Actively Recruiting
Investigational Trial to Evaluate Safety and Tolerability of Treprostinil in Children Diagnosed With PAH
Led by AOP Orphan Pharmaceuticals AG · Updated on 2025-05-31
20
Participants Needed
5
Research Sites
222 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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).
CONDITIONS
Official Title
Investigational Trial to Evaluate Safety and Tolerability of Treprostinil in Children Diagnosed With PAH
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Signed informed consent by parents or legal representatives and assent from appropriately aged participants
- Males or females from birth to under 18 years of age at consent
- Confirmed diagnosis of severe PAH classified as PH Group 1 requiring prostacyclin infusion treatment
- PAH diagnosis confirmed by right heart catheterisation at screening or historical catheterisation showing mPAP >20 mmHg and PVRI >3 Wood Units m²; ECHO confirmation allowed if catheterisation not possible
- Prostacyclin naive or previously treated with subcutaneous or intravenous treprostinil
- Participant is either non-childbearing potential or, if childbearing potential, has negative pregnancy test, is not lactating, and if sexually active agrees to use two reliable contraception methods until study completion and 30 days after last dose
You will not qualify if you...
- Known intolerance to prostacyclin analogues
- Pulmonary hypertension related to other conditions than specified
- Unrepaired congenital heart disease if surgery is planned within next 5 months
- Diagnosis of any lung disease
- Acutely decompensated heart failure within 30 days prior to screening
- Atrial septostomy or Potts shunt within 6 months prior to screening
- Clinically significant lab abnormalities preventing treprostinil therapy
- Moderate to severe hepatic dysfunction (liver function tests ≥3 times upper limit normal or Child Pugh class B or C)
- Pregnant or breastfeeding
- Hematological abnormalities (severe anemia, hemoglobin <10 g/dL, leukopenia, WBC <2500/µL)
- History of substance use disorder unless abstinent for at least 1 year
- Other severe acute or chronic medical or psychiatric conditions increasing risk or interfering with study
- Participation in another investigational drug or device trial within 30 days or 5 half-lives prior to screening
- Inability to handle pumps and infusion site without parent, family member, or guardian support at home or hospital setting
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 5 locations
1
Medizinische Universität Wien
Vienna, State of Vienna, Austria, 1090
Actively Recruiting
2
Necker-Enfants Malades Hospital, Paris
Paris, Paris, France, 75015
Actively Recruiting
3
Gottsegen National Cardiovascular Center
Budapest, Budapest, Hungary, H-1096
Actively Recruiting
4
Pediatric Cardiac Center
Bratislava, Slovakia
Actively Recruiting
5
Ramón y Cajal University Hospital
Madrid, Madrid, Spain, 28034
Actively Recruiting
Research Team
C
Clinical Project Manager
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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