Fabry Disease Clinical Trial
Official title:
Association of the Pulsatility Index and Vasomotor Reactivity With White Matter Lesions in Brain MRI of Fabry Disease Patients
Verified date | November 2023 |
Source | National and Kapodistrian University of Athens |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Observational |
We hypothesize that Fabry disease - FD is associated with elevated vascular resistance induced by cerebral small-vessel disease, indicating increased distal resistance to blood flow. The findings of this study may be used as a precursor for neuroimaging manifestations related to stroke in FD patients.
Status | Completed |
Enrollment | 45 |
Est. completion date | January 20, 2023 |
Est. primary completion date | July 1, 2022 |
Accepts healthy volunteers | Accepts Healthy Volunteers |
Gender | All |
Age group | 16 Years to 70 Years |
Eligibility | Inclusion Criteria: Fabry disease diagnosis, genetically confirmed Age> 16 years Exclusion Criteria: Insufficient temporal bone window MRI contra-indication Inability to cooperate for breath-holding test Detection of atrial fibrillation Refuse to sing informed consent |
Country | Name | City | State |
---|---|---|---|
Greece | Second Department of Neurology, "Attikon" University Hospital, School of Medicine, National and Kapodistrian University of Athens, Athens, Greece | Athens |
Lead Sponsor | Collaborator |
---|---|
National and Kapodistrian University of Athens |
Greece,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Prevalence of elevated pulsatility index of FD patients | to assess the prevalence of elevated pulsatility index in FD patients at a single time point. | 2 years | |
Primary | Prevalence of elevated vasomotor reactivity in FD patients. | to assess the prevalence of elevated vasomotor reactivity in FD patients at a single time point. | 2 years | |
Secondary | Association of pulsalitily index with leukoencephalopathy in FD patients. | To associate the pulsality index measured by TCD and TCCD with the presence of white matter lesions in brain MRI of FD patients. | 2 years | |
Secondary | Association of vasomotor reactivity with leukoencephalopathy in FD patients. | To associate the vasomotor reactivity measured by TCD and TCCD with the presence of white matter lesions in brain MRI of FD patients. | 2 years | |
Secondary | ?o compare the pulsatility index measured in FD patients against corresponding prospectively collected data from healthy individuals, stratified by age and sex. | For the secondary outcome, age and sex-matched healthy controls will be consecutively enrolled. After clinical evaluation, healthy controls will present no FD-associated manifestations, hence they will be FD negative. In addition, brain MRI will be performed and subjects with white matter disease and leukoencephalopathy will be excluded. Included controls will be leukoencephalopathy negative. TCD and TCCD evaluation will be performed in healthy controls, in order to measure pulsatility index and compare the results against FD patients. | 2 years | |
Secondary | ?o compare vasomotor reactivity measured in FD patients against corresponding prospectively collected data from healthy individuals, stratified by age and sex. | For the secondary outcome, age and sex-matched healthy controls will be consecutively enrolled. After clinical evaluation, healthy controls will present no FD-associated manifestations, hence they will be FD negative. In addition, brain MRI will be performed and subjects with white matter disease and leukoencephalopathy will be excluded. Included controls will be leukoencephalopathy negative. TCD and TCCD evaluation will be performed in healthy controls, in order to measure vasomotor reactivity and compare the results against FD patients. | 2 years |
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