Riociguat in Children With Pulmonary Arterial Hypertension (PAH)

This study is looking for children and young people aged between 6 and 17 years who have pulmonary arterial hypertension (PAH). This includes PAH that has no known cause (idiopathic), PAH that runs in families (heritable), or PAH linked to other conditions like certain autoimmune diseases or heart defects that have been fixed over six months ago.

You must have had your PAH officially diagnosed through a special heart test called a right heart catheterisation, which confirms the pressures in your lungs are high enough for PAH. It's important that you are already receiving standard treatments for PAH, like certain medications called Endothelin Receptor Antagonists or Prostacyclin Analogues, and have been on them for at least 12 weeks before starting this study. We're looking for patients who need extra help with their condition, or newly diagnosed patients starting their first PAH medicines.

For teenage girls who could become pregnant, it's essential that a pregnancy test is negative before joining the study. You'll also need to agree to talk about safe sex practices and contraception to prevent pregnancy during the study.

If you decide to take part, you'll first have some initial checks to make sure the study is right for you. Then, you'll start taking the study medicine, riociguat. This will last for a main period of up to 24 weeks.

During the first 8 weeks, doctors will slowly adjust your dose of riociguat to find the best and safest amount for you. This is called the 'titration phase'. After that, you'll continue taking that dose for another 16 weeks in the 'maintenance phase'. Throughout this time, you'll have regular visits to the hospital or clinic. These visits will involve blood tests, physical exams, and other checks to see how you're doing and how the medicine is affecting you. After the main 24 weeks, there's an option to continue taking the study medicine in a longer-term part of the study, if both you and your doctor agree.