Ongoing, recruitingPhase II and Phase III (Integrated)Interventional

A Phase 2/3, Multicenter, Randomized, Double-blind, Placebo-Controlled, Adaptive Design Study to Evaluate the Efficacy and Safety of MK-5475 in Adults with Pulmonary Arterial Hypertension

This research study is investigating a new drug, MK-5475, for adults living with pulmonary arterial hypertension (PAH). PAH is a condition where the blood vessels in your lungs become narrowed, making it harder for your heart to pump blood, which can make you feel breathless and tired. The study aims to find out if MK-5475 is safe and effective in improving some of the key symptoms and measurements of PAH. Researchers will compare MK-5475 to a 'placebo' (a dummy drug). They'll measure things like how far people can walk in six minutes and improvements in how the heart and lungs are working. This is a crucial step in hopefully finding new ways to help those affected by PAH.

At a glance

Status

Ongoing, recruiting

Phase

Phase II and Phase III (Integrated)

Sponsor

Merck Sharp & Dohme LLC

Enrolment target

Start

07 Jun 2023

Pulmonary arterial hypertension (PAH) in one of the following groups: Idiopathic PAH Heritable PAH Drug and toxin-induced PAH PAH associated with connective tissue disease HIV infection or congenital heart disease

What is this study about?

This study is exploring a new medicine called MK-5475, for adults who have a condition called pulmonary arterial hypertension (PAH). PAH affects the blood vessels in your lungs, causing them to narrow. This means your heart has to work much harder to pump blood through your lungs, which can lead to symptoms like breathlessness, tiredness, and dizziness. The purpose of this study is to carefully look at whether MK-5475 is safe to use and if it can help improve the health and well-being of people with PAH.

This is a 'Phase 2/3' study, which means it combines two important stages of research. In the Phase 2 part, the main goal is to carefully check how the medicine affects the pressure in the lung's blood vessels. In the Phase 3 part, the focus shifts to whether the medicine helps people feel better in their day-to-day lives, for example, by improving how far they can walk. To ensure the results are fair and reliable, some participants will receive the new medication (MK-5475), while others will receive a 'placebo' – which looks like the study drug but contains no active medicine. This comparison helps researchers understand if any improvements are genuinely due to the new medication.

The study involves various types of PAH, including those that are inherited, caused by certain drugs or toxins, or linked to other conditions like connective tissue disease, HIV, or specific heart problems you were born with. By conducting this research, doctors hope to gain a better understanding of how MK-5475 works and if it could become a new treatment option to help people with PAH live healthier and more comfortable lives.

Key takeaways

Investigating a new drug, MK-5475, for pulmonary arterial hypertension (PAH).
Compares MK-5475 to a placebo (dummy drug).
Aims to check safety and how it affects lung function and walking ability.
Open to adults 18+ with various types of PAH.
Participation involves regular clinic visits and health checks over several months.
You can withdraw from the study at any time.

Who may be eligible?

To join this study, you must be an adult aged 18 years or older. The study is open to both men and women.

You would need to have been diagnosed with pulmonary arterial hypertension (PAH). This includes different types of PAH, such as if the cause isn't known (idiopathic), if it runs in your family (heritable), or if it's caused by certain medicines, chemicals, or other health conditions like connective tissue disease, HIV, or certain heart problems you were born with.

There will be other detailed criteria that doctors check to make sure the study medication is suitable and safe for you, and that the research results will be accurate. If you are interested, a study doctor will discuss all these requirements with you to see if you might be a good fit.

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.

Are you 18 years old or older?
Have you been diagnosed with pulmonary arterial hypertension (PAH)?
Is your PAH idiopathic, heritable, or linked to drug/toxin use, connective tissue disease, HIV, or heart problems you were born with?
Are you able to attend regular clinic appointments for several months?
Are you willing to potentially receive either the new drug or a placebo?

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, you will be assigned by chance to either receive the new medicine (MK-5475) or a placebo (a dummy treatment). Neither you nor your study doctor will know which one you are receiving – this is to make sure the results are as unbiased as possible. Over a total period of 12 to 24 weeks, you'll have regular visits to the study clinic.

During these visits, the study team will carry out various health checks and tests. These might include measurements of your lung and heart function, how far you can walk in six minutes, and blood tests. You will also be asked about how you are feeling and if you have experienced any side effects. All of these assessments are important to track how the medication is working and to ensure your safety throughout the study. After the main treatment period, there will be follow-up appointments to continue monitoring your health.

Potential risks and benefits

Participating in this study might offer you a potential benefit by giving you access to a new experimental medication (MK-5475) that could potentially improve your PAH symptoms. However, as with any new drug, there are potential risks, including side effects that are not yet fully known. For those receiving the placebo, there's no direct treatment benefit from the study drug itself. All known and potential risks will be thoroughly explained to you before you decide to join. Remember, your participation is completely voluntary, and you have the right to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (7)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

—
Unverified
Greece
—
Unverified
Belgium
—
Unverified
Italy
—
Unverified
Poland
—
Unverified
France
—
Unverified
Sweden
—
Unverified
Germany

Common questions

What is pulmonary arterial hypertension (PAH)?

PAH is a serious condition where blood vessels in your lungs become narrow, making it hard for your heart to pump blood, causing shortness of breath and tiredness.

What is MK-5475?

MK-5475 is a new medication being tested in this study to see if it can help improve the health of adults with PAH.

What is a 'placebo'?

A placebo is a 'dummy' treatment that looks like the study drug but contains no active medicine. It helps researchers compare the new drug's effects.

How long will the study last?

The main treatment and assessment period will last between 12 and 24 weeks, followed by further follow-up appointments.

Can I leave the study if I change my mind?

Yes, you can choose to leave the study at any point, and it won't affect your ongoing medical care.