Study to Determine Safe Doses of Selexipag for Children with Pulmonary Arterial Hypertension

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What is this study about?

This clinical trial is focused on studying pulmonary arterial hypertension in children. This is a condition where the blood pressure in the arteries of the lungs is higher than normal, which can lead to heart problems. The treatment being tested is a medication called selexipag, which is taken as a film-coated tablet. Selexipag works by targeting specific receptors in the body to help relax and widen the blood vessels in the lungs, potentially improving blood flow and reducing the workload on the heart.

The purpose of the study is to confirm the appropriate doses of selexipag for children aged 2 to 18 years with pulmonary arterial hypertension. The study will observe how the medication is processed in the body and its effects over a period of time. Participants will take the medication orally, and the study will monitor their response to determine if the doses used in children achieve similar effects as those seen in adults.

Throughout the study, children will receive selexipag and will be closely monitored by healthcare professionals to ensure their safety and to assess how well the medication is tolerated. The study aims to gather information on the medication’s safety and how it behaves in the body, which will help in understanding the best way to use selexipag in treating children with this condition.

1 enrollment

Upon joining the study, the first step involves providing a signed and dated informed consent by the parent(s) or legally authorized representative(s). If developmentally capable, the child will also provide assent.

Eligibility is confirmed for children aged between 2 and 18 years, weighing at least 9 kg, with a confirmed diagnosis of pulmonary arterial hypertension (PAH) through historical right heart catheterization.

2 baseline visit

During the baseline visit, a thorough assessment is conducted to ensure all eligibility criteria are met. This includes confirming the PAH diagnosis and ensuring the child is in World Health Organization functional class II to III.

For females of childbearing potential, a negative pregnancy test is required, and agreement to undertake monthly pregnancy tests and use reliable contraception is necessary.

3 medication administration

The study involves taking a medication called selexipag, which is provided in the form of a film-coated tablet for oral use. The dosage and frequency are determined based on the child’s specific needs and are aimed at achieving similar exposure levels as in adults.

The medication is administered over a 12-week period, during which the dose may be gradually increased to find the optimal level.

4 monitoring and assessments

Throughout the trial, regular monitoring and assessments are conducted to evaluate the safety and tolerability of the medication. This includes checking for any side effects and measuring how the body processes the medication.

These assessments help in understanding the pharmacokinetics, which is how the drug is absorbed, distributed, metabolized, and excreted in the body.

5 end of study

At the end of the study, a final assessment is conducted to gather comprehensive data on the child’s response to the medication.

The study aims to confirm the appropriate starting dose of selexipag for children with PAH, ensuring it is safe and effective.

Who Can Join the Study?

Signed and dated informed consent by the parent(s) or legally authorized representative(s) and agreement from children who are able to understand and agree.
Males or females between the ages of 2 and 17 years at the start of the study, weighing at least 9 kg (about 20 pounds).
Confirmed diagnosis of Pulmonary Arterial Hypertension (PAH) through a test called right heart catheterization, which checks the pressure in the heart and lungs.
PAH must be due to one of the following causes:
- Idiopathic (unknown cause)
- Heritable (runs in families)
- Associated with congenital heart disease (heart problems present from birth)
- Drug or toxin-induced
- Associated with HIV
- Associated with connective tissue disease (affects tissues that support other parts of the body)
Must be in World Health Organization functional class II to III, which describes the severity of symptoms and how they affect daily activities.
Participants must be treated with specific medications for PAH, such as an endothelin receptor antagonist (ERA) or a phosphodiesterase type 5 (PDE-5) inhibitor, and the dose must have been stable for at least 3 months before joining the study. Alternatively, participants who are not suitable for these treatments can also join.
Females who can have children must have a negative pregnancy test at the start and agree to monthly tests. They must also use a reliable method of birth control if they are sexually active, from the start of the study until 30 days after stopping the study drug.

Who Cannot Join the Study?

Patients who are not within the age range of 2 to 18 years cannot participate.
Patients who do not have Pulmonary Arterial Hypertension cannot participate.
Patients who are part of a vulnerable population that the study does not include cannot participate.

Where you can join this trial?

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Verified Sites

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Other Sites

Site Name	City	Country
Hopital Des Enfants	Toulouse	France
Assistance Publique Hopitaux De Paris	Paris	France
Medical Center – University Of Freiburg	Freiburg Im Breisgau	Germany
Gottsegen National Cardiovascular Center	Budapest	Hungary

Trial status

Country	Status	Recruitment Start
France	Not recruiting	01.05.2018
Germany	Not recruiting	01.05.2018
Hungary	Not recruiting	01.05.2018

Trial locations

Investigated drugs:

Selexipag

Selexipag is a medication used in this clinical trial to help treat children with pulmonary arterial hypertension, which is a type of high blood pressure that affects the arteries in the lungs and the right side of the heart. The goal of using selexipag in this study is to find the right starting dose for children, based on how the medication works in adults. This medication helps to relax and widen the blood vessels in the lungs, which can lower blood pressure and improve symptoms. The study aims to understand how selexipag and its active form, known as ACT-333679, behave in the bodies of children, to ensure they receive a dose that is both safe and effective.

Investigated diseases:

Pulmonary arterial hypertension

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. Over time, this increased workload can cause the heart muscle to weaken and become less efficient. Symptoms often include shortness of breath, fatigue, and dizziness, which may worsen with physical activity. The progression of the disease can lead to further complications affecting the heart and lungs.

Trial ID:

2022-503042-42-00

Protocol code:

AC-065A203

Trial Phase:

Therapeutic exploratory (Phase II)

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Study to Determine Safe Doses of Selexipag for Children with Pulmonary Arterial Hypertension

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