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

NCT ID: NCT01028651

Last Updated: 2017-02-24

Basic Information

• Recruitment Status: COMPLETED
• Total Enrollment: 13 participants
• Study Classification: OBSERVATIONAL
• Study Start Date: 2011-01-31
• Study Completion Date: 2013-04-30

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.

Detailed Description

Treprostinil is approved as a continuous subcutaneous (SC) or intravenous (IV) infusion by the FDA for the treatment of WHO group I PAH with New York Heart Association (NYHA) Functional Class II, III or IV symptomatology. To date, treprostinil has not been studied in the setting of PoPH; however, it is commonly prescribed in this setting. This was an observational, open-label, multicenter study which documented the safety and efficacy profile of this agent in PoPH to facilitate orthotopic liver transplantation (OLT).

Conditions

Portopulmonary Hypertension; Pulmonary Arterial Hypertension; Pulmonary Hypertension

Study Design

• Observational Model Type: COHORT
• Study Time Perspective: PROSPECTIVE

Study Groups

Portopulmonary hypertension

Treprostinil

Intervention Type: 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.

Interventions

Treprostinil

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.

Intervention Type: DRUG

Other Intervention Names

Remodulin

Eligibility Criteria

Inclusion Criteria

• Patients must:

1. Had portal hypertension.

2. Be otherwise suitable candidates for OLT.

3. 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.

4. Treprostinil therapy must be recommended by the treating physician per standard of care.

5. Be NYHA Functional Class II, III, or IV.

6. Had pulmonary capillary wedge (PCW) pressure ≤18 mmHg and transpulmonary gradient (TPG) ≥15 mmHg.

Exclusion Criteria

• Patients must not:

1. Had received any any investigational therapy as part of a clinical trial for any indication within 30 days prior to enrollment.

2. 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.

3. Had renal failure requiring hemodialysis.

• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

University of California, Los Angeles

OTHER

Sponsor Role: collaborator

Brigham and Women's Hospital

OTHER

Sponsor Role: collaborator

University of Texas

OTHER

Sponsor Role: collaborator

Emory University

OTHER

Sponsor Role: collaborator

United Therapeutics

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Rajan Saggar, MD

Role: STUDY_DIRECTOR

University of California, Los Angeles

Micah Fisher, MD

Role: STUDY_DIRECTOR

Emory University

Aaron Waxman, MD, PhD

Role: STUDY_DIRECTOR

Brigham and Women's Hospital

Sonja Bartolome, MD

Role: STUDY_DIRECTOR

UT Southwestern Medical Center

Locations

University of California, Los Angeles

Los Angeles, California, United States

Emory Univeristy

Atlanta, Georgia, United States

Brigham and Women's Hospital

Boston, Massachusetts, United States

University of Texas, Southwestern Medical Center

Dallas, Texas, United States

Countries

United States

Other Identifiers

RIV-PH-414

Identifier Type: -

Identifier Source: org_study_id