Ongoing, recruitingTherapeutic exploratory (Phase II)Interventional

A Phase 2b, Randomized, Double-Blind, Multicenter, Placebo-Controlled Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of Treprostinil Palmitil Inhalation Powder in Participants with Pulmonary Arterial Hypertension

This study is looking into a new inhaled powder called Treprostinil Palmitil to treat a serious lung condition known as pulmonary arterial hypertension (PAH). In PAH, the blood vessels in your lungs become narrow, making it hard for your heart to pump blood, leading to shortness of breath and tiredness. This research is in an early stage (Phase 2b), meaning it's still exploring how well the medicine works and if it's safe. Participants will either receive one of three strengths of the new medicine or a placebo (a powder with no active medicine). Doctors will closely monitor health changes, side effects, and how the body handles the medicine over 16 weeks to understand its potential benefits and risks.

At a glance

Status

Ongoing, recruiting

Phase

Therapeutic exploratory (Phase II)

Sponsor

Insmed Inc.

Enrolment target

Start

19 Jun 2024

Pulmonary Arterial Hypertension

What is this study about?

This study is about a new medicine in powder form, inhaled through a device, for people with a serious lung condition called pulmonary arterial hypertension, or PAH. In PAH, the blood vessels in your lungs become thick and narrow, which makes it very hard for your heart to pump blood through them. This can lead to symptoms like feeling out of breath, tired, and having chest pain.

The new medicine being tested is called Treprostinil Palmitil Inhalation Powder. The main goal of this study is to see how well this new medicine works to improve the condition of your blood vessels in your lungs and if it's safe to use. Researchers are also trying to figure out the right amount of medicine to give to get the best results without too many side effects.

This is a 'Phase 2b' study, which means it's an important step where the medicine is given to a group of patients to explore its effectiveness and safety in more detail, after earlier general safety tests. We hope to understand if this medicine could be a good treatment option for people living with PAH in the future.

Key takeaways

This study is testing a new inhaled medicine for pulmonary arterial hypertension (PAH).
It aims to see how well the medicine works and if it's safe.
Participants will receive either the new medicine or a dummy powder.
The study lasts 16 weeks and involves regular check-ups.
It's open to adults aged 18 and over with PAH.
Your health will be closely monitored throughout the study.

Who may be eligible?

To join this study, you must be at least 18 years old. Both men and women can take part. You also need to have been diagnosed with pulmonary arterial hypertension.

There might be other specific health requirements or conditions that would mean you couldn't join, which a doctor will explain in detail. For example, certain other medical conditions or taking specific medications might prevent you from participating.

It's important to remember that the study team will review your medical history carefully to make sure the study is a safe and suitable option for 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.

Are you 18 years old or older?
Do you have a diagnosis of pulmonary arterial hypertension (PAH)?
Are you able to attend regular clinic visits for 16 weeks?
Are you comfortable with the idea of taking an inhaled medicine?
Are you willing to have medical tests like blood tests and ECGs?

Answer every question to see your result.

What does participation involve?

If you join this study, it will involve several visits to a clinic over a period of 16 weeks. You'll be given either the new inhaled medicine or a placebo powder (which looks the same but has no active medicine) to take regularly. Neither you nor your doctor will know which one you are receiving.

During your visits, you will have regular health checks. This could include things like walking tests (a 6-minute walk test to see how far you can walk in that time), blood tests, checking your blood pressure and heart rate, and an electrocardiogram (ECG) to monitor your heart. You'll also be asked about any side effects or how you are feeling. Throughout the 16 weeks, doctors will closely monitor your health to see how the medicine is affecting you and if you experience any side effects.

Potential risks and benefits

Taking part in this study could potentially offer some benefits, such as receiving a new medicine that might improve your condition, or receiving close medical attention and monitoring. However, as with any new medicine, there are potential risks, including experiencing side effects that could be mild or more serious. The full list of potential side effects will be discussed with you by the study doctor. You have the right to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (6)

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

—
Unverified
Belgium
—
Unverified
Italy
—
Unverified
Spain
—
Unverified
Denmark
—
Unverified
Austria
—
Unverified
Germany

Common questions

What is pulmonary arterial hypertension (PAH)?

PAH is a serious condition where the blood vessels in your lungs become narrowed, making it hard for blood to flow through. This causes your heart to work harder and can lead to shortness of breath and tiredness.

What does 'inhalation powder' mean?

It means the medicine comes in a very fine powder form that you breathe in using a special device, so it goes directly into your lungs.

What is a 'placebo'?

A placebo is a dummy medicine that looks exactly like the real medicine but contains no active ingredients. It helps researchers compare the effects of the new medicine against no treatment.

How long will I be in the study?

The main treatment period for this study is 16 weeks, and during this time you will have several clinic visits for check-ups and tests.

What is a '6-minute walk test'?

This is a simple test where you walk as far as you can in 6 minutes. It helps doctors see how your lung and heart function are affecting your ability to exercise.