E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Cystic Fibrosis |
Fibrosis quística |
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E.1.1.1 | Medical condition in easily understood language |
Cystic Fibrosis |
Fibrosis quística |
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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 safety and tolerability of Elexacaftor (ELX)/Tezacaftor (TEZ)/Ivacaftor (IVA) in subjects with Cystic Fibrosis (CF). |
Evaluar la seguridad y la tolerabilidad de elexacaftor (ELX)/tezacaftor (TEZ)/ivacaftor (IVA) en pacientes con fibrosis quística (FQ) |
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E.2.2 | Secondary objectives of the trial |
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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. For subjects being considered for resumption of participation in this study after enrolling in another Vertex study of investigational CFTR modulators (referred to as “another qualified Vertex study”): Completed the ETT visit in another qualified Vertex study before or on the same day as the Returning Visit in this study. If more than 30 days have elapsed since the ETT visit in the other qualified Vertex study, approval of the medical monitor is required. 6. Willing to remain on a stable CF treatment regimen (as defined in Section 9.5 of the protocol) through completion of study participation. |
1. El sujeto (o su representante legalmente designado y autorizado) firmará y fechará un formulario de consentimiento informado (FCI) y, cuando proceda, un formulario de asentimiento. 2. Está dispuesto y es capaz de cumplir con las visitas programadas, el plan de tratamiento, las restricciones del estudio, las pruebas analíticas, las pautas anticonceptivas y otros procedimientos del estudio. 3. No retiró el consentimiento de un estudio matriz. 4. Cumple al menos 1 de los siguientes criterios: • Haber completado el tratamiento con el fármaco del estudio en un estudio matriz. • Tuvo una o varias interrupciones del fármaco del estudio en un estudio matriz, pero no fue una interrupción permanente del fármaco del estudio, y completó las visitas del estudio hasta la última visita programada del periodo de tratamiento en el estudio matriz. 5. Para sujetos que se están considerando para reanudar su participación en este estudio después de inscribirse en otro estudio de Vertex de moduladores de CFTR en investigación (denominados “otro estudio de Vertex cualificado”): Completaron la visita de FAT en otro estudio de Vertex cualificado antes o el mismo día que la visita de regreso en este estudio. Si han transcurrido más de 30 días desde la visita de FAT en el otro estudio cualificado de Vertex, se requiere la aprobación del supervisor médico. 6. Dispuesto a seguir con una pauta de tratamiento estable para la FQ (según se define en la Sección 9.5) hasta la finalización de la participación en el estudio. |
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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. 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). 3. Pregnant and breast-feeding females. Female subjects must have a negative pregnancy test at the Day 1 Visit before receiving the first dose of 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. 5. For subjects being considered for resumption of participation in this study after enrolling in another qualified Vertex study, the following exclusion criteria also apply: • Subject received the first dose of study drug in the Treatment Period of another qualified Vertex study, • Subject has access to commercially available ELX/TEZ/IVA or is receiving managed access program supplied ELX/TEZ/IVA, or • Subject departed this study more than once to participate in another qualified Vertex study. |
1. Antecedentes de comorbilidad que, en opinión del investigador, pueden confundir los resultados del estudio o suponer un riesgo adicional en la administración del fármaco del estudio al sujeto. 2. Antecedentes de intolerancia al fármaco en un estudio original que suponga un riesgo adicional para el sujeto en opinión del investigador (p. ej., sujetos con antecedentes de alergia o hipersensibilidad al fármaco del estudio). 3. Mujeres embarazadas y en periodo de lactancia. Los sujetos de sexo femenino deben tener una prueba de embarazo negativa en la visita del día 1 antes de recibir la primera dosis del fármaco del estudio. 4. Participación actual en un ensayo con un fármaco en investigación distinto de un estudio matriz. Se permite la participación en un estudio no intervencionista (incluidos estudios observacionales, estudios de registro y estudios que requieran extracciones de sangre sin administración del fármaco del estudio) y la selección para otro estudio de Vertex. 5. Para los sujetos a los que se esté considerando para reanudar su participación en este estudio después de inscribirse en otro estudio de Vertex cualificado, también se aplicarán los siguientes criterios de exclusión: • El sujeto recibió la primera dosis del fármaco del estudio en el periodo de tratamiento de otro estudio de Vertex cualificado, • El sujeto tiene acceso a ELX/TEZ/IVA disponible comercialmente o está recibiendo ELX/TEZ/IVA suministrado por el programa de acceso gestionado, o bien • El sujeto abandonó este estudio más de una vez para participar en otro estudio de Vertex cualificado. |
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E.5 End points |
E.5.1 | Primary end point(s) |
Safety and tolerability of ELX/TEZ/IVA based on adverse events (AEs), clinical laboratory values, ECGs, vital signs, and pulse oximetry. |
La seguridad y la tolerabilidad de ELX/TEZ/IVA en función de acontecimientos adversos (AA), valores analíticos clínicos, electrocardiogramas (ECG), constantes vitales y pulsioximetría. |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
From signing of ICF until safety Follow up visit. |
Desde la firma de la HIP hasta la visita de seguimiento de seguridad |
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E.5.2 | Secondary end point(s) |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
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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 | No |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | No |
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 | Yes |
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 | No |
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.8.3 |
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 | 8 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 18 |
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 | No |
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 |
Belgium |
Netherlands |
Spain |
Czechia |
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E.8.7 | Trial has a data monitoring committee | No |
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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last scheduled visit (or scheduled contact) of the last subject |
Última visita programada (o contacto programado) del último sujeto |
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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 | 0 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 1 |
E.8.9.2 | In all countries concerned by the trial months | 10 |
E.8.9.2 | In all countries concerned by the trial days | 6 |