Active not recruitingPHASE3INTERVENTIONAL

A Study of Selexipag as Add-On Treatment to Standard of Care in Children With Pulmonary Arterial Hypertension

This research study is looking into a new use for a medicine called selexipag for children and teenagers (aged 2 to 17) who have a serious lung condition known as pulmonary arterial hypertension (PAH). PAH makes it hard for blood to flow through the lungs, leading to breathlessness and tiredness. Selexipag is already used in adults with PAH. The study wants to find out if adding selexipag to the standard treatments children are already receiving can prevent their PAH from getting worse, compared to those who receive their usual treatment plus a dummy medicine (placebo). Because PAH in children is rare and needs better treatments, this study is very important to gather more information and hopefully improve care for young patients.

At a glance

Status

Active not recruiting

Phase

PHASE3

Sponsor

Actelion

Enrolment target

138

Start

16 Jan 2020

Estimated completion

01 Oct 2027

Hypertension, Pulmonary

What is this study about?

This study is about a lung condition called pulmonary arterial hypertension, or PAH. In children with PAH, the blood vessels in their lungs become narrow and stiff, making it difficult for the heart to pump blood through them effectively. This can lead to serious health problems and needs careful management.

Doctors are always looking for better ways to treat PAH, especially in children. Currently, there aren't many medicines specifically approved for young people that work on a particular pathway in the body called the prostacyclin pathway. However, there's a medicine called selexipag that already helps adults with PAH by working on this pathway. Scientists believe that selexipag might also reduce scarring, prevent cells from growing too much, and stop blood clots from forming in the lung vessels.

This study aims to discover if adding selexipag to the treatments children are already taking for their PAH can make a difference. The main goal is to see if it can delay the disease from getting worse, compared to a dummy medicine (placebo). This research is vital because it could lead to an important new treatment option for children living with PAH, giving them a better quality of life.

Key takeaways

The study tests selexipag for children aged 2-17 with PAH.
It aims to see if selexipag can slow down disease progression.
Children will receive selexipag or a dummy medicine first, in addition to their current treatments.
The study includes regular check-ups over an estimated 8-year period.
Participation is voluntary, and you can withdraw at any time.

Who may be eligible?

This study is looking for children and teenagers between 2 and 17 years old who weigh at least 9 kilograms (about 20 pounds) and have a confirmed diagnosis of pulmonary arterial hypertension (PAH).

To be considered, your child's PAH must fall into certain types, including those that are inherited, those linked to some congenital heart conditions, or PAH that developed after certain heart surgeries or due to specific infections like HIV. They should also be in WHO functional class II or III, which describes how much their symptoms affect their daily life. Importantly, they must already be on at least one standard PAH treatment, such as an endothelin receptor antagonist or a phosphodiesterase type-5 inhibitor, and have been on a stable dose for at least three months.

Children generally cannot take part if their PAH is due to certain other serious conditions like liver problems or blood vessel blockages, or if they have a condition called Eisenmenger syndrome. Those who have previously taken selexipag, have other serious illnesses that shorten life expectancy, are pregnant or breastfeeding, or are allergic to selexipag or its ingredients will also not be able to join. It's crucial to discuss all your child's medical history with the study doctors.

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.

Is your child between 2 and 17 years old?
Does your child have a confirmed diagnosis of pulmonary arterial hypertension (PAH)?
Is your child currently taking at least one standard PAH medication, with a stable dose for 3 months or more?
Does your child NOT have PAH due to liver problems, certain blood clots, or Eisenmenger syndrome?
Is your child NOT pregnant or breastfeeding, and has not previously taken selexipag?

Answer every question to see your result.

What does participation involve?

If your child participates, they will first go through a 'screening period' for up to six weeks. This is to check if they are suitable for the study. If they are, they will then enter the main 'double-blind treatment period'. In this part, neither you nor the doctors will know if your child is receiving selexipag or a dummy medicine called a placebo, in addition to their current PAH treatments. The dose of the study medicine will start low and be gradually increased (up-titration) to find the right level, followed by a 'maintenance' period where they take a stable dose.

After the double-blind part, there will be an 'open-label extension period' for three years. In this phase, all children will receive selexipag. Throughout the study, there will be regular check-ups to monitor your child's health, how the medicine is affecting them, and how well it is working. The study team will also collect blood samples. Following the end of all treatment, there will be a final safety check-up about a month later. The entire study, from start to finish, is expected to last about 8 years.

Potential risks and benefits

Participating in a clinical trial may offer potential benefits, such as access to a new medicine that could help manage your child's PAH, and closer medical monitoring of their condition. However, there are also potential risks involved. Your child might experience side effects from the study medicine, or the medicine might not be effective for them. Like with any medicine, there's a chance of allergic reactions. The study involves regular hospital visits, tests, and taking medication, which can be time-consuming. An independent group of experts will regularly check the study data to ensure the safety of all participants. You and your child are entirely free to withdraw from the study at any time, for any reason, without it affecting their usual medical care.

Locations (116)

Phoenix Children's Hospital
Verified postcode
Phoenix, United States
UCLA Medical Center
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Los Angeles, United States
UCSF
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San Francisco, United States
Childrens Hospital Colorado
Verified postcode
Aurora, United States
Children's National Medical Center
Verified postcode
Washington D.C., United States
Congenital Heart Center of the University of Florida
Verified postcode
Gainesville, United States
Riley Hospital for Children
Verified postcode
Indianapolis, United States
Detroit Medical Center
Verified postcode
Detroit, United States
Cincinnati Children's Hospital Medical Center
Verified postcode
Cincinnati, United States
Childrens Hospital Of Philadelphia
Verified postcode
Philadelphia, United States
Texas Children's Hospital
Verified postcode
Houston, United States
Primary Children's Hospital
Verified postcode
Salt Lake City, United States

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 through them.

What is selexipag?

Selexipag is a medicine already used for adults with PAH that helps open up blood vessels in the lungs. This study is testing it for children.

Will my child definitely get the new medicine?

In the first part of the study, your child will either get selexipag or a dummy medicine (placebo), in addition to their usual care. Neither you nor the doctors will know which one.

How long will the study last?

The study, including screening, treatment, and follow-up, is expected to last about 8 years in total.

Can my child stop participating at any time?

Yes, you can choose to withdraw your child from the study at any point, and it will not affect their regular medical care.