ANZCTR - Registration

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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 endorsed by the ANZCTR. Before participating in a study, talk to your health care provider and refer to this information for consumers

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

Scientific title

Safety study of sibling cord blood cell infusion to children with cerebral palsy

Secondary ID [1]

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

Condition category

Condition code

Neurological

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]

Treatment: Other

Comparator / control treatment

No control group

Control group

Uncontrolled

Outcomes

Primary outcome [1]

Safety 1) Documentation of acute infusion reactions 2) Documentation of any instances of Graft-versus-Host Disease

Timepoint [1]

36 hours, 3 months, 12 months

Secondary outcome [1]

Preliminary analysis of change in gross motor function: Gross Motor Function Measure (GMFM-66)

Timepoint [1]

3 months, 12 months

Secondary outcome [2]

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]

1, 4 and 24 hours, 1 week, 1 and 3 months

Secondary outcome [3]

Preliminary analysis of change in fine motor function: Quality of Upper Extremity Skills Test (QUEST)

Timepoint [3]

3 months, 12 months

Secondary outcome [4]

Preliminary analysis of change in cognitive function: Age appropriate cognitive testing

Timepoint [4]

12 months

Secondary outcome [5]

Preliminary analysis of change in quality of life: Cerebral Palsy Quality of Life (CP-QoL-CHILD)

Timepoint [5]

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?

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

Accrual to date

Final

Recruitment in Australia

Recruitment state(s)

ACT,NSW,NT,QLD,SA,TAS,WA,VIC

Funding & Sponsors

Funding source category [1]

Charities/Societies/Foundations

Name [1]

Cerebral Palsy Alliance

Address [1]

187 Allambie Rd, Allambie Heights NSW 2100

Country [1]

Australia

Funding source category [2]

Commercial sector/Industry

Name [2]

Cell Care Australia

Address [2]

42 Corporate Drive, Heatherton VIC 3202

Country [2]

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]

None

Name [1]

Address [1]

Country [1]

Ethics approval

Ethics application status

Approved

Ethics committee name [1]

Royal Children's Hospital Human Research Ethics Committee

Ethics committee address [1]

Research, Ethics & Governance
The Royal Children's Hospital
50 Flemington Rd, Parkville VIC 3052

Ethics committee country [1]

Australia

Date submitted for ethics approval [1]

18/09/2014

Approval date [1]

19/08/2015

Ethics approval number [1]

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]

/AnzctrAttachments/370171-SCUBI-CP Protocol v10 06.03.17.pdf (Protocol)

Contacts

Principal investigator

Name

Prof Dinah Reddihough

Address

Developmental Disability & Rehabilitation Research Murdoch Childrens Research Institute 50 Flemington Rd, Parkville VIC 3052

Country

Australia

Phone

+613 9345 5898

Fax

[email protected]

Contact person for public queries

Name

Kylie Crompton

Address

Developmental Disability & Rehabilitation Research Murdoch Childrens Research Institute 50 Flemington Rd, Parkville VIC 3052

Country

Australia

Phone

+613 9936 6756

Fax

[email protected]

Contact person for scientific queries

Name

Kylie Crompton

Address

Developmental Disability & Rehabilitation Research Murdoch Childrens Research Institute 50 Flemington Rd, Parkville VIC 3052

Country

Australia

Phone

+613 9936 6756

Fax

[email protected]

Data sharing statement

Will the study consider sharing individual participant data?

Yes

Will there be any conditions when requesting access to individual participant data?

Persons/groups eligible to request access:
• Anyone who wishes to access it

Conditions for requesting access:
• -

What individual participant data might be shared?

• Assessment data (GMFM, QUEST, CPQoL-Child)
Adverse event data

What types of analyses could be done with individual participant data?

• Any purpose

When can requests for individual participant data be made (start and end dates)?

From:
2021

To:
2022

Where can requests to access individual participant data be made, or data be obtained directly?

• Access subject to approval by Principal Investigator

Are there extra considerations when requesting access to individual participant data?

What supporting documents are/will be available?

Doc. No.	Type	Citation	Link	Email	Other Details	Attachment
1281	Study protocol					Study-related document.pdf
1282	Informed consent form					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.

Trial Review

Documents added manually

Documents added automatically