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Macitentan

Clinical trials are studying Macitentan in different groups of patients, including children and adults with pulmonary arterial hypertension and people with inoperable chronic thromboembolic pulmonary hypertension. These studies look at safety, long-term effects, and how well treatment works. Some trials compare Macitentan with standard care, placebo, or different doses.

Table of Contents

Overview of the trial program

The trial data show studies of Macitentan in people with pulmonary hypertension, with a focus on pulmonary arterial hypertension and inoperable chronic thromboembolic pulmonary hypertension.[1][2][3][4]

The studies include both completed and authorised trials, and they are mainly designed to test safety, effectiveness, and long-term results.[1][2][3][4]

Conditions being studied

One group of trials studies pulmonary arterial hypertension, including a study in children and another in adults with symptomatic disease.[1][2]

Another trial studies inoperable chronic thromboembolic pulmonary hypertension, which means a long-term form of high blood pressure in the lungs caused by blood clots that cannot be treated with surgery.[4]

One authorised study is a long-term follow-up study for participants from pulmonary hypertension parent studies, showing interest in continued safety monitoring over time.[3]

Trial designs and phases

The set includes Phase 2 and Phase 3 trials.[1][2][3][4]

The pediatric study was a multicenter, open-label, randomized study with a single-arm extension period, which means it was done at more than one center, treatment assignment was known, and some participants continued into an added follow-up period.[1]

The CTEPH study was a Phase 2 interventional trial comparing initial dual oral treatment with standard-of-care oral monotherapy before balloon pulmonary angioplasty, also called BPA.[4]

The Phase 3 adult study compares Macitentan 75 mg with Macitentan 10 mg, while the long-term follow-up study focuses on safety in treated participants.[2][3]

Who can participate

The pediatric trial enrolled children with pulmonary arterial hypertension, including children younger than 2 years and those 2 years of age or older in the blood-level measurements described in the outcomes.[1]

The Phase 3 dose-comparison study targets participants with symptomatic pulmonary arterial hypertension.[2]

The CTEPH trial focused on newly diagnosed, treatment-naïve participants with inoperable disease, meaning they had not yet received treatment for this condition.[4]

The long-term follow-up study includes treated participants from pulmonary hypertension parent studies.[3]

Main outcomes being measured

In the pediatric study, the main outcome was trough plasma concentrations, which means the lowest drug level in the blood before the next dose, measured at steady state in children of different ages.[1]

The Phase 3 dose-comparison study measured the time to first CEC-adjudicated morbidity or mortality event, meaning the time until a serious disease-worsening event or death is confirmed by an expert committee.[2]

The long-term follow-up study measured treatment-emergent adverse events, serious adverse events, events leading to stopping treatment, and deaths from baseline until end of study.[3]

The CTEPH study measured pulmonary vascular resistance at week 16, expressed as a percentage of the starting value, to see how blood flow resistance in the lung vessels changed over time.[4]

Key studies in this set

NCT02932410 was a Phase 3 pediatric study of Macitentan versus standard of care, with goals to assess pharmacokinetics, safety, and efficacy in children with pulmonary arterial hypertension.[1]

2024-515669-32-00 is an authorised Phase 3 study comparing long-term efficacy and safety of Macitentan 75 mg versus 10 mg in pulmonary arterial hypertension, with 900 planned participants.[2]

2023-506791-27-00 is an authorised Phase 3 long-term follow-up study for treated participants, with 155 planned participants and a focus on safety outcomes.[3]

NCT04780932, also called IMPACT-CTEPH, was a completed Phase 2 study in inoperable chronic thromboembolic pulmonary hypertension, with 96 participants and a focus on pulmonary vascular resistance before BPA.[4]

What these trials may mean for patients

These studies show that researchers are testing Macitentan in different forms of pulmonary hypertension, not just one patient group.[1][2][3][4]

Some trials ask whether a different dose may work better, while others focus on safety, long-term follow-up, or how the treatment changes blood flow in the lungs.[2][3][4]

Together, the studies aim to build a clearer picture of how Macitentan is being evaluated in children and adults with pulmonary hypertension-related conditions.[1][2][3][4]

Trial ID Phase Condition studied Status Enrollment
NCT02932410 Phase 3 Pediatric pulmonary arterial hypertension Completed 162
2024-515669-32-00 Phase 3 Pulmonary arterial hypertension Authorised 900
2023-506791-27-00 Phase 3 Pulmonary arterial hypertension Authorised 155
NCT04780932 Phase 2 Inoperable chronic thromboembolic pulmonary hypertension Completed 96

FAQ

  • What conditions are being studied in Macitentan trials? The trials in this data study pulmonary arterial hypertension, including pediatric PAH, and inoperable chronic thromboembolic pulmonary hypertension. One study also looks at long-term follow-up in pulmonary hypertension parent studies.
  • Who can take part in these trials? Participants include children with pulmonary arterial hypertension, adults with symptomatic pulmonary arterial hypertension, and newly diagnosed, treatment-naïve people with inoperable CTEPH. Each trial has its own eligibility rules.
  • What phases are the Macitentan trials? The trials shown here are in Phase 2 and Phase 3. Phase 2 studies usually explore early signs of benefit, while Phase 3 studies test effectiveness and safety in larger groups.
  • What are the trials trying to measure? The studies measure things like drug levels in the blood, time to the first morbidity or mortality event, long-term safety, and pulmonary vascular resistance. These outcomes help show how the treatment performs over time.
  • Are the studies comparing Macitentan with other treatments? Yes. Some trials compare Macitentan with standard of care, placebo, or a different Macitentan dose. This helps researchers see whether one approach works better or is safer.

Ongoing Clinical Trials on Macitentan

  • Long-term Safety Study of Tadalafil and Macitentan for Patients with Pulmonary Arterial Hypertension or Chronic Thromboembolic Pulmonary Hypertension

    Recruiting

    1 1 1 1
    Investigated diseases:
    Investigated drugs:
    Bulgaria Hungary Poland

  • Study on Macitentan for Children with Pulmonary Arterial Hypertension

    Not recruiting

    1 1 1
    Investigated diseases:
    Investigated drugs:
    Hungary Poland Portugal Spain

  • Study Comparing the Effectiveness and Safety of Macitentan 75 mg and 10 mg for Patients with Pulmonary Arterial Hypertension

    Not recruiting

    1 1 1
    Investigated diseases:
    Investigated drugs:
    Austria Belgium Bulgaria Czechia Denmark France +10

  • Study on the Effects of Riociguat and Macitentan in Patients with Inoperable Chronic Thromboembolic Pulmonary Hypertension

    Not recruiting

    1 1
    Investigated diseases:
    Investigated drugs:
    France

Glossary

  • Pulmonary arterial hypertension (PAH): A type of high blood pressure in the arteries of the lungs. It can make the heart work harder and cause symptoms such as shortness of breath.
  • Pediatric: Related to children.
  • Inoperable: Not able to be treated with surgery.
  • Chronic thromboembolic pulmonary hypertension (CTEPH): A long-lasting form of high blood pressure in the lungs caused by blood clots that block blood flow.
  • Phase 2: An earlier stage of a clinical trial that looks at whether a treatment may work and checks safety in a smaller group.
  • Phase 3: A later stage of a clinical trial that tests a treatment in a larger group to learn more about how well it works and how safe it is.
  • Open-label: A study design where participants and researchers know which treatment is being given.
  • Randomized: Participants are assigned to treatment groups by chance.
  • Placebo: An inactive treatment used for comparison in a trial.
  • Pulmonary vascular resistance (PVR): How much resistance blood meets when flowing through the blood vessels in the lungs. Lower values can mean easier blood flow.
  • Morbidity or mortality event: A health problem or death used as a study outcome to measure serious disease worsening.
  • Adverse event: Any unwanted medical problem that happens during a study.

References