Ongoing, recruitingTherapeutic confirmatory (Phase III)Interventional

A Phase 3, Randomized, Double-Blind, Placebo_Controlled Study to Evaluate Sotatercept When Added to Maximum Tolerated Background Therapy in Participants With Pulmonary Arterial Hypertension (PAH) World Health Organization (WHO) Functional Class (FC) III or FC IV at High Risk of Mortality

This study is looking at a new medication called sotatercept for people aged 18 and over who have a serious lung condition called pulmonary arterial hypertension (PAH). PAH makes it difficult for your heart to pump blood through the lungs, causing breathlessness and tiredness. Participants will be those whose PAH is quite advanced. The study aims to find out if adding sotatercept to their current PAH treatments can help improve their health. Specifically, researchers will be checking if it can help people live longer, avoid needing a lung transplant, or prevent hospital stays due to their PAH getting worse. This is a "Phase 3" study, meaning it's a later stage of testing, often comparing the new medicine to a dummy treatment (placebo) to confirm its benefits and safety.

At a glance

Status

Ongoing, recruiting

Phase

Therapeutic confirmatory (Phase III)

Sponsor

Acceleron Pharma Inc.

Enrolment target

Start

27 Aug 2024

Pulmonary Arterial Hypertension (PAH)

What is this study about?

This study is named a "Phase 3 randomized, double-blind, placebo-controlled study" looking into a new treatment called sotatercept for people with an advanced lung condition called Pulmonary Arterial Hypertension (PAH). PAH is a serious condition where the blood vessels in the lungs become narrow, making it hard for the heart to pump blood through them. This can lead to symptoms like breathlessness, tiredness, and chest pain, and can put a strain on the heart. The study focuses on people whose PAH is quite severe, meaning they are at a higher risk of their condition worsening.

The main goal of this study is to see if adding sotatercept to the medications people are already taking for their PAH can make a difference. The researchers will be carefully observing participants to see if taking sotatercept helps them live longer, avoid needing a lung transplant (which is sometimes necessary for severe PAH), or prevents them from needing to go to hospital because their PAH has got worse. They will also look at how sotatercept affects overall survival.

To make sure the study results are fair and accurate, some participants will receive sotatercept, while others will receive a 'dummy' treatment called a placebo. Neither the patients nor their doctors will know who is getting which treatment until after the study is over. This is called 'double-blind' and helps prevent any bias. The study will also be looking at several other important health markers, such as how far people can walk in six minutes, their quality of life, and various measurements related to their heart and lung function, to get a full picture of the treatment's effects. The hope is that sotatercept could offer a new way to help people manage their PAH more effectively.

Key takeaways

This study tests a new medicine, sotatercept, for severe PAH.
It aims to improve survival, prevent transplants, and reduce hospital stays.
Participants will get either sotatercept or a placebo, added to their current PAH treatments.
The study involves regular clinic visits and health checks.
It's for adults (18+) with advanced PAH who are already on standard treatment.

Who may be eligible?

To be considered for this study, you would need to meet certain requirements. The study is open to both men and women, and you must be at least 18 years old – there's no upper age limit, meaning older adults are also welcome to participate if they meet the other criteria.

Crucially, you must have Pulmonary Arterial Hypertension (PAH) that is considered quite advanced, specifically if your doctor has described it as WHO Functional Class III or IV. This means your PAH is significantly affecting your daily activities and you are considered to be at a higher risk of serious health problems. You also need to already be on the best possible treatment for your PAH that your doctor recommends and that you can tolerate.

There will be other detailed medical checks to make sure the study is a good fit for you and that it is safe for you to take part. This will involve your study doctor reviewing your full medical history and current health status.

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)?
Has your doctor told you your PAH is quite advanced (WHO Functional Class III or IV)?
Are you currently taking standard medications for your PAH that you can tolerate?

Answer every question to see your result.

What does participation involve?

If you decide to join this study, you would be taking a new medication called sotatercept, which is given as an injection. This medication would be added to your current PAH treatments. The study is designed to compare sotatercept to a 'dummy' treatment (placebo), so you would have an equal chance of receiving either. Neither you nor your study doctor would know which one you are receiving during the study.

Taking part would involve regular visits to the study clinic for assessments. These visits would include physical examinations, blood tests, heart and lung function tests (like walking tests), and questionnaires about how you are feeling and your quality of life. The treatments and assessments would continue over a specific period, followed by a period of careful follow-up to monitor your health. The total duration of your involvement could be quite long, potentially lasting for several years, to fully assess the long-term effects of the treatment and understand key health outcomes.

Potential risks and benefits

Taking part in any medical study has potential benefits and risks. You might benefit from receiving a new treatment (sotatercept) that could improve your PAH, or you might find that the close monitoring you receive throughout the study helps your overall care. However, like all medications, sotatercept may have side effects, which would be explained to you in detail by the study team. Since you might receive a placebo, there's also a possibility you won't get the active treatment. You are always free 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.

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Unverified
Belgium
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Unverified
Netherlands
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Unverified
Germany
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Unverified
Italy
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Unverified
France
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Unverified
Spain

Common questions

What is Pulmonary Arterial Hypertension (PAH)?

PAH is a serious condition where blood vessels in the lungs become narrow, making it hard for the heart to pump blood through them, leading to breathlessness and tiredness.

What is sotatercept?

Sotatercept is an investigational new medicine being tested to see if it can improve the health of people with PAH.

What does 'Phase 3' mean for a study?

Phase 3 means this is a later stage of testing, often comparing a new medicine to existing treatments or a dummy treatment (placebo) to confirm its effectiveness and safety.

What is a 'placebo'?

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

How is sotatercept given?

Sotatercept is given as an injection, which you would receive as part of the study.