Active not recruitingPHASE3INTERVENTIONAL

Outcome Study Assessing a 75 Milligrams (mg) Dose of Macitentan in Patients With Pulmonary Arterial Hypertension

This clinical trial is investigating a higher dose of a medicine called macitentan (75mg) for people living with pulmonary arterial hypertension (PAH). PAH is a condition where the blood pressure in the arteries leading to the lungs is too high. The main goal of the study is to find out if the 75mg dose of macitentan is more effective than the currently used 10mg dose at delaying serious health events, like hospital stays or worsening symptoms, or death. Participants will receive either 10mg, 37.5mg, 75mg of macitentan, or a dummy pill (placebo). This is a Phase 3 study, meaning it's one of the final steps before a new treatment might become widely available.

At a glance

Status

Active not recruiting

Phase

PHASE3

Sponsor

Actelion

Enrolment target

935

Start

30 Jun 2020

Estimated completion

20 Feb 2029

Pulmonary Arterial Hypertension

What is this study about?

This research study is about a lung condition called Pulmonary Arterial Hypertension (PAH). In PAH, the blood vessels in your lungs become narrow, making it hard for your heart to pump blood through them. This can make you feel tired, dizzy, or short of breath. The study is particularly looking at a medicine called macitentan, which is already used to treat PAH in some doses.

The main aim of this study is to see if a higher dose of macitentan (75 milligrams, or mg) works better than the usual 10mg dose. "Better" in this case means if it can help people with PAH avoid serious health problems or even death for a longer time. They also want to compare these doses to a dummy pill (called a placebo) to understand the medicine's effects more clearly.

By taking part, you would be helping doctors and researchers understand more about how to best treat PAH. This could lead to new ways of managing the condition and improving the lives of people living with it in the future.

Key takeaways

This study is testing a higher dose (75mg) of macitentan for PAH.
It aims to see if the higher dose is better at preventing serious health problems than the current 10mg dose.
Participants will receive one of three doses of macitentan or a placebo.
This is a Phase 3 study, a key step towards potential new treatments.
You must have diagnosed PAH and meet specific health criteria to take part.
Closer medical monitoring is a potential benefit, but side effects are possible.

Who may be eligible?

To join this study, you generally need to be 18 years old or older. You must have been diagnosed with Pulmonary Arterial Hypertension (PAH) and be experiencing symptoms that fall into certain categories (WHO Functional Class II, III, or IV). Your PAH must also be of a specific type, such as idiopathic (meaning the cause isn't known), genetic, caused by drugs, or linked to conditions like connective tissue disease or HIV infection.

Your diagnosis of PAH will need to be confirmed by special heart measurements, showing specific pressure levels in your lungs and heart. You should also be able to walk for at least 50 meters (about 55 yards) in a 6-minute test. If you can walk further, up to 440 meters, you might still be eligible if your symptoms are more severe or you have a specific blood test result.

However, you won't be able to join if you have three or more specific risk factors for another heart condition called heart failure with preserved ejection fraction. The study will check your medical records for these details.

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 your PAH symptoms moderate to severe (WHO Functional Class II, III, or IV)?
Can you walk at least 50 meters in 6 minutes?
Do you NOT have three or more risk factors for another heart condition called heart failure with preserved ejection fraction?
Has your PAH diagnosis been confirmed by specific heart and lung pressure measurements?

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, you will be assigned to one of four groups: receiving 10mg of macitentan, 37.5mg of macitentan, 75mg of macitentan, or a placebo (a dummy pill that looks like the medication but contains no active drug). You won't know which group you are in, and neither will the study doctors, to make sure the results are as fair as possible. You will be given the study medication to take regularly.

Throughout the study, you will have regular clinic visits. These visits will involve various health checks, blood tests, and repeat assessments to monitor your condition and how you are responding to the treatment. This will likely include repeat 6-minute walking tests and other evaluations related to your heart and lung health. The total duration of your participation in the study will depend on how long it takes for enough participants to experience certain health events, so it could last for several years, with regular follow-up appointments.

Potential risks and benefits

Participating in a clinical trial might offer potential benefits, such as receiving a new or different dose of an established medication and closer medical monitoring than you might usually get. While macitentan is an existing treatment for PAH, the 75mg dose is new, so there's a chance it could be more effective. However, like all medications, macitentan can have side effects, and some are specific to different doses. As you might receive a placebo, there's also a chance you might not get any active treatment. The research team will explain all known potential risks and side effects to you. Remember, joining is entirely your choice, and you are free to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (274)

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

Mayo Clinic
Verified postcode
Phoenix, United States
Arizona Pulmonary Specialists, Ltd
Verified postcode
Scottsdale, United States
Scripps Memorial Hospital
Verified postcode
La Jolla, United States
USC Keck
Verified postcode
Los Angeles, United States
Jeffrey S. Sager, MD Medical Corporation
Verified postcode
Santa Barbara, United States
Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center
Verified postcode
Torrance, United States
National Jewish Health
Verified postcode
Denver, United States
Cleveland Clinic
Verified postcode
Weston, United States
Piedmont Healthcare
Verified postcode
Atlanta, United States
Northside Hospital
Verified postcode
Atlanta, United States
Advocate Christ Medical Center
Verified postcode
Oak Lawn, United States
Indiana University
Verified postcode
Indianapolis, United States

Common questions

What is Pulmonary Arterial Hypertension (PAH)?

PAH is a serious condition where the blood pressure in the arteries that go from your heart to your lungs becomes too high, making it harder for your heart to pump blood.

What is macitentan?

Macitentan is a medicine that is currently used to treat PAH. It works by helping to relax and widen the narrowed blood vessels in the lungs.

What does 'Phase 3' mean in a clinical trial?

Phase 3 is one of the final stages of testing a new treatment. It involves a larger number of patients and aims to confirm if the treatment is effective and safe before it can be made widely available.

What is a placebo?

A placebo is a dummy pill or treatment that looks exactly like the real medicine but contains no active ingredients. It's used in studies to compare the effects of the active drug.

Can I choose which dose of macitentan I receive?

No, you cannot choose. To ensure fair results, participants are randomly assigned to one of the treatment groups (different doses of macitentan or placebo) without knowing which one they are receiving.