Trial Review

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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
Date results information initially provided
5/02/2019
Type of registration
Prospectively registered

Titles & IDs
Public title
Stem Cells in Umbilical Blood Infusion for Cerebral Palsy
Scientific title
Safety study of sibling cord blood cell infusion to children with cerebral palsy
Secondary ID [1] 288592 0
ClinicalTrials.gov registry number: NCT03087110
Universal Trial Number (UTN)
U1111-1179-9253
Trial acronym
SCUBI-CP
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Cerebral palsy 297738 0
Condition category
Condition code
Neurological 297921 297921 0 0
Other neurological disorders

Intervention/exposure
Study type
Interventional
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.
Intervention code [1] 293988 0
Treatment: Other
Comparator / control treatment
No control group
Control group
Uncontrolled

Outcomes
Primary outcome [1] 297436 0
Safety
1) Documentation of acute infusion reactions
2) Documentation of any instances of Graft-versus-Host Disease
Timepoint [1] 297436 0
36 hours, 3 months, 12 months
Secondary outcome [1] 321048 0
Preliminary analysis of change in gross motor function:
Gross Motor Function Measure (GMFM-66)
Timepoint [1] 321048 0
3 months, 12 months
Secondary outcome [2] 321049 0
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
Timepoint [2] 321049 0
1, 4 and 24 hours, 1 week, 1 and 3 months
Secondary outcome [3] 322178 0
Preliminary analysis of change in fine motor function:
Quality of Upper Extremity Skills Test (QUEST)
Timepoint [3] 322178 0
3 months, 12 months
Secondary outcome [4] 322206 0
Preliminary analysis of change in cognitive function:
Age appropriate cognitive testing
Timepoint [4] 322206 0
12 months
Secondary outcome [5] 322207 0
Preliminary analysis of change in quality of life:
Cerebral Palsy Quality of Life (CP-QoL-CHILD)
Timepoint [5] 322207 0
3 months, 12 months

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
Minimum age
1 Years
Maximum age
16 Years
Sex
Both males and females
Can healthy volunteers participate?
No
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

Study design
Purpose of the study
Treatment
Allocation to intervention
Non-randomised trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
- Additional exclusion criterion for the first six participants: mild/moderate CP
- Staggered treatments to allow observation between participants
Phase
Not Applicable
Type of endpoint/s
Safety
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Date of first participant enrolment
Anticipated
7/04/2016
Actual
7/04/2016
Date of last participant enrolment
Anticipated
31/01/2017
Actual
6/06/2018
Date of last data collection
Anticipated
30/04/2020
Actual
30/04/2020
Sample size
Target
12
Accrual to date
Final
12
Recruitment in Australia
Recruitment state(s)
ACT,NSW,NT,QLD,SA,TAS,WA,VIC

Funding & Sponsors
Funding source category [1] 292939 0
Charities/Societies/Foundations
Name [1] 292939 0
Cerebral Palsy Alliance
Country [1] 292939 0
Australia
Funding source category [2] 292940 0
Commercial sector/Industry
Name [2] 292940 0
Cell Care Australia
Country [2] 292940 0
Australia
Primary sponsor type
Other
Name
Murdoch Childrens Research Institute
Address
The Royal Children's Hospital
50 Flemington Rd Parkville VIC 3052
Country
Australia
Secondary sponsor category [1] 291703 0
None
Name [1] 291703 0
Address [1] 291703 0
Country [1] 291703 0

Ethics approval
Ethics application status
Approved
Ethics committee name [1] 294444 0
Royal Children's Hospital Human Research Ethics Committee
Ethics committee address [1] 294444 0
Research, Ethics & Governance
The Royal Children's Hospital
50 Flemington Rd, Parkville VIC 3052
Ethics committee country [1] 294444 0
Australia
Date submitted for ethics approval [1] 294444 0
18/09/2014
Approval date [1] 294444 0
19/08/2015
Ethics approval number [1] 294444 0
34210

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.
Trial website
Trial related presentations / publications
Public notes
Attachments [1] 2774 2774 0 0
/AnzctrAttachments/370171-SCUBI-CP Protocol v10 06.03.17.pdf (Protocol)

Contacts
Principal investigator
Name 63742 0
Prof Dinah Reddihough
Address 63742 0
Developmental Disability & Rehabilitation Research
Murdoch Childrens Research Institute
50 Flemington Rd, Parkville VIC 3052
Country 63742 0
Australia
Phone 63742 0
+613 9345 5898
Fax 63742 0
Email 63742 0
[email protected]
Contact person for public queries
Name 63743 0
Dr Kylie Crompton
Address 63743 0
Developmental Disability & Rehabilitation Research
Murdoch Childrens Research Institute
50 Flemington Rd, Parkville VIC 3052
Country 63743 0
Australia
Phone 63743 0
+613 9936 6756
Fax 63743 0
Email 63743 0
[email protected]
Contact person for scientific queries
Name 63744 0
Dr Kylie Crompton
Address 63744 0
Developmental Disability & Rehabilitation Research
Murdoch Childrens Research Institute
50 Flemington Rd, Parkville VIC 3052
Country 63744 0
Australia
Phone 63744 0
+613 9936 6756
Fax 63744 0
Email 63744 0
[email protected]

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Yes
What data in particular will be shared?
Assessment data (GMFM, QUEST, CPQoL-Child)
Adverse event data
When will data be available (start and end dates)?
2021 - 2022
Available to whom?
Anyone who wishes to access it
Available for what types of analyses?
Any purpose
How or where can data be obtained?
Access subject to approval by Principal Investigator


What supporting documents are/will be available?

Doc. No.TypeCitationLinkEmailOther DetailsAttachment
1281Study protocol    370171-(Uploaded-04-02-2019-15-50-47)-Study-related document.pdf
1282Informed 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.