Treprostinil in Newborns With Pulmonary Hypertension; a Non-Interventional Study to Collect Data on Drug Utilization, Safety, and Effectiveness (NEPHY)

September 2, 2025 updated by: Ferrer Internacional S.A.

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).

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Recruiting

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Observational

Enrollment (Estimated)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
60

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact

The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.

Study Locations

  • France
      • Bron, France, 69500
        • Recruiting
        • CHU Lyon (HCL) - Hopital Femme Mère Enfant
        • Contact:
        • Principal Investigator:
          • Marine Butin, Medical Doctor
      • Grenoble, France, 38043
        • Recruiting
        • Réanimation néonatale CHU Grenoble Alpes - Hôpital Couple Enfant
        • Contact:
        • Principal Investigator:
          • Amélie Desrumaux, Medical Doctor
      • Lille, France, 59000
        • Recruiting
        • CHU Lille - Clinique de Néonatalogie
        • Contact:
        • Principal Investigator:
          • Kevin Leduc, Medical Doctor
      • Toulouse, France, 31300
        • Recruiting
        • CHU de Toulouse Hopital des enfants - Réanimation Néonatale
        • Contact:
        • Principal Investigator:
          • Sophie Breinig, Medical Doctor

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

  • Child

Accepts Healthy Volunteers

No

Sampling Method

Non-Probability Sample

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.

Description

Inclusion Criteria:

  1. 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.
  2. Aged up to 44 weeks after conception at treprostinil initiation.
  3. Pulmonary hypertension (PH) or suspicion of PH at the moment of treprostinil initiation.
  4. Parent(s) or legally authorized representative(s) provides non-opposition consent for the patient participation in the study.
  5. Newborn infants affiliated to French social security.

Exclusion Criteria:

  • None.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
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)
From baseline to Day 30 after last dose of treprostinil
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).
From baseline to Day 30 after last dose of treprostinil
Number of participants with treatment-related adverse events to evaluate safety.
Time Frame: From baseline to Day 30 after last dose of treprostinil
From baseline to Day 30 after last dose of treprostinil

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
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
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).
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.
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
From day 30 after treprostinil treatment to Year 2 after last dose of treprostinil

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Ferrer Internacional S.A.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
February 25, 2025

Primary Completion (Estimated)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
July 30, 2027

Study Completion (Estimated)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
December 31, 2027

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
May 21, 2024

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
July 11, 2024

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
July 12, 2024

Study Record Updates

Last Update Posted (Estimated)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
September 3, 2025

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
September 2, 2025

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
September 1, 2025

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • FREM-NIS-2301

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

UNDECIDED

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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