Back to resultsA 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)
Primary Purpose
Pulmonary Arterial Hypertension
Status
Recruiting
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
Treprostinil Palmitil
Placebo
Sponsored by
About this trial
This is an interventional treatment trial for Pulmonary Arterial Hypertension focused on measuring Treprostinil Palmitil
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.
Sites / Locations
- USA006Recruiting
- USA016Recruiting
- ARG006Recruiting
- ARG007Recruiting
- GER005Recruiting
- ITA002Recruiting
- JPN002Recruiting
- JPN003Recruiting
- MYS002Recruiting
- PHL002Recruiting
- GBR001Recruiting
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Treprostinil Palmitil Inhalation Powder (TPIP)
Arm Description
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.
Outcomes
Primary Outcome Measures
Number of Participants Who Experience at Least one Treatment Emergent Adverse Event (TEAE) and TEAEs by Severity
Secondary Outcome Measures
Absolute Change From Pre-Open Label Extension (OLE) Baseline in 6-Minute Walk Distance (6MWD)
Relative Change From Pre-OLE Baseline in 6MWD
Change From Pre-OLE Baseline in the Concentration of N-Terminal Fragment B-Type Natriuretic Peptide (NT-proBNP) in Blood
Change From Pre-OLE Baseline in the Registry to Evaluate Early and Long-Term PAH Disease Management (REVEAL) Lite 2.0 Score
Change From Pre-OLE Baseline in New York Heart Association/ World Health Organization (NYHA/WHO) Functional Capacity Class
Annualized Clinical Worsening Event Rate
Annualized clinical worsening event rate is defined as the total number of clinical worsening events that occurred during the treatment period divided by the total number of participant-years during the treatment period. Clinical worsening events are one of the following: All-cause death, or onset of TEAE with a fatal outcome occurring ≤ 14 days after study drug discontinuation; Hospitalization for right heart failure (for > 48 hours), heart-lung or lung transplant, or atrial septostomy; Addition (or increase in dose) of specified PAH-specific medications; Combined occurrence of events including ≥20% decrease in 6MWD, worsening WHO/NYHA functional capacity class, and appearance of or worsening of signs/symptoms of right heart failure from baseline.
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT05649748
Brief 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)
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
2. Study Status
Record Verification Date
June 2023
Overall Recruitment Status
Recruiting
Study Start Date
March 7, 2023 (Actual)
Primary Completion Date
December 31, 2026 (Anticipated)
Study Completion Date
December 31, 2026 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Insmed Incorporated
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Pulmonary Arterial Hypertension
Keywords
Treprostinil Palmitil
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2, Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
100 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
Treprostinil Palmitil Inhalation Powder (TPIP)
Arm Type
Experimental
Arm Description
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.
Intervention Type
Drug
Intervention Name(s)
Treprostinil Palmitil
Other Intervention Name(s)
INS1009
Intervention Description
Administered by oral inhalation, using a Plastiape capsule-based dry powder inhaler.
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
Administered by oral inhalation using a Plastiape capsule-based dry powder inhaler
Primary Outcome Measure Information:
Title
Number of Participants Who Experience at Least one Treatment Emergent Adverse Event (TEAE) and TEAEs by Severity
Time Frame
From screening up to last follow up visit (Up to approximately 26 months)
Secondary Outcome Measure Information:
Title
Absolute Change From Pre-Open Label Extension (OLE) Baseline in 6-Minute Walk Distance (6MWD)
Time Frame
Pre-OLE baseline (baseline of the lead-in TPIP study), Months 6, 12, 18, and24
Title
Relative Change From Pre-OLE Baseline in 6MWD
Time Frame
Pre-OLE baseline (baseline of the lead-in TPIP study), Months 6, 12, 18, and 24
Title
Change From Pre-OLE Baseline in the Concentration of N-Terminal Fragment B-Type Natriuretic Peptide (NT-proBNP) in Blood
Time Frame
Pre-OLE baseline (baseline of the lead-in TPIP study), Months 6, 12, 18, and 24
Title
Change From Pre-OLE Baseline in the Registry to Evaluate Early and Long-Term PAH Disease Management (REVEAL) Lite 2.0 Score
Time Frame
Pre-OLE baseline (baseline of the lead-in TPIP study), Months 6, 12, 18, and 24
Title
Change From Pre-OLE Baseline in New York Heart Association/ World Health Organization (NYHA/WHO) Functional Capacity Class
Time Frame
Pre-OLE baseline (baseline of the lead-in TPIP study), Months 6, 12, 18, and 24
Title
Annualized Clinical Worsening Event Rate
Description
Annualized clinical worsening event rate is defined as the total number of clinical worsening events that occurred during the treatment period divided by the total number of participant-years during the treatment period. Clinical worsening events are one of the following: All-cause death, or onset of TEAE with a fatal outcome occurring ≤ 14 days after study drug discontinuation; Hospitalization for right heart failure (for > 48 hours), heart-lung or lung transplant, or atrial septostomy; Addition (or increase in dose) of specified PAH-specific medications; Combined occurrence of events including ≥20% decrease in 6MWD, worsening WHO/NYHA functional capacity class, and appearance of or worsening of signs/symptoms of right heart failure from baseline.
Time Frame
Baseline up to Month 24 or early discontinuation
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
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.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Insmed Medical Information
Phone
1-844-446-7633
Email
medicalinformation@insmed.com
Facility Information:
Facility Name
USA006
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60611
Country
United States
Individual Site Status
Recruiting
Facility Name
USA016
City
Dallas
State/Province
Texas
ZIP/Postal Code
75246
Country
United States
Individual Site Status
Recruiting
Facility Name
ARG006
City
Rosario
State/Province
Santa Fe
ZIP/Postal Code
S2013KDS
Country
Argentina
Individual Site Status
Recruiting
Facility Name
ARG007
City
San Miguel de Tucuman
State/Province
Tucuman
ZIP/Postal Code
T4000AXL
Country
Argentina
Individual Site Status
Recruiting
Facility Name
GER005
City
Heidelberg
State/Province
Baden-Württemberg
ZIP/Postal Code
69126
Country
Germany
Individual Site Status
Recruiting
Facility Name
ITA002
City
Pavia
State/Province
Lombardia
ZIP/Postal Code
27100
Country
Italy
Individual Site Status
Recruiting
Facility Name
JPN002
City
Okayama-Shi
State/Province
Okayama
ZIP/Postal Code
701-1192
Country
Japan
Individual Site Status
Recruiting
Facility Name
JPN003
City
Suita-Shi
State/Province
Osaka
ZIP/Postal Code
564-8565
Country
Japan
Individual Site Status
Recruiting
Facility Name
MYS002
City
Kuantan
State/Province
Pahang
ZIP/Postal Code
25200
Country
Malaysia
Individual Site Status
Recruiting
Facility Name
PHL002
City
Makati City
ZIP/Postal Code
1229
Country
Philippines
Individual Site Status
Recruiting
Facility Name
GBR001
City
Bath
State/Province
Avon
ZIP/Postal Code
BA1 3NG
Country
United Kingdom
Individual Site Status
Recruiting
12. IPD Sharing Statement
Plan to Share IPD
No
Learn more about this trial
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)
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