Stem Cells in Umbilical Blood Infusion for CP

Murdoch Childrens Research Institute12 enrolled

Overview

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.

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.

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Cerebral Palsy

Interventions

Matched sibling donor cord blood cell infusionBIOLOGICAL

Single dose intravenous infusion of 12/12 HLA matched sibling donor cord blood cells (\>1x10\^7 cells/kg)

Eligibility

Sex: ALLMin age: 1 YearMax age: 16 Years

Medical Language ↔ Plain English

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 parent/guardian 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 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 parent/guardian consent

Locations (2)

Lady Cilento Children's Hospital

Brisbane, Queensland, Australia

The Royal Children's Hospital

Melbourne, Victoria, Australia

Outcomes

Primary Outcomes

Number of participants with abnormal clinical assessment and/or laboratory values

Safety

Time frame: 12 months

Secondary Outcomes

Preliminary analysis of change in gross motor function

Gross Motor Function Measure (GMFM-66)

Time frame: Baseline, 3 months

Preliminary analysis of change in gross motor function

Gross Motor Function Measure (GMFM-66)

Time frame: Baseline, 12 months

Preliminary analysis of change in fine motor function

Quality of Upper Extremity Skills Test (QUEST)

Time frame: Baseline, 3 months

Preliminary analysis of change in fine motor function

Quality of Upper Extremity Skills Test (QUEST)

Time frame: 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.

Time frame: Baseline, 12 months

Preliminary analysis of change in quality of life

Cerebral Palsy Quality of Life (CP-QoL-CHILD)

Time frame: Baseline, 3 months

Preliminary analysis of change in quality of life

Data from ClinicalTrials.gov

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