Trial Review

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Trial registered on ANZCTR


Registration number
ACTRN12623000424606
Ethics application status
Approved
Date submitted
28/03/2023
Date registered
28/04/2023
Date last updated
28/04/2023
Date data sharing statement initially provided
28/04/2023
Type of registration
Prospectively registered

Titles & IDs
Public title
Australian Marrow Failure Biobank
Scientific title
Australian Marrow Failure Biobank
Secondary ID [1] 308998 0
None
Universal Trial Number (UTN)
Trial acronym
AMFB
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Bone Marrow Failure Syndromes 329036 0
Condition category
Condition code
Human Genetics and Inherited Disorders 326015 326015 0 0
Other human genetics and inherited disorders
Blood 326016 326016 0 0
Haematological diseases

Intervention/exposure
Study type
Observational
Patient registry
True
Target follow-up duration
5
Target follow-up type
Years
Description of intervention(s) / exposure
The biobank is established to create repository of biological specimens from patients with acquired or inherited bone marrow failure syndromes including germline predisposition to haematological malignancy. Samples may be linked to patient records in the previously established Aplastic Anaemia and Other Bone Marrow Failure Syndromes Registry (AAR), which houses a comprehensive clinical data set.

Patients with bone marrow failure syndrome will be asked to provide the following samples:
BASELINE: 1x blood sample, 1x bone marrow aspirate sample and any one of the following germline samples: hair bulb, oral collection, nail clippings.
ANNUAL: 1x blood sample.
FORTUITOUS: 1x bone marrow aspirate sample for each bone marrow biopsy the patient undergoes.
Bespoke sample collection of valuable biological samples which are not included above are able to be sent to the biobank with prior ethics approval and discussion with the AMFB team to ensure that appropriate protocols exist to permit storage.

Every effort will be made for these research samples to be collected during routine visits and procedures. Patients will continue with annual collections unless they decide to withdraw.

Consented asymptomatic blood relatives of eligible patients who participate in the AMFB will have a singular baseline peripheral blood sample collected.
Intervention code [1] 325442 0
Not applicable
Comparator / control treatment
No control group
Control group
Uncontrolled

Outcomes
Primary outcome [1] 333874 0
Annual target of collection of 150 bone marrow aspirates and 300 peripheral blood samples.
Timepoint [1] 333874 0
At diagnosis, 12 months, annually thereafter.
Secondary outcome [1] 418595 0
N/A
Timepoint [1] 418595 0
N/A

Eligibility
Key inclusion criteria
For for individuals with BMFS:
- Established diagnosis of a BMFS or newly presenting diagnosis of BMFS
- Participant or substitute decision maker is willing and able to give informed consent
- Participant or substitute decision-maker is willing to participate in the Aplastic Anaemia and Other Bone Marrow Failure Syndromes Registry (AAR)

Due to the inherited nature of these conditions, blood relatives of a person with an established diagnosis of an inherited BMFS or newly presenting diagnosis of an inherited BMFS are also invited to participate in the AMFB where indicated.
Minimum age
No limit
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
Yes
Key exclusion criteria
For for individuals with BMFS:
- A verified medical diagnosis other than a BMFS (e.g. following chemotherapy or B12 deficiency)

Study design
Purpose
Natural history
Duration
Longitudinal
Selection
Convenience sample
Timing
Prospective
Statistical methods / analysis
The AMFB will be governed by the Data and Biologicals Access Committee (DBAC). The DBAC will be responsible for receiving and considering expressions of interest (EOI) from researchers for access to clinical data and biological specimens. The DBAC will confirm scientific quality and relevance to bone marrow failure research before approving an application. The researchers must have an approved HREC application and executed Data and Biospecimen Transfer Agreement before access is granted for AMFB samples.

Recruitment
Recruitment status
Not yet recruiting
Date of first participant enrolment
Anticipated
1/05/2023
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
200
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW,VIC
Recruitment hospital [1] 24048 0
Monash Medical Centre - Clayton campus - Clayton
Recruitment hospital [2] 24049 0
Monash Children’s Hospital - Clayton
Recruitment hospital [3] 24050 0
Peter MacCallum Cancer Centre - Melbourne
Recruitment hospital [4] 24051 0
Concord Repatriation Hospital - Concord
Recruitment postcode(s) [1] 39553 0
2139 - Concord
Recruitment postcode(s) [2] 39552 0
3000 - Melbourne
Recruitment postcode(s) [3] 39551 0
3168 - Clayton

Funding & Sponsors
Funding source category [1] 313211 0
Charities/Societies/Foundations
Name [1] 313211 0
Maddie Riewoldt's Vision
Country [1] 313211 0
Australia
Primary sponsor type
University
Name
Monash University
Address
Monash University
School of Public Health and Preventive Medicine,
Level 1
553 St Kilda Road,
Melbourne, VIC 3004
Country
Australia
Secondary sponsor category [1] 314931 0
None
Name [1] 314931 0
None
Address [1] 314931 0
Country [1] 314931 0

Ethics approval
Ethics application status
Approved
Ethics committee name [1] 312442 0
Monash Health HREC
Ethics committee address [1] 312442 0
Research Support Services
Level 2, i Block,
Monash Medical Centre
246 Clayton Road
CLAYTON VIC 3168
Ethics committee country [1] 312442 0
Australia
Date submitted for ethics approval [1] 312442 0
26/10/2022
Approval date [1] 312442 0
16/03/2023
Ethics approval number [1] 312442 0
RES-22-0000-667A

Summary
Brief summary
Approximately 5 million people are affected by bone marrow failure syndromes (BMFS) worldwide each year, but individually many of the BMFS are rare. In Australia there are 160 new diagnoses each year, of which 50% do not survive. The rarity of these conditions and the absence of coordinated data and sample collection present barriers to research into biology and optimal treatment of BMFS. In Australia, Maddie Riewoldt's Vision (MRV) has supported a national comprehensive clinical data set on newly diagnosed patients with BMFS through strategic funding of the Aplastic Anaemia Registry and Other Bone Marrow Failure Syndromes Registry (AAR). MRV seeks to build on this initiative by establishing a matched research sample set that will allow new research questions to be asked, provide a feasible and ready-to-go sample set that will support BMFS research. The purpose of the AMFB is to establish a national repository of samples from patients with BMFS to serve as a community resource for clinicians and researchers. Promote nationally consistent, comprehensive diagnostic (including molecular) testing for patients with BMFS. It is hoped that the research from this biobank will provide valuable information to improve the diagnosis, treatment, or care of people with BMFS.
Trial website
https://aaregistry.org.au/australian-marrow-failure-biobank/
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 124682 0
Prof Melissa Southey
Address 124682 0
School of Clinical Sciences at Monash Health
Medicine, Nursing and Health Sciences
Monash University
246 Clayton Road
Clayton Vic 3168
Country 124682 0
Australia
Phone 124682 0
+61 3 8572 2391
Fax 124682 0
Email 124682 0
melissa.southey@monash.edu
Contact person for public queries
Name 124683 0
Dr Lauren Young
Address 124683 0
Monash University
School of Public Health and Preventive Medicine,
Level 1
553 St Kilda Road,
Melbourne, VIC 3004
Country 124683 0
Australia
Phone 124683 0
+61 3 9903 0115
Fax 124683 0
Email 124683 0
lauren.young@monash.edu
Contact person for scientific queries
Name 124684 0
Prof Erica Wood
Address 124684 0
Monash University
School of Public Health and Preventive Medicine,
Level 1
553 St Kilda Road,
Melbourne, VIC 3004
Country 124684 0
Australia
Phone 124684 0
+61 3 99030051
Fax 124684 0
Email 124684 0
erica.wood@monash.edu

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Yes
What data in particular will be shared?
Individual participant samples will be coded with re-identifiable codes before access to researchers is granted. As such, no directly identifying data will be shared.
When will data be available (start and end dates)?
Samples will be available once an adequate repository of samples has been established.
No end date has been determined.
Available to whom?
Only researchers who provide a methodologically sound proposal and who have approval from relevant human research ethics committees and the AMFB Data and Biological Access Committee.
Available for what types of analyses?
Only to achieve aims related to those listed in the approved proposal.
How or where can data be obtained?
Researchers will be required to sign a Data and Biospecimen Transfer Agreement before receiving any samples. The cost of transporting samples will be covered by the researchers.
To initiate this process, please contact amfbiobank@monash.edu


What supporting documents are/will be available?

No Supporting Document Provided



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.