Den här översättningen är inte klar ännu. Den här sidan är just nu på engelska.

Gå till den engelska sidan

Cure My Disease

Don't miss your cure!

 Log in / Sign up

Trikafta's long-term safety tested in cystic fibrosis patients with rare mutations

NCT ID NCT05331183

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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 Sep 11, 2026 · Updated 8 times

Summary

This study looks at the long-term safety and effectiveness of the drug combination Trikafta (elexacaftor/tezacaftor/ivacaftor) in people with cystic fibrosis who have certain non-F508del gene mutations. About 297 participants who completed a prior study will continue taking the medication and be monitored for side effects, lung function, sweat chloride levels, and quality of life. The goal is to confirm that the drug remains safe and helpful over an extended period.

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

297 people

The number who actually took part.

Started

Nov 2022

Expected to finish

Apr 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

6 years 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: Part A: Completed study drug treatment in parent study or had study drug interruption(s) in parent study but completed study visits up to the last scheduled visit of the treatment period in the parent study Part B: Completed study drug treatment in Part A or had study drug interruption(s) in Part A but completed study visits up to the last scheduled visit of the treatment period of Part A Key Exclusion Criteria: History of study drug intolerance in the parent study Other protocol defined Inclusion/Exclusion criteria may apply.

Get updates

Get notified about this study

Sign up to get updates when this study changes or when new studies for Cystic fibrosis are added.

Vår säkerhetsrekommendation!

Genom att skicka in godkänner du våra Användarvillkor

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

  • Amsterdam UMC - locatie AMC - Afdeling Longgeneeskunde

    Amsterdam, Netherlands

  • Azienda Ospedaliera Regionale 'San Carlo', Potenza

    Potenza, Italy

  • Azienda Ospedaliera di Verona - Ospedale Civile Maggiore

    Verona, Italy

  • Azienda Ospedaliero Universitaria (AOU) San Luigi Gonzaga

    Orbassano, Italy

  • Azienda Ospedaliero Universitaria Federico II Napoli

    Naples, Italy

  • Azienda Ospedaliero Universitaria Ospedale Meyer

    Florence, Italy

  • Azienda Ospedaliero Universitaria delle Marche

    Ancona, Italy

  • British Columbia Children's Hospital

    Vancouver, Canada

  • CHU de Nice - Hôpital Pasteur 2

    Nice, France

  • CUB Hôpital Erasme

    Brussels, Belgium

  • Centre Hospitalier Intercommunal Creteil - Pulmonology

    Créteil, France

  • Centre de Perharidy, Roscoff

    Roscoff, France

  • Charité - Paediatric Pulmonology Department

    Berlin, Germany

  • Dr. von Haunersches Kinderspital - Pulmonology

    München, Germany

  • Erasmus Medisch Centrum - Pulmonology

    Rotterdam, Netherlands

  • Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico

    Milan, Italy

  • Fondazione IRCCS Ospedale Maggiore Policlinico, Mangiagalli e Regina Elena

    Milan, Italy

  • HagaZiekenhuis

    The Hague, Netherlands

  • Hopital Arnaud de Villeneuve - Service de Pneumologie et Maladies Respiratoires

    Montpellier, France

  • Hopital Bretonneau

    Tours, France

  • Hopital Cochin - Pulmonology

    Paris, France

  • Hopital Femme Mere-Enfant

    Bron, France

  • Hopital Larrey

    Toulouse, France

  • Hopital Necker Enfants Malades - Pulmonology

    Paris, France

  • Hopital Nord

    Marseille, France

  • Hopital Robert Debre - Pulmonology

    Paris, France

  • Hospital Infantil Universitario Nino Jesus

    Madrid, Spain

  • Hospital Saint Joan de Deu

    Barcelona, Spain

  • Hospital Universitari Vall d´Hebron Servicio de Broncoscopia

    Barcelona, Spain

  • Hospital Universitario 12 de Octubre - Pulmonology

    Madrid, Spain

  • Hospital Universitario Ramon y Cajal - Pulmonology

    Madrid, Spain

  • Hospital Universitario Virgen del Rocio

    Seville, Spain

  • Hospital Universitario de Jerez de la Frontera

    Jerez de la Frontera, Spain

  • Hospital Universitario y Politecnico La Fe - Pulmonology

    Valencia, Spain

  • Hospital Virgen de la Arrixaca

    Murcia, Spain

  • Hospital de Cruces

    Barakaldo, Spain

  • Hospital for Sick Children - Pulmonology

    Toronto, Canada

  • Hôpital Lyon Sud - Pulmonology

    Pierre-Bénite, France

  • Hôpital Nord Laennec

    Nantes, France

  • Hôpital Sud - Rennes

    Rennes, France

  • Hôpital américain de Reims - Département de pédiatrie A

    Reims, France

  • IRCCS Istituto Giannina Gaslini-Ospedale Pediatrico

    Genova, Italy

  • IRCSS Ospedale Pediatrico Bambino Gesu - Pulmonology

    Rome, Italy

  • Institut Cœur Poumon, CHU de Lille

    Lille, France

  • Institut d´Investigacio i Innovacio Parc Tauli

    Sabadell, Spain

  • Instytut Matki i Dziecka, Klinika Mukowiscydozy IMiD, Oddział Chorób Płuc SZPZOZ im.Dzieci Warszawy w Dziekanowie Leśnym

    Łomianki, Poland

  • Johannes Gutenberg-Universitaet

    Mainz, Germany

  • Justus-Liebig-Universität Gießen

    Giessen, Germany

  • Karolinska University Hospital - Pulmonology

    Stockholm, Sweden

  • Kinderspital Zuerich

    Zurich, Switzerland

  • Klinik für Kinder- und Jugendmedizin

    Münster, Germany

  • Klinika Detskych Infekcnich Nemoci

    Brno, Czechia

  • Klinikum Westbrandenburg (CF)

    Potsdam, Germany

  • Klinikum der Johann-Wolfgang-Goethe Universitaet - Pulmonology

    Frankfurt, Germany

  • La Paz Infantil

    Madrid, Spain

  • Le Centre de recherche du CHUM (CRCHUM)

    Montreal, Canada

  • Lungenheilkunde Munchen - Pasing

    München, Germany

  • McGill University Health Centre, Glen Site, Montreal Children's Hospital

    Montreal, Canada

  • Medizinische Hochschule Hannover

    Hanover, Germany

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

    Hanover, Germany

  • Medizinische Universität Innsbruck - Heilstättenschule Universitätsklinik

    Innsbruck, Austria

  • Motol University Hospital

    Prague, Czechia

  • Mukoviszidose-Zentrum am Universitätsklinikum Jena

    Jena, Germany

  • National Koranyi Institute for TBC and Pulmonology

    Budapest, Hungary

  • Oslo University Hospital, Department of Paediatric Medicine

    Oslo, Norway

  • Pediatric Hospital Polanki named of Maciej Płażyński

    Gdansk, Poland

  • Policlinico Universitario G. Martino

    Messina, Italy

  • Radboud University Medical Center - Pulmonology

    Nijmegen, Netherlands

  • Ruhrlandklinik

    Essen, Germany

  • Sahlgrenska Universitetssjukhuset - Göteborg CF-center

    Gothenburg, Sweden

  • St. Paul's Hospital - Pulmonology

    Vancouver, Canada

  • Stollery Children's Hospital

    Edmonton, Canada

  • UMCU, Locatie Wilhelmina Kinderziekenhuis

    Utrecht, Netherlands

  • UZ Brussel - Campus Jette

    Brussels, Belgium

  • Unidade Local de Saúde de Santa Maria, E.P.E. - Pulmonology

    Lisbon, Portugal

  • Universitair Ziekenhuis Antwerpen (UZA) - Antwerp University Hospital

    Edegem, Belgium

  • Universitair Ziekenhuis Gent

    Ghent, Belgium

  • Universitaire Ziekenhuizen Leuven - Campus Gasthuisberg

    Leuven, Belgium

  • Universite Catholique de Louvain - Pulmonology

    Woluwe-Saint-Lambert, Belgium

  • University Hospital Cologne (Koeln) - CF-Studienzentrum

    Cologne, Germany

  • Universitätsklinikum Halle (Saale) / Universitätsklinik und Poliklinik für Innere Medizin, Schwerpunkt Pneumologie

    Halle, Germany

More trials for these conditions

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