E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Pulmonary arterial hypertension related to congenital heart disease. |
|
E.1.1.1 | Medical condition in easily understood language |
Elevated pressure in the lung arteries as a consequence of congenital heart disease |
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E.1.1.2 | Therapeutic area | Diseases [C] - Cardiovascular Diseases [C14] |
MedDRA Classification |
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 determine whether the use of selexipag on top of current PAH medication improves exercise capacity in adult patients with pulmonary arterial hypertension related to congenital heart disease |
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E.2.2 | Secondary objectives of the trial |
1. Time-to-first-(PAH-related) morbidity event or death 2. Onset of (supra)ventricular arrhythmias 3. Worsening of WHO functional class 4. Increase right ventricular function parameters with echocardiography 5. Decrease in serum biomarkers (NT-pro-BNP) 6. Increase in Quality-of-Life scores 7. Number of adverse and serious adverse events
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
- Male and female patients ≥ 18 years old - Symptomatic PAH related to congenital heart disease of all complexities - WHO functional class II-III - Documented hemodynamic diagnosis of PAH by right heart catheterization or echocardiography, performed at time prior to screening - Patients not receiving treatment for PAH or those who are receiving endothelin-receptor antagonist, a phosphodiesterase type 5 inhibitor or both at a dose that has been stable for 3 months - Signed informed consent |
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E.4 | Principal exclusion criteria |
- WHO functional class IV - Patients who have received prostacyclin (analogs) treatment within 1 month before baseline visit or are scheduled to receive any of these drugs during the trial - Patients with moderate or severe hepatic impairment (Child-Pugh B and C) - Females who are lactating or pregnant or plan to become pregnant during the study (a pregnancy test is offered to every female patient within the fertile age) - Known hypersensitivity to any of the excipients of the drug formulations - Contraindication for trial medication - Contraindication for cardiopulmonary exercise test - Incapable of giving informed consent
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E.5 End points |
E.5.1 | Primary end point(s) |
percentage change in exercise capacity |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
At 6 months of treatment with selexipag and at 12 months of study |
|
E.5.2 | Secondary end point(s) |
1. Time-to-first-(PAH-related) morbidity event or death 2. Onset of (supra)ventricular arrhythmias 3. Worsening of WHO functional class 4. Increase right ventricular function parameters with echocardiography 5. Decrease in serum biomarkers (NT-pro-BNP) 6. Increase in Quality-of-Life scores 7. Number of adverse events and serious adverse events
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
At 6 months of treatment with selexipag and at 12 months of study |
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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 | 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 | 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 | Information not present in EudraCT |
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.8.3 |
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 | No |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | No |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
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
|
End of trial 1 year after the last patient is included |
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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 | 1 |
E.8.9.1 | In the Member State concerned months | 3 |
E.8.9.1 | In the Member State concerned days | 0 |