Clinical trial NCT06445673: A Prospective Non-interventional Observational Study to Observe Long-term Treatment and Outcomes in Pulmonary Arterial Hypertension (PAH) Patients
Official title: A Prospective, Multicenter, International Non-interventional, Observational Cohort Study to Observe Long-term Treatment Patterns and Real-word Outcomes in PAH Group I Patients Previously Participating in TripleTRE - TripleTRE-NIS
Study type: OBSERVATIONAL
Overall status: ENROLLING_BY_INVITATION
Conditions: Pulmonary Arterial Hypertension
Lead sponsor: AOP Orphan Pharmaceuticals AG
Sponsor class: INDUSTRY
First posted: 2024-06-06
Enrollment: 110
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: The study population will consist of patients who participated in the TripleTRE study and who have not been lost to follow-up and have given a written informed consent for their participation in the NIS.

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.


Eligibility criteria: 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)

Primary outcomes:
measure: Time to death or lung transplantation
timeFrame: up to year 3
measure: Time to first clinical worsening
timeFrame: up to year 3
measure: Total number of clinical worsenings
timeFrame: up to year 3
Secondary outcomes:
measure: Long-term change in 6MWD
timeFrame: up to year 3
measure: Long-term change in PAH treatment regimens
timeFrame: up to year 3
measure: Long-term change in risk status
timeFrame: up to year 3
measure: Long-term change in WHO FC
timeFrame: up to year 3
measure: Long-term change in BDS
timeFrame: up to year 3
measure: Long-term change in NT-proBNP/BNP
timeFrame: up to year 3
measure: Long-term change in Pulmonary Vascular Resistance (PVR)
timeFrame: up to year 3
measure: Long-term change in mean Pulmonary Arterial Pressure (mPAP)
timeFrame: up to year 3
measure: Long-term change in Right Atrial Pressure (RAP)
timeFrame: up to year 3
measure: Long-term change in mean Right Atrial Pressure (mRAP)
timeFrame: up to year 3
measure: Long-term change in Cardiac Index (CI)
timeFrame: up to year 3
measure: Long-term change in Cardiac Output (CO)
timeFrame: up to year 3
measure: Long-term change in Pulmonary capillary wedge pressure (PCWP)
timeFrame: up to year 3
measure: Long-term change in Stroke volume index (SVI)
timeFrame: up to year 3
measure: Long-term change in RV-PA coupling
timeFrame: up to year 3
measure: Long-term change in RV end-diastolic area (RVEDA)
timeFrame: up to year 3
measure: Long-term change in RV end-systolic area (RVESA)
timeFrame: up to year 3
measure: Long-term change in RV fractional area change (RVFAC)
timeFrame: up to year 3
measure: Long-term change in Right Atrium (RA) area
timeFrame: up to year 3
measure: Long-term change in Pericardial effusion
timeFrame: up to year 3
measure: Long-term change in Right ventricular ejection fraction (RVEF)
timeFrame: up to year 3
Other outcomes:
measure: Frequency and seriousness of adverse events (AE) and adverse drug reactions (ADR)
timeFrame: up to year 3
measure: Long-term change in QoL using European Quality of Life 5 Dimensions 5 Level Version (EQ-5D-5L) questionnaire
timeFrame: up to year 3
measure: Long-term change in QoL using PAH-specific emPHasis-10 questionnaire
timeFrame: up to year 3
Locations:
city: Linz
country: Austria
facility: Ordensklinikum Linz
city: Vienna
country: Austria
facility: Medical University Vienna
city: Olomouc
country: Czechia
facility: Fakultní Nemocnice Olomouc
city: Praha
country: Czechia
facility: Všeobecná fakultní nemocnice v Praze
city: Paris
country: France
facility: Hôpital Bicêtre-- Assistance Publique Hopitaux de Paris
city: Strasbourg
country: France
facility: Hôpitaux Universitaires de Strasbourg
city: Berlin
country: Germany
facility: DRK Kliniken Berlin Westend
city: Dresden
country: Germany
facility: University Hospital Carl Gustav Carus of Technical University Dresden
city: Greifswald
country: Germany
facility: Universitätsmedizin Greifswald
city: Budapest
country: Hungary
facility: Gottsegen National Cardiovascular lnstitute
city: Szeged
country: Hungary
facility: Medical University of Szeged
city: Rome
country: Italy
facility: Sapienza University of Rome
city: Kraków
country: Poland
facility: John Paul II Hospital Krakow
city: Otwock
country: Poland
facility: Fryderyk Chopin Hospital in European Health Centre Otwock
city: Bucharest
country: Romania
facility: Emergency Institute for Cardiovascular Diseases Prof. Dr. C.C.Iliescu
city: Târgu-Mureş
country: Romania
facility: Emergency Clinical County Hospital of Targu Mures
city: Barcelona
country: Spain
facility: Hospital Clinic of Barcelona
city: Madrid
country: Spain
facility: Hospital Ramon y Cajal
Study officials:
References: