Back to resultsA Study of Real-world Cohort of Pulmonary Arterial Hypertension (PAH) Participants (CARE PAH)
Primary Purpose
Pulmonary Arterial Hypertension
Status
Terminated
Phase
Phase 4
Locations
International
Study Type
Interventional
Intervention
PAH Therapies
Sponsored by
About this trial
This is an interventional other trial for Pulmonary Arterial Hypertension
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
Sites / Locations
- Bay Area Cardiology Associates, P.A. - Brandon Office
- University of South Florida
- Northwestern University
- University Of Iowa - Hospitals & Clinics
- University of Kansas Medical Center
- Kentuckiana Pulmonary Associates
- University of Maryland
- Tufts Medical Center
- University of Michigan
- Mayo Clinic - Rochester
- University of Missouri
- University of New Mexico
- University of Cinncinnati Physicans- UC Health Physicnas Office
- University of Pittsburgh Medical Center
- Houston Methodist Hospital
- Instituto Cardiovascular de Rosario
- Universidade Federal De Minas Gerais - Hospital das Clínicas
- Universidade Estadual Paulista 'Julio De Mesquita Filho'
- Irmandade Santa Casa de Misericordia de Porto Alegre
- Hospital Das Clinicas Da Faculdade De Medicina Da USP
- University of Alberta Hospital
- London Health Sciences Centre - Victoria Hospital
- Beijing Anzhen Hospital
- Beijing Fuwai Hospital
- Peking Union Medical College Hospital
- West China Hospital, Sichuan University
- Shanghai Pulmonary Hospital
- Universitatsklinikum Bonn
- Ospedale S.Giuseppe, Gruppo MultiMedica
- IRCCS Policlinico San Matteo, Università degli studi di Pavi
- Universita degli Studi di Roma 'La Sapienza' - Umberto I Policlinico di Roma
- National Hospital Organization Okayama Medical Center
- Pusan National University Hospital
- Gachon University Gil Medical Center
- Severance Hospital, Yonsei University Health System
- Asan Medical Center
- The Catholic University of Korea, Seoul St. Mary's Hospital
- National Heart Institute
- Vrije Universiteit Amsterdam (VU)
- Sint Antonius Ziekenhuis
- Erasmus MC
- Krakowski Szpital Specjalistyczny im Jana Pawla II
- Wojewodzki Szpital Specjalistyczny im. Stefana Kardynala Wyszynskiego SPZOZ
- CardioPulmonary Research, PSC
- National Heart Centre (NHC) Singapore
- Hosp. Univ. 12 De Octubre
- Hosp. Univ. Pta. de Hierro Majadahonda
- Hosp. Univ. Marques de Valdecilla
- Sahlgrenska Universitetsjukhuset
- Royal Free Hospital
- Hammersmith Hospital
- Freeman Hospital
- Papworth Hospital, Cambridge University
- Sheffield Teaching Hospitals NHS Foundation Trust Royal Hallamshire Hospital
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Participants with PAH
Arm Description
Participants with pulmonary arterial hypertension (PAH) who newly initiate any PAH therapy(ies) at the index date (date when a participant starts the first new PAH therapy after baseline assessments) in a routine clinical setting, either as first-line therapy, as replacement therapies, as concomitant with other PAH therapies, or have already been receiving macitentan 10 milligrams (mg) for at least 3 months prior to the index date. The primary data source for this study will be the medical records of each participant.
Outcomes
Primary Outcome Measures
Time to all Cause Death
Time to all cause death will be reported. All-cause death defined as deaths due to any cause.
Time to Death due to Pulmonary Arterial Hypertension (PAH) or First Hospitalization due to PAH
Time to death due to PAH or first hospitalization due to PAH will be reported.
Secondary Outcome Measures
Time to Death due to PAH
Time to death due to PAH will be reported.
Time to First all-cause Hospitalization
Time to first all-cause hospitalization will be reported.
Time to First Morbidity/Mortality Event
Time to first morbidity/mortality event will be reported. All-cause death, non-planned hospitalization due to PAH (including for worsening of PAH, atrial septostomy, lung transplantation with or without heart transplantation, or initiation of parenteral prostacyclins), PAH-related disease progression, defined as (both criteria must be satisfied): At least 15 percent (%) decrease in 6-minute walking distance (6MWD) from baseline or therapy change visit, and initiation of additional PAH therapy or worsening of World Health Organization (WHO) functional class (FC) will be collected for morbidity/mortality events assessment.
Time to Clinical Worsening
Time to clinical worsening will be reported. All-cause death, non-planned hospitalization due to PAH (including for worsening of PAH, atrial septostomy, lung transplantation with or without heart transplantation, or initiation of parenteral prostacyclins), PAH-related deterioration identified by at least 1 criterion: worsening of WHO FC, deterioration by at least 15% in exercise capacity, as measured by the 6MWD, any signs or symptoms of right-sided heart failure will be collected for clinical worsening assessment.
Medical Resource Utilization
Number per year of all-cause and PAH-related hospitalizations; number per year of in-patient hospital days for all causes and PAH-related causes; number per year of emergency room visits for all causes and for PAH-related causes that do not result in hospital admittance will be collected for assessment of medical resource utilization.
Change from Baseline in 6-minute Walk Distance (6MWD)
Change from baseline in 6MWD according to non-invasive criteria will be assessed as per the low (greater than [>] 440 meters [m]), intermediate (165-440m), and high-risk category (less than [<] 165m).
Change from Baseline in World Health Organization (WHO) Functional Class (FC)
Change from baseline in WHO FC according to non-invasive criteria will be assessed as per the low (I, II), intermediate (III), and high-risk category (IV).
Change from Baseline in N-terminal-pro-hormone Brain Natriuretic Peptide (NT-proBNP)
Change from baseline in NT-proBNP according to non-invasive criteria will be assessed as per the low (<300 nanogram per liters [ng/l]), intermediate (300-1400ng/l), and high-risk category (>1400 ng/l).
Time to Worsening in WHO FC
Time to worsening in WHO FC will be reported.
Change from Baseline in the Number of low-risk Noninvasive Criteria Based on WHO FC, 6MWD, and NT-proBNP
Change from baseline in the number of low-risk noninvasive criteria based on WHO FC, 6MWD, and NT-proBNP will be reported.
Time to all-cause Death Based on the Number of low-risk Noninvasive criteria Based on WHO FC, 6MWD, and NT-proBNP
Time to all-cause death based on the number of low-risk noninvasive criteria based on WHO FC, 6MWD, and NT-proBNP will be reported.
Change from Baseline in Number of Participants Within Each Overall Risk Category (Low, Intermediate, or High) According to the Noninvasive Criteria
Change from baseline in number of participants within each overall risk category (low, intermediate, or high) according to low-risk non-invasive criteria will be assessed as per the following parameters: WHO FC- low (I, II), intermediate (III), and high-risk category (IV); 6MWD- low (> 440m), intermediate (165-440m), and high-risk category (< 165m), and <165m; and NT-proBNP- low (<300ng/l), intermediate (300-1400ng/l), and high-risk category (>1400 ng/l).
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
Change from baseline in number of participants within each overall risk category (low, Intermediate, or High) according to REVEAL Lite 1 Variables will be reported. The REVEAL Lite 1 variables risk calculator determines the risk status, and the scores (ranges from 0 to 19) can be defined as: low risk as a score of less than or equal to (<=) 6, intermediate risk as a score of 7 or 8, and high risk as a score of greater than or equal to (>=) 9 for the survival rates.
Change from Baseline in Number of Participants Within Each Overall Risk Category (low, Intermediate, or High) According to REVEAL Lite 2 Variables
Change from baseline in number of participants within each overall risk category (low, Intermediate, or High) according to REVEAL Lite 2 Variables will be reported. The REVEAL Lite 2 variables risk calculator determines the risk status, and the scores (ranges from 1 to 14) can be defined as: low risk as a score of <= 5, intermediate risk as a score of 6 or 7, and high risk as a score of >= 8 for the survival rates.
Time to all-cause Death Based on the Risk Category Determined by the Noninvasive Criteria
Time to all-cause death based on the risk category determined by the noninvasive criteria will be reported.
Time to all-cause Death Based on the Risk Category Determined by the REVEAL Lite 1
Time to all-cause death based on the risk category determined by the REVEAL Lite 1 will be reported.
Time to all-cause Death Based on the Risk Category Determined by the REVEAL Lite 2
Time to all-cause death based on the risk category determined by the REVEAL Lite 2 will be reported.
Risk Assessment Strategies for Clinical Worsening or Death
The assessment of risk category for clinical worsening and death will be assessed by the following 3 risk assessment strategies: number of low-risk noninvasive criteria based on WHO FC, 6MWD, and NT-proBNP.
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
A modified version of the PAH-SYMPACT for Use in Clinical Practice (SYMPACT-CP), has been created for use in routine clinical practice, as an assessment to support symptom monitoring and guide treatment decisions by healthcare providers. In the SYMPACT-CP, the 24-hour recall period for the oxygen use item and the 11 symptoms items have been modified to a 1-week recall period. The SYMPACT-CP also includes 11 impact items (with a 1-week recall period). This modification was made with the intention of administering the questionnaire at a single timepoint.
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)
The EQ-5D-5L is an instrument to measure health-related quality of life consisting of a descriptive system and visual analogue scale (VAS). The descriptive system comprises 5 dimensions: mobility, self-care, usual activities, pain/discomfort, and anxiety/depression. Each dimension has 5 levels of perceived problems (1 indicating no problem, 2 indicating slight problems, 3 indicating moderate problems, 4 indicating severe problems, 5 indicating extreme problems). Participant selects an answer for each of 5 dimensions considering the response that best matches his or her health "today". The responses to the 5 dimensions are used to compute a single score ranging from 0 (worst health state) to 100 (better health state) representing the general health status of the individual.
Change from Baseline in Health-Related Quality of life Status as Assessed by 36-item Short-Form Health Survey (SF-36) v2 Acute Questionnaire
The SF-36 v2 acute questionnaire is a 36-item short form survey used to assess the participant's quality of life. In the SF-36 v2 Acute Questionnaire, participants are instructed to rate their health and capacity to perform activities of daily living in eight domains including physical functioning, physical role limitations, bodily pain, general health, vitality, social functioning, emotional role limitations, and mental health during the last week. The scores range from 0 (lowest or worst possible level of functioning) to 100 (highest or best possible level of functioning).
Change from Baseline in PAH-specific Medication Adherence as Assessed by Morisky Medication Adherence Scale-8 (MMAS-8) Score
The MMAS-8 is commonly used in standard clinical practice and a validated measure of treatment adherence. The MMAS-8 includes 8 questions assessing the extent of adherence or nonadherence and reasons for non-adherence.
Change from Baseline in Medication Adherence Questions of each Prescribed PAH Therapy Class
Change from baseline in medication adherence questions of each prescribed PAH therapy class will be reported.
Change from Baseline in PAH-specific Medication Adherence as Assessed by Pulmonary Arterial Hypertension-Symptoms and Impact (PAH-SYMPACT) Questionnaire
The PAH-SYMPACT questionnaire, applicable only for participants who enrolled in the PAH-SYMPACT substudy, is a PRO instrument and composed of 2 parts: the symptoms part and the Impacts part. There is no total symptom score; the 2 domains are: cardiopulmonary symptoms and cardiovascular symptoms. The Impacts part has 2 domains: Physical Impacts Domain and Cognitive/Emotional Impacts Domain which contain 7 and 4 items, respectively. Each item has 5-point Likert response scale (0=not at all, 1=mild, 2=moderate, 3=severe, and 4=very severe). The mean individual weekly symptom item scores are aggregated by domain, with the sum then being divided by the number of symptom items in the respective domain. This leads to the average weekly domain score ranging from 0-4 for each participant with higher scores indicating greater symptom severity or worse impact.
Change from Baseline in Patient Global Assessment of Disease Severity (PGA-S) of PAH
Change from baseline in PGA-S of PAH will be reported. It includes severity of PAH symptoms, such as, none, mild, moderate, severe and very severe.
PAH Symptoms and Impact using SYMPACT-CP Questionnaire
PAH symptoms and impact using SYMPACT-CP questionnaire will be reported.
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT04955990
Brief Title
A Study of Real-world Cohort of Pulmonary Arterial Hypertension (PAH) Participants
Acronym
CARE PAH
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
2. Study Status
Record Verification Date
April 2023
Overall Recruitment Status
Terminated
Why Stopped
The sponsor decided to terminate the study.
Study Start Date
October 14, 2021 (Actual)
Primary Completion Date
December 20, 2022 (Actual)
Study Completion Date
December 20, 2022 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Actelion
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Pulmonary Arterial Hypertension
7. Study Design
Primary Purpose
Other
Study Phase
Phase 4
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
227 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Participants with PAH
Arm Type
Experimental
Arm Description
Participants with pulmonary arterial hypertension (PAH) who newly initiate any PAH therapy(ies) at the index date (date when a participant starts the first new PAH therapy after baseline assessments) in a routine clinical setting, either as first-line therapy, as replacement therapies, as concomitant with other PAH therapies, or have already been receiving macitentan 10 milligrams (mg) for at least 3 months prior to the index date. The primary data source for this study will be the medical records of each participant.
Intervention Type
Drug
Intervention Name(s)
PAH Therapies
Intervention Description
PAH therapies will be administered in a routine clinical setting and data will be collected from participants who newly initiate any PAH therapy(ies), or have already been receiving macitentan 10 mg for at least 3 months prior to the index date. Participants will receive PAH therapies such as Macitentan, Ambrisentan, Bosentan, Tadalafil, Sildenafil, Riociguat, Selexipag, Epoprostenol, Iloprost, Treprostinil, and Beraprost.
Primary Outcome Measure Information:
Title
Time to all Cause Death
Description
Time to all cause death will be reported. All-cause death defined as deaths due to any cause.
Time Frame
Up to 6 years
Title
Time to Death due to Pulmonary Arterial Hypertension (PAH) or First Hospitalization due to PAH
Description
Time to death due to PAH or first hospitalization due to PAH will be reported.
Time Frame
Up to 6 years
Secondary Outcome Measure Information:
Title
Time to Death due to PAH
Description
Time to death due to PAH will be reported.
Time Frame
Up to 6 years
Title
Time to First all-cause Hospitalization
Description
Time to first all-cause hospitalization will be reported.
Time Frame
Up to 6 years
Title
Time to First Morbidity/Mortality Event
Description
Time to first morbidity/mortality event will be reported. All-cause death, non-planned hospitalization due to PAH (including for worsening of PAH, atrial septostomy, lung transplantation with or without heart transplantation, or initiation of parenteral prostacyclins), PAH-related disease progression, defined as (both criteria must be satisfied): At least 15 percent (%) decrease in 6-minute walking distance (6MWD) from baseline or therapy change visit, and initiation of additional PAH therapy or worsening of World Health Organization (WHO) functional class (FC) will be collected for morbidity/mortality events assessment.
Time Frame
Up to 6 years
Title
Time to Clinical Worsening
Description
Time to clinical worsening will be reported. All-cause death, non-planned hospitalization due to PAH (including for worsening of PAH, atrial septostomy, lung transplantation with or without heart transplantation, or initiation of parenteral prostacyclins), PAH-related deterioration identified by at least 1 criterion: worsening of WHO FC, deterioration by at least 15% in exercise capacity, as measured by the 6MWD, any signs or symptoms of right-sided heart failure will be collected for clinical worsening assessment.
Time Frame
Up to 6 years
Title
Medical Resource Utilization
Description
Number per year of all-cause and PAH-related hospitalizations; number per year of in-patient hospital days for all causes and PAH-related causes; number per year of emergency room visits for all causes and for PAH-related causes that do not result in hospital admittance will be collected for assessment of medical resource utilization.
Time Frame
Up to 6 years
Title
Change from Baseline in 6-minute Walk Distance (6MWD)
Description
Change from baseline in 6MWD according to non-invasive criteria will be assessed as per the low (greater than [>] 440 meters [m]), intermediate (165-440m), and high-risk category (less than [<] 165m).
Time Frame
Baseline up to 6 years
Title
Change from Baseline in World Health Organization (WHO) Functional Class (FC)
Description
Change from baseline in WHO FC according to non-invasive criteria will be assessed as per the low (I, II), intermediate (III), and high-risk category (IV).
Time Frame
Baseline up to 6 years
Title
Change from Baseline in N-terminal-pro-hormone Brain Natriuretic Peptide (NT-proBNP)
Description
Change from baseline in NT-proBNP according to non-invasive criteria will be assessed as per the low (<300 nanogram per liters [ng/l]), intermediate (300-1400ng/l), and high-risk category (>1400 ng/l).
Time Frame
Baseline up to 6 years
Title
Time to Worsening in WHO FC
Description
Time to worsening in WHO FC will be reported.
Time Frame
Up to 6 months
Title
Change from Baseline in the Number of low-risk Noninvasive Criteria Based on WHO FC, 6MWD, and NT-proBNP
Description
Change from baseline in the number of low-risk noninvasive criteria based on WHO FC, 6MWD, and NT-proBNP will be reported.
Time Frame
Baseline up to 6 years
Title
Time to all-cause Death Based on the Number of low-risk Noninvasive criteria Based on WHO FC, 6MWD, and NT-proBNP
Description
Time to all-cause death based on the number of low-risk noninvasive criteria based on WHO FC, 6MWD, and NT-proBNP will be reported.
Time Frame
Up to 6 years
Title
Change from Baseline in Number of Participants Within Each Overall Risk Category (Low, Intermediate, or High) According to the Noninvasive Criteria
Description
Change from baseline in number of participants within each overall risk category (low, intermediate, or high) according to low-risk non-invasive criteria will be assessed as per the following parameters: WHO FC- low (I, II), intermediate (III), and high-risk category (IV); 6MWD- low (> 440m), intermediate (165-440m), and high-risk category (< 165m), and <165m; and NT-proBNP- low (<300ng/l), intermediate (300-1400ng/l), and high-risk category (>1400 ng/l).
Time Frame
Baseline up to 6 years
Title
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
Description
Change from baseline in number of participants within each overall risk category (low, Intermediate, or High) according to REVEAL Lite 1 Variables will be reported. The REVEAL Lite 1 variables risk calculator determines the risk status, and the scores (ranges from 0 to 19) can be defined as: low risk as a score of less than or equal to (<=) 6, intermediate risk as a score of 7 or 8, and high risk as a score of greater than or equal to (>=) 9 for the survival rates.
Time Frame
Baseline up to 6 years
Title
Change from Baseline in Number of Participants Within Each Overall Risk Category (low, Intermediate, or High) According to REVEAL Lite 2 Variables
Description
Change from baseline in number of participants within each overall risk category (low, Intermediate, or High) according to REVEAL Lite 2 Variables will be reported. The REVEAL Lite 2 variables risk calculator determines the risk status, and the scores (ranges from 1 to 14) can be defined as: low risk as a score of <= 5, intermediate risk as a score of 6 or 7, and high risk as a score of >= 8 for the survival rates.
Time Frame
Baseline up to 6 years
Title
Time to all-cause Death Based on the Risk Category Determined by the Noninvasive Criteria
Description
Time to all-cause death based on the risk category determined by the noninvasive criteria will be reported.
Time Frame
Up to 6 years
Title
Time to all-cause Death Based on the Risk Category Determined by the REVEAL Lite 1
Description
Time to all-cause death based on the risk category determined by the REVEAL Lite 1 will be reported.
Time Frame
Up to 6 years
Title
Time to all-cause Death Based on the Risk Category Determined by the REVEAL Lite 2
Description
Time to all-cause death based on the risk category determined by the REVEAL Lite 2 will be reported.
Time Frame
Up to 6 years
Title
Risk Assessment Strategies for Clinical Worsening or Death
Description
The assessment of risk category for clinical worsening and death will be assessed by the following 3 risk assessment strategies: number of low-risk noninvasive criteria based on WHO FC, 6MWD, and NT-proBNP.
Time Frame
Up to 6 years
Title
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
Description
A modified version of the PAH-SYMPACT for Use in Clinical Practice (SYMPACT-CP), has been created for use in routine clinical practice, as an assessment to support symptom monitoring and guide treatment decisions by healthcare providers. In the SYMPACT-CP, the 24-hour recall period for the oxygen use item and the 11 symptoms items have been modified to a 1-week recall period. The SYMPACT-CP also includes 11 impact items (with a 1-week recall period). This modification was made with the intention of administering the questionnaire at a single timepoint.
Time Frame
Baseline up to 6 years
Title
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)
Description
The EQ-5D-5L is an instrument to measure health-related quality of life consisting of a descriptive system and visual analogue scale (VAS). The descriptive system comprises 5 dimensions: mobility, self-care, usual activities, pain/discomfort, and anxiety/depression. Each dimension has 5 levels of perceived problems (1 indicating no problem, 2 indicating slight problems, 3 indicating moderate problems, 4 indicating severe problems, 5 indicating extreme problems). Participant selects an answer for each of 5 dimensions considering the response that best matches his or her health "today". The responses to the 5 dimensions are used to compute a single score ranging from 0 (worst health state) to 100 (better health state) representing the general health status of the individual.
Time Frame
Baseline up to 6 years
Title
Change from Baseline in Health-Related Quality of life Status as Assessed by 36-item Short-Form Health Survey (SF-36) v2 Acute Questionnaire
Description
The SF-36 v2 acute questionnaire is a 36-item short form survey used to assess the participant's quality of life. In the SF-36 v2 Acute Questionnaire, participants are instructed to rate their health and capacity to perform activities of daily living in eight domains including physical functioning, physical role limitations, bodily pain, general health, vitality, social functioning, emotional role limitations, and mental health during the last week. The scores range from 0 (lowest or worst possible level of functioning) to 100 (highest or best possible level of functioning).
Time Frame
Baseline up to 6 years
Title
Change from Baseline in PAH-specific Medication Adherence as Assessed by Morisky Medication Adherence Scale-8 (MMAS-8) Score
Description
The MMAS-8 is commonly used in standard clinical practice and a validated measure of treatment adherence. The MMAS-8 includes 8 questions assessing the extent of adherence or nonadherence and reasons for non-adherence.
Time Frame
Baseline up to 6 years
Title
Change from Baseline in Medication Adherence Questions of each Prescribed PAH Therapy Class
Description
Change from baseline in medication adherence questions of each prescribed PAH therapy class will be reported.
Time Frame
Baseline up to 6 years
Title
Change from Baseline in PAH-specific Medication Adherence as Assessed by Pulmonary Arterial Hypertension-Symptoms and Impact (PAH-SYMPACT) Questionnaire
Description
The PAH-SYMPACT questionnaire, applicable only for participants who enrolled in the PAH-SYMPACT substudy, is a PRO instrument and composed of 2 parts: the symptoms part and the Impacts part. There is no total symptom score; the 2 domains are: cardiopulmonary symptoms and cardiovascular symptoms. The Impacts part has 2 domains: Physical Impacts Domain and Cognitive/Emotional Impacts Domain which contain 7 and 4 items, respectively. Each item has 5-point Likert response scale (0=not at all, 1=mild, 2=moderate, 3=severe, and 4=very severe). The mean individual weekly symptom item scores are aggregated by domain, with the sum then being divided by the number of symptom items in the respective domain. This leads to the average weekly domain score ranging from 0-4 for each participant with higher scores indicating greater symptom severity or worse impact.
Time Frame
Baseline up to 6 years
Title
Change from Baseline in Patient Global Assessment of Disease Severity (PGA-S) of PAH
Description
Change from baseline in PGA-S of PAH will be reported. It includes severity of PAH symptoms, such as, none, mild, moderate, severe and very severe.
Time Frame
Baseline up to 6 years
Title
PAH Symptoms and Impact using SYMPACT-CP Questionnaire
Description
PAH symptoms and impact using SYMPACT-CP questionnaire will be reported.
Time Frame
Baseline up to 6 years
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Actelion Clinical Trial
Organizational Affiliation
Actelion
Official's Role
Study Director
Facility Information:
Facility Name
Bay Area Cardiology Associates, P.A. - Brandon Office
City
Brandon
State/Province
Florida
ZIP/Postal Code
33511
Country
United States
Facility Name
University of South Florida
City
Tampa
State/Province
Florida
ZIP/Postal Code
33606
Country
United States
Facility Name
Northwestern University
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60611
Country
United States
Facility Name
University Of Iowa - Hospitals & Clinics
City
Iowa City
State/Province
Iowa
ZIP/Postal Code
52242
Country
United States
Facility Name
University of Kansas Medical Center
City
Kansas City
State/Province
Kansas
ZIP/Postal Code
66160
Country
United States
Facility Name
Kentuckiana Pulmonary Associates
City
Louisville
State/Province
Kentucky
ZIP/Postal Code
40202
Country
United States
Facility Name
University of Maryland
City
Baltimore
State/Province
Maryland
ZIP/Postal Code
21201
Country
United States
Facility Name
Tufts Medical Center
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02111-1526
Country
United States
Facility Name
University of Michigan
City
Ann Arbor
State/Province
Michigan
ZIP/Postal Code
48109-5000
Country
United States
Facility Name
Mayo Clinic - Rochester
City
Rochester
State/Province
Minnesota
ZIP/Postal Code
55905
Country
United States
Facility Name
University of Missouri
City
Columbia
State/Province
Missouri
ZIP/Postal Code
65212
Country
United States
Facility Name
University of New Mexico
City
Albuquerque
State/Province
New Mexico
ZIP/Postal Code
87131
Country
United States
Facility Name
University of Cinncinnati Physicans- UC Health Physicnas Office
City
Cincinnati
State/Province
Ohio
ZIP/Postal Code
45219
Country
United States
Facility Name
University of Pittsburgh Medical Center
City
Pittsburgh
State/Province
Pennsylvania
ZIP/Postal Code
15213
Country
United States
Facility Name
Houston Methodist Hospital
City
Houston
State/Province
Texas
ZIP/Postal Code
77030-1502
Country
United States
Facility Name
Instituto Cardiovascular de Rosario
City
Rosario
ZIP/Postal Code
S2000DSR
Country
Argentina
Facility Name
Universidade Federal De Minas Gerais - Hospital das Clínicas
City
Belo Horizonte
ZIP/Postal Code
30130-100
Country
Brazil
Facility Name
Universidade Estadual Paulista 'Julio De Mesquita Filho'
City
Botucatu
ZIP/Postal Code
18618-970
Country
Brazil
Facility Name
Irmandade Santa Casa de Misericordia de Porto Alegre
City
Porto Alegre
ZIP/Postal Code
90020-090
Country
Brazil
Facility Name
Hospital Das Clinicas Da Faculdade De Medicina Da USP
City
Sao Paulo
ZIP/Postal Code
05403-000
Country
Brazil
Facility Name
University of Alberta Hospital
City
Edmonton
State/Province
Alberta
ZIP/Postal Code
T6G 2R7
Country
Canada
Facility Name
London Health Sciences Centre - Victoria Hospital
City
London
State/Province
Ontario
ZIP/Postal Code
N6A 5W9
Country
Canada
Facility Name
Beijing Anzhen Hospital
City
Beijing
ZIP/Postal Code
100029
Country
China
Facility Name
Beijing Fuwai Hospital
City
Beijing
ZIP/Postal Code
100037
Country
China
Facility Name
Peking Union Medical College Hospital
City
Beijing
ZIP/Postal Code
100044
Country
China
Facility Name
West China Hospital, Sichuan University
City
Chengdu
ZIP/Postal Code
610041
Country
China
Facility Name
Shanghai Pulmonary Hospital
City
Shanghai
ZIP/Postal Code
200433
Country
China
Facility Name
Universitatsklinikum Bonn
City
Bonn
ZIP/Postal Code
53127
Country
Germany
Facility Name
Ospedale S.Giuseppe, Gruppo MultiMedica
City
Milano
ZIP/Postal Code
20123
Country
Italy
Facility Name
IRCCS Policlinico San Matteo, Università degli studi di Pavi
City
Pavia
ZIP/Postal Code
27100
Country
Italy
Facility Name
Universita degli Studi di Roma 'La Sapienza' - Umberto I Policlinico di Roma
City
Rome
ZIP/Postal Code
00161
Country
Italy
Facility Name
National Hospital Organization Okayama Medical Center
City
Okayama
ZIP/Postal Code
701-1192
Country
Japan
Facility Name
Pusan National University Hospital
City
Busan
ZIP/Postal Code
49241
Country
Korea, Republic of
Facility Name
Gachon University Gil Medical Center
City
Incheon
ZIP/Postal Code
21565
Country
Korea, Republic of
Facility Name
Severance Hospital, Yonsei University Health System
City
Seoul
ZIP/Postal Code
03722
Country
Korea, Republic of
Facility Name
Asan Medical Center
City
Seoul
ZIP/Postal Code
05505
Country
Korea, Republic of
Facility Name
The Catholic University of Korea, Seoul St. Mary's Hospital
City
Seoul
ZIP/Postal Code
6591
Country
Korea, Republic of
Facility Name
National Heart Institute
City
Kuala Lumpur
ZIP/Postal Code
50400
Country
Malaysia
Facility Name
Vrije Universiteit Amsterdam (VU)
City
Amsterdam
ZIP/Postal Code
1081 HV
Country
Netherlands
Facility Name
Sint Antonius Ziekenhuis
City
Nieuwegein
ZIP/Postal Code
3430 EM
Country
Netherlands
Facility Name
Erasmus MC
City
Rotterdam
ZIP/Postal Code
3015 CE
Country
Netherlands
Facility Name
Krakowski Szpital Specjalistyczny im Jana Pawla II
City
Kraków
ZIP/Postal Code
31-202
Country
Poland
Facility Name
Wojewodzki Szpital Specjalistyczny im. Stefana Kardynala Wyszynskiego SPZOZ
City
Lublin
ZIP/Postal Code
20-718
Country
Poland
Facility Name
CardioPulmonary Research, PSC
City
Guaynabo
ZIP/Postal Code
00928
Country
Puerto Rico
Facility Name
National Heart Centre (NHC) Singapore
City
Singapore
ZIP/Postal Code
169609
Country
Singapore
Facility Name
Hosp. Univ. 12 De Octubre
City
Cardiologia
ZIP/Postal Code
28041
Country
Spain
Facility Name
Hosp. Univ. Pta. de Hierro Majadahonda
City
Majadahonda
ZIP/Postal Code
28222
Country
Spain
Facility Name
Hosp. Univ. Marques de Valdecilla
City
Santander
ZIP/Postal Code
39008
Country
Spain
Facility Name
Sahlgrenska Universitetsjukhuset
City
Goeteborg
ZIP/Postal Code
413 46
Country
Sweden
Facility Name
Royal Free Hospital
City
London
ZIP/Postal Code
NW3 2QG
Country
United Kingdom
Facility Name
Hammersmith Hospital
City
London
ZIP/Postal Code
W12 0HS
Country
United Kingdom
Facility Name
Freeman Hospital
City
Newcastle upon Tyne
ZIP/Postal Code
NE7 7DN
Country
United Kingdom
Facility Name
Papworth Hospital, Cambridge University
City
Papworth Everard
ZIP/Postal Code
CB23 3RE
Country
United Kingdom
Facility Name
Sheffield Teaching Hospitals NHS Foundation Trust Royal Hallamshire Hospital
City
Sheffield
ZIP/Postal Code
S10 2JF
Country
United Kingdom
12. IPD Sharing Statement
Plan to Share IPD
Yes
IPD Sharing Plan Description
The data sharing policy of the Janssen Pharmaceutical Companies of Johnson and Johnson is available at www.janssen.com/clinical- trials/transparency. As noted on this site, requests for access to the study data can be submitted through Yale Open Data Access (YODA) project site at yoda.yale.edu
IPD Sharing URL
https://www.janssen.com/clinical-trials/transparency
Learn more about this trial
A Study of Real-world Cohort of Pulmonary Arterial Hypertension (PAH) Participants
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