RecruitingPhase 3NCT06350032

Investigational Trial to Evaluate Safety and Tolerability of Treprostinil in Children Diagnosed With PAH

Open-label, Single-arm, Non-controlled Trial to Evaluate Safety and Tolerability of Treprostinil Sodium in Children Below the Age of 18 Years Diagnosed With Pulmonary Arterial Hypertension (PAH)

Sponsor

AOP Orphan Pharmaceuticals AG

Enrollment

20 participants

Start Date

Jul 31, 2024

Study Type

INTERVENTIONAL

Conditions

Pulmonary Arterial Hypertension

Summary

The goal of this clinical trial is to evaluate safety and tolerability of preservative-free parenteral treprostinil in paediatric patients with PAH (PH Group 1) who are below 18 years of age. The main question it aims to answer is: • if preservative-free parenteral treprostinil is safe and tolerable in the treatment of paediatric PAH in patients who are either prostacyclin-naïve or have been previously treated with commercially available parenteral treprostinil formulations. Participants will receive either subcutaneous (SC) or intravenous (IV) preservative-free treprostinil and will be observed for 5 months (20 weeks ± 1 week).

Eligibility

Max Age: 18 Years

Plain Language Summary

Simplified for easier understanding

This study tests the safety and tolerability of a medication called treprostinil — delivered as a continuous infusion — in infants, children, and adolescents with pulmonary arterial hypertension (PAH), a serious condition where the blood pressure in the lungs is abnormally high. **You may be eligible if...** - You are a child from newborn to under 18 years of age - You have been diagnosed with severe PAH (Group 1 pulmonary hypertension) confirmed by a right heart catheterization or echocardiogram - Your doctor has determined that you need treatment with a prostacyclin infusion - You have not previously received a prostacyclin medication **You may NOT be eligible if...** - You have already been treated with a prostacyclin therapy - You have PAH caused by a different underlying condition not covered by Group 1 classification - Your parents or legal guardians are unable or unwilling to provide consent Talk to your doctor to see if this trial is right for you.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

DRUGpreservative-free parenteral treprostinil

Continuous infusion of either SC or IV preservative-free treprostinil. The dosing is not stipulated by the study protocol and will be done according to patient needs.