An open-label extension study to evaluate the safety, tolerability, and efficacy of long-term use of inhalation powder of treprostinil palmitil in participants with pulmonary arterial hypertension
Zusammenfassung der Studie
This is an open-label extension study, Phase 2/3, with multiple centers. Its objective is to evaluate treprostinil palmitil inhalation powder (TPIP), an experimental drug currently under investigation for the treatment of pulmonary arterial hypertension (PAH), to determine if TPIP is safe for long-term use in individuals with PAH. Additionally, to ascertain if TPIP is well tolerated (has few side effects), to learn more about TPIP and its mechanism of action in the human body and the substances it produces (“biomarkers”), to measure the amount of TPIP in the body (pharmacokinetics), and to gain further insight into the body's response to the study drug. The study will begin with an initial dose adjustment period of 3 weeks (from Day 1 to Day 21), after which each participant will receive a single dose of TPIP daily for the duration of the study. All individuals participating in the study will undergo the same study assessments. The study will last approximately 24 months from the reference visit (Day 1), end of the dose adjustment period (Week 3), until the end of the Follow-up visit/phone call (Visit 13 or 4 weeks after the administration of the last dose of TPIP at Month 24). At least 13 visits will take place at the study center, including, if applicable, a screening visit (Visit 1 occurring up to 30 days before the administration of the first dose of the study drug on Day 1), 11 visits during the treatment period of the study (Visits 2 to 12/Day 1 to Month 24), and 1 Follow-up visit/phone call (Visit 13/4 weeks after the administration of the last dose of TPIP at Month 24). The follow-up visit may occur either at the study center or by phone at the physician's discretion.
(BASEC)
Untersuchte Intervention
Treprostinil palmitil is an inactive prodrug of treprostinil, which is widely used in the treatment of pulmonary hypertension. Treprostinil palmitil inhalation powder (TPIP) is a dry powder formulation for inhalation currently being developed for the treatment of pulmonary arterial hypertension. TPIP is expected to provide prolonged release of treprostinil in the lungs with the aim of less frequent and more convenient dosing while reducing the frequency and severity of side effects.
(BASEC)
Untersuchte Krankheit(en)
Pulmonary Arterial Hypertension (PAH)
(BASEC)
1. Participants who have completed the end-of-treatment visit in the INS1009-202 study or any other preliminary study on TPIP in PAH. Participants for whom the open-label extension study (OLE) was not available at the time of the completion of the initial study are eligible for recruitment in the year following their end-of-treatment visit of the initial study. 2. Completion of the screening assessments of the reference visit to confirm eligibility to participate if more than 30 days have elapsed since the end of the study visit in the INS1009-201 study, the INS1009-202 study, or any other preliminary study on TPIP in PAH. 3. Ability to provide signed informed consent. (BASEC)
Ausschlusskriterien
1. Participants who have experienced any hypersensitivity or adverse reaction to the drug, or who have had an early withdrawal/termination in a previous study on TPIP in PAH that may indicate that continuing treatment with TPIP may pose an unreasonable risk to the participant. 2. Initiation of parenteral administration of prostacyclin analogs since the end of the INS1009-201, INS1009-202 studies or other TPIP studies. Initiation of inhaled and oral prostacyclin analogs or receptor agonists is allowed if stopped 24 hours prior to the start of administration of the study drug. 3. Pregnancy or breastfeeding. 4. Any medical or psychological condition including any relevant laboratory abnormalities at screening, which, in the investigator's opinion, suggests a new and/or poorly understood disease that may pose an unreasonable risk to the patient as a result of their participation in the study. (BASEC)
Studienstandort
Lausanne
(BASEC)
Sponsor
Insmed Incorporated
(BASEC)
Kontakt für weitere Auskünfte zur Studie
Name der bewilligenden Ethikkommission (bei multizentrischen Studien nur die Leitkommission)
Ethikkommission Waadt
(BASEC)
Datum der Bewilligung durch die Ethikkommission
27.01.2023
(BASEC)
ICTRP Studien-ID
NCT05649748 (ICTRP)
Offizieller Titel (Genehmigt von der Ethikkommission)
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 (BASEC)
Wissenschaftlicher Titel
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 (ICTRP)
Öffentlicher Titel
An Extension Study of Treprostinil Palmitil Inhalation Powder (TPIP) for Pulmonary Arterial Hypertension (PAH) (ICTRP)
Untersuchte Krankheit(en)
Pulmonary Arterial Hypertension
(ICTRP)
Untersuchte Intervention
Drug: Treprostinil Palmitil
Drug: Placebo
(ICTRP)
Studientyp
Interventional (ICTRP)
Studiendesign
Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label). (ICTRP)
Ein-/Ausschlusskriterien
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.
- Any new ventricular or supraventricular tachyarrhythmia except for paroxysmal atrial
fibrillation and any new symptomatic bradycardia.
- 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).
- New evidence of thromboembolic disease as assessed by ventilation/perfusion (VQ) scan,
pulmonary angiography, or pulmonary computed tomography (CT) scan.
- Active and current symptomatic coronavirus disease 2019 (COVID-19) or previous severe
disease and/or hospitalization due to COVID-19.
- Interval organ transplantation.
- New active liver disease or hepatic dysfunction.
- 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.
- 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.
- Current use of cigarettes (as defined by Centers for Disease Control and Prevention
[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.
- Participants who currently inhale marijuana (recreational or medical).
Note: Other inclusion/exclusion criteria may apply.
(ICTRP)
nicht verfügbar
Primäre und sekundäre Endpunkte
Number of Participants Who Experience at Least one Treatment Emergent Adverse Event (TEAE) and TEAEs by Severity
(ICTRP)
Absolute Change From Pre-Open Label Extension (OLE) Baseline in 6-Minute Walk Distance (6MWD)
Change From Pre-OLE Baseline in New York Heart Association/ World Health Organization (NYHA/WHO) Functional Capacity Class
Plasma Concentration Levels of Treprostinil Palmitil (TP) and Treprostinil (TRE)
Annualized Clinical Worsening Event Rate
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
Relative Change From Pre-OLE Baseline in 6MWD
(ICTRP)
Registrierungsdatum
06.12.2022 (ICTRP)
Einschluss des ersten Teilnehmers
07.03.2023 (ICTRP)
Sekundäre Sponsoren
nicht verfügbar
Weitere Kontakte
Insmed Medical Information, medicalinformation@insmed.com, 1-844-446-7633 (ICTRP)
Sekundäre IDs
2022-001951-18, 2023-505539-11-0, INS1009-203 (ICTRP)
Angaben zur Verfügbarkeit von individuellen Teilnehmerdaten
nicht verfügbar
Weitere Informationen zur Studie
https://trialsearch.who.int/Trial2.aspx?TrialID=NCT05649748 (ICTRP)
Ergebnisse der Studie
Zusammenfassung der Ergebnisse
nicht verfügbar
Link zu den Ergebnissen im Primärregister
nicht verfügbar