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

NCT06499363 | Recruiting

• 1 other identifier: FREM-NIS-2301
• Study Type: observational
• Target: 60
• Locations: 1 country
• Sites: 4

Brief 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).

Conditions

Persistent Pulmonary Hypertension of Newborn

Outcome Measures

Primary Outcomes (3)

• 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 Outcomes (3)

• 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.

  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

Eligibility Criteria

• Age: Up to 44 Weeks
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)
• 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.

You may qualify if:

• 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.

You may not qualify if:

• None.

Sponsors & Collaborators

• Ferrer Internacional S.A.: lead

Study Sites (4)

CHU Lyon (HCL) - Hopital Femme Mère Enfant

Bron, 69500, France

RECRUITING

Réanimation néonatale CHU Grenoble Alpes - Hôpital Couple Enfant

Grenoble, 38043, France

RECRUITING

CHU Lille - Clinique de Néonatalogie

Lille, 59000, France

RECRUITING

CHU de Toulouse Hopital des enfants - Réanimation Néonatale

Toulouse, 31300, France

RECRUITING

MeSH Terms

Conditions

Persistent Fetal Circulation Syndrome

Condition Hierarchy (Ancestors)

Hypertension, Pulmonary; Lung Diseases; Respiratory Tract Diseases; Infant, Newborn, Diseases; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Central Study Contacts

Ferrer MedInfo

CONTACT

609850565; medinfo@ferrer.com

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: OTHER
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 21, 2024
• First Posted: July 12, 2024
• Study Start: February 25, 2025
• Primary Completion (Estimated): July 30, 2027
• Study Completion (Estimated): December 31, 2027
• Last Updated: September 3, 2025

Record last verified: 2025-09

Locations

FR (4)