E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
The intended indication is Pulmonary Arterial Hypertension |
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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 |
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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 |
Primary Objective for AC-065A302 To demonstrate the effect of ACT-293987 on exercise capacity in patients with PAH
Primary Objective for the pool of studies AC-065A301 and AC-065A302 To demonstrate the effect of ACT-293987 on time to first clinical worsening in patients with PAH |
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E.2.2 | Secondary objectives of the trial |
Secondary Objective for the pool of studies AC-065A301 and AC-065A302 To evaluate the effects of ACT-293987 on: - Safety and tolerability
Secondary Objectives for the pool of studies AC-065A301 and AC-065A302 To evaluate the effects of ACT-293987 on: - Other secondary and exploratory efficacy endpoints |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
- Signed informed consent prior to initiation of any study-mandated procedure. - Male and female patients 18 years of age or older with symptomatic PAH. - Documented hemodynamic diagnosis of PAH. |
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E.4 | Principal exclusion criteria |
- Patients who have received prostacyclin (epoprostenol) or prostacyclin analogs (e.g., treprostinil, iloprost) within 1 month before Baseline Visit, or are scheduled to receive any of these compounds during the trial. - Patients with moderate or severe obstructive lung disease: FEV1/FVC < 70% or FEV1 < 65% of predicted value after bronchodilator administration. - Patients with moderate or severe restrictive lung disease: Total Lung Capacity < 60% of predicted value. - Patients with moderate or severe hepatic impairment (Child-Pugh B and C). - Patients with clinically-relevant left ventricular dysfunction. - Patients with clinically-significant chronic renal insufficiency (estimated creatinine clearance < 30 mL/min, or serum creatinine > 2.5 mg/dL). - Patients with body weight < 40 kg. |
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E.5 End points |
E.5.1 | Primary end point(s) |
- The change of 6MWD from Baseline to Week 16. - The time to first clinical worsening up to EOS. Clinical worsening will be adjudicated in a blinded fashion by an independent Critical Event Committee (CEC). |
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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 | Yes |
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 |
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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 | Yes |
E.8.1.1 | Randomised | Yes |
E.8.1.2 | Open | No |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | Yes |
E.8.1.5 | Parallel group | Yes |
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 | Yes |
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 | 4 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 35 |
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 | Information not present in EudraCT |
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 |
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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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End of study will coincide with Study Closure (if no premature drug discontinuation) |
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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 | 4 |
E.8.9.1 | In the Member State concerned months | 3 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 4 |
E.8.9.2 | In all countries concerned by the trial months | 3 |
E.8.9.2 | In all countries concerned by the trial days | 0 |