NCT05201144 A Trial of Phosphodiesterase-5 Inhibitor in Neonatal Congenital Diaphragmatic Hernia (TOP-CDH)
| NCT ID | NCT05201144 |
| Status | Recruiting |
| Phase | Phase 2 |
| Sponsor | University of Utah |
| Condition | Congenital Diaphragmatic Hernia |
| Study Type | INTERVENTIONAL |
| Enrollment | 40 participants |
| Start Date | 2022-09-15 |
| Primary Completion | 2026-03-31 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
In Phase 2, researchers evaluate early signs of effectiveness. You may be randomized to receive the active treatment or a comparator. Monitoring continues closely.
This trial targets 40 participants in total. It began in 2022-09-15 with a primary completion date of 2026-03-31.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief Summary
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.
Eligibility 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
Contact & Investigator
Frequently Asked Questions
Who can join the NCT05201144 clinical trial?
This trial is open to participants of all sexes, studying Congenital Diaphragmatic Hernia. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
What phase is the NCT05201144 trial and what does that mean for participants?
Phase 2 trials evaluate whether the treatment shows signs of effectiveness while continuing to monitor safety. More participants are enrolled than in Phase 1 to help refine the treatment protocol.
Is NCT05201144 currently recruiting?
Yes, NCT05201144 is actively recruiting participants. Contact the research team at carrie.rau@hsc.utah.edu for enrollment information.
Where is the NCT05201144 trial being conducted?
This trial is being conducted at Salt Lake City, United States.
Who is sponsoring the NCT05201144 clinical trial?
NCT05201144 is sponsored by University of Utah. The trial plans to enroll 40 participants.