Fabry DiseaseNCT04639999
Impact of MIgalastat TheRApy on CaRdiac Function in patiEnts With Fabry's Cardiomyopathy (MIRACRE-Fabry Trial)
An observational study of Fabry Disease, run by Yonsei University at 1 site in South Korea.
Trial 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. Read more about this study
- Who is it for?
- Ages 16 to 70 years
- Conditions the study is for
- Fabry Disease
- What is being tested?
- Echocardiography
- How many people
- 20 participants
- Where
- 1 siteSouth Korea
- Study dates
- 5 Nov 2020to Apr 2025
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 16 to 70 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Patients aged 16 ~ 70 years with Fabry disease confirmed by enzyme assay and gene test
Who may be able to join
- 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
Who cannot join
- 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)
Trial locations and contacts
1 site in 1 country.
South Korea1 site, 1 recruiting
- Recruiting
Severance hospital
Seoul
Geu-Ru Hong, Ph.D, 82+2-2228-8443, grhong@yuhs.ac
About this study
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.
Read the detailed description
- 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.
- Primary/Secondary Endpoint
- 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.
- 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
- 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.
- Conditions
- Fabry Disease
- Being tested
- Echocardiography
- Medical subject headings
- Fabry Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
EchocardiographyOther
LV vortex flow in Echocardiography
| Study group | Role | What they receive |
|---|---|---|
| Fabry's disease | Fabry's disease patients who were confirmed by enzyme assay and gene study Receives: Other: Echocardiography |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change of peak exercise E/E' by diastolic stress echocardiography
Measured: 2 years
Change of global longitudinal strain
Measured: 2 years
Change of LV vortex flow parameters
Measured: 2 years
Secondary outcomes (10)
Changes of extracellular volume by CMR (T1 mapping)
Measured: 2 years
Evaluation of the degree of the resting LV diastolic function
Measured: 2 years
Evaluation of global and regional LV strain
Measured: 2 years
Evaluation of LV mass index
Measured: 2 years
Evaluation of reduction of peak exercise E/E prime
Measured: 2 years
Changes of quality of life using Short Form 36
Measured: 2 years
Change of peak VO2 by diastolic stress echocardiography
Measured: 2 years
Change of exercise time by diastolic stress echocardiography
Measured: 2 years
Change of AT by diastolic stress echocardiography
Measured: 2 years
Change in T1 baseline (myo, ms) & T1 baseline (blood, ms) T1 postcontrast (myo, ms) & T1 baseline (blood, ms) by CMR
Measured: 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Nov 2020
- Study start5 Nov 2020
- Primary completionJan 2025 (estimated)
- Study completionApr 2025 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Fabry DiseaseStudies of the same condition, open and under way.
- More trials run by Yonsei UniversityThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Case only
- Time perspective
- Prospective
- Enrolment
- 20 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Yonsei University
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Geu-Ru Hong, Ph.D, Principal investigator, Severance Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04639999
- Sponsor's protocol ID
- 4-2020-1038
- Status last verified
- Oct 2021
- First submitted
- 10 Nov 2020
- Last updated
- 14 Oct 2021
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 14 Oct 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Impact of MIgalastat TheRApy on CaRdiac Function in patiEnts With Fabry's Cardiomyopathy (MIRACRE-Fabry Trial)
- ClinicalTrials.gov ID
- NCT04639999
- Acronym / sponsor ID
- 4-2020-1038
- Conditions
- Fabry Disease
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 20 participants (estimated)
- Lead sponsor
- Yonsei UniversityUniversity, hospital or other
- Study start
- 5 Nov 2020 (actual)
- Primary completion
- Jan 2025 (estimated)
- Study completion
- Apr 2025 (estimated)
- First posted
- 23 Nov 2020 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
- 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.
- Primary/Secondary Endpoint
- 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.
- 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
- 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.
Study design
- Observational model
- Case only
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Fabry's disease | – | Other: EchocardiographyFabry's disease patients who were confirmed by enzyme assay and gene study |
Who may qualify
- Ages
- 16 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Patients aged 16 ~ 70 years with Fabry disease confirmed by enzyme assay and gene test
Key inclusion criteria (3 listed)
- 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
Key exclusion criteria (8 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| EchocardiographyLV vortex flow in Echocardiography | Other | Fabry's disease |
Primary outcome
Change of peak exercise E/E' by diastolic stress echocardiography
Time frame: 2 years
Change of global longitudinal strain
Time frame: 2 years
Change of LV vortex flow parameters
Time frame: 2 years
10 secondary outcomes
Changes of extracellular volume by CMR (T1 mapping)
Time frame: 2 years
Evaluation of the degree of the resting LV diastolic function
Time frame: 2 years
Evaluation of global and regional LV strain
Time frame: 2 years
Evaluation of LV mass index
Time frame: 2 years
Evaluation of reduction of peak exercise E/E prime
Time frame: 2 years
Changes of quality of life using Short Form 36
Time frame: 2 years
Change of peak VO2 by diastolic stress echocardiography
Time frame: 2 years
Change of exercise time by diastolic stress echocardiography
Time frame: 2 years
Change of AT by diastolic stress echocardiography
Time frame: 2 years
Change in T1 baseline (myo, ms) & T1 baseline (blood, ms) T1 postcontrast (myo, ms) & T1 baseline (blood, ms) by CMR
Time frame: 2 years
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 Oct 2021.
- Individual participant dataSharing planned: Undecided
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Geu-Ru Hong, Ph.D principal investigator
Severance Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Fabry Disease, run by Yonsei University at 1 site in South Korea.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 14 Oct 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in South Korea. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Geu-Ru Hong, Ph.D (82+2-2228-8443, grhong@yuhs.ac), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change of peak exercise E/E' by diastolic stress echocardiography (time frame: 2 years). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 14 Oct 2021.