Overview
Sponsor-declared trial summary
Phase
Therapeutic confirmatory (Phase III)
Status
Ongoing, recruitment ended
Participants planned
323
Countries
13
Sites
74
Cystic Fibrosis
Part A and B: To evaluate the long-term safety and tolerability of elexacaftor (ELX)/tezacaftor (TEZ)/ivacaftor (IVA)
Key facts
- Sponsor
- Vertex Pharmaceuticals Inc.
- Participant type
- Pediatric, Patients
- Age range
- 0-17 years, 18-64 years
- Gender
- Male and Female
- Therapeutic area
- Diseases [C] - Respiratory Tract Diseases [C08]
- Trial duration
- 8 Nov 2022 → ongoing
- Decision date (initial)
- 2024-10-28
- Transition trial
- Yes
- Low-intervention
- No
- Rare-disease indication
- Yes
- Vulnerable population
- Yes
External identifiers
- EU CT number
- 2024-515637-14-00
- EudraCT number
- 2021-005914-33
Trial design
CTIS Part I — objectives, methods, condition coding
Main objective
Scope: Safety, Efficacy
Part A and B: To evaluate the long-term safety and tolerability of elexacaftor (ELX)/tezacaftor (TEZ)/ivacaftor (IVA)
Secondary objectives 1
- Part A Only: To evaluate the long-term efficacy and pharmacodynamics (PD) of ELX/TEZ/IVA
Conditions and MedDRA coding
Cystic Fibrosis
| Version | Level | Code | Term | System organ class |
|---|---|---|---|---|
| 20.0 | PT | 10011762 | Cystic fibrosis | 100000004850 |
Regulatory references
- EMA paediatric investigation plan (PIP)
- EMEA-002324-PIP01-17
- Plan to share IPD
- No
- IPD plan description
- Details on Vertex data sharing criteria and process for requesting access can be found at: https://www.vrtx.com/independent-research/clinical-trial-data-sharing
Eligibility criteria
Principal inclusion / exclusion criteria as submitted by sponsor
Inclusion criteria 5
- Subject (or the subject’s legally appointed and authorized representative) will sign and date an informed consent form (ICF) and, when appropriate, an assent form.
- Willing and able to comply with scheduled visits, treatment plan, study restrictions, laboratory tests, contraceptive guidelines (as applicable), and other study procedures. • For subjects <18 years of age: as judged by the investigator, parent or legal guardian must be able to understand protocol requirements, restrictions, and instructions and the parent or legal guardian should be able to ensure that the subject will comply with and is likely to complete the study as planned.
- Did not withdraw consent from the parent study.
- Part A: Meets at least 1 of the following criteria: • Completed study drug treatment in the parent study. • Had study drug interruption(s) in the parent study, but completed study visits up to the last scheduled visit of the Treatment Period of the parent study. 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 completed study visits up to the last scheduled visit of the Treatment Period of Part A.
- Willing to remain on a stable CF treatment regimen (other than CFTR modulators, as defined in Section 9.5) through completion of study participation.
Exclusion criteria 4
- History of any illness or any clinical condition that might confound the results of the study or pose an additional risk in administering study drug(s) to the subject.
- History of drug intolerance in the parent study that would pose an additional risk to the subject. (e.g., subjects with a history of allergy or hypersensitivity to the study drug).
- Pregnant and nursing females. Females of childbearing potential (Section 11.5.6.1) must have a negative pregnancy test at the Day 1 Visit (in Part A and Part B) before receiving the first dose of study drug.
- Current participation in an investigational drug trial (other than the parent study). Participation in a noninterventional study (including observational studies, registry studies, and studies requiring blood collections without administration of study drug) and screening for another Vertex study is permitted.
Endpoints
Primary and secondary outcome measures (English text)
Primary endpoints 1
- Parts A and B: Safety and tolerability based on adverse events (AEs), clinical laboratory values, ECGs, vital signs, and pulse oximetry
Secondary endpoints 6
- Part A Only: Absolute change from baseline in percent predicted forced expiratory volume in 1 second (ppFEV1)
- Absolute change from baseline in sweat chloride (SwCl)
- Absolute change from baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) respiratory domain (RD) score
- Absolute change from baseline in body mass index (BMI)
- Absolute change from baseline in weight
- Number of pulmonary exacerbations (PEx)
Investigational products
Investigational medicinal products (IMPs), comparators, placebo, auxiliary
Test 8
Kaftrio 37.5 mg/25 mg/50 mg film-coated tablets
PRD9418409 · Product
- Active substance
- Tezacaftor
- Substance synonyms
- VX-661, 1-(2,2-DIFLUORO-2H-1,3-BENZODIOXOL-5-YL)-N-{1-[(2R)-2,3-DIHYDROXYPROPYL]-6-FLUORO-2-(1-HYDROXY-2-METHYLPROPAN-2-YL)-1H-INDOL-5-YL}CYCLOPROPANE-1-CARBOXAMIDE, 1-(2,2-DIFLUORO-1,3-BENZODIOXOL-5-YL)-N-{1-[(2R)-2,3-DIHYDROXYPROPYL]-6-FLUORO-2-(1-HYDROXY-2-METHYLPROPAN-2-YL)-1HINDOL-5-YL}CYCLOPROPANECARBOXAMIDE
- Pharmaceutical form
- FILM-COATED TABLET
- Route of administration
- ORAL USE
- Max daily dose
- 50 mg milligram(s)
- Max total dose
- 50 mg milligram(s)
- Max treatment duration
- 192 Week(s)
- Authorisation status
- Authorised
- ATC code
- R07AX32 — -
- Marketing authorisation
- EU/1/20/1468/002
- MA holder
- VERTEX PHARMACEUTICALS (IRELAND) LIMITED
- MA country
- EU
- Paediatric formulation
- No
- Orphan designation
- Yes
- Orphan designation number
- EU/3/18/2116
- Modified vs. Marketing Authorisation
- Yes
- Modification description
- Packaged and labelled for clinical use and is QP released at the clinical site Fisher Clinical Services GmBH
Kaftrio 75 mg/50 mg/100 mg film-coated tablets
PRD8271954 · Product
- Active substance
- Tezacaftor
- Substance synonyms
- VX-661, 1-(2,2-DIFLUORO-2H-1,3-BENZODIOXOL-5-YL)-N-{1-[(2R)-2,3-DIHYDROXYPROPYL]-6-FLUORO-2-(1-HYDROXY-2-METHYLPROPAN-2-YL)-1H-INDOL-5-YL}CYCLOPROPANE-1-CARBOXAMIDE, 1-(2,2-DIFLUORO-1,3-BENZODIOXOL-5-YL)-N-{1-[(2R)-2,3-DIHYDROXYPROPYL]-6-FLUORO-2-(1-HYDROXY-2-METHYLPROPAN-2-YL)-1HINDOL-5-YL}CYCLOPROPANECARBOXAMIDE
- Pharmaceutical form
- FILM-COATED TABLET
- Route of administration
- ORAL USE
- Max daily dose
- 100 mg milligram(s)
- Max total dose
- 100 mg milligram(s)
- Max treatment duration
- 192 Week(s)
- Authorisation status
- Authorised
- ATC code
- R07AX32 — -
- Marketing authorisation
- EU/1/20/1468/001
- MA holder
- VERTEX PHARMACEUTICALS (IRELAND) LIMITED
- MA country
- EU
- Paediatric formulation
- No
- Orphan designation
- Yes
- Orphan designation number
- EU/3/18/2116
- Modified vs. Marketing Authorisation
- Yes
- Modification description
- Packaged and labelled for clinical use and is QP released at the clinical site Fisher Clinical Services GmBH
Kalydeco 150 mg film-coated tablets
PRD3203571 · Product
- Active substance
- Ivacaftor
- Substance synonyms
- VX-770, N-(2,4-DI-TERT-BUTYL-5-HYDROXYPHENYL)-1,4-DIHYDRO-4-OXOQUINOLINE-3-CARBOXAMIDE
- Pharmaceutical form
- FILM-COATED TABLET
- Route of administration
- ORAL USE
- Max daily dose
- 150 mg milligram(s)
- Max total dose
- 150 mg milligram(s)
- Max treatment duration
- 192 Week(s)
- Authorisation status
- Authorised
- ATC code
- R07AX02 — -
- Marketing authorisation
- EU/1/12/782/001
- MA holder
- VERTEX PHARMACEUTICALS (IRELAND) LIMITED
- MA country
- EU
- Paediatric formulation
- No
- Orphan designation
- Yes
- Orphan designation number
- EU/3/08/556
- Modified vs. Marketing Authorisation
- Yes
- Modification description
- Packaged and labelled for clinical use and is QP released at the clinical site Fisher Clinical Services GmBH
PRD7963761 · Product
- Active substance
- Ivacaftor
- Other product name
- IVACAFTOR
- Pharmaceutical form
- FILM-COATED TABLET
- Route of administration
- ORAL USE
- Max daily dose
- 150 mg milligram(s)
- Max total dose
- 150 mg milligram(s)
- Max treatment duration
- 192 Week(s)
- Authorisation status
- Not Authorised
- MA holder
- VERTEX PHARMACEUTICALS, INCORPORATED
- Paediatric formulation
- No
- Orphan designation
- Yes
- Orphan designation number
- EU/3/08/556
PRD7900328 · Product
- Active substance
- Ivacaftor
- Other product name
- IVACAFTOR
- Pharmaceutical form
- FILM-COATED TABLET
- Route of administration
- ORAL USE
- Max daily dose
- 75 mg milligram(s)
- Max total dose
- 75 mg milligram(s)
- Max treatment duration
- 192 Week(s)
- Authorisation status
- Not Authorised
- MA holder
- VERTEX PHARMACEUTICALS, INCORPORATED
- Paediatric formulation
- No
- Orphan designation
- Yes
- Orphan designation number
- EU/3/08/556
VX-445/VX-661/VX-770 fixed-dose combination tablet
PRD7975086 · Product
- Active substance
- Tezacaftor
- Other product name
- VX-445/TEZ/IVA; VX-445/Tezacaftor/Ivacaftor
- Pharmaceutical form
- FILM-COATED TABLET
- Route of administration
- ORAL USE
- Max daily dose
- 50 mg milligram(s)
- Max total dose
- 50 mg milligram(s)
- Max treatment duration
- 192 Week(s)
- Authorisation status
- Not Authorised
- MA holder
- VERTEX PHARMACEUTICALS, INCORPORATED
- Paediatric formulation
- No
- Orphan designation
- Yes
- Orphan designation number
- EU/3/18/2116
VX-445/VX-661/VX-770 film-coated fixed-dose combination tablet
PRD7400755 · Product
- Active substance
- Tezacaftor
- Other product name
- VX-445/TEZ/IVA; VX-445/Tezacaftor/Ivacaftor; Elexacaftor/Tezacaftor/Ivacaftor; ELX/TEZ/IVA
- Pharmaceutical form
- FILM-COATED TABLET
- Route of administration
- ORAL USE
- Max daily dose
- 100 mg milligram(s)
- Max total dose
- 100 mg milligram(s)
- Max treatment duration
- 192 Week(s)
- Authorisation status
- Not Authorised
- MA holder
- VERTEX PHARMACEUTICALS, INCORPORATED
- Paediatric formulation
- No
- Orphan designation
- Yes
- Orphan designation number
- EU/3/18/2116
Kalydeco 75 mg film-coated tablets
PRD8533015 · Product
- Active substance
- Ivacaftor
- Substance synonyms
- VX-770, N-(2,4-DI-TERT-BUTYL-5-HYDROXYPHENYL)-1,4-DIHYDRO-4-OXOQUINOLINE-3-CARBOXAMIDE
- Pharmaceutical form
- FILM-COATED TABLET
- Route of administration
- ORAL USE
- Max daily dose
- 75 mg milligram(s)
- Max total dose
- 75 mg milligram(s)
- Max treatment duration
- 192 Week(s)
- Authorisation status
- Authorised
- ATC code
- R07AX02 — -
- Marketing authorisation
- EU/1/12/782/007
- MA holder
- VERTEX PHARMACEUTICALS (IRELAND) LIMITED
- MA country
- EU
- Paediatric formulation
- No
- Orphan designation
- Yes
- Orphan designation number
- EU/3/08/556
- Modified vs. Marketing Authorisation
- Yes
- Modification description
- Packaged and labelled for clinical use and is QP released at the clinical site Fisher Clinical Services GmBH
Sponsors and contacts
Sponsor organisations, regulatory contacts, third parties
Vertex Pharmaceuticals Inc.
- Sponsor organisation
- Vertex Pharmaceuticals Inc.
- Address
- 50 Northern Avenue
- City
- Boston
- Postcode
- 02210-1862
- Country
- United States
Scientific contact point
- Organisation
- Vertex Pharmaceuticals Inc.
- Contact name
- Clinical Trials and Medical Info
Public contact point
- Organisation
- Vertex Pharmaceuticals Inc.
- Contact name
- Clinical Trials and Medical Info
Locations
13 EU/EEA countries · 74 investigational sites
By country
| Country | MS status | Planned subjects | Sites |
|---|---|---|---|
| Austria | Ongoing, recruitment ended | 2 | 1 |
| Belgium | Ongoing, recruitment ended | 31 | 6 |
| Czechia | Ongoing, recruitment ended | 8 | 2 |
| France | Ongoing, recruitment ended | 63 | 16 |
| Germany | Ended | 66 | 14 |
| Hungary | Ongoing, recruitment ended | 3 | 1 |
| Italy | Ongoing, recruitment ended | 44 | 11 |
| Netherlands | Ongoing, recruitment ended | 15 | 5 |
| Norway | Ended | 3 | 1 |
| Poland | Ongoing, recruitment ended | 12 | 2 |
| Portugal | Ongoing, recruitment ended | 6 | 1 |
| Spain | Ongoing, recruitment ended | 36 | 12 |
| Sweden | Ended | 6 | 2 |
| Rest of world
Canada, Switzerland
|
— | 28 | — |
Investigational sites
Medizinische Universitaet Innsbruck
Kinder-und Jugendheilkunde, Pädiatrie III, Anichstrasse 35, 6020, Innsbruck
UZ Leuven
/, Herestraat 49, 3000, Leuven
Universitair Ziekenhuis Gent
/, Corneel Heymanslaan 10, 9000, Gent
UZ Brussel
/, Laarbeeklaan 101, 1090, Jette
Cliniques Universitaires Saint-Luc
/, Hippokrateslaan 10, Batiment 54, Sint-Lambrechts-Woluwe
Hopital Erasme
/, Lennikse Baan 808, 1070, Anderlecht
Antwerp University Hospital
/, Drie Eikenstraat 655, 2650, Edegem
Fakultni Nemocnice Brno
Klinika dětských infekčních nemocí, Jihlavska 340/20, Bohunice, Brno
Fakultni Nemocnice Motol A Homolka
Ústav lékařské mikrobiologie 2. lékařské fakulty UK a centrum cystické fibrózy, V Uvalu 84/1, Motol, Prague
Centre Hospitalier Regional Universitaire De Tours
Service Pneumologie et Explorations Fonctionnelles Respiratoires, 2 Boulevard Tonnelle, 37000, Tours
Centre Hospitalier Intercommunal Creteil
Service de pédiatrie, 40 Avenue De Verdun, 94000, Creteil
Centre Hospitalier Universitaire De Nantes
Service Pneumologie Institut du Thorax, Boulevard Du Professeur Jacques Monod, 44800, Saint Herblain
Centre Hospitalier Universitaire De Montpellier
Service de pneumologie et Maladies Respiratoires, 371 Avenue Du Doyen Gaston Giraud, 34090, Montpellier
Hospices Civils De Lyon
Service de Pédiatrie, Allergologie, Mucoviscidose, 59 Boulevard Pinel, 69500, Bron
Centre Hospitalier Universitaire De Rennes
Service de pédiatrie, 16 Boulevard De Bulgarie, Bp 90349, Rennes
Centre Hospitalier Regional De Marseille
Service de pneumologie, 265 Chemin Des Bourrely, 13015, Marseille
Assistance Publique Hopitaux De Paris
Service de Gastro-entérologie, Mucoviscidose, Pneumologie, 48 Boulevard Serurier, 75019, Paris
Fondation Ildys
NA, Rue Alain Colas, Cs 31826, Brest Cedex 2
Hopital Cardiologique
Service de Pneumologie et Immuno-Allergologie, Boulevard Du Professeur Jules Leclercq, 59037, Lille Cedex
Centre Hospitalier Universitaire De Toulouse
Service de Pneumologie Allergologie, CRCM Adulte, 24 Chemin De Pouvourville, 31400, Toulouse
Centre Hospitalier Lyon Sud
Service de Médecine Interne- Centre de référence Mucoviscidose- Bâtiment 1A –2ème étage, 165 Chemin du Grand Revoyet, 69495 Pierre Bénite, Pierre Bénite
Assistance Publique Hopitaux De Paris
Dept. of pediatric pneumology and allergology - Rare diseases reference center, 149 Rue De Sevres, 75015, Paris
Assistance Publique Hopitaux De Paris
Service de Physiologie-Explorations Fonctionnelles, 27 Rue Du Faubourg Saint Jacques, 75014, Paris
Centre Hospitalier Universitaire De Nice
Unité de Recherche Clinique Pneumologie, 30 Voie Romaine, 06000, Nice
Centre Hospitalier Universitaire Reims
Département de pédiatrie A, 45 Rue Cognacq Jay, 51100, Reims
Universitaetsmedizin der Johannes Gutenberg-Universitaet Mainz KöR
Klinik und Poliklinik für Kinder- und Jugendmedizin Pädiatrische Pneumologie, Allergologie und Mukov, Langenbeckstrasse 1, Oberstadt, Mainz
University Hospital Cologne AöR
CF Center Cologne, Kerpener Strasse 62, Lindenthal, Cologne
Medizinische Hochschule Hannover
Klinik für Pädiatrische Pneumologie, Allergologie und Neonatologie, Carl-Neuberg-Strasse 1, Gross Buchholz, Hanover
Universitaetsklinikum Jena KöR
Klinik für Kinder und Jugendmedizin, Am Klinikum 1, Lobeda, Jena
Ruhrlandklinik Westdeutsches Lungenzentrum Am Universitaetsklinikum Essen gGmbH
Ruhrlandklinik - Westdeutsches Lungenzentrum am Universitätsklinikum Essen gGmbH, Tueschener Weg 40, Heidhausen, Essen
Klinikum der Universitaet Muenchen AöR
Paediatric Pneumology, Lindwurmstrasse 4, Ludwigsvorstadt-Isarvorstadt, Munich
Justus-Liebig-Universitaet Giessen
Abteilung Allgemeine Pädiatrie und Neonatologie Funktionsbereich Päd. Pneumologie und Allergologie, Feulgenstrasse 10-12, 35392, Giessen
Charite Universitaetsmedizin Berlin KöR
Department of Pediatric Respiratory Medicine, Immunology and Critical Care Medicine, Augustenburger Platz 1, Wedding, Berlin
Pneumological Study Center Munich West
Lungenheilkunde München-Pasing, Gleichmannstrasse 5, 81241, Munich
Universitaetsklinikum Frankfurt AöR
Pediatric Pulmonology, Allergy and Cystic Fibrosis, Theodor-Stern-Kai 7, 60590, Frankfurt Am Main
Universitaetsklinikum Muenster AöR
Klinik für Kinder- und Jugendmedizin Pädiatrische Pneumologie, Albert-Schweitzer-Campus 1, Sentrup, Muenster
Klinikum Ernst von Bergmann gGmbH
Kinder- und Jugendklinik - CF-Zentrum, Charlottenstrasse 72, Noerdliche Innenstadt, Potsdam
Medizinische Hochschule Hannover
Klinik für Pädiatrische Pneumologie, Allergologie und Neonatologie, Carl-Neuberg-Strasse 1, Gross Buchholz, Hanover
Universitaetsklinikum Halle (Saale) AöR
Universitätsklinik und Poliklinik für Innere Medizin I - Schwerpunkt Pneumologie, Ernst-Grube-Strasse 40, Kroellwitz, Halle Saale
Orszagos Koranyi Pulmonologiai Intezet
Cisztas Fibrozls Reszleg, Koranyi Frigyes Ut 1, 1121, Budapest XII
Ospedale Pediatrico Bambino Gesu
UO Fibrosi Cistica, Piazza Di Sant'onofrio 4, 00165, Rome
IRCCS Istituto Giannina Gaslini
Centro Fibrosi Cistica, Via Gerolamo Gaslini 5, 16147, Genoa
Azienda Ospedaliera Regionale San Carlo
Centro Regionale di Fibrosi Cistica, Via Potito Petrone, 85100, Potenza
Azienda Ospedaliero Universitaria Delle Marche
Centro Fibrosi Cistica, Via Conca 71, 60126, Ancona
Azienda Ospedaliera Universitaria Meyer IRCCS
Centro Fibrosi Cistica - Medicina Pediatrica, Viale Gaetano Pieraccini 24, 50139, Florence
Azienda Ospedaliera Universitaria Federico II Di Napoli
Unità di Geriatria e Fibrosi Cistica dell’Adulto, Via Sergio Pansini 5, 80131, Naples
Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico
SC Pneumologia - Centro Fibrosi Cistica, Via Francesco Sforza 28, 20122, Milan
Azienda Ospedaliera Universitaria Gaetano Martino Messina
Unità di Gastroenterologia Pediatrica e Fibrosi Cistica, Via Consolare Valeria N 1, 98124, Messina
Azienda Ospedaliera Universitaria Integrata Verona
Centro Fibrosi Cistica, Piazzale Aristide Stefani 1, 37126, Verona
Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico
Centro Fibrosi Cistica - Clinica Pediatrica, Via Francesco Sforza 28, 20122, Milan
Azienda Ospedaliero-Universitaria San Luigi Gonzaga
Pneumologia - Centro Fibrosi Cistica, Regione Gonzole 10, 10043, Orbassano
Universitair Medisch Centrum Utrecht
Department of Pediatric Pulmonology / Research, Heidelberglaan 100, 3584 CX, Utrecht
Radboud universitair medisch centrum Stichting
Department of pediatrics, Geert Grooteplein Zuid 10, 6525 GA, Nijmegen
VUMC Stichting
Department of Pulmonology, De Boelelaan 1117, 1081 HV, Amsterdam
Haga Hospital
Department Pulmonology, Els Borst-Eilersplein 275, 2545 AA, 's-Gravenhage
Erasmus Universitair Medisch Centrum Rotterdam (Erasmus MC)
Department of Pediatric Respiratory Medicine, Dr. Molewaterplein 40, 3015 GD, Rotterdam
Oslo University Hospital HF
Department of Paediatric Medicine, Taarnbygget, Kirkeveien 166, Oslo
Samodzielny Zespol Publicznych Zakladow Opieki Zdrowotnej Im. Dzieci Warszawy W Dziekanowie Lesnym
Oddział Chorób Płuc, Ul. Marii Konopnickiej 65, Dziekanow Lesny, Lomianki
Szpital Dzieciecy Polanki Im. Macieja Plazynskiego W Gdansku Sp. z o.o.
Oddział Pediatrii i Mukowiscydozy, Ul. Polanki 119, 80-308, Gdansk
Unidade Local De Saude De Santa Maria E.P.E.
Pneumology, Avenida Professor Egas Moniz, 1649-035, Lisbon
Hospital De Jerez De La Frontera
Servicio de Pediatría, Carretera De La Ronda Circunvalacion S/n, 11407, Jerez De La Frontera
Hospital Universitario La Paz
Servicio Neumologia pediatrica, Paseo De La Castellana 261, 28046, Madrid
Hospital Universitario Ramon Y Cajal
Servicio de Pediatría, Carretera Del Colmenar Viejo Km 9 100, Por El Pardo, Madrid
University Clinical Hospital Virgen De La Arrixaca
Servicio de Pediatría, Carretera Madrid-Cartagena S/N, El Palmar, Murcia
Hospital Universitari Vall D Hebron
Servicio Neumología, Passeig De La Vall D'Hebron 119-129, 08035, Barcelona
Hospital Universitario De Cruces
Neumologia Pediatrica, Cruces Plaza S/n, 48903, Barakaldo
Hospital Infantil Universitario Nino Jesus
Servicio Neumologia pediatrica, Avenida Menendez Pelayo 65, 28009, Madrid
Hospital Universitario 12 De Octubre
Servicio de Pediatría, Avenida De Cordoba Sn, 28041, Madrid
Hospital Universitario Y Politecnico La Fe
Unidad de Trasplante Pulmonar y Fibrosis Quística, Avenida Fernando Abril Martorell 106, 46026, Valencia
Parc Tauli Hospital Universitari
Servicio Medicina Pediatrica, Parc Del Tauli 1, 08208, Sabadell
Hospital Sant Joan De Deu Barcelona
Servicio Neumología, Passeig De Sant Joan De Deu 2, 08950, Esplugues De Llobregat
University Hospital Virgen Del Rocio S.L.
Unidad de Gestion Clínica de Neumología y Cirugía de Tórax. Unidad de Fibrosis Quistica., Avenida De Manuel Siurot S/n, 41013, Sevilla
Country notifications
Trial-start, recruitment-start, end and early-termination notifications submitted per Member State
| Country | Trial start | Trial end | Recruitment start | Recruitment end | Early termination |
|---|---|---|---|---|---|
| Austria | 2023-03-22 | 2023-04-11 | 2023-04-11 | ||
| Belgium | 2022-12-09 | 2023-01-03 | 2023-06-12 | ||
| Czechia | 2023-04-14 | 2023-04-24 | 2023-06-22 | ||
| France | 2022-11-08 | 2022-11-23 | 2023-07-03 | ||
| Germany | 2022-12-08 | 2022-12-16 | 2023-07-04 | ||
| Hungary | 2023-06-20 | 2023-06-27 | 2023-06-27 | ||
| Italy | 2023-02-02 | 2023-02-14 | 2023-07-05 | ||
| Netherlands | 2023-01-09 | 2023-01-12 | 2023-05-10 | ||
| Norway | 2023-06-05 | 2023-06-20 | 2023-06-27 | ||
| Poland | 2023-06-01 | 2023-06-14 | 2023-06-27 | ||
| Portugal | 2023-03-31 | 2023-04-10 | 2023-06-19 | ||
| Spain | 2022-12-01 | 2022-12-07 | 2023-06-28 | ||
| Sweden | 2023-04-28 | 2023-05-12 | 2023-05-24 |
Results and documents
Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial
Documents 243 files
Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.
| Type | Title | Version |
|---|---|---|
| Protocol (for publication) | D1_Protocol ENG - 2024-515637-14-00_Redacted | 2.3 |
| Protocol (for publication) | D4_Patient facing documents_CFQ-R_Placeholder | 1 |
| Recruitment arrangements (for publication) | K1_Recruitment Arrangements_ES_Placeholder Statement | 1.0 |
| Recruitment arrangements (for publication) | K1_Recruitment Arrangements_FR_Placeholder Statement | 1 |
| Recruitment arrangements (for publication) | K1_Recruitment Arrangements_Placeholder Statement | 1 |
| Recruitment arrangements (for publication) | K1_Recruitment Arrangements_Placeholder Statement_BE | 1.0 |
| Recruitment arrangements (for publication) | K1_Recruitment Arrangements_Placeholder Statement_CZ | 1.0 |
| Recruitment arrangements (for publication) | K1_Recruitment Arrangements_Placeholder Statement_DE | 1.0 |
| Recruitment arrangements (for publication) | K1_Recruitment Arrangements_Placeholder Statement_HU | 1 |
| Recruitment arrangements (for publication) | K1_Recruitment Arrangements_Placeholder Statement_PL | 1 |
| Recruitment arrangements (for publication) | K1_Recruitment Arrangements_Placeholder Statement_PT | 1 |
| Recruitment arrangements (for publication) | K1_Recruitment Arrangements_Placeholder Statement_SE | 1.0 |
| Recruitment arrangements (for publication) | K1_Recruitment Arrangements_Statement_AT_en | 1.0 |
| Recruitment arrangements (for publication) | K1_Recruitment Arrangements_Statement_NL_en | 1.0 |
| Recruitment arrangements (for publication) | K1_Recruitment Arrangements_Statement_NO_en | 1.0 |
| Subject information and informed consent form (for publication) | L1_ICF_Adult Extenuating Circumstances Addendum Part A Part B_HU_hu | 1 |
| Subject information and informed consent form (for publication) | L1_ICF_Adult Genetic_HU_hu | 2 |
| Subject information and informed consent form (for publication) | L1_ICF_Adult Part A_HU_hu | 1 |
| Subject information and informed consent form (for publication) | L1_ICF_Adult Part B_HU_hu | 1 |
| Subject information and informed consent form (for publication) | L1_ICF_Pregnancy_HU_hu | 2 |
| Subject information and informed consent form (for publication) | L1_List of submitted patient materials_HU_hu | 1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Adolescent Extenuating Circumstances Part A and B_AT_de | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Adolescent Extenuating Circumstances part A and B_BE_EN | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Adolescent Extenuating Circumstances part A and B_BE_FR | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Adolescent Extenuating Circumstances part A and B_BE_NL | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Adolescent Extenuating Circumstances Part A and B_ES_es | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Adolescent Extenuating Circumstances_NL_nl | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Adolescent Extenuating Circumstances_NO_no | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Adolescent Extenuating Circumstances_Part A and B_PL_pl | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Adolescent Extenuating Circumstances_Part A and B_PT_pt | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Adolescent Part A_12-13yrs_AT_de | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Adolescent Part A_14-17yrs_AT_de | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Adolescent Part A_14-17yrs_AT_de_redacted | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Adolescent Part A_BE_EN_Redacted | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Adolescent Part A_BE_FR_Redacted | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Adolescent Part A_BE_NL_Redacted | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Adolescent Part A_ES_es | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Adolescent Part A_NL_nl_redacted | 2.1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Adolescent Part A_NO_no_redacted | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Adolescent Part B_12-13yrs_AT_de | 2.1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Adolescent Part B_14-17yrs_AT_de | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Adolescent Part B_14-17yrs_AT_de_redacted | 2.1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Adolescent Part B_BE_EN_Redacted | 2.1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Adolescent Part B_BE_FR_Redacted | 2.1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Adolescent Part B_BE_NL_Redacted | 2.1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Adolescent Part B_ES_es | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Adolescent Part B_NL_nl_redacted | 2.1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Adolescent Part B_NO_no_redacted | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Adolescent_Part A_DE_de | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Adolescent_Part A_PL_pl | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Adolescent_Part A_PT_pt | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Adolescent_Part B_DE_de | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Adolescent_Part B_PL_pl | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Adolescent_Part B_PT_pt | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Adult -Parent Part A_ES_es_Redacted | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Adult -Parent Part B_ES_es_Redacted | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Adult Extenuating Circumstances Part A and B_AT_de | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Adult Parent Extenuating Circumstances Part A and B_BE_EN | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Adult Parent Extenuating Circumstances Part A and B_BE_FR | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Adult Parent Extenuating Circumstances Part A and B_BE_NL | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Adult Parent Extenuating Circumstances_NL_nl | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Adult Parent Extenuating Circumstances_NO_no | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Adult Parent Extenuating Circumstances_Part A and B_PL_pl | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Adult Parent Extenuating Circumstances_Part A and B_PT_pt | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Adult Parent_Part A_PL_pl_redacted | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Adult Parent_Part A_PT_pt | 2.1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Adult Parent_Part B_PL_pl_redacted | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Adult Parent_Part B_PT_pt | 2.1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Adult Part A_AT_de | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Adult Part A_AT_de_redacted | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Adult Part A_NL_nl_redacted | 2.1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Adult Part A_NL_redacted_Initial version | 1.1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Adult Part A_NO_no_redacted | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Adult Part B_AT_de | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Adult Part B_AT_de_redacted | 2.1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Adult Part B_NL_nl_redacted | 2.1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Adult Part B_NL_nl_redacted_Initial version | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Adult Part B_NO_no_redacted | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Adult_Part A_DE_de_redacted | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Adult_Part B_DE_de_redacted | 2.1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Adult-Parent Extenuating Circumstances Part A and B_ES_es | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Adult-Parent Part A_BE_EN_Redacted | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Adult-Parent Part A_BE_FR_Redacted | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Adult-Parent Part A_BE_NL_Redacted | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Adult-Parent Part B_BE_EN_Redacted | 2.1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Adult-Parent Part B_BE_FR_Redacted | 2.1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Adult-Parent Part B_BE_NL_Redacted | 2.1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Child Assent Part A_NO_no_redacted | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Child Assent Part B_NO_no_redacted | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Child Assent Part B_SE_se | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Child Extenuating Circumstances Part A and B_BE_EN | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Child Extenuating Circumstances Part A and B_BE_FR | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Child Extenuating Circumstances Part A and B_BE_NL | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Child Extenuating Circumstances_NO_no | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Child Extenuating Circumstances_Part A and B_PL_pl | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Child Part A_BE_EN | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Child Part A_BE_FR | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Child Part A_BE_NL | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Child Part A_NL_nl | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Child Part B_BE_EN | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Child Part B_BE_FR | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Child Part B_BE_NL | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Child Part B_NL_nl | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Child_Part A_DE_de | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Child_Part A_PL_pl | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Child_Part A_PT_pt | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Child_Part B_DE_de | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Child_Part B_PL_pl | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Child_Part B_PT_pt | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Data Privacy Annex_ES_es | 1.1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Greenphire Consent_BE_EN | 1.1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Greenphire Consent_BE_FR | 1.1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Greenphire Consent_BE_NL | 1.1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Greenphire ICF_DE_de | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Greenphire_AT_de | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Greenphire_NL_nl | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Greenphire_PL_pl | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Greenphire_PT_pt | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Main Adult Part B_FR_Fr_tracked changes | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Parent Extenuating Circumstances Part A and B_AT_de | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Parent Part A_AT_de_redacted | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Parent Part A_AT_de_redacted_initial | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Parent Part A_NL_nl_redacted | 2.1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Parent Part A_NO_no_redacted | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Parent Part B_AT_de_redacted | 2.1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Parent Part B_NL_nl_redacted | 2.1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Parent Part B_NO_no_redacted | 2.1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Parent_Part A_DE_de_redacted | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Parent_Part B_DE_de_redacted | 2.1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Pregnancy_AT_de | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Pregnancy_BE_EN_Redacted | 1.2 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Pregnancy_BE_FR_Redacted | 1.2 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Pregnancy_BE_NL_Redacted | 1.2 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Pregnancy_DE_de | 1.2 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Pregnancy_ES_es | 1.1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Pregnancy_NL_nl_redacted | 1.2 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Pregnancy_NO_no_redacted | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Pregnancy_PL_pl | 1.2 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Pregnancy_PT_pt | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Adolescent 12-14 yrs_Part A_CZ_cz | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Adolescent 12-14 yrs_Part B_CZ_cz | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Adolescent 15-17 yrs_Part A_CZ_cz | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Adolescent 15-17 yrs_Part B_CZ_cz | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Adolescent Assent Part A 12-14 yr _SE_sv | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Adolescent Assent Part A 15-17 yr_SE_sv | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Adolescent Assent Part B 12-14 yr_SE_sv | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Adolescent Assent Part B 15-17 yr_SE_sv | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Adolescent Extenuating Circumstances Part A and B_IT_it | 1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Adolescent Extenuating Circumstances Parts A and B_Fr_fr | 1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Adolescent Extenuating Circumstances_Part A and B_CZ_cz | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Adolescent Part A_Fr-fr | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Adolescent Part A_IT_it | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Adolescent Part B_Fr_fr | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Adolescent Part B_IT_it | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Adult Extenuating Circumstances Part A and B_IT_it | 1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Adult Extenuating Circumstances Parts A and B_Fr_fr | 1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Adult Extenuating Circumstances_Part A and B_CZ_cz | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Adult Part A_CZ_cz | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Adult Part A_HU_hu_redacted | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Adult Part A_IT_it_redacted | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Adult Part B_HU_hu_redacted | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Adult Part B_IT_it_redacted | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Adult Privacy_IT_it | 2.2 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Adult_Part B_CZ_cz | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Child Assent Part A_IT_it | 1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Child Assent Part A_SE_se | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Child Assent Part B _IT_it | 1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Child Extenuating Circumstances Part A and B_IT_it | 1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Child Extenuating Circumstances Parts A and B_Fr_fr | 1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Child Part A_Fr_fr | 1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Child Part B_Fr_fr | 1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Extenuating Circumstances Adult Parent Part A and B_SE_se | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Extenuating Circumstances Part A and B 12-14 yr _SE_se | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Extenuating Circumstances Part A and B 15-17yr_SE_se | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_GDPR Adult_CZ_cz | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_GDPR Parent_CZ_cz | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_GDPR Pregnant Partner_CZ_cz | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Greenphire ICF_IT_it | 1.1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Greenphire_CZ_cz | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Greenphire_ES_es | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Greenphire_HU_hu | 1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Greenphire_SE_se | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Main Adult Part A_Fr_fr_redacted | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Main Adult Part A_SE_sv | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Main Adult Part B_Fr_fr_redacted | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Main Adult Part B_SE_sv | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Main Parent Part A_Fr_fr_redacted | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Main Parent Part A_SE_sv | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Main Parent Part B_Fr_fr_redacted | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Main Parent Part B_SE_sv | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Parent Extenuating Circumstances Part A and B_Fr_fr | 1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Parent Extenuating Circumstances Part A and B_IT_it | 1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Parent Extenuating Circumstances_Part A and B_CZ_cz | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Parent Part A_CZ_cz | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Parent Part A_IT_it_redacted | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Parent Part B_CZ_cz | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Parent Part B_IT_it_redacted | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Parent Privacy_IT_it | 2.2 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Pregnancy ICF_Fr_fr | 1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Pregnancy Privacy_IT_it | 2.2 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Pregnancy_CZ_cz | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Pregnancy_IT_it | 1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Pregnancy_SE_se | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS_Adult Extenuating Circumstances Addendum Part A Part B_HU_hu | 1 |
| Subject information and informed consent form (for publication) | L1_SIS_Adult Genetic_HU_hu | 2 |
| Subject information and informed consent form (for publication) | L1_SIS_Pregnancy_HU_hu | 2 |
| Subject information and informed consent form (for publication) | L2_ Other subject information material_List of sites data protection officers and IRBs_Austria | 3.1 |
| Subject information and informed consent form (for publication) | L2_GP Letter_IT_it | 1 |
| Subject information and informed consent form (for publication) | L2_Other subject information material_Patient Alert Card_HU_hu | 1 |
| Summary of Product Characteristics (SmPC) (for publication) | G2_ SmPC Kaftrio | N/A |
| Summary of Product Characteristics (SmPC) (for publication) | G2_ SmPC Kalydeco | N/A |
| Summary of Product Characteristics (SmPC) (for publication) | G2_SmPC Kaftrio_Placeholder | N/A |
| Summary of Product Characteristics (SmPC) (for publication) | G2_SmPC Kalydeco_Placeholder | N/A |
| Synopsis of the protocol (for publication) | D1_Protocol lay synopsis CZ 2024-515637-14-00 | 2.3 |
| Synopsis of the protocol (for publication) | D1_Protocol lay synopsis de-AT 2024-515637-14-00 | 2.3 |
| Synopsis of the protocol (for publication) | D1_Protocol lay synopsis de-BE - 2024-515637-14-00 | 2.3 |
| Synopsis of the protocol (for publication) | D1_Protocol lay synopsis ENG - 2024-515637-14-00 | 2.3 |
| Synopsis of the protocol (for publication) | D1_Protocol lay synopsis ES 2024-515637-14-00 | 2.3 |
| Synopsis of the protocol (for publication) | D1_Protocol lay synopsis FR 2024-515637-14-00 | 2.3 |
| Synopsis of the protocol (for publication) | D1_Protocol lay synopsis fr-BE 2024-515637-14-00 | 2.3 |
| Synopsis of the protocol (for publication) | D1_Protocol lay synopsis HU 2024-515637-14-00 | 2.3 |
| Synopsis of the protocol (for publication) | D1_Protocol lay synopsis IT 2024-515637-14-00 | 2.3 |
| Synopsis of the protocol (for publication) | D1_Protocol lay synopsis NL 2024-515637-14-00 | 2.3 |
| Synopsis of the protocol (for publication) | D1_Protocol lay synopsis nl-BE 2024-515637-14-00 | 2.3 |
| Synopsis of the protocol (for publication) | D1_Protocol lay synopsis NO 2024-515637-14-00 | 2.3 |
| Synopsis of the protocol (for publication) | D1_Protocol lay synopsis PL 2024-515637-14-00 | 2.3 |
| Synopsis of the protocol (for publication) | D1_Protocol lay synopsis PT 2024-515637-14-00 | 2.3 |
| Synopsis of the protocol (for publication) | D1_Protocol lay synopsis SE 2024-515637-14-00 | 2.3 |
| Synopsis of the protocol (for publication) | D1_Protocol synopsis_ENG - 2024-515637-14-00 | 2.3 |
| Synopsis of the protocol (for publication) | D1_Protocol_synopsis CZ - 2024-515637-14-00 | 2.3 |
| Synopsis of the protocol (for publication) | D1_Protocol_synopsis de-AT - 2024-515637-14-00 | 2.3 |
| Synopsis of the protocol (for publication) | D1_Protocol_synopsis de-BE - 2024-515637-14-00 | 2.3 |
| Synopsis of the protocol (for publication) | D1_Protocol_synopsis ES - 2024-515637-14-00 | 2.3 |
| Synopsis of the protocol (for publication) | D1_Protocol_synopsis fr-BE - 2024-515637-14-00 | 2.3 |
| Synopsis of the protocol (for publication) | D1_Protocol_synopsis fr-FR - 2024-515637-14-00 | 2.3 |
| Synopsis of the protocol (for publication) | D1_Protocol_synopsis HU - 2024-515637-14-00 | 2.3 |
| Synopsis of the protocol (for publication) | D1_Protocol_synopsis IT - 2024-515637-14-00 | 2.3 |
| Synopsis of the protocol (for publication) | D1_Protocol_synopsis nl-BE - 2024-515637-14-00 | 2.3 |
| Synopsis of the protocol (for publication) | D1_Protocol_synopsis nl-NL- 2024-515637-14-00 | 2.3 |
| Synopsis of the protocol (for publication) | D1_Protocol_synopsis NO - 2024-515637-14-00 | 2.3 |
| Synopsis of the protocol (for publication) | D1_Protocol_synopsis PL - 2024-515637-14-00 | 2.3 |
| Synopsis of the protocol (for publication) | D1_Protocol_synopsis PT - 2024-515637-14-00 | 2.3 |
| Synopsis of the protocol (for publication) | D1_Protocol_synopsis SE - 2024-515637-14-00 | 2.3 |
Application history
7 submissions — initial application plus substantial / non-substantial modifications
| # | Type | Code | Submitted | Reference MS | Conclusion | Decision date |
|---|---|---|---|---|---|---|
| 1 | INITIAL | IN | 2024-09-19 | Germany | Acceptable 2024-10-17
|
2024-10-17 |
| 2 | SUBSTANTIAL MODIFICATION | SM-1 | 2024-11-26 | Germany | Acceptable 2025-03-12
|
2025-03-12 |
| 3 | SUBSTANTIAL MODIFICATION | SM-2 | 2025-04-09 | Germany | Acceptable 2025-06-10
|
2025-06-11 |
| 4 | SUBSTANTIAL MODIFICATION | SM-3 | 2025-07-22 | Germany | Acceptable 2025-10-13
|
2025-10-13 |
| 5 | SUBSTANTIAL MODIFICATION | SM-4 | 2025-10-22 | Acceptable | 2025-11-14 | |
| 6 | NON SUBSTANTIAL MODIFICATION | NSM-1 | 2026-03-16 | Acceptable | 2026-03-16 | |
| 7 | SUBSTANTIAL MODIFICATION | SM-5 | 2026-04-29 | Acceptable | 2026-05-13 |