Sildenafil for Diaphragmatic Hernia
What You Need to Know Before You Apply
What is the purpose of this trial?
Congenital diaphragmatic hernia (CDH) occurs in approximately 1 in 3000 US live births, similar to the incidence seen within the Utah Birth Defects cohort. The diaphragmatic defect compromises lung growth and alters pulmonary vascular development. This is reflected postnatally as respiratory failure, pulmonary hypertension (PH) and overall cardiopulmonary dysfunction, particularly post-repair. Currently, optimal management of post-repair PH remains poorly investigated. Sildenafil citrate is a highly selective phosphodiesterase-5 inhibitor that increases cGMP levels, leading to smooth muscle relaxation and an anti-proliferative effect within the pulmonary vasculature. It is used off-label for many neonatal PH disorders, including PH associated with bronchopulmonary dysplasia and idiopathic persistent PH. Most neonates with CDH born within the Mountain West referral basin are managed at a quaternary care center, Primary Children's Hospital (PCH). Of these neonates with PH, approximately 25% have been treated with off-label sildenafil. However, neither the PCH clinical care group nor others have developed/published a standardized approach for either initiating or discontinuing sildenafil therapy in this group of patients. Thus, the aim of this study is to assess the safety and effectiveness of sildenafil therapy for PH in neonates with CDH within the Utah cohort. Given the relatively short-term outcome and small sample size for this trial, the plan is to use this data to support a larger multicenter randomized trial targeting long-term cardiopulmonary outcomes of infants with CDH and post-repair PH.
Will I have to stop taking my current medications?
The trial does not specify if you need to stop taking your current medications, but you cannot participate if you are on fluconazole when starting the study drug.
Is sildenafil generally safe for humans?
How is the drug sildenafil unique in treating diaphragmatic hernia?
What data supports the effectiveness of the drug sildenafil for diaphragmatic hernia?
Research shows that sildenafil can help improve lung blood flow and reduce high blood pressure in the lungs (pulmonary hypertension) in infants with congenital diaphragmatic hernia. Studies in both humans and animals have demonstrated that sildenafil can stabilize and improve the condition by enhancing oxygenation and reducing the need for other treatments.56789
Are You a Good Fit for This Trial?
This trial is for newborns with a birth defect called congenital diaphragmatic hernia (CDH) who are in the NICU at Primary Children's Hospital. They must have had surgery to repair the hernia and show signs of pulmonary hypertension on an echocardiogram taken within 48-72 hours after surgery. Babies can't join if they didn't have surgery, need ECMO, or are allergic to sildenafil.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive sildenafil citrate 1mg/kg every 8 hours or placebo for up to 14 days
Follow-up
Participants are monitored for safety and effectiveness after treatment, including incidence of open-label sildenafil use and other outcomes
What Are the Treatments Tested in This Trial?
Interventions
- Placebo
- Sildenafil Oral Suspension
Sildenafil Oral Suspension is already approved in United States, European Union, Canada for the following indications:
- Erectile dysfunction
- Pulmonary arterial hypertension
- Pulmonary arterial hypertension
- Erectile dysfunction
- Pulmonary arterial hypertension
- Pulmonary arterial hypertension
- Erectile dysfunction
- Pulmonary arterial hypertension
- Pulmonary arterial hypertension
Find a Clinic Near You
Who Is Running the Clinical Trial?
University of Utah
Lead Sponsor