NCT05201144RecruitingPhase 2
A Trial of Phosphodiesterase-5 Inhibitor in Neonatal Congenital Diaphragmatic Hernia (TOP-CDH)
Source: ClinicalTrials.gov · Last updated 4 Apr 2025 · Not updated for over 12 months - ask the site whether it is still open
Brief summary (registry, in English)
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.
- Status (registry)
- Recruiting
- Phase (registry)
- Phase 2
- Study type
- Interventional
- Enrollment
- 40 (estimated)
- First submitted
- 7 Jan 2022
- Start date
- 15 Sept 2022
- Primary completion date
- 31 Mar 2026
- Lead sponsor
- University of Utah
- NCT number
- NCT05201144
What you commit to
- Study type: Interventional · Phase 2
- Planned participants: 40
- From 15 Sept 2022 to the primary completion date, 31 Mar 2026
- 1 site in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- Infants with CDH who do not undergo surgical repair
- Has concurrent severe congenital heart defect that requires neonatal cardiac repair
- Concurrent therapy with fluconazole at time of study drug initiation
- Receiving extracorporeal membrane oxygenation (ECMO) at the time of the study
Other illness
- Has a documented sildenafil allergy
Other
- Does not have an echocardiogram 48-72 hours post-repair
- Has LVEI \< 1.4 on above echocardiogram
- Inability to obtain parental consent within 24 hours of the echocardiogram
Who the study is looking for
- 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
Sex: All · Does not accept healthy volunteers
Sites (1)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Primary Children's Hospital Coordinator of this site? Confirm its status | Salt Lake City | United States | Recruiting |
This page is information, not medical advice. Discuss any trial with your doctor.