Study on Macitentan 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. Pulmonary arterial hypertension is a condition where the blood pressure in the arteries of the lungs is higher than normal, which can make it difficult for the heart to pump blood through the lungs. The study will use a medication called macitentan, which is being tested to see how it works compared to the usual treatments for this condition. Macitentan is provided in a form that can be easily dissolved, making it suitable for children.

The purpose of the study is to understand how macitentan is processed in the body, as well as to evaluate its safety and effectiveness in treating children with pulmonary arterial hypertension. The study will involve children taking macitentan or receiving standard care, and their progress will be monitored over time. The study will also include a period where all participants will receive macitentan to gather more information about its long-term effects.

Participants will be observed for changes in their condition, including how well they can perform physical activities and their overall quality of life. The study will also track any hospitalizations related to pulmonary arterial hypertension and monitor the participants’ health through regular check-ups. The trial is expected to continue until late 2025, providing valuable insights into the treatment of this condition in children.

1 joining the study

Upon joining the study, a signed informed consent by the parent(s) or legally designated representative is required. Additionally, assent from developmentally capable children is needed before any study procedures begin.

2 randomization

Participants are randomly assigned to receive either the study medication, macitentan, or the standard of care treatment. This process ensures that each participant has an equal chance of receiving either treatment.

3 medication administration

If assigned to the macitentan group, the medication is taken orally in the form of a dispersible tablet. The dosage and frequency are determined by the study team based on the participant’s age and weight.

4 initial assessment

Participants undergo an initial assessment to measure baseline health indicators. This includes checking the trough (pre-dose) plasma concentrations of macitentan and its active metabolite at specific weeks, depending on the participant’s age.

5 ongoing monitoring

Throughout the study, participants are monitored for safety and efficacy of the treatment. This includes regular health check-ups and assessments to track any changes in health status.

6 end of core period

The core period of the study concludes with a final assessment of the participant’s health and response to the treatment. This includes measuring changes in physical activity, quality of life, and other health indicators.

7 extension period

Participants may enter a single-arm extension period where they continue to receive the study medication. This phase allows for further assessment of the long-term effects of the treatment.

8 study completion

Upon completion of the study, participants undergo a final evaluation to assess the overall impact of the treatment. This includes a review of all collected data and health outcomes.

Who Can Join the Study?

Signed informed consent by the parent(s) or legally designated representative, and agreement from children who are able to understand, before starting any study-related procedures.
Children who are 1 month to under 18 years old.
Children with a body weight of at least 3.5 kg at the time of joining the study.
Children with a confirmed diagnosis of pulmonary arterial hypertension (PAH) through a past procedure called right heart catheterization. This procedure measures the pressure in the lungs and heart.
Children with PAH that fits into specific categories, including those with Down Syndrome, and caused by:
- Unknown reasons (idiopathic PAH)
- Inherited reasons (heritable PAH)
- Associated with congenital heart disease (CHD), which is a heart problem present at birth
- Caused by drugs or toxins
- Associated with HIV
- Associated with connective tissue disease (PAH-aCTD)
Children who are in World Health Organization Functional Class I to III, which describes the severity of their condition.
Children who have not received PAH-specific treatment before, or those who are on one or two PAH-specific treatments.
Girls who can have children must have a negative pregnancy test before starting and during the study, and must agree to use a reliable method of birth control if they are sexually active.

Who Cannot Join the Study?

Patients with any other serious medical condition that might interfere with the study.
Patients who have had a recent surgery or are planning to have surgery during the study period.
Patients who are currently participating in another clinical trial.
Patients who have a known allergy to the study medication or its ingredients.
Patients who are unable to follow the study procedures or instructions.
Patients who have a history of non-compliance with medical treatments.
Patients who are pregnant or breastfeeding.
Patients with a history of drug or alcohol abuse.
Patients with certain heart conditions that are not stable.
Patients with severe liver or kidney disease.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name	City	Country
Hospital Universitario Y Politecnico La Fe	Valencia	Spain
Unidade Local De Saude De Lisboa Ocidental E.P.E.	Carnaxide	Portugal
Centro Hospitalar Universitario Sao Joao E.P.E.	Porto	Portugal

Other Sites

Site Name	City	Country
Gottsegen National Cardiovascular Center	Budapest	Hungary
Wojewodzki Szpital Specjalistyczny We Wroclawiu	Wroclaw	Poland
Syampdx Kumegxtzw Imj Khyonx Julubhuxi Uajbmmtfugss Mmiffmhunw Iwu Khiccm Mswkiqjqqaytigh W Psuyqpwp	Poznan	Poland
Cgctnq Hawdykhmys E Udqhrqictzjoh Dr Czbygmc Ehvgkn	Coimbra	Portugal

Trial status

Country	Status	Recruitment Start
Hungary	Not recruiting	27.10.2023
Poland	Not recruiting	27.10.2023
Portugal	Not recruiting	27.10.2023
Spain	Not recruiting	27.10.2023

Trial locations

Investigated drugs:

Macitentan

Macitentan is a medication used in this clinical trial to help treat children with pulmonary arterial hypertension, which is a condition where there is high blood pressure in the arteries that supply the lungs. This medication works by relaxing and widening the blood vessels, which can help lower the blood pressure in the lungs and improve the ability to exercise. The goal of using macitentan in this trial is to see how well it works and how safe it is for children with this condition.

Standard of Care refers to the regular treatment that is typically given to patients with pulmonary arterial hypertension. This can include a variety of medications and therapies that are commonly used to manage the condition and its symptoms. In this trial, the standard of care is used as a comparison to see how well macitentan performs against the usual treatments that children with this condition receive.

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 a result, the heart must work harder to pump blood through these arteries. Over time, this increased workload can lead to the enlargement and weakening of the heart. 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 ID:

2023-509845-10-00

Protocol code:

AC-055-312

NCT ID:

NCT02932410

Trial Phase:

Therapeutic confirmatory (Phase III)

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Study on Macitentan for Children with Pulmonary Arterial Hypertension

What is this study about?

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