Clinical trial • Phase III • Respiratory | Rare Disease

tezacaftor, deutivacaftor, vanzacaftor for Cystic fibrosis

Phase III trial of tezacaftor, deutivacaftor, vanzacaftor for Cystic fibrosis. open-label, none/not specified-controlled. 513 participants.

Overview

Trial Therapeutic Area
Respiratory | Rare Disease
Trial Disease
Cystic fibrosis
Trial Stage
Phase III
Drug Modality
Small molecule
Paediatric Trial
Yes
Orphan Drug
Yes

Key dates

Initial CTIS Submission Date
16-05-2024
First CTIS Authorization Date
25-06-2024

Trial design

open-label, none/not specified-controlled Phase III trial in Czechia, Portugal, Italy and others.

Open Label
Yes
Comparator
None/Not specified
Target Sample Size
513

Eligibility

Recruits 513 paediatric patients.

Pregnancy Exclusion
Pregnant or breast-feeding at the time of enrollment in Part A.
Vulnerable Population
Vulnerable populations are included: consent must be signed by the subject or his/her legally appointed and authorized representative, and an assent form is required 'when appropriate'. Age-specific informed consent/assent materials are provided (adolescent assent forms and parent/guardian consent forms are present, including documents titled for adolescents 12-14 yrs and 15-17 yrs and parent/guardian ICFs). The trial documentation includes separate ICFs for adults, parents/guardians, adolescents (multiple age bands), and pregnancy information, indicating tailored consent/assent handling for minors and other vulnerable groups.

Inclusion criteria

  • {"criterion_text":"- Subject (or his or her legally appointed and authorized representative) will sign and date an informed consent form, and, when appropriate, an assent form.\n- Willing and able to comply with scheduled visits, treatment plan, study restrictions, laboratory tests, contraceptive guidelines, and other study procedures.\n- Part A: Did not withdraw consent from a parent study. Part B: Did not withdraw consent from Part A. Part C: Did not withdraw consent from Part B.\n- Part A: Meets at least 1 of the following criteria: •\tCompleted study drug treatment in a parent study. •\tHad study drug interruption(s) in a parent study, but did not permanently discontinue study drug, and completed study visits up to the last scheduled visit of the Treatment Period of a parent study. Part B: Meets at least 1 of the following criteria: •\tCompleted study drug treatment in Part A. •\tHad 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. Part C: Meets at least 1 of the following criteria: • Completed study drug treatment in Part B. • Had study drug interruption(s) in Part B, but did not permanently discontinue study drug, and completed study visits up to the last scheduled visit of the Treatment Period of Part B.\n- Willing to remain on a stable Cystic Fibrosis treatment regimen through completion of study participation."}

Exclusion criteria

  • {"criterion_text":"- New development of a comorbidity during the parent study, Part A, or Part B that might pose an additional risk in administering study drug to the subject. This includes, but is not limited to, the following: - Hepatic cirrhosis with portal hypertension, moderate hepatic impairment, or severe hepatic impairment, that might pose an additional risk in administering study drug to the subject. - Solid organ or hematological transplantation. - Any other comorbidity that, in the opinion of the investigator, might post an additional risk in administering study drug to the subject.\n- Pregnant or breast-feeding at the time of enrollment in Part A.\n- History of drug intolerance in a parent study that would pose an additional risk to the subject in the opinion of the investigator. (e.g., subjects with a history of allergy or hypersensitivity to the study drug.)\n- Current participation in an investigational drug trial (other than a parent study). Participation in a non-interventional study (including observational studies, registry studies, and studies requiring blood collections without administration of study drug) and screening for another Vertex study is permitted.\n- The subject or a close relative of the subject is the investigator or a sub-investigator, research assistant, pharmacist, study coordinator, or other staff directly involved with the conduct of the study at that site. However, an adult (aged 18 years or older) who is a relative of a study staff member may be enrolled in the study provided that: - the adult lives independently of and does not reside with the study staff member, and - the adult participates in the study at a site other than the site at which the family member is employed."}

Endpoints

Primary endpoints

  • {"endpoint_text":"- Safety and tolerability of long-term treatment with VX-121/TEZ/D-IVA based on adverse events, clinical laboratory values, ECGs, vital signs, and pulse oximetry","definition_or_measurement_approach":"Measured by adverse events, clinical laboratory values, ECGs, vital signs, and pulse oximetry as listed in the endpoint wording."}

Secondary endpoints

  • {"endpoint_text":"- Absolute change from baseline in percent predicted forced expiratory volume in 1 second (ppFEV1)","definition_or_measurement_approach":"Absolute change from baseline in ppFEV1 (percent predicted FEV1) as measured by spirometry."}
  • {"endpoint_text":"- Absolute change from baseline in sweat chloride (SwCl)","definition_or_measurement_approach":"Absolute change from baseline in sweat chloride concentration (SwCl) as measured by sweat chloride testing."}
  • {"endpoint_text":"- Number of pulmonary exacerbations (PEx)","definition_or_measurement_approach":"Count of pulmonary exacerbations (PEx) experienced by subjects during the study (event count)."}

Recruitment

Registry Or Advocacy Recruitment
True, Fondation Ildys
Planned Sample Size
513
Recruitment Window Months
61
Consent Approach
Informed consent is required from the subject or the subject's legally appointed and authorized representative; assent is required when appropriate. Age-specific informed consent and assent documents are provided (examples in the file list: adolescent assent forms for 12-14 yrs and 15-17 yrs, parent/guardian ICFs, adult ICFs, pregnancy information). Consent/assent materials are available in multiple languages as evidenced by protocol/ICF document titles (examples: English, Czech, Portuguese, Spanish, French, Hungarian, Italian, Dutch, Norwegian, Polish, Swedish, German).

Geography

Total Number Of Sites
81
Total Number Of Participants
331

Czechia

Earliest CTIS Part Ii Submission Date
31-05-2024
Latest Decision Or Authorization Date
04-02-2026
Processing Time Days
614
Number Of Sites
2
Number Of Participants
6

Sites

Site Name
Fakultni Nemocnice V Motole
Department Name
Ústav lékařské mikrobiologie 2. lékařské fakulty UK a centrum cystické fibrózy
Principal Investigator Name
Pavel Dřevínek
Principal Investigator Email
pavel.drevinek@lfmotol.cuni.cz
Contact Person Name
Pavel Dřevínek
Contact Person Email
pavel.drevinek@lfmotol.cuni.cz
Site Name
Fakultni Nemocnice Brno
Department Name
Klinika nemocí plicních a TBC
Principal Investigator Name
Lukáš Homola
Principal Investigator Email
homola.lukas@fnbrno.cz
Contact Person Name
Lukáš Homola
Contact Person Email
homola.lukas@fnbrno.cz

Portugal

Earliest CTIS Part Ii Submission Date
31-05-2024
Latest Decision Or Authorization Date
03-02-2026
Processing Time Days
613
Number Of Sites
3
Number Of Participants
8

Sites

Site Name
Unidade Local De Saude De Santo Antonio E.P.E.
Department Name
Pneumology
Principal Investigator Name
Telma Barbosa
Principal Investigator Email
telmab@gmail.com
Contact Person Name
Telma Barbosa
Contact Person Email
telmab@gmail.com
Site Name
Unidade Local de Saude de Sao Joao E.P.E.
Department Name
Pneumology
Principal Investigator Name
Adelina Amorim
Principal Investigator Email
adelinamorim@gmail.com
Contact Person Name
Adelina Amorim
Contact Person Email
adelinamorim@gmail.com
Site Name
Unidade Local De Saude De Santa Maria E.P.E.
Department Name
Pneumology
Principal Investigator Name
Pilar Azevedo
Principal Investigator Email
pilar.azevedo@hotmail.com
Contact Person Name
Pilar Azevedo
Contact Person Email
pilar.azevedo@hotmail.com

Italy

Earliest CTIS Part Ii Submission Date
31-05-2024
Latest Decision Or Authorization Date
03-02-2026
Processing Time Days
613
Number Of Sites
8
Number Of Participants
23

Sites

Site Name
Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico
Department Name
Centro Fibrosi Cistica - Clinica Pediatrica
Principal Investigator Name
Valeria Daccò
Principal Investigator Email
valeria.dacco@policlinico.mi.it
Contact Person Name
Valeria Daccò
Site Name
Azienda Ospedaliera Universitaria Meyer IRCCS
Department Name
Centro Fibrosi Cistica Pediatrica - Medicina Padiatrica
Principal Investigator Name
Vito Terlizzi
Principal Investigator Email
vito.terlizzi@meyer.it
Contact Person Name
Vito Terlizzi
Contact Person Email
vito.terlizzi@meyer.it
Site Name
Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico
Department Name
SC Pneumologia - Centro Fibrosi Cistica
Principal Investigator Name
Andrea Gramegna
Principal Investigator Email
andrea.gramegna@unimi.it
Contact Person Name
Andrea Gramegna
Contact Person Email
andrea.gramegna@unimi.it
Site Name
Azienda Ospedaliera Universitaria Integrata Verona
Department Name
Centro Fibrosi Cistica
Principal Investigator Name
Marco Cipolli
Principal Investigator Email
marco.cipolli@aovr.veneto.it
Contact Person Name
Marco Cipolli
Contact Person Email
marco.cipolli@aovr.veneto.it
Site Name
Azienda Ospedaliero-Universitaria San Luigi Gonzaga
Department Name
Pneumologia - Centro Fibrosi Cistica
Principal Investigator Name
Sara Demichelis
Principal Investigator Email
s.demichelis@sanluigi.piemonte.it
Contact Person Name
Sara Demichelis
Site Name
Azienda Ospedaliera Regionale San Carlo
Department Name
Centro Regionale di Fibrosi Cistica
Principal Investigator Name
Angela Pepe
Principal Investigator Email
angpepe01@gmail.com
Contact Person Name
Angela Pepe
Contact Person Email
angpepe01@gmail.com
Site Name
Azienda Ospedaliero Universitaria Delle Marche
Department Name
Centro Fibrosi Cistica
Principal Investigator Name
Benedetta Fabrizzi
Contact Person Name
Benedetta Fabrizzi
Site Name
IRCCS Istituto Giannina Gaslini
Department Name
Centro Fibrosi Cistica
Principal Investigator Name
Carlo Castellani
Principal Investigator Email
carlocastellani@gaslini.org
Contact Person Name
Carlo Castellani
Contact Person Email
carlocastellani@gaslini.org

Spain

Earliest CTIS Part Ii Submission Date
31-05-2024
Latest Decision Or Authorization Date
05-02-2026
Processing Time Days
615
Number Of Sites
8
Number Of Participants
21

Sites

Site Name
University Clinical Hospital Virgen De La Arrixaca
Department Name
Servicio de Pediatría
Principal Investigator Name
Pedro Mondéjar López
Principal Investigator Email
mondejarp@gmail.com
Contact Person Name
Pedro Mondéjar López
Contact Person Email
mondejarp@gmail.com
Site Name
Parc Tauli Hospital Universitari
Department Name
Servicio Medicina Pediátrica
Principal Investigator Name
Óscar Asensio de la Cruz
Principal Investigator Email
oasensio58@gmail.com
Contact Person Name
Óscar Asensio de la Cruz
Contact Person Email
oasensio58@gmail.com
Site Name
Hospital Universitari Vall D Hebron
Department Name
Departamento Respitarorio
Principal Investigator Name
Antonio Álvarez
Principal Investigator Email
antonio.alvarez@vallhebron.cat
Contact Person Name
Antonio Álvarez
Contact Person Email
antonio.alvarez@vallhebron.cat
Site Name
Sant Joan De Deu Barcelona Hospital
Department Name
Servicio Neumología
Principal Investigator Name
María Cols Roig
Principal Investigator Email
MCols@sjdhospitalbarcelona.org
Contact Person Name
María Cols Roig
Contact Person Email
MCols@sjdhospitalbarcelona.org
Site Name
Hospital Universitario Ramon Y Cajal
Department Name
Servicio de Pediatría
Principal Investigator Name
Enrique Blitz Castro
Principal Investigator Email
enrique.blitz@salud.madrid.org
Contact Person Name
Enrique Blitz Castro
Contact Person Email
enrique.blitz@salud.madrid.org
Site Name
Hospital Universitario Y Politecnico La Fe
Department Name
Unidad de Trasplante Pulmonar y Fibrosis Quística
Principal Investigator Name
Amparo Solé Jover
Principal Investigator Email
sole_amp@gva.es
Contact Person Name
Amparo Solé Jover
Contact Person Email
sole_amp@gva.es
Site Name
Hospital Universitario 12 De Octubre
Department Name
Servicio de Pediatría
Principal Investigator Name
Carmen Luna Paredes
Principal Investigator Email
cluna@salud.madrid.org
Contact Person Name
Carmen Luna Paredes
Contact Person Email
cluna@salud.madrid.org
Site Name
University Hospital Virgen Del Rocio S.L.
Department Name
Unidad de Gestion Clínica de Neumología y Cirugía de Tórax. Unidad de Fibrosis Quistica.
Principal Investigator Name
Esther Quintana Gallego
Principal Investigator Email
mariae.quintana.sspa@juntadeandalucia.es
Contact Person Name
Esther Quintana Gallego

Belgium

Earliest CTIS Part Ii Submission Date
31-05-2024
Latest Decision Or Authorization Date
02-02-2026
Processing Time Days
612
Number Of Sites
5
Number Of Participants
27

Sites

Site Name
UZ Leuven
Department Name
Campus Gasthuisberg
Principal Investigator Name
François Vermeulen
Principal Investigator Email
Francois.vermeulen@uzleuven.be
Contact Person Name
François Vermeulen
Contact Person Email
Francois.vermeulen@uzleuven.be
Site Name
Universitair Ziekenhuis Gent
Principal Investigator Name
Eva Van Braeckel
Principal Investigator Email
Eva.VanBraeckel@uzgent.be
Contact Person Name
Eva Van Braeckel
Contact Person Email
Eva.VanBraeckel@uzgent.be
Site Name
Hopital Erasme
Principal Investigator Name
Christiane Knoop
Principal Investigator Email
christiane.knoop@hubruxelles.be
Contact Person Name
Christiane Knoop
Site Name
UZ Brussel
Principal Investigator Name
Eef Vanderhelst
Principal Investigator Email
eef.vanderhelst@uzbrussel.be
Contact Person Name
Eef Vanderhelst
Contact Person Email
eef.vanderhelst@uzbrussel.be
Site Name
Cliniques Universitaires Saint-Luc
Principal Investigator Name
Sophie Gohy
Principal Investigator Email
sophie.gohy@uclouvain.be
Contact Person Name
Sophie Gohy
Contact Person Email
sophie.gohy@uclouvain.be

Hungary

Earliest CTIS Part Ii Submission Date
31-05-2024
Latest Decision Or Authorization Date
03-02-2026
Processing Time Days
613
Number Of Sites
2
Number Of Participants
30

Sites

Site Name
Orszagos Koranyi Pulmonologiai Intezet
Department Name
Cisztas Fibrozls Reszleg
Principal Investigator Name
Adrien Halasz
Principal Investigator Email
halasz.adrien@koranyi.hu
Contact Person Name
Adrien Halasz
Contact Person Email
halasz.adrien@koranyi.hu
Site Name
Reformatus Pulmonologiai Centrum
Department Name
Gyormektudogyogyaszati Osztaly
Principal Investigator Name
Istvan Laki
Principal Investigator Email
gyermektudo@gmail.com
Contact Person Name
Istvan Laki
Contact Person Email
gyermektudo@gmail.com

Netherlands

Earliest CTIS Part Ii Submission Date
31-05-2024
Latest Decision Or Authorization Date
09-02-2026
Processing Time Days
619
Number Of Sites
5
Number Of Participants
11

Sites

Site Name
Radboud universitair medisch centrum / RADBOUDUMC
Department Name
Amalia Children's Hospital, pediatrics
Principal Investigator Name
Jolt Roukema
Principal Investigator Email
jolt.roukema@radboudumc.nl
Contact Person Name
Jolt Roukema
Contact Person Email
jolt.roukema@radboudumc.nl
Site Name
Haga Hospital
Department Name
Pulmonology
Principal Investigator Name
Renske van der Meer
Principal Investigator Email
r.vandermeer@hagaziekenhuis.nl
Contact Person Name
Renske van der Meer
Contact Person Email
r.vandermeer@hagaziekenhuis.nl
Site Name
Stichting Amsterdam UMC
Department Name
Pulmonary Medicine
Principal Investigator Name
Josje Altenburg
Principal Investigator Email
j.altenburg@amsterdamumc.nl
Contact Person Name
Josje Altenburg
Contact Person Email
j.altenburg@amsterdamumc.nl
Site Name
Erasmus Universitair Medisch Centrum Rotterdam (Erasmus MC)
Department Name
Research Department Pulmonary Disease
Principal Investigator Name
Lieke Kamphuis
Principal Investigator Email
l.kamphuis@erasmusmc.nl
Contact Person Name
Lieke Kamphuis
Contact Person Email
l.kamphuis@erasmusmc.nl
Site Name
Universitair Medisch Centrum Utrecht
Department Name
Wilhelmina children's hospital, pediatrics
Principal Investigator Name
Kors van der Ent
Principal Investigator Email
k.vanderent@umcutrecht.nl
Contact Person Name
Kors van der Ent
Contact Person Email
k.vanderent@umcutrecht.nl

France

Earliest CTIS Part Ii Submission Date
31-05-2024
Latest Decision Or Authorization Date
03-02-2026
Processing Time Days
613
Number Of Sites
14
Number Of Participants
43

Sites

Site Name
Les Hopitaux Universitaires De Strasbourg
Department Name
Pédiatrie médico-chirurgicale
Principal Investigator Name
Laurence Weiss
Principal Investigator Email
laurence.weiss@chru-strasbourg.fr
Contact Person Name
Laurence Weiss
Site Name
Centre Hospitalier Intercommunal Creteil
Department Name
Service de Pédiatrie
Principal Investigator Name
Ralph Epaud
Principal Investigator Email
ralph.epaud@chicreteil.fr
Contact Person Name
Ralph Epaud
Contact Person Email
ralph.epaud@chicreteil.fr
Site Name
Fondation Ildys
Principal Investigator Name
Sophie Ramel
Principal Investigator Email
sophie.ramel@ildys.org
Contact Person Name
Sophie Ramel
Contact Person Email
sophie.ramel@ildys.org
Site Name
Robert Debre University Hospital
Department Name
Service de Gastro-entérologie, Mucoviscidose, Pneumologie
Principal Investigator Name
Véronique Houdouin
Principal Investigator Email
veronique.houdouin@aphp.fr
Contact Person Name
Véronique Houdouin
Contact Person Email
veronique.houdouin@aphp.fr
Site Name
Centre Hospitalier Universitaire De Montpellier
Department Name
Service de Pneumologie et Maladies Respiratoires
Principal Investigator Name
Raphaël Chiron
Principal Investigator Email
r-chiron@chu-montpellier.fr
Contact Person Name
Raphaël Chiron
Contact Person Email
r-chiron@chu-montpellier.fr
Site Name
Centre Hospitalier Universitaire De Bordeaux
Department Name
Pneumo-Pédiatrie
Principal Investigator Name
Stéphanie Bui
Principal Investigator Email
stephanie.bui@chu-bordeaux.fr
Contact Person Name
Stéphanie Bui
Contact Person Email
stephanie.bui@chu-bordeaux.fr
Site Name
Hospices Civils De Lyon
Department Name
Service de Médecine Interne
Principal Investigator Name
Isabelle Durieu
Principal Investigator Email
isabelle.durieu@chu-lyon.fr
Contact Person Name
Isabelle Durieu
Contact Person Email
isabelle.durieu@chu-lyon.fr
Site Name
Hospices Civils De Lyon
Department Name
Service de Pédiatrie, Allergologie, Mucoviscidose
Principal Investigator Name
Philippe Reix
Principal Investigator Email
philippe.reix@chu-lyon.fr
Contact Person Name
Philippe Reix
Contact Person Email
philippe.reix@chu-lyon.fr
Site Name
Centre Hospitalier Universitaire Rouen
Department Name
Pneumologie, Allergologie, Mucoviscidose Pédiatrique
Principal Investigator Name
Christophe Marguet
Principal Investigator Email
christophe.marguet@chu-rouen.fr
Contact Person Name
Christophe Marguet
Site Name
Assistance Publique Hopitaux De Paris
Department Name
Service de Physiologie-Explorations Fonctionnelles
Principal Investigator Name
Isabelle Fajac
Principal Investigator Email
isabelle.fajac@parisdescartes.fr
Contact Person Name
Isabelle Fajac
Site Name
Centre Hospitalier Regional Universitaire De Tours
Department Name
Service Pneumologie et Explorations Fonctionnelles Respiratoires
Principal Investigator Name
Julie Mankikian
Principal Investigator Email
j.mankikian@chu-tours.fr
Contact Person Name
Julie Mankikian
Contact Person Email
j.mankikian@chu-tours.fr
Site Name
Hospital Foch
Department Name
Service de Pneumologie (Adultes)
Principal Investigator Name
Dominique Grenet
Principal Investigator Email
d.grenet@hopital-foch.org
Contact Person Name
Dominique Grenet
Contact Person Email
d.grenet@hopital-foch.org
Site Name
Centre Hospitalier Universitaire De Nice
Department Name
Unité de Recherche Clinique Pneumologie
Principal Investigator Name
Sylvie Leroy
Principal Investigator Email
leroy.s2@chu-nice.fr
Contact Person Name
Sylvie Leroy
Contact Person Email
leroy.s2@chu-nice.fr
Site Name
Hopital Cardiologique
Department Name
Service de Pneumologie et Immuno-Allergologie
Principal Investigator Name
Olivier Le Rouzic
Principal Investigator Email
olivier.lerouzic@chru-lille.fr
Contact Person Name
Olivier Le Rouzic
Contact Person Email
olivier.lerouzic@chru-lille.fr

Austria

Earliest CTIS Part Ii Submission Date
31-05-2024
Latest Decision Or Authorization Date
09-02-2026
Processing Time Days
619
Number Of Sites
4
Number Of Participants
8

Sites

Site Name
Gemeinnuetzige Salzburger Landeskliniken Betriebsgesellschaft mbH
Department Name
Universitätsklinik für Pneumologie/Lungenheilkunde
Principal Investigator Name
Michael Studnicka
Principal Investigator Email
m.studnicka@salk.at
Contact Person Name
Michael Studnicka
Contact Person Email
m.studnicka@salk.at
Site Name
University Hospital Graz
Department Name
Division of Paediatric Pulmonology and Allergology and Cystic Fibrosis Centre
Principal Investigator Name
Ernst Eber
Principal Investigator Email
ernst.eber@medunigraz.at
Contact Person Name
Ernst Eber
Contact Person Email
ernst.eber@medunigraz.at
Site Name
Medizinische Universitaet Innsbruck
Department Name
Kinder-und Jugendheilkunde, Pädiatrie III
Principal Investigator Name
Dorothea Appelt
Principal Investigator Email
dorothea.appelt@i-med.ac.at
Contact Person Name
Dorothea Appelt
Contact Person Email
dorothea.appelt@i-med.ac.at
Site Name
Medical University Of Vienna
Department Name
Klinische Abteilung für Pädiatrische Pulmologie
Principal Investigator Name
Zsolt Szepfalusi
Principal Investigator Email
zsolt.szepfalusi@meduniwien.ac.at
Contact Person Name
Zsolt Szepfalusi

Germany

Earliest CTIS Part Ii Submission Date
31-05-2024
Latest Decision Or Authorization Date
04-02-2026
Processing Time Days
614
Number Of Sites
14
Number Of Participants
100

Sites

Site Name
Universitaetsklinikum Essen AöR
Department Name
Klinik für Kinderheilkunde III
Principal Investigator Name
Florian Stehling
Principal Investigator Email
florian.stehling@uk-essen.de
Contact Person Name
Florian Stehling
Contact Person Email
florian.stehling@uk-essen.de
Site Name
Universitaetsmedizin der Johannes Gutenberg-Universitaet Mainz KöR
Department Name
Klinik für Kinder-und Jugendmedizin, Pädiatrische Pneumologie, Allergologie und Mukoviszidose
Principal Investigator Name
Oliver Nitsche
Principal Investigator Email
oliver.nitsche@unimedizin-mainz.de
Contact Person Name
Oliver Nitsche
Site Name
Ruhrlandklinik Westdeutsches Lungenzentrum Am Universitaetsklinikum Essen gGmbH
Department Name
Westdeutsches Lungenzentrum
Principal Investigator Name
Sivagurunathan Sutharsan
Principal Investigator Email
sivagurunathan.sutharsan@rlk.uk-essen.de
Contact Person Name
Sivagurunathan Sutharsan
Site Name
Universitaetsklinikum Giessen und Marburg GmbH
Department Name
Zentrum fuer Kinderheilkunde und Jugendmedizin, Paed. Pneumologie
Principal Investigator Name
Azadeh Bagheri-Potthoff
Contact Person Name
Azadeh Bagheri-Potthoff
Site Name
Universitaetsklinikum Wuerzburg AöR
Department Name
Kinderklinik
Principal Investigator Name
Helge Hebestreit
Principal Investigator Email
hebestreit_h@ukw.de
Contact Person Name
Helge Hebestreit
Contact Person Email
hebestreit_h@ukw.de
Site Name
Medizinische Hochschule Hannover
Department Name
Klinik für Pädiatrische Pneumologie, Allergologie und Neonatologie
Principal Investigator Name
Felix Ringshausen
Principal Investigator Email
Ringshausen.Felix@mh-hannover.de
Contact Person Name
Felix Ringshausen
Site Name
Universitaetsklinikum Jena KöR
Department Name
Klinik für Kinder und Jugendmedizin
Principal Investigator Name
Michael Lorenz
Principal Investigator Email
michael.lorenz@med.uni-jena.de
Contact Person Name
Michael Lorenz
Contact Person Email
michael.lorenz@med.uni-jena.de
Site Name
Universitaetsklinikum Ulm AöR
Department Name
Klinik für Kinder- und Jugendmedizin Ambulanz für Mukoviszidose, Pneumologie, Allergologie
Principal Investigator Name
Sebastian Bode
Principal Investigator Email
Sebastian.Bode@uniklinik-ulm.de
Contact Person Name
Sebastian Bode
Site Name
Charite Universitaetsmedizin Berlin KöR
Department Name
Department of Pediatric Respiratory Medicine, Immunology and Critical Care Medicine
Principal Investigator Name
Mirjam Stahl
Principal Investigator Email
mirjam.stahl@charite.de
Contact Person Name
Mirjam Stahl
Contact Person Email
mirjam.stahl@charite.de
Site Name
Universitaetsklinikum Erlangen AöR
Department Name
Kinder und Jugendklinik
Principal Investigator Name
Sabina Schmitt-Grohé
Principal Investigator Email
sabina.schmitt-grohe@uk.erlangen.de
Contact Person Name
Sabina Schmitt-Grohé
Site Name
Universitaetsklinikum Frankfurt AöR
Department Name
Christiane Herzog CF-Zentrum
Principal Investigator Name
Wolfgang Gleiber
Principal Investigator Email
Wolfgang.gleiber@kgu.de
Contact Person Name
Wolfgang Gleiber
Contact Person Email
Wolfgang.gleiber@kgu.de
Site Name
Pneumological Study Center Munich-West
Department Name
Pneumologisches Studienzentrum
Principal Investigator Name
Rainald Fischer
Principal Investigator Email
rainald.fischer@gmail.com
Contact Person Name
Rainald Fischer
Contact Person Email
rainald.fischer@gmail.com
Site Name
Klinikum Ernst von Bergmann gGmbH
Department Name
Kinder- und Jugendklinik - CF-Zentrum
Principal Investigator Name
Carsten Schwarz
Principal Investigator Email
Carsten.Schwarz@klinikumwb.de
Contact Person Name
Carsten Schwarz
Contact Person Email
Carsten.Schwarz@klinikumwb.de
Site Name
Medizinische Hochschule Hannover
Department Name
Pädiatrisches Zentrum für Cystische Fibrose (Mukoviszidose)
Principal Investigator Name
Anna-Maria Dittrich
Principal Investigator Email
Dittrich.Anna-Maria@mh-hannover.de
Contact Person Name
Anna-Maria Dittrich

Sweden

Earliest CTIS Part Ii Submission Date
31-05-2024
Latest Decision Or Authorization Date
02-02-2026
Processing Time Days
612
Number Of Sites
3
Number Of Participants
20

Sites

Site Name
Sahlgrenska University Hospital-Vaestra Goetalandsregionen
Department Name
Allergi- lung och CF-mottagningen barn
Principal Investigator Name
Emily Krantz
Principal Investigator Email
emily.amundson@vgregion.se
Contact Person Name
Emily Krantz
Contact Person Email
emily.amundson@vgregion.se
Site Name
Karolinska University Hospital
Department Name
Stockholm CF Center K56-58
Principal Investigator Name
Isabelle de Monestrol
Contact Person Name
Isabelle de Monestrol
Site Name
Region Skane Skanes Universitetssjukhus
Department Name
Lungmottagning Cystisk fibros
Principal Investigator Name
Ulrika Lindberg
Principal Investigator Email
Ulrika.Lindberg@skane.se
Contact Person Name
Ulrika Lindberg
Contact Person Email
Ulrika.Lindberg@skane.se

Norway

Earliest CTIS Part Ii Submission Date
31-05-2024
Latest Decision Or Authorization Date
09-02-2026
Processing Time Days
619
Number Of Sites
2
Number Of Participants
4

Sites

Site Name
Helse Bergen HF
Department Name
Pediatrics and Adolescent Medicine
Principal Investigator Name
Mette Engan
Principal Investigator Email
mette.engan@helse-bergen.no
Contact Person Name
Mette Engan
Contact Person Email
mette.engan@helse-bergen.no
Site Name
Oslo University Hospital HF
Department Name
Department of Paediatric Medicine
Principal Investigator Name
Egil Bakkeheim
Principal Investigator Email
uxbake@ous-hf.no
Contact Person Name
Egil Bakkeheim
Contact Person Email
uxbake@ous-hf.no

Ireland

Earliest CTIS Part Ii Submission Date
31-05-2024
Latest Decision Or Authorization Date
03-02-2026
Processing Time Days
613
Number Of Sites
5
Number Of Participants
12

Sites

Site Name
St Vincent's University Hospital
Department Name
Respiratory Medicine
Principal Investigator Name
Edward McKone
Principal Investigator Email
edward.mckone@ucd.ie
Contact Person Name
Edward McKone
Contact Person Email
edward.mckone@ucd.ie
Site Name
University Hospital Limerick
Department Name
Paediatric Department
Principal Investigator Name
Barry Linnane
Principal Investigator Email
barry.linnane@hse.ie
Contact Person Name
Barry Linnane
Contact Person Email
barry.linnane@hse.ie
Site Name
Children's Health Ireland (Crumlin)
Department Name
Respiratory Medicine
Principal Investigator Name
Desmond Cox
Principal Investigator Email
des.cox@olchc.ie
Contact Person Name
Desmond Cox
Contact Person Email
des.cox@olchc.ie
Site Name
Children's Health Ireland (Temple Street)
Department Name
Respiratory department
Principal Investigator Name
Michael Williamson
Principal Investigator Email
michael.williamson@cuh.ie
Contact Person Name
Michael Williamson
Contact Person Email
michael.williamson@cuh.ie
Site Name
Cork University Hospital
Department Name
Department of Medicine
Principal Investigator Name
Barry Plant
Principal Investigator Email
b.plant@ucc.ie
Contact Person Name
Barry Plant
Contact Person Email
b.plant@ucc.ie

Poland

Earliest CTIS Part Ii Submission Date
31-05-2024
Latest Decision Or Authorization Date
08-02-2026
Processing Time Days
618
Number Of Sites
2
Number Of Participants
4

Sites

Site Name
Samodzielny Zespol Publicznych Zakladow Opieki Zdrowotnej Im. Dzieci Warszawy W Dziekanowie Lesnym
Department Name
Oddział Chorób Płuc
Principal Investigator Name
Dorota Sands
Principal Investigator Email
dorota.sands@imid.med.pl
Contact Person Name
Dorota Sands
Contact Person Email
dorota.sands@imid.med.pl
Site Name
Szpital Dzieciecy Polanki Im. Macieja Plazynskiego W Gdansku Sp. z o.o.
Department Name
Oddział Pediatrii i Mukowiscydozy
Principal Investigator Name
Ewa Sapiejka
Principal Investigator Email
e.sapiejka@wp.pl
Contact Person Name
Ewa Sapiejka
Contact Person Email
e.sapiejka@wp.pl

Greece

Earliest CTIS Part Ii Submission Date
31-05-2024
Latest Decision Or Authorization Date
02-02-2026
Processing Time Days
612
Number Of Sites
2
Number Of Participants
6

Sites

Site Name
Ippokratio General Hospital Of Thessaloniki
Department Name
3rd Pediatrics Department of Aristotle University of Thessaloniki
Principal Investigator Name
Elpis Chatziagorou
Principal Investigator Email
pqre@ippokratio.gr
Contact Person Name
Elpis Chatziagorou
Contact Person Email
pqre@ippokratio.gr
Site Name
Geniko Nosokomeio Thessalonikis George Papanikolaou
Department Name
Pulmonary Clinic of AUTH
Principal Investigator Name
Katerina Manika
Principal Investigator Email
ktmn05@yahoo.gr
Contact Person Name
Katerina Manika
Contact Person Email
ktmn05@yahoo.gr

Denmark

Earliest CTIS Part Ii Submission Date
31-05-2024
Latest Decision Or Authorization Date
05-02-2026
Processing Time Days
615
Number Of Sites
2
Number Of Participants
8

Sites

Site Name
Rigshospitalet
Department Name
Cystisk Fibrosis Center afs. 8632
Principal Investigator Name
Inger Hee Mabuza Mathiesen
Principal Investigator Email
lnger.hee.mabuza.mathiesen@regionh.dk
Contact Person Name
Inger Hee Mabuza Mathiesen
Site Name
Aarhus Universitetshospital
Department Name
Børn og Unge, Aarhus Universitetshospital
Principal Investigator Name
Hanne Olesen
Principal Investigator Email
hannoles@rm.dk
Contact Person Name
Hanne Olesen
Contact Person Email
hannoles@rm.dk

Sponsor

Primary sponsor

Full Name
Vertex Pharmaceuticals Inc.
Organisation Type
Pharmaceutical company
Country Of Registered Address
United States

Contract research organisations

Name
Syneos Health Inc.
Responsibilities
Study start-up activities; contact email spyros.pamfilos@syneoshealth.com

Third parties

  • {"country":"United States","full_name":"Syneos Health Inc.","duties_or_roles":"Study start-up activities","organisation_type":"Pharmaceutical company"}

Investigational products

Investigational Product Name
Alyftrek 125 mg/50 mg/10 mg film-coated tablets
Active Substance
tezacaftor, deutivacaftor, vanzacaftor
Modality
Small molecule
Routes Of Administration
ORAL USE
Route
oral
Authorisation Status
Marketing authorisation: EU/1/25/1943/002
Orphan Designation
Yes
Maximum Dose
20 mg (maxDailyDoseAmount field value: 20 mg)
Investigational Product Name
VX-121/VX-661/VX-561 Film-coated tablet
Active Substance
tezacaftor, deutivacaftor, vanzacaftor
Modality
Small molecule
Routes Of Administration
ORAL
Route
oral
Orphan Designation
Yes
Maximum Dose
20 mg (maxDailyDoseAmount field value: 20 mg)
Combination Treatment
Yes

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