ACUMEN: 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.
This study, called ACUMEN, is investigating a new form of melatonin given through a drip to babies who have experienced a type of brain injury called Hypoxic-Ischaemic Encephalopathy (HIE). These babies are already receiving cooling treatment, which helps protect their brain. The main goal of this early-stage study is to make sure the new melatonin treatment is safe at different doses. Researchers will carefully watch for any side effects and check how much melatonin is in the baby's blood. We also want to understand how the baby's body handles the medication. The hope is that this new treatment, alongside cooling, could potentially reduce long-term disabilities in these vulnerable babies. This is a very early step, focusing on safety before looking at how well it works.
At a glance
What is this study about?
When a baby is born, sometimes they don't get enough oxygen or blood flow to their brain. This can lead to a condition called Hypoxic-Ischaemic Encephalopathy, or HIE. HIE can cause brain damage, which might lead to long-term health problems like learning difficulties or physical disabilities. Currently, one of the main treatments for babies with HIE is 'therapeutic hypothermia,' which means gently cooling the baby's body to a lower temperature for a few days. This cooling helps protect the brain from further damage.
This study is looking at a new treatment called melatonin, which is a natural hormone that helps regulate sleep. In this study, melatonin is given through a drip (intravenously) to babies with HIE who are already receiving cooling treatment. The main aim of this first phase of the study is to check if this new way of giving melatonin is safe for these babies and to find the right amount (dose) to give. Researchers will closely monitor the babies for any side effects and measure how much melatonin is in their blood to ensure it's within a safe and helpful range. They will also check the alcohol levels in the blood, as a small amount of alcohol is used to help the melatonin dissolve.
This is a very early study, sometimes called a 'Phase I' study, which means it's one of the first times this specific melatonin treatment is being given to humans, specifically babies. If this study shows that the treatment is safe, it could open the door for future larger studies to see if melatonin, when used with cooling, can truly help to reduce the seriousness of long-term disabilities in babies affected by HIE. This research is an important step in trying to improve the outcomes for these babies.
Key takeaways
- This study is testing a new melatonin treatment for babies with brain injury (HIE).
- Its main aim is to check the safety of giving melatonin through a drip at different doses.
- Babies taking part will also receive standard cooling treatment.
- This is an early-stage (Phase I) study, focusing on safety before effectiveness.
- Close monitoring for side effects and medication levels will take place.
Who may be eligible?
This study is for babies who have been diagnosed with moderate to severe HIE shortly after birth. This means they have experienced a significant lack of oxygen or blood flow to their brain.
There are no specific age limits beyond being a newborn with this condition, and both baby boys and baby girls can take part. The medical team will assess each baby carefully to see if they meet all the specific criteria for joining the study.
- Is my baby a newborn?
- Has my baby been diagnosed with moderate or severe HIE?
- Is my baby currently receiving cooling treatment?
- Is my baby generally stable enough to participate in a research study?
This is a guide only — the research team will confirm whether you can take part.
What does participation involve?
If your baby is eligible and you agree for them to take part, they will receive the new melatonin treatment through a drip while also undergoing their standard cooling treatment. The study involves giving different doses of melatonin to different groups of babies. Doctors will take regular blood samples to check the melatonin levels and make sure they are safe, and to monitor for any alcohol in the blood that comes from the medication. Your baby will be very closely monitored by the medical team throughout the treatment period, which is planned for about 172 hours (just over a week) from the first dose until shortly after the last dose. There will also be brain scans (MRI) and continuous brain activity monitoring (aEEG/EEG) to help understand how the treatment might be working and to get ready for future studies.
Potential risks and benefits
Locations (1)
- —Ireland
Common questions
What is HIE?
HIE stands for Hypoxic-Ischaemic Encephalopathy. It's a type of brain injury that can happen to babies if they don't get enough oxygen or blood flow to their brain around the time of birth.
What is melatonin?
Melatonin is a natural hormone that our bodies produce, mainly known for helping us sleep. In this study, doctors are investigating if a man-made version of melatonin, given through a drip, could help protect baby's brains after HIE.
Why is this only Phase I?
Phase I means it's one of the first times this specific treatment is being given to babies. The main focus is to ensure it's safe and to find the best dose, before moving on to larger studies to see how well it works.
Will my baby still get cooling treatment?
Yes, all babies in this study will also receive the standard cooling treatment, which is currently the best available care for HIE.
How long will my baby be monitored?
Your baby will be very closely monitored for about 172 hours (just over a week) from the start of the treatment until after the final dose.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
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