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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT05649748




Registration number
NCT05649748
Ethics application status
Date submitted
6/12/2022
Date registered
14/12/2022

Titles & IDs
Public title
An Extension Study of Treprostinil Palmitil Inhalation Powder (TPIP) for Pulmonary Arterial Hypertension (PAH)
Scientific 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
Secondary ID [1] 0 0
2022-001951-18
Secondary ID [2] 0 0
INS1009-203
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Pulmonary Arterial Hypertension 0 0
Condition category
Condition code
Respiratory 0 0 0 0
Other respiratory disorders / diseases
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Cardiovascular 0 0 0 0
Hypertension

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Treprostinil Palmitil
Treatment: Drugs - Placebo

Experimental: Treprostinil Palmitil Inhalation Powder (TPIP) - Participants who are not transitioning immediately from other TPIP studies:INS1009-201(NCT04791514), INS1009-202(NCT05147805) and other lead-in studies, will be given TPIP, once daily (QD), starting with 80 micrograms (µg), up-titrated to highest tolerated dose between 80 µg and 640 µg during 3-week titration period that maybe increased upto maximum dose of 1280 µg QD post initial titration, per investigator's assessment. Overall treatment period=24 months.

Participants transitioning immediately from randomized blinded lead-in TPIP study and who previously received:

1. TPIP- will be given placebo QD(80 µg upto achieved TPIP dose from previous study)along with achieved TPIP dose from previous study in blinded manner during 3-week titration period.
2. Placebo- will be given TPIP QD (80 µg up to achieved placebo dose from previous study)along with achieved placebo dose from previous study in blinded manner during 3-week titration period.Overall treatment period=24 months.


Treatment: Drugs: Treprostinil Palmitil
Administered by oral inhalation, using a Plastiape capsule-based dry powder inhaler.

Treatment: Drugs: Placebo
Administered by oral inhalation using a Plastiape capsule-based dry powder inhaler
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Number of Participants Who Experience at Least one Treatment Emergent Adverse Event (TEAE) and TEAEs by Severity
Timepoint [1] 0 0
From screening up to last follow up visit (Up to approximately 26 months)
Secondary outcome [1] 0 0
Absolute Change From Pre-Open Label Extension (OLE) Baseline in 6-Minute Walk Distance (6MWD)
Timepoint [1] 0 0
Pre-OLE baseline (baseline of the lead-in TPIP study), Months 6, 12, 18, and 24
Secondary outcome [2] 0 0
Relative Change From Pre-OLE Baseline in 6MWD
Timepoint [2] 0 0
Pre-OLE baseline (baseline of the lead-in TPIP study), Months 6, 12, 18, and 24
Secondary outcome [3] 0 0
Change From Pre-OLE Baseline in the Concentration of N-Terminal Fragment B-Type Natriuretic Peptide (NT-proBNP) in Blood
Timepoint [3] 0 0
Pre-OLE baseline (baseline of the lead-in TPIP study), Months 6, 12, 18, and 24
Secondary outcome [4] 0 0
Change From Pre-OLE Baseline in the Registry to Evaluate Early and Long-Term PAH Disease Management (REVEAL) Lite 2.0 Score
Timepoint [4] 0 0
Pre-OLE baseline (baseline of the lead-in TPIP study), Months 6, 12, 18, and 24
Secondary outcome [5] 0 0
Change From Pre-OLE Baseline in New York Heart Association/ World Health Organization (NYHA/WHO) Functional Capacity Class
Timepoint [5] 0 0
Pre-OLE baseline (baseline of the lead-in TPIP study), Months 6, 12, 18, and 24
Secondary outcome [6] 0 0
Annualized Clinical Worsening Event Rate
Timepoint [6] 0 0
OLE Baseline (Day 1) up to Month 24 or early discontinuation
Secondary outcome [7] 0 0
Plasma Concentration Levels of Treprostinil Palmitil (TP) and Treprostinil (TRE)
Timepoint [7] 0 0
OLE Baseline (Day 1), Months 6, 12, 18, and 24

Eligibility
Key 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.
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key 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.

Study design
Purpose of the study
Treatment
Allocation to intervention
Not applicable
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
7/03/2023
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
31/12/2026
Actual
Sample size
Target
100
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Florida
Country [2] 0 0
United States of America
State/province [2] 0 0
Illinois
Country [3] 0 0
United States of America
State/province [3] 0 0
New York
Country [4] 0 0
United States of America
State/province [4] 0 0
Texas
Country [5] 0 0
Argentina
State/province [5] 0 0
Buenos Aires
Country [6] 0 0
Argentina
State/province [6] 0 0
Santa Fe
Country [7] 0 0
Argentina
State/province [7] 0 0
Tucuman
Country [8] 0 0
Argentina
State/province [8] 0 0
Córdoba
Country [9] 0 0
Austria
State/province [9] 0 0
Oberösterreich
Country [10] 0 0
Austria
State/province [10] 0 0
Wien
Country [11] 0 0
Belgium
State/province [11] 0 0
Brussels
Country [12] 0 0
Brazil
State/province [12] 0 0
Minas Gerais
Country [13] 0 0
Brazil
State/province [13] 0 0
Rio Grande Do Sul
Country [14] 0 0
Brazil
State/province [14] 0 0
Santa Catarina
Country [15] 0 0
Denmark
State/province [15] 0 0
Central Jutland
Country [16] 0 0
Germany
State/province [16] 0 0
Baden-Württemberg
Country [17] 0 0
Germany
State/province [17] 0 0
Bavaria
Country [18] 0 0
Germany
State/province [18] 0 0
Schleswig-Holstein
Country [19] 0 0
Italy
State/province [19] 0 0
Lombardia
Country [20] 0 0
Italy
State/province [20] 0 0
Monza
Country [21] 0 0
Italy
State/province [21] 0 0
Palermo
Country [22] 0 0
Italy
State/province [22] 0 0
Roma
Country [23] 0 0
Japan
State/province [23] 0 0
Hokkaido
Country [24] 0 0
Japan
State/province [24] 0 0
Hukuoka
Country [25] 0 0
Japan
State/province [25] 0 0
Ibaraki
Country [26] 0 0
Japan
State/province [26] 0 0
Okayama
Country [27] 0 0
Japan
State/province [27] 0 0
Osaka
Country [28] 0 0
Malaysia
State/province [28] 0 0
Kedah
Country [29] 0 0
Malaysia
State/province [29] 0 0
Pahang
Country [30] 0 0
Malaysia
State/province [30] 0 0
Selangor
Country [31] 0 0
Mexico
State/province [31] 0 0
Jalisco
Country [32] 0 0
Mexico
State/province [32] 0 0
Nuevo León
Country [33] 0 0
Mexico
State/province [33] 0 0
Mexico City
Country [34] 0 0
Mexico
State/province [34] 0 0
San Luis Potosi
Country [35] 0 0
Philippines
State/province [35] 0 0
National Capital Region
Country [36] 0 0
Philippines
State/province [36] 0 0
Makati City
Country [37] 0 0
Serbia
State/province [37] 0 0
Belgrade
Country [38] 0 0
Spain
State/province [38] 0 0
Santander
Country [39] 0 0
Spain
State/province [39] 0 0
Sevilla
Country [40] 0 0
United Kingdom
State/province [40] 0 0
Avon

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Insmed Incorporated
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Insmed Medical Information
Address 0 0
Country 0 0
Phone 0 0
1-844-446-7633
Fax 0 0
Email 0 0
[email protected]
Contact person for scientific queries

Data sharing statement


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results not provided in https://clinicaltrials.gov/study/NCT05649748