Current Environment:


This study assessed the safety and treatment effect of intravenous (IV) Remodulin as an add-on therapy in neonates with persistent pulmonary hypertension of the newborn (PPHN).


Persistent Pulmonary Hypertension of the Newborn

Recruitment Status


Detailed Description

This study was designed to investigate if the addition of Remodulin reduced the rate of clinical worsening (defined as the need for additional treatment targeting PPHN, need for extracorporeal mechanical oxygenation [ECMO], or death) in neonatal subjects with PPHN who did not show an adequate response to inhaled nitric oxide (iNO). This study was part of a pediatric investigation plan agreed upon by the EMA (EMEA 000207-PIP01-08-M08).

Eligibility Criteria

Inclusion Criteria:

Parent(s) or legal guardian provided consent for the subject to participate
Weight at least 2 kg at Screening
Gestational age of ≥34 weeks and ≤14 days old at Screening
Diagnosis of PPHN, which was either idiopathic in nature or associated with the following: meconium aspiration syndrome, pneumonia, respiratory distress syndrome, sepsis, birth hypoxia, perinatal encephalopathy, or unilateral congenital diaphragmatic hernia
Currently requiring ventilator support
Two consecutive oxygenation index (OI) of 15 or greater separated by at least 30 minutes, after receiving iNO for at least 3 hours
Echocardiographic (ECHO) evidence of pulmonary hypertension with elevated right ventricle pressure
Dedicated venous access for the administration of study drug (central line or peripherally inserted central venous catheter)

Exclusion Criteria:

Previous or concurrent use of a phosphodiesterase-5 inhibitor, endothelin receptor antagonist, or prostanoid
Significant congenital heart disease as detected by ECHO, minor valvular abnormalities, or expected transitional findings such as a patent foramen ovale, or patent ductus arteriosus.
Clinically significant, untreated active pneumothorax at Screening
Evidence of clinically significant bleeding at Screening
Necrotizing enterocolitis (≥Bells stage II at Screening)
Uncontrolled hypotension (mean systemic pressures ≤35 mmHg at Screening)
Uncontrolled coagulopathy and / or untreated thrombocytopenia (<50,000 platelets/µL at Screening)
History of severe (Grade 3 or 4) intracranial hemorrhage at Screening
Currently receiving extracorporeal mechanical oxygenation (ECMO) or had immediate plans to initiate ECMO
Expected duration on mechanical ventilation of <48 hours
Life expectancy was less than 2 months or had a lethal chromosomal anomaly
Contraindication to ECMO
Bilateral congenital diaphragmatic hernia
Active seizures at Screening
Currently participating in another clinical drug study


Intervention Type

Intervention Name


IV Remodulin




Phase 2



Min Age

1 Hour

Max Age

14 Days

Download Date

March 5, 2024

Principal Investigator


Primary Contact Information

For more information on this trial, visit


For more information and to contact the study team:

Remodulin as Add-on Therapy for the Treatment of Persistent Pulmonary Hypertension of the Newborn NCT02261883