Clinical trial NCT04955990: A Study of Real-world Cohort of Pulmonary Arterial Hypertension (PAH) Participants
Official title: An International, Non-Drug Interventional, Real-world Cohort of PAH Patients Newly Initiating PAH Therapy With Guideline-directed Assessments of Disease Severity
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE4
Conditions: Pulmonary Arterial Hypertension
Lead sponsor: Actelion
Sponsor class: INDUSTRY
First posted: 2021-07-09
Enrollment: 232
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: OTHER

Brief summary: This study is designed to describe pulmonary arterial hypertension (PAH) participants in terms of their clinical characteristics, therapies used, disease progression, and outcomes (example, death, hospitalization, risk category for predicted mortality risk, and patient-reported outcomes \[PROs\]) in real-world clinical practice. This study will collect high-quality real-world data that may be used as a stand-alone dataset or in combination with other studies to address relevant research questions (example, serve as an external control dataset to another study) to support development and access to PAH therapies, as well as to contribute to the knowledge base of PAH through publications.


Eligibility criteria: Inclusion Criteria:

* Symptomatic pulmonary arterial hypertension (PAH) in any PAH subtype
* PAH diagnosis confirmed by hemodynamic evaluation at rest at any time prior to or at the index date fulfilling all of the criteria below: a) Mean pulmonary artery pressure greater than (\>) 20 millimeters of mercury (mm Hg), and b) Pulmonary artery wedge pressure or left ventricular end diastolic pressure less than or equal to (\<=) 15 mm Hg, and c) Pulmonary vascular resistance greater than or equal to (\>=) 3 Wood Units (that is, \>= 240 dynes seconds per centimeters penta \[dyn∙sec/cm\^5\])
* Participant satisfies either a or b: a) Newly initiating 1 or more PAH therapy(ies) (as monotherapy or add-on therapy) at index date. These newly initiated PAH therapies should not have been used within 3 months of the index date; b) Taking macitentan 10 milligrams (mg) therapy (as monotherapy or in combination) with no changes in PAH therapy for within 3 months prior to the index date
* All mandated assessments must be performed and recorded at the baseline visit before the initiation of the new PAH therapy at the index date or enrollment in the study.
* For the pulmonary arterial hypertension-symptoms and impact (PAH-SYMPACT) substudy only: Participants initiating any endothelin receptor antagonist (ERA) or phosphodiesterase-5 inhibitor therapies at index date or at therapy change must provide consent to enroll in the optional PAH-SYMPACT substudy. Refusal to give consent for the optional PAH-SYMPACT substudy will not exclude a participant from participation in the main study

Exclusion Criteria:

* Participants enrolled in any interventional clinical trial with an investigational therapy in the 3-month period prior to index date
* Currently enrolled in an observational study sponsored or managed by a Janssen company
* Presence of moderate or severe obstructive lung disease (forced expiratory volume in 1 second \[FEV1\] / forced vital capacity \[FVC\] \<70%; and FEV1 \<60% of predicted after bronchodilator administration) in participants with a known or suspected history of significant lung disease, as documented by a spirometry test performed within 1 year prior to screening
* Presence of moderate or severe restrictive lung disease (for example, total lung capacity or FVC \<60 percent \[%\] of normal predicted value) in participants with a known or suspected history of significant lung disease, as documented by a spirometry test performed within 1 year prior to screening

Primary outcomes:
measure: Time to all Cause Death
timeFrame: Up to 6 years
measure: Time to Death due to Pulmonary Arterial Hypertension (PAH) or First Hospitalization due to PAH
timeFrame: Up to 6 years
Secondary outcomes:
measure: Time to Death due to PAH
timeFrame: Up to 6 years
measure: Time to First all-cause Hospitalization
timeFrame: Up to 6 years
measure: Time to First Morbidity/Mortality Event
timeFrame: Up to 6 years
measure: Time to Clinical Worsening
timeFrame: Up to 6 years
measure: Medical Resource Utilization
timeFrame: Up to 6 years
measure: Change from Baseline in 6-minute Walk Distance (6MWD)
timeFrame: Baseline up to 6 years
measure: Change from Baseline in World Health Organization (WHO) Functional Class (FC)
timeFrame: Baseline up to 6 years
measure: Change from Baseline in N-terminal-pro-hormone Brain Natriuretic Peptide (NT-proBNP)
timeFrame: Baseline up to 6 years
measure: Time to Worsening in WHO FC
timeFrame: Up to 6 months
measure: Change from Baseline in the Number of low-risk Noninvasive Criteria Based on WHO FC, 6MWD, and NT-proBNP
timeFrame: Baseline up to 6 years
measure: Time to all-cause Death Based on the Number of low-risk Noninvasive criteria Based on WHO FC, 6MWD, and NT-proBNP
timeFrame: Up to 6 years
measure: Change from Baseline in Number of Participants Within Each Overall Risk Category (Low, Intermediate, or High) According to the Noninvasive Criteria
timeFrame: Baseline up to 6 years
measure: Change from Baseline in Number of Participants Within Each Overall Risk Category (low, Intermediate, or High) According to Registry to Evaluate Early and Long-term PAH Disease Management (REVEAL) Lite 1 Variables
timeFrame: Baseline up to 6 years
measure: Change from Baseline in Number of Participants Within Each Overall Risk Category (low, Intermediate, or High) According to REVEAL Lite 2 Variables
timeFrame: Baseline up to 6 years
measure: Time to all-cause Death Based on the Risk Category Determined by the Noninvasive Criteria
timeFrame: Up to 6 years
measure: Time to all-cause Death Based on the Risk Category Determined by the REVEAL Lite 1
timeFrame: Up to 6 years
measure: Time to all-cause Death Based on the Risk Category Determined by the REVEAL Lite 2
timeFrame: Up to 6 years
measure: Risk Assessment Strategies for Clinical Worsening or Death
timeFrame: Up to 6 years
measure: Change from Baseline in Health-related Quality of Life (HRQoL) as Measured by Symptoms and Impact Questionnaire for Use in Clinical Practice (SYMPACT-CP) Questionnaire
timeFrame: Baseline up to 6 years
measure: Change from Baseline in Health Related Quality of Life Assessed by European Quality of Life (EuroQoL) Group, 5-Dimension, 5-Level Questionnaire (EQ-5D-5L)
timeFrame: Baseline up to 6 years
measure: Change from Baseline in Health-Related Quality of life Status as Assessed by 36-item Short-Form Health Survey (SF-36) v2 Acute Questionnaire
timeFrame: Baseline up to 6 years
measure: Change from Baseline in PAH-specific Medication Adherence as Assessed by Morisky Medication Adherence Scale-8 (MMAS-8) Score
timeFrame: Baseline up to 6 years
measure: Change from Baseline in Medication Adherence Questions of each Prescribed PAH Therapy Class
timeFrame: Baseline up to 6 years
measure: Change from Baseline in PAH-specific Medication Adherence as Assessed by Pulmonary Arterial Hypertension-Symptoms and Impact (PAH-SYMPACT) Questionnaire
timeFrame: Baseline up to 6 years
measure: Change from Baseline in Patient Global Assessment of Disease Severity (PGA-S) of PAH
timeFrame: Baseline up to 6 years
measure: PAH Symptoms and Impact using SYMPACT-CP Questionnaire
timeFrame: Baseline up to 6 years
Other outcomes:
Locations:
city: Brandon
country: United States
facility: Bay Area Cardiology Associates, P.A. - Brandon Office
state: Florida
city: Tampa
country: United States
facility: University of South Florida
state: Florida
city: Chicago
country: United States
facility: Northwestern University
state: Illinois
city: Iowa City
country: United States
facility: University Of Iowa - Hospitals & Clinics
state: Iowa
city: Kansas City
country: United States
facility: University of Kansas Medical Center
state: Kansas
city: Louisville
country: United States
facility: Kentuckiana Pulmonary Associates
state: Kentucky
city: Baltimore
country: United States
facility: University of Maryland
state: Maryland
city: Boston
country: United States
facility: Tufts Medical Center
state: Massachusetts
city: Ann Arbor
country: United States
facility: University of Michigan
state: Michigan
city: Rochester
country: United States
facility: Mayo Clinic - Rochester
state: Minnesota
city: Columbia
country: United States
facility: University of Missouri
state: Missouri
city: Albuquerque
country: United States
facility: University of New Mexico
state: New Mexico
city: Cincinnati
country: United States
facility: University of Cinncinnati Physicans- UC Health Physicnas Office
state: Ohio
city: Pittsburgh
country: United States
facility: University of Pittsburgh Medical Center
state: Pennsylvania
city: Houston
country: United States
facility: Houston Methodist Hospital
state: Texas
city: Rosario
country: Argentina
facility: Instituto Cardiovascular de Rosario
city: Belo Horizonte
country: Brazil
facility: Universidade Federal De Minas Gerais - Hospital das Clínicas
city: Botucatu
country: Brazil
facility: Universidade Estadual Paulista 'Julio De Mesquita Filho'
city: Porto Alegre
country: Brazil
facility: Irmandade Santa Casa de Misericordia de Porto Alegre
city: Sao Paulo
country: Brazil
facility: Hospital Das Clinicas Da Faculdade De Medicina Da USP
city: Edmonton
country: Canada
facility: University of Alberta Hospital
state: Alberta
city: London
country: Canada
facility: London Health Sciences Centre Victoria Hospital
state: Ontario
city: Beijing
country: China
facility: Beijing Anzhen Hospital
city: Beijing
country: China
facility: Beijing Fuwai Hospital
city: Beijing
country: China
facility: Peking Union Medical College Hospital
city: Chengdu
country: China
facility: West China Hospital Sichuan University
city: Shanghai
country: China
facility: Shanghai Pulmonary Hospital
city: Bonn
country: Germany
facility: Universitatsklinikum Bonn
city: Milano
country: Italy
facility: Ospedale S.Giuseppe, Gruppo MultiMedica
city: Pavia
country: Italy
facility: IRCCS Policlinico San Matteo, Università degli studi di Pavi
city: Rome
country: Italy
facility: Universita degli Studi di Roma 'La Sapienza' - Umberto I Policlinico di Roma
city: Okayama
country: Japan
facility: National Hospital Organization Okayama Medical Center
city: Busan
country: Korea, Republic of
facility: Pusan National University Hospital
city: Incheon
country: Korea, Republic of
facility: Gachon University Gil Medical Center
city: Seoul
country: Korea, Republic of
facility: Severance Hospital Yonsei University Health System
city: Seoul
country: Korea, Republic of
facility: Asan Medical Center
city: Seoul
country: Korea, Republic of
facility: The Catholic University of Korea Seoul St Mary s Hospital
city: Kuala Lumpur
country: Malaysia
facility: National Heart Institute
city: Amsterdam
country: Netherlands
facility: Vrije Universiteit Amsterdam (VU)
city: Nieuwegein
country: Netherlands
facility: Sint Antonius Ziekenhuis
city: Rotterdam
country: Netherlands
facility: Erasmus MC
city: Krakow
country: Poland
facility: Krakowski Szpital Specjalistyczny im Jana Pawla II
city: Lublin
country: Poland
facility: Wojewodzki Szpital Specjalistyczny im Stefana Kardynala Wyszynskiego SPZOZ
city: Guaynabo
country: Puerto Rico
facility: CardioPulmonary Research, PSC
city: Singapore
country: Singapore
facility: National Heart Centre (NHC) Singapore
city: Madrid
country: Spain
facility: Hosp. Univ. 12 de Octubre
city: Majadahonda
country: Spain
facility: Hosp. Univ. Pta. de Hierro Majadahonda
city: Santander
country: Spain
facility: Hosp. Univ. Marques de Valdecilla
city: Goeteborg
country: Sweden
facility: Sahlgrenska Universitetsjukhuset
city: London
country: United Kingdom
facility: Royal Free Hospital
city: London
country: United Kingdom
facility: Hammersmith Hospital
city: Newcastle upon Tyne
country: United Kingdom
facility: Freeman Hospital
city: Papworth Everard
country: United Kingdom
facility: Papworth Hospital, Cambridge University
city: Sheffield
country: United Kingdom
facility: Sheffield Teaching Hospitals NHS Foundation Trust Royal Hallamshire Hospital
Study officials:
affiliation: Actelion
name: Actelion Clinical Trial
role: STUDY_DIRECTOR
References: