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

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.
ClinicalTrials.gov Identifier: NCT03087110
Recruitment Status : Active, not recruiting
First Posted : March 22, 2017
Last Update Posted : July 18, 2018
Sponsor:
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

Tracking Information
First Submitted Date  ICMJE February 26, 2017
First Posted Date  ICMJE March 22, 2017
Last Update Posted Date July 18, 2018
Actual Study Start Date  ICMJE March 2016
Estimated Primary Completion Date September 2019   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: March 15, 2017)
Number of participants with abnormal clinical assessment and/or laboratory values [ Time Frame: 12 months ]
Safety
Original Primary Outcome Measures  ICMJE Same as current
Change History Complete list of historical versions of study NCT03087110 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: March 15, 2017)
  • 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 Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Stem Cells in Umbilical Blood Infusion for CP
Official Title  ICMJE 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 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.

Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Study Design  ICMJE Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Cerebral Palsy
Intervention  ICMJE 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)
Study Arms  ICMJE Experimental: Cord blood infusion
Matched sibling donor cord blood cell infusion
Intervention: Biological: Matched sibling donor cord blood cell infusion
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Active, not recruiting
Actual Enrollment  ICMJE
 (submitted: March 15, 2017)
12
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE February 2020
Estimated Primary Completion Date September 2019   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 1 Year to 16 Years   (Child)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Australia
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03087110
Other Study ID Numbers  ICMJE HREC/14/RCHM/38; RCH ID 34210
U1111-1179-9253 ( Other Identifier: WHO Universal Trial Number )
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party Murdoch Childrens Research Institute
Study Sponsor  ICMJE Murdoch Childrens Research Institute
Collaborators  ICMJE
  • The Royal Children's Hospital Melbourne
  • Children's Health Queensland
  • Monash Health
  • Sydney Children's Hospitals Network
  • Cerebral Palsy Alliance
Investigators  ICMJE
Principal Investigator: Dinah Reddihough, MBChB, MD Group leader
PRS Account Murdoch Childrens Research Institute
Verification Date July 2018

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP