Using Sildenafil to Treat Neonates with Congenital Diaphragmatic Hernia
A Trial of Phosphodiesterase-5 Inhibitor in Neonatal Congenital Diaphragmatic Hernia (TOP-CDH)
This study is testing if sildenafil can help newborns with congenital diaphragmatic hernia and high blood pressure in their lungs recover better after surgery.
Quick facts
| Phase | Phase 2 |
|---|---|
| Study type | Interventional |
| Enrollment | 40 (estimated) |
| Sex | All |
| Sponsor | University of Utah Academic / other |
| Locations | 1 site (Salt Lake City, Utah) |
| Trial ID | NCT05201144 on ClinicalTrials.gov |
What this trial studies
This clinical trial investigates the use of sildenafil citrate, a phosphodiesterase-5 inhibitor, in neonates diagnosed with congenital diaphragmatic hernia (CDH) who have undergone surgical repair. The study aims to assess the drug's effectiveness in managing pulmonary hypertension (PH) that often complicates recovery in these infants. Participants will receive either sildenafil or a placebo, and their responses will be monitored through echocardiograms to evaluate pulmonary vascular function. The trial is conducted at Primary Children's Hospital in Salt Lake City, Utah, focusing on improving outcomes for this vulnerable population.
Who should consider this trial
Good fit: Ideal candidates for this study are infants admitted to the NICU with a diagnosis of congenital diaphragmatic hernia who have undergone surgical repair and have specific echocardiogram results indicating pulmonary hypertension.
Not a fit: Patients who have not undergone surgical repair for congenital diaphragmatic hernia or have severe concurrent congenital heart defects may not benefit from this study.
Why it matters
Potential benefit: If successful, this treatment could significantly improve respiratory function and overall survival rates in neonates with congenital diaphragmatic hernia and associated pulmonary hypertension.
How similar studies have performed: While sildenafil has been used off-label for various neonatal pulmonary hypertension disorders, this specific application in post-surgical CDH management is novel and has not been extensively tested.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * Infants admitted to PCH NICU * Diagnosis of congenital diaphragmatic hernia (CDH) * Status post-surgical repair of diaphragmatic defect * Has an echocardiogram 48-72 hours after repair with left ventricular eccentricity index (LVEI) ≥ 1.4 * Parental consent obtained within 24 hours after the above echocardiogram Exclusion Criteria: * Infants with CDH who do not undergo surgical repair * Does not have an echocardiogram 48-72 hours post-repair * Has LVEI \< 1.4 on above echocardiogram * Has concurrent severe congenital heart defect that requires neonatal cardiac repair * Has a documented sildenafil allergy * Concurrent therapy with fluconazole at time of study drug initiation * Inability to obtain parental consent within 24 hours of the echocardiogram * Receiving extracorporeal membrane oxygenation (ECMO) at the time of the study
Where this trial is running
Salt Lake City, Utah
- Primary Children's Hospital — Salt Lake City, Utah, United States (Recruiting)
Study contacts
- Study coordinator: Carrie A Rau, RN
- Email: carrie.rau@hsc.utah.edu
- Phone: 801-213-3360
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.