Current Environment:

Summary

This pilot study aims to assess the safety and treatment effect of acute dosing with IV Remodulin in neonates with persistent pulmonary hypertension of the newborn (PPHN).

Conditions

Persistent Pulmonary Hypertension of the Newborn

Recruitment Status

Recruiting

Detailed Description

This study will enroll subjects with PPHN who do not show an adequate response to inhaled nitric oxide with the hypothesis that the addition of intravenous (IV) Remodulin will reduce the rate of clinical worsening as compared to standard of care. Additionally, this study aims to evaluate the treatment effect of Remodulin and better understand the dosing and pharmacokinetics in the neonatal population.

Eligibility Criteria

Inclusion Criteria:

Parent(s) or guardian provides consent for the subject to participate, as per institutional policy
At least 2 kg at Screening
Gestational age ≥ 34 weeks and ≤ 14 days old at Screening
Diagnosis of PPHN, which is either idiopathic in nature or associated with the following: meconium aspiration syndrome (MAS), pneumonia, respiratory distress syndrome (RDS), sepsis, birth hypoxia, perinatal encephalopathy or unilateral congenital diaphragmatic hernia (CDH)
Currently requiring ventilator support
Receiving iNO with two OIs of 15 or greater separated by at least 30 minutes after receiving iNO for at least 3 hours
Echocardiographic 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 (PDE5i), endothelin receptor antagonist (ERA), or prostanoid
Significant congenital heart disease (CHD) as detected by ECHO (excluding presence of minor defects such as small secundum atrial septal defect (ASD), minor valvular abnormalities, or expected transitional findings such as a patent foramen ovale (PFO), or patent ductus arteriosus (PDA). Subjects with small muscular, restrictive ventricular septal defect (VSD) may be enrolled
Clinically significant, untreated active pneumothorax at Screening
Evidence of clinically significant bleeding
Necrotizing entercolitis; ≥ Bells stage II at Screening
Uncontrolled hypotension; mean systemic pressures ≤ 35 mmHg at Screening.
Uncontrolled coagulopathy and / or untreated thrombocytopenia; defined as <50,000 platelets /µL at Screening
History of severe (Grade 3 or 4) intracranial hemorrhage
Currently receiving ECMO or has immediate plans to initiate ECMO
Expected duration on mechanical ventilation of less than 48 hours
Life expectancy is less than two months or has a lethal chromosomal anomaly
Contraindication to ECMO
Bilateral congenital diaphragmatic hernia
Active seizures at Screening
Currently participating in another clinical drug study (excluding observational registries)

Intervention

Intervention Type

Intervention Name

Drug

IV Remodulin

Drug

Placebo

Phase

Phase 2

Gender

All

Min Age

N/A

Max Age

14 Days

Download Date

April 14, 2022

Principal Investigator

N/A

Primary Contact Information

United Therapeutics Global Medical Information

919-485-8350

clinicaltrials@unither.com

For more information on this trial, visit clinicaltrials.gov.

Contact

For more information and to contact the study team: