E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Cystic Fibrosis |
fibrosi cistica |
|
E.1.1.1 | Medical condition in easily understood language |
Cystic Fibrosis |
fibrosi cistica |
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E.1.1.2 | Therapeutic area | Diseases [C] - Respiratory Tract Diseases [C08] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 20.0 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10011762 |
E.1.2 | Term | Cystic fibrosis |
E.1.2 | System Organ Class | 10010331 - Congenital, familial and genetic disorders |
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E.1.3 | Condition being studied is a rare disease | Yes |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
To evaluate the long-term safety and tolerability of VX121/tezacaftor/deutivacaftor (VX-121/TEZ/D-IVA) in subjects with cystic fibrosis (CF) |
Valutare la sicurezza e la tollerabilità a lungo termine di VX-121/tezacaftor/deutivacaftor (VX-121/TEZ/D-IVA) in soggetti con fibrosi cistica (FC) |
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E.2.2 | Secondary objectives of the trial |
To evaluate the long-term efficacy of VX-121/TEZ/D-IVA |
Valutare l’efficacia a lungo termine di VX-121/TEZ/D-IVA |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1.Subject (or his or her legally appointed and authorized representative) will sign and date an informed consent form (ICF), and, when appropriate, an assent form. 2.Willing and able to comply with scheduled visits, treatment plan, study restrictions, laboratory tests, contraceptive guidelines, and other study procedures. 3.Did not withdraw consent from a parent study. 4.Meets at least 1 of the following criteria: •Completed study drug treatment in a parent study. •Had 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. 5.Willing to remain on a stable CF treatment regimen through completion of study participation. |
1.Il soggetto (o il suo rappresentante legalmente nominato e autorizzato) firmerà e daterà un modulo di consenso informato (ICF) e, quando appropriato, un modulo di assenso. 2.disposto e in grado di rispettare le visite programmate, il piano di trattamento, le restrizioni dello studio, gli esami di laboratorio, le linee guida per la contraccezione e le altre procedure dello studio. 3.Non ha ritirato il proprio consenso da uno studio iniziale. 4.Soddisfa almeno uno dei seguenti criteri: -Ha completato il trattamento con il farmaco in studio in uno studio iniziale. -Ha subito interruzione(i) del trattamento con il farmaco in studio in uno studio iniziale, ma non ha interrotto definitivamente il farmaco in studio e ha completato le visite dello studio fino all'ultima visita programmata del periodo di trattamento di uno studio iniziale. 5.Disponibilità a mantenere un regime terapeutico stabile per la FC fino al completamento della partecipazione allo studio. |
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E.4 | Principal exclusion criteria |
1.History of any comorbidity that, in the opinion of the investigator, might confound the results of the study or pose an additional risk in administering study drug to the subject. 2.Pregnant or breast-feeding females. All female subjects must have a negative pregnancy test at the Day 1 Visit before receiving the first dose of study drug. 3.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.) 4.Current participation in an investigational drug trial (other than a 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. |
1.Anamnesi di qualsiasi comorbidità che, a giudizio dello sperimentatore, potrebbe confondere i risultati dello studio o comportare un rischio aggiuntivo nella somministrazione del farmaco di studio al soggetto. 2.Donne in gravidanza o in allattamento. Tutti i soggetti di sesso femminile devono avere un test di gravidanza negativo alla visita del giorno 1 prima di ricevere la prima dose di farmaco in studio. 3.Storia di intolleranza al farmaco in uno studio iniziale che, a giudizio dello sperimentatore, comporterebbe un rischio aggiuntivo per il soggetto. (ad esempio, soggetti con una storia di allergia o ipersensibilità al farmaco in studio). 4.Partecipazione in corso a uno studio farmacologico in fase di sperimentazione (diverso da uno studio iniziale). È consentita la partecipazione a uno studio non interventistico (compresi gli studi osservazionali, gli studi di registro e gli studi che richiedono un prelievo di sangue senza somministrazione del farmaco in studio) e lo screening per un altro studio Vertex. |
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E.5 End points |
E.5.1 | Primary end point(s) |
Safety and tolerability of long-term treatment with VX-121/TEZ/D-IVA based on adverse events (AEs), clinical laboratory values, ECGs, vital signs, and pulse oximetry |
Sicurezza e tollerabilità del trattamento a lungo termine con VX-121/TEZ/D-IVA basate su eventi avversi (AE), valori clinici di laboratorio, ECG, segni vitali e pulsossimetria |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
Day 1 through 96 weeks |
Dal 1° giorno fino a 96 settimane |
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E.5.2 | Secondary end point(s) |
Absolute change from baseline in percent predicted forced expiratory volume in 1 second (ppFEV1) Absolute change from baseline in sweat chloride (SwCl) Number of pulmonary exacerbations (PEx) |
Variazione assoluta dal basale in percentuale del valore previsto di volume espiratorio forzato in 1 secondo (ppFEV1) Variazione assoluta dal basale del cloruro contenuto nel sudore (SwCl) Numero di riacutizzazioni polmonari (PEx) |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
Day 1 through 96 weeks |
Dal 1° giorno fino a 96 settimane |
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E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | Yes |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | No |
E.6.7 | Pharmacodynamic | No |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | No |
E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | No |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | No |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | No |
E.7.3 | Therapeutic confirmatory (Phase III) | Yes |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | No |
E.8.1.1 | Randomised | No |
E.8.1.2 | Open | No |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | No |
E.8.1.5 | Parallel group | No |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | Yes |
E.8.1.7.1 | Other trial design description |
|
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | No |
E.8.2.3 | Other | No |
E.8.2.4 | Number of treatment arms in the trial | 1 |
E.8.3 |
The trial involves single site in the Member State concerned
| No |
E.8.4 | The trial involves multiple sites in the Member State concerned | Yes |
E.8.4.1 | Number of sites anticipated in Member State concerned | 9 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 95 |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | Yes |
E.8.6.2 | Trial being conducted completely outside of the EEA | Information not present in EudraCT |
E.8.6.3 | If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned |
Australia |
Canada |
Israel |
New Zealand |
United States |
Austria |
France |
Poland |
Sweden |
Netherlands |
Spain |
Switzerland |
Czechia |
Germany |
Greece |
Italy |
Belgium |
Denmark |
Hungary |
Ireland |
Norway |
Portugal |
United Kingdom |
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E.8.7 | Trial has a data monitoring committee | Yes |
E.8.8 |
Definition of the end of the trial and justification where it is not the last
visit of the last subject undergoing the trial
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E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 1 |
E.8.9.1 | In the Member State concerned months | 11 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 3 |
E.8.9.2 | In all countries concerned by the trial months | 0 |
E.8.9.2 | In all countries concerned by the trial days | 17 |