Trial registered on ANZCTR


Trial ID
ACTRN12613000186752
Ethics application status
Approved
Date submitted
13/02/2013
Date registered
15/02/2013
Date last updated
27/03/2014
Type of registration
Prospectively registered

Titles & IDs
Public title
Cord Reinfusion in Diabetes Pilot Study
Scientific title
A pilot study to assess the feasibility of reinfusion of cord blood in children at risk of type 1 diabetes.

Secondary ID [1] 281950 0
Nil known
Universal Trial Number (UTN)
U1111-1139-5067
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Type 1 diabetes 288368 0
Condition category
Condition code
Metabolic and Endocrine 288714 288714 0 0
Diabetes

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
The trial involves two stages. The first stage is to screen, with a blood test, those with a first or second degree relative with type 1 diabetes, for diabetes autoantibodies. The purpose of screening is to determine their risk of developing type 1 diabetes. The screening blood tests can be conducted every 12months for the duration of the study to monitor the development of said autoantibodies. Rescreening will be offerred to all participants during the 5 years study duration and will be conducted with the consent of the participant.

Those at high risk (positive for two or more autoantibodies) will be invited to participate in stage 2 where they will be given a reinfusion of their autologous cord blood via intravenous infusion. This will be a one off treatment that will take approximately five hours with the reinfusion taking 1 hour followed by a 4 hour observation period. Post reinfusion all participants will enter follow-up with visits and blood tests every 3 months for the first year and every 6 months the following two years after treatment to monitor effect.
Intervention code [1] 286513 0
Early detection / Screening
Intervention code [2] 286533 0
Prevention
Comparator / control treatment
For this pilot study there will be no placebo or control group.
Control group
Uncontrolled

Outcomes
Primary outcome [1] 288856 0
The primary outcome of this study is to determine the feasibility of using autologous cord blood in children at risk of developing type 1 diabetes. This will be done by assessing the incidence of persons with high-risk of developing type 1 diabetes who have stored cord blood, and assessing the processes involved in reinfusing autologous cord blood in this group. This data will be collected through medical assessments, assessment of past family and medical history and performing laboratory tests.
Timepoint [1] 288856 0
We envisage that by the 3rd year of recruitment and screening, we would have identified the 20 participants that will go onto receive the reinfusion. At this point, data analysis of the number of screening participants to the number of participants idenitified as being high-risk will be conducted. We will assess the processes in preparing and reinfusing the autologous cord blood unit and determine if these processes are feasible.
Secondary outcome [1] 301173 0
Determine safety of reinfusing autologous cord blood in this population by assessing occurance of adverse events through the collection of clinical assessments and blood tests.
Timepoint [1] 301173 0
Safety will be determined at the point of the reinfusion visit for each participant and ongoing as a participant group by analysing the data and clinical assessments collected on the day and in follow-up. Expected adverse events include pain and/or bruising from venepuncture, nausea, vomiting, fever, rash and itch. All these adverse events will be assessed through clinical assessment and reporting of same.

Eligibility
Key inclusion criteria
The key inclusion criteria for the study requires that potential participants must be aged 1-12 years old, be a first or second relative of a person with type 1 diabetes, have cord blood collected and stored from birth, and not have diabetes.
Minimum age
1 Years
Maximum age
12 Years
Gender
Both males and females
Can healthy volunteers participate?
Yes
Key exclusion criteria
Participants who have diabetes, do not have adequate quality and quantity of cord blood stored and impaired renal, liver or immunological function will be excluded.

Study design
Purpose of the study
Prevention
Allocation to intervention
Non-randomised trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Those identified from screening to be at high risk of developing type 1 diabetes (positive markers for two or more autoantibodies tested) will all receive a reinfusion of autologous cord blood if they consent to it being used.
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
There will be no randomisation.
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 0
Type of endpoint(s)
Safety
Statistical methods / analysis
As the primary outcome measure is feasibility of recruitment and screening of children who have cord blood (CB) stored and have a first or second degree relative with type 1 diabetes (T1D), or have previously been tested for islet antibodies, the results will be reported using descriptive statistics. Specifically we will determine:
- Response rate to recruitment from families whose child has CB stored at private and public cord banks; comparison of estimated versus actual numbers.
- Number of children with CB who have a first or second degree relative with T1D
- Number of children with CB who have previously been screened for islet autoantibodies.
These data will inform sample size calculations and expected response rates the proposed randomised controlled trial. Interim analysis of these data will occur once the targeted group for screening have been approached and responded at approximately 3 years into the study.

Recruitment
Recruitment status
Recruiting
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Current
Final
Recruitment in Australia
Recruitment state(s)
ACT,NSW,NT,QLD,SA,TAS,WA,VIC
Recruitment postcode(s) [1] 6319 0
2145 - Westmead
Recruitment postcode(s) [2] 6347 0
2000 - Sydney
Recruitment postcode(s) [3] 6348 0
3000 - Melbourne
Recruitment postcode(s) [4] 6349 0
4000 - Brisbane
Recruitment postcode(s) [5] 6350 0
5000 - Adelaide
Recruitment postcode(s) [6] 6351 0
6000 - Perth
Recruitment postcode(s) [7] 6352 0
7000 - Hobart

Funding & Sponsors
Funding source category [1] 286729 0
Commercial sector/Industry
Name [1] 286729 0
Cell Care Australia
Address [1] 286729 0
PO Box 833 Moorabbin VIC 3189 AUSTRALIA
Country [1] 286729 0
Australia
Primary sponsor type
Hospital
Name
Sydney Children's Hospitals Network (Westmead)
Address
Corner of Hawkesbury and Hainsworth Streets Westmead NSW 2145
Country
Australia
Secondary sponsor category [1] 285504 0
None
Name [1] 285504 0
Address [1] 285504 0
Country [1] 285504 0

Ethics approval
Ethics application status
Approved
Ethics committee name [1] 288801 0
Sydney Children's Hospitals Network HREC
Ethics committee address [1] 288801 0
Kids Research Institute
Children's Hospital at Westmead
Locked Bag 4001
WESTMEAD NSW 2145
Ethics committee country [1] 288801 0
Australia
Date submitted for ethics approval [1] 288801 0
Approval date [1] 288801 0
01/06/2012
Ethics approval number [1] 288801 0
EC00130

Summary
Brief summary
The CORD study is a pilot study aiming to determine the feasibility of reinfusing cord blood in children who are at risk of developing type 1 diabetes. The hypothesis is that autologous cord blood, when reinfused into children at high risk of developing type 1 diabetes, will resotre immune tolerance and prevent or mitigate the development of type 1 diabetes.

Participants aged 1-12 years with a relative with type 1 diabetes will be tested every 12 months for the development of islet antigens. Following screening, a target treatment group of 20 with two or more autoantibodies, will receive an infusion of their autologous cord blood. Participants will be followed for 3 years or until they develop diabetes.
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 37834 0
A/Prof Maria Craig
Address 37834 0
Institute of Endocrinology and Diabetes
The Children's Hospital at Westmead
Corner of Hawkesbury and Hainsworth Street
WESTMEAD NSW 2145
Country 37834 0
Australia
Phone 37834 0
+61 2 98453907
Fax 37834 0
Email 37834 0
m.craig@unsw.edu.au
Contact person for public queries
Name 37835 0
Miss Phuong Phan
Address 37835 0
Institute of Endocrinology and Diabetes
The Children's Hospital at Westmead
Corner of Hawkesbury and Hainsworth Street
WESTMEAD NSW 2145
Country 37835 0
Australia
Phone 37835 0
+61 2 98451233
Fax 37835 0
Email 37835 0
phuong.phan@health.nsw.edu.au
Contact person for scientific queries
Name 37836 0
A/Prof Maria Craig
Address 37836 0
Institute of Endocrinology and Diabetes
The Children's Hospital at Westmead
Corner of Hawkesbury and Hainsworth Street
WESTMEAD NSW 2145
Country 37836 0
Australia
Phone 37836 0
+61 2 98453907
Fax 37836 0
Email 37836 0
m.craig@unsw.edu.au