Assessing the safety and efficacy of Macitentan in patients with portopulmonary hypertension


Several drugs (blood vessel dilators), including macitentan, are currently approved for the treatment of pulmonary arterial hypertension (PAH) in many parts of the world. These drugs have significantly improved the outcome of the condition. However, none of these treatments have been evaluated in portopulmonary hypertension, a form of PAH caused by liver disease. The purpose of this study is to assess the safety and effectiveness of macitentan in portopulmonary hypertension by looking at several clinical indicators, including the pressures within the arteries of the lungs and a person’s capacity for exercise.

Keywords: Portopulmonary hypertension, PAH, macitentan

Study Sites

Keck School of Medicine, Los Angeles 90089

  • Men & Women
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What's involved?

Study length
  • Maximum 8 months long, including up to 28 days screening, 24 weeks' study and 30 days' post-study safety follow-up

Number of visits
  • 8

  • Macitentan or placebo

  • Blood draw for laboratory tests (monthly)

  • Invasive measurement of blood pressure in the lungs (2x)

  • 6-minute walk test (monthly)

  • Physical examination/vital signs, pregnancy test (monthly, if applicable)

  • Hepatic vein catheterization (if needed)

  • All study procedures are free and reasonable travel expenses are compensated

Medical cost coverage
  • None Required

Why participate?

Medical advances are impossible without volunteers.


Must have

  • Confirmed diagnosis of portopulmonary hypertension
  • Increased blood flow resistance in the lungs
  • Able to perform a 6-minute walk test

Can't have

  • Other likely causes of pulmonary hypertension
  • Severe or unstable liver disease
  • Transplanted liver
  • Other medications that interfere with study drug
  • IV, SC, oral prostacyclin or ERA treatment
  • Allergy to the study drug
  • Ongoing alcohol or drug abuse
  • Any other reason that might interfere with study conduct

About This Study

The study is being conducted in 28 centers across the US as well as 52 centers worldwide, including in Europe and Brazil. Participants will receive either macitentan (active treatment) or placebo (no study drug) for 12 weeks, and then all patients will receive macitentan for a further 12 weeks. Volunteer participation will help contribute to the first robust evidence for treating this disease with pulmonary arterial hypertension medications.

Study team

Fh1w9f3ctogktdjeqylh Collaborate with the PI

For questions about this study, contact:

Research study materials

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