Sildenafil for Bronchopulmonary Dysplasia
(SILDI-SAFE Trial)
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial aims to test the safety of sildenafil (commonly used for erectile dysfunction and pulmonary hypertension) in treating severe bronchopulmonary dysplasia (BPD) in premature infants. BPD is a serious lung condition affecting preemies who require extra breathing support. The study will compare different doses of sildenafil to a placebo to evaluate its effectiveness in these infants. Babies born before 29 weeks who still need breathing assistance in the NICU may qualify for this study. As a Phase 2 trial, this research measures the treatment's effectiveness in an initial, smaller group, offering a chance to contribute to important medical advancements.
Will I have to stop taking my current medications?
The trial does not specify if participants must stop taking their current medications, but it does exclude those who have recently taken certain drugs like sildenafil, vasopressors, inhaled nitric oxide, and milrinone. It's best to discuss your current medications with the study team.
Is there any evidence suggesting that this trial's treatments are likely to be safe?
Research shows that sildenafil is increasingly used in premature babies, despite limited safety information. Some studies suggest it might help reduce lung damage, offering hope for babies with bronchopulmonary dysplasia (BPD). Notably, one study found that sildenafil did not increase the risk of severe retinopathy of prematurity, a serious eye condition, in very low birth weight babies.
The FDA does not recommend sildenafil for children aged 1 to 17 with high blood pressure in the lungs, and no specific guidelines exist for its use in newborns. Researchers are still gathering safety data specifically for newborns. While sildenafil shows potential benefits, its safety in premature babies remains under study, and more information is needed.12345Why do researchers think this study treatment might be promising for BPD?
Most treatments for bronchopulmonary dysplasia focus on supportive care like oxygen therapy or mechanical ventilation. Unlike these standard approaches, sildenafil is unique because it targets pulmonary hypertension, a condition that often accompanies bronchopulmonary dysplasia, by relaxing blood vessels in the lungs to improve blood flow. Researchers are excited about sildenafil because it has the potential to directly address a root cause of the condition rather than just alleviating symptoms. Additionally, sildenafil's different dosages and delivery methods (both IV and enteral) allow for flexibility in treating varying severities of the condition. This innovative approach offers hope for more effective management of bronchopulmonary dysplasia in infants.
What evidence suggests that sildenafil might be an effective treatment for bronchopulmonary dysplasia?
This trial will compare sildenafil with a placebo to evaluate its effects on babies with severe bronchopulmonary dysplasia (BPD), a lung condition. Research has shown that sildenafil might improve breathing and lower pressure in the lung arteries. One study suggested it might reduce lung damage and support healthy lung blood vessels. Early experiences indicate that sildenafil could be effective for babies with BPD and high blood pressure in the lungs. However, more detailed studies are needed to confirm these benefits.13678
Who Is on the Research Team?
Matt Laughon, MD
Principal Investigator
UNC
Christoph Hornik, MD
Principal Investigator
Duke UMC
Are You a Good Fit for This Trial?
This trial is for premature infants in NICUs with severe bronchopulmonary dysplasia. Eligible babies are 32-44 weeks postmenstrual age, born before 29 weeks gestation, and need respiratory support. Parents must consent to participate. Infants can't join if they've had certain heart defects, sickle cell disease, recent vasopressors or nitric oxide use, high liver enzymes, or known sildenafil allergy.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive sildenafil or placebo every 8 hours for 28 days
Weaning
Participants are weaned off the study drug following the treatment period
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- Placebo
- Sildenafil
Trial Overview
The study tests the safety of Sildenafil compared to a placebo in helping premature infants with severe lung problems (BPD). It's randomized (babies are put into groups by chance), double-masked (neither doctors nor parents know which treatment the baby gets), and dose levels increase over time.
How Is the Trial Designed?
6
Treatment groups
Active Control
Placebo Group
Sildenafil (0.5 mg/kg IV or 1 mg/kg enteral) every 8 hours for 28 days
Sildenafil (2 mg/kg IV or 4 mg/kg enteral) every 8 hours for 28 days
Sildenafil (1 mg/kg IV or 2 mg/kg enteral) every 8 hours for 28 days
Placebo (IV or enteral) every 8 hours for 28 days
Placebo (IV or enteral) every 8 hours for 28 days
Placebo (IV or enteral) every 8 hours for 28 days
Sildenafil is already approved in United States, European Union, Canada, Japan for the following indications:
- Erectile dysfunction
- Pulmonary arterial hypertension
- Erectile dysfunction
- Pulmonary arterial hypertension
- Pulmonary arterial hypertension
- Erectile dysfunction
- Pulmonary arterial hypertension
- Erectile dysfunction
- Pulmonary arterial hypertension
Find a Clinic Near You
Who Is Running the Clinical Trial?
Christoph Hornik
Lead Sponsor
Christoph P Hornik, MD MPH
Lead Sponsor
National Heart, Lung, and Blood Institute (NHLBI)
Collaborator
University of North Carolina, Chapel Hill
Collaborator
Citations
Sildenafil for bronchopulmonary dysplasia and pulmonary ...
Our results show that sildenafil appears to be associated with improvements in estimates of PAP and respiratory scores; however, sildenafil showed no effect on ...
Efficacy of Sildenafil in Infants with Bronchopulmonary ...
We also found improvement in clinical outcomes at 14 weeks following sildenafil treatment initiation. However, since there is no control arm in ...
Safety of sildenafil in premature infants at risk ...
Sildenafil reduces lung injury and preserves normal vasculature in preclinical models, and improves outcomes in children with pulmonary hypertension, and thus ...
Safety of sildenafil in premature infants with severe ...
Sildenafil may therefore prevent the development of pulmonary hypertension associated with lung disease of prematurity by reducing pulmonary ...
5.
publications.aap.org
publications.aap.org/hospitalpediatrics/article/6/1/27/26295/Sildenafil-Treatment-of-Infants-WithSildenafil Treatment of Infants With Bronchopulmonary ...
Early clinical experience indicates that sildenafil treatment may be efficacious for infants with BPD-PH, with evidence suggesting that ...
Safety of Sildenafil in Premature Infants With Severe ...
This is a multicenter, randomized, placebo-controlled, sequential dose-escalating, double-masked, safety study of sildenafil in premature infants.
More safety data: what about efficacy of sildenafil?
The study did not show an increased risk for severe retinopathy of prematurity for very low birth weight infants on sildenafil.
Safety of sildenafil in premature infants with severe ...
The FDA does not recommend sildenafil for PH in children 1 to 17 years of age and there are no recommendations regarding its use in neonates and ...
Unbiased Results
We believe in providing patients with all the options.
Your Data Stays Your Data
We only share your information with the clinical trials you're trying to access.
Verified Trials Only
All of our trials are run by licensed doctors, researchers, and healthcare companies.