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

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: Recruiting
• Conditions: Persistent Pulmonary Hypertension of Newborn

• Study Type: Observational
• Enrollment (Estimated): 60

Contacts and Locations

• Study Contact: Name: Ferrer MedInfo; Phone Number: 609850565; Email: medinfo@ferrer.com

Study Locations

• France
  • Bron, France, 69500
    • Recruiting
    • CHU Lyon (HCL) - Hopital Femme Mère Enfant
    • Contact: Marine Butin, Medical Doctor; Phone Number: +33 (0)4 27 85 52 84; Email: marine.butin@chu-lyon.fr
    • Principal Investigator: Marine Butin, Medical Doctor
  • Grenoble, France, 38043
    • Recruiting
    • Réanimation néonatale CHU Grenoble Alpes - Hôpital Couple Enfant
    • Contact: Amélie Desrumaux, Medical Doctor; Phone Number: +33(0)4 76 76 55 03; Email: ADesrumaux@chu-grenoble.fr
    • Principal Investigator: Amélie Desrumaux, Medical Doctor
  • Lille, France, 59000
    • Recruiting
    • CHU Lille - Clinique de Néonatalogie
    • Contact: Kevin Leduc, Medical Doctor; Phone Number: +33 03 20 44 58 89; Email: Kevin.LEDUC@chu-lille.fr
    • Principal Investigator: Kevin Leduc, Medical Doctor
  • Toulouse, France, 31300
    • Recruiting
    • CHU de Toulouse Hopital des enfants - Réanimation Néonatale
    • Contact: Sophie Breinig, Medical Doctor; Phone Number: +33 (0)5 34 55 84 77; Email: breinig.s@chu-toulouse.fr
    • Principal Investigator: Sophie Breinig, Medical Doctor

Participation Criteria

Eligibility Criteria

• 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

How is the study designed?

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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)	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).	From baseline to Day 30 after last dose of treprostinil
Number of participants with treatment-related adverse events to evaluate safety.		From baseline to Day 30 after last dose of treprostinil

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Composite endpoint of absence of complications through Day 14 after the index date.		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.	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.		From day 30 after treprostinil treatment to Year 2 after last dose of treprostinil

Collaborators and Investigators

• Sponsor: Ferrer Internacional S.A.

Study record dates

Study Major Dates

• Study Start (Actual): February 25, 2025
• Primary Completion (Estimated): July 30, 2027
• Study Completion (Estimated): December 31, 2027

Study Registration Dates

• First Submitted: May 21, 2024
• First Submitted That Met QC Criteria: July 11, 2024
• First Posted (Actual): July 12, 2024

Study Record Updates

• Last Update Posted (Estimated): September 3, 2025
• Last Update Submitted That Met QC Criteria: September 2, 2025
• Last Verified: September 1, 2025

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Respiratory Tract Diseases; Lung Diseases; Infant, Newborn, Diseases; Hypertension, Pulmonary; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Persistent Fetal Circulation Syndrome
• Other Study ID 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