Ongoing, recruitingTherapeutic confirmatory (Phase III)Interventional

A multicenter, open-label, randomized study with single-arm extension period to assess the pharmacokinetics, safety and efficacy of macitentan versus standard of care in children with pulmonary arterial hypertension

This research study is for children with a serious lung condition called pulmonary arterial hypertension (PAH). It's a Phase 3 trial, which means the new medicine, macitentan, has already been tested in earlier stages. The main goal is to understand how the medicine works in children of different ages, checking how much of it is in their blood and if it's safe to use. Researchers will compare macitentan to the standard care children usually receive. They will also look at whether the medicine reduces hospital stays, improves physical activity, and boosts overall quality of life. This study aims to find better ways to help children living with PAH.

At a glance

Status

Ongoing, recruiting

Phase

Therapeutic confirmatory (Phase III)

Sponsor

Actelion Pharmaceuticals Ltd.

Enrolment target

Start

20 Aug 2024

Pediatric pulmonary arterial hypertension

What is this study about?

This study is all about helping children who have a serious lung condition called pulmonary arterial hypertension (PAH). In PAH, the blood vessels in the lungs become narrow, making it hard for the heart to pump blood. This can lead to tiredness, breathlessness, and other serious problems. The study is investigating a specific medicine called macitentan, which is already used in adults with PAH. Researchers want to understand if it can also be a good treatment option for children.

The main things the scientists want to find out are how the medicine behaves in children's bodies (how much gets into the blood), whether it's safe to use, and if it's effective in improving their health. They will be comparing macitentan to the treatments children usually receive. This type of study is called a 'Phase 3' trial, which means it’s one of the final steps before a new medicine can potentially be approved for wider use. By doing this research, doctors hope to find better and safer ways to help children with PAH live healthier and more active lives.

Beyond just checking the medicine's levels and safety, the study will also look at several important aspects of a child's health. For example, they'll be tracking if the medicine can reduce the number of times a child needs to be hospitalized because of their PAH. They'll also measure things like how much a child can move around and play (their physical activity), how their heart is working, and how they feel about their overall quality of life. All this information helps doctors understand the full picture of how macitentan might help children with PAH.

Key takeaways

A study for children of all ages with pulmonary arterial hypertension (PAH).
Investigating a medicine called macitentan, already used in adults.
Checking how the medicine acts in the body, its safety, and how well it works.
Comparing macitentan to current standard treatments.
Aims to improve quality of life, reduce hospital stays, and enhance physical activity.
Regular health checks and monitoring will be part of the study.

Who may be eligible?

This study is looking for children of all ages who have been diagnosed with pulmonary arterial hypertension (PAH). Both boys and girls can take part in this research.

To see if your child can join, the doctors in charge of the study will check some specific medical details about their condition. This helps make sure the study is safe and appropriate for all children involved, and that the results will be clear.

Since this is a medical study, it's really important to talk to your child's doctor or the study team to find out if your child meets all the requirements to participate.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Does your child have a confirmed diagnosis of pulmonary arterial hypertension (PAH)?
Is your child currently receiving treatment for PAH?
Is your child able to attend regular hospital or clinic visits?
Are you able to commit to the study's requirements over an extended period?
Do you want to explore new treatment options for your child's PAH?

Answer every question to see your result.

What does participation involve?

If your child takes part in this study, they will receive either the study medicine, macitentan, or their usual care. The study involves regular visits to the hospital or clinic where doctors will monitor your child closely. During these visits, there will be assessments like blood tests to check how much medicine is in their body, heart scans (echocardiograms) to see how their heart is working, and activity monitors to track their movement. They will also complete questionnaires about their quality of life. The total time your child will be involved in the core part of the study will vary, but there will be follow-up appointments and continued monitoring for a longer period. You can withdraw your child from the study at any time.

Potential risks and benefits

Participating in this study might offer your child the potential benefit of trying a new medicine that could improve their PAH symptoms, reduce their hospital stays, or improve their quality of life. However, like all medicines, macitentan can have side effects, and your child’s health might not improve. The study doctors will carefully monitor your child for any potential risks or side effects. You have the right to withdraw your child from the study at any point, without giving a reason, and this will not affect their usual medical care.

Locations (4)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

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Unverified
Portugal
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Unverified
Hungary
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Unverified
Spain
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Unverified
Poland

Common questions

What is pulmonary arterial hypertension (PAH)?

PAH is a serious condition where the blood vessels in the lungs become narrow, making it difficult for the heart to pump blood. This can lead to symptoms like shortness of breath and tiredness.

What is macitentan?

Macitentan is a medicine that helps to widen the blood vessels in the lungs, making it easier for blood to flow through. It’s already used for adults with PAH.

Why is this study important for children?

This study aims to find out if macitentan is safe and effective for children with PAH, as well as finding the right dose, potentially offering a new treatment option for them.

Will my child definitely get the new medicine?

No, this is a 'randomized' study, meaning some children will receive macitentan and others will continue with standard care. This helps researchers compare the treatments fairly.

How long will my child be in the study?

The study has a main period and a longer follow-up period. The exact duration will be explained by the study team, but it involves regular check-ups over an extended time.