Ambrisentan in Patients With Porto-pulmonary Hypertension A Multicenter Open Label Trial
Status:
Completed
Trial end date:
2020-03-01
Target enrollment:
Participant gender:
Summary
This is an Open Label, Multicenter, pilot clinical trial to assess the efficacy and safety of
an oral selective Endothelin Receptor Antagonist (ambrisentan) in patients with
portopulmonary hypertension.
Preliminary evidence suggests that ambrisentan is safe and effective in patients with
portopulmonary hypertension. The goal of therapy for these patients is to improve symptoms of
dyspnea and to improve pulmonary hemodynamics to a mean pulmonary artery pressure <35 mm Hg
in order to make patients eligible for liver transplantation. Therefore, the primary
endpoints for this study will include 6 minute walk distance (6MWD) and pulmonary vascular
resistance (PVR).
Eligible subjects will receive 5 mg ambrisentan once-daily for the first 4 weeks. After the
initial 4-week period, investigators will increase study drug dose to 10 mg once daily (both
5 mg and 10 mg doses are FDA approved). If 10 mg is not tolerated in the opinion of
investigator, then the investigator may decrease the dose back to 5 mg once daily. Primary
outcome is a change in both the 6 Minute Walk Distance and in Pulmonary Vascular Resistance
from baseline to Week 24. Subjects will be monitored with liver function tests (LFT) every 2
weeks for the first 8 weeks, then every 4 weeks thereafter. These safety laboratory tests may
be performed at a local phlebotomy laboratory or at the Investigator clinic. In addition, the
Investigator will assess each subject for safety and efficacy at Week 4, Week 12, and Week
24. Following Week 24, subjects will be assessed for safety and efficacy every 12 weeks.
Patients will be followed for a total of 1 year. After 1 year, if the Investigator feels that
continuing the treatment will be beneficial to the patients, they will be provided with
ambrisentan by Gilead Pharmaceuticals, free of charge.