An Open-Label Study to Assess the Safety, Pharmacokinetics, and Pharmacodynamics of Treprostinil Palmitil Inhalation Powder in Participants With Pulmonary Arterial Hypertension

Insmed Incorporated1 site in 1 country1 target enrollmentMarch 29, 2022

ConditionsPulmonary Arterial Hypertension

InterventionsTreprostinil Palmitil

DrugsTreprostinil Palmitil

Overview

Phase: Phase 2
Intervention: Treprostinil Palmitil
Conditions: Pulmonary Arterial Hypertension
Sponsor: Insmed Incorporated
Enrollment: 1
Locations: 1
Primary Endpoint: Number of Participants Who Experience a Treatment Emergent Adverse Event (TEAE)
Status: Terminated
Last Updated: 2 years ago

Overview Investigators Eligibility Criteria Arms & Interventions Outcomes Sites Similar Trials

Overview

Brief Summary

The main purpose of this study is to evaluate the safety and tolerability of single dose of treprostinil palmitil inhalation powder (TPIP) in participants with pulmonary arterial hypertension (PAH).

Registry

clinicaltrials.gov

Start Date

March 29, 2022

End Date

August 26, 2022

Last Updated

2 years ago

Study Type

Interventional

Study Design

Sequential

Sex

All

Investigators

Sponsor

Insmed Incorporated

Responsible Party

Sponsor

Eligibility Criteria

Inclusion Criteria

•Participant must be ≥ 18 years of age at the time of signing the informed consent
•Participants must have a diagnosis of World Health Organization Group 1 Pulmonary Hypertension (PH) (PAH) with the following characteristics
•Etiology of idiopathic, heritable, drug/toxin-induced or connective tissue disease (CTD)-related PAH
•Right heart catheterization with the following hemodynamic findings:
•Mean pulmonary arterial pressure (mPAP) \> 20 mmHg at rest,
•Pulmonary capillary wedge pressure (PCWP) ≤ 15 mmHg, and
•Pulmonary vascular resistance (PVR) of ≥ 3 Wood Units (WU)
•No change in pulmonary hypertension medications (eg, ambrisentan, bosentan, macitentan, sildenafil, tadalafil, riociguat) or dosage for at least 90 days prior to Screening
•No change in diuretic use or dosage for at least 30 days prior to Screening
•Body mass index (BMI) within the range 18.0 - 32.0 kg/m\^2 (inclusive)

Exclusion Criteria

•Any PH other than idiopathic, hereditary, drug/toxin-induced, or connective tissue disease (CTD) associated PAH (eg, congenital heart disease-associated PAH, portal hypertension-associated PAH, PH belonging to Groups 2 through 5)
•Allergy, or documented hypersensitivity or contraindication, to the ingredients of treprostinil palmitil inhalation powder (TPIP) or treprostinil (TRE)
•Previous intolerance to prostacyclin analogs or receptor agonists (eg, selexipag) per investigator discretion
•History of anaphylaxis or previously documented hypersensitivity reaction to any drug per Investigator discretion
•History of heart disease including left ventricular ejection fraction (LVEF) ≤ 40% or clinically significant valvular, constrictive, or atherosclerotic heart disease (myocardial infarction, etc)
•Active liver disease or hepatic dysfunction manifested as:
•Elevated liver function test results (ALT or AST \> 2 × ULN) at Screening
•Bilirubin \> 1.5 × ULN (isolated bilirubin \> 1.5 × ULN; ULN is acceptable if bilirubin is fractionated and direct bilirubin \< 35%) at Screening.
•Known hepatic or biliary abnormalities, not including Gilbert's syndrome or asymptomatic gallstones at Screening.
•History of HIV infection/positive HIV serology test result at Screening

Arms & Interventions

Treprostinil Palmitil Inhalation Powder

Participant received a single dose of TPIP 112.5 micrograms (μg) via oral inhalation on Day 1. The participant then entered into a 16-week Extended Use Treatment (EUT) Period during which TPIP, administered via oral inhalation, was titrated up to a mean daily dose of 320 μg.

Intervention: Treprostinil Palmitil

Outcomes

Primary Outcomes

Number of Participants Who Experience a Treatment Emergent Adverse Event (TEAE)

Time Frame: Up to 150 days

Secondary Outcomes

Time to Maximum Concentration (Tmax) of Treprostinil (TRE) in Plasma(Pre-dose and multiple timepoints post-dose up to Day 2)
Maximum Observed Concentration (Cmax) of Treprostinil (TRE) in Plasma(Pre-dose and multiple timepoints post-dose up to Day 2)
Change From Baseline in Pulmonary Vascular Resistance (PVR) After TPIP Administration(Day 1: Pre-treatment (Baseline), 8 and 24 hours post-treatment)
Area Under the Concentration-time Curve From Time 0 to Time of the Last Measurable Concentration (AUClast) of Treprostinil (TRE) in Plasma(Pre-dose and multiple timepoints post-dose up to Day 2)
Elimination Half-life (t1/2) of Treprostinil (TRE) in Plasma(Pre-dose and multiple timepoints post-dose up to Day 2)
Area Under the Concentration-time Curve From Time 0 to Infinity (AUC0-∞) of Treprostinil (TRE) in Pasma(Pre-dose and multiple timepoints post-dose up to Day 2)