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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/ct2/show/NCT05649748




Registration number
NCT05649748
Ethics application status
Date submitted
6/12/2022
Date registered
14/12/2022
Date last updated
13/06/2023

Titles & IDs
Public title
A Study to Assess the Safety, Tolerability, and Effectiveness of the Long-Term Use of Treprostinil Palmitil Inhalation Powder (TPIP) in Participants With 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 administered TPIP, once daily (QD), at a starting dose of 80 micrograms (µg), up-titrated to the highest tolerated dose between 80 µg and 640 µg during 3-week titration period. The overall treatment period will be 24 months. Participants who are transitioning immediately from a randomized blinded lead-in TPIP study and who previously received:
TPIP- will be administered placebo QD (80 µg up to achieved TPIP dose from previous study) along with the achieved TPIP dose from previous study in a blinded manner during 3-week titration period.
Placebo- will be administered TPIP QD (80 µg up to achieved placebo dose from previous study) along with the achieved placebo dose from previous study in a blinded manner during 3-week titration period. The overall treatment period will be 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, and24
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
Baseline up to Month 24 or early discontinuation

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.

Study design
Purpose of the study
Treatment
Allocation to intervention
N/A
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/Phase 3
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
Illinois
Country [2] 0 0
United States of America
State/province [2] 0 0
Texas
Country [3] 0 0
Argentina
State/province [3] 0 0
Santa Fe
Country [4] 0 0
Argentina
State/province [4] 0 0
Tucuman
Country [5] 0 0
Germany
State/province [5] 0 0
Baden-Württemberg
Country [6] 0 0
Italy
State/province [6] 0 0
Lombardia
Country [7] 0 0
Japan
State/province [7] 0 0
Okayama
Country [8] 0 0
Japan
State/province [8] 0 0
Osaka
Country [9] 0 0
Malaysia
State/province [9] 0 0
Pahang
Country [10] 0 0
Philippines
State/province [10] 0 0
Makati City
Country [11] 0 0
United Kingdom
State/province [11] 0 0
Avon

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

Ethics approval
Ethics application status

Summary
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.
Trial website
https://clinicaltrials.gov/ct2/show/NCT05649748
Trial related presentations / publications
Public notes
This record is viewable in the ANZCTR as it had previously listed Australia and/or New Zealand as a recruitment site, however these sites have since been removed

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



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/ct2/show/NCT05649748