Impact of MIgalastat TheRApy on CaRdiac Function in patiEnts With Fabry's Cardiomyopathy (MIRACRE-Fabry Trial)

  • STATUS
    Recruiting
  • End date
    Apr 23, 2025
  • participants needed
    20
  • sponsor
    Yonsei University
Updated on 23 October 2021
cardiomyopathy
strain
hypertrophy
migalastat
vortex

Summary

This is an observational study. No treatment or intervention will be assigned to the subjects. All patients will receive full standard of care concomitant medication for the treatment of their cardiac condition. 20 patients with genetically confirmed Anderson-Fabry disease who have a plan to start Migalastat will undergo 2D strain, diastolic stress echocardiography, LV vortex flow analysis, and CMR at baseline and after 2 year of treatment with Migalastat for follow-up.

Description

  1. Objectives - to evaluate the impact of chaperone therapy on LV diastolic function and flow in patients with Fabry's cardiomyopathy using LV 2D strain, diastolic stress echocardiography, LV vortex flow and CMR.
  2. Primary/Secondary Endpoint
    1. Primary endpoint:
      • Change of peak exercise E/E' by diastolic stress echocardiography, global longitudinal strain and LV vortex flow parameters at 2 year follow up.
    2. Secondary endpoints:
      • Changes of extracellular volume by CMR (T1 mapping) at 2 year follow up
      • Evaluation of the degree of the resting LV diastolic function
      • Evaluation of global and regional LV strain
      • Other echo-parameters; LV mass index at baseline, 2 year follow up, reduction of peak exercise E/E prime at 2 year follow up
      • Changes of quality of life using questionnaire
      • Change of peak VO2, exercise time, AT by diastolic stress echocardiography at 2 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 -Study Design: This is an observational study. No treatment or intervention will be assigned to the subjects. All patients will receive full standard of care concomitant medication for the treatment of their cardiac condition. 20 patients with genetically confirmed Anderson-Fabry disease who have a plan to start Migalastat will undergo 2D strain, diastolic stress echocardiography, LV vortex flow analysis, and CMR at baseline and after 2 year of treatment with Migalastat for follow-up.

Details
Condition Fabry's Disease, Fabry Disease
Treatment Echocardiography
Clinical Study IdentifierNCT04639999
SponsorYonsei University
Last Modified on23 October 2021

Eligibility

Yes No Not Sure

Inclusion Criteria

Patients aged 16 ~ 70 years with Fabry's disease who were confirmed by enzyme assay and gene study
Patients who have LV hypertrophy in 2D echocardiography (end diastolic septum and posterior wall thickness 12mm) or Patients who present with cardiac changes (indicative of disease progression such as decreased global longitudinal strain on 2D strain echocardiography or low native T1 mapping on cardiac MRI) even without LV wall thickness of 12mm
Patients provided with the written, informed consent to participate in this study

Exclusion Criteria

Contraindication for chaperone therapy (Migalastat)
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)
Clear my responses

How to participate?

Step 1 Connect with a study center
What happens next?
  • You can expect the study team to contact you via email or phone in the next few days.
  • Sign up as volunteer to help accelerate the development of new treatments and to get notified about similar trials.

You are contacting

Investigator Avatar

Primary Contact

site

0/250

Additional screening procedures may be conducted by the study team before you can be confirmed eligible to participate.

Learn more

If you are confirmed eligible after full screening, you will be required to understand and sign the informed consent if you decide to enroll in the study. Once enrolled you may be asked to make scheduled visits over a period of time.

Learn more

Complete your scheduled study participation activities and then you are done. You may receive summary of study results if provided by the sponsor.

Learn more

Similar trials to consider

Loading...

Browse trials for

Not finding what you're looking for?

Every year hundreds of thousands of volunteers step forward to participate in research. Sign up as a volunteer and receive email notifications when clinical trials are posted in the medical category of interest to you.

Sign up as volunteer

user name

Added by • 

 • 

Private

Reply by • Private
Loading...

Lorem ipsum dolor sit amet consectetur, adipisicing elit. Ipsa vel nobis alias. Quae eveniet velit voluptate quo doloribus maxime et dicta in sequi, corporis quod. Ea, dolor eius? Dolore, vel!

  The passcode will expire in None.
Loading...

No annotations made yet

Add a private note
  • abc Select a piece of text from the left.
  • Add notes visible only to you.
  • Send it to people through a passcode protected link.
Add a private note