A Long-term Open-Label Extension Study of Treprostinil Palmitil Inhalation Powder for Treatment of Pulmonary Hypertension Associated with Interstitial Lung Disease

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Insmed Inc.

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Respiratory Tract Diseases [C08]

Decision date (initial)

2026-07-05

Transition trial

No

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

Funding sources

Insmed Incorporated

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Pharmacokinetic, Efficacy, Pharmacodynamic

To evaluate the safety and tolerability of the long-term use of TPIP in participants with PHILD from Study INS1009-311

Secondary objectives 8

1. To evaluate the effect of the long-term use of TPIP on exercise capacity
2. To evaluate the effect of the long-term use of TPIP on pulmonary function
3. To evaluate the effect of the long-term use of TPIP on blood biomarkers of disease severity
4. To evaluate the effect of the long-term use of TPIP on ILD exacerbations
5. To evaluate the effect of the long-term use of TPIP on the clinical worsening rate
6. To evaluate the effect of the long-term use of TPIP on participant reported disease-related symptoms and associated physical activities
7. To evaluate the effect of the long-term use of TPIP on health-related quality of life
8. To evaluate the effect of the long-term use of TPIP on major morbidity and mortality

Conditions and MedDRA coding

Pulmonary Hypertension Associated with Interstitial Lung Disease

Study design 3 periods

Regulatory references

Scientific advice from competent authorities

European Medicines Agency

Plan to share IPD

No

IPD plan description

N/A

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 3

1. Participants who have completed the lead-in PH-ILD TPIP Study INS1009-311.
2. Capable of giving signed informed consent that includes compliance with the requirements and restrictions listed in the ICF and in this protocol.
3. Agree not to participate in any other interventional trials or use investigational drugs or devices while participating in the INS1009-312 study.

Exclusion criteria 8

1. Participants who experienced any AEs evaluated as causally related to TPIP by the Investigator in a lead-in study , which in the opinion of the Investigator, could pose an unreasonable risk of continued treatments for the participant.
2. Current use or expected need for PAH-approved therapy, including prostacyclin, prostacyclin analogues or other prostacyclin receptor agonists, endothelin receptor antagonists, and/or soluble guanylate cyclase stimulator, or any PH-ILD approved treprostinil therapy. Use of phosphodiesterase 5 inhibitors in line with applicable guidelines is allowed.
3. Pregnant or breastfeeding. Male and female (WOCBP) participants must use contraceptives that are consistent with local regulations regarding the methods of contraception for those participating in clinical studies (contraceptive guidance is located in Section 10.4). Female participants of childbearing potential must have a negative urine pregnancy test result at trial entry before the first dose of study drug. Additional requirements for pregnancy testing during and after study intervention are located in Section 8.4.5 and Section 8.5.5.
4. Any medical or psychological condition, including relevant laboratory abnormalities that, in the opinion of the Investigator, may present an unreasonable risk to the study participant as a result of his/her participation in this clinical trial, may impede their ability complete the study or the study assessments or confound the outcomes of the trial.
5. Diagnosis of Pulmonary Hypertension WHO Groups 1, 2, 4, or 5, or subtypes of PH WHO Group 3 other than interstitial lung disease (including combined pulmonary fibrosis and emphysema)
6. Evidence of left ventricular failure, HFpEF or postcapillary PH
7. Platelet count <50.0 x 103/μL at Enrollment, and/or unexplained coagulation abnormalities in repeated laboratory tests.
8. Known hypersensitivity or contraindication to treprostinil or TPIP or TPIP formulation excipients (eg, mannitol, leucine).

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. AEs, laboratory assessments, vital signs, physical examination, 12-lead ECG, and supplemental oxygen use

Secondary endpoints 9

1. Change in 6MWD measured post-dose from pre-OLE Baseline up to 104 weeks
2. Absolute and percent change from pre-OLE Baseline in FVC, FVC% pred, FEV1, and FEV1% pred up to 104 weeks
3. Change in NT-proBNP plasma concentration from pre-OLE Baseline up to 104 weeks
4. Annualized rate of ILD exacerbations during the OLE study (104 weeks)
5. Proportion of participants with a clinical worsening event. Clinical worsening events are defined as one of the following: • Hospitalization due to a cardiopulmonary indication related to the disease under study
6. • Deterioration of PH-ILD, defined as a combination of the following changes from Baseline attributable to the disease under study: − decrease in 6MWD ≥15%, confirmed by 2 tests at least 4 hours but not more than 1 week apart, AND − signs/symptoms of worsened right heart failure or worsened WHO/NYHA functional class. • Lung transplantation (except for when preplanned prior to the study) • Death from any cause
7. Mean change in L-PF total symptom domain score from pre-OLE Baseline up to 104 weeks Mean change from pre-OLE Baseline up to 104 weeks in: • L-PF cough domain score • L-PF dyspnea domain score • L-PF impact domain score
8. Mean change from pre-OLE Baseline up to 104 weeks in: • EQ-5D-5L index score • EQ-5D-5L VAS
9. Proportion of participants with a major morbidity or mortality event, defined as one of the following: • Hospitalization due to a cardiopulmonary indication related to the disease under study • Lung transplantation (except for when pre-planned prior to the study) • Death from any cause

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 4

Placebo 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Insmed Inc.

Sponsor organisation

Insmed Inc.

Address

700 Us Highway 202/206

City

Bridgewater

Postcode

08807-1704

Country

United States

Scientific contact point

Organisation

Insmed Inc.

Contact name

Natasha Makulova

Public contact point

Organisation

Insmed Inc.

Contact name

Medical Information

Third parties 20

Locations

12 EU/EEA countries · 66 investigational sites

By country

Investigational sites

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 97 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

1 submissions — initial application plus substantial / non-substantial modifications