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Completed

NCT Number: NCT03087110

Stem Cells in Umbilical Blood Infusion for CP

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.

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Key information

Age range

1 year–16 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1

Primary location

Lady Cilento Children's Hospital, Brisbane, Queensland, Australia

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About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Matched sibling donor cord blood cell infusion

Biological

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

Primary outcomes

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

    Time frame: 12 months

    Safety

Secondary outcomes

  1. Preliminary analysis of change in gross motor function

    Time frame: Baseline, 3 months

    Gross Motor Function Measure (GMFM-66)

  2. Preliminary analysis of change in gross motor function

    Time frame: Baseline, 12 months

    Gross Motor Function Measure (GMFM-66)

  3. Preliminary analysis of change in fine motor function

    Time frame: Baseline, 3 months

    Quality of Upper Extremity Skills Test (QUEST)

  4. Preliminary analysis of change in fine motor function

    Time frame: Baseline, 12 months

    Quality of Upper Extremity Skills Test (QUEST)

  5. 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.

  6. Preliminary analysis of change in quality of life

    Time frame: Baseline, 3 months

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

  7. Preliminary analysis of change in quality of life

    Time frame: Baseline,12 months

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

  8. 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

Sponsors and collaborators

Lead sponsor

Murdoch Childrens Research Institute

Other

Collaborators

  • Cerebral Palsy Alliance
  • Children's Health Queensland
  • Monash Health
  • Royal Children's Hospital
  • Sydney Children's Hospitals Network

Registry information

Official study title

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

Acronym: SCUBI-CP

Important dates

Study start
2016
Primary completion
2019
Study completion
2020
First posted
Mar 22, 2017
Registry last updated
Jan 20, 2021

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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