Fabry DiseaseNCT04602364
French Prospective, Observational Cohort Study of Patients With Fabry Disease Treated With Migalastat
An observational study of Fabry Disease, run by Amicus Therapeutics France SAS at 13 sites in France.
Trial summary
This is a noninterventional cohort study to evaluate the effects of migalastat, on long-term safety, effectiveness, and quality of life (QOL) in patients with Fabry disease. 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?
- Noninterventional
- How many people
- 48 participants
- Where
- 13 sitesFrance
- Study dates
- 15 Oct 2020to 30 Jun 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
- Child, Adult, Older adult
Study population: The Miga-Fab is open to participants with Fabry disease treated by migalastat. There is no predetermined sample size.
Who may be able to join
- Patients with Fabry disease aged 16 years or older
- eGFRCKD-EPI > 30 mL/min/1.73 m2
- treated with migalastat, or who are starting migalastat upon enrollment
- Patients with Fabry disease and/or parents/guardians (when applicable) who are able to understand and have provided a signed non-opposition form.
- Equipped with a web connection via a computer or tablet
Who cannot join
- Patients who are participating in a clinical trial of any investigational medicinal product or device at the time of enrollment.
- Patients already included in the present study
Trial locations and contacts
13 sites in 1 country.
13 sites in 1 country
France13 sites
CHU Angers
Angers
CHU de Bordeaux
Bordeaux
Hôpital Pellegrin
Bordeaux
CHU Caen
Caen
CHU Lille
Lille
Hôpital femme mère enfant
Lyon
Hôpital de la Conception | AP-HM
Marseille
CHU de Nancy
Nancy
CHU Nantes Hôtel Dieu
Nantes
Hôpital de la Croix Saint Simon
Paris
Hôpital Tenon AP-HP
Paris
Hôpitaux Universitaires de Strasbourg
Strasbourg
CHU Toulouse Rangueil
Toulouse
About this study
Official title: A French Prospective, Observational Cohort Study of Patients With Fabry Disease Treated With Migalastat - the MIGA-FAB Study
This is a noninterventional cohort study to evaluate the effects of migalastat, on long-term safety, effectiveness, and quality of life (QOL) in patients with Fabry disease.
Read the detailed description
Occurrence of key indicators of safety and effectiveness will be evaluated, such as cardiac, cerebrovascular and renal events, and overall survival. The study is designed to provide effectiveness and safety data by Q2 2023 which will cover a period up to 5 years after the migalastat launch date.
This will involve a retrospective data collection up to migalastat initiation (for patients already receiving migalastat) and a prospective follow-up from 1 to 3.5 years (depending on the time of enrollment) in migalastat-treated patients with Fabry disease who have a GLA mutation amenable to migalastat.
All visits will be scheduled and conducted according to the clinical site's standard of care. Standard of care is defined as a diagnostic and customary clinical treatment/practice process that a clinician chooses according to their clinical judgement for a Fabry disease patient. There are no study- required visits, tests or clinical assessments.
- Conditions
- Fabry Disease
- Being tested
- Noninterventional
- Medical subject headings
- Fabry Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
NoninterventionalOther
Not applicable; Noninterventional study
| Study group | Role | What they receive |
|---|---|---|
| Miga-Fab patients | Miga-Fab is a French prospective, observational cohort study of patients with Fabry disease treated with migalastat Receives: Other: Noninterventional |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety - SAEs
Evaluation of the occurrence ie the number of SAEs
Measured: up to 60 months
Safety - vital signs
Evaluation of resting blood pressure
Measured: up to 60 months
Effectiveness - Fabry Associated Clinical Events (FACEs)
Evaluation of the occurrence of the FACEs ie total number of cardiac, cerebrovascular, and renal events
Measured: up to 60 months
Effectiveness - survival
Survival among all patients enrolled, as assessed by recorded patient death from any cause
Measured: From date of inclusion until the date of death from any cause, assessed up to 60 months
SF-12 12-Item Short Form Health Survey
Evaluation of QOL by the 12-Item Short Form Health Survey (SF-12) ; the higher the score the worse the quality of life is
Measured: up to 60 months
BPI
Brief pain inventory questionnaire ; the higher the score the more intense the pain is
Measured: up to 60 months
FABPRO-GI
Fabry Disease Patient-Reported Outcome-Gastro intestinal Signs and Symptoms Questionnaire ; the higher the score the more importante the GI symptoms are
Measured: inclusion to last visit
Cardiac echo imagery
Echocardiogram (Echo) Left Ventricular Mass Index (LVMI)
Measured: up to 60 months
Treatment compliance
Patient adherence evaluation (% of taken intakes per month) as reported monthly through self-reports of forgotten intakes by the patient
Measured: up to 60 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Oct 2020
- Study start15 Oct 2020
- Primary completion30 Jun 2023
- Study completion30 Jun 2023
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
- 48 participants (actual)
Sponsor and investigators
- Lead sponsor
- Amicus Therapeutics France SAS
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Olivier Lidove, Dr., Principal investigator, Groupe Hospitalier Diaconnesses Croix Saint Simon
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04602364
- Sponsor's protocol ID
- MIGA-FAB
- Status last verified
- May 2024
- First submitted
- 22 Jan 2020
- Last updated
- 23 May 2024
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 23 May 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A French Prospective, Observational Cohort Study of Patients With Fabry Disease Treated With Migalastat - the MIGA-FAB Study
- ClinicalTrials.gov ID
- NCT04602364
- Acronym / sponsor ID
- MIGA-FAB
- Conditions
- Fabry Disease
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 48 participants (actual)
- Lead sponsor
- Amicus Therapeutics France SASIndustry (pharma, biotech, devices)
- Study start
- 15 Oct 2020 (actual)
- Primary completion
- 30 Jun 2023 (actual)
- Study completion
- 30 Jun 2023 (actual)
- First posted
- 26 Oct 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 a noninterventional cohort study to evaluate the effects of migalastat, on long-term safety, effectiveness, and quality of life (QOL) in patients with Fabry disease.
Detailed description
Occurrence of key indicators of safety and effectiveness will be evaluated, such as cardiac, cerebrovascular and renal events, and overall survival. The study is designed to provide effectiveness and safety data by Q2 2023 which will cover a period up to 5 years after the migalastat launch date.
This will involve a retrospective data collection up to migalastat initiation (for patients already receiving migalastat) and a prospective follow-up from 1 to 3.5 years (depending on the time of enrollment) in migalastat-treated patients with Fabry disease who have a GLA mutation amenable to migalastat.
All visits will be scheduled and conducted according to the clinical site's standard of care. Standard of care is defined as a diagnostic and customary clinical treatment/practice process that a clinician chooses according to their clinical judgement for a Fabry disease patient. There are no study- required visits, tests or clinical assessments.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Miga-Fab patients | – | Other: NoninterventionalMiga-Fab is a French prospective, observational cohort study of patients with Fabry disease treated with migalastat |
Who may qualify
- Ages
- 16 years and older
- Sex
- All
- Age groups
- Child, Adult, Older adult
- Study population
- The Miga-Fab is open to participants with Fabry disease treated by migalastat. There is no predetermined sample size.
Key inclusion criteria (5 listed)
- Patients with Fabry disease aged 16 years or older
- eGFRCKD-EPI > 30 mL/min/1.73 m2
- treated with migalastat, or who are starting migalastat upon enrollment
- Patients with Fabry disease and/or parents/guardians (when applicable) who are able to understand and have provided a signed non-opposition form.
- Equipped with a web connection via a computer or tablet
Key exclusion criteria (2 listed)
- Patients who are participating in a clinical trial of any investigational medicinal product or device at the time of enrollment.
- Patients already included in the present study
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 |
|---|---|---|
| NoninterventionalNot applicable; Noninterventional study | Other | Miga-Fab patients |
Primary outcome
Safety - SAEs
Time frame: up to 60 months
Evaluation of the occurrence ie the number of SAEs
Safety - vital signs
Time frame: up to 60 months
Evaluation of resting blood pressure
Effectiveness - Fabry Associated Clinical Events (FACEs)
Time frame: up to 60 months
Evaluation of the occurrence of the FACEs ie total number of cardiac, cerebrovascular, and renal events
Effectiveness - survival
Time frame: From date of inclusion until the date of death from any cause, assessed up to 60 months
Survival among all patients enrolled, as assessed by recorded patient death from any cause
SF-12 12-Item Short Form Health Survey
Time frame: up to 60 months
Evaluation of QOL by the 12-Item Short Form Health Survey (SF-12) ; the higher the score the worse the quality of life is
BPI
Time frame: up to 60 months
Brief pain inventory questionnaire ; the higher the score the more intense the pain is
FABPRO-GI
Time frame: inclusion to last visit
Fabry Disease Patient-Reported Outcome-Gastro intestinal Signs and Symptoms Questionnaire ; the higher the score the more importante the GI symptoms are
Cardiac echo imagery
Time frame: up to 60 months
Echocardiogram (Echo) Left Ventricular Mass Index (LVMI)
Treatment compliance
Time frame: up to 60 months
Patient adherence evaluation (% of taken intakes per month) as reported monthly through self-reports of forgotten intakes by the patient
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 May 2024.
- Individual participant dataSharing planned: No
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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.
Olivier Lidove, Dr. principal investigator
Groupe Hospitalier Diaconnesses Croix Saint Simon
13 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 Amicus Therapeutics France SAS at 13 sites in France.
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 5 inclusion and 2 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: finished, as of the sponsor's last update on 23 May 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 13 sites in France. 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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Safety - SAEs (time frame: up to 60 months). The study lists 9 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 23 May 2024.