Pulmonary Arterial HypertensionPhase 2NCT04791514
A Study of Treprostinil Palmitil Inhalation Powder (TPIP) In Pulmonary Arterial Hypertension (PAH)
A Phase 2 study testing Treprostinil Palmitil in people with Pulmonary Arterial Hypertension, run by Insmed Incorporated at 1 site in United States.
Trial summary
The main purpose of this study is to evaluate the safety and tolerability of single dose of treprostinil palmitil inhalation powder (TPIP) in participants with pulmonary arterial hypertension (PAH). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Pulmonary Arterial Hypertension
- What is being tested?
- Treprostinil Palmitil
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 1 participants
- Where
- 1 siteUnited States
- Study dates
- 29 Mar 2022to 26 Aug 2022
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Participant must be ≥ 18 years of age at the time of signing the informed consent
- Participants must have a diagnosis of World Health Organization Group 1 Pulmonary Hypertension (PH) (PAH) with the following characteristics
- Etiology of idiopathic, heritable, drug/toxin-induced or connective tissue disease (CTD)-related PAH
- Right heart catheterization with the following hemodynamic findings:
- Mean pulmonary arterial pressure (mPAP) > 20 mmHg at rest,
- Pulmonary capillary wedge pressure (PCWP) ≤ 15 mmHg, and
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- Pulmonary vascular resistance (PVR) of ≥ 3 Wood Units (WU)
- No change in pulmonary hypertension medications (eg, ambrisentan, bosentan, macitentan, sildenafil, tadalafil, riociguat) or dosage for at least 90 days prior to Screening
- No change in diuretic use or dosage for at least 30 days prior to Screening
- Body mass index (BMI) within the range 18.0 - 32.0 kg/m^2 (inclusive)
- Male participants: Male participants and their female partners of childbearing potential must agree to use highly effective contraception from Study Day 1 to at least 90 days after dosing
- Female participants: Women of child-bearing potential (WOCPB, defined as premenopausal, not surgically sterile for at least 3 months prior to Screening) must use a highly effective contraception method and agree to be tested for pregnancy from at Screening, Baseline, and 30 days after dosing
- Capable of giving signed informed consent which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in the protocol.
Who cannot join
- Any PH other than idiopathic, hereditary, drug/toxin-induced, or connective tissue disease (CTD) associated PAH (eg, congenital heart disease-associated PAH, portal hypertension-associated PAH, PH belonging to Groups 2 through 5)
- Allergy, or documented hypersensitivity or contraindication, to the ingredients of treprostinil palmitil inhalation powder (TPIP) or treprostinil (TRE)
- Previous intolerance to prostacyclin analogs or receptor agonists (eg, selexipag) per investigator discretion
- History of anaphylaxis or previously documented hypersensitivity reaction to any drug per Investigator discretion
- History of heart disease including left ventricular ejection fraction (LVEF) ≤ 40% or clinically significant valvular, constrictive, or atherosclerotic heart disease (myocardial infarction, etc)
- Active liver disease or hepatic dysfunction manifested as:
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- Elevated liver function test results (ALT or AST > 2 × ULN) at Screening
- Bilirubin > 1.5 × ULN (isolated bilirubin > 1.5 × ULN; ULN is acceptable if bilirubin is fractionated and direct bilirubin < 35%) at Screening.
- Known hepatic or biliary abnormalities, not including Gilbert's syndrome or asymptomatic gallstones at Screening.
- History of HIV infection/positive HIV serology test result at Screening
- History of active/chronic Hepatitis B or C/ positive hepatitis B or C serology test result at Screening
- History of abnormal bleeding or bruising
- Known or suspected immunodeficiency disorder, including history of invasive opportunistic infections (eg, tuberculosis, histoplasmosis, listeriosis, coccidioidomycosis, pneumocystosis, aspergillosis) despite infection resolution, or otherwise recurrent infections of abnormal frequency, or prolonged infections suggesting an immune-compromised status, as judged by the investigator
- Active and current symptomatic infection by SARS CoV 2
- Participants with current or recent (past 4 weeks) lower respiratory tract infection
- History of malignancy in the past 5 years, with exception of completely treated in situ carcinoma of the cervix and completely treated non-metastatic squamous or basal cell carcinoma of the skin
- Participants receiving triple combination therapy for PAH consisting of endothelin receptor agonists, phosphoesterase type 5 inhibitors, and guanylate cyclase stimulators (riociguat)
- Participants receiving prostanoids/prostacyclin agonists
- Participants receiving potent CYP2C8 inhibitors, such as gemfibrozil
- Have participated in any other interventional clinical studies within 30 days of Baseline
- Current or history of substance and/or alcohol abuse
- Current user of cigarettes or e-cigarettes
- Pregnant or breastfeeding
Trial locations and contacts
1 site in 1 country.
About this study
Official title: An Open-Label Study to Assess the Safety, Pharmacokinetics, and Pharmacodynamics of Treprostinil Palmitil Inhalation Powder in Participants With Pulmonary Arterial Hypertension
The main purpose of this study is to evaluate the safety and tolerability of single dose of treprostinil palmitil inhalation powder (TPIP) in participants with pulmonary arterial hypertension (PAH).
- Conditions
- Pulmonary Arterial Hypertension
- Being tested
- Treprostinil Palmitil
- Keywords
- Pulmonary Arterial Hypertension, Treprostinil Palmitil Inhalation Powder, Treprostinil Palmitil
- Medical subject headings
- Pulmonary Arterial Hypertension
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Treprostinil PalmitilDrug
Also called INS1009
Administered by oral inhalation using a Plastiape capsule-based dry powder inhaler
| Study group | Role | What they receive |
|---|---|---|
| Treprostinil Palmitil Inhalation Powder | Experimental | Participant received a single dose of TPIP 112.5 micrograms (μg) via oral inhalation on Day 1. The participant then entered into a 16-week Extended Use Treatment (EUT) Period during which TPIP, administered via oral inhalation, was titrated up to a mean daily dose of 320 μg. Receives: Drug: Treprostinil Palmitil |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants Who Experience a Treatment Emergent Adverse Event (TEAE)
Measured: Up to 150 days
Secondary outcomes (6)
Change From Baseline in Pulmonary Vascular Resistance (PVR) After TPIP Administration
Measured: Day 1: Pre-treatment (Baseline), 8 and 24 hours post-treatment
Maximum Observed Concentration (Cmax) of Treprostinil (TRE) in Plasma
Measured: Pre-dose and multiple timepoints post-dose up to Day 2
Time to Maximum Concentration (Tmax) of Treprostinil (TRE) in Plasma
Measured: Pre-dose and multiple timepoints post-dose up to Day 2
Area Under the Concentration-time Curve From Time 0 to Time of the Last Measurable Concentration (AUClast) of Treprostinil (TRE) in Plasma
Measured: Pre-dose and multiple timepoints post-dose up to Day 2
Area Under the Concentration-time Curve From Time 0 to Infinity (AUC0-∞) of Treprostinil (TRE) in Pasma
Measured: Pre-dose and multiple timepoints post-dose up to Day 2
Elimination Half-life (t1/2) of Treprostinil (TRE) in Plasma
Measured: Pre-dose and multiple timepoints post-dose up to Day 2
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Mar 2021
- Study start29 Mar 2022
- Primary completion26 Aug 2022
- Study completion26 Aug 2022
Why it stopped: Low enrolment
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Pulmonary Arterial HypertensionStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 1 participants (actual)
Sponsor and investigators
- Lead sponsor
- Insmed Incorporated
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04791514
- Sponsor's protocol ID
- INS1009-201
- Status last verified
- Aug 2023
- First submitted
- 8 Mar 2021
- Last updated
- 22 Sep 2023
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 22 Sep 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-Label Study to Assess the Safety, Pharmacokinetics, and Pharmacodynamics of Treprostinil Palmitil Inhalation Powder in Participants With Pulmonary Arterial Hypertension
- ClinicalTrials.gov ID
- NCT04791514
- Acronym / sponsor ID
- INS1009-201
- Conditions
- Pulmonary Arterial Hypertension
- Keywords
- Pulmonary Arterial Hypertension, Treprostinil Palmitil Inhalation Powder, Treprostinil Palmitil
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Low enrolment
- Enrolment
- 1 participants (actual)
- Lead sponsor
- Insmed IncorporatedIndustry (pharma, biotech, devices)
- Study start
- 29 Mar 2022 (actual)
- Primary completion
- 26 Aug 2022 (actual)
- Study completion
- 26 Aug 2022 (actual)
- First posted
- 10 Mar 2021 (actual)
- Results
- Posted 22 Sep 2023
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The main purpose of this study is to evaluate the safety and tolerability of single dose of treprostinil palmitil inhalation powder (TPIP) in participants with pulmonary arterial hypertension (PAH).
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Treprostinil Palmitil Inhalation Powder | Experimental | Drug: Treprostinil PalmitilParticipant received a single dose of TPIP 112.5 micrograms (μg) via oral inhalation on Day 1. The participant then entered into a 16-week Extended Use Treatment (EUT) Period during which TPIP, administered via oral inhalation, was titrated up to a mean daily dose of 320 μg. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (13 listed)
- Participant must be ≥ 18 years of age at the time of signing the informed consent
- Participants must have a diagnosis of World Health Organization Group 1 Pulmonary Hypertension (PH) (PAH) with the following characteristics
- Mean pulmonary arterial pressure (mPAP) > 20 mmHg at rest,
- Pulmonary capillary wedge pressure (PCWP) ≤ 15 mmHg, and
- Pulmonary vascular resistance (PVR) of ≥ 3 Wood Units (WU)
Key exclusion criteria (23 listed)
- Any PH other than idiopathic, hereditary, drug/toxin-induced, or connective tissue disease (CTD) associated PAH (eg, congenital heart disease-associated PAH, portal hypertension-associated PAH, PH belonging to Groups 2 through 5)
- Allergy, or documented hypersensitivity or contraindication, to the ingredients of treprostinil palmitil inhalation powder (TPIP) or treprostinil (TRE)
- Previous intolerance to prostacyclin analogs or receptor agonists (eg, selexipag) per investigator discretion
- History of anaphylaxis or previously documented hypersensitivity reaction to any drug per Investigator discretion
- History of heart disease including left ventricular ejection fraction (LVEF) ≤ 40% or clinically significant valvular, constrictive, or atherosclerotic heart disease (myocardial infarction, etc)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Treprostinil PalmitilAlso: INS1009Administered by oral inhalation using a Plastiape capsule-based dry powder inhaler | Drug | Treprostinil Palmitil Inhalation Powder |
Primary outcome
Number of Participants Who Experience a Treatment Emergent Adverse Event (TEAE)
Time frame: Up to 150 days
6 secondary outcomes
Change From Baseline in Pulmonary Vascular Resistance (PVR) After TPIP Administration
Time frame: Day 1: Pre-treatment (Baseline), 8 and 24 hours post-treatment
Maximum Observed Concentration (Cmax) of Treprostinil (TRE) in Plasma
Time frame: Pre-dose and multiple timepoints post-dose up to Day 2
Time to Maximum Concentration (Tmax) of Treprostinil (TRE) in Plasma
Time frame: Pre-dose and multiple timepoints post-dose up to Day 2
Area Under the Concentration-time Curve From Time 0 to Time of the Last Measurable Concentration (AUClast) of Treprostinil (TRE) in Plasma
Time frame: Pre-dose and multiple timepoints post-dose up to Day 2
Area Under the Concentration-time Curve From Time 0 to Infinity (AUC0-∞) of Treprostinil (TRE) in Pasma
Time frame: Pre-dose and multiple timepoints post-dose up to Day 2
Elimination Half-life (t1/2) of Treprostinil (TRE) in Plasma
Time frame: Pre-dose and multiple timepoints post-dose up to Day 2
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Sep 2023.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Treprostinil Palmitil in people with Pulmonary Arterial Hypertension, run by Insmed Incorporated at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 13 inclusion and 23 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 22 Sep 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 22 Sep 2023). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Participants Who Experience a Treatment Emergent Adverse Event (TEAE) (time frame: Up to 150 days). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 22 Sep 2023.