Clinical Trial: Remodulin as Add-on Therapy for the Treatment of Persistent Pulmonary Hypertension of the Newborn

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

Official Title: Intravenous Remodulin (Treprostinil) as Add-on Therapy for the Treatment of Persistent Pulmonary Hypertension of the Newborn: A Randomized, Placebo-Controlled, Safety and

Brief 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).

Detailed Summary: 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.
Sponsor: United Therapeutics

Current Primary Outcome: Evaluate the rate of clinical worsening in neonates with PPHN [ Time Frame: Up to Day 14 ]

Efficacy will be assessed by a composite endpoint of clinical worsening as defined by the following:

  • Initiation of additional pulmonary vasodilator therapy
  • Initiation of extracorporeal mechanical oxygenation (ECMO) per institutional policies
  • Death


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Time to discontinuation of inhaled nitric oxide (iNO) [ Time Frame: Up to Day 56 ]
  • Change in oxygenation index (OI) [ Time Frame: Hour 12, hour 24, hour 72, Day 7 and Day 14/ or prior to study drug discontiuation ]
    OI= [MAP(mmHg) x FiO2(%) / PaO2(mmHg)] x 100
  • Time on mechanical ventilation [ Time Frame: Up to Day 56 ]
  • Time to initiation of ECMO [ Time Frame: Up to Day 56 ]
  • Mean treprostinil plasma concentration per dose achieved [ Time Frame: 24 hours after initiation of Remodulin and immediately prior to wean ]
    Two blood samples will be collected from each patient for treprostinil pharmacokinetic (PK) analysis. Plasma samples will be analyzed for treprostinil using a validated bioanalytical plasma assay.
  • Safety [ Time Frame: up to Day 56 ]
    Assessment of adverse events, change in vital signs, and change in labs.
  • Change in partial pressure of oxygen in arterial blood (PaO2) / fraction of inspired oxygen (FiO2) [P/F ratio] [ Time Frame: Hour 12, hour 24, and hour 72 ]
  • Change in N-terminal pro-Brain Natriuretic Peptide (NT-proBNP) [ Time Frame: Day 7, Day 14, prior to study drug wean, study drug discontinuation ]
  • Change in pre and post-ductal oxygen saturation (SpO2) [ Time Frame: Hour 6, hour 12, hour 24, and hour 72 ]


Original Secondary Outcome: Same as current

Information By: United Therapeutics

Dates:
Date Received: October 30, 2013
Date Started: May 2015
Date Completion: January 2018
Last Updated: April 5, 2017
Last Verified: April 2017