Sildenafil for Neonatal Asphyxia

(SANE-02 Trial)

Age: < 18

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 1

Waitlist Available

Sponsor: McGill University Health Centre/Research Institute of the McGill University Health Centre

No Placebo Group

Trial Summary

What is the purpose of this trial?

The investigators will determine the maximum tolerable dose of sildenafil and establish the pharmacokinetic and pharmacodynamic profile of sildenafil in human asphyxiated neonates treated with hypothermia. They will use a 3+3 design to escalate the sildenafil dose up to 6 mg/kg/day (3mg/kg/dose q12h) in asphyxiated neonates demonstrating brain injury despite hypothermia treatment and assess whether we observe any beneficial effects of sildenafil on their brain and cardiopulmonary hemodynamics, without causing serious adverse events

Research Team

Eligibility Criteria

This trial is for newborns who are at least 36 weeks old, weigh over 1800 grams, and have suffered from birth asphyxia leading to brain injury. They must show signs of distress like low Apgar scores or poor blood gas levels and have evidence of brain injury on an MRI. Babies with complex heart disease, genetic syndromes, or certain types of bleeding in the brain can't participate.

Inclusion Criteria

The baby showed signs of distress after birth, such as low Apgar score or abnormal blood gas results.

My newborn has signs of severe brain injury confirmed by tests.

A brain MRI showed signs of injury within the first 2 days of life.

Exclusion Criteria

My newborn has a genetic syndrome.

My newborn has brain malformations.

My newborn had a brain bleed detected by MRI on their second day.

Treatment Details

Interventions

• Sildenafil Citrate (Phosphodiesterase-5 (PDE-5) Inhibitor)

Trial Overview The study is testing different doses of Sildenafil Citrate up to a maximum of 6 mg/kg/day in newborns with brain injuries from asphyxia despite being cooled (hypothermia treatment). Researchers want to find the highest dose babies can tolerate without serious side effects and see if it helps their brains and hearts.

Participant Groups

Group I: Sildenafil Experimental Treatment 1 Intervention

Who Is Running the Clinical Trial?

McGill University Health Centre/Research Institute of the McGill University Health Centre

Lead Sponsor

Trials

476

Recruited

170,000+

Dr. Lucie Opatrny

McGill University Health Centre/Research Institute of the McGill University Health Centre

President and Executive Director since 2023

MDCM and Master's in Epidemiology and Biostatistics from McGill University, Master's in Healthcare Management and Diploma in Advanced Negotiation from Harvard University

Dr. Patrizia Cavazzoni

McGill University Health Centre/Research Institute of the McGill University Health Centre

Chief Medical Officer

MD from McGill University, residency in Psychiatry and fellowship in Mood Disorders at the University of Ottawa