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

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ClinicalTrials.gov Identifier: NCT02469181
Recruitment Status : Recruiting
First Posted : June 11, 2015
Last Update Posted : January 11, 2019
Sponsor:
Information provided by (Responsible Party):
Yonsei University

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
Fabry's Disease

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.

      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

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Study Type : Observational
Estimated 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)
Study Start Date : October 2014
Estimated Primary Completion Date : October 2020
Estimated Study Completion Date : October 2020


Group/Cohort
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).



Primary Outcome Measures :
  1. Changes of peak exercise E(velocity of early)/E'(Early diastolic) by diastolic stress echocardiography [ Time Frame: 1 year after the echocardiography ]

Secondary Outcome Measures :
  1. Change of peak VO2 by diastolic stress echocardiography [ Time Frame: 1 year after the echocardiography ]
  2. Change of peak exercise time by diastolic stress echocardiography [ Time Frame: 1 year after the echocardiography ]
  3. LV(left ventricular) vortex flow analysis by contract echocardiography [ Time Frame: 1 year after the echocardiography ]
  4. Changes of extracellular volume by Cardiac MRI(CMR) [ Time Frame: 1 year after the CMR ]

Biospecimen Retention:   Samples Without DNA
Lyso GL-3


Information from the National Library of Medicine

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Ages Eligible for Study:   20 Years to 75 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Patients aged 20~75years with Fabry's disease who were confirmed by enzyme assay and gene study
Criteria

Inclusion Criteria:

  • Patients aged 20~75 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)

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02469181


Contacts
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Contact: Geu Ru Hong, M.D., Ph.D. 82-2-2228-8443 grhong@yuhs.ac

Locations
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Korea, Republic of
Division of Cardiology, Yonsei Cardiovascular Hospital, Yonsei University College of Medicine Recruiting
Seoul, Korea, Republic of, 120-752
Contact: Geu-Ru Hong, MD, PhD    82-2-2228-8443    GRHONG@yuhs.ac   
Sponsors and Collaborators
Yonsei University
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Responsible Party: Yonsei University
ClinicalTrials.gov Identifier: NCT02469181    
Other Study ID Numbers: 4-2014-0679
First Posted: June 11, 2015    Key Record Dates
Last Update Posted: January 11, 2019
Last Verified: January 2019
Additional relevant MeSH terms:
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Fabry Disease
Cardiomyopathies
Heart Diseases
Cardiovascular Diseases
Sphingolipidoses
Lysosomal Storage Diseases, Nervous System
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Cerebral Small Vessel Diseases
Cerebrovascular Disorders
Vascular Diseases
Genetic Diseases, X-Linked
Genetic Diseases, Inborn
Metabolism, Inborn Errors
Lipidoses
Lipid Metabolism, Inborn Errors
Lysosomal Storage Diseases
Metabolic Diseases
Lipid Metabolism Disorders