Fabry DiseaseGALAFABNCT03949920
A Study of Migalastat in Fabry Disease
An observational study of Fabry Disease, run by Manchester University NHS Foundation Trust at 2 sites in United Kingdom.
Trial summary
Fabry disease is a rare metabolic condition characterised by the widespread deposition of sphingolipids in multiple organ systems. Cardiac involvement is common, it occurs in fifty percent of patients and it is the leading cause of death. Read more about this study
- Who is it for?
- Ages 16 years and older
- Conditions the study is for
- Fabry Disease
- What is being tested?
- Migalastat
- How many people
- 21 participants
- Where
- 2 sitesUnited Kingdom
- Study dates
- 16 May 2019to 31 Dec 2023
- 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 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Patients with Fabry disease commencing Migalastat as part of routine care.
Who may be able to join
- Confirmed Fabry disease Aged 16 or over Beginning clinical treatment with Migalastat
Who cannot join
- Contraindication to cardiac MRI
Trial locations and contacts
2 sites in 1 country.
United Kingdom2 sites
Manchester University Foundation Trust
Manchester, M139LT
Salford Royal NHS Foundation Trust
Manchester, M6 8HD
About this study
Official title: A Prospective Observational Study Investigating the Impact of Migalastat on Cardiovascular Structure and Function in Fabry Disease
Fabry disease is a rare metabolic condition characterised by the widespread deposition of sphingolipids in multiple organ systems. Cardiac involvement is common, it occurs in fifty percent of patients and it is the leading cause of death. Despite this, heart and blood vessel (cardiovascular system) manifestations of Fabry disease remain poorly characterised, and it remains unclear which patients benefit from therapy, or when therapy should be initiated. Migalastat is increasingly used to treat fabry disease however the impact of Migalastat on the cardiovascular system is poorly understood. Detailed assessment of the impact of Migalastat on heart and blood vessel structure and function is urgently needed. This observational study will use state of the art, non-invasive investigations to provide greater understanding of the cardiovascular manifestations of Fabry disease and the effects of Migalastat. It will provide insight into which patients respond more effectively to Migalastat, which in turn will facilitate personalisation of therapy, optimisation of the timing of therapy initiation and more cost-effective care.
Read the detailed description
This is a prospective longitudinal observational study of patients starting Migalastat as part of routine care. Participants will be recruited from outpatient clinics and have a number of investigations before starting therapy and after twelve months of therapy.
Investigations will include a detailed assessment of symptoms and clinical features, blood tests, echocardiography, detailed cardiac MRI scans, heart rhythm monitoring and exercise capacity assessment.
Parameters will be assessed at baseline and at twelve months.
- Conditions
- Fabry Disease
- Being tested
- Migalastat
- Keywords
- Fabry, Anderson-Fabry, Migalastat
- Medical subject headings
- Fabry Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MigalastatDrug
Participants will be starting Migalastat as part of clinical care. Their initiation onto Migalastat is not determined by this study or it's protocol. Interventions as part of the study will be limited to the study investigations.
What is being measured
The primary outcome is the main question the study is designed to answer.
Indexed Left Ventricular Mass (grams/m2)
LV mass, indexed to body surface area, assessed using cardiac MRI.
Measured: 12 months
Secondary outcomes (12)
Change in BSA-indexed LV volumes measured using cardiac MRI, from baseline to 12 months (mls/m2)
Measured: 12 months
Change LV ejection fraction, measured using cardiac MRI (%)
Measured: 12 months
Change in myocardial extracellular volume, measured using cardiac MRI (%)
Measured: 12 months
Change in myocardial tissue T1 and T2 times measured using cardiac MRI (ms)
Measured: 12 months
Change in pulmonary artery systolic pressure, measured using echocardiography (mmHg)
Measured: 12 months
Change in myocardial PCr/ATP ratio, measured using cardiac MRI spectroscopy (ratio)
Measured: 12 months
Change in the number of extra heart beats in a 24 hour period, measured using ambulatory heart monitoring. (number)
Measured: 12 months
Change in exercise capacity - Six minute walk test (meters)
Measured: 12 months
20. Change in health status (quality of life), measured using change in SF-36 score. (score)
Measured: 12 months
Change in Lyso-GB3 blood test levels (nmol/L)
Measured: 12 months
Change in Troponin blood test levels (ng/L)
Measured: 12 months
Change in NT pro BNP blood test levels (pg/L)
Measured: 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 May 2019
- Study start16 May 2019
- Primary completion31 Dec 2023 (estimated)
- Study completion31 Dec 2023 (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.
- 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
- Cohort
- Time perspective
- Prospective
- Enrolment
- 21 participants (actual)
Sponsor and investigators
- Lead sponsor
- Manchester University NHS Foundation Trust
- Sponsor type
- Other government
- Collaborators
- Northern Care Alliance NHS Foundation Trust
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03949920
- Sponsor's protocol ID
- B00544
- Other IDs
- 19/NW/0099
- Acronym
- GALAFAB
- Status last verified
- May 2023
- First submitted
- 18 Apr 2019
- Last updated
- 31 May 2023
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No: There is no plan to share IPD with other researchers.
Study at a glance
From the registry record, last updated by the sponsor on 31 May 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Prospective Observational Study Investigating the Impact of Migalastat on Cardiovascular Structure and Function in Fabry Disease
- ClinicalTrials.gov ID
- NCT03949920
- Acronym / sponsor ID
- GALAFAB / B00544
- Conditions
- Fabry Disease
- Keywords
- Fabry, Anderson-Fabry, Migalastat
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 21 participants (actual)
- Lead sponsor
- Manchester University NHS Foundation TrustOther government
- Collaborators
- Northern Care Alliance NHS Foundation Trust
- Study start
- 16 May 2019 (actual)
- Primary completion
- 31 Dec 2023 (estimated)
- Study completion
- 31 Dec 2023 (estimated)
- First posted
- 14 May 2019 (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.
Fabry disease is a rare metabolic condition characterised by the widespread deposition of sphingolipids in multiple organ systems. Cardiac involvement is common, it occurs in fifty percent of patients and it is the leading cause of death. Despite this, heart and blood vessel (cardiovascular system) manifestations of Fabry disease remain poorly characterised, and it remains unclear which patients benefit from therapy, or when therapy should be initiated. Migalastat is increasingly used to treat fabry disease however the impact of Migalastat on the cardiovascular system is poorly understood. Detailed assessment of the impact of Migalastat on heart and blood vessel structure and function is urgently needed. This observational study will use state of the art, non-invasive investigations to provide greater understanding of the cardiovascular manifestations of Fabry disease and the effects of Migalastat. It will provide insight into which patients respond more effectively to Migalastat, which in turn will facilitate personalisation of therapy, optimisation of the timing of therapy initiation and more cost-effective care.
Detailed description
This is a prospective longitudinal observational study of patients starting Migalastat as part of routine care. Participants will be recruited from outpatient clinics and have a number of investigations before starting therapy and after twelve months of therapy.
Investigations will include a detailed assessment of symptoms and clinical features, blood tests, echocardiography, detailed cardiac MRI scans, heart rhythm monitoring and exercise capacity assessment.
Parameters will be assessed at baseline and at twelve months.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
Who may qualify
- Ages
- 16 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Patients with Fabry disease commencing Migalastat as part of routine care.
Key inclusion criteria (1 listed)
- Confirmed Fabry disease Aged 16 or over Beginning clinical treatment with Migalastat
Key exclusion criteria (1 listed)
- Contraindication to cardiac MRI
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 |
|---|---|---|
| MigalastatParticipants will be starting Migalastat as part of clinical care. Their initiation onto Migalastat is not determined by this study or it's protocol. Interventions as part of the study will be limited to the study investigations. | Drug | – |
Primary outcome
Indexed Left Ventricular Mass (grams/m2)
Time frame: 12 months
LV mass, indexed to body surface area, assessed using cardiac MRI.
12 secondary outcomes
Change in BSA-indexed LV volumes measured using cardiac MRI, from baseline to 12 months (mls/m2)
Time frame: 12 months
Change LV ejection fraction, measured using cardiac MRI (%)
Time frame: 12 months
Change in myocardial extracellular volume, measured using cardiac MRI (%)
Time frame: 12 months
Change in myocardial tissue T1 and T2 times measured using cardiac MRI (ms)
Time frame: 12 months
Change in pulmonary artery systolic pressure, measured using echocardiography (mmHg)
Time frame: 12 months
Change in myocardial PCr/ATP ratio, measured using cardiac MRI spectroscopy (ratio)
Time frame: 12 months
Change in the number of extra heart beats in a 24 hour period, measured using ambulatory heart monitoring. (number)
Time frame: 12 months
Change in exercise capacity - Six minute walk test (meters)
Time frame: 12 months
20. Change in health status (quality of life), measured using change in SF-36 score. (score)
Time frame: 12 months
Change in Lyso-GB3 blood test levels (nmol/L)
Time frame: 12 months
Change in Troponin blood test levels (ng/L)
Time frame: 12 months
Change in NT pro BNP blood test levels (pg/L)
Time frame: 12 months
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 31 May 2023.
- Individual participant dataSharing planned: No. There is no plan to share IPD with other researchers.
Related trials for Fabry Disease
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A Global Prospective Observational Study of Women With Fabry Disease and Their Infants During Pregnancy and Breastfeeding
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Active, not recruiting
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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.
The record lists no contact. Use the official record to reach the sponsor.
2 sites 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 Manchester University NHS Foundation Trust at 2 sites in United Kingdom.
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 1 inclusion and 1 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 31 May 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in United Kingdom. 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?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Indexed Left Ventricular Mass (grams/m2) (time frame: 12 months). 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 31 May 2023.