E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Hipertensión arterial pulmonar (PAH) en niños. |
|
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 12.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10064908 |
E.1.2 | Term | Associated with (APAH) |
|
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 12.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10064909 |
E.1.2 | Term | Idiopathic (IPAH) |
|
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 12.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10064910 |
E.1.2 | Term | Familial (FPAH) |
|
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 |
Evaluar la seguridad a largo plazo, tolerabilidad y eficacia de la formulación pediátrica de bosentan dos veces frente a tres veces al día en niños con hipertensión arterial pulmonar (PAH) |
|
E.2.2 | Secondary objectives of the trial |
No hay objetivo secundario. |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. Pacientes que completaron el estudio principal FUTURE 3 (AC-052-373) o discontinuaron prematuramente debido a la progresión de la PAH, si bosentan no fue permanentemente discontinuado. 2. Pacientes que toleraron la presentación pediátrica de bosentan y para los que bosentan se considera beneficioso por parte del investigador al final del estudio principal FUTURE 3 (AC-052-373). 3. Consentimiento informado firmado por los padres o representantes legales antes de cualquier procedimiento requerido por el estudio. |
|
E.4 | Principal exclusion criteria |
1. Intolerancia o hipersensibilidad conocida a bosentan o a cualquiera de los excipientes del comprimido dispersable de Tracleer 2. Cualquier anomalía clínicamente significativa de laboratorio que impida la continuación de la terapia con bosentan 3. Embarazo 4. Valores de la AST y/o ALT > 3 veces el límite superior del valor normal (ULN) 5. Lesión hepática moderada a grave, es decir de la clase Child-Pugh, B ó C 6. Discontinuación prematura y permanente del fármaco del estudio durante el estudio principal FUTURE 3 (AC-052-373) 7. Cualquier violación grave del protocolo del estudio principal FUTURE 3 (AC 052 373). |
|
E.5 End points |
E.5.1 | Primary end point(s) |
No se ha definido el objetivo principal para este estudio de extensión exploratorio abierto. |
|
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 | 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 | Yes |
E.6.13.1 | Other scope of the trial description |
|
E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | No |
E.7.1.1 | First administration to humans | Information not present in EudraCT |
E.7.1.2 | Bioequivalence study | Information not present in EudraCT |
E.7.1.3 | Other | Information not present in EudraCT |
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 | 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 | 2 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 20 |
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 |
|
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
|
Proporcionado en el protocolo |
|
E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 3 |
E.8.9.1 | In the Member State concerned months | 8 |
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 | 8 |
E.8.9.2 | In all countries concerned by the trial days | 0 |