Ongoing Clinical Trials for Hypoxic-Ischaemic Encephalopathy
Currently, there are 4 clinical trials investigating new treatments for hypoxic-ischaemic encephalopathy in newborns. These studies are exploring innovative approaches including stem cell therapy, pain medication management, melatonin treatment, and blood flow-enhancing medications, all used alongside standard cooling therapy. The trials are taking place in France, Italy, and Ireland.
Clinical trial locations
- France
- Ireland
- Italy
Study of Autologous Cord Blood Stem Cells Treatment for Newborns with Hypoxic-Ischemic Encephalopathy
This trial is investigating a novel regenerative therapy using a baby’s own umbilical cord blood stem cells to treat brain injury caused by oxygen deprivation at birth. The study focuses on determining whether this treatment is safe and feasible for newborns.
Main inclusion criteria: The trial accepts newborns who show signs of brain dysfunction within 6 hours after birth, with a severity score of 2 or higher on the Sarnat scale. Babies must be receiving standard cooling therapy and may need to have abnormal brain activity recordings. The mother must test negative for HIV, HTLV, hepatitis B and C, and syphilis. Parents must provide written consent for participation.
Main exclusion criteria: The study excludes newborns with severe congenital abnormalities, confirmed genetic disorders, metabolic disorders, or severe organ failure. Babies who did not have their cord blood collected at birth, those with active bleeding or severe blood clotting disorders, and those with severe systemic infections cannot participate. Infants who have received other experimental treatments or cannot receive the full course of cooling treatment are also excluded.
Trial focus: The main goal is to assess the safety and feasibility of using autologous cord blood stem cells as a treatment for brain injury in newborns. The stem cells are collected from the baby’s own umbilical cord at birth, processed, and then given back through intravenous infusion alongside standard cooling therapy. Children will be monitored for up to two years after treatment to evaluate their development and any effects of the therapy.
Investigational treatment: The trial uses autologous cord blood stem cells collected from the baby’s umbilical cord blood at birth. These stem cells have the potential to repair damaged tissue and promote healing. The cells are processed and administered back to the baby at a dose of 50 million cells per kilogram of body weight through intravenous infusion.
Study of Fentanyl Continuous Infusion in Newborns with Hypoxic Ischemic Brain Injury During Cooling Therapy
This research examines how the pain medication fentanyl works in newborns with brain injury who are receiving cooling therapy. The study aims to understand how these infants’ bodies process this medication during their treatment.
Main inclusion criteria: Newborns must require cooling treatment for oxygen deprivation at birth and be born at or after 35 weeks of pregnancy with a birth weight over 1.8 kg. They must have at least one condition indicating severe oxygen deprivation, such as a low Apgar score at 5 minutes, ongoing resuscitation needs at 10 minutes, or abnormal blood test results within the first hour. Babies must show signs of brain dysfunction, have moderate to severe brain activity abnormalities or seizures, and start cooling treatment within 6 hours of birth. Parents must provide written consent.
Main exclusion criteria: The study excludes infants who do not meet criteria for cooling treatment or are not receiving fentanyl for sedation and pain control. Babies without a history of oxygen deprivation at birth, those receiving other pain medications besides fentanyl, and those with severe birth defects or active bleeding disorders cannot participate. Infants with severe liver or kidney dysfunction or those participating in other trials are also excluded.
Trial focus: The primary goal is to understand how fentanyl moves through the body of newborns receiving cooling therapy. The medication is given first as a single dose of 2 micrograms per kilogram, followed by continuous administration at 1 microgram per kilogram per hour. Blood samples are collected at specific times to measure medication levels, and doctors carefully monitor for any side effects.
Investigational treatment: Fentanyl is a potent opioid pain medication used to manage pain and provide sedation. In this trial, it is administered as a continuous infusion to help keep babies comfortable during their cooling treatment and reduce stress.
Study of intravenous melatonin safety and dosing in newborns with moderate to severe hypoxic-ischaemic encephalopathy receiving therapeutic hypothermia treatment
This trial tests a new intravenous form of melatonin given alongside standard cooling treatment to determine if it is safe and to find the appropriate dose for future studies.
Main inclusion criteria: Babies must be admitted to the Neonatal Intensive Care Unit with moderate-severe brain injury and qualify for cooling treatment according to hospital guidelines. They must be born at or after 36 completed weeks of pregnancy and be clinically stable before receiving the study medication, with proper IV access, normal blood pressure, controlled seizures, and stable vital signs. Blood pressure monitoring through an arterial line must be in place. Parents or legal guardians must provide informed consent.
Main exclusion criteria: The study excludes infants older than 6 hours of life, those with major birth defects or genetic disorders, and babies with severe bleeding, liver, or kidney problems. Newborns with severe blood pressure instability, known allergies to melatonin, or those who have received other experimental treatments cannot participate. Infants with conditions that would interfere with study procedures are also excluded.
Trial focus: The study aims to determine if giving high doses of intravenous melatonin is safe for newborns with moderate to severe brain injury and to identify the most appropriate dose. The medical team monitors brain activity using specialized equipment, takes blood samples to measure drug levels, performs MRI scans between days 4 and 10, and follows the babies’ development using various assessment methods.
Investigational treatment: The trial uses a novel intravenous formulation of melatonin, a hormone that naturally regulates sleep-wake cycles. In this study, it is being tested as a neuroprotective agent to help reduce brain damage in newborns who experienced oxygen deprivation at birth. The medication works as a powerful antioxidant to protect brain cells and is given alongside cooling treatment.
Study on Sildenafil and Hypothermia for Newborns with Hypoxic-Ischemic Encephalopathy
This clinical trial explores whether combining sildenafil with controlled cooling is more effective than cooling alone in improving survival without brain injuries in newborns.
Main inclusion criteria: Newborns must be born at or after 36 weeks of pregnancy and diagnosed with brain injury caused by oxygen deprivation. They must be treated with cooling therapy, maintaining a body temperature of 33.5 ± 0.5 degrees Celsius. The experimental treatment must start more than 1 hour after cooling begins and within the first 12 hours of life. The newborn must have social security coverage, and one parent or legal guardian must provide written consent.
Main exclusion criteria: The study excludes patients with conditions other than oxygen-related brain injury, those who are not newborns born after 36 weeks of pregnancy, and those not undergoing cooling treatment. Patients who are not eligible for intravenous sildenafil treatment cannot participate.
Trial focus: The study is conducted in two steps. The first examines how the body processes sildenafil in affected newborns. The second step compares outcomes of babies receiving the combination treatment to those receiving a placebo, focusing on survival without brain lesions visible on MRI scans. Throughout the study, researchers monitor brain activity, heart function, and assess neurodevelopmental outcomes over a 2-year follow-up period.
Investigational treatment: Sildenafil is a medication that relaxes blood vessels to improve blood flow and oxygen delivery to tissues. It is classified as a phosphodiesterase type 5 inhibitor. In this trial, sildenafil is given intravenously alongside controlled cooling to see if it can improve survival without brain damage by enhancing blood flow to the injured brain.
Summary
The current landscape of clinical trials for hypoxic-ischaemic encephalopathy demonstrates a diverse approach to treating this serious newborn condition. All four trials use therapeutic hypothermia as a foundation, with each investigating different additional treatments to potentially improve outcomes.
France is conducting two trials, exploring both regenerative medicine through cord blood stem cells and blood flow enhancement with sildenafil. Italy is focusing on understanding pain medication management during cooling therapy, while Ireland is investigating melatonin as a neuroprotective agent. This geographic distribution reflects international collaboration in addressing this challenging condition.
The trials represent different therapeutic strategies: regenerative medicine, pharmacokinetic optimization, neuroprotection, and blood flow enhancement. Each study includes comprehensive monitoring and follow-up assessments, with some tracking development for up to two years. These trials collectively aim to improve survival rates and reduce long-term neurological complications in affected newborns.