E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Indication: Symptomatic 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 |
To compare the safety and tolerability of inhaled iloprost delivered by power disc-15 or power disc-6 up to 12 weeks treatment in patients with symptomatic PAH.
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E.2.2 | Secondary objectives of the trial |
To compare the inhalation times of inhaled iloprost delivered by I-neb using the power disc-15 or power disc-6 up to 12 weeks treatment in patients with symptomatic PAH. |
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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 with symptomatic idiopathic, familial pulmonary arterial hypertension or pulmonary hypertension associated with HIV or drugs/toxins (APAH) in NYHA functional class II to IV who have completed study AC-063A301,
3.Women of childbearing potential* with a negative urine pre-treatment pregnancy test and who consistently and correctly use (from screening and up to 28 days after discontinuation of study drug) a reliable method of contraception with a Pearl index of <1% (oral hormonal contraceptive, implant, vaginal hormone ring, or intrauterine system [IUS]), during the entire study duration and for at least 1 month after last study drug intake. Their partner, if not vasectomised, must use a condom in addition.
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E.4 | Principal exclusion criteria |
1.Pulmonary arterial hypertension related to any condition other than those specified in the inclusion criteria,
2.Pulmonary arterial hypertension associated with significant venous or capillary involvement (PCWP > 15 mmHg), known pulmonary veno-occlusive disease, or pulmonary capillary hemangiomatosis,
3.HIV seropositive with any of the following: •Active opportunistic infections within 3 months prior to randomization •Changes in antiretroviral regimen within 3 months of randomization •Using inhaled pentamidine
4.Moderate to severe obstructive lung disease: forced expiratory volume in 1 second/forced vital capacity (FEV1/FVC) < 70% and FEV1 < 65% of predicted value after bronchodilator administration,
5.Moderate to severe restrictive lung disease: total lung capacity (TLC) < 60% of predicted value,
6.Pregnant or breast-feeding women, 7.Systemic hypertension (systolic blood pressure > 160 mmHg or diastolic blood pressure > 100 mmHg on repeated measurement),
8.Systolic blood pressure < 95 mmHg,
9.Moderate to severe hepatic impairment, i.e., Child-Pugh Class B or C,
10.Chronic renal insufficiency defined by serum creatinine > 2.5 mg/dL (221 μmol/L) or ongoing dialysis,
11.Clinically relevant bleeding disorder or active bleeding,
12.Known hypersensitivity to iloprost or any of its excipients.
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E.5 End points |
E.5.1 | Primary end point(s) |
•Treatment-emergent adverse events (AEs) from the first inhalation of study drug to the day before the end of 12-week treatment period or up to last inhalation in case of early discontinuation •Treatment-emergent serious adverse events (SAEs) from the first inhalation of study drug to the day before the end of 12-week treatment period or up to last inhalation in case of early discontinuation •AEs leading to premature discontinuation of study drug.
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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 | No |
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 | No |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | Yes |
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 | Information not present in EudraCT |
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 | 2 |
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 | 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 | 9 |
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 | 9 |
E.8.9.2 | In all countries concerned by the trial days | 0 |