Safety of Sildenafil in Premature Infants
SIL02
3 other identifiers
interventional
109
2 countries
15
Brief Summary
Describe the safety of sildenafil in premature infants at risk of bronchopulmonary dysplasia and determine preliminary effectiveness and pharmacokinetics (PK) of sildenafil. Funding Source - FDA Office of Orphan Products Development (OOPD).
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P50-P75 for phase_2
Started Apr 2018
Longer than P75 for phase_2
15 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
April 17, 2017
CompletedFirst Posted
Study publicly available on registry
May 5, 2017
CompletedStudy Start
First participant enrolled
April 2, 2018
CompletedPrimary Completion
Last participant's last visit for primary outcome
March 1, 2025
CompletedStudy Completion
Last participant's last visit for all outcomes
March 1, 2025
CompletedResults Posted
Study results publicly available
March 11, 2026
CompletedMarch 30, 2026
December 1, 2025
6.9 years
April 17, 2017
December 18, 2025
March 10, 2026
Conditions
Outcome Measures
Primary Outcomes (1)
Safety as Determined by Adverse Event Experienced by Participants
Description of safety of sildenafil in premature infants and assessed by frequency and incidence of adverse events (AEs) and serious adverse events. Both non-serious and serious adverse events were collected from the time of informed consent through Day 14 after the last study intervention. Serious adverse events were additionally collected through 28 days after the last study intervention. Retinopathy of prematurity (ROP), whether serious or non-serious, was collected through hospital discharge or transfer; hospitalization duration varied based on clinical course, the longest duration was up to 575 days.
Nonserious AEs were collected through Day 14 post last intervention; SAEs through Day 28. Retinopathy of prematurity was assessed through discharge/transfer, up to 575 days after first treatment.
Secondary Outcomes (6)
Volume of Distribution
Pharmacokinetic samples were collected after any dose following completion of 7 days of study drug administration through Day 28 of study drug administration.
Clearance
Pharmacokinetic samples were collected after any dose following completion of 7 days of study drug administration through Day 28 of study drug administration.
Half-Life
Pharmacokinetic samples were collected after any dose following completion of 7 days of study drug administration through Day 28 of study drug administration.
Area Under the Curve (AUC)
Pharmacokinetic samples were collected after any dose following completion of 7 days of study drug administration through Day 28 of study drug administration.
Peak Plasma Concentration
Pharmacokinetic samples were collected after any dose following completion of 7 days of study drug administration through Day 28 of study drug administration.
- +1 more secondary outcomes
Study Arms (6)
Sildenafil cohort 1
EXPERIMENTALWithin cohort 1 infants will be randomized using a 3:1 scheme to receive sildenafil or placebo. Infants randomized to sildenafil will receive 0.125 mg/kg daily every 8 hours intravenously (IV), or 0.25 mg/kg daily every 8 hours enterally for 28 days.
Placebo cohort 1
PLACEBO COMPARATORInfants randomized to the placebo treatment group will receive the equivalent of dextrose 5% (sugar water) to be administered IV or enteral use.
Sildenafil cohort 2
EXPERIMENTALCohort 2 infants will receive sildenafil 0.5 mg/kg daily every 8 hours intravenously (IV) or 1 mg/kg daily every 8 hours enterally for 28 days.
Placebo cohort 2
PLACEBO COMPARATORInfants randomized to the placebo treatment group will receive the equivalent of dextrose 5% (sugar water) to be administered IV or enteral use.
Sildenafil cohort 3
EXPERIMENTALCohort 3 infants will receive sildenafil 1 mg/kg daily every 8 hours intravenously (IV) or 2 mg/kg daily every 8 hours enterally for 28 days.
Placebo cohort 3
PLACEBO COMPARATORInfants randomized to the placebo treatment group will receive the equivalent of dextrose 5% (sugar water) to be administered IV or enteral use.
Interventions
Infants will be randomized using a 3:1 scheme to receive sildenafil or placebo.
Infants randomized to the placebo group will receive the equivalent volume of dextrose 5% for IV use or enteral use (if receiving enteral study drug).
Eligibility Criteria
You may qualify if:
- Receiving positive airway pressure (nasal continuous airway pressure, nasal intermittent positive pressure ventilation, or nasal cannula flow \> 1LPM) or mechanical ventilation (high frequency or conventional)
- \<29 weeks gestational age at birth
- (inclusive) days postnatal age at time of randomization
You may not qualify if:
- Currently receiving vasopressors
- Currently receiving inhaled nitric oxide
- Baseline mean arterial pressure \< gestational age (in weeks) plus postnatal age (in weeks) within 2 hours of sildenafil administration
- Known allergy to sildenafil
- Known sickle cell disease
- Aspartate Aminotransferase (AST) \> 225 U/L \< 72 hours prior to randomization
- Alanine Aminotransferase (ALT) \> 150 U/L \< 72 hours prior to randomization
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (15)
University of Arkansas for Medical Sciences
Little Rock, Arkansas, 72205, United States
University of Florida College of Medicine Jacksonville-Wolfson Children's Hospital
Jacksonville, Florida, 32209, United States
University of Florida Jacksonville Shands Medical Center
Jacksonville, Florida, 32209, United States
University of Illinois at Chicago
Chicago, Illinois, 60612, United States
Wesley Medical Center
Wichita, Kansas, 67214, United States
University of Kentucky
Lexington, Kentucky, 40356, United States
Ochsner Baptist Medical Center
New Orleans, Louisiana, 70115, United States
University of Mississippi Medical Center
Jackson, Mississippi, 39216, United States
Children's Hospital of Nevada at UMC
Las Vegas, Nevada, 89106, United States
Monmouth Medical Center
Long Branch, New Jersey, 07740, United States
Cohen Children's Medical Center of NY
New Hyde Park, New York, 11040, United States
Golisano Children's Hospital - University of Rochester Medical Center
Rochester, New York, 14642, United States
WakeMed Health and Hospitals
Raleigh, North Carolina, 27610, United States
University of Oklahoma
Oklahoma City, Oklahoma, 73104, United States
Health Sciences Centre Hospital
Winnipeg, Manitoba, R3A 1R9, Canada
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Results Point of Contact
- Title
- Talaya McCright-Gill
- Organization
- Duke University
Study Officials
- PRINCIPAL INVESTIGATOR
Matthew M Laughon, MD, MPH
University of North Carolina, Chapel Hill
Publication Agreements
- PI is Sponsor Employee
- Yes
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- RANDOMIZED
- Masking
- QUADRUPLE
- Who Masked
- PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- OTHER
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
April 17, 2017
First Posted
May 5, 2017
Study Start
April 2, 2018
Primary Completion
March 1, 2025
Study Completion
March 1, 2025
Last Updated
March 30, 2026
Results First Posted
March 11, 2026
Record last verified: 2025-12
Data Sharing
- IPD Sharing
- Will not share