New inhaled drug shows promise for rare lung condition

NCT ID NCT05147805

Disease control Sponsor: Insmed Incorporated Source: ClinicalTrials.gov ↗

First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This phase 2b trial tested an inhaled powder form of treprostinil palmitil in 102 adults with pulmonary arterial hypertension (PAH), a condition where high blood pressure in the lungs makes it hard to breathe. The study compared the drug to a placebo to see if it could lower lung blood vessel resistance over 16 weeks. It also tracked walking distance and side effects.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance: Treprostinil palmitil inhalation powder
What this could lead to: If successful, this could lead to a new inhaled treatment option to help control pulmonary arterial hypertension by improving blood flow in the lungs.
What could go wrong: This is a mid-stage trial with only 102 participants, so results may not confirm effectiveness or safety. The drug may not work better than placebo or could cause side effects.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

102 people

The number who actually took part.

Started

Aug 2022

Finished

Mar 2025

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 to 75 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: * Participants must be ≥ 18 to ≤ 75 years at the time of signing the informed consent form (ICF). * Participants must have a diagnosis of World Health Organization (WHO) Group 1 Pulmonary Hypertension (PH) \[pulmonary arterial hypertension (PAH)\] in any of the following subtypes: 1. Idiopathic 2. Heritable 3. Drug/toxin-induced or connective tissue disease (CTD)-associated PAH 4. Congenital heart disease-related with simple systemic-to-pulmonary shunt at least 1 year following repair. * PAH diagnosis for at least 3 months. * Participants must be on stable PH therapy consisting of up to 2 medications from the following classes: 1. Endothelin receptor antagonists (eg, ambrisentan, bosentan, macitentan) 2. Phosphoesterase type 5 inhibitors (eg, sildenafil, tadalafil) 3. Guanylate cyclase stimulator (eg, riociguat) * No change in PH medications (eg, ambrisentan, bosentan, macitentan, sildenafil, tadalafil, riociguat) or dosage for at least 30 days prior to Screening. * No change in long-term diuretic use or dosage for at least 30 days prior to Screening. * Body Mass Index (BMI) within the range 18.0-37.0 kg/m\^2 (inclusive). * Male participants: Male participants who are not sterile and have female partners of childbearing potential, must be using effective contraception from Day 1 to at least 90 days after the last dose of study drug. * Female participants: Women must be postmenopausal (defined as no menses for 12 months without an alternative medical cause), surgically sterile, (ie, post-tubal ligation for at least 12 months) or using highly effective contraception methods (ie, methods that alone or in combination achieve \<1% unintended pregnancy rates per year when used consistently and correctly) from Day 1 to at least 90 days after the last dose of study drug. * Male participants with pregnant or non-pregnant woman of childbearing potential partner must use a condom in order to avoid potential exposure to embryo/fetus. * Capable of giving signed informed consent which includes compliance with the requirements and restrictions listed in the ICF and in the protocol. Exclusion Criteria: * History of PH other than idiopathic, hereditary, drug/toxin-induced, repaired simple congenital heart disease, or CTD-associated PAH (eg, complex, congenital heart disease-associated PAH, portal hypertension-associated PAH, PH belonging to Groups 2 through 5). * Allergy, or documented hypersensitivity or contraindication, to TPIP or Treprostinil or mannitol (an excipient of the TPIP formulation). * Any known ventricular or supraventricular tachyarrhythmia except for paroxysmal atrial fibrillation and any symptomatic bradycardia. * History of heart disease including left ventricular ejection fraction (LVEF) ≤ 40% or clinically significant valvular, constrictive, or symptomatic atherosclerotic heart disease (eg, stable angina, myocardial infarction, etc). * Participation in a cardio-pulmonary rehabilitation program within 1 month of Screening Visit. * Evidence of thromboembolic disease as assessed by ventilation-perfusion (VQ) scan, pulmonary angiography, or pulmonary computed tomography (CT) scan. * Active liver disease or hepatic dysfunction. * History of HIV infection. * Established diagnosis of hepatitis B viral infection, or positive for hepatitis B surface antigen (HBsAg) at the time of Screening. * Established diagnosis of hepatitis C viral infection at the time of screening. * Active and current symptomatic coronavirus disease 2019 (COVID-19) or previous severe disease and/or hospitalization due to COVID-19. * Use of live attenuated vaccines within 30 days of the Screening Visit. * Participants with Down's Syndrome. * History of abnormal bleeding or bruising. * History of solid organ transplantation. * 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. * History of alcohol or drug abuse within 6 months prior to Screening. * Acute or chronic impairment (other than dyspnea), limiting the ability to comply with study requirements, in particular with 6-minute walk test (eg, angina pectoris, claudication, musculoskeletal disorder, need for walking aids). * Participants with current or recent (past 30 days) lower respiratory tract infection. * History of malignancy in the past 5 years, with exception of completely treated in situ carcinoma of the cervix and completely treated non-metastatic squamous or basal cell carcinoma of the skin. * Change in PH medication (endothelin receptor agonists, phosphoesterase type 5 inhibitors, and guanylate cyclase stimulators or diuretics) between Screening and Baseline. * Have participated in any other interventional clinical studies within 30 days prior to Screening. * Current use of cigarettes (as defined by Centers for Disease Control and Prevention) or e-cigarettes. * Participants who currently inhale marijuana (recreational or medical). * Pregnant or breastfeeding. Note: Other inclusion/exclusion criteria may apply.

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Conditions

The condition(s) this trial relates to.

pulmonary arterial hypertension

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Pulmonary arterial hypertension

Contacts and locations

Locations

ARG001

Córdoba, X5000FPQ, Argentina
ARG002

Rosario, Santa Fe Province, S2013DSR, Argentina
ARG004

Córdoba, X5000DCE, Argentina
ARG006

Rosario, Santa Fe Province, S2013KDS, Argentina
ARG007

San Miguel de Tucumán, Tucumán Province, T4000AXL, Argentina
ARG008

Cuiudad Autónoma de Buenos Aires, C1430EGF, Argentina
ARG009

Quilmes, Buenos Aires, B1878GEG, Argentina
AUS001

Woolloongabba, Queensland, 4102, Australia
AUS002

Hobart, Tasmania, 7000, Australia
AUS003

Adelaide, South Australia, 5000, Australia
AUS004

Milton, Queensland, 4064, Australia
AUS005

New Lambton Heights, New South Wales, 2305, Australia
AUT001

Vienna, 1090, Austria
AUT002

Linz, Upper Austria, 4020, Austria
BEL001

Liège, 4000, Belgium
BEL002

Leuven, Vlaams Brabant, 3000, Belgium
BEL003

Anderlecht, Brussels Capital, 1070, Belgium
BRA001

São Paulo, 05403-000, Brazil
BRA002

Blumenau, Santa Catarina, 80440-080, Brazil
BRA003

Belo Horizonte, Minas Gerais, 30130, Brazil
BRA004

Belo Horizonte, Minas Gerais, 30441-070, Brazil
BRA006

Porto Alegre, Rio Grande do Sul, 90035-003, Brazil
BRA007

Passo Fundo, Rio Grande do Sul, 99010-120, Brazil
CHE002

Lausanne, Vaud (fr), CH-1011, Switzerland
DNK001

Aarhus N, Central Jutland, 8200, Denmark
ESP001

Santander, Cantabria, 39008, Spain
ESP002

Barcelona, 8025, Spain
ESP003

Seville, 41009, Spain
ESP004

Toledo, 45007, Spain
ESP006

Palma de Mallorca, Balearic Islands, 07010, Spain
ESP007

Las Palmas, 35010, Spain
ESP008

Madrid, 28046, Spain
GBR001

Bath, Avon, BA1 3NG, United Kingdom
GBR002

Glasgow, Lanarkshire, G81 4HX, United Kingdom
GBR003

Newcastle upon Tyne, Tyne and Wear, NE3 3HD, United Kingdom
GBR004

London, NW3 2Q, United Kingdom
GBR006

London, London, City of, W12 0HS, United Kingdom
GER001

Halle, Saxony-Anhalt, 6120, Germany
GER002

Lübeck, Schleswig-Holstein, 23562, Germany
GER003

Munich, 80639, Germany
GER005

Heidelberg, Baden-Wurttemberg, 69126, Germany
GER006

Dresden, Saxony, 01307, Germany
GER007

Berlin, 12683, Germany
ITA001

Palermo, Sicily, 90127, Italy
ITA002

Pavia, Lombardy, 27100, Italy
ITA003

Naples, Campania, 80131, Italy
ITA004

Roma, 00161, Italy
ITA005

Monza, Lombardy, 20900, Italy
ITA006

Milan, Lombardy, 20123, Italy
JPN001

Kagoshima, Kagoshima-ken, 890-8520, Japan
JPN002

Okayama, Okayama-ken, 701-1154, Japan
JPN003

Suita-Shi, Ôsaka, 564-8565, Japan
JPN004

Sapporo, Hokkaidô, 060-8648, Japan
JPN005

Sapporo, Hokkaidô, 060-8543, Japan
JPN006

Tsukuba, Ibaraki, 305-8576, Japan
JPN007

Kurume-Shi, Hukuoka, 830-0011, Japan
JPN008

Shinjuku-Ku, Tokyo, 160-8582, Japan
JPN009

Nagasaki, Nagasaki, 852-8501, Japan
MEX001

Sertoma, 64718, Mexico
MEX003

Mexico City, Mexico City, 14080, Mexico
MEX004

San Luis Potosí City, 78200, Mexico
MEX005

Lomas de Guevara, Jalisco, 44657, Mexico
MYS002

Kuantan, Pahang, 25200, Malaysia
MYS003

Kajang, Selangor, 43000, Malaysia
MYS004

Sungai Buloh, Selangor, 47000, Malaysia
MYS005

Alor Star, Kedah, 05460, Malaysia
PHL001

Quezon City, National Capital Region, 1100, Philippines
PHL002

Makati City, 1229, Philippines
SRB001

Belgrade, 11000, Serbia
SRB003

Belgrade, 11000, Serbia
SRB004

Belgrade, Belgrade, 116550, Serbia
USA001

Chicago, Illinois, 60612, United States
USA002

West Hollywood, California, 90048, United States
USA003

Kansas City, Kansas, 66160, United States
USA005

Jacksonville, Florida, 32224, United States
USA006

Chicago, Illinois, 60611, United States
USA007

Orlando, Florida, 32803, United States
USA008

Gainesville, Florida, 32610, United States
USA009

Atlanta, Georgia, 30309, United States
USA010

Winter Park, Florida, 32792-3800, United States
USA011

Tampa, Florida, 33601, United States
USA012

Denison, Texas, 75020, United States
USA013

Indianapolis, Indiana, 46260, United States
USA014

Iowa City, Iowa, 52242, United States
USA016

Dallas, Texas, 75246, United States
USA017

New York, New York, 10032-1559, United States
USA018

Houston, Texas, 77030, United States
USA021

Tucson, Arizona, 85724, United States
USA022

Scottsdale, Arizona, 85258, United States
USA023

Sacramento, California, 95817, United States
USA025

Phoenix, Arizona, 85006, United States
USA102

New York, New York, 10021, United States

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