Treprostinil for newborns with persistent pulmonary hypertension

Treprostinil in Newborns With Pulmonary Hypertension; a Noninterventional Study to Collect Data on Drug Utilization, Safety, and Effectiveness.

This observational project will see if treprostinil used in newborns with persistent pulmonary hypertension appears safe and helpful when used in routine care in French hospitals.

Quick facts

• Study type: Observational
• Enrollment: 60 (estimated)
• Ages: N/A to 44 Weeks
• Sex: All
• Sponsor: Ferrer Internacional S.A. (industry)
• Locations: 4 sites (Bron and 3 other locations)
• Trial ID: NCT06499363 on ClinicalTrials.gov

What this trial studies

This is a non-interventional, observational registry collecting real-world clinical data on newborns up to 44 weeks post-conception who received at least one IV or SC dose of treprostinil within the five years before data collection. Clinical sites will extract dosing, route, timing, baseline status, adverse events, and short-term outcomes from medical records at participating French tertiary neonatal centers. Parents or legal representatives provide consent for inclusion and no changes to clinical care are required by the protocol. The dataset will be analyzed descriptively to characterize utilization patterns, safety signals, and clinical outcomes in this population.

Who should consider this trial

Good fit: Newborn infants (up to 44 weeks after conception) with pulmonary hypertension or suspected PH who received at least one IV or SC dose of treprostinil within five years and whose parent or legal representative consents, and who are affiliated with French social security, are ideal candidates.

Not a fit: Infants who were never treated with treprostinil, treated outside the five-year window, not affiliated with French social security, or lacking consent will not be included and therefore will not directly benefit from this dataset.

Why it matters

Potential benefit: If successful, the study could clarify real-world safety and outcome patterns for treprostinil in PPHN and help clinicians make better-informed treatment decisions.

How similar studies have performed: Existing evidence for treprostinil in PPHN is limited largely to case reports and small series, so randomized or large-scale evidence is lacking and this real-world dataset is relatively novel.

Eligibility criteria

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.

Where this trial is running

Bron and 3 other locations

• CHU Lyon (HCL) - Hopital Femme Mère Enfant — Bron, France (RECRUITING)
• Réanimation néonatale CHU Grenoble Alpes - Hôpital Couple Enfant — Grenoble, France (RECRUITING)
• CHU Lille - Clinique de Néonatalogie — Lille, France (RECRUITING)
• CHU de Toulouse Hopital des enfants - Réanimation Néonatale — Toulouse, France (RECRUITING)

Study contacts

• Study coordinator: Ferrer MedInfo

Conditions: Persistent Pulmonary Hypertension of Newborn