E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Pulmonary Arterial Hypertension (PAH) |
Hipertensión arterial pulmonar (HAP) |
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E.1.1.1 | Medical condition in easily understood language |
PAH includes rare, chronic cardiopulmonary diseases involving common pathologic features of inappropriate cell growth resulting in increased resistance to blood flow through the pulmonary vasculature. |
La HAP incluye patologías cardiopulmonares raras y crónicas que implican patologías de crecimiento celular inadecuado, provoca mayor resistencia al flujo sanguíneo a través de la vasculatura pulmonar. |
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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 | 21.1 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10064911 |
E.1.2 | Term | Pulmonary arterial hypertension |
E.1.2 | System Organ Class | 10038738 - Respiratory, thoracic and mediastinal disorders |
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E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 20.0 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10077729 |
E.1.2 | Term | Pulmonary arterial hypertension WHO functional class III |
E.1.2 | System Organ Class | 10038738 - Respiratory, thoracic and mediastinal disorders |
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E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 20.0 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10077730 |
E.1.2 | Term | Pulmonary arterial hypertension WHO functional class IV |
E.1.2 | System Organ Class | 10038738 - Respiratory, thoracic and mediastinal disorders |
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E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 20.0 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10077740 |
E.1.2 | Term | Pulmonary arterial hypertension WHO functional class II |
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 |
The objective of the study is to establish the long-term safety and tolerability of AV-101. The long-term effects of AV-101 on efficacy measures will also be assessed |
El objetivo principal del estudio es establecer la seguridad a largo plazo de AV-101. También se evaluarán los efectos a largo plazo de AV-101 en las mediciones de la eficacia |
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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 |
Key Inclusion Criteria: (Additional criteria, may apply, per the protocol) To be eligible, a participant is required to be or have: • Consented to participate in the LTE and has successfully completed the placebo-controlled 24-week Study AV-101-002. • Female subjects of childbearing potential who have agreed to continue to use a highly effective form of contraception during the LTE and for at least 30 days after completing or discontinuing study treatment (highly effective forms of contraception. |
Criterios clave de inclusión: (criterios adicionales pueden aplicar, de acuerdo con el protocolo) Para ser apto, se requiere que un participante: • Haya dado su consentimiento para participar en la ELP y haya completado con éxito el estudio controlado con placebo de 24 semanas AV-101-002. • Sea una mujer en edad fértil que haya acordado seguir utilizando una forma muy eficaz de anticoncepción durante la ELP y al menos 30 días después de completar o interrumpir el tratamiento del estudio |
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E.4 | Principal exclusion criteria |
Key Exclusion Criteria: Subjects meeting any of the following criteria: • The Investigator believes that it would not be in the best interest of the subject to be included in the LTE e.g., for clinical or social reasons. • Subjects who were not compliant with study medication in AV-101-002 as assessed by the Investigator |
Criterios clave de exclusión: Los sujetos que cumplan cualquiera de los siguientes criterios: • El investigador cree que, por ejemplo, por motivos clínicos o sociales, no sería lo mejor para el sujeto que fuera incluido en la ELP. • Los sujetos que no cumplían con la medicación del estudio en AV-101-002 según la evaluación del investigador. |
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E.5 End points |
E.5.1 | Primary end point(s) |
Safety and tolerability of AV-101. Other Pre-specified Outcome Measures: Change from baseline in the 6MWD [Time Frame: Baseline, up to study completion] Change from baseline in NT-proBNP [Time Frame: Baseline, up to study completion] Change from baseline in Transthoracic Echo parameters of right ventricular (RV) function [Time Frame: Baseline, up to study completion] Time to Clinical Worsening [Time Frame: up to study completion] |
Seguridad y tolerabilidad de AV-101 Otros medidas pre-especificadas de resultados: Cambio con respecto al inicio en la 6MWD [Marco de tiempo: línea de base, hasta la finalización del estudio] Cambio en el NT-proBNP desde el inicio [Marco de tiempo: línea de base, hasta la finalización del estudio] Cambio con respecto al inicio en los parámetros de la función ventricular derecha (VD) del ecocardiograma transtorácico [Marco de tiempo: línea de base, hasta la finalización del estudio] Tiempo hasta el empeoramiento clínico de los acontecimientos [Marco de tiempo: hasta la finalización del estudio] |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
Study Completion |
Finalización del estudio |
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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 | Yes |
E.6.6 | Pharmacokinetic | No |
E.6.7 | Pharmacodynamic | No |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | Yes |
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 | Yes |
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 | Yes |
E.8.1.7.1 | Other trial design description |
Extensión a largo plazo |
Long-term extension |
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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 | 3 |
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 | 6 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 43 |
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 |
Brazil |
Canada |
Chile |
China |
Colombia |
Israel |
Mexico |
Puerto Rico |
Singapore |
South Africa |
United States |
France |
Poland |
Sweden |
Netherlands |
Spain |
Czechia |
Germany |
Greece |
Italy |
Belgium |
Ireland |
Portugal |
Turkey |
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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LVLS A subject has completed the study if he/she has completed all study assessments, including the last visit or the last scheduled procedure shown in the Schedule of Assessments. |
LVLS Un participante ha completado el estudio si ha completado todas las evaluaciones del estudio, incluida la última visita o el último procedimiento programado que se muestra en el Calendario de evaluaciones |
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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 | 3 |
E.8.9.1 | In the Member State concerned months | 1 |
E.8.9.1 | In the Member State concerned days | 29 |
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 | 1 |
E.8.9.2 | In all countries concerned by the trial days | 29 |