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

Last updated: June 23, 2021
Sponsor: Yonsei University
Overall Status: Active - Recruiting

Phase

N/A

Condition

Fabry Disease

Kidney Disease

Circulation Disorders

Treatment

N/A

Clinical Study ID

NCT02469181
4-2014-0679
  • Ages 16-75
  • All Genders

Study 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.

Eligibility Criteria

Inclusion

Inclusion Criteria:

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

Exclusion

Exclusion Criteria:

  • Contraindication for agalsidase beta enzyme replacement treatment
  • Patients who cannot perform supine bicycle stress echocardiography, contrastechocardiography or cardiac MRI
  • Hemodynamically significant valvular heart disease or arrythmias
  • History of acute myocardial infarction or congestive heart failure with reduced LVejection 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)

Study Design

Total Participants: 20
Study Start date:
May 21, 2015
Estimated Completion Date:
October 31, 2024

Study 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.

      1. Secondary endpoint

    • Changes of extracellular volume by CMR(T1 mapping) at 1 year follow up (RECAP-F trial) ② Evaluation of the degree of the resting LV diastolic function ③ 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 ⑤ Change of 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 : 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).

  1. Study procedures : Examinations as described below will be done before ERT and 1 year later

Connect with a study center

  • Division of Cardiology, Yonsei Cardiovascular Hospital, Yonsei University College of Medicine

    Seoul, 120-752
    Korea, Republic of

    Active - Recruiting

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