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Ongoing, recruitingTherapeutic confirmatory (Phase III)Interventional

A Phase 3, Randomized, Double-blind, Placebo-controlled Study to Evaluate Sotatercept When Added to Background Pulmonary Arterial Hypertension (PAH) Therapy in Newly Diagnosed Intermediate- and High-risk PAH Patients

This important study is investigating a new medication called sotatercept for individuals who have recently received a diagnosis of Pulmonary Arterial Hypertension (PAH) and are considered to have an intermediate or high risk. PAH is a serious condition affecting the blood vessels in the lungs. Participants will be randomly assigned to receive either sotatercept or a placebo (a dummy medicine with no active ingredients), alongside their usual PAH treatments. The main goal is to see if sotatercept can help prevent serious health events like hospital stays, the need for lung transplants, or worsening of the condition, and if it improves how people feel and function over time. This is a large-scale study designed to confirm the medicine's effectiveness and safety.

At a glance

Status
Ongoing, recruiting
Phase
Therapeutic confirmatory (Phase III)
Sponsor
Acceleron Pharma Inc.
Enrolment target
253
Start
24 Sep 2024

What is this study about?

This research study is about a new medicine called sotatercept for people who have recently been diagnosed with a lung condition called Pulmonary Arterial Hypertension, or PAH. PAH makes it harder for your heart to pump blood through your lungs, which can make you feel tired and out of breath. If you've been told you have an intermediate or high risk with your PAH, this study might be especially relevant.

In this study, some people will receive the new medicine, sotatercept, while others will receive a placebo, which looks just like the study medicine but doesn't have any active ingredients. This is a common way to test new medicines fairly, so we can see if the new medicine really makes a difference compared to not taking it. Both groups will also continue to receive their usual PAH medications.

Researchers want to see if sotatercept can help prevent serious problems that can happen with PAH, such as needing to go to the hospital, needing a lung transplant, or if your condition gets worse. They will also look at how sotatercept affects your physical abilities, how you feel day-to-day, and other important health markers. This is a 'Phase 3' study, which means it's one of the final steps before a medicine might be made available to everyone, aiming to confirm its benefits and safety on a larger group of patients.

Key takeaways

  • Tests a new medicine, sotatercept, for recently diagnosed PAH.
  • Compares sotatercept to a dummy medicine (placebo).
  • Looks to prevent serious health events and improve well-being.
  • Participants continue their usual PAH treatments.
  • Suitable for people aged 18+ with intermediate to high-risk PAH.
  • A Phase 3 study, aiming to confirm effectiveness and safety.

Who may be eligible?

To join this study, people generally need to be 18 years old or older. The study is open to both men and women.

It's specifically for those who have been diagnosed with Pulmonary Arterial Hypertension (PAH) relatively recently. You would also be considered for the study if your doctors believe your PAH is at an intermediate or high risk. This helps researchers focus on people who might benefit most from a new treatment.

There will be other specific health requirements that your study doctor will discuss with you to make sure the study is a good fit for your health and safety.

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.

  1. Are you 18 years old or older?
  2. Have you been diagnosed with Pulmonary Arterial Hypertension (PAH)?
  3. Has your doctor told you that your PAH is considered intermediate or high risk?
  4. Are you able to continue using your current background PAH treatments?
Answer every question to see your result.

What does participation involve?

If you decide to join this study, you'll be given either the study medicine, sotatercept, or a dummy medicine called a placebo. You won't know which one you're getting, and neither will your study doctor. You'll continue to take your usual PAH medications throughout the study.

Taking part will involve regular visits to the clinic for check-ups. During these visits, you might have blood tests, heart checks, and exercise tests to see how you're feeling and how the medicine is affecting your body. The study will look at how you're doing over time, including if you need to go to the hospital for your PAH or if your condition changes. The total length of your participation would be discussed with you by the study team.

Potential risks and benefits

Taking part in any medical study has potential benefits and potential risks. You might benefit if sotatercept helps improve your PAH, but it's also possible you could receive the placebo and not experience any direct benefit from the study medicine. As with any new medicine, there might be side effects, which the study team will monitor closely. You will be fully informed of all known and potential risks before you decide to participate. Your safety and well-being are the top priority. Remember, joining is entirely voluntary, and you have the right to withdraw from the study at any time, for any reason, without affecting your standard medical care.

Locations (13)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.
  • Unverified
    Spain
  • Unverified
    Poland
  • Unverified
    France
  • Unverified
    Belgium
  • Unverified
    Portugal
  • Unverified
    Czechia
  • Unverified
    Netherlands
  • Unverified
    Germany
  • Unverified
    Croatia
  • Unverified
    Denmark
  • Unverified
    Italy
  • Unverified
    Austria

Common questions

What is Pulmonary Arterial Hypertension (PAH)?

PAH is a serious condition where the blood vessels in your lungs become narrow, stiff, or blocked. This makes it harder for blood to flow through, causing high blood pressure in the lungs and making your heart work harder.

What does 'randomized, double-blind, placebo-controlled' mean?

It means you'll be randomly assigned to get either the new medicine or a dummy pill (placebo), and neither you nor your doctor will know which one you're taking. This helps ensure the results are fair and accurate.

What is sotatercept?

Sotatercept is a new medicine being investigated for PAH. It's given as an injection and works in a new way to help improve the blood vessels in the lungs.

What is a 'Phase 3' study?

Phase 3 is an advanced stage of clinical research. It means the medicine has already shown promise in earlier stages, and this study aims to confirm its effectiveness and safety in a larger group of people before it can be considered for wider use.

Will I still take my regular PAH medicines?

Yes, if you join this study, you will continue to take your usual PAH treatments. Sotatercept is being tested as an additional medicine to take alongside your background therapy.

How to find out more

Always speak to your GP or specialist before deciding to take part in a study.

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