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).
Signed informed consent by the parents or the legal representatives and written assent from appropriately aged participants
Males or females from birth to under 18 years of age at the time informed consent was signed
Confirmed diagnosis of severe PAH classified as PH Group 1 requiring a treatment with prostacyclin infusion
Mean pulmonary arterial pressure (mPAP) >20 mmHg
Known intolerance to prostacyclin analogues
PH related to conditions other than specified above
Unrepaired congenital heart disease if surgery is planned within next 5 months
Subjects diagnosed with any lung disease
Single group
preservative-free parenteral treprostinil
DrugContinuous 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.
Frequency and seriousness of adverse events and adverse drug reactions
The frequency and seriousness of adverse events and adverse drug reactions during the first 5 months (20 weeks ± 1 weeks) of treatment according to Common Terminology Criteria for Adverse Events (CTCAE, version 5.0).
Change from baseline in quality of Life (QoL)
Change from baseline in quality of Life (QoL) as assessed by the Pediatric Quality of Life Inventory (PedsQoL Version 4.0) questionnaire. Rating from 0 (never) to 4 (almost always) per question. Questionnaire will be completed by pediatric patient (version appropriate for age group) and parents/caregivers.
Change from baseline in 6-minute walk distance (6MWD)
Change from baseline in 6MWD (patients \> 6 years)
Change from baseline in World Health Organization Functional Class (WHO FC)
Change in WHO FC class assessment based on current PH guidelines ranging from class I to class IV, whereas class IV means severe limitations.
Change from baseline in echocardiography (ECHO) parameters - RA/RV enlargement
right atrium (RA) / right ventricle (RV)
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