Sildenafil to Repair Brain Injury Secondary to Birth Asphyxia

Summary

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

Timeline

Start

2019-09-19

Primary completion

2021-12-31

Completion

2024-12

Publications

• Wintermark P, Lapointe A, Altit G, Steinhorn R, Rampakakis E, Meid AD, Burhenne J, Bajraktari-Sylejmani G, Khairy M, Adamo MT, Gilbert G, Toffoli D, Zavalkoff S, Luu TM, Hailu E, Haefeli WE. Testing Higher Doses of Sildenafil to Repair Brain Injury Secondary to Birth Asphyxia: An Open-Label Dose-Finding Phase 1b Clinical Trial-Sildenafil Administration to Treat Neonatal Encephalopathy-Study 02. J Pediatr. 2025 Oct;285:114701. doi: 10.1016/j.jpeds.2025.114701. Epub 2025 Jun 23.

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Indications