A Phase 3, Randomized, DoubleBlind, Placebo-controlled, Multicenter, Parallel-group Study to Evaluate the Efficacy and Safety of Once Daily Treprostinil Palmitil Inhalation Powder in Participants with Pulmonary Arterial Hypertension (PALM-PAH)
Pulmonary arterial hypertension (PAH) is a condition where high blood pressure in the lungs makes it difficult for the heart to pump blood, causing breathlessness and fatigue. Existing inhaled treatments often need to be taken several times a day, which can be disruptive. This Phase 3 clinical study tests an experimental treatment called treprostinil palmitil inhalation powder. It is designed to widen blood vessels in the lungs while needing to be inhaled only once daily. Researchers are comparing this new powder against a placebo (an identical-looking dummy powder with no active medicine). They will measure how far participants can walk in six minutes after 24 weeks of treatment. They will also track changes in daily symptoms, blood markers, and overall heart health. The goal is to find out if this once-daily inhaler is safe, effective, and easier for people living with PAH to use.
At a glance
What is this study about?
Pulmonary arterial hypertension (PAH) is a serious, long-term condition caused by narrowing and stiffening of the blood vessels that carry blood from the heart to the lungs. This raises blood pressure inside the lungs and forces the right side of the heart to work much harder than normal. Over time, people with PAH can experience severe breathlessness, chest discomfort, dizziness, and extreme tiredness during everyday activities.
Treprostinil is an established type of medicine known as a prostacyclin analogue. It acts like a natural substance in the body that relaxes and widens tight blood vessels, lowering lung pressure and easing the strain on the heart. While some inhaled versions of treprostinil already exist, they typically require multiple doses throughout the day. This trial, called PALM-PAH, is testing a new formulation called treprostinil palmitil, which stays in the lungs longer and only needs to be inhaled once a day.
In this study, participants will be randomly chosen to receive either the active medicine or an inactive dummy powder (placebo). Over 24 weeks, researchers will measure improvements in exercise capacity using a six-minute walking test. They will also look at heart-related blood tests, questionnaires about daily life and breathing, and whether the medicine helps keep patients out of hospital. If proven successful, this once-daily inhaler could offer a more manageable and effective treatment option for people living with PAH.
Key takeaways
- The PALM-PAH trial is testing a once-daily inhaled powder for pulmonary arterial hypertension.
- The main aim is to see if the medicine helps people walk further in a six-minute walk test after 24 weeks.
- Participants have an equal chance of receiving either the active study drug or an inactive placebo powder.
- The study is for adults aged 18 and older who have a confirmed diagnosis of PAH.
- Joining is entirely voluntary, and you can leave the study at any stage without affecting your routine medical care.
Who may be eligible?
This trial is open to adults aged 18 and older who have been diagnosed with pulmonary arterial hypertension (PAH). Both men and women can take part.
To be considered, participants must be able to perform routine clinic walking tests and be willing to use an inhaler once every day. In most cases, participants will already be receiving standard, approved medications for their PAH and must be on a steady dose before starting the study.
You may not be eligible if you have other serious lung or heart conditions, if your PAH is caused by certain other medical issues, or if you have recently started or changed major heart therapies. Women who are pregnant or breastfeeding are generally unable to take part. The study doctor will carry out a thorough medical review to ensure it is completely safe for you to join.
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 have been diagnosed with pulmonary arterial hypertension (PAH).
- I am currently on stable medical treatment for my condition.
- I am able to walk and perform a six-minute walking test.
- I am able and willing to use a daily dry powder inhaler.
- I am not currently pregnant or breastfeeding.
What does participation involve?
Taking part in this study lasts for at least 24 weeks. If you join, you will be randomly assigned by a computer to receive either the active treprostinil palmitil powder or a matching placebo powder. Neither you nor your doctor will know which one you are receiving. You will inhale the powder once a day using a small dry powder inhaler device. Throughout the study, you will attend regular hospital appointments so the team can monitor your health. These visits will include six-minute walk tests, blood tests, heart checks (such as ECGs or scans), and questionnaires asking about your breathing and how PAH affects your daily routine. You will be closely supported by the study team, and you can reach out to them between visits if you feel unwell.
Potential risks and benefits
Locations (12)
- —UnverifiedGermany
- —UnverifiedHungary
- —UnverifiedBelgium
- —UnverifiedFrance
- —UnverifiedItaly
- —UnverifiedSweden
- —UnverifiedAustria
- —UnverifiedPoland
- —UnverifiedLithuania
- —UnverifiedSpain
- —UnverifiedRomania
- —UnverifiedGreece
Common questions
What is a placebo and why is it used in this study?
A placebo is a dummy treatment designed to look, smell, and taste identical to the real medicine, but it contains no active drug. Comparing the active powder to a placebo helps researchers clearly see whether health improvements are genuinely caused by the new medicine.
Will I have to stop taking my current PAH medicines?
No. In trials like this, the study treatment is usually added to your existing, stable background therapies. Your study doctor will review your current prescriptions to confirm they are safe to continue.
How is the study medicine taken?
It is taken once daily by breathing in a fine dry powder from a small capsule using a handheld inhaler device, similar to standard asthma or COPD inhalers.
Does taking part in this clinical trial cost anything?
No. The study medication, inhaler devices, and all trial-related tests are provided free of charge. Most studies also provide reimbursement for your travel expenses to and from the hospital.
What happens if my condition gets worse during the trial?
Your safety is the top priority. The medical team tracks your health closely. If your PAH gets worse, your doctors will intervene immediately and may adjust your treatment or withdraw you from the trial to give you the care you need.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
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