Using melatonin to protect the brain in newborns with HIE during cooling treatment

Melatonin as a Neuroprotective Therapy in Neonates With HIE Undergoing Hypothermia

Early Phase 1 Interventional University of Florida · NCT02621944

This study is testing whether melatonin can help protect the brains of newborns with Hypoxic-Ischemic Encephalopathy during cooling treatment to see if it improves their long-term development.

Quick facts

PhaseEarly Phase 1
Study typeInterventional
Enrollment70 (estimated)
AgesN/A to 6 Hours
SexAll
SponsorUniversity of Florida Academic / other
Locations2 sites (Gainesville, Florida and 1 other locations)
Trial IDNCT02621944 on ClinicalTrials.gov

What this trial studies

This clinical trial investigates the neuroprotective effects of melatonin in neonates diagnosed with Hypoxic-Ischemic Encephalopathy (HIE) who are undergoing therapeutic hypothermia. The study will enroll thirty infants and utilize a dose escalation approach, starting with a low dose of melatonin and increasing it to determine the safest and most effective dosage. Researchers will monitor serum melatonin levels, adverse events, and long-term developmental outcomes at 18-22 months of age, while also assessing the impact of melatonin on inflammation and oxidative stress related to brain injury. The goal is to identify a potential adjunct therapy that could improve neurological outcomes in affected infants.

Who should consider this trial

Good fit: Ideal candidates for this study are infants over 36 weeks gestation with signs of encephalopathy and who have undergone therapeutic hypothermia within 6 hours of birth.

Not a fit: Patients with suspected metabolic disorders, meningitis, or significant congenital anomalies may not benefit from this study.

Why it matters

Potential benefit: If successful, this study could lead to improved neurological outcomes for neonates suffering from HIE.

How similar studies have performed: While the use of melatonin in this context is novel, other studies have explored its neuroprotective properties, suggesting potential for success.

Eligibility criteria

Show full inclusion / exclusion criteria
Inclusion Criteria:

* Eligible infants are \>36 0/7th weeks gestation,
* pH (cord or neonatal) \<7.0,
* base deficit \>16 mEq/L,
* no available blood gas,
* a cord blood/first hour of life blood gas with pH \> 7.0 and \< 7.15,
* base deficit between 10 and 15.9 mEq/L,
* infants must have a history of an acute perinatal event,
* either a 10-minute Apgar \< 5 or a continued need for ventilation,
* All infants must have signs of encephalopathy within 6 hours of age using the modified Sarnat scoring system,
* neonates cooled within 6 hours of birth will be included in the study.

Exclusion Criteria:

* suspected inborn errors of metabolism (elevated ammonia) and hypoglycemia,
* clinical signs and symptoms consistent with meningitis detected upon sepsis evaluation,
* a diagnosis of congenital abdominal surgical problems along with multiple congenital anomalies and/or chromosomal abnormalities.

Where this trial is running

Gainesville, Florida and 1 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
Conditions Hypoxic Ischemic Encephalopathy
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.