E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Pulmonary Hypertension |
Hipertensión pulmonar |
|
E.1.1.1 | Medical condition in easily understood language |
Raised blood pressure in the arteries that supply the lungs |
Aumento de la presión arterial en las arterias que suministran los pulmones |
|
E.1.1.2 | Therapeutic area | Diseases [C] - Cardiovascular Diseases [C14] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 19.1 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10037400 |
E.1.2 | Term | Pulmonary hypertension |
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 provide continuing open-label treatment with bardoxolone methyl as part of this extended access program while collecting ongoing safety and tolerability data of bardoxolone methyl |
Ofrecer tratamiento abierto continuado con bardoxolona metilo como parte de este programa de acceso ampliado, además de recoger datos sobre la seguridad y tolerabilidad de bardoxolona metilo |
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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 |
Treatment-compliant patients who are participating in qualifying ongoing studies and have completed required End-of-Treatment and/or Follow-up visits in a prior clinical study with bardoxolone methyl |
Pacientes que cumplan el tratamiento, estén participando en estudios calificadores en curso y hayan completado las visitas necesarias de final de tratamiento o de seguimiento en un estudio clínico previo con bardoxolona metilo |
|
E.4 | Principal exclusion criteria |
1. Participation in other investigational clinical studies involving interventional products being tested or used in a way different from the approved form or when used for an unapproved indication 2. Patients who have an ongoing SAE from a clinical study that is assessed by the investigator as related to bardoxolone methyl 3. Unwilling to practice acceptable methods of birth control (both males who have partners of childbearing potential and females of childbearing potential) while taking study drug 4. Women who are pregnant or breastfeeding 5. Patient is, in the opinion of the investigator, unable to comply with the requirements of the study protocol or is unsuitable for the study for any reason 6. Known hypersensitivity to any component of the study drug |
1. Participación en otros estudios clínicos de investigación en donde se evalúen o utilicen productos de intervención de una manera diferente a la aprobada o para una indicación no autorizada. 2. Pacientes con un AAG en marcha en un estudio clínico que el investigador considere relacionado con bardoxolona metilo. 3. Negativa a utilizar métodos anticonceptivos aceptables (tanto varones con parejas en edad fértil como mujeres en edad fértil) durante el tratamiento con el fármaco del estudio. 4. Mujeres embarazadas o lactantes. 5. Pacientes que, en opinión del investigador, no puedan cumplir los requisitos del protocolo del estudio o no sean idóneos para participar en el estudio por alguna otra razón. 6. Hipersensibilidad conocida a alguno de los componentes del fármaco del estudio. |
|
E.5 End points |
E.5.1 | Primary end point(s) |
Frequency, intensity, and relationship to study drug of adverse events (AEs) and serious adverse events (SAEs), and change from baseline in the following assessments: physical examinations, vital sign measurements, BNP, N-terminal pro-brain natriuretic peptide (NT-proBNP), and weight |
Frecuencia, intensidad y relación con el fármaco del estudio de acontecimientos adversos (AA) y acontecimientos adversos graves (AAG) y cambios con respecto al momento basal en las evaluaciones siguientes: exploraciones físicas, mediciones de las constantes vitales, péptido natriurético de tipo B (BNP), propéptido natriurético cerebral N-terminal (NT-proBNP) y peso |
|
E.5.1.1 | Timepoint(s) of evaluation of this end point |
When bardoxolone methyl becomes commercially available or when the patient withdraws from the study. |
Cuando bardoxolona metilo se comercialice o cuando el paciente se retire del estudio |
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E.5.2 | Secondary end point(s) |
|
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 | 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.8.2.4 | 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 | 5 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 21 |
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 |
Argentina |
Australia |
Belgium |
Brazil |
Canada |
Chile |
Czech Republic |
Germany |
Israel |
Japan |
Mexico |
Netherlands |
Spain |
United Kingdom |
United States |
|
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
|
After the Week 24 visit, patients will continue to be assessed in person every 24 weeks until bardoxolone methyl becomes commercially available . End of study visit should occur witin 30 days after bardoxolone methyl becomes available through commercial channels. |
Después de la visita de la semana 24, se seguirá evaluando a los pacientes en persona cada 24 semanas hasta que bardoxolona metilo esté comercializado. La visita de final del estudio deberá realizarse en los 30 días siguientes a la comercialización de bardoxolona metilo. |
|
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 |