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

Not yet recruiting

• Conditions: Persistent Pulmonary Hypertension of Newborn

• Registration Number: NCT06499363
• Lead Sponsor: Ferrer Internacional S.A.

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).

Detailed Description

Not available

Study Timeline

• Status Verified: 2024-05
• Study First Submitted: 2024-05-21
• Study First Submitted QC: 2024-07-11
• Last Update Submitted: 2024-07-11
• Study First Posted: 2024-07-12
• Last Update Posted: 2024-07-12
• Study Start: 2024-07-21
• Primary Completion: 2027-07-30
• Study Completion: 2027-12-31

Recruitment & Eligibility

• Status: NOT_YET_RECRUITING
• Sex: All
• Target Recruitment: 60

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 & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Characteristics of neonates receiving treprostinil in the clinical practice.	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)
Number of participants with treatment-related adverse events to evaluate safety.	From baseline to Day 30 after last dose of treprostinil
Treprostinil-specific treatment patterns.	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).

Secondary Outcome Measures

Name	Time	Method
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
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).	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.