Using sildenafil to treat brain injury in newborns after asphyxia
Treatment of Neonatal Encephalopathy With Oral Sildenafil Suspension to Repair Brain Injury Secondary to Birth Asphyxia: A Randomized, Double-blind, Placebo-controlled Clinical Trial to Evaluate Safety and Efficacy (Phase II Study)
This study is testing if sildenafil can help newborns with brain injuries from lack of oxygen at birth recover better after treatment with cooling therapy.
Quick facts
| Phase | Phase 2 |
|---|---|
| Study type | Interventional |
| Enrollment | 60 (estimated) |
| Ages | 0 Minutes to 48 Hours |
| Sex | All |
| Sponsor | McGill University Health Centre/Research Institute of the McGill University Health Centre Academic / other |
| Locations | 2 sites (Montreal, Quebec and 1 other locations) |
| Trial ID | NCT06098833 on ClinicalTrials.gov |
What this trial studies
This clinical trial investigates the use of sildenafil, a drug commonly used for pulmonary hypertension, to repair brain injury in neonates suffering from hypoxic-ischemic encephalopathy (HIE) due to birth asphyxia. The study will enroll neonates who have undergone therapeutic hypothermia and will randomly assign them to receive either sildenafil or a placebo for seven days. The primary aim is to evaluate the efficacy of sildenafil in reducing brain injury as measured by MRI, while secondary aims include assessing the safety of the drug and its effects on cardiopulmonary hemodynamics. This multicenter, double-blind, placebo-controlled trial aims to provide critical insights into a potential new treatment for a condition with currently limited options.
Who should consider this trial
Good fit: Ideal candidates for this study are neonates with moderate to severe hypoxic-ischemic encephalopathy who have undergone therapeutic hypothermia.
Not a fit: Patients who do not meet the criteria for induced hypothermia or have severe comorbidities may not benefit from this study.
Why it matters
Potential benefit: If successful, this treatment could significantly improve brain recovery and outcomes for newborns affected by birth asphyxia.
How similar studies have performed: While sildenafil has shown promise in small studies for similar conditions, this specific application in neonates with HIE is novel and untested.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * Male and female neonates meeting the criteria for induced hypothermia: * Gestational age ≥36weeks and birth weight ≥1800g; * Evidence of fetal distress, i.e., history of an acute perinatal event, cord pH ≤7.0 or base deficit * 16 mEq/L; * Evidence of neonatal distress, such as an Apgar score ≤5 at 10 minutes, postnatal blood gas pH obtained within the first hour of life ≤ 7.0 or base deficit ≤ - 16 mEq/L, or a continued need for ventilation initiated at birth and continued for at least 10 minutes; * Evidence of moderate to severe neonatal encephalopathy by an abnormal neurological exam and/or an amplitude-integrated electroencephalogram (aEEG). They will receive whole-body cooling to an esophageal temperature of 33.5°C, initiated within the first 6 hours of life, continued for 72 hours, and then they will be slowly rewarmed using standard protocol. * Evidence of brain injury on a brain magnetic resonance imaging (MRI) performed on day 2 of life. Exclusion Criteria: * Neonates with complex congenital heart disease * Neonates with cerebral malformations * Neonates with genetic syndrome * Neonates with intraventricular and/or intraparenchymal hemorrhage on MRI performed on day 2 of life * Moribund infants not expected to survive
Where this trial is running
Montreal, Quebec and 1 other locations
- Montreal Children's Hospital — Montreal, Quebec, Canada (Recruiting)
- Centre Hospitalier Universitaire Sainte-Justine — Montreal, Quebec, Canada (Recruiting)
Study contacts
- Principal investigator: Pia Wintermark, MD — Research Institute of the McGill University Health Centre (The Institute)
- Study coordinator: Pia Wintermark, MD
- Email: pia.wintermark@mcgill.ca
- Phone: +1-514-412-4452
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.