protocolSection identificationModule nctId: NCT06350032, orgStudyIdInfo id: TREPaed, secondaryIdInfos id: 2023-505082-91-00, type: CTIS, briefTitle: Investigational Trial to Evaluate Safety and Tolerability of Treprostinil in Children Diagnosed With PAH, statusModule overallStatus: NOT_YET_RECRUITING, startDateStruct date: 2024-05, primaryCompletionDateStruct date: 2028-11, completionDateStruct date: 2028-11, studyFirstPostDateStruct date: 2024-04-05, lastUpdatePostDateStruct date: 2024-04-05, sponsorCollaboratorsModule leadSponsor name: AOP Orphan Pharmaceuticals AG, class: INDUSTRY, descriptionModule briefSummary: 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 treatment-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)., conditionsModule conditions: Pulmonary Arterial Hypertension, designModule studyType: INTERVENTIONAL, phases: PHASE3, designInfo allocation: NA, interventionModel: SINGLE_GROUP, primaryPurpose: TREATMENT, maskingInfo masking: NONE, enrollmentInfo count: 20, type: ESTIMATED, armsInterventionsModule interventions name: preservative-free parenteral treprostinil, outcomesModule primaryOutcomes measure: Frequency and seriousness of adverse events and adverse drug reactions, otherOutcomes measure: Change from baseline in quality of Life (QoL), otherOutcomes measure: Change from baseline in 6-minute walk distance (6MWD), otherOutcomes measure: Change from baseline in World Health Organization Functional Class (WHO FC), otherOutcomes measure: Change from baseline in echocardiography (ECHO) parameters - RA/RV enlargement, otherOutcomes measure: Change from baseline in echocardiography (ECHO) parameters - RV systolic dysfunction, otherOutcomes measure: Change from baseline in echocardiography (ECHO) parameters - RV/LV end-systolic ratio (PSAX), otherOutcomes measure: Change from baseline in echocardiography (ECHO) parameters - tricuspid annular plane systolic excursion (TAPSE), otherOutcomes measure: Change from baseline in echocardiography (ECHO) parameters - S/D ratio (TR jet), otherOutcomes measure: Change from baseline in echocardiography (ECHO) parameters - Pulmonary Artery Acceleration Time (PAAT), otherOutcomes measure: Change from baseline in echocardiography (ECHO) parameters - pericardial effusion, otherOutcomes measure: Change from baseline in echocardiography (ECHO) parameters - eccentricity index, otherOutcomes measure: Change from baseline in echocardiography (ECHO) parameters - acceleration time, otherOutcomes measure: Change from baseline in plasma N-terminal pro-brain natriuretic peptide (NT-proBNP) levels, otherOutcomes measure: Treprostinil plasma concentration, eligibilityModule sex: ALL, maximumAge: 18 Years, stdAges: CHILD, stdAges: ADULT, contactsLocationsModule locations facility: Medizinische Universität Wien, city: Vienna, zip: 1090, country: Austria, contacts name: Sulaimi Albinni, MD, role: PRINCIPAL_INVESTIGATOR, geoPoint lat: 48.20849, lon: 16.37208, locations facility: Necker-Enfants Malades Hospital, Paris, city: Paris, zip: 75015, country: France, contacts name: Damien Bonnet, Prof, role: PRINCIPAL_INVESTIGATOR, geoPoint lat: 48.85341, lon: 2.3488, locations facility: Gottsegen National Cardiovascular Center, city: Budapest, zip: H-1096, country: Hungary, contacts name: Laszlo Ablonczy, MD, role: PRINCIPAL_INVESTIGATOR, geoPoint lat: 47.49801, lon: 19.03991, locations facility: Ramón y Cajal University Hospital, city: Madrid, zip: 28034, country: Spain, contacts name: Maria J del Cerro, Prof, role: PRINCIPAL_INVESTIGATOR, geoPoint lat: 40.4165, lon: -3.70256, hasResults: False