Persistent Pulmonary Hypertension of NewbornNEPHYNCT06499363
Treprostinil in Newborns With Pulmonary Hypertension; a Non-Interventional Study to Collect Data on Drug Utilization, Safety, and Effectiveness
An observational study of Persistent Pulmonary Hypertension of Newborn, run by Ferrer Internacional S.A. at 4 sites in France.
Trial summary
This observational study will seek to describe the uses and safety and efficacy outcomes of treprostinil in clinical practice in patients with persistent pulmonary hypertension (PPHN). Read more about this study
- Who is it for?
- Ages Up to 44 weeks
- Conditions the study is for
- Persistent Pulmonary Hypertension of Newborn
- How many people
- 60 participants
- Where
- 4 sitesFrance
- Study dates
- 25 Feb 2025to 31 Dec 2027
- Phase
- Observational
- 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 44 weeks
- All sexes
- No healthy volunteers
- Child
Study population: Approximately 60 newborns with PH (of whom at least 40 newborns with persistent pulmonary hypertension of the newborn [PPHN]) are expected to be included.
Who may be able to join
- Newborn infants who received at least one dose of IV or SC treprostinil as per clinical practice within 5 years from the moment of data collection.
- Aged up to 44 weeks after conception at treprostinil initiation.
- Pulmonary hypertension (PH) or suspicion of PH at the moment of treprostinil initiation.
- Parent(s) or legally authorized representative(s) provides non-opposition consent for the patient participation in the study.
- Newborn infants affiliated to French social security.
Who cannot join
- None.
Trial locations and contacts
4 sites in 1 country. Contact the study team or a site near you to ask about taking part.
France4 sites, 4 recruiting
- Recruiting
CHU Lyon (HCL) - Hopital Femme Mère Enfant
Bron, 69500
Marine Butin, Medical Doctor, +33 (0)4 27 85 52 84, marine.butin@chu-lyon.fr
Marine Butin, Medical Doctor
- Recruiting
Réanimation néonatale CHU Grenoble Alpes - Hôpital Couple Enfant
Grenoble, 38043
Amélie Desrumaux, Medical Doctor, +33(0)4 76 76 55 03, ADesrumaux@chu-grenoble.fr
Amélie Desrumaux, Medical Doctor
- Recruiting
CHU Lille - Clinique de Néonatalogie
Lille, 59000
Kevin Leduc, Medical Doctor, +33 03 20 44 58 89, Kevin.LEDUC@chu-lille.fr
Kevin Leduc, Medical Doctor
- Recruiting
CHU de Toulouse Hopital des enfants - Réanimation Néonatale
Toulouse, 31300
Sophie Breinig, Medical Doctor, +33 (0)5 34 55 84 77, breinig.s@chu-toulouse.fr
Sophie Breinig, Medical Doctor
About this study
Official title: Treprostinil in Newborns With Pulmonary Hypertension; a Noninterventional Study to Collect Data on Drug Utilization, Safety, and Effectiveness.
This observational study will seek to describe the uses and safety and efficacy outcomes of treprostinil in clinical practice in patients with persistent pulmonary hypertension (PPHN).
- Medical subject headings
- Persistent Fetal Circulation Syndrome
What is being measured
The primary outcome is the main question the study is designed to answer.
Characteristics of neonates receiving treprostinil in the clinical practice.
Sex (male/female) ; Gestational Age (weeks) ; Birth Weight (g) ; Apgar (1 and 5 min, 0-10) and Time from birth to treprostinil initiation (h)
Measured: From baseline to Day 30 after last dose of treprostinil
Treprostinil-specific treatment patterns.
Prior concomitant treatments, treprostinil route, treprostinil dose(s) (mg/ml) and treprostinil treatment duration (days).
Measured: From baseline to Day 30 after last dose of treprostinil
Number of participants with treatment-related adverse events to evaluate safety.
Measured: From baseline to Day 30 after last dose of treprostinil
Secondary outcomes (3)
Composite endpoint of absence of complications through Day 14 after the index date.
Measured: From baseline to Day 30 after last dose of treprostinil
Description of the clinical outcomes from treprostinil start day (i.e., Day 0) to Day 14 and at Day 30 after last dose of treprostinil.
Evolution of pre- and post-ductal oxygen saturation (SpO2); oxygen partial pressure (PO2); clinical laboratory parameters and fraction of inspired oxygen (FiO2) at times 0h, 24h, 48h, 72h, Day 7, Day 14, and Day 30 after last dose of treprostinil.
Measured: 0h, 24h, 48h, 72h, Day 7, Day 14, and Day 30 after last dose of treprostinil (if applicable).
Description of neurodevelopmental progress over 2 years after treprostinil initiation through a Global Clinical Impression Scale.
Measured: From day 30 after treprostinil treatment to Year 2 after last dose of treprostinil
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Jul 2024
- Study start25 Feb 2025
- Primary completion30 Jul 2027 (estimated)
- Study completion31 Dec 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Persistent Pulmonary Hypertension of NewbornStudies 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
- Observational
- Observational model
- Cohort
- Time perspective
- Other
- Enrolment
- 60 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Ferrer Internacional S.A.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06499363
- Sponsor's protocol ID
- FREM-NIS-2301
- Acronym
- NEPHY
- Status last verified
- Sep 2025
- First submitted
- 21 May 2024
- Last updated
- 3 Sep 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 3 Sep 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Treprostinil in Newborns With Pulmonary Hypertension; a Noninterventional Study to Collect Data on Drug Utilization, Safety, and Effectiveness.
- ClinicalTrials.gov ID
- NCT06499363
- Acronym / sponsor ID
- NEPHY / FREM-NIS-2301
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 60 participants (estimated)
- Lead sponsor
- Ferrer Internacional S.A.Industry (pharma, biotech, devices)
- Study start
- 25 Feb 2025 (actual)
- Primary completion
- 30 Jul 2027 (estimated)
- Study completion
- 31 Dec 2027 (estimated)
- First posted
- 12 Jul 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.
This observational study will seek to describe the uses and safety and efficacy outcomes of treprostinil in clinical practice in patients with persistent pulmonary hypertension (PPHN).
Study design
- Observational model
- Cohort
- Time perspective
- Other
Who may qualify
- Ages
- Up to 44 weeks
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
- Study population
- Approximately 60 newborns with PH (of whom at least 40 newborns with persistent pulmonary hypertension of the newborn [PPHN]) are expected to be included.
Key inclusion criteria (5 listed)
- Newborn infants who received at least one dose of IV or SC treprostinil as per clinical practice within 5 years from the moment of data collection.
- Aged up to 44 weeks after conception at treprostinil initiation.
- Pulmonary hypertension (PH) or suspicion of PH at the moment of treprostinil initiation.
- Parent(s) or legally authorized representative(s) provides non-opposition consent for the patient participation in the study.
- Newborn infants affiliated to French social security.
Key exclusion criteria (1 listed)
- None.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Characteristics of neonates receiving treprostinil in the clinical practice.
Time frame: From baseline to Day 30 after last dose of treprostinil
Sex (male/female) ; Gestational Age (weeks) ; Birth Weight (g) ; Apgar (1 and 5 min, 0-10) and Time from birth to treprostinil initiation (h)
Treprostinil-specific treatment patterns.
Time frame: From baseline to Day 30 after last dose of treprostinil
Prior concomitant treatments, treprostinil route, treprostinil dose(s) (mg/ml) and treprostinil treatment duration (days).
Number of participants with treatment-related adverse events to evaluate safety.
Time frame: From baseline to Day 30 after last dose of treprostinil
3 secondary outcomes
Composite endpoint of absence of complications through Day 14 after the index date.
Time frame: From baseline to Day 30 after last dose of treprostinil
Description of the clinical outcomes from treprostinil start day (i.e., Day 0) to Day 14 and at Day 30 after last dose of treprostinil.
Time frame: 0h, 24h, 48h, 72h, Day 7, Day 14, and Day 30 after last dose of treprostinil (if applicable).
Description of neurodevelopmental progress over 2 years after treprostinil initiation through a Global Clinical Impression Scale.
Time frame: From day 30 after treprostinil treatment to Year 2 after last dose of treprostinil
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 3 Sep 2025.
- Individual participant dataSharing planned: Undecided
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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.
4 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Persistent Pulmonary Hypertension of Newborn, run by Ferrer Internacional S.A. at 4 sites in France.
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 5 inclusion and 1 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 3 Sep 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites in France. 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.
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 Ferrer MedInfo (609850565, medinfo@ferrer.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Characteristics of neonates receiving treprostinil in the clinical practice. (time frame: From baseline to Day 30 after last dose of treprostinil). The study lists 3 primary outcomes in all. 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 3 Sep 2025.