N/A
N=13
A Study to Assess the Safety and Efficacy of Treprostinil to Facilitate Liver Transplantation in Patients With Portopulmonary Hypertension
Portopulmonary Hypertension · Pulmonary Arterial Hypertension · Pulmonary Hypertension
Bottom Line
View on ClinicalTrials.gov: NCT01028651 ↗Enrolled (actual)
13
Serious AEs
61.5%
Results posted
Feb 2017
Primary outcome: Primary: Number of Subjects Who Achieved Hemodynamic Parameters Appropriate for Orthotopic Liver Transplantation Candidacy at Week 24. — 1; 12 participants
Study Design & Population
- Study type
- Observational
- Phase
- N/A
- Interventions
- Treprostinil (Drug)
- Age
- Pediatric, Adult, Older Adult
- Sex
- All
- Sponsor
- United Therapeutics
- Primary completion
- Apr 2013
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Subjects Who Achieved Hemodynamic Parameters Appropriate for Orthotopic Liver Transplantation Candidacy at Week 24. |
1; 12 | — |
| SECONDARY Change in Hemodynamic Parameters (Via Right Heart Catheterization [RHC]) at Rest From Baseline to Week 24 |
-3.8; -2.0; -4.0; -2.0; -0.7; 7.0 | — |
| SECONDARY Change in Heart Rate at Rest From Baseline to Week 24 |
0.5 | — |
| SECONDARY Change in Cardiac Output at Rest From Baseline to Week 24 |
0.3 | — |
| SECONDARY Change in Arterial and Venous Oxygen Saturation at Rest From Baseline to Week 24 |
-2.0; 0.5 | — |
| SECONDARY Change in Pulmonary Vascular Resistance (PVR) at Rest From Baseline to Week 24 |
-0.3 | — |
| SECONDARY Change in 6-minute Walk Distance (6MWD) From Baseline to Weeks 12 and 24. |
54.5; 12.0 | — |
| SECONDARY Change in Echocardiogram Parameters (Right Atrium and Right Ventricle Area) From Baseline to Weeks 12 and 24 |
-0.1; -1.7; -1.0; -7.4 | — |
| SECONDARY Change in Echocardiogram Parameters (Right Ventricle Diameter) From Baseline to Weeks 12 and 24 |
-5.0; 3.0 | — |
| SECONDARY Change in Echocardiogram Parameters (Right Ventricular Systolic Pressure) From Baseline to Weeks 12 and 24 |
-12; -5.0 | — |
| SECONDARY Change in Echocardiogram Parameters (Tricuspid Annular Plane Systolic Excursion) From Baseline to Weeks 12 and 24 |
0.5; 0.1 | — |
| SECONDARY Change in Quality of Life From Baseline to Weeks 12 and 24 |
5.7; 10.5; 7.9; 9.0; 3.6; 6.0 | — |
| SECONDARY Change in Plasma Brain N-terminal Pro-brain Natriuretic Peptide (NT-proBNP) From Baseline to Weeks 12 and 24 |
-628; -478 | — |
Summary
This was a multicenter, prospective, observational, open-label study. Patients meeting inclusion/exclusion criteria received treatment with treprostinil as recommended by their treating physicians and were followed according to standard of care. This observational study collected clinical data and biologic specimens from patients who were treated for portopulmonary hypertension (PoPH), with a goal of achieving hemodynamic parameters appropriate for orthotopic liver transplantation candidacy, including mean pulmonary arterial pressure (mPAP) less than 35 mmHg and pulmonary vascular resistance (PVR) less than 3 Wood-units (WU) at Week 24 in patients with severe PoPH.
Eligibility Criteria
Inclusion Criteria
- Patients must:
- Had portal hypertension.
- Be otherwise suitable candidates for OLT.
- Had severe pulmonary arterial hypertension (PAH) defined as a resting mean pulmonary arterial pressure (mPAP) >35 mmHg and pulmonary vascular resistance (PVR) ≥3 Wood Units (WU) by right heart catheterization (RHC) performed as part of standard of care evaluation within 90 days of enrollment.
- Treprostinil therapy must be recommended by the treating physician per standard of care.
- Be NYHA Functional Class II, III, or IV.
- Had pulmonary capillary wedge (PCW) pressure ≤18 mmHg and transpulmonary gradient (TPG) ≥15 mmHg.
Exclusion Criteria
- Patients must not:
- Had received any any investigational therapy as part of a clinical trial for any indication within 30 days prior to enrollment.
- Had a change in dose of treatment for PAH (bosentan [Tracleer], ambrisentan [Letairis], tadalafil [Adcirca], or sildenafil [Revatio]), within 30 days prior to enrollment. That is, subjects may have been treated with any of these agents provided the dose was stable for at least 30 days prior to enrollment.
- Had renal failure requiring hemodialysis.
Data sourced from ClinicalTrials.gov (NCT01028651). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.