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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/study/NCT03199001
Registration number
NCT03199001
Ethics application status
Date submitted
19/06/2017
Date registered
26/06/2017
Titles & IDs
Public title
Native T1 Mapping by Cardiovascular Magnetic Resonance Imaging in Rare Diseases
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Scientific title
Native T1 Mapping by Cardiovascular Magnetic Resonance Imaging in Rare Diseases- A New Method to Improve Patient Care
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Secondary ID [1]
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14/0354
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Universal Trial Number (UTN)
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Trial acronym
FABRY400
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Fabry Disease
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Condition category
Condition code
Human Genetics and Inherited Disorders
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Other human genetics and inherited disorders
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Metabolic and Endocrine
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Metabolic disorders
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Metabolic and Endocrine
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0
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Other metabolic disorders
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Other
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0
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Research that is not of generic health relevance and not applicable to specific health categories listed above
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Intervention/exposure
Study type
Observational
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Patient registry
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Target follow-up duration
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Target follow-up type
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Description of intervention(s) / exposure
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Presence of storage in Fabry cardiomyopathy
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Assessment method [1]
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Presence or absence of storage (measured in milliseconds) by T1 mapping by CMR
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Timepoint [1]
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1 hour
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Secondary outcome [1]
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Presence of inflammation in Fabry cardiomyopathy
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Assessment method [1]
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Presence or absence of inflammation (measured in milliseconds) by T2 mapping by CMR
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Timepoint [1]
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1 hour
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Secondary outcome [2]
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Change in storage measure
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Assessment method [2]
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Change in storage measure (measured in milliseconds) by T1 mapping by CMR
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Timepoint [2]
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12 months
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Eligibility
Key inclusion criteria
* Gene-positive Fabry Disease
* Male or female
* Age at least 9 years
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Minimum age
9
Years
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Maximum age
No limit
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Sex
Both males and females
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Can healthy volunteers participate?
No
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Key exclusion criteria
* Any absolute contraindication to CMR
* Pregnancy
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Study design
Purpose
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Duration
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Selection
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Timing
Prospective
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Statistical methods / analysis
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Recruitment
Recruitment status
UNKNOWN
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
19/02/2015
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
19/02/2019
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Actual
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Sample size
Target
400
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Accrual to date
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Final
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Recruitment in Australia
Recruitment state(s)
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Recruitment hospital [1]
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University of Sydney - Sydney
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Recruitment postcode(s) [1]
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- Sydney
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Recruitment outside Australia
Country [1]
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United Kingdom
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State/province [1]
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Birmingham
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Country [2]
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United Kingdom
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State/province [2]
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London
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Funding & Sponsors
Primary sponsor type
Other
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Name
University College, London
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Address
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Country
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Other collaborator category [1]
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Other
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Name [1]
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University Hospital Birmingham
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Address [1]
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Country [1]
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Other collaborator category [2]
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Other
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Name [2]
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University of Sydney
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Address [2]
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Country [2]
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Ethics approval
Ethics application status
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Summary
Brief summary
Fabry Disease (FD) is a rare, X-linked lysosomal storage disorder leading to left ventricular hypertrophy, myocardial fibrosis, arrhythmia and heart failure. Cardiac involvement is the leading cause of death in FD. Treatment with enzyme replacement therapy is expensive, may be poorly targeted and there are difficulties in early detection and disease monitoring. T1 mapping signal change is a potential remarkable biomarker for FD. Fabry400 is a multicentre study aiming to understand the biology of Fabry Disease and its relationship to non-invasive multi parametric mapping by CMR.
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Trial website
https://clinicaltrials.gov/study/NCT03199001
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Trial related presentations / publications
Sado DM, White SK, Piechnik SK, Banypersad SM, Treibel T, Captur G, Fontana M, Maestrini V, Flett AS, Robson MD, Lachmann RH, Murphy E, Mehta A, Hughes D, Neubauer S, Elliott PM, Moon JC. Identification and assessment of Anderson-Fabry disease by cardiovascular magnetic resonance noncontrast myocardial T1 mapping. Circ Cardiovasc Imaging. 2013 May 1;6(3):392-8. doi: 10.1161/CIRCIMAGING.112.000070. Epub 2013 Apr 5. Pica S, Sado DM, Maestrini V, Fontana M, White SK, Treibel T, Captur G, Anderson S, Piechnik SK, Robson MD, Lachmann RH, Murphy E, Mehta A, Hughes D, Kellman P, Elliott PM, Herrey AS, Moon JC. Reproducibility of native myocardial T1 mapping in the assessment of Fabry disease and its role in early detection of cardiac involvement by cardiovascular magnetic resonance. J Cardiovasc Magn Reson. 2014 Dec 5;16(1):99. doi: 10.1186/s12968-014-0099-4. Nappi C, Altiero M, Imbriaco M, Nicolai E, Giudice CA, Aiello M, Diomiaiuti CT, Pisani A, Spinelli L, Cuocolo A. First experience of simultaneous PET/MRI for the early detection of cardiac involvement in patients with Anderson-Fabry disease. Eur J Nucl Med Mol Imaging. 2015 Jun;42(7):1025-31. doi: 10.1007/s00259-015-3036-3. Epub 2015 Mar 26. Vijapurapu R, Nordin S, Baig S, Liu B, Rosmini S, Augusto J, Tchan M, Hughes DA, Geberhiwot T, Moon JC, Steeds RP, Kozor R. Global longitudinal strain, myocardial storage and hypertrophy in Fabry disease. Heart. 2019 Mar;105(6):470-476. doi: 10.1136/heartjnl-2018-313699. Epub 2018 Oct 3.
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Public notes
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Contacts
Principal investigator
Name
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Address
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Country
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Phone
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Fax
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Email
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Contact person for public queries
Name
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James Moon, MD
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Address
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Country
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Phone
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Fax
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Email
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[email protected]
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Contact person for scientific queries
Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
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No/undecided IPD sharing reason/comment
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What supporting documents are/will be available?
No Supporting Document Provided
Results publications and other study-related documents
No documents have been uploaded by study researchers.
Results not provided in
https://clinicaltrials.gov/study/NCT03199001