A Study of Real-world Cohort of Pulmonary Arterial Hypertension (PAH) Participants

NCT04955990 | Terminated Phase 4

• 2 other identifiers: CR10900767896062PAH4005
• Study Type: interventional
• Target: 232
• Locations: 17 countries
• Sites: 54

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.

Conditions

Pulmonary Arterial Hypertension

Outcome Measures

Primary Outcomes (2)

• Time to all Cause Death

  Time to all cause death will be reported. All-cause death defined as deaths due to any cause.

  Up to 6 years

• 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.

  Up to 6 years

Secondary Outcomes (26)

• Time to Death due to PAH

  Up to 6 years

• Time to First all-cause Hospitalization

  Up to 6 years

• Time to First Morbidity/Mortality Event

  Up to 6 years

• Time to Clinical Worsening

  Up to 6 years

• Medical Resource Utilization

  Up to 6 years

Study Arms (1)

Participants with PAH

EXPERIMENTAL

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.

Other: PAH Therapies

Interventions

PAH Therapies OTHER

No Drug will be administered as part of this study. Real world data will be collected in participants who newly initiate PAH therapies in routine clinical setting or have already been receiving macitentan 10 mg for at least 3 months prior to the index date.

Participants with PAH

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Actelion: lead

Study Sites (54)

Bay Area Cardiology Associates, P.A. - Brandon Office

Brandon, Florida, 33511, United States

Location

University of South Florida

Tampa, Florida, 33606, United States

Location

Northwestern University

Chicago, Illinois, 60611, United States

Location

University Of Iowa - Hospitals & Clinics

Iowa City, Iowa, 52242, United States

Location

University of Kansas Medical Center

Kansas City, Kansas, 66160, United States

Location

Kentuckiana Pulmonary Associates

Louisville, Kentucky, 40202, United States

Location

University of Maryland

Baltimore, Maryland, 21201, United States

Location

Tufts Medical Center

Boston, Massachusetts, 02111-1526, United States

Location

University of Michigan

Ann Arbor, Michigan, 48109-5000, United States

Location

Mayo Clinic - Rochester

Rochester, Minnesota, 55905, United States

Location

University of Missouri

Columbia, Missouri, 65212, United States

Location

University of New Mexico

Albuquerque, New Mexico, 87131, United States

Location

University of Cinncinnati Physicans- UC Health Physicnas Office

Cincinnati, Ohio, 45219, United States

Location

University of Pittsburgh Medical Center

Pittsburgh, Pennsylvania, 15213, United States

Location

Houston Methodist Hospital

Houston, Texas, 77030-1502, United States

Location

Instituto Cardiovascular de Rosario

Rosario, S2000DSR, Argentina

Location

Universidade Federal De Minas Gerais - Hospital das Clínicas

Belo Horizonte, 30130-100, Brazil

Location

Universidade Estadual Paulista 'Julio De Mesquita Filho'

Botucatu, 18618-970, Brazil

Location

Irmandade Santa Casa de Misericordia de Porto Alegre

Porto Alegre, 90020-090, Brazil

Location

Hospital Das Clinicas Da Faculdade De Medicina Da USP

São Paulo, 05403 900, Brazil

Location

University of Alberta Hospital

Edmonton, Alberta, T6G 2R7, Canada

Location

London Health Sciences Centre Victoria Hospital

London, Ontario, N6A 5W9, Canada

Location

Beijing Anzhen Hospital

Beijing, 100029, China

Location

Beijing Fuwai Hospital

Beijing, 100037, China

Location

Peking Union Medical College Hospital

Beijing, 100044, China

Location

West China Hospital Sichuan University

Chengdu, 610041, China

Location

Shanghai Pulmonary Hospital

Shanghai, 200433, China

Location

Universitatsklinikum Bonn

Bonn, 53127, Germany

Location

Ospedale S.Giuseppe, Gruppo MultiMedica

Milan, 20123, Italy

Location

IRCCS Policlinico San Matteo, Università degli studi di Pavi

Pavia, 27100, Italy

Location

Universita degli Studi di Roma 'La Sapienza' - Umberto I Policlinico di Roma

Rome, 00161, Italy

Location

National Hospital Organization Okayama Medical Center

Okayama, 701-1192, Japan

Location

National Heart Institute

Kuala Lumpur, 50400, Malaysia

Location

Vrije Universiteit Amsterdam (VU)

Amsterdam, 1081 HV, Netherlands

Location

Sint Antonius Ziekenhuis

Nieuwegein, 3430 EM, Netherlands

Location

Erasmus MC

Rotterdam, 3015 CE, Netherlands

Location

Krakowski Szpital Specjalistyczny im Jana Pawla II

Krakow, 31 202, Poland

Location

Wojewodzki Szpital Specjalistyczny im Stefana Kardynala Wyszynskiego SPZOZ

Lublin, 20 718, Poland

Location

CardioPulmonary Research, PSC

Guaynabo, 00928, Puerto Rico

Location

National Heart Centre (NHC) Singapore

Singapore, 169609, Singapore

Location

Pusan National University Hospital

Busan, 49241, South Korea

Location

Gachon University Gil Medical Center

Incheon, 21565, South Korea

Location

Severance Hospital Yonsei University Health System

Seoul, 03722, South Korea

Location

Asan Medical Center

Seoul, 05505, South Korea

Location

The Catholic University of Korea Seoul St Mary s Hospital

Seoul, 6591, South Korea

Location

Hosp. Univ. 12 de Octubre

Madrid, 28041, Spain

Location

Hosp. Univ. Pta. de Hierro Majadahonda

Majadahonda, 28222, Spain

Location

Hosp. Univ. Marques de Valdecilla

Santander, 39008, Spain

Location

Sahlgrenska Universitetsjukhuset

Gothenburg, 413 46, Sweden

Location

Royal Free Hospital

London, NW3 2QG, United Kingdom

Location

Hammersmith Hospital

London, W12 0HS, United Kingdom

Location

Freeman Hospital

Newcastle upon Tyne, NE7 7DN, United Kingdom

Location

Papworth Hospital, Cambridge University

Papworth Everard, CB23 3RE, United Kingdom

Location

Sheffield Teaching Hospitals NHS Foundation Trust Royal Hallamshire Hospital

Sheffield, S10 2JF, United Kingdom

Location

MeSH Terms

Conditions

Pulmonary Arterial Hypertension

Condition Hierarchy (Ancestors)

Hypertension, Pulmonary; Lung Diseases; Respiratory Tract Diseases

Study Officials

• Actelion Clinical Trial

  Actelion

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NA
• Masking: NONE
• Purpose: OTHER
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 29, 2021
• First Posted: July 9, 2021
• Study Start: October 14, 2021
• Primary Completion: December 20, 2022
• Study Completion: December 20, 2022
• Last Updated: February 3, 2025

Record last verified: 2025-01

Data Sharing

• IPD Sharing: Will share

Locations

KR (5); CA (2); JP (1); PR (1); ES (3); CN (5); PL (2); AR (1); MY (1); DE (1); US (15); IT (3); SG (1); BR (4); GB (5); NL (3); SE (1)