Clinical trial NCT04567602: A Study of Pulmonary Arterial Hypertension Participants Treated With Macitentan or Selexipag
Official title: Non-Interventional Study on Pulmonary Arterial Hypertension Patients Treated With Macitentan or Selexipag: Experience From an Italian Cohort (INSPECTIO)
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Pulmonary Arterial Hypertension
Lead sponsor: Janssen-Cilag S.p.A.
Sponsor class: INDUSTRY
First posted: 2020-09-28
Enrollment: 186
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Study population consist of participants with confirmed diagnosis of PAH.

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).


Eligibility criteria: 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

Primary outcomes:
measure: Change from Baseline in the Number of the Non-Invasive Low-Risk Criteria to 12 Months
timeFrame: Baseline and 12 Months
Secondary outcomes:
measure: Number of Participants with Improved, Stable or Worsened Risk profile from Baseline to 12 and 24 Months
timeFrame: Baseline up to 12 and 24 Months
measure: Change in Progression in the Number of Participants with Low/High Intermediate Risk
timeFrame: Baseline up to 24 Months
measure: Change from Baseline in 6MWD
timeFrame: Baseline up to 24 Months
measure: Change from Baseline in BNP or NT-proBNP
timeFrame: Baseline up to 24 Months
measure: 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
timeFrame: Baseline up to 12 and 24 Months
measure: Adherence to European Society of Cardiology and European Respiratory Society (ESC/ERS) guidelines and 6th World Symposium on Pulmonary Hypertension (WSPH)
timeFrame: Up to 24 Months
measure: Change from Baseline in Echocardiographic Parameters
timeFrame: Baseline up to 24 Months
measure: Change from Baseline in Hemodynamic Parameters
timeFrame: Baseline up to 24 Months
measure: Hospitalization Rate due to Worsening of PAH
timeFrame: Up to 24 Months
measure: Overall Survival (OS)
timeFrame: Up to 24 Months
measure: Change from Baseline in Health-Related Quality of Life (HRQoL) as Measured with Emphasis-10 Questionnaire
timeFrame: Baseline up to 24 Months
measure: Change from Baseline in Participants with Narrative plots
timeFrame: Baseline and 12 Months
measure: Change from Baseline in Caregivers with Narrative Plots
timeFrame: Baseline and 12 Months
measure: Number of Participants with Adverse Events (AEs) and Adverse Drug Reactions (ADRs)
timeFrame: Up to 24 Months
Other outcomes:
Locations:
city: Acquaviva Delle Fonti
country: Italy
facility: Generale Regionale F. Miulli
city: Ancona
country: Italy
facility: A.O. Universitaria Ospedali Riuniti di Ancona
city: Bari
country: Italy
facility: Azienda Ospedaliero Universitaria Consorziale Policlinico di Bari
city: Bari
country: Italy
facility: Ospedale Di Venere
city: Bolzano
country: Italy
facility: Ospedale di Bolzano
city: Brescia
country: Italy
facility: ASST Spedali Civili Brescia
city: Cagliari
country: Italy
facility: Azienda Ospedaliera G. Brotzu
city: Catanzaro
country: Italy
facility: AO di Catanzaro Pugliese Ciaccio
city: Chieti
country: Italy
facility: ASL2 Lanciano - Vasto - Chieti - Ospedale 'SS Annunziata' di Chieti
city: Firenze
country: Italy
facility: AOU Careggi
city: Foggia
country: Italy
facility: Azienda Ospedaliero Universitaria Ospedali Riuniti di Foggia
city: Genova
country: Italy
facility: Ospedale Policlinico San Martino IRCCS
city: Ivrea
country: Italy
facility: Presidio Ospedaliero di Ivrea
city: Macerata
country: Italy
facility: UOC Oncologia Ospedale Provinciale di Macerata
city: Milano
country: Italy
facility: Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico
city: Milano
country: Italy
facility: IRCCS Ospedale San Raffaele
city: Milano
country: Italy
facility: Centro Cardiologico Monzino
city: Napoli
country: Italy
facility: Ospedale Monaldi
city: Novara
country: Italy
facility: Università del Piemonte Orientale - Ospedale Maggiore della Carità di Novara
city: Padova
country: Italy
facility: AOU di Padova
city: Palermo
country: Italy
facility: ISMETT Istituto Mediterraneo Trapianti e Terapie ad Alta Specializzazione
city: Pavia
country: Italy
facility: Fondazione IRCCS Policlinico San Matteo
city: Pisa
country: Italy
facility: Fondazione Toscana Gabriele Monasterio CNR
city: Roma
country: Italy
facility: AOU Policlinico Umberto I
city: Roma
country: Italy
facility: Policinico A Gemelli
city: San Donato Milanese
country: Italy
facility: IRCCS Policlinico San Donato
city: Sassari
country: Italy
facility: Presidio SS Annunziata AOU Sassari
city: Torino
country: Italy
facility: Azienda Ospedaliera Città della Salute e della Scienza di Torino
city: Udine
country: Italy
facility: ASUI Santa Maria della Misericordia di Udine
city: Verona
country: Italy
facility: Ospedale Borgo Roma
Study officials:
affiliation: Janssen-Cilag S.p.A.
name: Janssen-Cilag S.p.A., Italy Clinical Trial
role: STUDY_DIRECTOR
References: