Fabry's DiseaseNCT02469181
Impact of Enzyme Replacement Therapy on Cardiac Function in Patients With Fabry's Cardiomyopathy (RECAFTURE Trial)
An observational study of Fabry's Disease, run by Yonsei University at 1 site in South Korea.
Trial 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. Read more about this study
- Who is it for?
- Ages 16 to 75 years
- Conditions the study is for
- Fabry's Disease
- How many people
- 20 participants
- Where
- 1 siteSouth Korea
- Study dates
- 21 May 2015to Oct 2024
- 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 75 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Patients aged 16~75years with Fabry's disease who were confirmed by enzyme assay and gene study
Who may be able to join
- 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
Who cannot join
- 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)
Trial locations and contacts
1 site in 1 country.
South Korea1 site, 1 recruiting
- Recruiting
Division of Cardiology, Yonsei Cardiovascular Hospital, Yonsei University College of Medicine
Seoul, 120-752
Geu-Ru Hong, MD, PhD, 82-2-2228-8443, GRHONG@yuhs.ac
About this study
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)
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.
Read the detailed description
- 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.
- 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.
- 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
- 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).
- Study procedures : Examinations as described below will be done before ERT and 1 year later
- Conditions
- Fabry's Disease
- Medical subject headings
- Fabry Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| 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). |
What is being measured
The primary outcome is the main question the study is designed to answer.
Changes of peak exercise E(velocity of early)/E'(Early diastolic) by diastolic stress echocardiography
Measured: 1 year after the echocardiography
Secondary outcomes (4)
Change of peak VO2 by diastolic stress echocardiography
Measured: 1 year after the echocardiography
Change of peak exercise time by diastolic stress echocardiography
Measured: 1 year after the echocardiography
LV(left ventricular) vortex flow analysis by contract echocardiography
Measured: 1 year after the echocardiography
Changes of extracellular volume by Cardiac MRI(CMR)
Measured: 1 year after the CMR
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Jun 2015 (estimated)
- Study start21 May 2015
- Primary completionOct 2024 (estimated)
- Study completionOct 2024 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Fabry's 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
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02469181
- Sponsor's protocol ID
- 4-2014-0679
- Status last verified
- Jun 2021
- First submitted
- 3 Jun 2015
- Last updated
- 24 Jun 2021
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 24 Jun 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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)
- ClinicalTrials.gov ID
- NCT02469181
- Acronym / sponsor ID
- 4-2014-0679
- Conditions
- Fabry's Disease
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 20 participants (estimated)
- Lead sponsor
- Yonsei UniversityUniversity, hospital or other
- Study start
- 21 May 2015 (actual)
- Primary completion
- Oct 2024 (estimated)
- Study completion
- Oct 2024 (estimated)
- First posted
- 11 Jun 2015 (estimated)
- 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.
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
- 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.
- 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.
- 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
- 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).
- Study procedures : Examinations as described below will be done before ERT and 1 year later
Study design
- Observational model
- Case only
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| 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). |
Who may qualify
- Ages
- 16 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Patients aged 16~75years with Fabry's disease who were confirmed by enzyme assay and gene study
Key inclusion criteria (3 listed)
- 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
Key exclusion criteria (8 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Changes of peak exercise E(velocity of early)/E'(Early diastolic) by diastolic stress echocardiography
Time frame: 1 year after the echocardiography
4 secondary outcomes
Change of peak VO2 by diastolic stress echocardiography
Time frame: 1 year after the echocardiography
Change of peak exercise time by diastolic stress echocardiography
Time frame: 1 year after the echocardiography
LV(left ventricular) vortex flow analysis by contract echocardiography
Time frame: 1 year after the echocardiography
Changes of extracellular volume by Cardiac MRI(CMR)
Time frame: 1 year after the CMR
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Jun 2021.
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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.
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's 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 24 Jun 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, M.D., 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?
Changes of peak exercise E(velocity of early)/E'(Early diastolic) by diastolic stress echocardiography (time frame: 1 year after the echocardiography). 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 24 Jun 2021.