Fabry DiseaseNCT03230591
Impact of Agalsidase Alfa Therapy on Cardiac funcTION in Patients With Fabry's Cardiomyopathy
An observational study of Fabry Disease, run by Yonsei University at 1 site in South Korea.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 16 to 75 years
- Conditions the study is for
- Fabry Disease
- What is being tested?
- Echocardiography
- How many people
- 25 participants
- Where
- 1 siteSouth Korea
- Study dates
- 12 Jul 2017to Jan 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 75 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Patients aged 16 ~ 75 years with Fabry disease confirmed by enzyme assay and gene test
Who may be able to join
- Patients aged 16~ 75 years with Fabry disease confirmed by enzyme assay and gene test
- Patients who have LVH 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 CMR)
- Patients provided written informed consent to participate in this study
Who cannot join
- Contraindication for enzyme replacement treatment with Agalsidase Alfa
- Patients who cannot receive supine bicycle stress echocardiography, contrast echocardiography or CMR
- Patients with hemodynamically significant valvular heart disease or arrhythmias
- Patients who have history of acute myocardial infarction or congestive heart failure with reduced LV ejection fraction of less than 35%
- Patients who had any cerebrovascular accident in the prior 6 months
- Scheduled or planned surgery in the next 6 months
- Patients with chronic liver cirrhosis
- Patients who are allergic to contrast agent (e.g. 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, 03722
Geu-Ru Hong, MD, PhD, 82-2-2228-8443, GRHONG@yuhs.ac
About this study
Official title: Impact of Agalsidase Alfa Therapy on Cardiac funcTION in Patients With Fabry's Cardiomyopathy (ACTION-Fabry Trial)
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. 25 patients with genetically confirmed Anderson-Fabry disease who have a plan to start ERT with Agalsidase Alfa will undergo 2D strain, diastolic stress echocardiography, LV vortex flow analysis, and CMR at baseline and after 1 year of treatment with ERT with Agalsidase Alfa for follow-up.
Read the detailed description
- Objectives - The purpose of this study is to evaluate the impact of ERT with Agalsidase Alfa 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 1) Primary endpoint:
- Change from baseline in peak exercise E/E' by diastolic stress echocardiography, global longitudinal strain and LV vortex flow parameters at 1 year 2) Secondary endpoints:
- Changes from baseline in extracellular volume by CMR (T1 mapping) at 1 year follow up
- Changes from baseline in evaluation of the degree of the resting LV diastolic function
- Changes from baseline in other echo-parameters; LV mass index, reduction of peak exercise E/E prime at 1 year follow up
- Changes from baseline in quality of life using questionnaire ⑤ Change from baseline in 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: 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. 25 patients with genetically confirmed Anderson-Fabry disease who have a plan to start ERT with Agalsidase Alfa will undergo 2D strain, diastolic stress echocardiography, LV vortex flow analysis, and CMR at baseline and after 1 year of treatment with ERT with Agalsidase Alfa for follow-up.
- Conditions
- Fabry Disease
- Being tested
- Echocardiography
- Keywords
- Fabry's disease, Agalsidase Alfa, Cardiomyopathy, Enzyme replacement therapy, Diastolic stress echocardiography, LV vortex flow, Cardiac MRI
- Medical subject headings
- Fabry Disease, Cardiomyopathies
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.
peak exercise E/E' by diastolic stress echocardiography
Change from baseline in peak exercise E/E' by diastolic stress echocardiography
Measured: 1 year
global longitudinal strain
Measured: 1 year
Secondary outcomes (3)
extracellular volume by CMR
Changes from baseline in extracellular volume by CMR (T1 mapping) at 1 year follow up
Measured: 1 year
evaluation of the degree of the resting LV diastolic function
Changes from baseline in evaluation of the degree of the resting LV diastolic function
Measured: 1 year
quality of life using questionnaire
Changes from baseline in quality of life using questionnaire
Measured: 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Jul 2017
- Study start12 Jul 2017
- Primary completionJan 2025 (estimated)
- Study completionJan 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
- 25 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
- NCT03230591
- Sponsor's protocol ID
- 4-2017-0471
- Status last verified
- Jun 2021
- First submitted
- 21 Jul 2017
- Last updated
- 24 Jun 2021
- Results posted
- 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 24 Jun 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Impact of Agalsidase Alfa Therapy on Cardiac funcTION in Patients With Fabry's Cardiomyopathy (ACTION-Fabry Trial)
- ClinicalTrials.gov ID
- NCT03230591
- Acronym / sponsor ID
- 4-2017-0471
- Conditions
- Fabry Disease
- Keywords
- Fabry's disease, Agalsidase Alfa, Cardiomyopathy, Enzyme replacement therapy, Diastolic stress echocardiography, LV vortex flow, Cardiac MRI
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 25 participants (estimated)
- Lead sponsor
- Yonsei UniversityUniversity, hospital or other
- Study start
- 12 Jul 2017 (actual)
- Primary completion
- Jan 2025 (estimated)
- Study completion
- Jan 2025 (estimated)
- First posted
- 26 Jul 2017 (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.
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. 25 patients with genetically confirmed Anderson-Fabry disease who have a plan to start ERT with Agalsidase Alfa will undergo 2D strain, diastolic stress echocardiography, LV vortex flow analysis, and CMR at baseline and after 1 year of treatment with ERT with Agalsidase Alfa for follow-up.
Detailed description
- Objectives - The purpose of this study is to evaluate the impact of ERT with Agalsidase Alfa 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 1) Primary endpoint:
- Change from baseline in peak exercise E/E' by diastolic stress echocardiography, global longitudinal strain and LV vortex flow parameters at 1 year 2) Secondary endpoints:
- Changes from baseline in extracellular volume by CMR (T1 mapping) at 1 year follow up
- Changes from baseline in evaluation of the degree of the resting LV diastolic function
- Changes from baseline in other echo-parameters; LV mass index, reduction of peak exercise E/E prime at 1 year follow up
- Changes from baseline in quality of life using questionnaire ⑤ Change from baseline in 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: 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. 25 patients with genetically confirmed Anderson-Fabry disease who have a plan to start ERT with Agalsidase Alfa will undergo 2D strain, diastolic stress echocardiography, LV vortex flow analysis, and CMR at baseline and after 1 year of treatment with ERT with Agalsidase Alfa 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 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Patients aged 16 ~ 75 years with Fabry disease confirmed by enzyme assay and gene test
Key inclusion criteria (3 listed)
- Patients aged 16~ 75 years with Fabry disease confirmed by enzyme assay and gene test
- Patients who have LVH 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 CMR)
- Patients provided written informed consent to participate in this study
Key exclusion criteria (8 listed)
- Contraindication for enzyme replacement treatment with Agalsidase Alfa
- Patients who cannot receive supine bicycle stress echocardiography, contrast echocardiography or CMR
- Patients with hemodynamically significant valvular heart disease or arrhythmias
- Patients who have history of acute myocardial infarction or congestive heart failure with reduced LV ejection fraction of less than 35%
- Patients who had any cerebrovascular accident 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
peak exercise E/E' by diastolic stress echocardiography
Time frame: 1 year
Change from baseline in peak exercise E/E' by diastolic stress echocardiography
global longitudinal strain
Time frame: 1 year
3 secondary outcomes
extracellular volume by CMR
Time frame: 1 year
evaluation of the degree of the resting LV diastolic function
Time frame: 1 year
quality of life using questionnaire
Time frame: 1 year
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.
- 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.
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 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, MD, 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?
peak exercise E/E' by diastolic stress echocardiography (time frame: 1 year). The study lists 2 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 24 Jun 2021.