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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/NCT03199001




Registration number
NCT03199001
Ethics application status
Date submitted
19/06/2017
Date registered
26/06/2017
Date last updated
26/06/2017

Titles & IDs
Public title
Native T1 Mapping by Cardiovascular Magnetic Resonance Imaging in Rare Diseases
Scientific title
Native T1 Mapping by Cardiovascular Magnetic Resonance Imaging in Rare Diseases- A New Method to Improve Patient Care
Secondary ID [1] 0 0
14/0354
Universal Trial Number (UTN)
Trial acronym
FABRY400
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Fabry Disease 0 0
Condition category
Condition code
Other 0 0 0 0
Research that is not of generic health relevance and not applicable to specific health categories listed above
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Metabolic and Endocrine 0 0 0 0
Metabolic disorders
Metabolic and Endocrine 0 0 0 0
Other metabolic disorders

Intervention/exposure
Study type
Observational
Patient registry
Target follow-up duration
Target follow-up type
Description of intervention(s) / exposure
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Presence of storage in Fabry cardiomyopathy - Presence or absence of storage (measured in milliseconds) by T1 mapping by CMR
Timepoint [1] 0 0
1 hour
Secondary outcome [1] 0 0
Presence of inflammation in Fabry cardiomyopathy - Presence or absence of inflammation (measured in milliseconds) by T2 mapping by CMR
Timepoint [1] 0 0
1 hour
Secondary outcome [2] 0 0
Change in storage measure - Change in storage measure (measured in milliseconds) by T1 mapping by CMR
Timepoint [2] 0 0
12 months

Eligibility
Key inclusion criteria
- Gene-positive Fabry Disease

- Male or female

- Age at least 9 years
Minimum age
9 Years
Maximum age
No limit
Gender
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
- Any absolute contraindication to CMR

- Pregnancy

Study design
Purpose
Duration
Selection
Timing
Prospective
Statistical methods / analysis

Recruitment
Recruitment status
Unknown status
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
University of Sydney - Sydney
Recruitment postcode(s) [1] 0 0
- Sydney
Recruitment outside Australia
Country [1] 0 0
United Kingdom
State/province [1] 0 0
Birmingham
Country [2] 0 0
United Kingdom
State/province [2] 0 0
London

Funding & Sponsors
Primary sponsor type
Other
Name
University College, London
Address
Country
Other collaborator category [1] 0 0
Other
Name [1] 0 0
University Hospital Birmingham
Address [1] 0 0
Country [1] 0 0
Other collaborator category [2] 0 0
Other
Name [2] 0 0
University of Sydney
Address [2] 0 0
Country [2] 0 0

Ethics approval
Ethics application status

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.
Trial website
https://clinicaltrials.gov/show/NCT03199001
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;16:99. 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.
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
James Moon, MD
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
j.moon@ucl.ac.uk
Contact person for scientific queries

Summary results
For IPD and results data, please see https://clinicaltrials.gov/show/NCT03199001