Remodulin as Add-on Therapy for the Treatment of Persistent Pulmonary Hypertension of the Newborn
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT02261883|
Recruitment Status : Recruiting
First Posted : October 10, 2014
Last Update Posted : March 30, 2021
|Condition or disease||Intervention/treatment||Phase|
|Persistent Pulmonary Hypertension of the Newborn||Drug: IV Remodulin Drug: Placebo||Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||70 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)|
|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 Efficacy Study|
|Study Start Date :||May 2015|
|Estimated Primary Completion Date :||December 2022|
|Estimated Study Completion Date :||December 2022|
Active Comparator: IV Remodulin
IV Remodulin will be initiated at 1 ng/kg/min. The dose will be increased in up to 2 ng/kg/min increments every 2 hrs until the OI is <10 (in the absence of dose-limiting side effects).
Drug: IV Remodulin
Treprostinil is a chemically stable tricyclic analogue of prostacyclin.
Other Name: treprostinil
Placebo Comparator: Placebo
Placebo will be initiated at 1 ng/kg/min. The dose will be increased in up to 2 ng/kg/min increments every 2 hrs until the OI is <10 (in the absence of dose-limiting side effects).
- 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
- 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 ]
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02261883
|Contact: United Therapeutics Global Medical Informationfirstname.lastname@example.org|