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Clinical Trial Details — Status: Recruiting

Administrative data

NCT number NCT03230591
Other study ID # 4-2017-0471
Secondary ID
Status Recruiting
Phase
First received
Last updated
Start date July 12, 2017
Est. completion date January 2025

Study information

Verified date June 2021
Source Yonsei University
Contact Geu-Ru Hong, MD, Ph.D
Phone 82-2-2228-8443
Email grhong@yuhs.ac
Is FDA regulated No
Health authority
Study type Observational

Clinical Trial Summary

Study Design: This is an observational study. No treatment or intervention will be assigned to the subjects. All patients will receive full standard of care concomitant medication for the treatment of their cardiac condition. 25 patients with genetically confirmed Anderson-Fabry disease who have a plan to start ERT with Agalsidase Alfa will undergo 2D strain, diastolic stress echocardiography, LV vortex flow analysis, and CMR at baseline and after 1 year of treatment with ERT with Agalsidase Alfa for follow-up.


Description:

1. Objectives - The purpose of this study is to evaluate the impact of ERT with Agalsidase Alfa on LV diastolic function and flow in patients with Fabry's cardiomyopathy using LV 2D strain, diastolic stress echocardiography, LV vortex flow and CMR. 2. Primary / Secondary Endpoint 1) Primary endpoint: - Change from baseline in peak exercise E/E' by diastolic stress echocardiography, global longitudinal strain and LV vortex flow parameters at 1 year 2) Secondary endpoints: - Changes from baseline in extracellular volume by CMR (T1 mapping) at 1 year follow up - Changes from baseline in evaluation of the degree of the resting LV diastolic function - Changes from baseline in other echo-parameters; LV mass index, reduction of peak exercise E/E prime at 1 year follow up - Changes from baseline in quality of life using questionnaire ⑤ Change from baseline in peak VO2, exercise time, AT by diastolic stress echocardiography at 1 year follow up ⑥ Change in T1 baseline (myo, ms) & T1 baseline (blood, ms), T1 postcontrast (myo, ms) & T1 baseline (blood, ms) by CMR 3. Study Methods 1) Study Design: This is an observational study. No treatment or intervention will be assigned to the subjects. All patients will receive full standard of care concomitant medication for the treatment of their cardiac condition. 25 patients with genetically confirmed Anderson-Fabry disease who have a plan to start ERT with Agalsidase Alfa will undergo 2D strain, diastolic stress echocardiography, LV vortex flow analysis, and CMR at baseline and after 1 year of treatment with ERT with Agalsidase Alfa for follow-up.


Recruitment information / eligibility

Status Recruiting
Enrollment 25
Est. completion date January 2025
Est. primary completion date January 2025
Accepts healthy volunteers No
Gender All
Age group 16 Years to 75 Years
Eligibility Inclusion Criteria: - Patients aged 16~ 75 years with Fabry disease confirmed by enzyme assay and gene test - Patients who have LVH in 2D echocardiography (end diastolic septum and posterior wall thickness = 12mm) or Patients who present with cardiac changes (indicative of disease progression such as decreased global longitudinal strain on 2D strain echocardiography or low native T1 mapping on CMR) - Patients provided written informed consent to participate in this study Exclusion Criteria: - Contraindication for enzyme replacement treatment with Agalsidase Alfa - Patients who cannot receive supine bicycle stress echocardiography, contrast echocardiography or CMR - Patients with hemodynamically significant valvular heart disease or arrhythmias - Patients who have history of acute myocardial infarction or congestive heart failure with reduced LV ejection fraction of less than 35% - Patients who had any cerebrovascular accident in the prior 6 months - Scheduled or planned surgery in the next 6 months - Patients with chronic liver cirrhosis - Patients who are allergic to contrast agent (e.g. Definity?, Lantheus Medical Imaging, North Billerica, MA, USA)

Study Design


Related Conditions & MeSH terms


Intervention

Other:
Echocardiography
LV vortex flow in Echocardiography

Locations

Country Name City State
Korea, Republic of Division of Cardiology, Yonsei Cardiovascular Hospital, Yonsei University College of Medicine Seoul

Sponsors (1)

Lead Sponsor Collaborator
Yonsei University

Country where clinical trial is conducted

Korea, Republic of, 

Outcome

Type Measure Description Time frame Safety issue
Primary peak exercise E/E' by diastolic stress echocardiography Change from baseline in peak exercise E/E' by diastolic stress echocardiography 1 year
Primary global longitudinal strain 1 year
Secondary extracellular volume by CMR Changes from baseline in extracellular volume by CMR (T1 mapping) at 1 year follow up 1 year
Secondary evaluation of the degree of the resting LV diastolic function Changes from baseline in evaluation of the degree of the resting LV diastolic function 1 year
Secondary quality of life using questionnaire Changes from baseline in quality of life using questionnaire 1 year
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