Evaluating safety of preservative-free treprostinil in children with pulmonary arterial hypertension

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)

This study is testing whether a new preservative-free version of treprostinil is safe for children with pulmonary arterial hypertension.

Quick facts

• Phase: Phase 3
• Study type: Interventional
• Enrollment: 20 (estimated)
• Ages: N/A to 18 Years
• Sex: All
• Sponsor: AOP Orphan Pharmaceuticals AG (Industry-sponsored)
• Locations: 5 sites (Vienna, State of Vienna and 4 other locations)
• Trial ID: NCT06350032 on ClinicalTrials.gov

What this trial studies

This clinical trial aims to assess the safety and tolerability of preservative-free parenteral treprostinil in pediatric patients diagnosed with pulmonary arterial hypertension (PAH). Participants, aged from birth to under 18 years, will receive either subcutaneous or intravenous treprostinil and will be monitored for a duration of 20 weeks. The study focuses on both treatment-naïve patients and those previously treated with other formulations of treprostinil. The goal is to determine if the preservative-free formulation is safe for use in this vulnerable population.

Who should consider this trial

Good fit: Ideal candidates include children under 18 years with a confirmed diagnosis of severe PAH requiring prostacyclin infusion.

Not a fit: Patients with conditions that prevent confirmation of PAH via right heart catheterization or echocardiogram may not benefit from this study.

Why it matters

Potential benefit: If successful, this study could provide a safer treatment option for children suffering from pulmonary arterial hypertension.

How similar studies have performed: Previous studies have indicated the efficacy of treprostinil in treating PAH, but this specific preservative-free formulation is being evaluated for the first time in children.

Eligibility criteria

Inclusion Criteria:

1. Signed informed consent by the parents or the legal representatives and written assent from appropriately aged participants
2. Males or females from birth to under 18 years of age at the time informed consent was signed
3. Confirmed diagnosis of severe PAH classified as PH Group 1 requiring a treatment with prostacyclin infusion
4. 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.
5. Prostacyclin naïve or patients pre-treated with SC or IV treprostinil prior to screening
6. 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)

Exclusion Criteria:

1. Known intolerance to prostacyclin analogues
2. PH related to conditions other than specified above
3. Unrepaired congenital heart disease if surgery is planned within next 5 months
4. Subjects diagnosed with any lung disease
5. Acutely decompensated heart failure within previous 30 days from screening
6. Subjects who have had an atrial septostomy or potts shunt within the previous 6 months of screening
7. Any clinically significant laboratory abnormality that precludes initiation or continuation of treprostinil therapy
8. 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
9. Subjects who are pregnant or breastfeeding
10. Haematological abnormalities (e.g., severe anaemia, Hgb \<10 g/dL, leukopenia, White Blood Cells (WBC) \<2500/μL)
11. History of substance use disorder, unless a proof of abstinence ≥1 year is provided
12. 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
13. 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
14. 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 this trial is running

Vienna, State of Vienna and 4 other locations

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

Study contacts

• Study coordinator: Clinical Project Manager

Conditions Pulmonary Arterial Hypertension