Clinical trial NCT05649748: An Extension Study of Treprostinil Palmitil Inhalation Powder (TPIP) for Pulmonary Arterial Hypertension (PAH)
Official title: An Open-Label Extension Study to Assess the Safety, Tolerability, and Effectiveness of the Long-Term Use of Treprostinil Palmitil Inhalation Powder in Participants With Pulmonary Arterial Hypertension
Study type: INTERVENTIONAL
Overall status: ACTIVE_NOT_RECRUITING
Phase: PHASE2
PHASE3
Conditions: Pulmonary Arterial Hypertension
Keywords: Treprostinil Palmitil
Lead sponsor: Insmed Incorporated
Sponsor class: INDUSTRY
First posted: 2022-12-14
Enrollment: 91
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: The primary purpose of the study is to evaluate the safety and tolerability of the long-term use of TPIP in participants with PAH from studies INS1009-201 (NCT04791514), INS1009-202 (NCT05147805) and other lead-in studies of TPIP in participants with PAH.


Eligibility criteria: Inclusion Criteria:

* Male and female participants who completed end of treatment study visit in Study INS1009-201, INS1009-202, or any other lead-in PAH TPIP study. Participants for whom the OLE study was not available at the time of their completion of the lead-in study are eligible for enrolment within one year of their lead-in end of treatment visit.
* Complete baseline screening assessments to confirm eligibility to participate if more than 30 days have elapsed since the end of the study visit in Study INS1009-201, INS1009-202, or any other lead-in PAH TPIP study.

Exclusion Criteria:

* Initiation of parenteral administration of prostacyclin analogues (eg, TRE, epoprostenol) since the completion of studies INS1009-201, INS1009-202 or other TPIP studies. Initiation of inhaled prostacyclin analogues (eg, TRE \[Tyvaso\] or iloprost) and oral prostacyclin analogues (eg, TRE \[Orenitram\]) or receptor agonists (eg, selexipag) are permitted if stopped 24 hours prior to the start of study drug administration.
* Any new ventricular or supraventricular tachyarrhythmia except for paroxysmal atrial fibrillation and any new symptomatic bradycardia.
* New-onset 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).
* New evidence of thromboembolic disease as assessed by ventilation/perfusion (VQ) scan, pulmonary angiography, or pulmonary computed tomography (CT) scan.
* Active and current symptomatic coronavirus disease 2019 (COVID-19) or previous severe disease and/or hospitalization due to COVID-19.
* Interval organ transplantation.
* New active liver disease or hepatic dysfunction.
* Interval malignancy with exception of completely treated in situ carcinoma of the cervix and completely treated non-metastatic squamous or basal cell carcinoma of the skin.
* Use of any investigational drug/device or participation in any investigational study within 30 days prior to screening, not including TPIP of the lead-in study.
* Current use of cigarettes (as defined by Centers for Disease Control and Prevention \[CDC\]) or e-cigarettes: An adult who has smoked at least 100 cigarettes in his or her lifetime, who smokes either every day or some days.
* Participants who currently inhale marijuana (recreational or medical).

Note: Other inclusion/exclusion criteria may apply.

Primary outcomes:
measure: Number of Participants Who Experience at Least one Treatment Emergent Adverse Event (TEAE) and TEAEs by Severity
timeFrame: From screening up to last follow up visit (Up to approximately 26 months)
Secondary outcomes:
measure: Absolute Change From Pre-Open Label Extension (OLE) Baseline in 6-Minute Walk Distance (6MWD)
timeFrame: Pre-OLE baseline (baseline of the lead-in TPIP study), Months 6, 12, 18, and 24
measure: Relative Change From Pre-OLE Baseline in 6MWD
timeFrame: Pre-OLE baseline (baseline of the lead-in TPIP study), Months 6, 12, 18, and 24
measure: Change From Pre-OLE Baseline in the Concentration of N-Terminal Fragment B-Type Natriuretic Peptide (NT-proBNP) in Blood
timeFrame: Pre-OLE baseline (baseline of the lead-in TPIP study), Months 6, 12, 18, and 24
measure: Change From Pre-OLE Baseline in the Registry to Evaluate Early and Long-Term PAH Disease Management (REVEAL) Lite 2.0 Score
timeFrame: Pre-OLE baseline (baseline of the lead-in TPIP study), Months 6, 12, 18, and 24
measure: Change From Pre-OLE Baseline in New York Heart Association/ World Health Organization (NYHA/WHO) Functional Capacity Class
timeFrame: Pre-OLE baseline (baseline of the lead-in TPIP study), Months 6, 12, 18, and 24
measure: Annualized Clinical Worsening Event Rate
timeFrame: OLE Baseline (Day 1) up to Month 24 or early discontinuation
measure: Plasma Concentration Levels of Treprostinil Palmitil (TP) and Treprostinil (TRE)
timeFrame: OLE Baseline (Day 1), Months 6, 12, 18, and 24
Other outcomes:
Locations:
city: Jacksonville
country: United States
facility: USA005
state: Florida
city: Tampa
country: United States
facility: USA011
state: Florida
city: Chicago
country: United States
facility: USA006
state: Illinois
city: Chicago
country: United States
facility: USA001
state: Illinois
city: New York
country: United States
facility: USA102
state: New York
city: Dallas
country: United States
facility: USA016
state: Texas
city: Quilmes
country: Argentina
facility: ARG009
state: Buenos Aires
city: Rosario
country: Argentina
facility: ARG006
state: Santa Fe
city: San Miguel de Tucuman
country: Argentina
facility: ARG007
state: Tucuman
city: Córdoba
country: Argentina
facility: ARG004
city: Córdoba
country: Argentina
facility: ARG001
city: Linz
country: Austria
facility: AUT002
state: Oberösterreich
city: Wien
country: Austria
facility: AUT001
city: Brussels
country: Belgium
facility: BEL003
city: Belo Horizonte
country: Brazil
facility: BRA004
state: Minas Gerais
city: Porto Alegre
country: Brazil
facility: BRA006
state: Rio Grande Do Sul
city: Blumenau
country: Brazil
facility: BRA002
state: Santa Catarina
city: Aarhus
country: Denmark
facility: DNK001
state: Central Jutland
city: Heidelberg
country: Germany
facility: GER005
state: Baden-Württemberg
city: München
country: Germany
facility: GER003
state: Bavaria
city: Lübeck
country: Germany
facility: GER002
state: Schleswig-Holstein
city: Pavia
country: Italy
facility: ITA002
state: Lombardia
city: Monza
country: Italy
facility: ITA005
city: Palermo
country: Italy
facility: ITA001
city: Roma
country: Italy
facility: ITA004
city: Sapporo-shi
country: Japan
facility: JPN005
state: Hokkaido
city: Sapporo
country: Japan
facility: JPN004
state: Hokkaido
city: Kurume-shi
country: Japan
facility: JPN007
state: Hukuoka
city: Tsukuba
country: Japan
facility: JPN006
state: Ibaraki
city: Okayama-Shi
country: Japan
facility: JPN002
state: Okayama
city: Suita-Shi
country: Japan
facility: JPN003
state: Osaka
city: Alor Setar
country: Malaysia
facility: MYS005
state: Kedah
city: Kuantan
country: Malaysia
facility: MYS002
state: Pahang
city: Sungai Buloh
country: Malaysia
facility: MYS004
state: Selangor
city: Lomas De Guevara
country: Mexico
facility: MEX005
state: Jalisco
city: Monterrey
country: Mexico
facility: MEX001
state: Nuevo León
city: Mexico City
country: Mexico
facility: MEX003
city: San Luis Potosi
country: Mexico
facility: MEX004
city: Quezon City
country: Philippines
facility: PHL001
state: National Capital Region
city: Makati City
country: Philippines
facility: PHL002
city: Belgrade
country: Serbia
facility: SRB001
city: Belgrade
country: Serbia
facility: SRB003
city: Santander
country: Spain
facility: ESP001
city: Sevilla
country: Spain
facility: ESP003
city: Bath
country: United Kingdom
facility: GBR001
state: Avon
Study officials:
References: