E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Pulmonary Arterial Hypertension |
Ipertensione polmonare arteriosa |
|
E.1.1.1 | Medical condition in easily understood language |
Pulmonary Arterial Hypertension is a condition in which the pressure in the blood vessels going to the lungs (the pulmonary arteries) is higher than normal |
Ipertensione polmonare arteriosa è una condizione in cui la pressione nei vasi sanguigni che vanno ai polmoni (arterie polmonari) è più alta del normale |
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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 | 14.1 |
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.1.3 | Condition being studied is a rare disease | Yes |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
To assess safety and tolerability of Epoprostenol for injection in patients with PAH |
Valutare la sicurezza e la tollerabilità dell’epoprostenolo iniettabile nei pazienti affetti da IAP |
|
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. Signed informed consent prior to initiation of any study mandated procedure 2. Patients who completed participation in study AC 066A301 3. Patients who have not obtained access (i.e. reimbursement) to commercial EFI at the time of ending participation in study AC-066A301 |
1. Firma del consenso informato prima dell’inizio delle procedure previste dallo studio 2. Pazienti che hanno completato la partecipazione allo studio AC 066A301 3. Pazienti che al termine della partecipazione allo studio AC-066A301 non hanno avuto accesso (cioè non hanno ottenuto il rimborso) all’epoprostenolo iniettabile disponibile in commercio |
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E.4 | Principal exclusion criteria |
1. Patients who prematurely discontinued study drug in study AC- 066A301 2. Patients for whom continued treatment with EFI is no longer considered appropriate 3. Patients with a clinical deterioration in study AC 066A301 defined by a decrease in Cardiac Index below 2.5 L/min/m2 |
1. Pazienti che hanno interrotto anticipatamente l’assunzione del farmaco in studio nello studio AC-066A301 2. Pazienti nei quali non si ritiene più opportuno continuare il trattamento con epoprostenolo iniettabile 3. Pazienti con peggioramento clinico nello studio AC 066A301 definito come una diminuzione dell’indice cardiaco al di sotto di a 2.5 L/min/m2 |
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E.5 End points |
E.5.1 | Primary end point(s) |
Tolerability / Safety endpoints: • Treatment-emergent adverse events (AEs) up to 24 hours post-EOT • Adverse events leading to premature discontinuation of study drug • Treatment-emergent serious AEs (SAEs) up to 30 days post-EOT |
Tollerabilità / Sicurezza: • Eventi avversi (AE) provocati dal trattamento fino a 24 ore post-EOT • Eventi avversi che provocano l’interruzione anticipata dell’assunzione del farmaco in studio • AE seri (SAE) provocati dal trattamento fino a 30 giorni post-EOT |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
- Monthly phone call during the study - End-of-Study Treatment (EOT) visit - 24 hours following EOT (phone call) - 30 days following EOT (phone call) |
- Telefonate mensili durante lo studio - Visita di fine trattamento (EOT) - 24 ore dopo la EOT (telefonata) - 30 giorni dopo la EOT (telefonata) |
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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 | 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 |
Tolerability, explaratory study phase 3b |
Studio esplorativo di fase 3 b per la tollerabilità |
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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) | 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 | No |
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) | 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.8.2.4 | 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 | No |
E.8.4.1 | Number of sites anticipated in Member State concerned | 1 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 6 |
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 |
|
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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E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 0 |
E.8.9.1 | In the Member State concerned months | 18 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 0 |
E.8.9.2 | In all countries concerned by the trial months | 18 |
E.8.9.2 | In all countries concerned by the trial days | 0 |