Participants with Pulmonary Arterial Hypertension MedDRA version: 21.1 Level: PT Classification code 10064911 Term: Pulmonary arterial hypertension System Organ Class: 10038738 - Respiratory, thoracic and mediastinal disorders
Conditions
Sponsors
Insmed Incorporated
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Participants who completed the end of treatment visit in Study INS1009-201, Study 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. 2. 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, Study INS1009-202, or any other lead-in PAH TPIP in study. 3. Capable of giving signed informed consent. Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 75 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 24
Exclusion criteria
Exclusion criteria: 1. Participants who experienced any hypersensitivity or adverse drug reaction or were withdrawn early/discontinued in a previous PAH TPIP study, which, in the opinion of the Investigator, could indicate that continued treatment with TPIP may present an unreasonable risk for the participant. 2. 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. 3. Pregnant or breastfeeding. 4. Any medical or psychological condition, including relevant laboratory abnormalities, at screening that, in the opinion of the Investigator, suggest a new and/or insufficiently understood disease that may present an unreasonable risk to the study participant as a result of participation in the study. 5. QTcF interval > 480 ms on resting ECG at screening, not including participants with right bundle branch block (RBBB) leading to a prolongation of the QRS. 6. Any new ventricular or supraventricular tachyarrhythmia except for paroxysmal atrial fibrillation and any new symptomatic bradycardia. 7. 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). 8. New evidence of thromboembolic disease as assessed by VQ scan, pulmonary angiography, or pulmonary CT scan. 9. Deterioration in renal function to estimated glomerular filtration rate 2 × ULN) and/or • Bilirubin > 1.5 × ULN (isolated bilirubin > 1.5 × ULN; ULN is acceptable if bilirubin is fractionated and direct bilirubin < 35%) 11. Active and current symptomatic COVID-19 or previous severe disease and/or hospitalization due to COVID-19 (Section 10.1.3.1.3). 12. History of abnormal bleeding or bruising with a platelet count of < 50,000/µL at screening. 13. Interval organ transplantation. 14. Any clinically significant abnormal laboratory values at screening or diseases or disorders (eg, cardiovascular, pulmonary, gastrointestinal, liver, kidney, neurological, musculoskeletal, endocrine, metabolic, psychiatric, physical impairment) that, in the opinion of the Investigator, may put the participant at risk by participating in the study, or interfere with the participant's treatment, assessment, or influence the results of the study, or have compliance issues with the study or have a planned or anticipated major surgical procedure during the study. 15. 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. 16. 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. 17. Current use of cigarettes (as defined by 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 (Glossary, CDC Tobacco Glossary, 2017). 18. Participants who currently inhale marijuana (re
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To evaluate the safety and tolerability of the long-term use of TPIP in participants with PAH from studies INS1009-201, INS1009-202 and other lead-in studies of TPIP in participants with PAH.;Secondary Objective: -To evaluate the effect of the long-term use of TPIP on exercise capacity in participants with PAH. - To evaluate the effect of the long-term use of TPIP on blood biomarkers of disease severity in participants with PAH. -To evaluate the effect of the long-term use of TPIP on the mortality risk in participants with PAH. -To evaluate the effect of the long-term use of TPIP on the clinical status of participants with PAH. To evaluate the effect of the long-term use of TPIP on the clinical worsening rate in participants with PAH. -To further evaluate the PK profile of the long-term use of TPIP in participants with PAH.;Primary end point(s): Frequency and severity of TEAEs ;Timepoint(s) of evaluation of this end point: Throughout the study. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): -Change in 6MWD (absolute and relative). -Change in the concentration of NT-proBNP in blood. - Change in the REVEAL Lite 2.0 score. -Annualized rate of clinical worsening events (as defined in the protocol) -Plasma concentration levels of TP and TRE.;Timepoint(s) of evaluation of this end point: - From pre-OLE baseline to Month 6, Month 12, Month 18, and Month 24 - From pre-OLE baseline* to Month 6, Month 12, Month 18, and Month 24 - From pre-OLE baseline* to Month 6, Month 12, Month 18, and Month 24 -Throughout the study -Throughout the study | — |
Countries
Argentina, Australia, Austria, Belgium, Brazil, Denmark, Germany, Italy, Japan, Malaysia, Mexico, Philippines, Serbia, Spain, Switzerland, United Kingdom, United States
Contacts
Public ContactRegulatory Affairs Manager EU
Insmed Switzerland GmbH
Outcome results
None listed