The Fabry Registry is an ongoing, international multi-center, strictly observational program that tracks the routine clinical outcomes for patients with Fabry disease, irrespective of treatment status. No experimental intervention is involved; patients in the Registry undergo clinical assessments and receive care as determined by the patient's treating physician.
The primary objectives of the Registry are:
* To enhance the understanding of the variability, progression, and natural history of Fabry disease, including heterozygous females with the disease;
* To assist the Fabry medical community with the development of recommendations for monitoring patients and reports on patient outcomes to help optimize patient care;
* To characterize and describe the Fabry population as a whole;
* To evaluate the long-term safety and effectiveness of Fabrazyme®
Fabry Pregnancy Sub-registry: This Sub-registry is a multicenter, international, longitudinal, observational, and voluntary program designed to track pregnancy outcomes for any pregnant woman enrolled in the Fabry Registry, regardless of whether she is receiving disease-specific therapy (such as enzyme replacement therapy with agalsidase beta) and irrespective of the commercial product with which she may be treated. Data from the Sub-registry are also used to fulfill various global regulatory requirements, to support product development/reimbursement, and for other research and non-research-related purposes. No experimental intervention is given; thus a patient will undergo clinical assessments and receive standard of care treatment as determined by the patient's physician. If a patient consents to this Sub-registry, information about the patient's medical and obstetric history, pregnancy, and birth will be collected, and, if a patient consents to data collection for her infant, data on infant growth through month 36 postpartum will be collected.
Who can participate
Inclusion Criteria
* Fabry Registry: All patients with a confirmed diagnosis of Fabry disease who have signed the informed consent and patient authorization form(s) are eligible for inclusion. Confirmed diagnosis is defined as a documented deficiency in plasma or leukocyte αGAL (alpha-galactosidase) enzyme activity and/or mutation(s) in the gene coding for αGAL.
* Fabry Pregnancy Sub-registry:
* Eligible women must:
* be enrolled in the Fabry Registry.
* be pregnant, or have been pregnant with appropriate medical documentation available.
* provide a signed informed consent and authorization form(s) to participate in the Sub-Registry prior to any Sub-Registry-related data collection being performed.
Exclusion Criteria Fabry Registry: There are no exclusion criteria. Fabry Pregnancy Sub-registry: There are no exclusion criteria.
Locations
41
Argentina (1)
Investigational Site Number : 153280
Berazategui , Buenos Aires
Australia (1)
Investigational Site Number : 036013
Westmead , New South Wales
Belgium (1)
Investigational Site Number : 150722
Antwerp
Brazil (1)
NPDM - Núcleo de Pesquisa e Desenvolvimento de Medicamentos- Site Number : 076007
Fortaleza , Ceará
Bulgaria (1)
Investigational Site Number : 153169
Sofia
COMPLETED
Canada (1)
Investigational Site Number : 124014
Calgary , Alberta
Chile (1)
Investigational Site Number : 152004
Antofagasta
China (1)
Investigational Site Number : 153165
Beijing
TERMINATED
Colombia (1)
Investigational Site Number : 170001
Bogotá
Croatia (1)
Investigational Site Number : 1910001
Rijeka
COMPLETED
Czech Republic (1)
Investigational Site Number : 153126
Prague
Denmark (1)
Investigational Site Number : 151229
Copenhagen
COMPLETED
Estonia (1)
Investigational Site Number : 153106
Tartu
Finland (1)
Investigational Site Number : 153024
Turku
France (1)
Investigational Site Number : 250005
Bordeaux
Hong Kong (1)
Investigational Site Number : 344001
Kowloon
Hungary (1)
Investigational Site Number : 3480002
Budapest
India (1)
Investigational Site Number : 356003
Ahmedabad
Italy (1)
Investigational Site Number : 380013
Florence , Firenze
Japan (1)
Investigational Site Number : 392007
Kobe , Hyōgo
ACTIVE_NOT_RECRUITING
Lithuania (1)
Investigational Site Number : 154244
Vilnius
Malaysia (1)
Investigational Site Number : 458001
Kuala Lumpur
Netherlands (1)
Investigational Site Number : 151171
Amsterdam
TERMINATED
Norway (1)
Investigational Site Number : 153141
Bergen
Peru (1)
Investigational Site Number : 604001
Lima
Philippines (1)
Investigational Site Number : 608001
City of Muntinlupa
Poland (1)
Investigational Site Number : 150575
Warsaw
Romania (1)
Investigational Site Number : 154164
Brasov
Russia (1)
Investigational Site Number : 643001
Moscow
Saudi Arabia (1)
Investigational Site Number : 682002
Al-Hasa
Serbia (1)
Investigational Site Number : 6880001
Novi Sad
Singapore (1)
Investigational Site Number : 702001
Singapore
Slovakia (1)
Investigational Site Number : 703001
Bratislava
COMPLETED
South Korea (1)
Investigational Site Number : 154273
Suwon , Gyeonggi-do
Spain (1)
Investigational Site Number : 154146
A Coruña , A Coruña [La Coruña]
COMPLETED
Sweden (1)
Investigational Site Number : 153050
Gothenburg
Taiwan (1)
Investigational Site Number : 151500
Chiayi City
Thailand (1)
Investigational Site Number : 764003
Bangkok
United Kingdom (1)
Investigational Site Number : 151340
Cambridge , Cambridgeshire
United States (1)
University of Alabama Birmingham- Nephrology- Site Number : 840018