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

Recruiting now · Phase 3

Conditions studied: Pulmonary Arterial Hypertension

In brief

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).

Key facts

Study ID: NCT06350032
Run by: AOP Orphan Pharmaceuticals AG
People needed: 20
Starts: 2024-07-31
Expected to finish: 2028-11-01
Last updated by the study team: 2025-05-31

Who can join

Age: any, up to 18. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

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
Current diagnosis of PAH confirmed by right heart catheterisation (RHC) at screening or by historical RHC prior to screening with following haemodynamic findings:
Mean pulmonary arterial pressure (mPAP) >20 mmHg
Pulmonary vascular resistance Index (PVRI) >3 Wood Units (WU) m² If RHC is not possible due to medical reasons (e.g. neonates and infants), the confirmation by ECHO at the screening is sufficient.
Prostacyclin naïve or patients pre-treated with SC or IV treprostinil prior to screening
A subject is eligible to participate in this study, as assessed by the investigator, if they are of:
Non-childbearing potential (i.e., physiologically incapable of becoming pregnant); or,
Child-bearing potential - has a negative pregnancy test and is not lactating and, if sexually active, agrees to continue to use 2 reliable methods of contraception until study completion and for at least 30 days following the last dose of study drug. Examples of reliable birth control methods include true abstinence as a lifestyle choice (periodic sexual abstinence method is not acceptable); the use of oral contraceptives; a reliable barrier method of birth control (diaphragms with contraceptive jelly; cervical caps with contraceptive jelly; condoms with contraceptive foam; intrauterine devices)

You may not qualify if…

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
Acutely decompensated heart failure within previous 30 days from screening
Subjects who have had an atrial septostomy or potts shunt within the previous 6 months of screening
Any clinically significant laboratory abnormality that precludes initiation or continuation of treprostinil therapy
Moderate to severe hepatic dysfunction as defined by elevated liver function tests (aspartate aminotransferase or alanine aminotransferase) ≥3 times the upper limit of normal at Screening, or Child Pugh class B or C hepatic disease
Subjects who are pregnant or breastfeeding
Haematological abnormalities (e.g., severe anaemia, Hgb <10 g/dL, leukopenia, White Blood Cells (WBC) <2500/μL)
History of substance use disorder, unless a proof of abstinence ≥1 year is provided
Other concurrent severe acute or chronic medical or psychiatric condition or laboratory abnormality that may increase the risk associated with study participation or investigational product administration or may interfere with the interpretation of study results and, in the judgment of the investigator, would make the subject inappropriate for entry into this study
Participation in another clinical trial of an investigational drug or device (including with placebo) within 30 days or 5 half-lives prior to screening, which-ever is longer
Patients not able to handle pumps and infusion site if there is no parent, family member, guardian present in their household taking over pump handling or if they are not treated in hospital set-up

Where it is running

Medizinische Universität Wien — Vienna, State of Vienna, Austria (enrolling)
Necker-Enfants Malades Hospital, Paris — Paris, Paris, France (enrolling)
Gottsegen National Cardiovascular Center — Budapest, Budapest, Hungary (enrolling)
Pediatric Cardiac Center — Bratislava, Slovakia (enrolling)
Ramón y Cajal University Hospital — Madrid, Madrid, Spain (enrolling)

Full record on ClinicalTrials.gov

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