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Study on Selexipag and Bosentan for Children with Pulmonary Arterial Hypertension

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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial is focused on studying Pulmonary Arterial Hypertension (PAH), a condition where the blood pressure in the arteries of the lungs is higher than normal. The study is testing a medication called Selexipag, which is being used as an additional treatment to the standard care for children aged 2 to under 18 years who have PAH. The purpose of the study is to see if adding Selexipag can help delay the progression of the disease compared to a placebo.

Participants in the study will receive either Selexipag or a placebo, in addition to their usual treatment for PAH. The study is designed to be double-blind, meaning neither the participants nor the researchers will know who is receiving the actual medication and who is receiving the placebo. This helps ensure that the results are not biased. The study will also include an open-label extension period, where all participants may receive Selexipag after the initial phase. The treatment will be given in the form of film-coated tablets, taken orally.

The study will last for a period of up to 96 weeks, during which the participants' health and response to the treatment will be closely monitored. The main goal is to determine how long it takes for the disease to progress in participants taking Selexipag compared to those taking a placebo. This information will help researchers understand the effectiveness and safety of Selexipag as an add-on treatment for children with PAH.

The research process

The trial runs in 7 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Joining the study

    Upon joining the study, the parent or legal guardian must sign an informed consent form indicating understanding of the study's purpose and procedures. If the child is capable of understanding, typically at age 7 or older, their assent is also required.

  2. Step 2

    Randomization

    Participants are randomly assigned to receive either the study medication, selexipag, or a placebo. This process is double-blind, meaning neither the participant nor the study team knows which treatment is being administered.

  3. Step 3

    Medication administration

    Participants will take the study medication, selexipag, in the form of a film-coated tablet. The medication is taken orally. The dosage and frequency will be determined by the study protocol and communicated to the participant by the study team.

  4. Step 4

    Treatment period

    The treatment period involves regular administration of the study medication as per the prescribed dosage and frequency. Participants will continue their standard care treatment alongside the study medication.

  5. Step 5

    Monitoring and assessments

    Throughout the study, participants will undergo regular monitoring and assessments to evaluate the efficacy and safety of the treatment. This includes physical examinations, laboratory tests, and other necessary evaluations as determined by the study protocol.

  6. Step 6

    Open-label extension

    After the initial study period, participants may enter an open-label extension phase where all participants receive selexipag. This phase allows further assessment of the medication's long-term effects.

  7. Step 7

    End of study

    The study concludes with a final assessment to determine the overall impact of the treatment. Participants will be informed about the study's findings and any further steps if necessary.

Who can join the trial?

11 criteria

  • Parent(s) or legal guardians must sign a form to show they understand the study and agree for their child to participate. Children who can understand the study (usually 7 years and older) must also agree to participate.
  • The child must be between the ages of 2 and 18 years old and weigh at least 9 kg (about 20 pounds).
  • The child must have a confirmed diagnosis of Pulmonary Arterial Hypertension (PAH), which is a type of high blood pressure affecting the lungs and heart. This diagnosis must be confirmed by a past medical test called Right Heart Catheterization (RHC).
  • The child must have PAH that falls under specific categories, including:
    • Idiopathic PAH (IPAH): PAH with no known cause.
    • Heritable PAH (HPAH): PAH that runs in families.
    • PAH associated with congenital heart disease (PAH-aCHD): PAH related to heart defects present at birth.
    • PAH caused by drugs or toxins.
    • PAH associated with HIV.
    • The child must be in World Health Organization Functional Class (WHO FC) II or III, which describes the severity of their symptoms.
    • The child must be receiving at least one specific treatment for PAH, such as an endothelin receptor antagonist (ERA) or a PDE-5 inhibitor, and the treatment dose must have been stable for at least 3 months before starting the study.
    • If the child is a female who can become pregnant, she must have a negative pregnancy test before starting the study and agree to regular pregnancy tests during the study. She must also agree to use effective birth control if she is sexually active.

Who cannot join the trial?

10 criteria

  • Patients who have a different condition than Pulmonary Arterial Hypertension cannot participate.
  • Patients who are not within the specified age range for the study cannot participate.
  • Patients who are not able to follow the study procedures or take the study medication cannot participate.
  • Patients who have other serious health conditions that might interfere with the study cannot participate.
  • Patients who are pregnant or breastfeeding cannot participate.
  • Patients who are currently participating in another clinical trial cannot participate.
  • Patients who have had a recent surgery or are planning to have surgery during the study cannot participate.
  • Patients who have allergies to the study medication or its ingredients cannot participate.
  • Patients who have a history of drug or alcohol abuse cannot participate.
  • Patients who have been diagnosed with a mental health condition that might interfere with the study cannot participate.
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Investigated drugs

Selexipag is a medication used in this clinical trial to help treat children with a condition called Pulmonary Arterial Hypertension (PAH). PAH is a type of high blood pressure that affects the arteries in the lungs and the right side of the heart. Selexipag works by relaxing and widening the blood vessels in the lungs, which can help lower the blood pressure in these vessels and improve the ability to exercise. In this trial, Selexipag is being tested to see if it can help slow down the progression of the disease when added to the standard treatments that children with PAH are already receiving.

What is already known about the treatment

Selexipag – Selexipag is administered orally in tablet form and is currently being studied in clinical trials for its effectiveness in treating Pulmonary Arterial Hypertension (PAH) in children. It is an investigational drug that is not yet widely available in medical practice but is being evaluated for its potential benefits. The main therapeutic indication for Selexipag is to help manage PAH, a condition characterized by high blood pressure in the arteries of the lungs. At the molecular level, Selexipag works by targeting specific pathways in the body to relax and widen blood vessels, which helps to lower blood pressure in the lungs. It is classified pharmacologically as a prostacyclin receptor agonist, which means it mimics the action of a natural substance in the body that helps to dilate blood vessels.

Investigated diseases

Pulmonary Arterial Hypertension – Pulmonary Arterial Hypertension is a condition characterized by high blood pressure in the arteries that supply the lungs. This disease causes the small arteries in the lungs to become narrow or blocked, leading to increased resistance to blood flow. As the condition progresses, the heart must work harder to pump blood through the lungs, which can lead to heart strain and enlargement. Over time, this increased workload can weaken the heart muscle and affect its ability to function properly. Symptoms often include shortness of breath, fatigue, dizziness, and swelling in the ankles or legs. The progression of the disease can vary, with symptoms gradually worsening over time.
Trial detailsLast updated 2 Oct 2026
Age0-17PhasePhase IIITrial ID2022-501012-34-00Protocol codeAC-065A310Estimated enrolment213 patientsSponsorActelion Pharmaceuticals Ltd.

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