Stem Cells in Umbilical Blood Infusion for CP

Study Overview
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Brief Summary
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This study will investigate the safety of single dose intravenous infusion of cord blood cells which were cryopreserved after the birth of a brother or sister to a child with cerebral palsy.

Detailed Description
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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. 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. Therefore, this trial will study the safety of infusing matched sibling cord blood cells to children with cerebral palsy.

Official Title
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Safety Study of Sibling Cord Blood Cell Infusion to Children With Cerebral Palsy

Conditions
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Cerebral Palsy

Intervention / Treatment
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* Biological: Matched sibling donor cord blood cell infusion


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Diagnosis of any type of CP CP of any severity A record of sibling CBU in storage at a TGA accredited private cord blood bank Ability to travel to one of the trial centres Ability to participate in assessments Informed consent by parent/guardian Exclusion Criteria: presence of progressive neurological disease known genetic disorder known brain dysplasia immune system disorder or immune deficiency syndrome infectious disease markers showing up on virology screen evidence of cord blood unit contamination, or fewer than 10^7 cells/kg body mass ventilator support ill health, or if the participant's medical condition does not allow safe travel previous cell therapy Botulinum toxin A within 3 months before or after infusion surgery within 3 months before or after infusion cannot obtain parent/guardian consent

Ages Eligible for Study
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Minimum Age: 1 Year
Maximum Age: 16 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Cord blood infusion<br>Matched sibling donor cord blood cell infusion | Biological: Matched sibling donor cord blood cell infusion<br>* Single dose intravenous infusion of 12/12 HLA matched sibling donor cord blood cells (>1x10^7 cells/kg)<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number of participants with abnormal clinical assessment and/or laboratory values | Safety | 12 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Preliminary analysis of change in gross motor function | Gross Motor Function Measure (GMFM-66) | Baseline, 3 months | 
| Preliminary analysis of change in gross motor function | Gross Motor Function Measure (GMFM-66) | Baseline, 12 months | 
| Preliminary analysis of change in fine motor function | Quality of Upper Extremity Skills Test (QUEST) | Baseline, 3 months | 
| Preliminary analysis of change in fine motor function | Quality of Upper Extremity Skills Test (QUEST) | Baseline, 12 months | 
| Preliminary analysis of change in cognitive function | Age appropriate cognitive testing: Bayley Scales of Infant and Toddler Development (BSID-III), Wechsler Preschool Primary Scale of Intelligence (WPPSI-IV), Wechsler Intelligence Scale for Children (WISC-V). Change from baseline will be compared using z-scores across measures. | Baseline, 12 months | 
| Preliminary analysis of change in quality of life | Cerebral Palsy Quality of Life (CP-QoL-CHILD) | Baseline, 3 months | 
| Preliminary analysis of change in quality of life | Cerebral Palsy Quality of Life (CP-QoL-CHILD) | Baseline,12 months | 
| Digital PCR analysis of peripheral blood cellular DNA to determine the fraction of donor DNA in circulation | Chimerism study to detect the longevity of infused cells | 3 months |