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

Sponsor
Yonsei University (Other)
Overall Status
Recruiting
CT.gov ID
NCT02469181
Collaborator
(none)
20
Enrollment
1
Location
112.4
Anticipated Duration (Months)
0.2
Patients Per Site Per Month

Study Details

Study Description

Brief 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.

Condition or Disease Intervention/Treatment Phase

    Detailed 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

    1. 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

    Study Design

    Study Type:
    Observational
    Anticipated Enrollment :
    20 participants
    Observational Model:
    Case-Only
    Time Perspective:
    Prospective
    Official Title:
    Impact of Enzyme Replacement Therapy on Cardiac Function in Patients With Fabry's Cardiomyopathy : A Prospective Multi-modality Imaging Study Using Diastolic Stress Echocardiography, LV Vortex Flow and Cardiac MRI(RECAFTURE Trial)
    Actual Study Start Date :
    May 21, 2015
    Anticipated Primary Completion Date :
    Oct 1, 2024
    Anticipated Study Completion Date :
    Oct 1, 2024

    Arms and Interventions

    Arm Intervention/Treatment
    FD(Fabry disease) group

    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).

    Outcome Measures

    Primary Outcome Measures

    1. Changes of peak exercise E(velocity of early)/E'(Early diastolic) by diastolic stress echocardiography [1 year after the echocardiography]

    Secondary Outcome Measures

    1. Change of peak VO2 by diastolic stress echocardiography [1 year after the echocardiography]

    2. Change of peak exercise time by diastolic stress echocardiography [1 year after the echocardiography]

    3. LV(left ventricular) vortex flow analysis by contract echocardiography [1 year after the echocardiography]

    4. Changes of extracellular volume by Cardiac MRI(CMR) [1 year after the CMR]

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    16 Years to 75 Years
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    No
    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)

    Contacts and Locations

    Locations

    Site City State Country Postal Code
    1 Division of Cardiology, Yonsei Cardiovascular Hospital, Yonsei University College of Medicine Seoul Korea, Republic of 120-752

    Sponsors and Collaborators

    • Yonsei University

    Investigators

    None specified.

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    None provided.
    Responsible Party:
    Yonsei University
    ClinicalTrials.gov Identifier:
    NCT02469181
    Other Study ID Numbers:
    • 4-2014-0679
    First Posted:
    Jun 11, 2015
    Last Update Posted:
    Jun 24, 2021
    Last Verified:
    Jun 1, 2021
    Additional relevant MeSH terms:
    Fabry Disease
    Cardiomyopathies

    Study Results

    No Results Posted as of Jun 24, 2021