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Stem Cells in Umbilical Blood Infusion for CP (SCUBI-CP)

This study is ongoing, but not recruiting participants.
Sponsor:
ClinicalTrials.gov Identifier:
NCT03087110
First Posted: March 22, 2017
Last Update Posted: July 6, 2017
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
Collaborators:
The Royal Children's Hospital Melbourne
Children's Health Queensland
Monash Health
Sydney Children's Hospitals Network
Cerebral Palsy Alliance
Information provided by (Responsible Party):
Murdoch Childrens Research Institute
  Purpose
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.

Condition Intervention Phase
Cerebral Palsy Biological: Matched sibling donor cord blood cell infusion Phase 1

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Safety Study of Sibling Cord Blood Cell Infusion to Children With Cerebral Palsy

Resource links provided by NLM:


Further study details as provided by Murdoch Childrens Research Institute:

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


Secondary Outcome Measures:
  • 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


Enrollment: 12
Actual Study Start Date: March 2016
Estimated Study Completion Date: September 2019
Estimated Primary Completion Date: September 2019 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Cord blood infusion
Matched sibling donor cord blood cell infusion
Biological: Matched sibling donor cord blood cell infusion
Single dose intravenous infusion of 12/12 HLA matched sibling donor cord blood cells (>1x10^7 cells/kg)

Detailed Description:

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.

  Eligibility

Information from the National Library of Medicine

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Ages Eligible for Study:   1 Year to 16 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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
  Contacts and Locations
Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03087110


Locations
Australia, Queensland
Lady Cilento Children's Hospital
Brisbane, Queensland, Australia, 4101
Australia, Victoria
The Royal Children's Hospital
Melbourne, Victoria, Australia, 3052
Sponsors and Collaborators
Murdoch Childrens Research Institute
The Royal Children's Hospital Melbourne
Children's Health Queensland
Monash Health
Sydney Children's Hospitals Network
Cerebral Palsy Alliance
Investigators
Principal Investigator: Dinah Reddihough, MBChB, MD Group leader
  More Information

Responsible Party: Murdoch Childrens Research Institute
ClinicalTrials.gov Identifier: NCT03087110     History of Changes
Other Study ID Numbers: HREC/14/RCHM/38; RCH ID 34210
U1111-1179-9253 ( Other Identifier: WHO Universal Trial Number )
First Submitted: February 26, 2017
First Posted: March 22, 2017
Last Update Posted: July 6, 2017
Last Verified: July 2017
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:
Cerebral Palsy
Brain Damage, Chronic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases