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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/study/NCT03305250
Registration number
NCT03305250
Ethics application status
Date submitted
21/09/2017
Date registered
9/10/2017
Titles & IDs
Public title
Arrhythmia Burden, Risk of Sudden Cardiac Death and Stroke in Patients With Fabry Disease
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Scientific title
Arrhythmia Burden, Risk of Sudden Cardiac Death and Stroke in Patients With Fabry Disease: the Role of Implantable Loop Recorders
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Secondary ID [1]
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FD-ILR-001
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Universal Trial Number (UTN)
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Trial acronym
RaILRoAD
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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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Other metabolic disorders
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Cardiovascular
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Coronary heart disease
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Cardiovascular
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0
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Diseases of the vasculature and circulation including the lymphatic system
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Cardiovascular
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Other cardiovascular diseases
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Treatment: Devices - Implantable Loop Recorder
Active comparator: Interventional Arm - Using an Implantable Loop Recorder fo continuous rhythm monitoring and home follow-up. This will be combined with standard care procedure, which will include annual ECG, 24 hour Holter/5 day ECG monitoring and further investigation dependent on symptom status.
No intervention: Standard of Care Arm - The standard of care with annual ECG, 24 hour Holter/5 day ECG monitoring and further investigation dependent on symptom status.
Treatment: Devices: Implantable Loop Recorder
An implantable loop recorder (ILR), also known as an insertable cardiac monitor, is a small device (smaller than a AAA battery) that is inserted under the skin on the front of the chest. The ILR is inserted using local anesthetic as an out-patient procedure and lasts approximately 30 minutes. The ILR captures a continuous ECG of your heart activity, which allows doctors to detect any abnormal heart rhythms at any point. If you have the ILR, you will have the device for 3 years, after which it will be removed under local anesthetic during an out-patient procedure, again lasting approximately 30 minutes. The ILR device is completely safe and shouldn't affect your day to day living.
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Intervention code [1]
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Treatment: Devices
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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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First occurrence of atrial fibrillation (AF) requiring anticoagulation
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Assessment method [1]
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This will include all descriptions of AF, which can be defined as:
1. paroxysmal - self-terminating episodes lasting between 48 hours to 7 days
2. persistent - intermittent episodes lasting between 7 days to 1 year
3. permanent - episodes lasting longer than 1 year
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Timepoint [1]
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Total monitoring time period in study - 3 years
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Primary outcome [2]
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First occurrence of bradyarrhythmia requiring cardiac pacing
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Assessment method [2]
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This would include:
1. Symptomatic significant AV block.
2. Mobitz type 2 AV block or complete heart block irrespective of symptoms.
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Timepoint [2]
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Total monitoring time period in study - 3 years
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Primary outcome [3]
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First occurrence of supraventricular arrhythmia requiring drug treatment or ablation.
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Assessment method [3]
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Timepoint [3]
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Total monitoring time period in study - 3 years
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Primary outcome [4]
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First occurrence of non-sustained ventricular tachyarrhythmia requiring drug treatment, ICD implantation or ablation
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Assessment method [4]
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This is classified as three or more ventricular beats at a rate \>120bpm, for a duration of less than 30 seconds.
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Timepoint [4]
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Total monitoring time period in study - 3 years
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Secondary outcome [1]
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Frequency of arrhythmia in patients with and without late gadolinium enhancement (LGE)
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Assessment method [1]
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The study will aim at quantifying the extent of LGE deposited with myocardial tissue on cardiac MRI scanning. This will subsequently be correlated with the burden of arrhythmia detected to assess for potential risk factors.
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Timepoint [1]
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3 years
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Secondary outcome [2]
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Frequency of arrhythmia according to location of myocardial fibrosis (inferolateral vs. non-inferolateral)
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Assessment method [2]
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The study will aim to correlate the location of myocardial fibrosis with the presence or absence of cardiac arrhythmia to define location of fibrosis as a potential risk factor for arrhythmia.
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Timepoint [2]
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3 years
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Secondary outcome [3]
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Frequency of arrhythmia in those patients with a QRS duration greater or less than 120ms
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Assessment method [3]
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Timepoint [3]
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3 years
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Secondary outcome [4]
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Frequency of arrhythmia in those with an atrial size above or below indexed normal range for age and sex
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Assessment method [4]
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Timepoint [4]
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3 years
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Eligibility
Key inclusion criteria
* Patients with genotypically or enzymatically confirmed FD
* Adults > 18 years of age
* Evidence of cardiac involvement from FD involving either:
* Any ECG abnormality associated with FD
* Low T1 on CMR (below centre-specific normal range according to sex)
* LVH on transthoracic echo (defined as MWT >12mm)
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Minimum age
18
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
* Patient with an existing cardiac device (PPM, ICD or ILR).
* Known dual pathology:
* Known coronary artery disease (positive non-invasive imaging, confirmed myocardial infarction, percutaneous or surgical revascularisation). Patients >40 years old with symptoms that could be from coronary artery disease will have this excluded
* Known cardiomyopathy disease causing mutation (e.g. SCN5, MYBPC3)
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Study design
Purpose of the study
Diagnosis
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Allocation to intervention
Randomised controlled trial
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Open (masking not used)
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Who is / are masked / blinded?
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Intervention assignment
Parallel
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Other design features
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Phase
NA
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Active, not recruiting
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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
18/09/2019
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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
1/07/2027
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Actual
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Sample size
Target
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Accrual to date
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Final
169
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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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West Midlands
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Country [2]
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United Kingdom
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State/province [2]
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Cambridge
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Country [3]
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United Kingdom
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State/province [3]
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Cardiff
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Country [4]
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United Kingdom
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State/province [4]
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London
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Country [5]
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United Kingdom
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State/province [5]
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Manchester
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Country [6]
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United Kingdom
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State/province [6]
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Sheffield
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Funding & Sponsors
Primary sponsor type
Other
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Name
University Hospital Birmingham NHS Foundation Trust
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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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Royal Free Hospital NHS Foundation Trust
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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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Northern Care Alliance NHS Foundation Trust
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Address [2]
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Country [2]
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Other collaborator category [3]
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Other
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Name [3]
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University of Sydney
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Address [3]
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Other collaborator category [4]
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Other
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Name [4]
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Cambridge University Hospitals NHS Foundation Trust
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Address [4]
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Other collaborator category [5]
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Government body
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Name [5]
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Cardiff and Vale University Health Board
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Address [5]
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Country [5]
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Other collaborator category [6]
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Other
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Name [6]
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Sheffield Teaching Hospitals NHS Foundation Trust
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Address [6]
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Country [6]
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Ethics approval
Ethics application status
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Summary
Brief summary
Fabry disease (FD) is a genetic disorder that leads to progressive accumulation of fat or 'sphingolipid' within the tissues, including the heart muscle and conductive tissue. Improvements in the detection of FD, together with more organised clinical services for rare diseases, has led to a rapid growth in the disease prevalence. Earlier and more frequent diagnosis of asymptomatic individuals before development of the disease itself has focused attention on early detection of organ involvement and closer monitoring of disease progression. Moreover, the introduction of enzyme replacement therapy within the last two decades has changed the natural history of FD as follows: a) increased life expectancy; b) improved morbidity; c) modification of the main cause of morbidity and mortality from renal (kidney) to cardiovascular (heart) events, including heart failure, abnormal heart rhythms, stroke and sudden death. Although symptoms such as palpitations and blackouts are extremely common, information on the frequency of proven abnormal heart rhythms is limited. In addition, the rate and appropriateness of implantation of life-saving devices is very variable, including pacemakers to boost the heart when too slow and cardio-defibrillators that stop the heart when too fast. The main markers of risk in similar diseases such as hypertrophic cardiomyopathy cannot be used in FD. While patients are routinely followed up in clinic with heart tracings and echocardiography (ultrasound of the heart), a recent small study has emphasised that these tests under-estimate the burden of abnormal heart rhythms in patients with advanced FD. The use of continuous heart monitoring with an implantable loop recorder (ILR) has led to a significant change in treatment in 13 out of 15 of FD patients. The investigators believe that more frequent use of ILRs will identify a greater need for change in therapy in many more patients than currently treated, with the aim of reducing morbidity and mortality in this patient cohort. In addition this will provide valuable data to inform an estimate of future risk for these patients.
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Trial website
https://clinicaltrials.gov/study/NCT03305250
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Trial related presentations / publications
Vijapurapu R, Kozor R, Hughes DA, Woolfson P, Jovanovic A, Deegan P, Rusk R, Figtree GA, Tchan M, Whalley D, Kotecha D, Leyva F, Moon J, Geberhiwot T, Steeds RP. A randomised controlled trial evaluating arrhythmia burden, risk of sudden cardiac death and stroke in patients with Fabry disease: the role of implantable loop recorders (RaILRoAD) compared with current standard practice. Trials. 2019 May 31;20(1):314. doi: 10.1186/s13063-019-3425-1.
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Public notes
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Contacts
Principal investigator
Name
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Richard Steeds, MD
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Address
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University Hospital Birmingham NHS Foundation Trust
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Phone
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Fax
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Email
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Contact person for public queries
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Address
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Phone
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Fax
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Email
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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
There is no plan to make individual participant data available to other researchers. Data analysis conducted using anonymised patient data will be shared through publications.
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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/NCT03305250