NCT04955990TerminatedPhase 4
An International, Non-Drug Interventional, Real-world Cohort of PAH Patients Newly Initiating PAH Therapy With Guideline-directed Assessments of Disease Severity
Actelion107 sites in 17 countries232 target enrollmentStarted: October 14, 2021Last updated:
Conditions
Trial Snapshot
- Phase
- Phase 4
- Status
- Terminated
- Sponsor
- Enrollment
- 232
- Locations
- 107
- Primary Endpoint
- Time to all Cause Death
Study Overview
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.
Study Design
- Study Type
- Interventional
- Allocation
- Na
- Intervention Model
- Single Group
- Primary Purpose
- Other
- Masking
- None
Eligibility Criteria
- Ages
- 18 Years to — (Adult, Older Adult)
- Sex
- All
- Accepts Healthy Volunteers
- No
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
Outcomes
Primary Outcomes
Time to all Cause Death
Time Frame: Up to 6 years
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 Frame: Up to 6 years
Time to death due to PAH or first hospitalization due to PAH will be reported.
Secondary Outcomes
- Time to First Morbidity/Mortality Event(Up to 6 years)
- Time to First all-cause Hospitalization(Up to 6 years)
- Change from Baseline in 6-minute Walk Distance (6MWD)(Baseline up to 6 years)
- Time to Worsening in WHO FC(Up to 6 months)
- Time to Death due to PAH(Up to 6 years)
- Time to Clinical Worsening(Up to 6 years)
- Medical Resource Utilization(Up to 6 years)
- Change from Baseline in World Health Organization (WHO) Functional Class (FC)(Baseline up to 6 years)
- Risk Assessment Strategies for Clinical Worsening or Death(Up to 6 years)
- 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(Baseline up to 6 years)
- Change from Baseline in N-terminal-pro-hormone Brain Natriuretic Peptide (NT-proBNP)(Baseline up to 6 years)
- Change from Baseline in the Number of low-risk Noninvasive Criteria Based on WHO FC, 6MWD, and NT-proBNP(Baseline up to 6 years)
- 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(Baseline up to 6 years)
- Change from Baseline in Number of Participants Within Each Overall Risk Category (Low, Intermediate, or High) According to the Noninvasive Criteria(Baseline up to 6 years)
- Time to all-cause Death Based on the Risk Category Determined by the REVEAL Lite 1(Up to 6 years)
- Change from Baseline in Medication Adherence Questions of each Prescribed PAH Therapy Class(Baseline up to 6 years)
- Time to all-cause Death Based on the Number of low-risk Noninvasive criteria Based on WHO FC, 6MWD, and NT-proBNP(Up to 6 years)
- Time to all-cause Death Based on the Risk Category Determined by the Noninvasive Criteria(Up to 6 years)
- Change from Baseline in Health-Related Quality of life Status as Assessed by 36-item Short-Form Health Survey (SF-36) v2 Acute Questionnaire(Baseline up to 6 years)
- Change from Baseline in PAH-specific Medication Adherence as Assessed by Pulmonary Arterial Hypertension-Symptoms and Impact (PAH-SYMPACT) Questionnaire(Baseline up to 6 years)
- Change from Baseline in Patient Global Assessment of Disease Severity (PGA-S) of PAH(Baseline up to 6 years)
- Time to all-cause Death Based on the Risk Category Determined by the REVEAL Lite 2(Up to 6 years)
- Change from Baseline in PAH-specific Medication Adherence as Assessed by Morisky Medication Adherence Scale-8 (MMAS-8) Score(Baseline up to 6 years)
- PAH Symptoms and Impact using SYMPACT-CP Questionnaire(Baseline up to 6 years)
- Change from Baseline in Number of Participants Within Each Overall Risk Category (low, Intermediate, or High) According to REVEAL Lite 2 Variables(Baseline up to 6 years)
- 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)(Baseline up to 6 years)
Investigators
Study Sites (107)
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