RecruitingPhase 3

A Study to Evaluate the Efficacy and Safety of Once Daily Treprostinil Palmitil Inhalation Powder (TPIP) in Participants With Pulmonary Arterial Hypertension (PAH)

A Phase 3, Randomized, Double-Blind, Placebo-controlled, Multicenter, Parallel-group Study to Evaluate the Efficacy and Safety of Once Daily Treprostinil Palmitil Inhalation Powder in Participants With Pulmonary Arterial Hypertension

Who can join

As published by the study on ClinicalTrials.gov. "Not stated" means the study hasn't said — ask the study team.

Age
18 Years to 75 Years
Sex
All sexes
Healthy volunteers
Not accepted
Compensation
Not stated

Other requirements are listed under Eligibility below. Only the study team can confirm whether you can join.

What to expect

No one can promise a trial will help you personally — that is what the research is trying to find out.

What participation involves

Not stated in the study description — ask the study team about visits and procedures.

Compensation
Not stated
  • Study-related exams and procedures are typically provided at no cost. This is how many trials work in general — confirm the details for this study with the study team.
  • Participants are monitored closely by the research team throughout the study.
  • Some participants may receive access to an investigational treatment, which is still being studied.
  • Your participation helps researchers learn whether this approach could help future patients.

About this study

In plain language

This study tests TPIP, a powder breathed in once a day, in adults with pulmonary arterial hypertension (PAH), or high blood pressure in the lung arteries. It compares TPIP with a placebo, which has no active drug, over 24 weeks to see how it affects exercise ability and to check its safety.

The primary objective of this study is to evaluate the effect of 24-weeks of once daily treatment with TPIP compared with placebo on exercise capacity in adults with PAH.

Compensation is read from the study's own description and may be missing — always confirm with the study team.

Eligibility

Who can join

  • Have PAH for at least 3 months before screening.
  • Have PAH with no known cause, inherited PAH, or PAH linked to drugs, toxins, or connective tissue disease. PAH linked to a simple heart defect is allowed if the defect was repaired at least 1 year ago.
  • Have PAH in function class II, III, or IV, meaning symptoms limit physical activity or occur at rest.
  • Take 1 to 3 allowed PAH medicines. These include endothelin receptor blockers, PDE5 blockers, riociguat, or sotatercept.
  • For allowed medicines other than sotatercept, have taken them for at least 90 days, with no dose change in the last 30 days.
  • If taking sotatercept, have used it for at least 6 months with no dose change for 3 months. Have no significant active bleeding in the past 3 months, no major bleeding events or risks in the past 6 months, and a platelet count of at least 100,000 per microliter. Platelets help blood clot.
  • Walk 150 to 450 meters in each of two 6-minute walking tests at screening.
  • Have a right-heart catheter test, which uses a thin tube to measure heart and lung blood pressures. Results must show mean lung artery pressure above 20 mmHg, heart filling pressure of 15 mmHg or less, and lung blood vessel resistance of at least 5 Wood units. A test from the past 6 months may count if PAH medicines and doses have not changed.

Who can't join

  • Have another type of lung high blood pressure, including PAH linked to HIV, complex heart defects, high pressure in liver blood vessels, blocked small lung veins, or schistosomiasis infection.
  • Have serious disease of the left side of the heart, unstable heart disease from poor blood flow, or an unstable heartbeat.
  • Have blocked airflow on a breathing test: the amount breathed out in 1 second divided by the total amount breathed out is below 0.7.
  • Have lung disease that limits how much air you can breathe out, with a breathing test result below 70% of the expected normal value.
  • Have long-term disease from blood clots in the lungs, or have had a new lung blood clot within 6 months before screening.
  • Have a known allergy or another medical reason not to take treprostinil, TPIP, or its ingredients, such as mannitol or leucine.
  • Have another medical or mental health condition, or abnormal test results, that the study doctor thinks could make the study unsafe, prevent completion, or affect the results.

Simplified for readability. The official criteria below are what the study team uses.

Official eligibility criteria

Inclusion Criteria * Participants must have a diagnosis of World Health Organisation (WHO) Group 1 pulmonary hypertension (PAH) in any of the following subtypes, in accordance with European Society of Cardiology European Respiratory Society (ESC/ERS) Guidelines: * Idiopathic PAH * Heritable PAH * Drug/toxin-induced PAH * Connective tissue disease (CTD)-associated PAH * PAH associated with congenital heart disease-related to simple systemic-to-pulmonary shunt at least 1 year following repair. * PAH diagnosis for at least 3 months prior to Screening. * New York Heart Association (NYHA) or World Health Organization (WHO) functional class II-IV. * Participants must be on stable PAH therapy consisting of 1 to 3 medications from the following classes: * Endothelin receptor antagonists (eg, ambrisentan, bosentan, macitentan) for at least 90 days prior to Screening with the last 30 days on stable dose * Phosphodiesterase type 5 inhibitors (eg, sildenafil, tadalafil) for at least 90 days prior to Screening with the last 30 days on stable dose * Guanylate cyclase stimulator (eg, riociguat) for at least 90 days prior to Screening with the last 30 days on stable dose * Activin signaling inhibitor (e.g., sotatercept) for at least 6 months prior to Screening, with the last 3 months on stable dose and meeting all the following conditions: * no active clinically significant bleeding (eg, epistaxis and gingival bleeding requiring medical interventions) within the past 3 months. * no history of major bleeding events or risks (eg, gastrointestinal or intracranial bleeding) within the past 6 months. * platelet counts ≥100,000 per microlitre (μL) at Screening * For both 6-minute walk tests (6MWTs), the values of 6-minute walk distance (6MWD) should be ≥ 150 and ≤ 450 meters at Screening. * Right heart catheterization (RHC) at Screening (or within 6 months prior to Screening, if available). Prior RHC may be used provided there has been no change in background PAH therapy and doses. The RHC must meet all of the following hemodynamic criteria: * Mean pulmonary arterial pressure (PAP) \>20 millimetre of mercury (mmHg) at rest. * pulmonary capillary wedge pressure (PCWP) or left ventricular end-diastolic pressure (LVEDP) ≤15 mmHg. * pulmonary vascular resistance (PVR) of ≥5 wood units (WU). Exclusion Criteria * Diagnosis of PH WHO Groups 2, 3, 4, or 5, or subtypes of PH WHO Group 1 other than described in inclusion criterion 2 (eg, human immunodeficiency virus (HIV), complex congenital heart disease-associated PAH, portal hypertension-associated PAH, pulmonary veno-occlusive disease, Schistosomiasis associated PAH). * Clinically significant left heart disease, including left-sided valvular disease, left ventricular systolic or diastolic dysfunction, echocardiographic findings suggestive of post-capillary pulmonary hypertension, unstable ischemic heart disease, or unstable arrhythmias. * Evidence of airflow obstruction defined by forced expiratory volume in 1 second (FEV1) per forced vital capacity (FVC) \<0.7. * Evidence of significant restrictive lung disease as evidenced by FVC \<70% predicted normal. * Evidence of chronic thromboembolic disease or recent (within 6 months of Screening) acute pulmonary embolism. * Known hypersensitivity or contraindication to treprostinil or TPIP or TPIP formulation excipients (e.g., mannitol, leucine). * Any other medical or psychological condition including relevant laboratory abnormalities at Screening that, in the opinion of the Investigator, suggest a new and/or insufficiently understood disease and/or may present an unreasonable risk to the study participant as a result of his/her participation in this clinical trial, may impede their ability complete the study or the study assessments or confound the outcomes of the trial. Note: Other protocol-defined inclusion/exclusion criteria may apply.

Study sites (11)

CA

KS

NE

NY

PA

SC