A Study to Evaluate the Efficacy, Safety and Pharmacokinetics of Treprostinil Palmitil Inhalation Powder in Participants With Pulmonary Arterial Hypertension

NCT05147805 | Completed Phase 2 DMC FDA Drug

• 3 other identifiers: INS1009-2022021-001528-162023-505541-99-00
• Study Type: interventional
• Target: 102
• Locations: 16 countries
• Sites: 90

Brief Summary

The main objective of the study is to assess the effect of treprostinil palmitil inhalation powder (TPIP) compared with placebo on pulmonary vascular resistance.

Conditions

Pulmonary Arterial Hypertension

Keywords

Treprostinil Palmitil; Pulmonary Arterial Hypertension

Outcome Measures

Primary Outcomes (1)

• Change from Baseline in Pulmonary Vascular Resistance at Week 16

  Baseline to Week 16

Secondary Outcomes (24)

• Change from Baseline in 6-Minute Walk Test Distance at Week 5, Week 10 and Week 16

  Baseline and Week 5, Week 10 and Week 16

• Percent Change from Baseline in 6-Minute Walk Test Distance at Week 5, Week 10 and Week 16

  Baseline and Week 5, Week 10 and Week 16

• Number of Participants Who Experience a Treatment-emergent Adverse Event (AE)

  Day 1 up to Week 20

• Number of Participants Who Experience a Clinically Significant Change from Baseline in Clinical Laboratory Evaluations

  Baseline to Week 16

• Number of Participants Who Experience a Clinically Significant Change from Baseline in Vital Sign Measurements

  Baseline to Week 16

Study Arms (2)

Treprostinil Palmitil Inhalation Powder

EXPERIMENTAL

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.

Drug: Treprostinil Palmitil

Placebo

PLACEBO COMPARATOR

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

Drug: Placebo

Interventions

Treprostinil Palmitil DRUG

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

Also known as: INS1009

Treprostinil Palmitil Inhalation Powder

Placebo DRUG

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

Placebo

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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:
• Idiopathic
• Heritable
• Drug/toxin-induced or connective tissue disease (CTD)-associated PAH
• 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:
• Endothelin receptor antagonists (eg, ambrisentan, bosentan, macitentan)
• Phosphoesterase type 5 inhibitors (eg, sildenafil, tadalafil)
• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Insmed Incorporated: lead

Study Sites (92)

USA025

Phoenix, Arizona, 85006, United States

Location

USA022

Scottsdale, Arizona, 85258, United States

Location

USA021

Tucson, Arizona, 85724, United States

Location

USA023

Sacramento, California, 95817, United States

Location

USA002

West Hollywood, California, 90048, United States

Location

USA008

Gainesville, Florida, 32610, United States

Location

USA005

Jacksonville, Florida, 32224, United States

Location

USA007

Orlando, Florida, 32803, United States

Location

USA011

Tampa, Florida, 33601, United States

Location

USA010

Winter Park, Florida, 32792-3800, United States

Location

USA009

Atlanta, Georgia, 30309, United States

Location

USA006

Chicago, Illinois, 60611, United States

Location

USA001

Chicago, Illinois, 60612, United States

Location

USA013

Indianapolis, Indiana, 46260, United States

Location

USA014

Iowa City, Iowa, 52242, United States

Location

USA003

Kansas City, Kansas, 66160, United States

Location

USA102

New York, New York, 10021, United States

Location

USA017

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

Location

USA016

Dallas, Texas, 75246, United States

Location

USA012

Denison, Texas, 75020, United States

Location

USA018

Houston, Texas, 77030, United States

Location

ARG009

Quilmes, Buenos Aires, B1878GEG, Argentina

Location

ARG002

Rosario, Santa Fe Province, S2013DSR, Argentina

Location

ARG006

Rosario, Santa Fe Province, S2013KDS, Argentina

Location

ARG007

San Miguel de Tucumán, Tucumán Province, T4000AXL, Argentina

Location

ARG004

Córdoba, X5000DCE, Argentina

Location

ARG001

Córdoba, X5000FPQ, Argentina

Location

ARG008

Cuiudad Autónoma de Buenos Aires, C1430EGF, Argentina

Location

AUS005

New Lambton Heights, New South Wales, 2305, Australia

Location

AUS004

Milton, Queensland, 4064, Australia

Location

AUS001

Woolloongabba, Queensland, 4102, Australia

Location

AUS003

Adelaide, South Australia, 5000, Australia

Location

AUS002

Hobart, Tasmania, 7000, Australia

Location

AUT002

Linz, Upper Austria, 4020, Austria

Location

AUT001

Vienna, 1090, Austria

Location

BEL003

Anderlecht, Brussels Capital, 1070, Belgium

Location

BEL002

Leuven, Vlaams Brabant, 3000, Belgium

Location

BEL001

Liège, 4000, Belgium

Location

BRA003

Belo Horizonte, Minas Gerais, 30130, Brazil

Location

BRA004

Belo Horizonte, Minas Gerais, 30441-070, Brazil

Location

BRA007

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

Location

BRA006

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

Location

BRA002

Blumenau, Santa Catarina, 80440-080, Brazil

Location

BRA001

São Paulo, 05403-000, Brazil

Location

DNK001

Aarhus N, Central Jutland, 8200, Denmark

Location

GER005

Heidelberg, Baden-Wurttemberg, 69126, Germany

Location

GER006

Dresden, Saxony, 01307, Germany

Location

GER001

Halle, Saxony-Anhalt, 6120, Germany

Location

GER002

Lübeck, Schleswig-Holstein, 23562, Germany

Location

GER007

Berlin, 12683, Germany

Location

GER003

Munich, 80639, Germany

Location

ITA003

Naples, Campania, 80131, Italy

Location

ITA006

Milan, Lombardy, 20123, Italy

Location

ITA005

Monza, Lombardy, 20900, Italy

Location

ITA002

Pavia, Lombardy, 27100, Italy

Location

ITA001

Palermo, Sicily, 90127, Italy

Location

ITA004

Roma, 00161, Italy

Location

JPN005

Sapporo, Hokkaidô, 060-8543, Japan

Location

JPN004

Sapporo, Hokkaidô, 060-8648, Japan

Location

JPN007

Kurume-Shi, Hukuoka, 830-0011, Japan

Location

JPN006

Tsukuba, Ibaraki, 305-8576, Japan

Location

JPN001

Kagoshima, Kagoshima-ken, 890-8520, Japan

Location

JPN009

Nagasaki, Nagasaki, 852-8501, Japan

Location

JPN002

Okayama, Okayama-ken, 701-1154, Japan

Location

JPN008

Shinjuku-Ku, Tokyo, 160-8582, Japan

Location

JPN003

Suita-Shi, Ôsaka, 564-8565, Japan

Location

MYS005

Alor Star, Kedah, 05460, Malaysia

Location

MYS002

Kuantan, Pahang, 25200, Malaysia

Location

MYS003

Kajang, Selangor, 43000, Malaysia

Location

MYS004

Sungai Buloh, Selangor, 47000, Malaysia

Location

MEX005

Lomas de Guevara, Jalisco, 44657, Mexico

Location

MEX003

Mexico City, Mexico City, 14080, Mexico

Location

MEX004

San Luis Potosí City, 78200, Mexico

Location

MEX001

Sertoma, 64718, Mexico

Location

PHL001

Quezon City, National Capital Region, 1100, Philippines

Location

PHL002

Makati City, 1229, Philippines

Location

SRB004

Belgrade, Belgrade, 116550, Serbia

Location

SRB001

Belgrade, 11000, Serbia

Location

SRB003

Belgrade, 11000, Serbia

Location

ESP006

Palma de Mallorca, Balearic Islands, 07010, Spain

Location

ESP001

Santander, Cantabria, 39008, Spain

Location

ESP002

Barcelona, 8025, Spain

Location

ESP007

Las Palmas, 35010, Spain

Location

ESP008

Madrid, 28046, Spain

Location

ESP003

Seville, 41009, Spain

Location

ESP004

Toledo, 45007, Spain

Location

CHE002

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

Location

GBR001

Bath, Avon, BA1 3NG, United Kingdom

Location

GBR002

Glasgow, Lanarkshire, G81 4HX, United Kingdom

Location

GBR006

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

Location

GBR003

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

Location

GBR004

London, NW3 2Q, United Kingdom

Location

MeSH Terms

Conditions

Pulmonary Arterial Hypertension

Condition Hierarchy (Ancestors)

Hypertension, Pulmonary; Lung Diseases; Respiratory Tract Diseases

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 24, 2021
• First Posted: December 7, 2021
• Study Start: August 24, 2022
• Primary Completion: March 27, 2025
• Study Completion: March 27, 2025
• Last Updated: March 30, 2026

Record last verified: 2026-03

Data Sharing

• IPD Sharing: Will not share

Locations

BR (6); IT (6); ME (4); JP (9); AU (5); ES (7); DE (6); BE (3); SE (3); PH (2); GB (5); AR (7); DK (1); US (21); MY (4); CH (1)