Fabry Disease Registry & Pregnancy Sub-registry

Fabry Disease Clinical Trial

Official title:

Fabry Disease Registry Protocol

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT00196742
• Other study ID #: DIREGC07006
• Secondary ID: AGAL19211U1111-1
• Status: Recruiting
• Start date: July 31, 2001
• Est. completion date: January 31, 2034

Study information

• Verified date: April 2024
• Source: Sanofi
• Contact: Trial Transparency
• Phone: 800-633-1610
• Email: Contact-Us[@]sanofi.com
• Is FDA regulated: No
• Study type: Observational [Patient Registry]

Clinical Trial Summary

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 9000
• Est. completion date: January 31, 2034
• Est. primary completion date: January 31, 2034
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

Eligibility

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 aGAL (alpha-galactosidase) enzyme activity and/or mutation(s) in the gene coding for aGAL.

• 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.

Related Conditions & MeSH terms

• Fabry Disease

Locations

Country	Name	City	State
Argentina	Investigational Site Number : 153130	Buenos Aires
Argentina	Investigational Site Number : 153172	Caba	Buenos Aires
Argentina	Investigational Site Number : 153194	Catamarca
Argentina	Investigational Site Number : 153032	Ciudad Autónoma de Buenos Aires	Buenos Aires
Argentina	Investigational Site Number : 032005	La Rioja
Argentina	Investigational Site Number : 032004	Santa Fe
Australia	Investigational Site Number : 151002	Adelaide	South Australia
Australia	Investigational Site Number : 153063	Herston	Queensland
Australia	Investigational Site Number : 153040	Parkville	Victoria
Australia	Investigational Site Number : 153090	Perth	Western Australia
Australia	Investigational Site Number : 036013	Westmead	New South Wales
Australia	Investigational Site Number : 151069	Westmead	New South Wales
Belgium	Investigational Site Number : 150722	Antwerpen
Belgium	Investigational Site Number : 153133	Antwerpen
Belgium	Investigational Site Number : 153001	Brussels
Belgium	Investigational Site Number : 150517	Bruxelles
Belgium	Investigational Site Number : 0560008	Jette
Belgium	Investigational Site Number : 151668	Leuven
Belgium	Investigational Site Number : 154275	Liège
Belgium	Investigational Site Number : 153100	Seraing
Brazil	Medicordis Site Number : 076009	Brasília	Distrito Federal
Brazil	PUC Campinas - Sociedade Campineira de Educaçao e Instruçao Site Number : 076013	Campinas	São Paulo
Brazil	Hospital Geral e Maternidade de Cuiabá Site Number : 076006	Cuiabá	Mato Grosso
Brazil	Instituto do Rim do Paraná Site Number : 076004	Curitiba	Paraná
Brazil	Clínica Senhor do Bonfim LTDA Site Number : 076008	Feira de Santana	Bahia
Brazil	Hospital Universitário Professor Polydoro Ernani de São Thiago - UFSC Site Number : 076012	Florianopolis	Santa Catarina
Brazil	Serviço de Terapia Renal de Ourinhos Site Number : 076011	Ourinhos	São Paulo
Brazil	Instituto de Medicina Integral Professor Fernando Figueira - IMIP Site Number : 076005	Recife	Pernambuco
Brazil	Instituto de Genética e Erros Inatos do Metabolismo - IGEIM Site Number : 076001	São Paulo
Brazil	Saúde Center Clínica - Clínica Médica Nossa Senhora de Fátima Site Number : 076002	Tapejara	Rio Grande Do Sul
Brazil	CTDR - Centro de Tratamento de Doenças Renais EIRELI Site Number : 076007	Uberlândia	Minas Gerais
Bulgaria	Investigational Site Number : 100001	Sofia
Bulgaria	Investigational Site Number : 153169	Sofia
Canada	Investigational Site Number : 124014	Calgary	Alberta
Canada	Investigational Site Number : 124002	Edmonton	Alberta
Canada	Investigational Site Number : 124008	Halifax	Nova Scotia
Canada	Investigational Site Number : 124021	London	Ontario
Canada	Investigational Site Number : 124022	Moncton	New Brunswick
Canada	Investigational Site Number : 124013	Montreal	Quebec
Canada	Investigational Site Number : 124001	Sherbrooke	Quebec
Canada	Investigational Site Number : 124023	St. John's	Newfoundland and Labrador
Canada	Investigational Site Number : 124006	Toronto	Ontario
Canada	Investigational Site Number : 124009	Toronto	Ontario
Canada	Investigational Site Number : 124010	Vancouver	British Columbia
Canada	Investigational Site Number : 124011	Vancouver	British Columbia
Canada	Investigational Site Number : 124019	Winnipeg	Manitoba
Chile	Investigational Site Number : 152004	Antofagasta
Chile	Investigational Site Number : 152001	Coquimbo
Chile	Investigational Site Number : 152003	Iquique
Chile	Investigational Site Number : 152005	Vallenar
China	Investigational Site Number : 1560003	Hangzhou
China	Investigational Site Number : 1560002	Jinan
China	Investigational Site Number : 1560004	Nanjing
Colombia	Investigational Site Number : 170001	Bogotá
Croatia	Investigational Site Number : 1910001	Rijena
Croatia	Investigational Site Number : 1910002	Zagreb
Czechia	Investigational Site Number : 153126	Praha 2
Denmark	Investigational Site Number : 153041	Copenhagen
Denmark	Investigational Site Number : 151229	København Ø
Estonia	Investigational Site Number : 153106	Tartu
Finland	Investigational Site Number : 153024	Turku
France	Investigational Site Number : 250005	Bordeaux
France	Investigational Site Number : 250002	BRON Cedex
France	Investigational Site Number : 250001	Garches
France	Investigational Site Number : 250007	Lille
France	Investigational Site Number : 250003	LIMOGES Cedex
France	Investigational Site Number : 250006	Marseille
France	Investigational Site Number : 250004	Saint Priest en Jarez
France	Investigational Site Number : 250009	Strasbourg
Germany	Investigational Site Number : 153013	Berlin
Germany	Investigational Site Number : 154150	Gießen
Germany	Investigational Site Number : 153147	Münster
Germany	Investigational Site Number : 153011	Würzburg
Hong Kong	Investigational Site Number : 344001	Kowloon
Hungary	Investigational Site Number : 3480002	Budapest
Hungary	Investigational Site Number : 3480003	Létavértes
Hungary	Investigational Site Number : 3480005	Pécs
Hungary	Investigational Site Number : 3480004	Sopron
India	Investigational Site Number : 356003	Ahmedabad
India	Investigational Site Number : 356001	Kolkata
India	Investigational Site Number : 3560004	Secunderabad
India	Investigational Site Number : 356002	Vellore
Indonesia	Investigational Site Number : 360001	Jakarta
Italy	Investigational Site Number : 380002	Bassano Del Grappa (VI)
Italy	Investigational Site Number : 380004	Firenze
Italy	Investigational Site Number : 380013	Firenze
Italy	Investigational Site Number : 380005	Genova
Italy	Investigational Site Number : 380006	Milano
Italy	Investigational Site Number : 380009	Milano
Italy	Investigational Site Number : 380003	Modena
Italy	Investigational Site Number : 380010	Monza (MB)
Italy	Investigational Site Number : 380008	Napoli
Italy	Investigational Site Number : 380007	Rimini
Italy	Investigational Site Number : 380012	Roma
Italy	Investigational Site Number : 380011	Torino
Italy	Investigational Site Number : 380001	Udine
Italy	Investigational Site Number : 380014	Vittoria (RG)
Japan	Investigational Site Number : 153205	Abeno-ku	Osaka
Japan	Investigational Site Number : 153217	Chiba
Japan	Investigational Site Number : 392007	Kobe-shi, Hyogo
Japan	Investigational Site Number : 153201	Nangoku-shi	Kochi
Japan	Investigational Site Number : 392006	Niigata-shi	Niigata
Japan	Investigational Site Number : 153204	Setagaya	Tokyo
Korea, Republic of	Investigational Site Number : 154246	Busan
Korea, Republic of	Investigational Site Number : 153191	Daejeon
Korea, Republic of	Investigational Site Number : 153225	Jeonnam
Korea, Republic of	Investigational Site Number : 151149	Seoul
Korea, Republic of	Investigational Site Number : 153180	Seoul
Korea, Republic of	Investigational Site Number : 153188	Seoul
Korea, Republic of	Investigational Site Number : 153192	Seoul
Korea, Republic of	Investigational Site Number : 153196	Seoul
Korea, Republic of	Investigational Site Number : 153212	Seoul
Korea, Republic of	Investigational Site Number : 153222	Seoul
Korea, Republic of	Investigational Site Number : 154273	Suwon-si
Korea, Republic of	Investigational Site Number : 154148	Yangsan	Gyeongsangnam-do
Korea, Republic of	Investigational Site Number : 153178	Yangsan-si	Gyeongsangnam-do
Kuwait	Investigational Site Number : 154225	Kuwait
Lithuania	Investigational Site Number : 154244	Vilnius
Lithuania	Investigational Site Number : 154652	Vilnius
Malaysia	Investigational Site Number : 458001	Jalan Pahang
Malaysia	Investigational Site Number : 458002	Kuala Lumpur
Norway	Investigational Site Number : 153141	Bergen
Peru	Investigational Site Number : 604002	Callao
Philippines	Investigational Site Number : 608001	Manila
Poland	Investigational Site Number : 150575	Warszawa
Portugal	Investigational Site Number : 620005	Lisboa
Portugal	Investigational Site Number : 620001	Porto
Romania	Investigational Site Number : 154099	Bucuresti
Romania	Investigational Site Number : 154164	Bucuresti
Romania	Investigational Site Number : 153081	Cluj-Napoca
Russian Federation	Investigational Site Number : 643001	Moscow
Russian Federation	Investigational Site Number : 643003	Nizhny Novgorod
Russian Federation	Investigational Site Number : 643002	Saint-Peterburg
Saudi Arabia	Investigational Site Number : 682002	Al Mubarraz
Serbia	Investigational Site Number : 6880001	Novi Sad
Singapore	Investigational Site Number : 702001	Singapore
Slovakia	Investigational Site Number : 703001	Bratislava
Slovenia	Investigational Site Number : 705001	Slovenj Gradec
Spain	Investigational Site Number : 154135	Barakaldo
Spain	Investigational Site Number : 153067	Barcelona
Spain	Investigational Site Number : 153077	Barcelona
Spain	Investigational Site Number : 153237	Barcelona
Spain	Investigational Site Number : 154123	Barcelona
Spain	Investigational Site Number : 154147	Barcelona
Spain	Investigational Site Number : 154204	Barcelona
Spain	Investigational Site Number : 154146	La Coruña
Spain	Investigational Site Number : 154159	La Coruña
Spain	Investigational Site Number : 154136	Tarragona
Spain	Investigational Site Number : 153213	Vigo
Sweden	Investigational Site Number : 153050	Göteborg
Sweden	Investigational Site Number : 151484	Stockholm
Taiwan	Investigational Site Number : 154093	Chiayi
Taiwan	Investigational Site Number : 152152	Hualien
Taiwan	Investigational Site Number : 152244	Kaohsiung
Taiwan	Investigational Site Number : 153088	Kaohsiung City
Taiwan	Investigational Site Number : 152246	Kaohsiung Hsien,
Taiwan	Investigational Site Number : 151534	New Taipei City
Taiwan	Investigational Site Number : 151500	Putz City, Chia-yi
Taiwan	Investigational Site Number : 151181	Taichung
Taiwan	Investigational Site Number : 151179	Taichung City
Taiwan	Investigational Site Number : 152153	Tainan
Taiwan	Investigational Site Number : 153248	Tainan
Taiwan	Investigational Site Number : 151182	Taipei
Taiwan	Investigational Site Number : 152226	Taipei
Taiwan	Investigational Site Number : 152321	Taipei
Taiwan	Investigational Site Number : 153158	Tao Yuan County
Thailand	Investigational Site Number : 764001	Bangkok
Thailand	Investigational Site Number : 764003	Bangkok
Thailand	Investigational Site Number : 764004	Bangkok
Thailand	Investigational Site Number : 764005	Khon Kaen
United Kingdom	Investigational Site Number : 154196	Belfast
United Kingdom	Investigational Site Number : 152287	Birmingham
United Kingdom	Investigational Site Number : 151340	Cambridge	Cambridgeshire
United Kingdom	Investigational Site Number : 150303	London
United Kingdom	Investigational Site Number : 151123	London
United Kingdom	Investigational Site Number : 151646	London
United Kingdom	Investigational Site Number : 150455	Manchester
United Kingdom	Investigational Site Number : 152361	Salford
United States	University of Michigan Pediatrics Site Number : 840107	Ann Arbor	Michigan
United States	John Hopkins Site Number : 840044	Baltimore	Maryland
United States	Ochsner Medical Center Site Number : 840120	Baton Rouge	Louisiana
United States	Billings Clinic Site Number : 840118	Billings	Montana
United States	University of Alabama Birmingham- Nephrology Site Number : 840018	Birmingham	Alabama
United States	University of Alabama Birmingham- Nephrology Site Number : 840073	Birmingham	Alabama
United States	Boston Children's Hospital Site Number : 840092	Boston	Massachusetts
United States	Brigham and Women's Hospital-Neurology Site Number : 840103	Boston	Massachusetts
United States	Massachusetts General Hospital-Genetics Site Number : 840062	Boston	Massachusetts
United States	Carolinas Medical Center Site Number : 840065	Charlotte	North Carolina
United States	University of Virginia School of Medicine-Pediatrics Site Number : 840078	Charlottesville	Virginia
United States	Ann and Robert Lurie Children's Hospital Site Number : 840013	Chicago	Illinois
United States	Rush University Medical Center Genetics Site Number : 840079	Chicago	Illinois
United States	Cincinnati Children's Hospital-Genetics Site Number : 840033	Cincinnati	Ohio
United States	Cleveland Clinic Foundation Pediatrics Site Number : 840048	Cleveland	Ohio
United States	UHCMC Site Number : 840119	Cleveland	Ohio
United States	Nationwide Children's Hospital Site Number : 840091	Columbus	Ohio
United States	Ohio State University Department of Genetics Site Number : 840097	Columbus	Ohio
United States	University of Missouri Health System Department of Genetics Site Number : 840031	Columibia	Missouri
United States	University Hematology Oncology Site Number : 840075	Coral Springs	Florida
United States	Baylor Research Center Site Number : 840058	Dallas	Texas
United States	Geisinger Site Number : 840111	Danville	Pennsylvania
United States	Emory University School of Medicine- Human Genetics Site Number : 840060	Decatur	Georgia
United States	University of Colorado at Denver Genetics Site Number : 840068	Denver	Colorado
United States	Children's Hospital of Michigan Site Number : 840066	Detroit	Michigan
United States	LSD Data Registry Site LLC Site Number : 840094	Dublin	Ohio
United States	Duke University Medical Center Genetics Dept Site Number : 840045	Durham	North Carolina
United States	O&O Alpan, LLC Site Number : 840025	Fairfax	Virginia
United States	Cook Children's Health Care System Site Number : 840024	Fort Worth	Texas
United States	University of Florida Dept of Genetics Site Number : 840083	Gainesville	Florida
United States	Infusion Associates Site Number : 840050	Grand Rapids	Michigan
United States	Spectrum for Health Site Number : 840019	Grand Rapids	Michigan
United States	Greenwood Genetcs Site Number : 840055	Greenville	South Carolina
United States	Joseph M. Sanzari Children's Hospital Site Number : 840101	Hackensack	New Jersey
United States	Shodair Children's Hospital Site Number : 840090	Helena	Montana
United States	Penn State University Medical Center-Neurology Site Number : 840104	Hershey	Pennsylvania
United States	Indianapolis University School of Medicine Site Number : 840027	Indianapolis	Indiana
United States	University of Iowa Site Number : 840032	Iowa City	Iowa
United States	University of California at Irvine Site Number : 840036	Irvine	California
United States	The Atwal Clinic Site Number : 840112	Jacksonville	Florida
United States	University of Florida Pediatrics Genetics Site Number : 840121	Jacksonville	Florida
United States	University Of Kansas Medical Center Site Number : 840071	Kansas City	Kansas
United States	Children's Specialty Center of Nevada Site Number : 840008	Las Vegas	Nevada
United States	Arkansas Childrens Hospital Site Number : 840109	Little Rock	Arkansas
United States	University of Arkansas for Medical Sciences Site Number : 840113	Little Rock	Arkansas
United States	Southern California Permanente Medical Group Site Number : 840108	Los Angeles	California
United States	UCLA School Of Medicine Site Number : 840088	Los Angeles	California
United States	USC Health Sciences Center Dept of Genetics Site Number : 840082	Los Angeles	California
United States	University of Louisville- Genetics Site Number : 840030	Louisville	Kentucky
United States	Northwell Health Site Number : 840102	Manhasset	New York
United States	University Of Miami SOM Site Number : 840006	Miami	Florida
United States	Children's Hospital of Wisconsin-Pediatrics Site Number : 840054	Milwaukee	Wisconsin
United States	Children's Health Care Site Number : 840114	Minneapolis	Minnesota
United States	Children's Hospital and Clinics of Minnesota Site Number : 840046	Minneapolis	Minnesota
United States	University of Minnesota Medical Center Pediatrics Site Number : 840076	Minneapolis	Minnesota
United States	Atlantic Health System Site Number : 840099	Morristown	New Jersey
United States	Vanderbilt University Hospital-Pediatrics Site Number : 840049	Nashville	Tennessee
United States	St. Peter's University Hospital Site Number : 840016	New Brunswick	New Jersey
United States	Yale Site Number : 840047	New Haven	Connecticut
United States	Tulane University Medical Center Site Number : 840001	New Orleans	Louisiana
United States	Icahn School of Medicine Site Number : 840080	New York	New York
United States	Metropolitan Hospital Center Site Number : 840110	New York	New York
United States	New York University School Of Medicine Site Number : 840040	New York	New York
United States	Children's Hospital of the Kings Daughters Site Number : 840072	Norfolk	Virginia
United States	Children's Hospital Oakland Site Number : 840029	Oakland	California
United States	University of Nebraska Medical Center- Pediatrics Site Number : 840084	Omaha	Nebraska
United States	Children's Hospital of Orange County Site Number : 840074	Orange	California
United States	Stanford Unviersity MC Dept of Genetics Site Number : 840022	Palo Alto	California
United States	St. Joseph's Children's Hospital Site Number : 840057	Paterson	New Jersey
United States	Children's Hospital of Philadelphia HUP Medical Genetics Site Number : 840089	Philadelphia	Pennsylvania
United States	Children's Hospital of Philadelphia Site Number : 840034	Philadelphia	Pennsylvania
United States	Phoenix Children's Hospital Site Number : 840003	Phoenix	Arizona
United States	University of Pittsburgh Site Number : 840023	Pittsburgh	Pennsylvania
United States	Maine Medical Center Pediatrics Site Number : 840064	Portland	Maine
United States	Oregon Health & Science University Site Number : 840116	Portland	Oregon
United States	Rhode Island Hospital Genetics Site Number : 840053	Providence	Rhode Island
United States	University of Rochester Medical Center SOM Site Number : 840105	Rochester	New York
United States	Kaiser Permanente Hospital Site Number : 840042	Sacramento	California
United States	UC Davis MIND Institute Site Number : 840010	Sacramento	California
United States	Washington University of St. Louis Site Number : 840100	Saint Louis	Missouri
United States	University of Utah Department of Medical Genetics Site Number : 840043	Salt Lake City	Utah
United States	Loma Linda University Children's Hospital Site Number : 840117	San Bernardino	California
United States	University of California at San Diego Site Number : 840007	San Diego	California
United States	University of California at San Francisco Site Number : 840051	San Francisco	California
United States	Seattle Children's Hospital Site Number : 840028	Seattle	Washington
United States	University Of Washington Medical Center Site Number : 840059	Seattle	Washington
United States	Baystate Health Site Number : 840002	Springfield	Massachusetts
United States	State University of New York Site Number : 840052	Syracuse	New York
United States	Multicare Health System ¿ Mary Bridge Childrens Hospital Site Number : 840115	Tacoma	Washington
United States	University of Florida-Genetics Site Number : 840096	Tampa	Florida
United States	University of Arizona Site Number : 840015	Tucson	Arizona
United States	New York Medical College Site Number : 840039	Valhalla	New York
United States	Children's National Medical Center Site Number : 840067	Washington	District of Columbia
Vietnam	Investigational Site Number : 154212	Hanoi

Sponsors (1)

Lead Sponsor	Collaborator
Genzyme, a Sanofi Company

Countries where clinical trial is conducted

United States,  Vietnam,  Argentina,  Australia,  Belgium,  Brazil,  Bulgaria,  Canada,  Chile,  China,  Colombia,  Croatia,  Czechia,  Denmark,  Estonia,  Finland,  France,  Germany,  Hong Kong,  Hungary,  India,  Indonesia,  Italy,  Japan,  Korea, Republic of,  Kuwait,  Lithuania,  Malaysia,  Norway,  Peru,  Philippines,  Poland,  Portugal,  Romania,  Russian Federation,  Saudi Arabia,  Serbia,  Singapore,  Slovakia,  Slovenia,  Spain,  Sweden,  Taiwan,  Thailand,  United Kingdom, 

References & Publications (3)

Germain DP, Brand E, Burlina A, Cecchi F, Garman SC, Kempf J, Laney DA, Linhart A, Marodi L, Nicholls K, Ortiz A, Pieruzzi F, Shankar SP, Waldek S, Wanner C, Jovanovic A. Phenotypic characteristics of the p.Asn215Ser (p.N215S) GLA mutation in male and female patients with Fabry disease: A multicenter Fabry Registry study. Mol Genet Genomic Med. 2018 Apr 12;6(4):492-503. doi: 10.1002/mgg3.389. Online ahead of print. — View Citation

Hopkin RJ, Cabrera G, Charrow J, Lemay R, Martins AM, Mauer M, Ortiz A, Patel MR, Sims K, Waldek S, Warnock DG, Wilcox WR. Risk factors for severe clinical events in male and female patients with Fabry disease treated with agalsidase beta enzyme replacement therapy: Data from the Fabry Registry. Mol Genet Metab. 2016 Sep;119(1-2):151-9. doi: 10.1016/j.ymgme.2016.06.007. Epub 2016 Jun 13. — View Citation

Wilcox WR, Feldt-Rasmussen U, Martins AM, Ortiz A, Lemay RM, Jovanovic A, Germain DP, Varas C, Nicholls K, Weidemann F, Hopkin RJ. Improvement of Fabry Disease-Related Gastrointestinal Symptoms in a Significant Proportion of Female Patients Treated with Agalsidase Beta: Data from the Fabry Registry. JIMD Rep. 2018;38:45-51. doi: 10.1007/8904_2017_28. Epub 2017 May 17. — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Fabry Registry: To evaluate the long-term safety and effectiveness of Fabrazyme®	The primary purpose of this Registry is to describe the development and progression of Fabry disease in a representative global population. As Fabry is not a well-described disease this longitudinal program has a wide variety of "primary" outcomes including antibody testing as well as complication outcomes (eg, QoL, cognitive testing) which are measured over time. Additionally, as it is subject to what is collected by clinical sites at the time of visit it is unknown the amount of data that will be available for analyses.	33 years
Primary	Fabry Pregnancy Sub-registry: pregnancy outcomes, including complications and infant growth	The primary objective of this Sub-registry is to track pregnancy outcomes, including complications and infant growth, in all women with Fabry disease during pregnancy, regardless of whether they receive disease-specific therapy, such as ERT with agalsidase beta.; This Sub-registry augments the routine monitoring and data collection recommended by the Fabry Registry. The Sub-registry aims to collect patient assessments from the ante- and perinatal periods and postpartum follow-up. Neonatal assessments and periodic pediatric assessments will also be collected.	33 years
Secondary	Fabry Register: Monitor factors associated with the efficacy of Fabry disease treatments	A secondary endpoint is to monitor those factors associated with the efficacy of Fabry disease treatments.	33 years