A Study to Assess the Safety and Efficacy of Treprostinil to Facilitate Liver Transplantation in Patients With Portopulmonary Hypertension

NCT01028651 | Completed Results

• 1 other identifier: RIV-PH-414
• Study Type: observational
• Target: 13
• Locations: 1 country
• Sites: 4

Brief 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.

Conditions

Portopulmonary Hypertension; Pulmonary Arterial Hypertension; Pulmonary Hypertension

Keywords

PAH; Pulmonary Hypertension; Remodulin; Treprostinil; Quality of Life

Outcome Measures

Primary Outcomes (1)

• Number of Subjects Who Achieved Hemodynamic Parameters Appropriate for Orthotopic Liver Transplantation Candidacy at Week 24.

  The primary efficacy endpoint was the number of subjects who achieved a mean pulmonary arterial pressure (mPAP) less than 35 mmHg and a pulmonary vascular resistance (PVR) less than 3 Wood units (WU) at Week 24 in patients with severe portopulmonary hypertension (PoPH).

  24 Weeks

Secondary Outcomes (12)

• Change in Hemodynamic Parameters (Via Right Heart Catheterization [RHC]) at Rest From Baseline to Week 24

  24 weeks

• Change in Heart Rate at Rest From Baseline to Week 24

  24 weeks

• Change in Cardiac Output at Rest From Baseline to Week 24

  24 weeks

• Change in Arterial and Venous Oxygen Saturation at Rest From Baseline to Week 24

  24 weeks

• Change in Pulmonary Vascular Resistance (PVR) at Rest From Baseline to Week 24

  24 weeks

Study Arms (1)

Portopulmonary hypertension

Drug: Treprostinil

Interventions

Treprostinil DRUG

Remodulin is supplied in concentrations of 1, 2.5 , 5, and 10 mg/mL and can be administered as supplied or diluted for intravenous (IV) infusion prior to administration. Remodulin is indicated for subcutaneous (SC) or IV use only as a continuous infusion. Remodulin is preferably infused subcutaneously, but can be administered by a central IV line if the SC route is not tolerated. The infusion rate is initiated at 1.25 ng/kg/min. If this initial dose cannot be tolerated because of systemic effects, the infusion rate should be reduced to 0.625 ng/kg/min.

Also known as: Remodulin

Portopulmonary hypertension

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Probability Sample

Study Population

Subjects were recruited from 4 liver transplantation centers in the US, referred for portopulmonary hypertension.

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• United Therapeutics: lead
• University of California, Los Angeles: collaborator
• Brigham and Women's Hospital: collaborator
• University of Texas: collaborator
• Emory University: collaborator

Study Sites (4)

University of California, Los Angeles

Los Angeles, California, 90024, United States

Location

Emory Univeristy

Atlanta, Georgia, 30322, United States

Location

Brigham and Women's Hospital

Boston, Massachusetts, 02115, United States

Location

University of Texas, Southwestern Medical Center

Dallas, Texas, 75235, United States

Location

MeSH Terms

Conditions

Pulmonary Arterial Hypertension; Hypertension, Pulmonary

Interventions

treprostinil

Condition Hierarchy (Ancestors)

Lung Diseases; Respiratory Tract Diseases; Hypertension; Vascular Diseases; Cardiovascular Diseases

Results Point of Contact

• Title: Kevin Laliberte, PharmD
• Organization: United Therapeutics Corp.

KLaliberte@unither.com

919-425-8176

Study Officials

• Rajan Saggar, MD

  University of California, Los Angeles

  STUDY DIRECTOR

• Micah Fisher, MD

  Emory University

  STUDY DIRECTOR

• Aaron Waxman, MD, PhD

  Brigham and Women's Hospital

  STUDY DIRECTOR

• Sonja Bartolome, MD

  University of Texas Southwestern Medical Center

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

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

Study Record Dates

• First Submitted: December 8, 2009
• First Posted: December 9, 2009
• Study Start: January 1, 2011
• Primary Completion: April 1, 2013
• Study Completion: April 1, 2013
• Last Updated: February 24, 2017
• Results First Posted: February 24, 2017

Record last verified: 2017-01

Locations

US (4)