What is the mechanism of action of this treatment?

Common treatments for Pulmonary Arterial Hypertension (PAH) include prostacyclin analogs, phosphodiesterase-5 inhibitors (PDE5Is), and endothelin receptor antagonists (ERAs). Prostacyclin analogs, such as treprostinil, dilate blood vessels and inhibit platelet aggregation, reducing pulmonary vascular resistance and improving blood flow. PDE5Is, like sildenafil, enhance nitric oxide effects, leading to vasodilation and better hemodynamics. ERAs, such as bosentan, block endothelin-1, a vasoconstrictor, reducing vascular resistance. These mechanisms are vital for PAH patients as they alleviate symptoms, improve exercise capacity, and potentially enhance survival.

What side effects are associated with this type of intervention?

Common side effects of prostacyclin analogs like treprostinil and iloprost, which are used to treat Pulmonary Arterial Hypertension, include headache, nausea, vomiting, diarrhea, flushing, jaw pain, and arthralgias. More severe adverse effects can include infusion site pain (for subcutaneous administration), central venous catheter infections, and complications related to the delivery system such as thrombosis and pump malfunction. Inhaled formulations may also cause cough and throat irritation.

What prior FDA approvals exist?

The FDA has approved several prostacyclin analogs for the treatment of Pulmonary Arterial Hypertension (PAH). These include Epoprostenol, Treprostinil, and Iloprost. Epoprostenol is administered intravenously and has shown significant improvements in hemodynamic parameters and survival rates. Treprostinil is available in multiple formulations, including intravenous, subcutaneous, oral, and inhaled, offering flexibility in administration while improving exercise capacity and symptoms. Iloprost is an inhaled prostacyclin analog that targets lung vasculature and requires frequent dosing. These treatments work by dilating blood vessels and inhibiting platelet aggregation, similar to the investigational Treprostinil Palmitil Inhalation Powder.

What other types of research exist?

Promising novel alternatives to Treprostinil Palmitil Inhalation Powder for PAH patients include: <ol> <li>Selexipag: An oral selective IP prostacyclin receptor agonist that has shown to improve exercise capacity and delay disease progression.</li> <li>Riociguat: A soluble guanylate cyclase stimulator that improves pulmonary hemodynamics and exercise capacity.</li> <li>Macitentan: An endothelin receptor antagonist that has demonstrated efficacy in improving long-term outcomes and delaying disease progression.</li> <li>Sotatercept: A novel agent in late-stage clinical trials that targets the transforming growth factor-beta (TGF-β) pathway, showing promise in improving pulmonary vascular resistance and exercise capacity.</li> </ol>

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Prostacyclin Analogue

Treprostinil Palmitil for Pulmonary Arterial Hypertension

Phase 2

Recruiting

Research Sponsored by Insmed Incorporated

Eligibility Criteria Checklist

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

Must have

Participants must be on stable PH therapy with specific medications and no changes in medication or dosage for at least 30 days prior to Screening.

Participants must have a diagnosis of World Health Organization (WHO) Group 1 Pulmonary Hypertension (PH) [pulmonary arterial hypertension (PAH)] in specific subtypes.

Must not have

Evidence of thromboembolic disease, active liver disease, HIV infection, hepatitis B or C viral infections, or symptomatic COVID-19.

History of PH other than specified subtypes.

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 5 years

Summary

This trial is testing an inhalable medicine called TPIP to see if it can help people with high blood pressure in their lungs by making it easier for blood to flow through their lung arteries.

Who is the study for?

Adults aged 18-75 with Pulmonary Arterial Hypertension (PAH) who are on stable PH therapy can join this trial. They must not smoke, have a history of certain heart diseases or infections, and women should use effective contraception. Participants need to be able to perform physical tests like walking.

What is being tested?

The study is testing Treprostinil Palmitil inhalation powder against a placebo to see if it improves blood flow resistance in the lungs for PAH patients. The participants will either receive the actual drug or a placebo without knowing which one they're getting.

What are the potential side effects?

While specific side effects aren't listed here, common ones for inhaled PAH treatments include coughing, headaches, nausea, dizziness, and potential risks associated with inhaling medication directly into the lungs.

Eligibility Criteria

Inclusion Criteria

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

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I've been on the same pulmonary hypertension medication for at least 30 days.

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I have been diagnosed with a specific type of high blood pressure in the lungs.

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I am between 18 and 75 years old.

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I am a man and will use birth control if my partner can have children.

Exclusion Criteria

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

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I have a history of blood clots, liver disease, HIV, hepatitis B or C, or symptomatic COVID-19.

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I have a history of pulmonary hypertension, but not the specified types.

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I have specific heart conditions or recently joined a heart-lung rehab program.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 5 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 5 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 5 years for reporting.

Treatment Details

Trial Design

2Treatment groups

Experimental Treatment

Placebo Group

Group I: Treprostinil Palmitil Inhalation PowderExperimental Treatment1 Intervention

Participants will be administered TPIP once per day at a starting dose of 80 micrograms (μg). Participants will be up-titrated to the highest tolerated dose for each individual participant of between 80 μg and 640 μg during the initial 3 weeks of treatment. The overall treatment period will be 16 weeks.

Group II: PlaceboPlacebo Group1 Intervention

Participants will be administered a placebo matching TPIP once per day for 16 weeks.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Treprostinil Palmitil

2022

Completed Phase 2

~40

0 / 7Eligibility 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

Common treatments for Pulmonary Arterial Hypertension (PAH) include prostacyclin analogs, phosphodiesterase-5 inhibitors (PDE5Is), and endothelin receptor antagonists (ERAs). Prostacyclin analogs, such as treprostinil, dilate blood vessels and inhibit platelet aggregation, reducing pulmonary vascular resistance and improving blood flow. PDE5Is, like sildenafil, enhance nitric oxide effects, leading to vasodilation and better hemodynamics. ERAs, such as bosentan, block endothelin-1, a vasoconstrictor, reducing vascular resistance. These mechanisms are vital for PAH patients as they alleviate symptoms, improve exercise capacity, and potentially enhance survival.