NCT06350032

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)

• Status: Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 20 (estimated)
• Sponsor: AOP Orphan Pharmaceuticals AG · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

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).

Detailed description

In this study, a preservative-free treprostinil solution provided in a single-use vial will be used. Efficacy of treprostinil for the treatment of PAH in pulmonary hypertension (PH) Group 1 in children is reported throughout literature. However, since the removal of the preservative might impact the safe use of the parenteral solution for infusion, the safety profile of the newly developed preservative-free treprostinil solution will be assessed within this trial.

Conditions

• Pulmonary Arterial Hypertension

Interventions

Type	Name	Description
DRUG	preservative-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.

Timeline

• Start date: 2024-07-31
• Primary completion: 2028-11-01
• Completion: 2028-11-01
• First posted: 2024-04-05
• Last updated: 2025-05-31

Locations

5 sites across 5 countries: Austria, France, Hungary, Slovakia, Spain