C5001001 - A Phase 1/2, Randomized, Double-blind, Placebo-controlled Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Single Escalating Doses of PF-07868489 in Healthy Adult Participants And, Additionally, Clinical Activity of Repeat Doses in Participants With Pulmonary Arterial Hypertension
Pulmonary arterial hypertension (PAH) is a condition that causes high blood pressure in the blood vessels supplying the lungs, making it hard to breathe and causing tiredness. This study is testing an experimental medicine called PF-07868489 to see if it is safe and effective. The trial is split into two parts. First, healthy adult volunteers receive single doses to check how the body handles the drug and to spot any side effects. Next, adults with PAH receive repeated doses over 24 weeks. Researchers want to see if the drug lowers blood pressure in the lungs and helps people walk further in six minutes. Participants are randomly chosen to receive either the study drug or a dummy treatment (placebo). Everyone is closely looked after by doctors with regular heart tests, blood tests, and health checks throughout the study.
At a glance
What is this study about?
Pulmonary arterial hypertension, often shortened to PAH, is a serious condition where the blood vessels carrying blood from the heart to the lungs become stiff and narrow. This raises blood pressure inside the lungs and forces the right side of the heart to work much harder. Over time, everyday tasks like walking up stairs or carrying shopping can make people feel breathless, dizzy, and exhausted.
This clinical trial is testing a new drug known as PF-07868489. Scientists hope this medicine may help relax lung blood vessels, relieve strain on the heart, and improve quality of life. However, before any new medicine can become widely available, it must be carefully tested in humans to see how it works and whether it causes any unwanted side effects.
The study has two key stages. In the first stage, healthy volunteers take single, carefully measured doses so doctors can track how the body absorbs and clears the medicine. In the second stage, people living with PAH receive regular doses for 24 weeks. Doctors will measure whether the medicine lowers lung resistance and helps participants walk further during a timed walking test.
Because this is an early-stage study, the primary focus is participant safety. The trial uses a 'placebo control', which means some people will receive an inactive dummy medicine instead of the real drug. This is the gold standard method to ensure that any health improvements or side effects are truly caused by the study medicine.
Key takeaways
- Tests a new medicine (PF-07868489) for pulmonary arterial hypertension.
- Open to adults aged 18 and over, including healthy volunteers and people with PAH.
- Compares the active drug against a dummy medicine (placebo).
- Tracks safety, blood flow in the lungs, and walking distance over 24 weeks.
- Taking part is voluntary, and you can withdraw at any time.
Who may be eligible?
This trial is designed for adults aged 18 and over. Because it is split into different stages, the study team is looking for two different types of participants.
The first group includes healthy adult volunteers who do not have major underlying health problems. These volunteers help researchers understand the basic safety of the drug before it is tested more widely.
The second group includes adults who have been diagnosed with pulmonary arterial hypertension. If you have PAH, the study doctors will check whether your current health, lung function, and routine medicines make you a suitable match for the trial.
There are detailed rules about who can join. For instance, pregnant or breastfeeding women may not be able to take part, and you may be excluded if you have certain other heart or lung conditions. The trial team will review your full medical history with you.
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.
- I am aged 18 or older.
- I am either a healthy adult volunteer OR I have a diagnosed case of pulmonary arterial hypertension (PAH).
- I am willing to attend regular hospital appointments for check-ups and tests.
- I am able to undergo blood tests, heart tracings (ECGs), and walking tests.
- I understand that I might receive a placebo rather than the active medicine.
What does participation involve?
Taking part in this study involves several stages over a period of roughly six months or more. First, you will attend a screening visit where doctors check your heart, lungs, and general health with blood tests and heart tracings (ECGs) to see if the study is safe for you.
If you join the PAH part of the study, you will take the study medicine or a placebo for 24 weeks. You will need to attend regular clinic visits for health checks. These include checking your vital signs (such as blood pressure and pulse), having blood samples drawn, and completing a 6-minute walk test to see how far you can comfortably walk.
Doctors will also measure the blood pressure and resistance inside your lung vessels to see if the medicine is working. You will be closely supported by a dedicated research team, and you will have follow-up checks after you finish taking the study medicine to ensure you remain well.
Potential risks and benefits
Locations (7)
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- —UnverifiedSpain
Common questions
What is a placebo?
A placebo looks and tastes just like the study drug but contains no active medicine. It allows doctors to see whether changes in health are genuinely caused by the new drug.
Will I have to pay to take part?
No, clinical trials in the UK do not cost you anything. The study medication, tests, and doctor visits are provided free of charge, and travel expenses are often covered.
Can I leave the study if I change my mind?
Yes, taking part is completely voluntary. You can stop participating at any point, and your normal healthcare will continue as usual.
What is the 6-minute walk test?
It is a simple, safe test where you walk at your own pace along a flat corridor for six minutes. It helps doctors measure your stamina and lung capacity.
Will I know if I am taking the real medicine or the placebo?
No, this is a 'double-blind' trial. Neither you nor the study doctors will know which treatment you are receiving until the study is finished, ensuring fair results.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
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