NEOSTeM (NEOnatal STroke Mesenchymal Stem Cell) Trial: Assessing the Safety of Mesenchymal Stem Cells for Babies with Stroke
A Phase I Dose Escalation Safety Trial of Repeated Mesenchymal Stem Cell Treatment for Neonatal Stroke
Sydney Children's Hospital Network
12 participants
May 3, 2024
Interventional
Conditions
Summary
Neonatal stroke occurs in up to 1 in 2,500 live births and can be defined as disturbances in the blood supply to the brain and includes an acute ischemic event. Up to 60% of babies diagnosed with stroke will have atypical neurological development and brain injury, including epilepsy and cerebral palsy. Current standard of care focusses on diagnosis and prompt stabilisation of symptoms such as seizures. There are no specific interventions to protect the developing brain or prevent the progression of injury. However, stem cells, particularly mesenchymal stem cells (MSCs), are a potential treatment for neonatal stroke, where early therapy may minimise lifelong impairment. This Phase I dose escalation trial assesses the safety of early and repeated systemic (intravenous) administration of MSCs for neonatal stroke at both 2- and 5-million cells/kg body weight.
Eligibility
Inclusion Criteria4
- Term/near-term infant greater than or equal to 35 weeks’ gestation
- Diagnosis of a stroke within the first 21 days of life, characterized by predominantly unilateral ischemic lesion within the territory of the middle cerebral artery, as confirmed by MRI
- Infant available for treatment within 72 hours from stroke confirmation
- Informed consent obtained from the parent
Exclusion Criteria8
- Born <35 weeks’ gestation;
- Stroke diagnosis made after 21 days of life;
- Any proven or suspected congenital anomaly; chromosomal disorder or metabolic disorder;
- Presence of an infection of the central nervous system;
- Requiring or likely to require Extracorporeal Membrane Oxygenation (ECMO; contraindicated for cell therapy) life support;
- Know positive test for HIV 1, HIV 2, hepatitis B virus, hepatitis C virus, or any other infection which in the opinion of the Investigator is likely to impact on the ability of the patient to participate in the study;
- Venous thromboembolism currently receiving/requiring anti-coagulation;
- No realistic prospect of survival (e.g. severe brain injury), at the discretion of the attending physician.
Interested in this trial?
Get notified about updates and connect with the research team.
Interventions
Allogenic mesenchymal stem/stromal cells (MSCs). Supplied and formulated in cryoprotectant medium. The product is cryopreserved in the vapour phase of liquid nitrogen (cells prepared/manufactured years in advance). Prior to intravenous administration, the product is rapid-thawed at the clinical site in a neonatal intensive care setting. Participants are treated in this dose-escalations study at 2- (low dose) or 5-million (high dose) cells/kg body weight. Enrollment across the two groups occurs sequentially. Treatment is administered twice. The first dose will commence within 72 hours of stroke confirmation via MRI, with the second 48-72 hours later. Intervention administration, dosing and adherence are recorded in site logs.
Locations(2)
View Full Details on ANZCTR
For the most up-to-date information, visit the official listing.
ACTRN12624000145505