Trial Review

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Trial registered on ANZCTR


Registration number
ACTRN12623000461695
Ethics application status
Approved
Date submitted
30/03/2023
Date registered
4/05/2023
Date last updated
4/05/2023
Date data sharing statement initially provided
4/05/2023
Type of registration
Retrospectively registered

Titles & IDs
Public title
Aplastic Anaemia and Other Bone Marrow Failure Syndromes Registry (AAR)
Scientific title
Aplastic Anaemia and Other Bone Marrow Failure Syndromes Registry (AAR)
Secondary ID [1] 309315 0
None
Universal Trial Number (UTN)
Trial acronym
AAR
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Aplastic Anaemia 329506 0
Bone Marrow Failure Syndrome 329507 0
Condition category
Condition code
Human Genetics and Inherited Disorders 326440 326440 0 0
Other human genetics and inherited disorders
Blood 326441 326441 0 0
Haematological diseases

Intervention/exposure
Study type
Observational
Patient registry
True
Target follow-up duration
50
Target follow-up type
Years
Description of intervention(s) / exposure
This is a patient registry collecting information on patients with a diagnosis of aplastic anaemia or any other acquired or inherited bone marrow failure syndrome. Data to be collected includes, but is not limited to: demographics, family history, co-morbidities, clinical features, full blood examination, bone marrow assessments, diagnosis, treatment received, treatment outcomes and overall survival. Information will be collected by review of medical records. Patients are not required to complete any questionnaires.
Intervention code [1] 325751 0
Diagnosis / Prognosis
Comparator / control treatment
No control group
Control group
Uncontrolled

Outcomes
Primary outcome [1] 334296 0
Incidence of acquired and inherited bone marrow failure syndromes in Australia by collecting diagnosis from the patient medical record.
Timepoint [1] 334296 0
At diagnosis. Follow up will occur at 6 months, 12 months and annual thereafter.
There is no end date to participation, patients will continue in follow up unless they withdraw.
Secondary outcome [1] 420171 0
N/A
Timepoint [1] 420171 0
N/A

Eligibility
Key inclusion criteria
Patients with diagnostic features of aplastic anaemia (AA) on bone marrow biopsy (pancytopenia with a hypocellular bone marrow, absence of an abnormal marrow infiltrate and no increase in reticulin), where other causes of bone marrow failure (e.g. following chemotherapy or B12 deficiency) have been excluded. Patients with AA that is acquired or due to inherited factors are included on the registry.
Minimum age
No limit
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
Patients who have chosen to ‘opt-off’ the registry.

Study design
Purpose
Natural history
Duration
Longitudinal
Selection
Convenience sample
Timing
Both
Statistical methods / analysis
Analysis plans will be reviewed by the Steering Committee and by a suitably qualified statistician prior to data from the AAR being analysed.
A detailed pre-determined statistical plan will be developed prior to planned analyses of registry data.

Recruitment
Recruitment status
Recruiting
Date of first participant enrolment
Anticipated
Actual
28/06/2013
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
1000
Accrual to date
352
Final
Recruitment in Australia
Recruitment state(s)
ACT,NSW,QLD,SA,TAS,WA,VIC
Recruitment hospital [1] 24369 0
The Alfred - Melbourne
Recruitment hospital [2] 24370 0
Austin Health - Austin Hospital - Heidelberg
Recruitment hospital [3] 24371 0
Box Hill Hospital - Box Hill
Recruitment hospital [4] 24372 0
Calvary Mater Newcastle - Waratah
Recruitment hospital [5] 24373 0
The Canberra Hospital - Garran
Recruitment hospital [6] 24374 0
The Children's Hospital at Westmead - Westmead
Recruitment hospital [7] 24375 0
Concord Repatriation Hospital - Concord
Recruitment hospital [8] 24376 0
Fiona Stanley Hospital - Murdoch
Recruitment hospital [9] 24377 0
Flinders Medical Centre - Bedford Park
Recruitment hospital [10] 24378 0
Barwon Health - Geelong Hospital campus - Geelong
Recruitment hospital [11] 24379 0
Gosford Hospital - Gosford
Recruitment hospital [12] 24380 0
John Hunter Children's Hospital - New Lambton
Recruitment hospital [13] 24381 0
Liverpool Hospital - Liverpool
Recruitment hospital [14] 24382 0
Monash Medical Centre - Clayton campus - Clayton
Recruitment hospital [15] 24384 0
Nepean Hospital - Kingswood
Recruitment hospital [16] 24385 0
Peter MacCallum Cancer Centre - Melbourne
Recruitment hospital [17] 24386 0
Prince of Wales Hospital - Randwick
Recruitment hospital [18] 24388 0
Perth Children's Hospital - Nedlands
Recruitment hospital [19] 24389 0
Princess Alexandra Hospital - Woolloongabba
Recruitment hospital [20] 24390 0
The Queen Elizabeth Hospital - Woodville
Recruitment hospital [21] 24391 0
Queensland Children's Hospital - South Brisbane
Recruitment hospital [22] 24392 0
The Royal Adelaide Hospital - Adelaide
Recruitment hospital [23] 24393 0
Royal Brisbane & Womens Hospital - Herston
Recruitment hospital [24] 24394 0
The Royal Childrens Hospital - Parkville
Recruitment hospital [25] 24395 0
Royal Hobart Hospital - Hobart
Recruitment hospital [26] 24396 0
Royal Melbourne Hospital - City campus - Parkville
Recruitment hospital [27] 24397 0
Royal North Shore Hospital - St Leonards
Recruitment hospital [28] 24398 0
Royal Perth Hospital - Perth
Recruitment hospital [29] 24399 0
Royal Prince Alfred Hospital - Camperdown
Recruitment hospital [30] 24400 0
Sir Charles Gairdner Hospital - Nedlands
Recruitment hospital [31] 24401 0
St George Hospital - Kogarah
Recruitment hospital [32] 24402 0
St Vincent's Hospital (Melbourne) Ltd - Fitzroy
Recruitment hospital [33] 24403 0
St Vincent's Hospital (Darlinghurst) - Darlinghurst
Recruitment hospital [34] 24404 0
Sydney Children's Hospital - Randwick
Recruitment hospital [35] 24405 0
The Townsville Hospital - Douglas
Recruitment hospital [36] 24406 0
Westmead Hospital - Westmead
Recruitment hospital [37] 24407 0
Wollongong Hospital - Wollongong
Recruitment hospital [38] 24408 0
Womens and Childrens Hospital - North Adelaide
Recruitment postcode(s) [1] 39984 0
2010 - Darlinghurst
Recruitment postcode(s) [2] 39969 0
2031 - Randwick
Recruitment postcode(s) [3] 39981 0
2050 - Camperdown
Recruitment postcode(s) [4] 39979 0
2065 - St Leonards
Recruitment postcode(s) [5] 39959 0
2139 - Concord
Recruitment postcode(s) [6] 39958 0
2145 - Westmead
Recruitment postcode(s) [7] 39965 0
2170 - Liverpool
Recruitment postcode(s) [8] 39982 0
2217 - Kogarah
Recruitment postcode(s) [9] 39963 0
2250 - Gosford
Recruitment postcode(s) [10] 39956 0
2298 - Waratah
Recruitment postcode(s) [11] 39964 0
2305 - New Lambton
Recruitment postcode(s) [12] 39986 0
2500 - Wollongong
Recruitment postcode(s) [13] 39957 0
2605 - Garran
Recruitment postcode(s) [14] 39968 0
2747 - Kingswood
Recruitment postcode(s) [15] 39953 0
3004 - Melbourne
Recruitment postcode(s) [16] 39977 0
3052 - Parkville
Recruitment postcode(s) [17] 39983 0
3065 - Fitzroy
Recruitment postcode(s) [18] 39954 0
3084 - Heidelberg
Recruitment postcode(s) [19] 39955 0
3128 - Box Hill
Recruitment postcode(s) [20] 39966 0
3168 - Clayton
Recruitment postcode(s) [21] 39962 0
3220 - Geelong
Recruitment postcode(s) [22] 39976 0
4029 - Herston
Recruitment postcode(s) [23] 39974 0
4101 - South Brisbane
Recruitment postcode(s) [24] 39972 0
4102 - Woolloongabba
Recruitment postcode(s) [25] 39985 0
4814 - Douglas
Recruitment postcode(s) [26] 39975 0
5000 - Adelaide
Recruitment postcode(s) [27] 39987 0
5006 - North Adelaide
Recruitment postcode(s) [28] 39973 0
5011 - Woodville
Recruitment postcode(s) [29] 39961 0
5042 - Bedford Park
Recruitment postcode(s) [30] 39980 0
6000 - Perth
Recruitment postcode(s) [31] 39971 0
6009 - Nedlands
Recruitment postcode(s) [32] 39960 0
6150 - Murdoch
Recruitment postcode(s) [33] 39978 0
7000 - Hobart

Funding & Sponsors
Funding source category [1] 313511 0
Charities/Societies/Foundations
Name [1] 313511 0
Maddie Riewoldt's Vision
Country [1] 313511 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] 315287 0
None
Name [1] 315287 0
None
Address [1] 315287 0
Country [1] 315287 0

Ethics approval
Ethics application status
Approved
Ethics committee name [1] 312709 0
Monash Health HREC
Ethics committee address [1] 312709 0
Research Support Services
Level 2, i Block,
Monash Medical Centre
246 Clayton Road
CLAYTON VIC 3168
Ethics committee country [1] 312709 0
Australia
Date submitted for ethics approval [1] 312709 0
16/09/2020
Approval date [1] 312709 0
29/09/2020
Ethics approval number [1] 312709 0
13139A
Ethics committee name [2] 312902 0
Children and Adolescent Health Service HREC
Ethics committee address [2] 312902 0
Office 5E, Perth Children’s Hospital
15 Hospital Avenue
Nedlands WA 6009
Ethics committee country [2] 312902 0
Australia
Date submitted for ethics approval [2] 312902 0
09/02/2021
Approval date [2] 312902 0
28/07/2021
Ethics approval number [2] 312902 0
RGS2439
Ethics committee name [3] 312939 0
RPAH HREC
Ethics committee address [3] 312939 0
Research Development Office
Royal Prince Alfred Hospital
Missenden Road
CAMPERDOWN NSW 2050
Ethics committee country [3] 312939 0
Australia
Date submitted for ethics approval [3] 312939 0
05/09/2012
Approval date [3] 312939 0
20/12/2012
Ethics approval number [3] 312939 0
X12-0311, HREC/12/RPAH/475

Summary
Brief summary
Aplastic Anaemia (AA) and Bone Marrow Failure Syndromes (BMFS) are rare diseases with significant morbidity and mortality. Currently there is little Australian data on the incidence of both acquired AA and BMFS, the therapies utilised or clinical outcomes.
The relative rarity of this disease makes both the accrual of data and material to support scientific studies, and the establishment of high quality randomised prospective trials challenging.
Furthermore, the existing literature concerning clinical outcomes of patients with AA may be biased by preferential reporting and publication of only good outcomes and/or serious or unusual events.
The national registry for all Australian patients with AA and BMFS provides an important community resource to address these issues.
Trial website
https://aaregistry.org.au/
Trial related presentations / publications
Public notes
All sites (with the exception of Western Australian sites) are approved by the Monash Health HREC under the NMA scheme. Western Australian sites are approved by Children and Adolescent Health Service HREC.

The registry was established in 2012 with single site ethics approvals at each individual site. Pre-NMA approval was obtained on 20/12/2012. Since the NMA approval on 29/09/2020, all previous approvals have been superseded.

Contacts
Principal investigator
Name 125594 0
Prof Erica Wood
Address 125594 0
Monash University
School of Public Health and Preventive Medicine,
Level 1
553 St Kilda Road,
Melbourne, VIC 3004
Country 125594 0
Australia
Phone 125594 0
+61 3 99030051
Fax 125594 0
Email 125594 0
erica.wood@monash.edu
Contact person for public queries
Name 125595 0
Ms Vanessa Fox
Address 125595 0
Monash University
School of Public Health and Preventive Medicine,
Level 1
553 St Kilda Road,
Melbourne, VIC 3004
Country 125595 0
Australia
Phone 125595 0
+61 3 99030532
Fax 125595 0
Email 125595 0
vanessa.fox@monash.edu
Contact person for scientific queries
Name 125596 0
Dr Lucy Fox
Address 125596 0
Peter MacCallum Cancer Centre
305 Grattan St, Melbourne VIC 3000
Country 125596 0
Australia
Phone 125596 0
+61 3 8559 7731
Fax 125596 0
Email 125596 0
lucy.fox@petermac.org

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
No/undecided IPD sharing reason/comment
Patient confidentiality


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.