A Study of Pulmonary Arterial Hypertension Participants Treated With Macitentan or Selexipag

Pulmonary Arterial Hypertension Clinical Trial

— INSPECTIO  

Official title:

Non-Interventional Study on Pulmonary Arterial Hypertension Patients Treated With Macitentan or Selexipag: Experience From an Italian Cohort (INSPECTIO)

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT04567602
• Other study ID #: CR108855
• Secondary ID: 67896049PAH4009
• Status: Completed
• Start date: October 6, 2020
• Est. completion date: March 5, 2024

Study information

• Verified date: May 2024
• Source: Janssen-Cilag S.p.A.
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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).

Recruitment information / eligibility

• Status: Completed
• Enrollment: 186
• Est. completion date: March 5, 2024
• Est. primary completion date: January 25, 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Familial Primary Pulmonary Hypertension
• Hypertension
• Pulmonary Arterial Hypertension

Intervention

Drug:
• PAH medication
There will be no specific interventions.

Locations

Country	Name	City	State
Italy	Generale Regionale F. Miulli	Acquaviva Delle Fonti
Italy	A.O. Universitaria Ospedali Riuniti di Ancona	Ancona
Italy	Azienda Ospedaliero Universitaria Consorziale Policlinico di Bari	Bari
Italy	Ospedale Di Venere	Bari
Italy	Ospedale di Bolzano	Bolzano
Italy	ASST Spedali Civili Brescia	Brescia
Italy	Azienda Ospedaliera G. Brotzu	Cagliari
Italy	AO di Catanzaro Pugliese Ciaccio	Catanzaro
Italy	ASL2 Lanciano - Vasto - Chieti - Ospedale 'SS Annunziata' di Chieti	Chieti
Italy	AOU Careggi	Firenze
Italy	Azienda Ospedaliero Universitaria Ospedali Riuniti di Foggia	Foggia
Italy	Ospedale Policlinico San Martino IRCCS	Genova
Italy	Presidio Ospedaliero di Ivrea	Ivrea
Italy	UOC Oncologia Ospedale Provinciale di Macerata	Macerata
Italy	Centro Cardiologico Monzino	Milano
Italy	Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico	Milano
Italy	IRCCS Ospedale San Raffaele	Milano
Italy	Ospedale Monaldi	Napoli
Italy	Università del Piemonte Orientale - Ospedale Maggiore della Carità di Novara	Novara
Italy	AOU di Padova	Padova
Italy	ISMETT Istituto Mediterraneo Trapianti e Terapie ad Alta Specializzazione	Palermo
Italy	Fondazione IRCCS Policlinico San Matteo	Pavia
Italy	Fondazione Toscana Gabriele Monasterio CNR	Pisa
Italy	AOU Policlinico Umberto I	Roma
Italy	Policinico A Gemelli	Roma
Italy	IRCCS Policlinico San Donato	San Donato Milanese
Italy	Presidio SS Annunziata AOU Sassari	Sassari
Italy	Azienda Ospedaliera Città della Salute e della Scienza di Torino	Torino
Italy	ASUI Santa Maria della Misericordia di Udine	Udine
Italy	Ospedale Borgo Roma	Verona

Sponsors (1)

Lead Sponsor	Collaborator
Janssen-Cilag S.p.A.

Country where clinical trial is conducted

Italy, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Change from Baseline in the Number of the Non-Invasive Low-Risk Criteria to 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.	Baseline and 12 Months
Secondary	Number of Participants with Improved, Stable or Worsened Risk profile from Baseline 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.	Baseline up to 12 and 24 Months
Secondary	Change in Progression in the Number of Participants with Low/High Intermediate Risk	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.	Baseline up to 24 Months
Secondary	Change from Baseline in 6MWD	Change from baseline in 6MWD will be reported.	Baseline up to 24 Months
Secondary	Change from Baseline in BNP or NT-proBNP	Change from baseline in BNP or NT-proBNP will be reported.	Baseline up to 24 Months
Secondary	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	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.	Baseline up to 12 and 24 Months
Secondary	Adherence to European Society of Cardiology and European Respiratory Society (ESC/ERS) guidelines and 6th World Symposium on Pulmonary Hypertension (WSPH)	Adherence to ESC/ERS guidelines and 6th WSPH will be reported.	Up to 24 Months
Secondary	Change from Baseline in Echocardiographic Parameters	Change in echocardiographic parameters will be reported as assessed by echocardiogram.	Baseline up to 24 Months
Secondary	Change from Baseline in Hemodynamic Parameters	Change in hemodynamic parameters will be reported.	Baseline up to 24 Months
Secondary	Hospitalization Rate due to Worsening of PAH	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.	Up to 24 Months
Secondary	Overall Survival (OS)	OS is defined as the duration/time from the start of study treatment to date of death due to any cause.	Up to 24 Months
Secondary	Change from Baseline in Health-Related Quality of Life (HRQoL) as Measured with Emphasis-10 Questionnaire	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.	Baseline up to 24 Months
Secondary	Change from Baseline in Participants with Narrative plots	Participants will be invited to fill in two narrative plots; one at the enrollment visit and one at the month 12 visit.	Baseline and 12 Months
Secondary	Change from Baseline in Caregivers with Narrative Plots	Caregivers will be invited to fill in two narrative plots; one at the enrollment visit and one at the month 12 visit.	Baseline and 12 Months
Secondary	Number of Participants with Adverse Events (AEs) and Adverse Drug Reactions (ADRs)	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.	Up to 24 Months