E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
symptomatic pulmonary arterial hypertension |
|
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 |
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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 | 17.1 |
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 the long-term safety and tolerability of ACT 064992 in patients with symptomatic pulmonary arterial hypertension (PAH). |
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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 |
Signed informed consent prior to initiation of any study-mandated procedure.
• Patients with pulmonary arterial hypertension and having completed the event-driven study,
AC-055-302/SERAPHIN, or
Patients who have experienced a morbidity event and for whom a written approval to roll
over into this study has been obtained from the Sponsor.
• Men or women ≥ 12 years of age:
Only sites that have adequate pediatric pulmonary experience are allowed to enroll
patients between 12 and 17 year of age.
• Women of childbearing potential* with a negative pre-treatment serum
pregnancy test and who consistently and correctly use (from screening and up to
28 days after study treatment discontinuation) 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, your partner, if not
vasectomised, must use a condom in addition.
*A woman is considered to have childbearing potential unless she meets at least one of
the following criteria:
− previous bilateral salpingo-oophorectomy or hysterectomy,
− premature ovarian failure confirmed by a specialist gynecologist,
− pre-pubescence, XY genotype, Turner syndrome, uterine agenesis,
− age > 50 years and not treated with any kind of HRT for at least 2 years
prior to screening with amenorrhea for at least 24 consecutive months
prior to screening and a serum FSH level of > 40 IU/L at screening. |
|
E.4 | Principal exclusion criteria |
•Any major violation of protocol AC-055-302/SERAPHIN.
•Pregnancy or breast-feeding.
•AST and/or ALT > 3 times the upper limit of the normal range.
•Any known factor or disease that might interfere with treatment compliance, study conduct or interpretation of the results, such as drug or alcohol dependence or psychiatric disease.
•Known hypersensitivity to ACT-064992 or any of the excipients. |
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E.5 End points |
E.5.1 | Primary end point(s) |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
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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 | 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 | 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) | 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 | 10 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 51 |
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 |
Argentina |
Australia |
Austria |
Belarus |
Belgium |
Bulgaria |
Canada |
Chile |
China |
Colombia |
Croatia |
France |
Germany |
Hong Kong |
Hungary |
India |
Israel |
Italy |
Malaysia |
Mexico |
Netherlands |
Peru |
Poland |
Romania |
Russian Federation |
Serbia |
Singapore |
Slovakia |
South Africa |
Sweden |
Taiwan |
Thailand |
Ukraine |
United Kingdom |
United States |
|
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
|
|
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 | 0 |
E.8.9.1 | In the Member State concerned days | |
E.8.9.2 | In all countries concerned by the trial years | 5 |
E.8.9.2 | In all countries concerned by the trial months | 0 |