An international, non-drug interventional, real-world cohort of PAH patients newly initiating PAH therapy with guideline-directed assessments of disease severity

Completed

• Conditions: Increased bloodpressure in the arteries of the lung; Pulmonary arterial hypertension; 10019280; 10038716

• Registration Number: NL-OMON52230
• Lead Sponsor: Actelion Pharmaceuticals

Brief Summary

Trial ended prematurely

Detailed Description

Not available

Study Timeline

• Results First Posted: 2023-10-06
• Last Update Posted: 23 April 2024

Recruitment & Eligibility

• Status: Completed
• Sex: Not specified
• Target Recruitment: 12

Inclusion Criteria

1. Signed ICF.
2. Male or female participants age >=18 years.
3. Symptomatic PAH in any PAH subtype.
4. 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 >20 mmHg, AND
b) Pulmonary artery wedge pressure or left ventricular end diastolic pressure
<=15 mmHg, AND
c) Pulmonary vascular resistance >= 3 Wood Units (ie, >=240 dyn*sec/cm5).
5. Newly initiating 1 or more PAH therapy(ies) (as monotherapy or add-on
therapy) at index date.
6. All mandated assessments must be performed and recorded at the baseline
visit before the initiation of the new PAH therapy.
7. Participants must agree to follow study protocol, including all mandated
visits and mandated assessments.
8. For the PAH-SYMPACT substudy only: Participants initiating any endothelin
receptor antagonist (ERA) or phosphodiesterase-5 inhibitor therapies at index
date 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.

Exclusion Criteria

1. Participants enrolled in any interventional clinical trial with an
investigational therapy in the 3-month period prior to index date.
2. Any PAH therapy initiated at index date was used by the participant
(including different route of administration of the same compound) within 3
months prior to index date for any reason. (Administration for vasoreactivity
testing is permitted.)
3. presence or known presence of moderate or severe obstructive lung disease
4. presence or known presence of moderate or severe restrictive lung disease

Study & Design

• Study Type: Observational invasive
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
<p>The primary objectives of this study are:<br /><br>• To describe the time to all-cause death in the overall study population and<br /><br>within each cohort (see the Cohort Definitions section).<br /><br>• To describe the time to death due to PAH or first hospitalization due to PAH<br /><br>in the overall study population and within each cohort.</p><br>