate hepatic impairment, or severe hepatic impairment that might pose an additional risk in administering study drug * History of solid organ, hematological transplantation, or cancer * History of drug intolerance in 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

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The places running it

    37 sites in 10 countries. The list below names each one and where it is.

  2. The official record

    The full official record for this study. This one lists no contact details, but it is the first place any would appear.

    Open the record ↗

  3. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Locations

  • American Family Childrens Hospital

    Madison, Wisconsin, 53792, United States

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

    Chicago, Illinois, 60611, United States

  • Boston Children's Hospital

    Boston, Massachusetts, 02115, United States

  • British Columbia Children's Hospital

    Vancouver, Canada

  • Charite Paediatric Pulmonology Department

    Berlin, Germany

  • Children's Health Care d/b/a Children's Hospitals and Clinics of Minnesota

    Minneapolis, Minnesota, 55404, United States

  • Children's Health Queensland Hospital and Health Service

    South Brisbane, Australia

  • Children's Hospital of Colorado - Pulmonology

    Aurora, Colorado, 80045, United States

  • Children's Hospital of Orange County

    Orange, California, 92868, United States

  • Children's Hospital of Pittsburgh - Pulmonology

    Pittsburgh, Pennsylvania, 15224, United States

  • Cincinnati Children's Hospital Medical Center - Pulmonology

    Cincinnati, Ohio, 45229, United States

  • Cohen Children's Medical Center - Lakeville Road

    Lake Success, New York, 11042, United States

  • Great Ormond Street Hospital for Children

    London, United Kingdom

  • Hopital Femme Mere-Enfant

    Bron, France

  • Hopital Necker Enfants Malades - Pulmonology

    Paris, France

  • Hospital for Sick Children - Pulmonology

    Toronto, Canada

  • Inselspital Bern

    Bern, Switzerland

  • JW Riley Hospital for Children - Pulmonology

    Indianapolis, Indiana, 46202, United States

  • Karolinska University Hospital - Pulmonology

    Stockholm, Sweden

  • Kinderspital Zurich - Abteilung Pneumologie

    Zurich, Switzerland

  • Medizinische Hochschule Hannover - Clinic for Pediatric Pneumology, Allergology and Neonatology, CF-Centre

    Hanover, Germany

  • Nationwide Children's Hospital

    Columbus, Ohio, 43205, United States

  • Noah's Ark Children's Hospital for Wales

    Cardiff, United Kingdom

  • Sahlgrenska Universitetssjukhuset - Göteborg CF-center

    Gothenburg, Sweden

  • Sophia Children's Hospital

    Rotterdam, Netherlands

  • St. Louis Children's Hospital - Pulmonology

    St Louis, Missouri, 63110, United States

  • Stanford University - Palo Alto - Pulmonology

    Palo Alto, California, 94304, United States

  • Starship Child Health

    Grafton, New Zealand

  • Texas Children's Hospital - Wallace Tower - Pulmonology

    Houston, Texas, 77030, United States

  • The Children's Mercy Hospital

    Kansas City, Missouri, 64108, United States

  • The Emory Clinic - Clifton Road - Pulmonology

    Atlanta, Georgia, 30322, United States

  • The Kids Research Institute Australia

    Nedlands, Australia

  • The Royal Children's Hospital Melbourne

    Parkville, Australia

  • UH Cleveland Medical Center - Pulmonology

    Cleveland, Ohio, 44106, United States

  • Universitatsklinikum Essen

    Essen, Germany

  • Vermont Lung Center

    Colchester, Vermont, 05446, United States

  • Women's & Children's Hospital

    North Adelaide, Australia

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