Status: Ongoing
First registered on:
12/09/2017
Last updated on:
23/03/2023
1. Study identification
EU PAS Register NumberEUPAS20599
Official titleA Prospective, Observational Registry of Patients with Fabry Disease
Study title acronymAT1001-030
Study typeObservational study
Brief description of the studyThis is a prospective, multi centre, multinational, observational, safety, effectiveness, and outcomes registry enrolling approximately 450 Fabry disease patients (approximately 250 patients in the primary migalastat treated group, 100 patients in the ERT-treated group, and 100 patients in the untreated group). Additionally, patients who previously participated in a migalastat clinical trial, long-term (>24 months treatment) migalastat patients and paediatric patients are eligible to enrol. These subpopulations will not count towards the 450-patient target. All patients will be followed for up to 5 years after enrolment.
This is a registry to evaluate the effects of treatment on long-term safety, effectiveness, and quality of life (QOL) in patients with Fabry disease, with a main focus on migalastat, a medicine with a novel mechanism of action. Occurrence of key indicators of safety and effectiveness will be evaluated, such as cardiac, cerebrovascular and renal events, serious adverse events, and overall survival. A comparison of these events will be evaluated over a period of 5 years in migalastat-treated, ERT-treated (with amenable and non-amenable mutations), and untreated patients with Fabry disease who have amenable mutations. The subpopulations identified above will be analysed separately from the primary migalastat-treated, ERT-treated and untreated groups.
Was this study requested by a regulator?Yes: EMA
Is the study required by a Risk Management Plan (RMP)?
EU RMP category 3 (required)
Regulatory procedure number (RMP Category 1 and 2 studies only)
Other study registration identification numbers and URLs as applicableEU/1/15/1082/001
2. Research centres and Investigator details
Coordinating study entity
Centre nameAmicus Therapeutics UK Limited
Centre locationMarlow, UK
Details of (Primary) lead investigator
Title Mrs
Last name Jasmine
First name Rutecki
Is this study being carried out with the collaboration of a research network?
No
Other centres where this study is being conducted
Multiple centres
In total how many centres are involved in this Study?80
Countries in which this study is being conducted
International study
Argentina
Australia
Austria
Canada
Denmark
Finland
Germany
Greece
Hungary
Ireland
Israel
Italy
Portugal
Spain
Switzerland
United Kingdom
United States
3. Study timelines: initial administrative steps, progress reports and final report
PlannedActual
Date when funding contract was signed01/12/2016
Start date of data collection03/05/201808/08/2018
Start date of data analysis
Date of interim report, if expected
Date of final study report03/05/2029
4. Sources of funding
Please provide estimates of the percentage of funding by source for this study
Names(s)Approximate % funding
Pharmaceutical companiesAmicus Therapeutics UK Limited100
Charities
Government body
Research councils
EU funding scheme
5. Contact details for enquiries
Scientific Enquiries
Title Mr
Last name Joseph
First name Guiliano
Address line 13675 Market Street
Address line 2
Address line 3
CityPhiladelphia
Postcode19104
CountryUnited States
Phone number (incl. country code)16096622000
Alternative phone number
Fax number (incl. country code)
Public Enquiries
Title Mr
Last name Joseph
First name Guiliano
Address line 13675 Market Street
Address line 2
Address line 3
CityPhiladelphia
Postcode19104
CountryUnited States
Phone number (incl. country code)16096622000
Alternative phone number
Fax number (incl. country code)
6. Study drug(s) information
Product NameGalafold
CountryUnited Kingdom
Substance INN(s)MIGALASTAT
7. Medical conditions to be studied
Medical condition(s)Yes
Fabry's disease
8. Population under study
Age
Adolescents (12 - 17 years)
Adults (18 - 44 years)
Adults (45 - 64 years)
Adults (65 - 74 years)
Adults (75 years and over)
Sex
Male
Female
Other population
Renal impaired
Pregnant women
9. Number of subjects
Estimated total number of subjects450
Additional information
The 450 patients include approximately 250 in the primary migalastat-treated, 100 in the ERT-treated, and 100 in the untreated groups. Additionally, patients who previously participated in a migalastat clinical trial, long-term (>24 months treatment) migalastat patients and paediatric patients are eligible to enrol. These subpopulations will not count towards the 450-patient target.
10. Source of data
Is this study being carried out with an established data source?No
Sources of data
Prospective patient-based data collection
Routine primary care electronic patient registry
Spontaneous reporting
11. Scope of the study
What is the scope of the study?
Disease epidemiology
Effectiveness evaluation
Safey
Primary scope : Safey
12. Main objective(s)
What is the main objective of the study?
This is a registry to evaluate the effects of treatment on long-term safety, effectiveness, andquality of life (QOL) in patients with Fabry disease, with a main focus on migalastat.
Occurrence of key indicators of safety and effectiveness will be evaluated, such as serious adverse events, cardiac,
cerebrovascular and renal events, and overall survival.
Are there primary outcomes?No
Are there secondary outcomes?No
13. Study design
What is the design of the study?
This is a prospective, multi centre, multinational, observational, safety, effectiveness, and
14. Follow-up of patients
Will patients be followed up?Yes
Please describe duration of follow up
All patients will be followed for up to 5 years after enrolment.
15. Data analysis plan
Please provide a brief summary of the analysis method
Descriptive statistics will be presented. No formal hypothesis testing will be performed. All
data will be summarized by treatment group. Continuous variables will be summarized using
the number of observations (n), mean, standard deviation (SD), median, minimum, and
maximum. Categorical data will be summarized using counts and percents. For event data,
ie, SAEs and the Fabry Associate Clinical Events (FACEs) of cardiac events, cerebrovascular events and renal events, exposure-adjusted incidence rates will be presented as counts of patients with a new event per 100 person-years exposure, with 95% confidence intervals using Ulm's method. An analysis of recurrent events will be conducted for SAEs and each specific FACE event and will be presented as total counts of each event per 100 person-years. A Cox proportional hazards ratio model will be used for the summary of survival data and will be adjusted for
age at baseline and any previous cardiovascular events.
16. ENCePP seal
Are you requesting the ENCePP seal for this study?
No
17. Full protocol
Not submitted
18. Study Results
Not submitted
Please list the 5 most relevant publications using data from your study
ReferenceLink to web-publication
None
19. Other relevant documents
Conflict(s) of interest of
investigator(s)Not submitted
Composition of Steering Group and
ObserversNot submitted
Other documentsNot
submitted
Signed Code of
Conduct Checklist
Not submitted
Signed Code of Conduct Declaration
Not submitted
Signed Checklist for Study
Protocols
Not submitted
