Study on Treprostinil Palmitil Inhalation Powder for Patients with Pulmonary Arterial Hypertension

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What is this study about?

This clinical trial is focused on studying a condition known as Pulmonary Arterial Hypertension (PAH). PAH is a type of high blood pressure that affects the arteries in the lungs and the right side of the heart. The study is testing a treatment called Treprostinil Palmitil Inhalation Powder (TPIP), which is a medication taken through inhalation. The purpose of the study is to evaluate how effective and safe TPIP is compared to a placebo in reducing the resistance in the blood vessels of the lungs, which is known as pulmonary vascular resistance.

Participants in the study will be randomly assigned to receive either TPIP or a placebo. The study will last for 16 weeks, during which participants will use an inhaler device to take the medication or placebo. The inhaler is a dry powder inhaler, which means it delivers the medication in the form of a fine powder that is inhaled into the lungs. Throughout the study, participants will have regular check-ups to monitor their health and the effects of the treatment. These check-ups will include tests like the six-minute walk test, which measures how far a person can walk in six minutes, and other assessments to track changes in their condition.

The study aims to provide valuable information about the potential benefits of TPIP for people with PAH. By comparing the effects of TPIP with a placebo, researchers hope to understand how well the treatment works and its safety profile. This information could help in developing better treatment options for those living with PAH. Participants will be closely monitored to ensure their safety and well-being throughout the study period.

1 initial assessment

Upon joining the study, an initial assessment is conducted to confirm eligibility. This includes reviewing medical history, physical examination, and laboratory tests.

A right heart catheterization is performed to measure specific heart pressures and confirm the diagnosis of pulmonary arterial hypertension.

2 baseline measurements

Baseline measurements are taken, including a six-minute walk test (6MWT) to assess exercise capacity.

A blood test is conducted to measure NT-proBNP, a marker related to heart function.

3 randomization and treatment initiation

Participants are randomly assigned to receive either the treprostinil palmitil inhalation powder or a placebo.

The medication is administered via inhalation, with dosage strengths of 80 µg, 160 µg, or 320 µg, depending on the group assignment.

4 treatment period

The treatment period lasts for 16 weeks. During this time, participants continue to take the inhalation powder as directed.

Regular follow-up visits are scheduled to monitor health status, including vital signs, ECG, and laboratory tests.

5 interim assessments

At weeks 5 and 10, additional assessments are conducted, including the six-minute walk test and blood tests to measure NT-proBNP levels.

These assessments help track changes in exercise capacity and heart function over time.

6 final assessment

At the end of the 16-week treatment period, a final assessment is conducted.

This includes a repeat of the six-minute walk test, blood tests, and a review of any side effects experienced during the study.

Who Can Join the Study?

Participants must be between 18 and 75 years old at the time of signing the informed consent form.
Participants need to complete at least two walking tests during the screening phase, with distances between 150 and 450 meters, and both results should be within 15% of each other.
Participants must undergo a right heart catheterization during screening or within 30 days prior, showing specific heart pressure measurements:
- Mean Pulmonary Artery Pressure (PAP) of at least 25 mmHg at rest.
- Pulmonary Capillary Wedge Pressure (PCWP) of 15 mmHg or less.
- Pulmonary Vascular Resistance (PVR) of at least 5 Wood Units (WU).
Body Mass Index (BMI) should be between 18.0 and 37.0 kg/m².
Both male and female participants must use contraceptives according to local regulations:
- Male participants who are not sterile and have female partners capable of becoming pregnant must use effective contraception from the start of the study to at least 90 days after the last dose of the study drug.
- Female participants must be postmenopausal, surgically sterile, or using highly effective contraception methods from the start of the study to at least 90 days after the last dose of the study drug.
Female participants capable of becoming pregnant must have a negative serum pregnancy test at screening and a negative urine pregnancy test at baseline.
Male participants with partners capable of becoming pregnant must use a condom to prevent potential exposure to the embryo or fetus.
Participants must be able to give signed informed consent.
Participants must be able to understand and comply with the study requirements.
Participants must have a diagnosis of WHO Group 1 Pulmonary Arterial Hypertension (PAH) in one of the following subtypes:
- Idiopathic (unknown cause).
- Heritable (genetic).
- Drug or toxin-induced.
- Connective tissue disease-associated PAH.
- Congenital heart disease-related with a simple systemic-to-pulmonary shunt at least 1 year after repair.
Participants must have had a PAH diagnosis for at least 3 months.
Participants must be classified as New York Heart Association (NYHA) or WHO functional class II or III, which indicates the severity of their symptoms.
Medical history, physical examination, vital signs, ECG, and lab results should match the degree of PAH and treatment.
Participants must be on stable PAH therapy with up to two medications from the following classes:
- Endothelin receptor antagonists (e.g., ambrisentan, bosentan, macitentan).
- Phosphodiesterase type 5 inhibitors (e.g., sildenafil, tadalafil).
- Guanylate cyclase stimulator (e.g., riociguat).
No changes in PAH medications or dosage for at least 30 days before screening.
No changes in long-term diuretic use dosage for at least 30 days before screening (single or rescue doses allowed, as per the investigator).
Participants must have documented lung function tests showing:
- Pre-bronchodilator predicted Forced Expiratory Volume in 1 second (FEV1) of at least 70%.
- FEV1/Forced Vital Capacity (FVC) ratio of at least 70% within 1 year of screening. If not available, pulmonary function testing will be done during screening.

Who Cannot Join the Study?

Patients who have a different condition than Pulmonary Arterial Hypertension cannot participate.
Patients who are not within the specified age range cannot participate.
Patients who are not part of the specified clinical trial group cannot participate.
Patients who are considered part of a vulnerable population cannot participate.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name	City	Country
Medical University Of Vienna	Vienna	Austria
Technische Universitaet Dresden	Dresden	Germany
Katholieke Universiteit te Leuven	Leuven	Belgium
Université Libre de Bruxelles – Hôpital Erasme	Brussels	Belgium

Other Sites

Site Name	City	Country
Centre hospitalier universitaire de Liege	Liege	Belgium
Fondazione IRCCS San Gerardo Dei Tintori	Monza	Italy
El Hospital Universitario De Gran Canaria Dr. Negrin	Las Palmas De Gran Canaria	Spain
BG Klinikum Unfallkrankenhaus Berlin gGmbH	Berlin	Germany
Universitaetsklinikum Schleswig-Holstein AöR	Kiel	Germany
Fondazione IRCCS Policlinico San Matteo	Pavia	Italy
Thoraxklinik Heidelberg gGmbH	Heidelberg	Germany
Istituto Mediterraneo Per I Trapianti E Terapie Ad Alta Specializzazione S.r.l.	Palermo	Italy
Servei De Salut De Les Illes Balears	Palma	Spain
Krankenhaus Neuwittelsbach	Munich	Germany
Oytcroalcpcuol Lhvk Gjsv	Linz	Austria
Huxmmrze Uwetmlzghnmgw Mihzmoe Dz Vtlbbylwft	Santander	Spain
Agvpdt Ufuyauxhfx Hcefrjmj	Aarhus	Denmark
Uspiagutxn Djnlf Svgqb Dc Rahl Lo Smiabfxc	Rome	Italy
Fcserurhu Pmdk Lw Irdoqpffjlozc Bbuabgidk Dko Hecmonfi Udwtwwrlrycvc Ly Pgq	Madrid	Spain
Hxlqlxsi Vxrk divggbko	Barcelona	Spain

Trial status

Country	Status	Recruitment Start
Austria	Not recruiting	22.12.2021
Belgium	Not recruiting	22.12.2021
Denmark	Not recruiting	22.12.2021
Germany	Not recruiting	22.12.2021
Italy	Not recruiting	22.12.2021
Spain	Not recruiting	22.12.2021

Trial locations

Investigated drugs:

Treprostinil Palmitil

Treprostinil Palmitil Inhalation Powder (TPIP) is a medication being studied for its effects on pulmonary arterial hypertension. It is designed to help reduce the resistance in the blood vessels of the lungs, which can improve blood flow and reduce the workload on the heart. This medication is inhaled, allowing it to directly target the lungs where it is needed. The study aims to evaluate how effective and safe this treatment is for patients with this condition.

Investigated diseases:

Pulmonary arterial hypertension

Pulmonary Arterial Hypertension – This is a condition characterized by high blood pressure in the arteries that supply the lungs. It occurs when the small arteries in the lungs become narrowed or blocked, making it harder for blood to flow through them. As a result, the heart has to work harder to pump blood through these arteries, which can lead to the heart becoming weak or enlarged over time. Symptoms often include shortness of breath, fatigue, dizziness, and swelling in the ankles or legs. The disease can progress gradually, with symptoms worsening as the pressure in the pulmonary arteries increases. It is considered a rare disease and can significantly impact daily activities and quality of life.

Trial ID:

2023-505541-99-00

Protocol code:

INS1009-202

NCT ID:

NCT05147805

Trial Phase:

Therapeutic exploratory (Phase II)

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Study on Treprostinil Palmitil Inhalation Powder for Patients with Pulmonary Arterial Hypertension

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