E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Patients with Idiopathic Pulmonary Fibrosis |
Pacientes con Fibrosis Pulmonar Idiopática |
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E.1.1.1 | Medical condition in easily understood language |
Progressive, fatal lung disease with an unknown cause |
Enfermedad pulmonar progresiva, mortal con causa desconocida |
|
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 | 14.0 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10021240 |
E.1.2 | Term | Idiopathic pulmonary fibrosis |
E.1.2 | System Organ Class | 10038738 - Respiratory, thoracic and mediastinal disorders |
|
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 14.0 |
E.1.2 | Level | SOC |
E.1.2 | Classification code | 10038738 |
E.1.2 | Term | Respiratory, thoracic and mediastinal disorders |
E.1.2 | System Organ Class | 10038738 - Respiratory, thoracic and mediastinal 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 assess the long-term safety and tolerability of macitentan in patients with Idiopathic Pulmonary Fibrosis |
Evaluar la seguridad y tolerabilidad a largo plazo de macitentan en pacientes con fibrosis pulmonar idiopática. |
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E.2.2 | Secondary objectives of the trial |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
- Signed informed consent prior to initiation of any study-related procedure. - Patients with IPF and having completed the double-blind AC- 055B201/MUSIC study as scheduled, i.e., having remained in the study until the sponsor-declared end-of study, whether or not study treatment was prematurely discontinued. - Women of childbearing potential must have a negative pre-treatment serum pregnancy test and must use a reliable method of contraception during study treatment and for at least 28 days after study treatment termination. |
- Consentimiento informado firmado antes de cualquier procedimiento requerido por el estudio. - Pacientes con IPF y que hayan completado el estudio doble ciego AC-055B201/MUSIC según estaba programado, es decir, que hayan permanecido en el estudio hasta que el promotor declaró el final del estudio (EOS), independientemente de si el tratamiento del estudio fue discontinuado prematuramente. - Las mujeres con potencial reproductivo deben tener una prueba de embarazo negativa anterior al tratamiento y deben utilizar un método anticonceptivo fiable durante el tratamiento del estudio y durante al menos 28 días después de la finalización del tratamiento del estudio. |
|
E.4 | Principal exclusion criteria |
- Any major violation of protocol AC-055B201/MUSIC. - Premature discontinuation of study treatment during the AC- 055B201/MUSIC study due to an adverse event (AE) assessed as related to the use of macitentan, excluding events of IPF worsening. - Pregnancy or breast-feeding. - AST and/or ALT > 3 times the upper limit of the normal range. - Any known factor or disease that might interfere with treatment compliance, study conduct or interpretation of the results, such as drug or alcohol dependence or psychiatric disease. - Known hypersensitivity to drugs of the same class as macitentan, or any of the excipients. |
- Cualquier violación grave del protocolo AC-055B201/MUSIC. - Discontinuación prematura del tratamiento del estudio durante el estudio AC 055B201/MUSIC debido a un acontecimiento adverso (AE) evaluado como relacionado al uso de macitentan, excluyendo acontecimientos de empeoramiento de la IPF. - Embarazo o lactancia. - AST y/o ALT >3 veces el límite superior de los rangos normales. - Cualquier factor o enfermedad conocida que pudiese interferir con el cumplimiento del tratamiento, realización del estudio o interpretación de los resultados, como la dependencia a alcohol o drogas o enfermedad psiquiátrica. - Hipersensibilidad conocida a fármacos de la misma clase que macitentan, o a alguno de sus excipientes. |
|
E.5 End points |
E.5.1 | Primary end point(s) |
- AEs leading to premature discontinuation of study drug. - Treatment-emergent SAEs up to 28 days after study drug discontinuation. - Occurrence of liver aminotransferase (ALT and/or AST) abnormality (> 3 and < or = 5 X ULN; > 5 and < or = 8 X ULN; > 8 X ULN) up to 28 days after study drug discontinuation. |
- AEs que conduzcan a la interrupción prematura del fármaco del estudio. -SAEs tratamiento emergentes hasta 28 días después de la discontinuación del fármaco del estudio. - Aparición de alteraciones en las aminotransferasas hepáticas (ALT y/o AST)(> 3 y < o = 5 X ULN; > 5 y < o = 8 X ULN; > 8 X ULN) hasta los 28 días posteriores a la interrupción del fármaco del estudio. |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
- 6-monthly safety visit - Liver aminotransferase and pregnancy (if applicable): monthly - Follow-up period 28 days after treatment discontinuation |
- visitas de seguridad cada 6 meses - aminotransferasa hepática y embarazo (si aplica): mensual - periodo de seguimiento a los 28 días después de la interrupción del tratamiento |
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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 | Yes |
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 | Yes |
E.6.13.1 | Other scope of the trial description |
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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) | Yes |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | No |
E.8.1.1 | Randomised | Information not present in EudraCT |
E.8.1.2 | Open | Information not present in EudraCT |
E.8.1.3 | Single blind | Information not present in EudraCT |
E.8.1.4 | Double blind | Information not present in EudraCT |
E.8.1.5 | Parallel group | Information not present in EudraCT |
E.8.1.6 | Cross over | Information not present in EudraCT |
E.8.1.7 | Other | Information not present in EudraCT |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | Information not present in EudraCT |
E.8.2.2 | Placebo | Information not present in EudraCT |
E.8.2.3 | Other | Information not present in EudraCT |
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 | 4 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 17 |
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 |
Australia |
Canada |
Israel |
South Africa |
Turkey |
United States |
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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
|
Last patient last visit |
Ultima visita del último paciente |
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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 | 5 |
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 | 5 |
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 | 0 |