E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Pulmonary arterial hypertension (PAH) |
|
E.1.1.1 | Medical condition in easily understood language |
PAH is a chronic condition in which the pressure in the blood vessels that
go from the heart to the lungs (the pulmonary arteries) is higher than
normal. |
|
E.1.1.2 | Therapeutic area | Diseases [C] - Respiratory Tract Diseases [C08] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 20.0 |
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 |
|
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 main objective of the study is to evaluate the long-term safety of macitentan 10 mg in subjects with pulmonary arterial hypertension. |
|
E.2.2 | Secondary objectives of the trial |
No secondary objectives were defined for this study. |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. Signed informed consent to take part in the study before any studymandated
procedure.
2. Subjects currently treated with macitentan in a clinical study that is
about to come to an end in France.
3. Women of childbearing potential are able to take part in the study if
the following applies:
a. Urine pregnancy test is negative at Enrollment.
b. Agreement to perform monthly urine or serum pregnancy tests during
the study and up to at least 30 days after the study treatment
discontinuation.
c. Agreement to adhere to the planned contraception scheme from
Enrollment up to at least 30 days after study treatment discontinuation. |
|
E.4 | Principal exclusion criteria |
1. Hemoglobin < 80 g/L.
2. Serum aspartate (AST) and/or alanine (ALT) aminotransferases more
than three times the upper limit of normal range.
3. Known and documented history of severe drug-induced hepatic
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impairment (with or without cirrhosis).
4. Pregnant, planning to be become pregnant, or breastfeeding.
5. Known hypersensitivity to macitentan, its excipients, or drugs of the
same class.
6. Planned or current treatment with another investigational treatment
up to 3 months prior to Enrollment.
7. 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. |
|
E.5 End points |
E.5.1 | Primary end point(s) |
The long-term safety of macitentan will be evaluated by the following
endpoints:
1. Treatment-emergent adverse events (AEs) leading to premature
discontinuation of study treatment.
2. Treatment-emergent serious adverse events (SAEs).
3. Pregnancies with maternal exposure to macitentan. |
|
E.5.1.1 | Timepoint(s) of evaluation of this end point |
1. for AEs during treatment phase at months 6, 12, 18, 24, 30, 36 and at
End-of-Treatment (EoT) visit, End-of-Study (EoS) visit and any
unscheduled visits
2. for SAEs during treatment phase at months 6, 12, 18, 24, 30, 36 and
at EoT visit, at EoS visit and any unscheduled visits
3. pregnancy tests performed at Screening and Enrollment (Day1) and
then monthly during treatment phase and at EoT visit |
|
E.5.2 | Secondary end point(s) |
|
E.5.2.1 | Timepoint(s) of evaluation of this end point |
|
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 | 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.8.2.4 | Number of treatment arms in the trial | 1 |
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 | 18 |
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 | 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
|
|
E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 3 |
E.8.9.1 | In the Member State concerned months | |
E.8.9.1 | In the Member State concerned days | |