A Prospective Non-interventional Observational Study to Observe Long-term Treatment and Outcomes in Pulmonary Arterial Hypertension (PAH) Patients

• Conditions: Pulmonary Arterial Hypertension

• Registration Number: NCT06445673
• Lead Sponsor: AOP Orphan Pharmaceuticals AG

Brief Summary

The goal of this observational study is to learn about the long-term development and outcomes of different treatment patterns of patients who initially participated in the TripleTRE study.

The primary objective of this non-interventional follow-up study is to assess the long-term real-world clinical outcomes, including disease progression and survival rates, in patients who initially participated in and completed the randomized TripleTRE trial.

Planned observation duration per patient is a minimum of 3 years.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2024-05-29
• Study First Submitted QC: 2024-06-04
• Study First Posted: 2024-06-06
• Study Start: 2024-07-31
• Status Verified: 2024-08
• Last Update Submitted: 2024-08-06
• Last Update Posted: 2024-08-07
• Primary Completion: 2030-06
• Study Completion: 2030-06

Recruitment & Eligibility

• Status: ENROLLING_BY_INVITATION
• Sex: All
• Target Recruitment: 110

Inclusion Criteria

1. Patients previously participating in the randomized TripleTRE trial who are able and willing to provide a signed informed consent for study participation in NIS

Exclusion Criteria

1. Lost to follow-up patients of TripleTRE study
2. Patients who discontinued all medicinal PAH treatments (e.g. after successful lung transplantation)
3. Patients who withdrew from the initial TripleTRE trial due to significant non-compliance with trial requirements (not adhering to therapy, not coming to hospital visits)

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Time to death or lung transplantation	up to year 3
Time to first clinical worsening	up to year 3	clinical worsening is defined as fulfilling on of the following criteria: * PAH related death (including all deaths where PAH cannot be excluded as cause); * Lung transplantation due to PAH; * PAH-related hospitalization; * Post baseline decrease in 6MWD by 15%; * Post baseline worsening of WHO FC
Total number of clinical worsenings	up to year 3	clinical worsening is defined as fulfilling on of the following criteria: * PAH related death (including all deaths where PAH cannot be excluded as cause); * Lung transplantation due to PAH; * PH-related hospitalization; * Post baseline decrease in 6MWD by 15%; * Post baseline worsening of WHO FC

Secondary Outcome Measures

Name	Time	Method
Long-term change in WHO FC	up to year 3	World Health Organization Functional Class (WHO FC) is categorized from I (no symptoms) to IV (severe dyspnea and symptoms)
Long-term change in BDS	up to year 3	BDS - Borg Dyspnea Score, ranging from 0 (no exertion) to 10 (maximum)
Long-term change in Cardiac Output (CO)	up to year 3	Cardiac output (CO) measured in liters per minute Parameter will be collected according to clinical routine
Long-term change in RV-PA coupling	up to year 3	RV-PA coupling estimated by the ratio of tricuspid annular plane systolic excursion by pulmonary artery systolic pressure Parameter will be collected according to clinical routine
Long-term change in RV end-systolic area (RVESA)	up to year 3	RV end-systolic area (RVESA) measured in square centimeters Parameter will be collected according to clinical routine
Long-term change in 6MWD	up to year 3	6-Minute Walking Distance (6MWD) will be measured in meters
Long-term change in PAH treatment regimens	up to year 3	any change or addition of new PH medication including dosing in time
Long-term change in risk status	up to year 3	Risk status measured by simplified four-strata risk-assessment tool
Long-term change in NT-proBNP/BNP	up to year 3	NT-proBNP/BNP: N-terminal pro Brain Natriuretic Peptide
Long-term change in Pulmonary Vascular Resistance (PVR)	up to year 3	PVR to be measured in WU Parameter will be collected according to clinical routine
Long-term change in Stroke volume index (SVI)	up to year 3	Parameter will be collected according to clinical routine
Long-term change in RV end-diastolic area (RVEDA)	up to year 3	RV end-diastolic area (RVEDA) measured in square centimeters Parameter will be collected according to clinical routine
Long-term change in mean Pulmonary Arterial Pressure (mPAP)	up to year 3	mPAP to be measured in mmHg Parameter will be collected according to clinical routine
Long-term change in Right Atrial Pressure (RAP)	up to year 3	RAP to be measured in mmHg Parameter will be collected according to clinical routine
Long-term change in mean Right Atrial Pressure (mRAP)	up to year 3	mean Right Atrial Pressure (mRAP) to be measured in mmHg Parameter will be collected according to clinical routine
Long-term change in Cardiac Index (CI)	up to year 3	Cardiac index (CI) measured in liters per minute per square meter Parameter will be collected according to clinical routine
Long-term change in Pulmonary capillary wedge pressure (PCWP)	up to year 3	Pulmonary capillary wedge pressure (PCWP) measured in mmHg Parameter will be collected according to clinical routine
Long-term change in RV fractional area change (RVFAC)	up to year 3	RV fractional area change (RVFAC) calculated in % Parameter will be collected according to clinical routine
Long-term change in Right ventricular ejection fraction (RVEF)	up to year 3	Parameter will be collected according to clinical routine
Long-term change in Right Atrium (RA) area	up to year 3	Right Atrium (RA) area in square centimeters Parameter will be collected according to clinical routine
Long-term change in Pericardial effusion	up to year 3	Pericardial effusion assessment will be done and rated as yes/no Parameter will be collected according to clinical routine

Trial Locations

Locations (18)

Hôpitaux Universitaires de Strasbourg; 🇫🇷 Strasbourg, France

Ordensklinikum Linz; 🇦🇹 Linz, Austria

Medical University Vienna; 🇦🇹 Vienna, Austria

Fakultní Nemocnice Olomouc; 🇨🇿 Olomouc, Czechia

Všeobecná fakultní nemocnice v Praze; 🇨🇿 Praha, Czechia

Hôpital Bicêtre-- Assistance Publique Hopitaux de Paris; 🇫🇷 Paris, France

DRK Kliniken Berlin Westend; 🇩🇪 Berlin, Germany

University Hospital Carl Gustav Carus of Technical University Dresden; 🇩🇪 Dresden, Germany

Gottsegen National Cardiovascular lnstitute; 🇭🇺 Budapest, Hungary

Universitätsmedizin Greifswald; 🇩🇪 Greifswald, Germany

Sapienza University of Rome; 🇮🇹 Rome, Italy

Medical University of Szeged; 🇭🇺 Szeged, Hungary

John Paul II Hospital Krakow; 🇵🇱 Kraków, Poland

Fryderyk Chopin Hospital in European Health Centre Otwock; 🇵🇱 Otwock, Poland

Hospital Clinic of Barcelona; 🇪🇸 Barcelona, Spain

Hospital Ramon y Cajal; 🇪🇸 Madrid, Spain

Emergency Institute for Cardiovascular Diseases Prof. Dr. C.C.Iliescu; 🇷🇴 Bucharest, Romania

Emergency Clinical County Hospital of Targu Mures; 🇷🇴 Târgu-Mureş, Romania