E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
pulmonary arterial hypertension (PAH) (idiopathic PAH, familial PAH, and PAH associated with collagen disease, corrcted congenital vitium or anorexigen |
|
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 9.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10064911 |
E.1.2 | Term | Pulmonary arterial hypertension |
|
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 9.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10065150 |
E.1.2 | Term | Associated with pulmonary arterial hypertension |
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E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 9.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10065151 |
E.1.2 | Term | Idiopathic pulmonary arterial hypertension |
|
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 9.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10065152 |
E.1.2 | Term | Familial pulmonary arterial hypertension |
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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 primary objective of the study is to collect and evaluate long-term safety and tolerability data of ACT-293987 (NS-304)- treatment in subjects with pulmonary arterial hypertension (PAH). After Visit 5 (24 weeks) safety will be assessed every 3 months. |
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E.2.2 | Secondary objectives of the trial |
The secondary objectives of the open-extension study are the assessments of efficacy of ACT-293987 (NS-304) regarding the following variable: 6-minute walk test (6MWT). Further objectives of the open-extension study are: assessments of afficacy of ACT-293987 (NS-304) regarding the following variables: - time to aggravation of PAH - proportion of subjects with aggravation of PAH - Borg dyspnoea score - New York Heart Association (NYHA) functional class - Plasma brain natriuretic peptide (BNP) concentrations After Visit 5 (24 weeks) efficacy will be assessed every 6 months. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1) Subjects who have completed the preceding double-blind study (NS-304/-02) 2) Subjects who have signed informed consent for participation in the open-extension study 3) Subjects who are able and willing to refrain from sunbathing, prolonged sun exposure and artificial sunlight exposure such as solarium use or UVA/UVB treatment, and to limit skin and eye exposure to sunlight using appropriate precautions (protective clothing, sunscreen and sunglasses) from the first dose until 14 days after study drug discontinuation. |
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E.4 | Principal exclusion criteria |
1) Subjects with medically abnormal findings (ECG, physical examination, AEs) at the end of visit 7 of the preceding double-blind study that investigators considers not appropriate to enter this study |
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E.5 End points |
E.5.1 | Primary end point(s) |
Safety assessment is the primary endpoint (vital signs: blood pressure, pulse rate; treatments-emergent AEs, clinical laboratory data: hematology, blood chemistry, and urinanalysis; physical examination and 12-lead electrocardiogramm (ECG) parameters). |
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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 | 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) | Yes |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
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.8.3 |
The trial involves single site in the Member State concerned
| Yes |
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 | 8 |
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 | Information not present in EudraCT |
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
|
end of the trial is 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 | 5 |
E.8.9.1 | In the Member State concerned months | |
E.8.9.1 | In the Member State concerned days | |
E.8.9.2 | In all countries concerned by the trial years | 5 |