Pulmonary Arterial HypertensionPhase 3NCT06350032
Investigational Trial to Evaluate Safety and Tolerability of Treprostinil in Children Diagnosed With PAH
A Phase 3 study testing preservative-free parenteral treprostinil in people with Pulmonary Arterial Hypertension, run by AOP Orphan Pharmaceuticals AG at 5 sites in 5 countries.
Trial summary
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. Read more about this study
- Who is it for?
- Ages Up to 18 years
- Conditions the study is for
- Pulmonary Arterial Hypertension
- What is being tested?
- preservative-free parenteral treprostinil
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- 5 sitesin 5 countries
- Study dates
- 31 Jul 2024to Nov 2028
- Phase
- Phase 3
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Up to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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.
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- 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)
Who cannot join
- 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
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- 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
Trial locations and contacts
5 sites in 5 countries. Contact the study team or a site near you to ask about taking part.
5 sites in 5 countries
Austria1 site, 1 recruiting
- Recruiting
Medizinische Universität Wien
Vienna, State of Vienna, 1090
Sulaimi Albinni, MD
France1 site, 1 recruiting
- Recruiting
Necker-Enfants Malades Hospital, Paris
Paris, Paris, 75015
Damien Bonnet, Prof
Hungary1 site, 1 recruiting
- Recruiting
Gottsegen National Cardiovascular Center
Budapest, Budapest, H-1096
Laszlo Ablonczy, MD
Slovakia1 site, 1 recruiting
- Recruiting
Pediatric Cardiac Center
Bratislava
Martin Zahorec, MD, PhD, +421 02/59320 152, martin.zahorec@nusch.sk
Spain1 site, 1 recruiting
- Recruiting
Ramón y Cajal University Hospital
Madrid, Madrid, 28034
Maria J del Cerro, Prof
About this study
Official title: 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)
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).
Read the detailed description
In this study, a preservative-free treprostinil solution provided in a single-use vial will be used. Efficacy of treprostinil for the treatment of PAH in pulmonary hypertension (PH) Group 1 in children is reported throughout literature. However, since the removal of the preservative might impact the safe use of the parenteral solution for infusion, the safety profile of the newly developed preservative-free treprostinil solution will be assessed within this trial.
- Conditions
- Pulmonary Arterial Hypertension
- Being tested
- preservative-free parenteral treprostinil
- Medical subject headings
- Pulmonary Arterial Hypertension
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
preservative-free parenteral treprostinilDrug
Continuous 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.
| Study group | Role | What they receive |
|---|---|---|
| preservative-free parenteral treprostinil | Experimental | Patients will be treated with either SC or IV preservative-free treprostinil formulation. Dosing is not stipulated by the study protocol and will be done according to investigators discretion based on patient needs. Receives: Drug: preservative-free parenteral treprostinil |
What is being measured
The primary outcome is the main question the study is designed to answer.
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).
Measured: 5 months (20 weeks ± 1 weeks)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Apr 2024
- Study start31 Jul 2024
- Primary completionNov 2028 (estimated)
- Study completionNov 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Pulmonary Arterial HypertensionStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 20 participants (estimated)
Sponsor and investigators
- Lead sponsor
- AOP Orphan Pharmaceuticals AG
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06350032
- Sponsor's protocol ID
- TREPaed
- Other IDs
- 2023-505082-91-00, 2023
- Status last verified
- Oct 2026
- First submitted
- 19 Mar 2024
- Last updated
- 29 Oct 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 29 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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)
- ClinicalTrials.gov ID
- NCT06350032
- Acronym / sponsor ID
- TREPaed
- Conditions
- Pulmonary Arterial Hypertension
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 20 participants (estimated)
- Lead sponsor
- AOP Orphan Pharmaceuticals AGIndustry (pharma, biotech, devices)
- Study start
- 31 Jul 2024 (actual)
- Primary completion
- Nov 2028 (estimated)
- Study completion
- Nov 2028 (estimated)
- First posted
- 5 Apr 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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).
Detailed description
In this study, a preservative-free treprostinil solution provided in a single-use vial will be used. Efficacy of treprostinil for the treatment of PAH in pulmonary hypertension (PH) Group 1 in children is reported throughout literature. However, since the removal of the preservative might impact the safe use of the parenteral solution for infusion, the safety profile of the newly developed preservative-free treprostinil solution will be assessed within this trial.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| preservative-free parenteral treprostinil | Experimental | Drug: preservative-free parenteral treprostinilPatients will be treated with either SC or IV preservative-free treprostinil formulation. Dosing is not stipulated by the study protocol and will be done according to investigators discretion based on patient needs. |
Who may qualify
- Ages
- Up to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (10 listed)
- 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:
- Prostacyclin naïve or patients pre-treated with SC or IV treprostinil prior to screening
Key exclusion criteria (14 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| preservative-free parenteral treprostinilContinuous 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. | Drug | preservative-free parenteral treprostinil |
Primary outcome
Frequency and seriousness of adverse events and adverse drug reactions
Time frame: 5 months (20 weeks ± 1 weeks)
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).
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 Oct 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
5 sites in 5 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing preservative-free parenteral treprostinil in people with Pulmonary Arterial Hypertension, run by AOP Orphan Pharmaceuticals AG at 5 sites in 5 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 10 inclusion and 14 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 29 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 sites in Austria, France, Hungary, Slovakia, Spain. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Clinical Project Manager (4366488375206, trepaed@aop-health.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Frequency and seriousness of adverse events and adverse drug reactions (time frame: 5 months (20 weeks ± 1 weeks)). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 29 Oct 2026.