NCT03913221

Caffeine for Hypoxic-Ischemic Encephalopathy

Pharmacokinetics and Safety of Caffeine in Neonates With Hypoxic-Ischemic Encephalopathy

• Status: Completed
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 17 (actual)
• Sponsor: University of North Carolina, Chapel Hill · Academic / Other
• Sex: All
• Age: 24 Hours
• Healthy volunteers: Not accepted

Summary

Hypoxic-ischemic encephalopathy (HIE) due to perinatal asphyxia is common and often fatal. Therapeutic hypothermia reduces mortality and morbidity in infants with HIE. Even with the widespread use of therapeutic hypothermia, \~60% of infants with HIE die or have neurodevelopmental impairment. As a result, there is an urgent, unmet public health need to develop adjuvant therapies to improve survival and neurodevelopmental outcomes in this population. Caffeine may offer neuroprotection for infants with HIE by blocking adenosine receptors in the brain and reducing neuronal cell death. In animal models of HIE, caffeine reduces white matter brain injury. Drugs in the same class as caffeine (i.e., methylxanthines) have been shown to be protective against acute kidney injury in the setting of HIE. However, their safety and efficacy have not been studied in the setting of therapeutic hypothermia and their effect on neurological outcomes is not known. Since these drugs reduce injury to the kidney in infants with HIE, they may also reduce injury to the brain. This phase I study will evaluate the pharmacokinetics, safety, and preliminary effectiveness of caffeine as an adjuvant therapy to improve neurodevelopmental outcomes in infants with HIE.

Conditions

• Hypoxic-Ischemic Encephalopathy

Interventions

Type	Name	Description
DRUG	Caffeine Citrate 5 mg/kg	Loading dose of caffeine 20 mg/kg IV followed by two daily doses of 5 mg/kg IV.
DRUG	Caffeine Citrate 10 mg/kg	Loading dose of caffeine 20 mg/kg IV followed by two daily doses of 10 mg/kg IV.

Timeline

• Start date: 2019-08-14
• Primary completion: 2023-01-01
• Completion: 2024-12-31
• First posted: 2019-04-12
• Last updated: 2025-03-27
• Results posted: 2024-09-19

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study