None listed
Conditions
Brief summary
Cerebral palsy (CP) is the most common physical disability of childhood, affecting 2 per 1000 live births across the world. CP describes permanent non-progressive motor disorders arising from damage to the developing brain. CP is often associated with epilepsy, difficulties in speech, sight, hearing, sensation, perception, behaviour or cognition. There is no cure for CP. Preclinical studies of different types of stem cells in models of acute brain injury similar to CP have shown significant functional improvement. The variety of stem cells available in umbilical cord blood (UCB), an ethically uncomplicated source of stem cells, has led to a focus on UCB stem cell therapy as a quick-to-clinic option. Previous studies indicate that autologous or unrelated donor UCBC infusion is safe and feasible for children with CP, and may lead to improved motor functioning, but there is no information about the safety and effects of matched sibling cord blood available. Therefore, this trial will study the safety of infusing matched sibling cord blood cells to children with cerebral palsy. Additionally, we will assess how long the cells remain within the recipient through sensitive chimerism assays, as we hypothesise that the cells may be rejected within 24 hours of infusion. Finally, we will trial the use of a range of outcome measures in this context.
Interventions
Single intravenous infusion of > 10 million cryopreserved matched sibling cord blood cells. Cells must be in storage at -196degC, with no bacterial contamination, in a TGA accredited cord blood bank.
Sponsors
Murdoch Childrens Research Institute
Study design
Allocation
Non-randomised trial
Intervention model
Single group
Primary purpose
Treatment
Masking
Open (masking not used)
Eligibility
Sex/Gender
All
Age
1 Years to 16 Years
Healthy volunteers
No
Inclusion criteria
1. Diagnosis of any type of CP 2. CP of any severity 3. A record of sibling CBU in storage at a TGA accredited private cord blood bank 4. Ability to travel to one of the trial centres 5. Ability to participate in assessments 6. Informed consent by parents
Exclusion criteria
1. presence of progressive neurological disease 2. known genetic disorder 3. known brain dysplasia 4. immune system disorder or immune deficiency syndrome 5. infectious disease markers showing up on the virology screen 6. evidence of cord blood unit contamination, or fewer than 10^7 cells/kg body mass 7. ventilator support 8. ill health, or if the participant’s medical condition does not allow safe travel 9. previous cell therapy 10. Botulinum toxin A within 3 months before or after infusion 11. Surgery within 3 months before or after infusion 12. Cannot obtain parental consent
Outcome results
None listed