With you, we are ready to give CTEPH even more attention.

The MACiTEPH Study is a clinical research study which is evaluating an investigational medication for inoperable chronic thromboembolic pulmonary hypertension (CTEPH).

As part of this study medical professionals will provide study-required monitoring of your CTEPH and overall health.


Chronic thromboembolic pulmonary hypertension (CTEPH) is a rare condition that impacts less than 1% of the world’s population. While the preferred treatment is surgery, it’s not always possible because some people’s CTEPH is inoperable.

Another treatment option is an intervention procedure called balloon pulmonary angioplasty (BPA) where balloons are used to open the arteries in the lungs. However, even with surgery or BPA, a person’s CTEPH can persist or recur.

About the MACiTEPH Study

Doctors with the MACiTEPH research study are evaluating an investigational medication for people with CTEPH that is either inoperable or persistent after surgery or BPA. Specifically, doctors want to look at its effect on your ability to exercise over time and compare the results with a placebo. A placebo looks like the investigational medication but contains no active medication.

The investigational medication has been approved for other uses by the FDA at a lower dose. The doctors are evaluating the safety and effectiveness of the investigational medication in people with CTEPH.

The results of this study will provide more information about the investigational medication when it is given to adults with CTEPH.

Who is eligible to be in the MACiTEPH Study?

To pre-qualify for this study, you must be:

18 to 80 years of age

Diagnosed with CTEPH that is either inoperable, OR persistent/recurrent following surgery and/or BPA

All study-required visits, tests and medications will be provided at no cost. In addition, reimbursement for study-required travel may be available.

Short Study Summary

Medical condition

Chronic Thromboembolic Pulmonary Hypertension (CTEPH)

Study duration

Up to 6 years

Study locations

This is a global study which will enroll participants across North America, South America, Europe, and Asia-Pacific



What does it mean to take part in the MACiTEPH Study?

If you are eligible and agree to be in this study, you will be randomly assigned (by chance) to receive either the investigational medication or placebo. A placebo looks like the investigational medication and is given in the same manner but does not have any active medication. You have an equal chance of being assigned to either treatment group (investigational medication or placebo). If you are currently taking medicine to treat your CTEPH, you may be able to continue taking it while in the study.

Neither you nor the study team will know which study medication you are receiving. However, in the event of an emergency, that information can be provided to the study doctor.

You will take the study medication once a day as instructed by the study doctor. After the treatment phase, you may have the possibility to join the open-label part of the study. During the open-label part of the study all participants will receive the investigational medication. During your study participation, you will be asked to attend regular study clinic visits and receive phone calls from the study team. This will allow the study team to evaluate your health and talk with you about the study. You may be in the study for up to 6 years; however, you are free to withdraw from the study at any time.

What are the possible benefits and risks related to the MACiTEPH Study?

It's possible that your CTEPH could improve while you're in this study, but that is not guaranteed. Your condition may also stay the same or worsen. However, your study participation may help people with CTEPH in the future.

It’s also possible you could experience one or more side effects during this study. Before you begin the study, the study doctor will talk with you about any potential study-related risks and side effects. Your health will be closely monitored throughout the study. You may be in the study for up to 6 years; however, you are free to withdraw from the study at any time.

Frequently Asked Questions

Clinical studies evaluate whether a medicine, treatment, or device is safe and effective for humans.

Clinical studies are scientific studies in which investigational medicines and treatments are tested to make sure they are safe and effective for people who may need them. They are one of the most important steps in bringing potential medications to patients.

Clinical research adds to medical knowledge and helps bring potential medications to people with medical conditions. In order to make potential medications available to the public they need be studied in clinical trials.

Clinical trials rely on the participation of volunteers in order to succeed. On average, it can take up to 8 years for an investigational medication to reach the public.

All investigational medical treatments and medications have gone through clinical trials to make sure that they are safe and effective.

Clinical studies can be sponsored, or funded, by pharmaceutical companies, academic medical centers, volunteer groups, or health care providers.

Every clinical study is led by a principal investigator, who is often a medical doctor. Clinical studies also have a research team that may include doctors, nurses, social workers, and other health care professionals.

Phase I

The first study in humans. These are small studies with 20-100 participants, mostly in healthy volunteers. The main objectives are to investigate:

• Safety of the study medication

• How the study medication is absorbed by the body and what dosage should be used

• How the study medication is removed from the body

• Potential side effects

Phase II

Small studies with around 100-500 participants. The main objectives are to investigate:

• Ongoing safety

• Whether the study medication works for a particular disease

• The best dose of the study medication

Phase III

Large studies with around 500 or more participants. These are the main studies for final approval by health authorities. The main objectives are to investigate:

• Safety and side effects in bigger populations

• Whether the study medication works for a particular disease

• How the treatment compares to already existing standard therapies

Phase IV

Large studies in patients after the study medication has been approved by regulatory authorities for prescription

use (or public use if it isn't a medicine that requires a prescription). The main objectives are to investigate:

• Side effects during day-to-day use in the population

• Risks and benefits over a longer period of time

Before taking part in a trial, you will be asked to read and sign an informed consent document to ensure:

1. You agree to volunteer

2. You understand the study, including all the study procedures, risks and potential side effects of the study medication.

3. You understand that you can leave the study at any time for any reason.

During the consenting process, you will have the opportunity to ask the site staff any questions you may have. Taking part in a clinical trial typically involves taking tests to determine if you are a match for the study. If you qualify, you will visit the clinic regularly to receive the investigational medication, undergo tests or procedures, and assess your disease. The study staff will monitor your progress and well-being.