Pulmonary Arterial Hypertension
Conditions
Brief summary
The purpose of the study is to evaluate the change from baseline to 12 months after study enrollment in the number of the following non-invasive risk criteria: World Health Organization Functional Class (WHO/FC), 6-minute walk distance (6MWD), Brain Natriuretic Peptide (BNP) or N-terminal pro-brain Natriuretic Peptide (NT-proBNP).
Interventions
DRUGPAH medication
There will be no specific interventions.
Sponsors
Janssen-Cilag S.p.A.
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Participants in the clinical classification of pulmonary hypertension Group 1 (PAH) * Participants with idiopathic, heritable and associated forms of PAH (connective tissue disease, congenital heart disease corrected at least 1-year ago, human immunodeficiency virus infection, drug use or toxin, and porto-pulmonary) * Confirmed diagnosis of PAH with hemodynamic study right heart catheterization (RHC) at enrolment or at latest follow-up (not later than twelve months from enrolment date) * Participants with a low or intermediate mortality risk profile, as per clinical judgement based on a multiparametric approach including clinical, functional exercise, right ventricular function and hemodynamic parameters * Participants currently in treatment with selexipag and/or macitentan (in monotherapy or in combination therapy)
Exclusion criteria
* Participants in Group 1 that are responders to the vasoreactivity test * Participants with pulmonary veno-occlusive disease (PVOD), defined on the basis of the following chest findings at high-resolution computed tomography (HRCT): centrilobular ground-glass opacities, smooth thickening of interlobular septa, mediastinal lymph node enlargement * Participants already in treatment with subcutaneous/intravenous prostanoids. Iloprost interventional (IV) (treatment of digital ulcers in systemic sclerosis \[SSc\] participants according to european league against rheumatism \[EULAR\] guidelines) is allowed * Participants currently enrolled in an interventional study
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Change from Baseline in the Number of the Non-Invasive Low-Risk Criteria to 12 Months | Baseline and 12 Months | Change from baseline in the number of the non-invasive low-risk criteria will be assessed according to the following parameters: world health organization (WHO)/functional class (FC)- I, II, 6-minute walk distance (6MWD) greater than (\>) 440 meters (m), and brain natriuretic peptide (BNP) less than (\<) 50 nanogram per liter (ng/l) or N-terminal-pro-hormone brain natriuretic Peptide (NT-proBNP) \<300 ng/l. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change in Progression in the Number of Participants with Low/High Intermediate Risk | Baseline up to 24 Months | Change in risk profile for the low/high intermediate risk participants according to following cut-off values for low intermediate risk/high intermediate risk, respectively: (6MWD \[greater than or equal to \[\>=\] or \< 300m\], Peak oxygen consumption \[VO2\] \[\>= or \< 50% predicted\], BNP \[less than or equal to \[\<=\] or \> 175 ng/l\], or NT-proBNP \[\<= or \> 850 ng/l\], right atrial area \[RA\] \[\<= or \> 22 centimeter \[cm\]\] and right atrial pressure \[RAP\] \[\<= or \> 11 millimeter of mercury \[mmHg\]\] or stroke volume index (SVI) (\> or \<= 38 milliliter per meter square \[ml/m2\])). The low intermediate risk is defined by a presence of at least three low intermediate parameters and a high intermediate risk is defined by a presence of at least three high intermediate parameters. |
| Change from Baseline in 6MWD | Baseline up to 24 Months | Change from baseline in 6MWD will be reported. |
| Change from Baseline in BNP or NT-proBNP | Baseline up to 24 Months | Change from baseline in BNP or NT-proBNP will be reported. |
| Change from Baseline in Registry to Evaluate Early and Long-term PAH Disease Management (REVEAL) 2.0 Risk Score up to 12 and 24 Months | Baseline up to 12 and 24 Months | The REVEAL risk calculator determines the risk status and the scores can be defined as: low risk as a score of \<= 6, intermediate risk as a score of 7 or 8, and high risk as a score of \>= 9 for the survival rates. |
| Adherence to European Society of Cardiology and European Respiratory Society (ESC/ERS) guidelines and 6th World Symposium on Pulmonary Hypertension (WSPH) | Up to 24 Months | Adherence to ESC/ERS guidelines and 6th WSPH will be reported. |
| Change from Baseline in Echocardiographic Parameters | Baseline up to 24 Months | Change in echocardiographic parameters will be reported as assessed by echocardiogram. |
| Number of Participants with Improved, Stable or Worsened Risk profile from Baseline to 12 and 24 Months | Baseline up to 12 and 24 Months | Risk profile is assessed as improved, stable or worsened where improved indicates 1 or more non-invasive parameters from intermediate to low risk profile; stable indicates no change in the parameters; and worsened indicates 1 or more non-invasive parameters move from low or intermediate to intermediate or high range risk. |
| Hospitalization Rate due to Worsening of PAH | Up to 24 Months | The hospitalization rate due to PAH worsening will be calculated based on the number of participants with at least one hospitalization. The calculation of the rate will be based on the number of events over the whole follow-up time that is exposure time. |
| Overall Survival (OS) | Up to 24 Months | OS is defined as the duration/time from the start of study treatment to date of death due to any cause. |
| Change from Baseline in Health-Related Quality of Life (HRQoL) as Measured with Emphasis-10 Questionnaire | Baseline up to 24 Months | The emPHasis-10 is a short and easy questionnaire that consists of 10 items which address breathlessness, fatigue, control and confidence. Each item is scored on a semantic differential six-point scale (0-5), with contrasting adjectives at each end. A total emPHasis-10 score is derived using simple aggregation of the 10 items. emPHasis-10 scores range from 0 to 50, higher scores indicate worse quality of life. |
| Change from Baseline in Participants with Narrative plots | Baseline and 12 Months | Participants will be invited to fill in two narrative plots; one at the enrollment visit and one at the month 12 visit. |
| Change from Baseline in Caregivers with Narrative Plots | Baseline and 12 Months | Caregivers will be invited to fill in two narrative plots; one at the enrollment visit and one at the month 12 visit. |
| Number of Participants with Adverse Events (AEs) and Adverse Drug Reactions (ADRs) | Up to 24 Months | An AE is any untoward medical occurrence in a participant enrolled in a clinical study that does not necessarily have a causal relationship with the pharmaceutical/biological agent under study. An ADR is defined as a response to a medicinal (investigational or non-investigational) product that is noxious and unintended. |
| Change from Baseline in Hemodynamic Parameters | Baseline up to 24 Months | Change in hemodynamic parameters will be reported. |
Countries
Italy
Outcome results
None listed