The INSIGNIA-PAH Clinical Trial will test an investigational drug in adults ages 18 to 75 who have pulmonary arterial hypertension (PAH). PAH is a type of high blood pressure that affects arteries in the lungs and the right side of the heart. This clinical trial is comparing the investigational drug with a placebo, both of which are a dry powder. A placebo looks like the investigational trial drug but does not contain any active medicine. You will breathe the powder into your lungs from your mouth through an inhaler.
Learn about the safety of the investigational drug and how well participants tolerate side effects associated with the investigational drug
Find the dose of the investigational drug that should be given to participants
This trial has 4 parts:
Screening
(up to 4 weeks long)
You will have medical tests to see if you qualify for the trial. You will visit the trial site 1 or more times.
Trial Treatment Base Period
(about 12 weeks long)
You will take the investigational drug or a placebo during this phase, go to the trial site about 4 times, and have 1 phone visit.
Trial Treatment Extension Period
(about 2 years long)
This period is optional. You will continue to take the investigational drug, go to the trial site about 28 times, and have about 2 phone visits.
Follow-up
(about 2 weeks long)
After you stop participating in the trial, you will have 1 phone visit.
Will I receive the investigational trial drug if I take part in the Trial Treatment base period part of the study?
Participants in the Trial Treatment base period will be randomly placed (assigned by chance) into 1 of 4 treatment groups. You will have the same chance of being placed into one of these groups:
3 of the groups will get different doses of the investigational drug
1 group will get a placebo
In the Trial Treatment Extension Period (about 2 years long), all participants will get the investigational drug.
INSIGNIA-PAH will be carried out in 2 parts, Phase 2 and Phase 3. Currently this study is enrolling our Phase 2 part of the trial. In Phase 2, we will research the investigational drug to determine the appropriate amount of drug to give to participants.
The Phase 3 part of the trial is not currently enrolling. Phase 3 clinical trials compare the safety and effectiveness of the investigational drug against the current standard treatment. Each phase of this trial includes a Base Period and an Extension Period.
Trial start and end dates
Trial start date
May 19, 2021
Estimated primary completion date
February 9, 2026
Estimated trial completion date
January 19, 2028
How to Qualify
You may be able to take part in this clinical trial if you are 18 to 75 years old and have PAH. There are more requirements to be in this clinical trial. The trial doctor will talk with you about all the requirements for the clinical trial to see if you may qualify.
If you qualify and decide to participate:
You will receive the trial treatment and all trial-related medical tests at no cost
Your PAH symptoms and overall health will be closely monitored by a trial doctor with experience in PAH
You may help researchers learn more about PAH and the investigational drug
During the clinical trial you will continue to take your current PAH medicines
Participation in the clinical trial is voluntary, and you are free to leave the clinical trial at any time. Your privacy will be maintained throughout the clinical trial.
Pulmonary arterial hypertension is a type of high blood pressure that affects arteries in the lungs and the right side of the heart. It is caused by blood vessels in the lungs that become narrow, blocked, or destroyed. This slows down the flow of blood through the lungs and makes the heart work harder to pump blood through the lungs. The heart becomes weak and fails.1
People with early stages of PAH may not have any noticeable symptoms. As the condition progresses, they may have symptoms like other lung diseases such as asthma or chronic obstructive pulmonary disease (COPD).2
The most common PAH symptoms include:2
Increased shortness of breath
Fatigue
Edema, or swelling of the feet and legs
Dizziness and fainting spells
Chest pain
Heart palpitations (racing or pounding)
PAH is a rare condition that is most common in women ages 30 to 60. Although the cause is usually unknown, about 15% to 20% of people with PAH have inherited the condition.3
The INSIGNIA-PAH Clinical Trial is being conducted to test an investigational drug in adults (ages 18 to 75) with PAH to learn about the safety of the investigational drug, how well participants tolerate side effects associated with the investigational drug, how well the investigational drug works on PAH symptoms, and how the investigational drug impacts physical activity. Researchers also want to find the dose of the investigational drug that should be given to participants.
If you qualify and decide to take part, you will be in the clinical trial for up to 16 weeks (about 4 months). You will visit the trial site up to 5 times and have 1 phone visit. After the 16-weeks period is completed, you have the option to participate in the trial extension period, which is about 2 years.
During the clinical trial, you will continue to take your current PAH medicines.
This trial has 4 parts:
Screening (up to 4 weeks long): You will have medical tests to see if you qualify for the trial. You will visit the trial site 1 or more times.
Trial Treatment Base Period (about 12 weeks long): You will take the investigational drug or a placebo during this phase, go to the trial site about 4 times, and have 1 phone visit.
Trial Treatment Extension Period (about 2 years long): This period is optional. You will continue to take the investigational drug, go to the trial site about 28 times, and have about 2 phone visits.
Follow-up (about 2 weeks long): After you stop participating in the trial, you will have 1 phone visit.
Before you agree to participate, the trial team will review all aspects of the clinical trial with you. If you decide to take part, you will be given a document called an Informed Consent Form that provides, in writing, the clinical trial’s purpose, assessments, procedures, potential benefits and risks, and precautions. You will have the opportunity to ask questions and decide if participating is right for you.
If you are eligible to participate, the trial team at the location you select can provide more information and answer your questions.
A placebo looks like the investigational drug but does not contain any active medicine. In this clinical trial, you will be randomly placed (assigned by chance) into 1 of 4 treatment groups. There is a:
3 in 4 (75%) chance you will receive the investigational drug
1 in 4 (25%) chance you will receive a placebo
All participants who qualify for the long-term extension will receive the investigational drug for up to 2 years.
Privacy, Withdrawing, Costs, and Permission
The trial team respects and protects your privacy and will not share your information except as required by law and will store your personal information with codes that do not identify you. The Informed Consent Form (completed by you prior to participation) will provide more information about how your privacy will be maintained.
Your participation in the clinical trial is entirely voluntary and you may withdraw for any reason, at any time. If you do decide to withdraw early, you will be asked to notify the trial team before doing so. You will be asked to return to the trial site at least once to complete a final visit and return any unused drug.
No. You will get the trial treatment at no cost. You will also get all clinical trial-related medical tests at no cost.
No, your doctor does not have to give permission for you to participate. However, either you or the trial doctor, with your permission, may contact your personal doctor to discuss your participation before you begin and keep your doctor up to date about your progress.
About Clinical Trials
A clinical trial is a research study that tries to answer questions about how medicines work in the people who take them. Researchers run studies to test whether an investigational medication is safe and effective. These studies may help doctors find new ways to help prevent, detect, or treat health problems. Clinical trials may also be referred to as research studies.
Participant safety is the priority. There are rules in place to help protect the rights, safety, and well-being of people who volunteer for research studies. These rules are put in place to make sure studies follow strict scientific and ethical guidelines.
Before a clinical research study can begin, a review board or ethics committee must review the study. In the U.S., this group is called an IRB or institutional review board. An IRB is made up of doctors, scientists, and members of the community.
Only patients who meet all eligibility criteria for a clinical trial may take part. The trial team at the site you select will review your medical history and current medical status against the eligibility criteria. They will determine if you are eligible to participate. You may also be asked to provide information from your medical records to help the trial team determine whether or not you may be eligible.
An investigational drug is a drug that hasn’t yet been approved for use in the general public. In order to be approved, the investigational drug must be tested in clinical trials to see if it is safe and effective for treating the target disease in certain groups of people.
The investigational drug in the INSIGNIA-PAH Clinical Trial is being tested for people with PAH to see if it may help improve your blood pressure that affects your heart and lungs.
What can you do next?
If you think this clinical trial might be a good fit and you are interested in taking part, take the next step to see if you are eligible.
Discuss with your doctor or care team
Print this page with details about the trial or email it to your doctor to discuss the clinical trial during your next visit.
Pulmonary hypertension. Mayo Clinic. Accessed May 8, 2022. https://www.mayoclinic.org/diseases-conditions/pulmonary-hypertension/symptoms-causes/syc-20350697
Pulmonary arterial hypertension symptoms and diagnosis. American Lung Association. Accessed May 8, 2022. https://www.lung.org/lung-health-diseases/lung-disease-lookup/pulmonary-arterial-hypertension/symptoms-diagnosis
Learn About Pulmonary Arterial Hypertension. American Lung Association. Accessed May 8, 2022. https://www.lung.org/lung-health-diseases/lung-disease-lookup/pulmonary-arterial-hypertension/learn-about-pulmonary-arterial-hypertension
