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RecruitingPhase 2

A Trial of Phosphodiesterase-5 Inhibitor in Neonatal Congenital Diaphragmatic Hernia (TOP-CDH)

Who can join

As published by the study on ClinicalTrials.gov. "Not stated" means the study hasn't said — ask the study team.

Age
No age limits stated
Sex
All sexes
Healthy volunteers
Not accepted
Compensation
Not stated

Other requirements are listed under Eligibility below. Only the study team can confirm whether you can join.

What to expect

No one can promise a trial will help you personally — that is what the research is trying to find out.

What participation involves

Not stated in the study description — ask the study team about visits and procedures.

Compensation
Not stated
  • Study-related exams and procedures are typically provided at no cost. This is how many trials work in general — confirm the details for this study with the study team.
  • Participants are monitored closely by the research team throughout the study.
  • Some participants may receive access to an investigational treatment, which is still being studied.
  • Your participation helps researchers learn whether this approach could help future patients.

About this study

In plain language

This study tests how safe and effective sildenafil is for treating high blood pressure in the lungs of newborns after surgery for congenital diaphragmatic hernia (CDH). CDH is a birth defect in the muscle between the chest and belly that can affect lung growth. The study is for babies treated at Primary Children's Hospital.

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.

Compensation is read from the study's own description and may be missing — always confirm with the study team.

Eligibility

Who can join

  • Admitted to the newborn intensive care unit at Primary Children's Hospital.
  • Has CDH, a birth defect in the muscle between the chest and belly.
  • Has had surgery to repair CDH.
  • Has a heart ultrasound 48–72 hours after surgery showing an LVEI of 1.4 or higher. LVEI measures the shape of a heart chamber.
  • A parent gives consent within 24 hours after that heart ultrasound.

Who can't join

  • Has CDH but does not have surgery to repair it.
  • Does not have a heart ultrasound 48–72 hours after surgery.
  • Has an LVEI below 1.4 on that heart ultrasound.
  • Has a severe heart birth defect that needs heart surgery as a newborn.
  • Has a recorded allergy to sildenafil.
  • Is taking fluconazole when the study medicine starts.
  • A parent's consent cannot be obtained within 24 hours after the heart ultrasound.
  • Is receiving ECMO, a machine treatment that supports the heart and lungs, at the time of the study.

Simplified for readability. The official criteria below are what the study team uses.

Official 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

Study sites (1)

UT