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

NCT ID: NCT02469181

Last Updated: 2021-06-24

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

UNKNOWN

Total Enrollment

20 participants

Study Classification

OBSERVATIONAL

Study Start Date

2015-05-21

Study Completion Date

2024-10-31

Brief Summary

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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.

Detailed Description

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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) 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).

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

Conditions

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Fabry's Disease

Study Design

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Observational Model Type

CASE_ONLY

Study Time Perspective

PROSPECTIVE

Study Groups

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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).

No interventions assigned to this group

Eligibility Criteria

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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)
Minimum Eligible Age

16 Years

Maximum Eligible Age

75 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Yonsei University

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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

Seoul, , South Korea

Site Status RECRUITING

Countries

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South Korea

Central Contacts

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Geu Ru Hong, M.D., Ph.D.

Role: CONTACT

82-2-2228-8443

Facility Contacts

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Geu-Ru Hong, MD, PhD

Role: primary

82-2-2228-8443

Other Identifiers

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4-2014-0679

Identifier Type: -

Identifier Source: org_study_id

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