Clinical Trial: Stem Cells in Umbilical Blood Infusion for CP

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

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

Brief Summary: 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 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.

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.


Sponsor: Murdoch Childrens Research Institute

Current Primary Outcome: Number of participants with abnormal clinical assessment and/or laboratory values [ Time Frame: 12 months ]

Safety


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Preliminary analysis of change in gross motor function [ Time Frame: Baseline, 3 months ]
    Gross Motor Function Measure (GMFM-66)
  • Preliminary analysis of change in gross motor function [ Time Frame: Baseline, 12 months ]
    Gross Motor Function Measure (GMFM-66)
  • Preliminary analysis of change in fine motor function [ Time Frame: Baseline, 3 months ]
    Quality of Upper Extremity Skills Test (QUEST)
  • Preliminary analysis of change in fine motor function [ Time Frame: Baseline, 12 months ]
    Quality of Upper Extremity Skills Test (QUEST)
  • Preliminary analysis of change in cognitive function [ Time Frame: Baseline, 12 months ]
    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.
  • Preliminary analysis of change in quality of life [ Time Frame: Baseline, 3 months ]
    Cerebral Palsy Quality of Life (CP-QoL-CHILD)
  • Preliminary analysis of change in quality of life [ Time Frame: Baseline,12 months ]
    Cerebral Palsy Quality of Life (CP-QoL-CHILD)
  • Digital PCR analysis of peripheral blood cellular DNA to determine the fraction of donor DNA in circulation [ Time Frame: 3 months ]
    Chimerism study to detect the longevity of infused cells


Original Secondary Outcome: Same as current

Information By: Murdoch Childrens Research Institute

Dates:
Date Received: February 26, 2017
Date Started: March 2016
Date Completion: September 2019
Last Updated: March 15, 2017
Last Verified: March 2017