A Phase 2 interventional study of Sildenafil and Ora-Blend in Neonatal Encephalopathy, sponsored by Pia Wintermark. Recruiting at 3 sites in 2 countries. Open to participants aged 0 Minutes to 48 Hours. Per ClinicalTrials.gov, last updated 2026-09-17.
Sponsored by Pia Wintermark · Phase 2, Interventional, and Treatment
Around the time of birth, some babies experience a condition called asphyxia, which means that their brain and other organs do not receive enough blood and/or oxygen to work properly. This life-threatening condition accounts for nearly 1 out of 4 deaths of all babies around the world, and often leads to severe brain damage, cerebral palsy, epilepsy, and trouble with learning and functioning in everyday life. At this time, no treatment is available to repair the brain damage caused by asphyxia. Excitingly, a drug called sildenafil (Viagra®) is already given safely to babies who suffer from increased blood pressure in their lungs' vessels. Recent studies using a laboratory model of asphyxia at birth suggest that sildenafil may also repair the brain damage caused by asphyxia. Similarly, recent small studies have shown that it is both feasible and safe to give sildenafil to human babies, who suffered from asphyxia at birth. These studies also highlight the first promising signs that sildenafil may improve how the brains of these babies work, which is consistent with the abovementioned laboratory studies.
On the basis of these previous researches, the investigators predict that sildenafil can repair the damage to a baby's brain. The investigators will test whether sildenafil can be safely given to a large group of human babies who suffer from asphyxia at birth, and will confirm whether sildenafil improves or not how their brains and hearts/lungs work.
This project will enable to determine whether sildenafil is a promising treatment for repairing brain damage in babies who suffer from asphyxia at birth. This project may also provide new solutions for these babies to improve their future life.
The investigators will enroll neonates with HIE treated with TH from NICUs in a multicentre, randomized, double-blind, placebo-controlled phase 2 clinical trial to evaluate the safety and efficacy of sildenafil to repair brain injury. Neonates with moderate-severe HIE on admission and with brain injury on a day-2 brain MRI (during TH) will be randomized to sildenafil or placebo (allocation 2:1) for 7 consecutive days.
Aim 1: Evaluate the efficacy of sildenafil to improve brain injury (primary outcome). The investigators will determine whether sildenafil reduces brain injury on a day-30 MRI compared to the baseline day-2 MRI.
Aim 2: Determine the safety of sildenafil (secondary outcome). The investigators will assess the safety of sildenafil by recording the incidence of adverse events.
Aim 3: Evaluate the efficacy of sildenafil to improve cardiopulmonary hemodynamics (secondary outcome). The investigators will determine whether sildenafil improves pulmonary pressure and right/left ventricular function on day 4 of life (after TH completion) compared to baseline day-2 measurements
106 studies on the registry are indexed under Asphyxia Neonatorum; 19 are open to participants now.
This study's planned enrollment of 60 is below the median of 173 across 73 interventional studies indexed under Asphyxia Neonatorum.
Browse Asphyxia Neonatorum studies →Pia Wintermark is the lead sponsor of 2 studies on the registry; 1 is open to participants now.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Evidence of fetal distress, i.e., history of an acute perinatal event, cord pH ≤7.0 or base deficit
Exclusion Criteria:
Sildenafil per os twice a day for seven consecutive days (from day 2 of life to day 9 of life) if brain injury on day 2 of life (dose 1=2mg/kg/dose, dose 2=2.5mg/kg/dose, and doses 3-14=3mg/kg/dose)
Drug: Sildenafil
Ora-Blend per os twice a day for seven consecutive days (from day 2 of life to day 9 of life) if brain injury on day 2 of life (dose 1=2mg/kg/dose, dose 2=2.5mg/kg/dose, and doses 3-14=3mg/kg/dose)
Drug: Ora-Blend
Sildenafil per os twice a day for seven consecutive days (from day 2 of life to day 9 of life) if brain injury on day 2 of life (dose 1=2mg/kg/dose, dose 2=2.5mg/kg/dose, and doses 3-14=3mg/kg/dose)
Ora-Blend per os twice a day for seven consecutive days (from day 2 of life to day 9 of life) if brain injury on day 2 of life (dose 1=2mg/kg/dose, dose 2=2.5mg/kg/dose, and doses 3-14=3mg/kg/dose)
Extent of brain injury
Primary outcome to explore efficacy (brain injury)
Time frame: Day 30 of life, compared to day 2 of life
Serious adverse events
Close monitoring for adverse events such as death, hypotension, persistent pulmonary hypertension, altered renal or hepatic function, etc to assess the safety of sildenafil
Time frame: Day 1 to 10 of life
Ejection fraction (EF) in % (reflecting left ventricular function) and tricuspid annular plane systolic excursion (TAPSE) in cm (reflecting right ventricular function)
Secondary outcome to explore efficacy (cardiopulmonary hemodynamics)
Time frame: Day 2 to 4 of life
Plan to share: No
Eligibility is decided by the study team. Share this record with your doctor or contact the team directly.
Contact study teamGet an email when the registry record changes — status, dates, results — or when someone posts here.
Sign in to followQuestions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.
Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.
Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.
Studies of the drug tested here: Sildenafil
Pia Wintermark