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.
במבט חטוף
על מה המחקר הזה?
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.
נקודות עיקריות
- 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.
- ההשתתפות היא בהתנדבות, ואתה יכול לפרוש בכל עת.
מי עשוי להיות זכאי?
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.
האם מחקר זה יכול להתאים לך?
ענה על השאלות המהירות הללו כדי לבדוק אם אתה עשוי להיות זכאי. זהו מדריך בלבד — צוות המחקר מקבל את ההחלטה הסופית.
- 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?
מה כרוך בהשתתפות?
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.
סיכונים ותועלות פוטנציאליים
מיקומים (116)
- בית החולים לילדים פיניקסמיקוד מאומתפניקס, ארצות הברית
- UCLA Medical Centerמיקוד מאומתלוס אנג'לס, ארצות הברית
- UCSFמיקוד מאומתסן פרנסיסקו, ארצות הברית
- בית החולים לילדים קולורדומיקוד מאומתאורורה, ארצות הברית
- המרכז הרפואי הלאומי לילדיםמיקוד מאומתוושינגטון די.סי., ארצות הברית
- Congenital Heart Center of the University of Floridaמיקוד מאומתגיינסוויל, ארצות הברית
- Riley Hospital for Childrenמיקוד מאומתאינדיאנפוליס, ארצות הברית
- Detroit Medical Centerמיקוד מאומתדטרויט, ארצות הברית
- מרכז רפואי בית חולים לילדים סינסינטימיקוד מאומתסינסינטי, ארצות הברית
- Childrens Hospital Of Philadelphiaמיקוד מאומתפילדלפיה, ארצות הברית
- בית החולים לילדים של טקססמיקוד מאומתיוסטון, ארצות הברית
- Primary Children's Hospitalמיקוד מאומתסולט לייק סיטי, ארצות הברית
שאלות נפוצות
מהי יתר לחץ דם ריאתי עורקי (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.
כמה זמן יימשך המחקר?
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.
כיצד לגלות עוד
תמיד התייעץ עם רופא המשפחה או המומחה שלך לפני שתחליט להשתתף במחקר.
דיון
דיון קהילתי
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