Impact of Enzyme Replacement Therapy on Cardiac Function in Patients With Fabry's Cardiomyopathy (RECAFTURE Trial)

  • STATUS
    Recruiting
  • End date
    Oct 4, 2024
  • participants needed
    20
  • sponsor
    Yonsei University
Updated on 4 July 2021
cardiomyopathy
replacement therapy
hypertrophy
vortex

Summary

The purpose of this study is to evaluate the impact of ERT on LV diastolic function and flow in patients with Fabry's cardiomyopathy using diastolic stress echocardiography, LV vortex flow and CMR.

Description

  1. Objectives -The purpose of this study is to evaluate the impact of ERT on LV diastolic function and flow in patients with Fabry's cardiomyopathy using diastolic stress echocardiography, LV vortex flow and CMR.
  2. Primary / Secondary Endpoint 1) Primary endpoint
    • Changes of peak exercise E/E' by diastolic stress echocardiography (RECAP-F STRESS trial) and LV vortex flow parameters (RECAP-F FLOW trial) at 1 year follow up. 2) Secondary endpoint
    • Changes of extracellular volume by CMR(T1 mapping) at 1 year follow up (RECAP-F trial) Evaluation of the degree of the resting LV diastolic function Other echo-parameters; LV mass index at baseline, 1 year follow up, reduction of peak exercise E/E prime at 1 year follow up
    • Changes of quality of life using questionnaire Change of peak VO2, exercise time, AT by diastolic stress echocardiography at 1 year follow up 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 : 28 patients with newly diagnosed genetically confirmed Anderson-Fabry's disease will undergo diastolic stress echocardiography, LV vortex flow analysis, and cardiac MRI before enzyme replacement therapy(ERT) (baseline study) and after 1 year of treatment with agalsidese beta at the dose of 1mg/kg (follow-up study). 2) Study procedures : Examinations as described below will be done before ERT and 1 year later

Details
Condition Fabry's Disease, Fabry Disease
Clinical Study IdentifierNCT02469181
SponsorYonsei University
Last Modified on4 July 2021

Eligibility

Yes No Not Sure

Inclusion Criteria

Patients aged 16~75 years with Fabry's disease who were confirmed by enzyme assay and gene study
All patients should have LV hypertrophy in 2D echocardiography (end diastolic septum and posterior wall thickness 12mm)
Patients provided with the written, informed consent to participate in this study

Exclusion Criteria

Contraindication for agalsidase beta enzyme replacement treatment
Patients who cannot perform supine bicycle stress echocardiography, contrast echocardiography or cardiac MRI
Hemodynamically significant valvular heart disease or arrythmias
History of acute myocardial infarction or congestive heart failure with reduced LV ejection fraction of less than 35%
CVA in the prior 6 months
Scheduled or planned surgery in the next 6 months
Chronic liver cirrhosis
Allergy to contrast agent (Definity, Lantheus Medical Imaging, North Billerica, MA, USA)
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