A Study of Treprostinil Palmitil Inhalation Powder (TPIP) In Pulmonary Arterial Hypertension (PAH)

NCT04791514 | Terminated Phase 2 Results FDA Drug

• 1 other identifier: INS1009-201
• Study Type: interventional
• Target: 1
• Locations: 1 country
• Sites: 1

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).

Conditions

Pulmonary Arterial Hypertension

Keywords

Pulmonary Arterial Hypertension; Treprostinil Palmitil Inhalation Powder; Treprostinil Palmitil

Outcome Measures

Primary Outcomes (1)

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

  Up to 150 days

Secondary Outcomes (6)

• Change From Baseline in Pulmonary Vascular Resistance (PVR) After TPIP Administration

  Day 1: Pre-treatment (Baseline), 8 and 24 hours post-treatment

• Maximum Observed Concentration (Cmax) of Treprostinil (TRE) in Plasma

  Pre-dose and multiple timepoints post-dose up to Day 2

• Time to Maximum Concentration (Tmax) 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 Time of the Last Measurable Concentration (AUClast) 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

Study Arms (1)

Treprostinil Palmitil Inhalation Powder

EXPERIMENTAL

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.

Drug: Treprostinil Palmitil

Interventions

Treprostinil Palmitil DRUG

Administered by oral inhalation using a Plastiape capsule-based dry powder inhaler

Also known as: INS1009

Treprostinil Palmitil Inhalation Powder

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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)
• Male participants: Male participants and their female partners of childbearing potential must agree to use highly effective contraception from Study Day 1 to at least 90 days after dosing
• Female participants: Women of child-bearing potential (WOCPB, defined as premenopausal, not surgically sterile for at least 3 months prior to Screening) must use a highly effective contraception method and agree to be tested for pregnancy from at Screening, Baseline, and 30 days after dosing
• Capable of giving signed informed consent which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in the protocol.

You may not qualify if:

• 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
• History of active/chronic Hepatitis B or C/ positive hepatitis B or C serology test result at Screening
• History of abnormal bleeding or bruising
• Known or suspected immunodeficiency disorder, including history of invasive opportunistic infections (eg, tuberculosis, histoplasmosis, listeriosis, coccidioidomycosis, pneumocystosis, aspergillosis) despite infection resolution, or otherwise recurrent infections of abnormal frequency, or prolonged infections suggesting an immune-compromised status, as judged by the investigator
• Active and current symptomatic infection by SARS CoV 2
• Participants with current or recent (past 4 weeks) lower respiratory tract infection

Sponsors & Collaborators

• Insmed Incorporated: lead

Study Sites (1)

USA002

New York, New York, 10021-9800, United States

Location

MeSH Terms

Conditions

Pulmonary Arterial Hypertension

Condition Hierarchy (Ancestors)

Hypertension, Pulmonary; Lung Diseases; Respiratory Tract Diseases

Limitations and Caveats

The study was terminated by the Sponsor due to low enrolment. As only 1 participant was enrolled in this study, no data is reported here, in order to protect and maintain participant privacy/confidentiality.

Results Point of Contact

• Title: Insmed Medical Information
• Organization: Insmed Incorporated

medicalinformation@insmed.com

1-844-446-7633

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: LTE60
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 8, 2021
• First Posted: March 10, 2021
• Study Start: March 29, 2022
• Primary Completion: August 26, 2022
• Study Completion: August 26, 2022
• Last Updated: September 22, 2023
• Results First Posted: September 22, 2023

Record last verified: 2023-08

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)