Pulmonary Arterial Hypertension (PAH)
Conditions
Keywords
Pulmonary Arterial Hypertension, Treprostinil, Parenteral Treprostinil, Subcutaneous Treprostinil, Observational Registry
Brief summary
TRESPECT is an international, multicenter, observational registry designed to describe the real-world use, effectiveness, and tolerability of parenteral treprostinil in adults with newly diagnosed pulmonary arterial hypertension (PAH). The registry will include patients who initiated subcutaneous or intravenous treprostinil within 12 months of PAH diagnosis. Both retrospective and prospective data will be collected from routine clinical care. Data will include patient characteristics, PAH treatment patterns, treprostinil dosing and route of administration, functional status, exercise capacity, biomarkers, echocardiographic and hemodynamic parameters, clinical events, and adverse events. Participants will be followed for at least 36 months or until a registry termination criterion is met. The registry is expected to enroll approximately 100 to 300 patients across specialized PAH centers in several European countries.
Interventions
DRUGtreprostinil
Parenteral treprostinil administered by continuous subcutaneous or intravenous infusion as part of routine clinical care. The decision to initiate treatment, route of administration, starting dose, dose titration, treatment duration, and use of concomitant PAH therapies are determined by the treating physician and are not assigned by the registry. Data on treatment initiation, doses at approximately 2 and 4 weeks, longitudinal dosing, route of administration, tolerability, local site reactions, pain, treatment changes, and clinical outcomes are collected during registry follow-up.
Sponsors
AOP Orphan Pharmaceuticals AG
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Adult patient (≥18 years) * Confirmed diagnosis of PAH (i.e. Group 1 as defined by ESC/ERS guidelines) * Initiated on parenteral Treprostinil (SC or IV) anytime from 1/1/2024 * Informed consent (prospective data or retrospective chart review, per local regulations)
Exclusion criteria
* Participation in any interventional trials affecting PAH (including patients from TripleTRE study) * Non-PAH forms of pulmonary hypertension (e.g., ESC-ERS Group 2-5)
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Mean Change From Baseline in 6-Minute Walk Distance at Month 12 | Baseline and Month 12 | Mean change in 6-minute walk distance from baseline to Month 12. The 6-minute walk distance is measured in meters. A positive change indicates improvement in exercise capacity. |
| Number and Percentage of Participants by Change in New York Heart Association Functional Class at Month 12 | Baseline and Month 12 | Number and percentage of participants whose New York Heart Association Functional Class improved, remained unchanged, or worsened from baseline to Month 12. Functional Class ranges from Class I to Class IV, with a higher class indicating greater functional limitation. |
| Number and Percentage of Participants With Local Administration-Site Reactions During the First 12 Months | From baseline through Month 12 | Number and percentage of participants experiencing at least one local administration-site reaction associated with subcutaneous or intravenous treprostinil application during the first 12 months after treatment initiation. |
| Mean Local Pain Score During the First 12 Months | Baseline, Week 2, Week 4, Month 3, Month 6, Month 9, and Month 12 | Local pain associated with parenteral treprostinil application will be assessed on a numerical rating scale from 0 to 10. Higher scores indicate greater pain. |
Outcome results
None listed