Trial Review

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Trial registered on ANZCTR


Registration number
ACTRN12614001073695
Ethics application status
Approved
Date submitted
16/09/2014
Date registered
7/10/2014
Date last updated
24/02/2022
Date data sharing statement initially provided
24/02/2022
Type of registration
Prospectively registered

Titles & IDs
Public title
A Multi-centre, Observational Study of Patients with Microangiopathic Thrombocytopenia by the Asian-Pacific Microangiopathic Thrombocytopenia (APMAT) Network
Scientific title
A Multi-centre, Observational Study by the Asian-Pacific Microangiopathic Thrombocytopenia (APMAT) Network To Determine The Clinical Characteristics, Laboratory Features, Treatments, and Clinical Outcomes in Patients with Microangiopathic Thrombocytopenia
Secondary ID [1] 285350 0
None
Universal Trial Number (UTN)
U1111-1161-8119
Trial acronym
APMAT 1
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Microangiopathic Thrombocytopenia 293074 0
Condition category
Condition code
Blood 293349 293349 0 0
Other blood disorders

Intervention/exposure
Study type
Observational
Patient registry
True
Target follow-up duration
6
Target follow-up type
Months
Description of intervention(s) / exposure
This is an observational study of previously diagnosed and treated microangiopathic thrombocytopenia (MAT) patients. MAT is a rare collection of disorders caused by the formation of intravascular aggregates of platelets resulting in microcirculatory thrombosis, red blood cell destruction and vital end-organ damage. The study will collect clinical and laboratory data to assess the disease characteristics, laboratory features, treatment strategies employed, and clinical outcome. Each participant will be observed for 6 months once they are enrolled in the study.
Intervention code [1] 290268 0
Not applicable
Comparator / control treatment
Not applicable
Control group
Uncontrolled

Outcomes
Primary outcome [1] 293183 0
To determine the clinical characteristics of microangiopathic thrombocytopenia including symptoms, potential precipitating factors, outcome, genetic, autoantibody and immune complex factors. Data will be collected by reviewing patients' medical records.
Timepoint [1] 293183 0
Follow-up of patients: 6 months;
Collection of data for all patients: Approx 18 months
Primary outcome [2] 293240 0
To determine the treatments given for microangiopathic thrombocytopenia. Data will be collected by reviewing patients' medical records.
Timepoint [2] 293240 0
Follow-up of patients: 6 months;
Collection of data for all patients: Approx 18 months
Primary outcome [3] 293241 0
To determine laboratory features microangiopathic thrombocytopenia patients. Methods for analysis include standard coagulation tests and cell counts, flow cytometry, immunoassays, activity assays and/or genotyping.
Timepoint [3] 293241 0
Follow-up of patients: 6 months;
Collection of data for all patients: Approx 18 months
Secondary outcome [1] 310539 0
To assess the value of ADAMTS13 testing in predicting disease relapse. Immunoassays and activity assays will be performed on blood samples collected at enrolment and six months post-enrolment.
Timepoint [1] 310539 0
Follow-up of patients: 6 months;
Collection of data for all patients: Approx 18 months
Secondary outcome [2] 310540 0
To assess the value of ADAMTS13 genetic mutations in predicting disease relapse. Genotyping will be performed on blood samples collected at enrolment and six months post-enrolment.
Timepoint [2] 310540 0
Follow-up of patients: 6 months;
Collection of data for all patients: Approx 18 months
Secondary outcome [3] 310541 0
To establish a MAT Biobank for further testing of novel assays used for the diagnosis and scientific understanding of MAT.
Timepoint [3] 310541 0
18 months
Secondary outcome [4] 310665 0
To facilitate basic disease mechanism and translational clinical research.
Timepoint [4] 310665 0
3 years

Eligibility
Key inclusion criteria
Patients presenting to a study centre and diagnosed with microangiopathic thrombocytopenia as defined by but not limited to anaemia, thrombocytopenia (<150 x10^9/L) and evidence of fragmented red blood cells (schistocytes) on blood film.
Minimum age
1 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
Patients who have non-microangiopathic thrombocytopenia and/or thrombosis where an alternate diagnosis is likely.

Patients who are unable or unwilling to provide informed consent.

Study design
Purpose
Natural history
Duration
Longitudinal
Selection
Defined population
Timing
Retrospective
Statistical methods / analysis
The sample size of this study (300) was selected to provide an observational overview of MAT patients in the APMAT Network. Interpretation of the data will be descriptive and no comparative interpretation of the data will be made. Where applicable, descriptive statistics in the form of mean (+/-SD), median and range for continuous variables and proportions (%) for categorical variables will be conducted.
The original number of 200 was erroneously included in the pervious version.

Recruitment
Recruitment status
Recruiting
Date of first participant enrolment
Anticipated
22/09/2014
Actual
1/01/2015
Date of last participant enrolment
Anticipated
31/12/2022
Actual
Date of last data collection
Anticipated
30/03/2022
Actual
Sample size
Target
300
Accrual to date
81
Final
Recruitment in Australia
Recruitment state(s)
ACT,NSW,QLD,SA,WA,VIC
Recruitment hospital [1] 2992 0
The Alfred - Prahran
Recruitment hospital [2] 2993 0
Calvary Mater Newcastle - Waratah
Recruitment hospital [3] 2994 0
Prince of Wales Hospital - Randwick
Recruitment hospital [4] 2995 0
The Royal Adelaide Hospital - Adelaide
Recruitment hospital [5] 2996 0
Royal Prince Alfred Hospital - Camperdown
Recruitment hospital [6] 2997 0
Princess Alexandra Hospital - Woolloongabba
Recruitment hospital [7] 2998 0
Liverpool Hospital - Liverpool
Recruitment hospital [8] 2999 0
Austin Health - Austin Hospital - Heidelberg
Recruitment hospital [9] 3000 0
Royal Melbourne Hospital - City campus - Parkville
Recruitment hospital [10] 5785 0
St Vincent's Private Hospital (Darlinghurst) - Darlinghurst
Recruitment hospital [11] 5786 0
Westmead Hospital - Westmead
Recruitment hospital [12] 5787 0
Hollywood Private Hospital - Nedlands
Recruitment hospital [13] 21832 0
The Canberra Hospital - Garran
Recruitment postcode(s) [1] 36889 0
2605 - Garran
Recruitment outside Australia
Country [1] 6356 0
China
State/province [1] 6356 0
Country [2] 6357 0
Taiwan, Province Of China
State/province [2] 6357 0
Country [3] 6358 0
Japan
State/province [3] 6358 0
Country [4] 6359 0
Korea, Republic Of
State/province [4] 6359 0
Country [5] 6360 0
Singapore
State/province [5] 6360 0
Country [6] 6361 0
Malaysia
State/province [6] 6361 0
Country [7] 6362 0
India
State/province [7] 6362 0
Country [8] 6363 0
Hong Kong
State/province [8] 6363 0
Country [9] 6364 0
Thailand
State/province [9] 6364 0
Country [10] 6365 0
New Zealand
State/province [10] 6365 0

Funding & Sponsors
Funding source category [1] 289966 0
Other Collaborative groups
Name [1] 289966 0
Australasian Society of Thrombosis and Haemostasis (ASTH)
Country [1] 289966 0
Australia
Funding source category [2] 289967 0
Commercial sector/Industry
Name [2] 289967 0
Alexion Pharmaceuticals Australasia Pty Limited
Country [2] 289967 0
Australia
Funding source category [3] 289968 0
Commercial sector/Industry
Name [3] 289968 0
Takeda
Country [3] 289968 0
United States of America
Primary sponsor type
Other Collaborative groups
Name
ASTH
Address
c/o PO Box 217
Glen Iris
Victoria 3146
Country
Australia
Secondary sponsor category [1] 288656 0
Other Collaborative groups
Name [1] 288656 0
Asia-Pacific Society on Thrombosis and Haemostasis (APSTH)
Address [1] 288656 0
c/o PO Box 217
Glen Iris
Victoria 3146
Country [1] 288656 0
Australia

Ethics approval
Ethics application status
Approved
Ethics committee name [1] 291678 0
Hollywood Private Hospital Research Ethics Committee
Ethics committee address [1] 291678 0
Ethics committee country [1] 291678 0
Australia
Date submitted for ethics approval [1] 291678 0
13/05/2014
Approval date [1] 291678 0
14/08/2014
Ethics approval number [1] 291678 0
HPH383
Ethics committee name [2] 295003 0
NEAF: Hunter New England Human Research Ethics Committee
Ethics committee address [2] 295003 0
Ethics committee country [2] 295003 0
Australia
Date submitted for ethics approval [2] 295003 0
17/07/2015
Approval date [2] 295003 0
25/02/2016
Ethics approval number [2] 295003 0
15/07/15/3.03
Ethics committee name [3] 295004 0
Health and Disability Ethics Committees
Ethics committee address [3] 295004 0
Ethics committee country [3] 295004 0
New Zealand
Date submitted for ethics approval [3] 295004 0
26/02/2015
Approval date [3] 295004 0
08/05/2015
Ethics approval number [3] 295004 0
15/NTA/25
Ethics committee name [4] 310429 0
Kementerian Keshihatan Malaysis
Ethics committee address [4] 310429 0
Ethics committee country [4] 310429 0
Malaysia
Date submitted for ethics approval [4] 310429 0
05/01/2016
Approval date [4] 310429 0
05/02/2016
Ethics approval number [4] 310429 0
NMRR-14-1883-22766(IIR)

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

Contacts
Principal investigator
Name 51482 0
Prof Ross Baker
Address 51482 0
Perth Blood Institute,
Hollywood Specialist Centre
3/95 Monash Avenue
Nedlands
WA 6009
Country 51482 0
Australia
Phone 51482 0
+61 892004904
Fax 51482 0
Email 51482 0
[email protected]
Contact person for public queries
Name 51483 0
Jim Tiao
Address 51483 0
Perth Blood Institute,
Hollywood Specialist Centre
3/95 Monash Avenue
Nedlands
WA 6009
Country 51483 0
Australia
Phone 51483 0
+61 93601319
Fax 51483 0
Email 51483 0
[email protected]
Contact person for scientific queries
Name 51484 0
Ross Baker
Address 51484 0
Perth Blood Institute,
Hollywood Specialist Centre
3/95 Monash Avenue
Nedlands
WA 6009
Country 51484 0
Australia
Phone 51484 0
+61 892004904
Fax 51484 0
Email 51484 0
[email protected]

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Yes
What data in particular will be shared?
clinical data collected at enrolment
When will data be available (start and end dates)?
IPD availability at the conclusion of the study up to 31/12/2027. Anticipated study conclusion date 31/12/2022.
Available to whom?
data is only available to researchers upon written request and approval by the research study steering committee
Available for what types of analyses?
meta-analysis
How or where can data be obtained?
data is available upon written request and approval by the research study steering committee. Upon approval, data requests can be made by contacting the study coordinator (email: [email protected])


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.