ACUMEN
Research type
Research Study
Full title
Phase I Dose Escalation and Cohort Expansion study to affirm the safety of pharmacological doses of a novel formulation of intravenous melatonin in babies with hypoxic-ischaemic encephalopathy (HIE) to augment therapeutic hypothermia (HT) treatment; to reduce the incidence and severity of disability in babies with moderate-severe HIE.
IRAS ID
1011409
Contact name
Felicia Ikeji
Contact email
Sponsor organisation
The Comprehensive Clinical Trials Unit at University College London
ISRCTN Number
ISRCTN61218504
Research summary
The ACUMEN trial is a research study aimed at improving treatments for newborn babies with a condition called hypoxic-ischemic encephalopathy (HIE). HIE is a type of brain injury that occurs when a baby does not receive enough oxygen and blood flow around the time of birth. While cooling therapy (therapeutic hypothermia) is currently the standard treatment, many babies still experience long-term challenges, including developmental delays and learning difficulties.
This study will test the safety and potential benefits of a drug called melatonin. Melatonin, a naturally occurring hormone, has shown promise in protecting brain cells in preclinical studies. The drug will be tested in its intravenous (IV) form to see if it can reach effective levels in the blood safely and tolerably for newborns.
The study will involve up to 60 participants with HIE who have been admitted to NICU and recruited from hospitals in the UK, Republic of Ireland, and Australia. Participants must meet eligibility criteria to take part and will receive the drug alongside standard cooling therapy. The study drug will be given as an IV infusion over six doses (1 loading and 5 maintenance doses) over a 72h hour period. Babies will be closely monitored during their hospital stay to ensure their safety and evaluate the drug’s effects. Blood samples (for melatonin, ethanol, acetaldehyde (sentinel participants only) and biomarker analysis), MRI/MRS brain imaging, and brain activity monitoring will help researchers understand how the drug works and its impact.
This research is funded by the Medical Research Council (MRC) and supported by hospitals and universities across the three countries. Results from this trial will inform whether this formulation of melatonin could be used in future trials to improve outcomes for babies with HIE.
REC name
London - Central Research Ethics Committee
REC reference
25/LO/0170
Date of REC Opinion
11 Apr 2025
REC opinion
Further Information Favourable Opinion