Study on the Safety and Tolerability of Treprostinil Sodium for Children with Pulmonary Arterial Hypertension

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What is this study about?

This clinical trial is focused on studying Pulmonary Arterial Hypertension (PAH), a condition where the blood pressure in the arteries of the lungs is higher than normal. The trial will use a treatment called treprostinil sodium, which is administered as a solution for infusion. The study will include different formulations of this treatment, known by their code names: TREPaed_0.5, TREPaed_1, TREPaed_2.5, TREPaed_5, and TREPaed_10. These formulations are designed for use in children under 18 years of age.

The purpose of the study is to evaluate the safety and tolerability of treprostinil sodium in children with Pulmonary Arterial Hypertension. Participants in the study will receive the treatment either through subcutaneous (under the skin) or intravenous (into a vein) methods. The study will monitor the frequency and seriousness of any adverse events or reactions to the drug over a period of time. Additionally, changes in the quality of life and other health parameters will be assessed to understand the impact of the treatment.

The study is open-label, meaning that both the researchers and participants will know which treatment is being administered. It is a single-arm trial, which means all participants will receive the same treatment without a comparison group. The trial is expected to run for several years, allowing researchers to gather comprehensive data on the effects of treprostinil sodium in treating Pulmonary Arterial Hypertension in children.

1 joining the trial

Upon joining the trial, informed consent must be signed by the parents or legal representatives. Participants who are old enough will also provide written assent.

Eligibility is confirmed for children from birth to under 18 years with a diagnosis of severe pulmonary arterial hypertension (PAH).

2 initial assessment

A diagnosis of PAH is confirmed through right heart catheterization or echocardiography if catheterization is not possible.

Participants are assessed for their ability to participate, including a pregnancy test for those of child-bearing potential.

3 treatment initiation

The treatment involves the administration of treprostinil sodium as a solution for infusion.

The medication is administered either subcutaneously or intravenously, depending on the participant’s condition and previous treatments.

4 treatment duration

The treatment continues for a period of 5 months (20 weeks ± 1 week).

During this time, the frequency and seriousness of any adverse events or drug reactions are monitored.

5 ongoing assessments

Throughout the trial, changes from baseline in quality of life, physical capacity, and heart function are evaluated.

These assessments include questionnaires, physical tests, and blood tests to measure various health indicators.

6 end of treatment

At the end of the treatment period, a final assessment is conducted to evaluate the overall impact of the medication.

Participants are monitored for any lasting effects or changes in their condition.

Who Can Join the Study?

Signed informed consent by the parents or the legal representatives and written agreement from participants who are old enough to understand.
Males or females from birth to under 18 years of age at the time informed consent was signed.
Confirmed diagnosis of severe Pulmonary Arterial Hypertension (PAH), which is a type of high blood pressure affecting the lungs and heart, requiring treatment with a medication called prostacyclin infusion.
Current diagnosis of PAH confirmed by a test called right heart catheterisation (RHC), which checks the pressure in the heart and lungs, or by an ultrasound of the heart (ECHO) if RHC is not possible. The test should show:
- Mean pulmonary arterial pressure (mPAP) greater than 20 mmHg.
- Pulmonary vascular resistance Index (PVRI) greater than 3 Wood Units (WU) m².
Patients who have never been treated with prostacyclin or those who have been treated with treprostinil through the skin (SC) or into a vein (IV) before the study.
Participants must be assessed by the investigator to be:
- Not able to become pregnant (non-childbearing potential); or,
- Able to become pregnant (child-bearing potential) but has a negative pregnancy test, is not breastfeeding, and if sexually active, agrees to use two reliable methods of birth control until the study ends and for at least 30 days after the last dose of the study drug. Reliable birth control methods include complete abstinence, oral contraceptives, or barrier methods like condoms with contraceptive foam.

Who Cannot Join the Study?

Patients who are not diagnosed with Pulmonary Arterial Hypertension (PAH). PAH is a condition where there is high blood pressure in the arteries of the lungs.
Patients who are 18 years of age or older.
Patients who have not been treated with or are not new to treatment with treprostinil, a medication used for PAH.
Patients who are not part of the vulnerable population, which means those who might need special care or protection.

Where you can join this trial?

Verified and Recommended Sites

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Verified Sites

Site Name	City	Country	Status
Medical University Of Vienna	Vienna	Austria

Other Sites

Site Name	City	Country
Gottsegen National Cardiovascular Center	Budapest	Hungary
Národny ustav srdcovych a cievnych chorob a.s.	Bratislava	Slovakia
Hopital Beaujon	Clichy	France
Kewusaid dal Ufaoelharleh Muotkhsg Aqs	Munich	Germany
Heulooad Vjaj dplcjyuv	Barcelona	Spain

Trial status

Country	Status	Recruitment Start
Austria	Recruiting	01.01.2024
France	Recruiting	01.01.2024
Germany	Recruiting	01.01.2024
Hungary	Recruiting	01.01.2024
Slovakia	Recruiting	01.01.2024
Spain	Recruiting	01.01.2024

Trial locations

Investigated drugs:

Treprostinil Sodium

Treprostinil is a medication used in this trial to treat children with pulmonary arterial hypertension (PAH). It helps to relax and widen the blood vessels in the lungs, which can lower blood pressure and improve blood flow. This trial aims to evaluate the safety and tolerability of a preservative-free form of treprostinil in children under 18 years old who have PAH.

Pulmonary Arterial Hypertension (PAH) – 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 heart strain and enlargement. Over time, this increased pressure can cause symptoms such as 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 continues to rise. It is a chronic condition that requires ongoing management to help control symptoms and improve quality of life.

Trial ID:

2023-505082-91-00

Protocol code:

TREPaed

Trial Phase:

Therapeutic confirmatory (Phase III)

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Study on the Safety and Tolerability of Treprostinil Sodium for Children with Pulmonary Arterial Hypertension

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