Extension of the Psychometric Validation Study ORCHESTRA in Patients With PAH

Study Overview
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Brief Summary
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Prospective, multi-center, open-label, single-arm, Phase 3b extension study of macitentan in patients with PAH. To assess the long-term safety of macitentan in patients with pulmonary arterial hypertension (PAH) beyond treatment in the AC-055-310 study.

Detailed Description
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To assess the long-term safety of macitentan in patients with pulmonary arterial hypertension (PAH) beyond treatment in the AC-055-310 study.

Official Title
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An Extension of AC-055-310, a Multi-center, Open-label, Single-arm, Phase 3b Study of Macitentan in Patients With Pulmonary Arterial Hypertension to Psychometrically Validate the French, Italian and Spanish Versions of the PAH-SYMPACT™

Conditions
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Pulmonary Arterial Hypertension

Intervention / Treatment
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* Drug: Macitentan


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Signed informed consent prior to any study-mandated procedure. Patients with PAH who completed the ORCHESTRA study (AC-055-310) as scheduled Women of childbearing potential (as defined below) must: Have a negative serum pregnancy test at Visit 1 (i.e., Visit 4 of study AC 055 310) and agree to perform monthly serum pregnancy tests. Agree to use two reliable methods of contraception in parallel, from Visit 1 until 1 month after study drug discontinuation (see details below). A female is considered to have childbearing potential unless she meets at least one of the following criteria: Previous bilateral salpingo and/or oophorectomy, or hysterectomy. Premature ovarian failure confirmed by a specialist. Pre-pubescence, XY genotype, Turner syndrome, uterine agenesis. Postmenopausal, defined as 12 consecutive months with no menses without an alternative medical cause. Of the two contraceptive methods that must be used, one must be from Group 1, and one must be from Group 2, defined as follows: Group 1: Oral, implantable, transdermal or injectable hormonal contraceptives, intrauterine devices, female sterilization (tubal ligation or non-surgical sterilization, e.g., permanent contraception with Essure procedure), or partner's sterilization (vasectomy). If a hormonal contraceptive is chosen from this group, it must be taken for at least one month prior to enrollment. Alternatively, if the Essure procedure is chosen as a contraceptive method, a hysterosalpingogram must have been performed to confirm correct location of the microinserts and tubal occlusion (as per manufacturer's recommendations). Group 2: Female or male condoms, diaphragm or cervical cap, any of them in combination with a spermicide. Sexual abstinence, rhythm methods, or contraception by the partner alone are not considered as acceptable methods of contraception for this study. Exclusion Criteria: Patients who prematurely discontinued study drug in study AC-055-310. Females who are lactating or pregnant (positive Visit 1 pregnancy test) or plan to become pregnant during the study. AST and/or ALT more than 3 X ULN.

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 80 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Macitentan<br>10 mg once daily | Drug: Macitentan<br>* 10 mg once daily<br>* Other names: ACT-064992;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| To assess the long-term safety of macitentan in patients with pulmonary arterial hypertension (PAH) beyond treatment in the AC-055-310 study. | Treatment-emergent adverse events (AEs) AEs leading to premature discontinuation of study drug Treatment-emergent serious adverse events (SAEs) Proportion of patients with treatment-emergent ALT and/or AST abnormality (> 3, > 5, and > 8 x ULN) associated or not with total bilirubin > 2 x ULN. Proportion of patients with treatment-emergent hemoglobin abnormality (≦ 100 g/L, and ≦ 80 g/L) | Baseline to end of treatment visit (around 6 months on average) | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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PAH-SYMPACT, PAH, pulmonary arterial hypertension, psychometric instrument