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

Pulmonary Arterial Hypertension Clinical Trial

— TripleTRE-NIS  

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

Clinical Trial Details — Status: Enrolling by invitation

Administrative data

• NCT number: NCT06445673
• Other study ID #: TREV1-10P.NIS
• Status: Enrolling by invitation
• Start date: June 2024
• Est. completion date: June 2030

Study information

• Verified date: June 2024
• Source: AOP Orphan Pharmaceuticals AG
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

Recruitment information / eligibility

• Status: Enrolling by invitation
• Enrollment: 110
• Est. completion date: June 2030
• Est. primary completion date: June 2030
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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)

Related Conditions & MeSH terms

• Familial Primary Pulmonary Hypertension
• Hypertension
• Pulmonary Arterial Hypertension

Locations

Country	Name	City	State
Austria	Ordensklinikum Linz	Linz
Austria	Medical University Vienna	Vienna
Czechia	Fakultní Nemocnice Olomouc	Olomouc
Czechia	Všeobecná fakultní nemocnice v Praze	Praha
France	Hôpital Bicêtre-- Assistance Publique Hopitaux de Paris	Paris
France	Hôpitaux Universitaires de Strasbourg	Strasbourg
Germany	DRK Kliniken Berlin Westend	Berlin
Germany	University Hospital Carl Gustav Carus of Technical University Dresden	Dresden
Germany	Universitätsmedizin Greifswald	Greifswald
Hungary	Gottsegen National Cardiovascular lnstitute	Budapest
Hungary	Medical University of Szeged	Szeged
Italy	Sapienza University of Rome	Rome
Poland	John Paul II Hospital Krakow	Kraków
Poland	Fryderyk Chopin Hospital in European Health Centre Otwock	Otwock
Romania	Emergency Institute for Cardiovascular Diseases Prof. Dr. C.C.Iliescu	Bucharest
Romania	Emergency Clinical County Hospital of Targu Mures	Târgu-Mures
Spain	Hospital Clinic of Barcelona	Barcelona
Spain	Hospital Ramon y Cajal	Madrid

Sponsors (1)

Lead Sponsor	Collaborator
AOP Orphan Pharmaceuticals AG

Countries where clinical trial is conducted

Austria,  Czechia,  France,  Germany,  Hungary,  Italy,  Poland,  Romania,  Spain, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Other	Frequency and seriousness of adverse events (AE) and adverse drug reactions (ADR)	This includes: Adverse events (S)AEs; Adverse drug reactions (S)ADRs related to any of PAH treatments; Adverse events related to underlying diagnosis, PAH	up to year 3
Other	Long-term change in QoL using European Quality of Life 5 Dimensions 5 Level Version (EQ-5D-5L) questionnaire	The EQ-5D-5L questionnaire consists of 2 parts - the EQ-5D-5L descriptive system and the EQ Visual Analogue scale. The descriptive system comprises 5 dimensions (mobility, self-care, usual activities, pain/discomfort, anxiety/depression). Each dimension has 5 levels: no problems, slight problems, moderate problems, severe problems and extreme problems. The patient is asked to indicate his/her health state by ticking (or placing a cross) in the box against the most appropriate statement in each of the 5 dimensions. This decision results in a 1-digit number expressing the level selected for that dimension. The digits for 5 dimensions can be combined in a 5-digit number describing the respondent's health state. It should be noted that the numerals 1-5 have no arithmetic properties and should not be used as a cardinal score.	up to year 3
Other	Long-term change in QoL using PAH-specific emPHasis-10 questionnaire	Emphasis meaning something of special importance or significance. Please translate using the most appropriate term. The PH in emPHasis represents the condition Pulmonary Hypertension. The number 10 refers to the number of items in the questionnaire.; This questionnaire is designed to determine how Pulmonary Hypertension (PH) affects patient's life. It refers to how PH affects or the impact that PH has on the patient's life.	up to year 3
Primary	Time to death or lung transplantation		up to year 3
Primary	Time to first clinical worsening	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	up to year 3
Primary	Total number of clinical worsenings	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	up to year 3
Secondary	Long-term change in 6MWD	6-Minute Walking Distance (6MWD) will be measured in meters	up to year 3
Secondary	Long-term change in PAH treatment regimens	any change or addition of new PH medication including dosing in time	up to year 3
Secondary	Long-term change in risk status	Risk status measured by simplified four-strata risk-assessment tool	up to year 3
Secondary	Long-term change in WHO FC	World Health Organization Functional Class (WHO FC) is categorized from I (no symptoms) to IV (severe dyspnea and symptoms)	up to year 3
Secondary	Long-term change in BDS	BDS - Borg Dyspnea Score, ranging from 0 (no exertion) to 10 (maximum)	up to year 3
Secondary	Long-term change in NT-proBNP/BNP	NT-proBNP/BNP: N-terminal pro Brain Natriuretic Peptide	up to year 3
Secondary	Long-term change in Pulmonary Vascular Resistance (PVR)	PVR to be measured in WU Parameter will be collected according to clinical routine	up to year 3
Secondary	Long-term change in mean Pulmonary Arterial Pressure (mPAP)	mPAP to be measured in mmHg Parameter will be collected according to clinical routine	up to year 3
Secondary	Long-term change in Right Atrial Pressure (RAP)	RAP to be measured in mmHg Parameter will be collected according to clinical routine	up to year 3
Secondary	Long-term change in mean Right Atrial Pressure (mRAP)	mean Right Atrial Pressure (mRAP) to be measured in mmHg Parameter will be collected according to clinical routine	up to year 3
Secondary	Long-term change in Cardiac Index (CI)	Cardiac index (CI) measured in liters per minute per square meter Parameter will be collected according to clinical routine	up to year 3
Secondary	Long-term change in Cardiac Output (CO)	Cardiac output (CO) measured in liters per minute Parameter will be collected according to clinical routine	up to year 3
Secondary	Long-term change in Pulmonary capillary wedge pressure (PCWP)	Pulmonary capillary wedge pressure (PCWP) measured in mmHg Parameter will be collected according to clinical routine	up to year 3
Secondary	Long-term change in Stroke volume index (SVI)	Parameter will be collected according to clinical routine	up to year 3
Secondary	Long-term change in RV-PA coupling	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	up to year 3
Secondary	Long-term change in RV end-diastolic area (RVEDA)	RV end-diastolic area (RVEDA) measured in square centimeters Parameter will be collected according to clinical routine	up to year 3
Secondary	Long-term change in RV end-systolic area (RVESA)	RV end-systolic area (RVESA) measured in square centimeters Parameter will be collected according to clinical routine	up to year 3
Secondary	Long-term change in RV fractional area change (RVFAC)	RV fractional area change (RVFAC) calculated in % Parameter will be collected according to clinical routine	up to year 3
Secondary	Long-term change in Right Atrium (RA) area	Right Atrium (RA) area in square centimeters Parameter will be collected according to clinical routine	up to year 3
Secondary	Long-term change in Pericardial effusion	Pericardial effusion assessment will be done and rated as yes/no Parameter will be collected according to clinical routine	up to year 3
Secondary	Long-term change in Right ventricular ejection fraction (RVEF)	Parameter will be collected according to clinical routine	up to year 3