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

NCT06445673 | Enrolling By Invitation

• 1 other identifier: TREV1-10P.NIS
• Study Type: observational
• Target: 110
• Locations: 9 countries
• Sites: 18

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.

Conditions

Pulmonary Arterial Hypertension

Outcome Measures

Primary Outcomes (3)

• Time to death or lung transplantation

  up to year 3

• 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

• 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 Outcomes (21)

• Long-term change in 6MWD

  up to year 3

• Long-term change in PAH treatment regimens

  up to year 3

• Long-term change in risk status

  up to year 3

• Long-term change in WHO FC

  up to year 3

• Long-term change in BDS

  up to year 3

Other Outcomes (3)

• Frequency and seriousness of adverse events (AE) and adverse drug reactions (ADR)

  up to year 3

• Long-term change in QoL using European Quality of Life 5 Dimensions 5 Level Version (EQ-5D-5L) questionnaire

  up to year 3

• Long-term change in QoL using PAH-specific emPHasis-10 questionnaire

  up to year 3

Study Arms (1)

TripleTRE NIS patients

all patients previously treated within the TripleTRE trial who are willing to participate in this NIS

Eligibility Criteria

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

You may qualify if:

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

You may not qualify if:

• Lost to follow-up patients of TripleTRE study
• Patients who discontinued all medicinal PAH treatments (e.g. after successful lung transplantation)
• 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)

Sponsors & Collaborators

• AOP Orphan Pharmaceuticals AG: lead

Study Sites (18)

Ordensklinikum Linz

Linz, Austria

Location

Medical University Vienna

Vienna, Austria

Location

Fakultní Nemocnice Olomouc

Olomouc, Czechia

Location

Všeobecná fakultní nemocnice v Praze

Prague, Czechia

Location

Hôpital Bicêtre-- Assistance Publique Hopitaux de Paris

Paris, France

Location

Hôpitaux Universitaires de Strasbourg

Strasbourg, France

Location

DRK Kliniken Berlin Westend

Berlin, Germany

Location

University Hospital Carl Gustav Carus of Technical University Dresden

Dresden, Germany

Location

Universitätsmedizin Greifswald

Greifswald, Germany

Location

Gottsegen National Cardiovascular lnstitute

Budapest, Hungary

Location

Medical University of Szeged

Szeged, Hungary

Location

Sapienza University of Rome

Rome, Italy

Location

John Paul II Hospital Krakow

Krakow, Poland

Location

Fryderyk Chopin Hospital in European Health Centre Otwock

Otwock, Poland

Location

Emergency Institute for Cardiovascular Diseases Prof. Dr. C.C.Iliescu

Bucharest, Romania

Location

Emergency Clinical County Hospital of Targu Mures

Târgu Mureş, Romania

Location

Hospital Clinic of Barcelona

Barcelona, Spain

Location

Hospital Ramon y Cajal

Madrid, Spain

Location

MeSH Terms

Conditions

Pulmonary Arterial Hypertension

Condition Hierarchy (Ancestors)

Hypertension, Pulmonary; Lung Diseases; Respiratory Tract Diseases

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 29, 2024
• First Posted: June 6, 2024
• Study Start: July 31, 2024
• Primary Completion (Estimated): June 1, 2030
• Study Completion (Estimated): June 1, 2030
• Last Updated: August 7, 2024

Record last verified: 2024-08

Data Sharing

• IPD Sharing: Will not share

Locations

DE (3); PL (2); AU (2); ES (2); IT (1); FR (2); CZ (2); RO (2); HU (2)