A Clinical Study of to Confirm the Doses of Selexipag in Children With Pulmonary Arterial Hypertension
This study is looking at a medicine called selexipag, also known as Uptravi, for children aged 2 to 17 who have a serious lung condition called pulmonary arterial hypertension (PAH). The main aim is to figure out the best starting dose for these children. We want to make sure children get a similar amount of the medicine in their body as adults do, while being safe and effective. Researchers will look at how the body handles the medicine in different age groups to confirm the doses. The study will involve around 60 children and will help ensure that selexipag can be used safely and effectively in younger patients with PAH.
At a glance
What is this study about?
This study is about a medicine called selexipag, also known as Uptravi. It's for children aged 2 to 17 who have a condition called pulmonary arterial hypertension (PAH). PAH affects the blood vessels in the lungs, making them narrow and stiff, which makes it harder for the heart to pump blood. This can lead to tiredness, breathlessness, and other serious problems.
The main goal of this study is to find the right starting dose of selexipag for children. This is important because children's bodies handle medicines differently from adults. Researchers want to make sure that children receive a similar amount of the medicine in their system as adults do, to get the best treatment effect without causing too many side effects. They will closely watch how the medicine works in the body at different starting doses.
The study will involve about 60 children, split into three age groups. The research team will carefully check the medicine's levels in the body to make sure the doses are correct and safe for each age group. This careful approach helps doctors confidently prescribe the correct amount of selexipag for children with PAH in the future.
Key takeaways
- This study is testing selexipag (Uptravi) in children with pulmonary arterial hypertension (PAH).
- The main goal is to find the correct starting dose for children aged 2-17.
- About 60 children will take part, split into three age groups.
- Doses will be gradually increased to find the best amount for each child.
- Close medical checks and monitoring will happen throughout the study.
- You can withdraw your child from the study at any time.
Who may be eligible?
To join this study, children must be between 2 and 17 years old and weigh at least 9 kilograms (about 20 pounds). They must have been diagnosed with pulmonary arterial hypertension (PAH). This diagnosis needs to be confirmed by a special heart test called a right heart catheterization at some point before the study begins. Children can have different types of PAH, including those that are inherited, those linked to heart problems from birth, or those caused by certain drugs or other illnesses.
Children currently taking other PAH medicines like ERA or PDE-5 inhibitors can join, as long as they've been on a stable dose for at least three months. If a girl who can have children is thinking of joining, she must have a negative pregnancy test, agree to monthly tests, and use reliable contraception if she is sexually active throughout the study and for 30 days afterwards.
However, some conditions mean a child can't take part. This includes certain types of PAH like those related to liver problems or specific advanced heart conditions. Children with moderate to large holes in their heart that cause blood to flow incorrectly, or those with very complex heart conditions from birth, like certain 'blue baby' conditions or after a specific heart operation called a Fontan procedure, will not be able to join.
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 aged between 2 and 17 years old?
- Does your child weigh at least 9 kilograms (about 20 pounds)?
- Has your child been diagnosed with pulmonary arterial hypertension (PAH)?
- If your child is a girl who could become pregnant, will she use reliable contraception if sexually active and have monthly pregnancy tests?
- Does your child NOT have certain specific severe heart defects or types of PAH (e.g., related to liver problems or complex congenital heart conditions)?
- Is your child currently taking stable doses of other PAH medicines (ERA or PDE-5 inhibitors), or are they not suitable for these therapies?
What does participation involve?
If your child takes part, they will receive the study medicine, selexipag, in tablet form. Initially, the dose will be low and gradually increased over the first 12 weeks to find the best dose for your child. This is called 'up-titration' and is similar to how adults start on this medication.
Children will be grouped into one of three age ranges: 12 to 17 years, 6 to 11 years, or 2 to 5 years. The starting dose will depend on your child's body weight. Throughout the study, there will be regular visits to the clinic where doctors and nurses will do check-ups, take blood samples to measure the medicine's levels, and monitor your child's overall health and any side effects. These assessments help ensure the medicine is working and is safe. The study will likely involve routine clinic visits, but the exact number and frequency will be explained by the study team. We aim to find the right dose for children, and the total duration your child will be in the study will be explained in detail by the study team.
Potential risks and benefits
Locations (34)
- Children'S Hospital Cardiac Care Center University Of ColoradoVerified postcodeAurora, United States
- University of Iowa HospitalVerified postcodeIowa City, United States
- Seattle Children's HospitalVerified postcodeSeattle, United States
- State Institution Republican Scientific And Practical Center For Pediatric SurgeryVerified postcodeMinsk, Belarus
- Health Institution 4Th City Children'S Clinical HospitalVerified postcodeMinsk, Belarus
- UZ GentVerified postcodeGhent, Belgium
- Centre Hospitalier Sainte JustineVerified postcodeMontreal, Canada
- Beijing Anzhen HospitalVerified postcodeBeijing, China
- Shanghai Childrens Medical CenterVerified postcodeShanghai, China
- CHU Arnaud de VilleneuveVerified postcodeMontpellier, France
- Hôpital Necker - Enfants MaladesVerified postcodeParis, France
- Chu Hopital Des EnfantsVerified postcodeToulouse, France
Common questions
What is pulmonary arterial hypertension (PAH)?
PAH is a rare but serious lung condition where blood vessels in the lungs become narrowed, making it harder for the heart to pump blood, leading to breathlessness and tiredness.
What is selexipag (Uptravi)?
Selexipag is a medicine used to treat PAH. It works by relaxing and widening the blood vessels in the lungs to improve blood flow.
Why is this study needed for children?
Children's bodies process medicines differently from adults. This study helps find the correct and safest starting dose of selexipag for children with PAH.
Will my child know which dose they are getting?
The study team will explain how the starting dose is chosen for your child based on their age and weight, and how it will be carefully increased.
Can I take my child out of the study if we change our minds?
Yes, you can withdraw your child from the study at any time without giving a reason, and it will not affect their regular medical care.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
Discussion
Community discussion
Powered by our forum at community.patient.info. Please be respectful — this is not medical advice.