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Trial registered on ANZCTR
Registration number
ACTRN12616000403437
Ethics application status
Approved
Date submitted
10/03/2016
Date registered
30/03/2016
Date last updated
11/02/2021
Date data sharing statement initially provided
5/02/2019
Type of registration
Prospectively registered
Titles & IDs
Public title
Stem Cells in Umbilical Blood Infusion for Cerebral Palsy
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Scientific title
Safety study of sibling cord blood cell infusion to children with cerebral palsy
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Secondary ID [1]
288592
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ClinicalTrials.gov registry number: NCT03087110
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Universal Trial Number (UTN)
U1111-1179-9253
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Trial acronym
SCUBI-CP
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Cerebral palsy
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Condition category
Condition code
Neurological
297921
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0
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Other neurological disorders
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Single intravenous infusion of > 10 million cryopreserved matched sibling cord blood cells. Cells must be in storage at -196degC, with no bacterial contamination, in a TGA accredited cord blood bank.
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Intervention code [1]
293988
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Treatment: Other
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Comparator / control treatment
No control group
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Control group
Uncontrolled
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Outcomes
Primary outcome [1]
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Safety
1) Documentation of acute infusion reactions
2) Documentation of any instances of Graft-versus-Host Disease
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Assessment method [1]
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Timepoint [1]
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36 hours, 3 months, 12 months
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Secondary outcome [1]
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Preliminary analysis of change in gross motor function:
Gross Motor Function Measure (GMFM-66)
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Assessment method [1]
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Timepoint [1]
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3 months, 12 months
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Secondary outcome [2]
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Chimerism studies to detect the longevity of infused cells:
Copy number deletion analysis of peripheral blood cellular DNA to determine the fraction of donor DNA at each timepoint
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Assessment method [2]
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Timepoint [2]
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1, 4 and 24 hours, 1 week, 1 and 3 months
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Secondary outcome [3]
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Preliminary analysis of change in fine motor function:
Quality of Upper Extremity Skills Test (QUEST)
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Assessment method [3]
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Timepoint [3]
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3 months, 12 months
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Secondary outcome [4]
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Preliminary analysis of change in cognitive function:
Age appropriate cognitive testing
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Assessment method [4]
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Timepoint [4]
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12 months
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Secondary outcome [5]
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Preliminary analysis of change in quality of life:
Cerebral Palsy Quality of Life (CP-QoL-CHILD)
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Assessment method [5]
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Timepoint [5]
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3 months, 12 months
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Eligibility
Key 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 parents
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Minimum age
1
Years
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Maximum age
16
Years
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Sex
Both males and females
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Can healthy volunteers participate?
No
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Key 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 the 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 parental consent
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Study design
Purpose of the study
Treatment
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Allocation to intervention
Non-randomised trial
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Open (masking not used)
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Who is / are masked / blinded?
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Intervention assignment
Single group
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Other design features
- Additional exclusion criterion for the first six participants: mild/moderate CP
- Staggered treatments to allow observation between participants
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Phase
Not Applicable
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Type of endpoint/s
Safety
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Statistical methods / analysis
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Recruitment
Recruitment status
Completed
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Date of first participant enrolment
Anticipated
7/04/2016
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Actual
7/04/2016
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Date of last participant enrolment
Anticipated
31/01/2017
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Actual
6/06/2018
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Date of last data collection
Anticipated
30/04/2020
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Actual
30/04/2020
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Sample size
Target
12
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Accrual to date
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Final
12
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Recruitment in Australia
Recruitment state(s)
ACT,NSW,NT,QLD,SA,TAS,WA,VIC
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Funding & Sponsors
Funding source category [1]
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Charities/Societies/Foundations
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Name [1]
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Cerebral Palsy Alliance
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Address [1]
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187 Allambie Rd, Allambie Heights NSW 2100
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Country [1]
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Australia
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Funding source category [2]
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Commercial sector/Industry
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Name [2]
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Cell Care Australia
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Address [2]
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42 Corporate Drive, Heatherton VIC 3202
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Country [2]
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Australia
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Primary sponsor type
Other
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Name
Murdoch Childrens Research Institute
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Address
The Royal Children's Hospital
50 Flemington Rd Parkville VIC 3052
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Country
Australia
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Secondary sponsor category [1]
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None
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Name [1]
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Address [1]
291703
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Country [1]
291703
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Ethics approval
Ethics application status
Approved
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Ethics committee name [1]
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Royal Children's Hospital Human Research Ethics Committee
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Ethics committee address [1]
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Research, Ethics & Governance The Royal Children's Hospital 50 Flemington Rd, Parkville VIC 3052
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Ethics committee country [1]
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Australia
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Date submitted for ethics approval [1]
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18/09/2014
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Approval date [1]
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19/08/2015
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Ethics approval number [1]
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34210
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Summary
Brief 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. CP is often associated with epilepsy, difficulties in speech, sight, hearing, sensation, perception, behaviour or cognition. There is no cure for CP. 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 available. Therefore, this trial will study the safety of infusing matched sibling cord blood cells to children with cerebral palsy. Additionally, we will assess how long the cells remain within the recipient through sensitive chimerism assays, as we hypothesise that the cells may be rejected within 24 hours of infusion. Finally, we will trial the use of a range of outcome measures in this context.
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Trial website
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Trial related presentations / publications
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Public notes
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Attachments [1]
2774
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/AnzctrAttachments/370171-SCUBI-CP Protocol v10 06.03.17.pdf
(Protocol)
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Contacts
Principal investigator
Name
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Prof Dinah Reddihough
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Address
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Developmental Disability & Rehabilitation Research
Murdoch Childrens Research Institute
50 Flemington Rd, Parkville VIC 3052
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Country
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Australia
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Phone
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+613 9345 5898
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Fax
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Email
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dinah.reddihough@rch.org.au
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Contact person for public queries
Name
63743
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Kylie Crompton
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Address
63743
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Developmental Disability & Rehabilitation Research
Murdoch Childrens Research Institute
50 Flemington Rd, Parkville VIC 3052
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Country
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Australia
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Phone
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+613 9936 6756
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Fax
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Email
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scubi.cp@mcri.edu.au
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Contact person for scientific queries
Name
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Kylie Crompton
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Address
63744
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Developmental Disability & Rehabilitation Research
Murdoch Childrens Research Institute
50 Flemington Rd, Parkville VIC 3052
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Country
63744
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Australia
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Phone
63744
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+613 9936 6756
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Fax
63744
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Email
63744
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kylie.crompton@mcri.edu.au
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Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Yes
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What data in particular will be shared?
Assessment data (GMFM, QUEST, CPQoL-Child)
Adverse event data
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When will data be available (start and end dates)?
2021 - 2022
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Available to whom?
Anyone who wishes to access it
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Available for what types of analyses?
Any purpose
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How or where can data be obtained?
Access subject to approval by Principal Investigator
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What supporting documents are/will be available?
No Supporting Document Provided
Doc. No.
Type
Citation
Link
Email
Other Details
Attachment
1281
Study protocol
370171-(Uploaded-04-02-2019-15-50-47)-Study-related document.pdf
1282
Informed consent form
370171-(Uploaded-04-02-2019-15-51-21)-Study-related document.pdf
Results publications and other study-related documents
Documents added manually
No documents have been uploaded by study researchers.
Documents added automatically
No additional documents have been identified.
Download to PDF