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

Part of paid clinical trials in Salt Lake City, Utah.

Sponsor
University of Utah
Study ID
NCT05201144
Phase
PHASE2
Status
Recruiting
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Conditions

  • Congenital Diaphragmatic Hernia
  • Pulmonary Hypertension

Eligibility Criteria

Sex
ALL
Age
N/A - N/A
Healthy Volunteers
Not accepted

Interventions

  • Sildenafil Oral Suspension — DRUG
    Sildenafil citrate is a highly selective PDE-5 inhibitor found in pulmonary vascular smooth muscle cells. Sildenafil acts by increasing cGMP levels in the nitric oxide pathway, leading to smooth muscle relaxation and an anti-proliferative effect within the pulmonary vasculature.
  • Placebo — OTHER
    Equal volume of placebo

Study Details

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.

Key Dates

Start date
Sep 15, 2022
Status verified
Jan 2025
Primary completion
Mar 31, 2026
Completion
Sep 30, 2026

Study Design

Enrollment
40 participants (estimated)
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT

Arms

  • Active Comparator: Sildenafil citrate
    Sildenafil citrate 1mg/kg every 8 hours (PO or NG) for up to 14 days
  • Placebo Comparator: Placebo
    Equivalent volume of Ora-sweet©/Ora-plus© every 8 hours (PO or NG) for up to 14 days

Primary Outcome Measure

Change in Left Ventricular Eccentricity Index (LVEI) on echocardiogram after 14 days of study treatment compared to baseline echocardiogram as compared to placebo. [ Time Frame: 14 days ]

Central Contacts

Locations (1)

FacilityCityStateZIPSite coordinators
Primary Children's HospitalSalt Lake CityUtah84113
Michelle Yang, MD
[email protected]
801-581-7052
Kimberlee Weaver-Lewis, RN
[email protected]
801-507-7675
Michelle Yang, MD (PRINCIPAL_INVESTIGATOR)

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Primary Children's Hospital· Salt Lake City, UT

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