A Study To Evaluate Safety And Efficacy Of IV Sildenafil In The Treatment Of Neonates With Persistent Pulmonary Hypertension Of The Newborn
A MULTI-CENTRE, RANDOMIZED, PLACEBO-CONTROLLED, DOUBLE-BLIND, TWO-ARMED, PARALLEL GROUP STUDY TO EVALUATE EFFICACY AND SAFETY OF IV SILDENAFIL IN THE TREATMENT OF NEONATES WITH PERSISTENT PULMONARY HYPERTENSION OF THE NEWBORN (PPHN) OR HYPOXIC RESPIRATORY FAILURE AND AT RISK FOR PPHN, WITH A LONG TERM FOLLOW-UP INVESTIGATION OF DEVELOPMENTAL PROGRESS 12 AND 24 MONTHS AFTER COMPLETION OF STUDY TREATMENT
2 other identifiers
interventional
59
12 countries
42
Brief Summary
This study will evaluate whether IV sildenafil can reduce the time on inhaled nitric oxide treatment and reduce the failure rate of available treatments for persistent pulmonary hypertension of the newborn.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for phase_3
Started Aug 2013
Longer than P75 for phase_3
42 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
September 17, 2012
CompletedFirst Posted
Study publicly available on registry
November 2, 2012
CompletedStudy Start
First participant enrolled
August 5, 2013
CompletedPrimary Completion
Last participant's last visit for primary outcome
October 17, 2018
CompletedResults Posted
Study results publicly available
March 25, 2020
CompletedStudy Completion
Last participant's last visit for all outcomes
September 28, 2020
CompletedAugust 16, 2021
August 1, 2021
5.2 years
September 17, 2012
October 16, 2019
August 12, 2021
Conditions
Keywords
Outcome Measures
Primary Outcomes (2)
Time on Inhaled Nitric Oxide (iNO) Treatment After Initiation of Intravenous (IV) Study Drug For Participants Without Treatment Failure
Time in days, on iNO treatment, for participants without iNO treatment failure, was calculated 14 days from the initiation of IV study drug or hospital discharge, whichever occurred first. iNO treatment failure was defined as need for additional treatment targeting PPHN, need for extra corporeal membrane oxygenation (ECMO), or death during the study.
14 days from the initiation of IV study drug or hospital discharge, whichever occurs first (maximum of 14 days)
Treatment Failure Rate
Treatment failure rate was defined as percentage of participants who needed additional treatment targeting PPHN, needed ECMO, or died during the study.
14 days from the initiation of IV study drug or hospital discharge, whichever occurs first (maximum of 14 days)
Secondary Outcomes (30)
Time From Initiation of Intravenous (IV) Study Drug to Final Weaning of Mechanical Ventilation
14 days from the initiation of IV study drug or hospital discharge, whichever occurs first (maximum of 14 days)
Time From Initiation of Intravenous (IV) Study Drug to First Treatment Failure
14 days from the initiation of IV study drug or hospital discharge, whichever occurs first (maximum of 14 days)
Percentage of Participants With Individual Components of Treatment Failure
14 days from the initiation of IV study drug or hospital discharge, whichever occurs first (maximum of 14 days)
Change From Baseline in Oxygenation Index (OI) at Hours 6, 12 and 24 Post-Infusion
Baseline, Hours 6, 12 and 24 after start of infusion
Change From Baseline in Differential Saturation at Hours 6, 12 and 24 Post-Infusion
Baseline, Hours 6, 12 and 24 after start of infusion
- +25 more secondary outcomes
Study Arms (2)
placebo
PLACEBO COMPARATORiv placebo of normal saline or 10% dextrose
sildenafil
EXPERIMENTALActive study drug
Interventions
IV placebo or 0.9% sodium chloride or 10% dextrose. Infusion rate based on weight.
loading dose of 0.1 mg/kg over 30 minutes followed by maintenance dose of 0.03 mg/kg/h. To infuse minimum 48 hours and maximum of 14 days.
Eligibility Criteria
You may qualify if:
- Neonates with persistent pulmonary hypertension of the newborn
- Age \<=96 hours and \>=34 weeks gestational age
- Oxygenation Index \>15 and \<60
- Concurrent treatment with inhaled nitric oxide and \>=50% oxygen
You may not qualify if:
- Prior or immediate need for extracorporeal membrane oxygenation or cardiopulmonary resuscitation
- Expected duration of mechanical ventilation \<48 hours
- Profound hypoxemia
- Life-threatening or lethal congenital anomaly
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (42)
Arkansas Children's Hospital
Little Rock, Arkansas, 72202, United States
University of California Davis Medical Center
Sacramento, California, 95817, United States
University of California Davis
Sacramento, California, 95817, United States
University of California Davis Medical Center
Sacramento, California, 95825, United States
Children´s National Medical Center
Washington D.C., District of Columbia, 20010, United States
Indiana University Health Methodist Hospital
Indianapolis, Indiana, 46202, United States
Riley Hospital for Children at IU Health
Indianapolis, Indiana, 46202, United States
Sydney and Lois Eskenazi Hospital
Indianapolis, Indiana, 46202, United States
Children's Mercy Hospitals & Clinics
Kansas City, Missouri, 64108, United States
Duke University Medical Center (DUMC)
Durham, North Carolina, 27710, United States
Fairview Hospital
Cleveland, Ohio, 44111, United States
Cleveland Clinic
Cleveland, Ohio, 44195, United States
OU Follow-Up Program, PREMIEr Clinic, Children's Hospital
Oklahoma City, Oklahoma, 73104, United States
OU Neonatal Intensive Care Unit at Children's Hospital
Oklahoma City, Oklahoma, 73104, United States
The University of Oklahoma Health Sciences Center
Oklahoma City, Oklahoma, 73104, United States
Henry Zarrow Neonatal Intensive Care Unit, Children's Hospital at Saint Francis
Tulsa, Oklahoma, 74136, United States
Warren Cancer Research Foundation
Tulsa, Oklahoma, 74136, United States
Vanderbilt Children's Hospital
Nashville, Tennessee, 37232, United States
Seattle Children's Research Institute
Seattle, Washington, 98101, United States
Seattle Childrens Hospital
Seattle, Washington, 98105, United States
Universitair Ziekenhuis Antwerpen
Edegem, 2650, Belgium
UZ Gent
Ghent, 9000, Belgium
CHUL du CHU de Quebec
Québec, Quebec, G1V 4G2, Canada
Aarhus Universitetshospital, Skejby
Aarhus N, 8200, Denmark
Neonatalklinikken Rigshospitalet, 5024
Copenhagen Ø, 2100, Denmark
Centre Hospitalier et Regional de Lille - Hopital Jeanne de Flandre
Lille, 59037, France
Centre Hospitalier et Régional de Lille,
Lille, 59037, France
Hôpital de la Conception Assistance Publique-Hôpitaux de Marseille
Marseille, 13385, France
CHU Robert Debré
Paris, 75019, France
Hopital NECKER - Enfants Malades
Paris, 75743, France
University Hospital of Leipzig
Leipzig, 04103, Germany
Neonatologia Fondazione IRCCS Policlinico San Matteo
Pavia, 27100, Italy
Radboud University Nijmegen Medical Centre
Nijmegen, 6525 GA, Netherlands
Erasmus MC, Sophia Children's hospital
Rotterdam, 3015 CN, Netherlands
Haukeland University Hospital
Bergen, Haukeland, 5000, Norway
Hospital Sant Joan de Deu
Esplugues de Llobregat, Barcelona / Spain, 08950, Spain
Hospital General Universitario Gregorio Marañon
Madrid, 28007, Spain
Karolinska University Hospital
Stockholm, 171 76, Sweden
St. Michael's Hospital
Bristol, BS2 8EG, United Kingdom
Leicester Royal Infirmary
Leicester, LE1 5WW, United Kingdom
Glenfield Hospital, University Hospitals of Leicester NHS Trust
Leicester, LE3 9QP, United Kingdom
Great Ormond Street Hospital NHS Foundation Trust
London, WC1N 3JH, United Kingdom
Related Publications (1)
Pierce CM, Zhang MH, Jonsson B, Iorga D, Cheruvu N, Balagtas CC, Steinhorn RH. Efficacy and Safety of IV Sildenafil in the Treatment of Newborn Infants with, or at Risk of, Persistent Pulmonary Hypertension of the Newborn (PPHN): A Multicenter, Randomized, Placebo-Controlled Trial. J Pediatr. 2021 Oct;237:154-161.e3. doi: 10.1016/j.jpeds.2021.05.051. Epub 2021 May 27.
PMID: 34052232DERIVED
Related Links
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Results Point of Contact
- Title
- Pfizer ClinicalTrials.gov Call Center
- Organization
- Pfizer Inc.
Study Officials
- STUDY DIRECTOR
Pfizer CT.gov Call Center
Pfizer
Publication Agreements
- PI is Sponsor Employee
- No
- Restriction Type
- OTHER
- Restrictive Agreement
- Yes
Study Design
- Study Type
- interventional
- Phase
- phase 3
- Allocation
- RANDOMIZED
- Masking
- DOUBLE
- Who Masked
- PARTICIPANT, INVESTIGATOR
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
September 17, 2012
First Posted
November 2, 2012
Study Start
August 5, 2013
Primary Completion
October 17, 2018
Study Completion
September 28, 2020
Last Updated
August 16, 2021
Results First Posted
March 25, 2020
Record last verified: 2021-08
Data Sharing
- IPD Sharing
- Will share
Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical\_trials/trial\_data\_and\_results/data\_requests.