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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT03087110




Registration number
NCT03087110
Ethics application status
Date submitted
26/02/2017
Date registered
22/03/2017

Titles & IDs
Public title
Stem Cells in Umbilical Blood Infusion for CP
Scientific title
Safety Study of Sibling Cord Blood Cell Infusion to Children With Cerebral Palsy
Secondary ID [1] 0 0
U1111-1179-9253
Secondary ID [2] 0 0
HREC/14/RCHM/38; RCH ID 34210
Universal Trial Number (UTN)
Trial acronym
SCUBI-CP
Linked study record

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

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Other - Matched sibling donor cord blood cell infusion

Experimental: Cord blood infusion - Matched sibling donor cord blood cell infusion


Treatment: Other: 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)
Intervention code [1] 0 0
Treatment: Other
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Number of participants with abnormal clinical assessment and/or laboratory values
Timepoint [1] 0 0
12 months
Secondary outcome [1] 0 0
Preliminary analysis of change in gross motor function
Timepoint [1] 0 0
Baseline, 3 months
Secondary outcome [2] 0 0
Preliminary analysis of change in gross motor function
Timepoint [2] 0 0
Baseline, 12 months
Secondary outcome [3] 0 0
Preliminary analysis of change in fine motor function
Timepoint [3] 0 0
Baseline, 3 months
Secondary outcome [4] 0 0
Preliminary analysis of change in fine motor function
Timepoint [4] 0 0
Baseline, 12 months
Secondary outcome [5] 0 0
Preliminary analysis of change in cognitive function
Timepoint [5] 0 0
Baseline, 12 months
Secondary outcome [6] 0 0
Preliminary analysis of change in quality of life
Timepoint [6] 0 0
Baseline, 3 months
Secondary outcome [7] 0 0
Preliminary analysis of change in quality of life
Timepoint [7] 0 0
Baseline,12 months
Secondary outcome [8] 0 0
Digital PCR analysis of peripheral blood cellular DNA to determine the fraction of donor DNA in circulation
Timepoint [8] 0 0
3 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 parent/guardian
Minimum age
1 Year
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 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

Study design
Purpose of the study
Treatment
Allocation to intervention
Not applicable
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
Phase
Phase 1
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
1/03/2016
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
1/12/2020
Sample size
Target
Accrual to date
Final
12
Recruitment in Australia
Recruitment state(s)
QLD,VIC
Recruitment hospital [1] 0 0
Lady Cilento Children's Hospital - Brisbane
Recruitment hospital [2] 0 0
The Royal Children's Hospital - Melbourne
Recruitment postcode(s) [1] 0 0
4101 - Brisbane
Recruitment postcode(s) [2] 0 0
3052 - Melbourne

Funding & Sponsors
Primary sponsor type
Other
Name
Murdoch Childrens Research Institute
Address
Country
Other collaborator category [1] 0 0
Other
Name [1] 0 0
The Royal Children's Hospital
Address [1] 0 0
Country [1] 0 0
Other collaborator category [2] 0 0
Other
Name [2] 0 0
Children's Health Queensland
Address [2] 0 0
Country [2] 0 0
Other collaborator category [3] 0 0
Other
Name [3] 0 0
Monash Health
Address [3] 0 0
Country [3] 0 0
Other collaborator category [4] 0 0
Other
Name [4] 0 0
Sydney Children's Hospitals Network
Address [4] 0 0
Country [4] 0 0
Other collaborator category [5] 0 0
Other
Name [5] 0 0
Cerebral Palsy Alliance
Address [5] 0 0
Country [5] 0 0

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Dinah Reddihough, MBChB, MD
Address 0 0
Group leader
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Yes
What data in particular will be shared?
The de-identified data set collected for this analysis of the SCUBI-CP trial will be available six months after publication of the primary outcome. The study protocol, analysis plan and consent forms will also be available. The data may be obtained from the Murdoch Children's Research Institute. Prior to releasing any data the following are required: a data access agreement must be signed between relevant parties, the SCUBI-CP Trial Steering Committee must see and approve the analysis plan describing how the data will be analysed, there must be an agreement around appropriate acknowledgement and any additional costs involved must be covered. Should the Trial Steering Committee be unavailable, this role is delegated to the Murdoch Children's Research Institute. Data will only be shared with a recognised research institution which has approved the proposed analysis plan.

Supporting document/s available: Study protocol, Statistical analysis plan (SAP), Informed consent form (ICF)
When will data be available (start and end dates)?
6 months after publication of primary outcome
Available to whom?
1) Data access agreement; 2) approval by Trial Steering Committee; 3) recognised research institutions.
Available for what types of analyses?
How or where can data be obtained?


What supporting documents are/will be available?




Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results not provided in https://clinicaltrials.gov/study/NCT03087110