An Adaptive Program of IKT-001 in Pulmonary Arterial Hypertension (PAH)

To be part of this study, you need to be an adult between 18 and 75 years old and have a specific diagnosis of Pulmonary Arterial Hypertension (PAH), known as WHO Group 1. This includes types like PAH that happens without a clear cause, PAH that runs in families, or PAH linked to certain drugs, connective tissue diseases, or a repaired heart condition present since birth.

You also need to be on stable treatment for your PAH, such as specific medications like endothelin receptor antagonists, phosphodiesterase-5 inhibitors, prostacyclins, or soluble guanylate cyclase stimulators, for at least 3 months before joining. Your body mass index (BMI) should be between 18.5 and 35. You'll need to have had specific tests to measure the pressure in your lungs that show your condition fits the study requirements, and you should be able to walk a certain distance in six minutes (between 100 and 475 metres).

There are also reasons why you might not be able to join. For example, if you have other types of PAH (WHO Groups 2, 3, 4, or 5) or specific Group 1 types like HIV-associated PAH. You would also be excluded if you have very high or very low blood pressure, have fainted recently, or have certain heart problems or a history of specific heart rhythm issues. Having some implanted heart monitoring devices would also prevent you from taking part.

If you decide to take part in this study, you will be in it for a while, and it will involve several visits to the study clinic. First, there's a 'screening period' to make sure you're suitable for the study based on your health and tests. After this, if you qualify, you will start the main treatment phase.

During the study, you'll be given either the new medicine, IKT-001, or a placebo (a dummy drug). You'll need to take this medicine regularly as instructed. You'll have scheduled visits to the clinic for various health checks. These will likely include physical examinations, blood tests, heart checks like ECGs, and repeat lung pressure measurements. You may also be asked to do walking tests (like the 6-minute walk test) to see how your symptoms are changing.

The study team will carefully monitor your health and any side effects you might experience. It's important to attend all your appointments and report any new symptoms or concerns. The total length of your participation, including follow-up, will be explained fully by the study team, but these types of studies can often last for many months.