What side effects are associated with this type of intervention?

Common treatments for Pulmonary Arterial Hypertension, such as treprostinil, epoprostenol, and PDE5 inhibitors like sildenafil, have several known side effects. Treprostinil, whether inhaled, subcutaneous, or intravenous, can cause headache, nausea, vomiting, diarrhea, flushing, jaw pain, and injection site pain. Epoprostenol, administered intravenously, may lead to similar side effects, including headache, jaw pain, and gastrointestinal issues, along with risks associated with central venous catheter use, such as infections. PDE5 inhibitors like sildenafil can cause headaches, flushing, dyspepsia, and visual disturbances. These side effects are important considerations when choosing a treatment plan for PAH.

What prior FDA approvals exist?

The FDA has approved several treatments for Pulmonary Arterial Hypertension (PAH) that are similar to treprostinil, a vasodilator. These include other prostanoids like epoprostenol and iloprost, which also work by dilating pulmonary arteries to reduce pressure and improve blood flow. Epoprostenol is often administered intravenously, while iloprost can be inhaled. Additionally, phosphodiesterase-5 inhibitors (PDE5Is) such as sildenafil and tadalafil have been approved for PAH treatment, improving exercise capacity and hemodynamics. Combination therapies, such as inhaled treprostinil with oral agents like bosentan or sildenafil, have also demonstrated significant improvements in exercise capacity and quality of life.

What other types of research exist?

Promising alternatives to Treprostinil for PAH patients include: <ol> <li>Sildenafil: A PDE5 inhibitor that improves pulmonary hemodynamics and exercise capacity.</li> <li>Tadalafil: Another PDE5 inhibitor that increases 6-minute walk distance (6MWD) and time to clinical worsening.</li> <li>Riociguat: A soluble guanylate cyclase stimulator that improves exercise capacity and WHO functional class.</li> <li>Macitentan: An endothelin receptor antagonist that has shown benefits in improving functional class and survival.</li> <li>Selexipag: An oral prostacyclin receptor agonist that delays disease progression and reduces the risk of hospitalization.</li> </ol>

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Prostacyclin Analogue

Inhaled Treprostinil for Pulmonary Hypertension

Q: What is the mechanism of action of this treatment?

The most common treatments for Pulmonary Arterial Hypertension (PAH) include vasodilators like treprostinil, which dilate the pulmonary arteries to reduce pulmonary arterial pressure and improve blood flow. This is crucial for PAH patients as it helps alleviate symptoms and improve exercise capacity. Other treatments include phosphodiesterase-5 inhibitors (PDE5Is) like sildenafil, which enhance nitric oxide signaling to relax blood vessels, and endothelin receptor antagonists (ERAs) like bosentan, which block endothelin-1 to prevent vasoconstriction. These mechanisms collectively aim to reduce the strain on the heart and improve the quality of life for PAH patients.

Phase 2

Waitlist Available

Led By Yogesh N Reddy, M.B.B.S

Research Sponsored by Mayo Clinic

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

≥ 18 Years of Age

Patients with suspected Pulmonary Arterial Hypertension related to Interstitial Lung Disease (ILD-PAH) undergoing right heart catheterization.

Must not have

< 18 years old.

Timeline

Screening 3 weeks

Treatment Varies

Follow Up baseline, 3-months

Awards & highlights

No Placebo-Only Group

Summary

This trial is testing an inhaled medication called treprostinil on patients with a lung condition that makes it hard for them to exercise. The medication helps by improving blood flow in the lungs. Researchers want to see if it makes exercise easier and improves quality of life after a period of use. Treprostinil has been shown to improve exercise capacity and is more convenient due to its longer-lasting effects.

Who is the study for?

Adults over 18 with suspected PAH-ILD undergoing right heart catheterization, and healthy volunteers without lung disease or pulmonary hypertension. It's not for anyone under 18.

What is being tested?

The study is testing the drug treprostinil inhaled by patients with PAH-ILD to see how it affects their ability to exercise and quality of life after three months. Some may undergo optional muscle biopsies or RHC while exercising.

What are the potential side effects?

Treprostinil may cause side effects such as headache, diarrhea, nausea, rash, jaw pain, flushing, increased cough or breathlessness during use.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

Select...

I am 18 years old or older.

Select...

I am suspected to have high blood pressure in the lungs due to lung disease and will undergo a heart procedure.

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I am healthy with no lung, heart, muscle diseases or high blood pressure in the lungs.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

Select...

I am under 18 years old.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ baseline, 3-months

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~baseline, 3-months

This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline, 3-months for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Change in cardiac output reserve (Qc)

Secondary study objectives

Change in New York Heart Association (NYHA) functional class

Change in exercise pulmonary artery pressure

Change in exercise pulmonary vascular resistance

+5 more

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

2Treatment groups

Experimental Treatment

Group I: PAH-ILD PatientsExperimental Treatment3 Interventions

Patients diagnosed with PAH-ILD will undergo RHC while exercising, receive inhaled treprostinil, and undergo an optional muscle biopsy.

Group II: Healthy VolunteersExperimental Treatment2 Interventions

Healthy volunteers will undergo RHC while exercising and an optional muscle biopsy.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Treprostinil

2021

Completed Phase 4

~180

0 / 4Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

The most common treatments for Pulmonary Arterial Hypertension (PAH) include vasodilators like treprostinil, which dilate the pulmonary arteries to reduce pulmonary arterial pressure and improve blood flow. This is crucial for PAH patients as it helps alleviate symptoms and improve exercise capacity. Other treatments include phosphodiesterase-5 inhibitors (PDE5Is) like sildenafil, which enhance nitric oxide signaling to relax blood vessels, and endothelin receptor antagonists (ERAs) like bosentan, which block endothelin-1 to prevent vasoconstriction. These mechanisms collectively aim to reduce the strain on the heart and improve the quality of life for PAH patients.