Clinical trial NCT00196742: Fabry Disease Registry & Pregnancy Sub-registry
Official title: Fabry Disease Registry Protocol
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Fabry Disease
Keywords: alpha Galactosidase A
aGAL (alpha-galactosidase)
Fabry
GL3 (globotriaosylceramide)
Anderson-Fabry Disease
angiokeratomas
GLA deficiency (gene deficiency)
errors in metabolism
Lead sponsor: Sanofi
Sponsor class: INDUSTRY
First posted: 2005-09-20
Enrollment: 9000
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: OTHER
Sampling method: NON_PROBABILITY_SAMPLE
Study population: All patients with a confirmed diagnosis of Fabry disease are eligible for inclusion in the Registry.

For Fabry Pregnancy Sub-registry: It is recommended that pregnancy data be collected on eligible women regardless of infant enrollment. In the event of patients having multiple pregnancies, data collection for each individual pregnancy is encouraged for all women enrolled in this Sub-Registry.

Brief 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.


Eligibility criteria: 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.

Primary outcomes:
measure: Fabry Registry: To evaluate the long-term safety and effectiveness of Fabrazyme®
timeFrame: 33 years
measure: Fabry Pregnancy Sub-registry: pregnancy outcomes, including complications and infant growth
timeFrame: 33 years
Secondary outcomes:
measure: Fabry Register: Monitor factors associated with the efficacy of Fabry disease treatments
timeFrame: 33 years
Other outcomes:
Locations:
city: Birmingham
country: United States
facility: University of Alabama Birmingham- Nephrology- Site Number : 840018
state: Alabama
city: Birmingham
country: United States
facility: University of Alabama Birmingham- Nephrology- Site Number : 840073
state: Alabama
city: Phoenix
country: United States
facility: Phoenix Children's Hospital- Site Number : 840003
state: Arizona
city: Tucson
country: United States
facility: University of Arizona- Site Number : 840015
state: Arizona
city: Little Rock
country: United States
facility: Arkansas Childrens Hospital- Site Number : 840109
state: Arkansas
city: Little Rock
country: United States
facility: University of Arkansas for Medical Sciences- Site Number : 840113
state: Arkansas
city: Irvine
country: United States
facility: University of California at Irvine- Site Number : 840036
state: California
city: Los Angeles
country: United States
facility: Southern California Permanente Medical Group- Site Number : 840108
state: California
city: Los Angeles
country: United States
facility: USC Health Sciences Center Dept of Genetics- Site Number : 840082
state: California
city: Los Angeles
country: United States
facility: UCLA School Of Medicine- Site Number : 840088
state: California
city: Oakland
country: United States
facility: Children's Hospital Oakland- Site Number : 840029
state: California
city: Orange
country: United States
facility: Children's Hospital of Orange County- Site Number : 840074
state: California
city: Palo Alto
country: United States
facility: Stanford Unviersity MC Dept of Genetics- Site Number : 840022
state: California
city: Sacramento
country: United States
facility: UC Davis MIND Institute- Site Number : 840010
state: California
city: Sacramento
country: United States
facility: Kaiser Permanente Hospital- Site Number : 840042
state: California
city: San Bernardino
country: United States
facility: Loma Linda University Children's Hospital- Site Number : 840117
state: California
city: San Diego
country: United States
facility: University of California at San Diego- Site Number : 840007
state: California
city: San Francisco
country: United States
facility: University of California at San Francisco- Site Number : 840051
state: California
city: New Haven
country: United States
facility: Yale - Site Number : 840047
state: Connecticut
city: Washington
country: United States
facility: Children's National Medical Center- Site Number : 840067
state: District of Columbia
city: Coral Springs
country: United States
facility: University Hematology Oncology- Site Number : 840075
state: Florida
city: Gainesville
country: United States
facility: University of Florida Dept of Genetics- Site Number : 840083
state: Florida
city: Jacksonville
country: United States
facility: University of Florida Pediatrics Genetics- Site Number : 840121
state: Florida
city: Jacksonville
country: United States
facility: The Atwal Clinic- Site Number : 840112
state: Florida
city: Miami
country: United States
facility: University Of Miami SOM- Site Number : 840006
state: Florida
city: Tampa
country: United States
facility: University of Florida-Genetics- Site Number : 840096
state: Florida
city: Decatur
country: United States
facility: Emory University School of Medicine- Human Genetics- Site Number : 840060
state: Georgia
city: Aurora
country: United States
facility: University of Colorado at Denver Genetics- Site Number : 840068
state: Illinois
city: Chicago
country: United States
facility: Ann and Robert Lurie Children's Hospital- Site Number : 840013
state: Illinois
city: Chicago
country: United States
facility: Rush University Medical Center Genetics- Site Number : 840079
state: Illinois
city: Indianapolis
country: United States
facility: Indianapolis University School of Medicine- Site Number : 840027
state: Indiana
city: Iowa City
country: United States
facility: University of Iowa- Site Number : 840032
state: Iowa
city: Kansas City
country: United States
facility: University of Kansas Medical Center- Site Number : 840071
state: Kansas
city: Louisville
country: United States
facility: University of Louisville- Genetics- Site Number : 840030
state: Kentucky
city: Baton Rouge
country: United States
facility: Ochsner Medical Center- Site Number : 840120
state: Louisiana
city: New Orleans
country: United States
facility: Tulane University Medical Center- Site Number : 840001
state: Louisiana
city: Portland
country: United States
facility: Maine Medical Center Pediatrics- Site Number : 840064
state: Maine
city: Baltimore
country: United States
facility: John Hopkins- Site Number : 840044
state: Maryland
city: Boston
country: United States
facility: Massachusetts General Hospital-Genetics- Site Number : 840062
state: Massachusetts
city: Boston
country: United States
facility: Brigham and Women's Hospital-Neurology- Site Number : 840103
state: Massachusetts
city: Boston
country: United States
facility: Boston Children's Hospital- Site Number : 840092
state: Massachusetts
city: Springfield
country: United States
facility: Baystate Health- Site Number : 840002
state: Massachusetts
city: Ann Arbor
country: United States
facility: University of Michigan Pediatrics- Site Number : 840107
state: Michigan
city: Detroit
country: United States
facility: Children's Hospital of Michigan- Site Number : 840066
state: Michigan
city: Grand Rapids
country: United States
facility: Spectrum for Health- Site Number : 840019
state: Michigan
city: Grand Rapids
country: United States
facility: Infusion Associates- Site Number : 840050
state: Michigan
city: Minneapolis
country: United States
facility: Children's Health Care- Site Number : 840114
state: Minnesota
city: Minneapolis
country: United States
facility: Children's Hospital and Clinics of Minnesota- Site Number : 840046
state: Minnesota
city: Minneapolis
country: United States
facility: University of Minnesota Medical Center Pediatrics- Site Number : 840076
state: Minnesota
city: Columibia
country: United States
facility: University of Missouri Health System Department of Genetics- Site Number : 840031
state: Missouri
city: Saint Louis
country: United States
facility: Washington University of St. Louis- Site Number : 840100
state: Missouri
city: Billings
country: United States
facility: Billings Clinic - Billings- Site Number : 840118
state: Montana
city: Helena
country: United States
facility: Shodair Children's Hospital- Site Number : 840090
state: Montana
city: Omaha
country: United States
facility: University of Nebraska Medical Center- Pediatrics- Site Number : 840084
state: Nebraska
city: Las Vegas
country: United States
facility: Children's Specialty Center of Nevada- Site Number : 840008
state: Nevada
city: Camden
country: United States
facility: Cooper Health Center- Site Number : 840098
state: New Jersey
city: Hackensack
country: United States
facility: Joseph M. Sanzari Children's Hospital- Site Number : 840101
state: New Jersey
city: Morristown
country: United States
facility: Atlantic Health System- Site Number : 840099
state: New Jersey
city: New Brunswick
country: United States
facility: St. Peter's University Hospital- Site Number : 840016
state: New Jersey
city: Paterson
country: United States
facility: St. Joseph's Children's Hospital- Site Number : 840057
state: New Jersey
city: Manhasset
country: United States
facility: Northwell Health- Site Number : 840102
state: New York
city: New York
country: United States
facility: New York University School Of Medicine- Site Number : 840040
state: New York
city: New York
country: United States
facility: Metropolitan Hospital Center- Site Number : 840110
state: New York
city: New York
country: United States
facility: Icahn School of Medicine at Mount Sinai- Site Number : 840080
state: New York
city: Rochester
country: United States
facility: University of Rochester Medical Center SOM- Site Number : 840105
state: New York
city: Syracuse
country: United States
facility: State University of New York- Site Number : 840052
state: New York
city: Valhalla
country: United States
facility: New York Medical College- Site Number : 840039
state: New York
city: Charlotte
country: United States
facility: Carolinas Medical Center- Site Number : 840065
state: North Carolina
city: Durham
country: United States
facility: Duke University Medical Center Genetics Dept- Site Number : 840045
state: North Carolina
city: Cincinnati
country: United States
facility: Cincinnati Children's Hospital-Genetics- Site Number : 840033
state: Ohio
city: Cleveland
country: United States
facility: UHCMC- Site Number : 840119
state: Ohio
city: Cleveland
country: United States
facility: Cleveland Clinic Foundation Pediatrics- Site Number : 840048
state: Ohio
city: Columbus
country: United States
facility: Nationwide Children's Hospital- Site Number : 840091
state: Ohio
city: Columbus
country: United States
facility: Ohio State University Department of Genetics- Site Number : 840097
state: Ohio
city: Dublin
country: United States
facility: LSD Data Registry Site LLC- Site Number : 840094
state: Ohio
city: Portland
country: United States
facility: Oregon Health and Science University- Site Number : 840116
state: Oregon
city: Portland
country: United States
facility: Oregon Health and Scinece University- Site Number : 840038
state: Oregon
city: Danville
country: United States
facility: Geisinger- Site Number : 840111
state: Pennsylvania
city: Hershey
country: United States
facility: Penn State University Medical Center-Neurology- Site Number : 840104
state: Pennsylvania
city: Philadelphia
country: United States
facility: Children's Hospital of Philadelphia HUP Medical Genetics- Site Number : 840089
state: Pennsylvania
city: Philadelphia
country: United States
facility: Children's Hospital of Philadelphia- Site Number : 840034
state: Pennsylvania
city: Pittsburgh
country: United States
facility: University of Pittsburgh- Site Number : 840023
state: Pennsylvania
city: Providence
country: United States
facility: Rhode Island Hospital Genetics- Site Number : 840053
state: Rhode Island
city: Greenville
country: United States
facility: Greenwood Genetcs- Site Number : 840055
state: South Carolina
city: Nashville
country: United States
facility: Vanderbilt University Hospital-Pediatrics- Site Number : 840049
state: Tennessee
city: Dallas
country: United States
facility: Baylor Research Center- Site Number : 840058
state: Texas
city: Fort Worth
country: United States
facility: Cook Children's Health Care System- Site Number : 840024
state: Texas
city: Salt Lake City
country: United States
facility: University of Utah Department of Medical Genetics- Site Number : 840043
state: Utah
city: Charlottesville
country: United States
facility: University of Virginia School of Medicine-Pediatrics- Site Number : 840078
state: Virginia
city: Fairfax
country: United States
facility: O&O Alpan, LLC- Site Number : 840025
state: Virginia
city: Norfolk
country: United States
facility: Children's Hospital of the Kings Daughters- Site Number : 840072
state: Virginia
city: Seattle
country: United States
facility: Seattle Children's Hospital- Site Number : 840028
state: Washington
city: Seattle
country: United States
facility: University Of Washington Medical Center- Site Number : 840059
state: Washington
city: Tacoma
country: United States
facility: Multicare Health System ¿ Mary Bridge Childrens Hospital- Site Number : 840115
state: Washington
city: Milwaukee
country: United States
facility: Children's Hospital of Wisconsin-Pediatrics- Site Number : 840054
state: Wisconsin
city: Ciudad Autónoma de Buenos Aires
country: Argentina
facility: Investigational Site Number : 153032
state: Buenos Aires
city: Buenos Aires
country: Argentina
facility: Investigational Site Number : 153172
city: Buenos Aires
country: Argentina
facility: Investigational Site Number : 153130
city: Catamarca
country: Argentina
facility: Investigational Site Number : 153194
city: La Rioja
country: Argentina
facility: Investigational Site Number : 032005
city: Santa Fe
country: Argentina
facility: Investigational Site Number : 032004
city: Westmead
country: Australia
facility: Investigational Site Number : 036013
state: New South Wales
city: Westmead
country: Australia
facility: Investigational Site Number : 151069
state: New South Wales
city: Brisbane
country: Australia
facility: Investigational Site Number : 153063
state: Queensland
city: Adelaide
country: Australia
facility: Investigational Site Number : 151002
state: South Australia
city: Parkville
country: Australia
facility: Investigational Site Number : 153040
state: Victoria
city: Perth
country: Australia
facility: Investigational Site Number : 153090
state: Western Australia
city: Antwerp
country: Belgium
facility: Investigational Site Number : 150722
city: Antwerp
country: Belgium
facility: Investigational Site Number : 153133
city: Brussels
country: Belgium
facility: Investigational Site Number : 153001
city: Brussels
country: Belgium
facility: Investigational Site Number : 0560008
city: Brussels
country: Belgium
facility: Investigational Site Number : 150517
city: Leuven
country: Belgium
facility: Investigational Site Number : 151668
city: Liège
country: Belgium
facility: Investigational Site Number : 154275
city: Seraing
country: Belgium
facility: Investigational Site Number : 153100
city: Feira de Santana
country: Brazil
facility: Clínica Senhor do Bonfim LTDA- Site Number : 076008
state: Bahia
city: Brasília
country: Brazil
facility: Medicordis- Site Number : 076009
state: Distrito Federal
city: Cuiabá
country: Brazil
facility: Hospital Geral e Maternidade de Cuiabá- Site Number : 076006
state: Mato Grosso
city: Uberlândia
country: Brazil
facility: CTDR - Centro de Tratamento de Doenças Renais EIRELI- Site Number : 076007
state: Minas Gerais
city: Curitiba
country: Brazil
facility: Instituto do Rim do Paraná- Site Number : 076004
state: Paraná
city: Recife
country: Brazil
facility: Instituto de Medicina Integral Professor Fernando Figueira - IMIP - Site Number : 076005
state: Pernambuco
city: Tapejara
country: Brazil
facility: Saúde Center Clínica - Clínica Médica Nossa Senhora de Fátima - Site Number : 076002
state: Rio Grande Do Sul
city: Florianopolis
country: Brazil
facility: Hospital Universitário Professor Polydoro Ernani de São Thiago - UFSC- Site Number : 076012
state: Santa Catarina
city: Campinas
country: Brazil
facility: Hospital e Maternidade Celso Pierro - PUC-Campinas- Site Number : 076013
state: São Paulo
city: Ourinhos
country: Brazil
facility: Serviço de Terapia Renal de Ourinhos- Site Number : 076011
state: São Paulo
city: São Paulo
country: Brazil
facility: Instituto de Genética e Erros Inatos do Metabolismo - IGEIM- Site Number : 076001
city: Sofia
country: Bulgaria
facility: Investigational Site Number : 153169
city: Sofia
country: Bulgaria
facility: Investigational Site Number : 100001
city: Calgary
country: Canada
facility: Investigational Site Number : 124014
state: Alberta
city: Edmonton
country: Canada
facility: Investigational Site Number : 124002
state: Alberta
city: Vancouver
country: Canada
facility: Investigational Site Number : 124010
state: British Columbia
city: Vancouver
country: Canada
facility: Investigational Site Number : 124011
state: British Columbia
city: Winnipeg
country: Canada
facility: Investigational Site Number : 124019
state: Manitoba
city: Moncton
country: Canada
facility: Investigational Site Number : 124022
state: New Brunswick
city: St. John's
country: Canada
facility: Investigational Site Number : 124023
state: Newfoundland and Labrador
city: Halifax
country: Canada
facility: Investigational Site Number : 124008
state: Nova Scotia
city: Kingston
country: Canada
facility: Investigational Site Number : 124005
state: Ontario
city: London
country: Canada
facility: Investigational Site Number : 124021
state: Ontario
city: Toronto
country: Canada
facility: Investigational Site Number : 124006
state: Ontario
city: Toronto
country: Canada
facility: Investigational Site Number : 124009
state: Ontario
city: Montreal
country: Canada
facility: Investigational Site Number : 124013
state: Quebec
city: Sherbrooke
country: Canada
facility: Investigational Site Number : 124001
state: Quebec
city: Antofagasta
country: Chile
facility: Investigational Site Number : 152004
city: Coquimbo
country: Chile
facility: Investigational Site Number : 152001
city: Iquique
country: Chile
facility: Investigational Site Number : 152003
city: Santiago
country: Chile
facility: Investigational Site Number : 152002
city: Vallenar
country: Chile
facility: Investigational Site Number : 152005
city: Beijing
country: China
facility: Investigational Site Number : 153165
city: Hangzhou
country: China
facility: Investigational Site Number : 1560003
city: Jinan
country: China
facility: Investigational Site Number : 1560002
city: Nanjing
country: China
facility: Investigational Site Number : 1560004
city: Shanghai
country: China
facility: Investigational Site Number : 153163
city: Bogotá
country: Colombia
facility: Investigational Site Number : 170001
city: Rijena
country: Croatia
facility: Investigational Site Number : 1910001
city: Zagreb
country: Croatia
facility: Investigational Site Number : 1910002
city: Praha 2
country: Czechia
facility: Investigational Site Number : 153126
city: Copenhagen
country: Denmark
facility: Investigational Site Number : 153041
city: København Ø
country: Denmark
facility: Investigational Site Number : 151229
city: Tartu
country: Estonia
facility: Investigational Site Number : 153106
city: Turku
country: Finland
facility: Investigational Site Number : 153024
city: Bordeaux
country: France
facility: Investigational Site Number : 250005
city: BRON Cedex
country: France
facility: Investigational Site Number : 250002
city: Garches
country: France
facility: Investigational Site Number : 250001
city: Lille
country: France
facility: Investigational Site Number : 250007
city: Limoges
country: France
facility: Investigational Site Number : 250003
city: Marseille
country: France
facility: Investigational Site Number : 250006
city: St-priest-en-jarez
country: France
facility: Investigational Site Number : 250004
city: Strasbourg
country: France
facility: Investigational Site Number : 250009
city: Berlin
country: Germany
facility: Investigational Site Number : 153013
city: Gießen
country: Germany
facility: Investigational Site Number : 154150
city: Münster
country: Germany
facility: Investigational Site Number : 153147
city: Würzburg
country: Germany
facility: Investigational Site Number : 153011
city: Kowloon
country: Hong Kong
facility: Investigational Site Number : 344001
city: Budapest
country: Hungary
facility: Investigational Site Number : 3480002
city: Létavértes
country: Hungary
facility: Investigational Site Number : 3480003
city: Pécs
country: Hungary
facility: Investigational Site Number : 3480005
city: Sopron
country: Hungary
facility: Investigational Site Number : 3480004
city: Ahmedabad
country: India
facility: Investigational Site Number : 356003
city: Kolkata
country: India
facility: Investigational Site Number : 356001
city: Secunderabad
country: India
facility: Investigational Site Number : 3560004
city: Vellore
country: India
facility: Investigational Site Number : 356002
city: Jakarta
country: Indonesia
facility: Investigational Site Number : 360001
city: Bassano Del Grappa (VI)
country: Italy
facility: Investigational Site Number : 380002
city: Firenze
country: Italy
facility: Investigational Site Number : 380004
city: Firenze
country: Italy
facility: Investigational Site Number : 380013
city: Genova
country: Italy
facility: Investigational Site Number : 380005
city: Milano
country: Italy
facility: Investigational Site Number : 380009
city: Milano
country: Italy
facility: Investigational Site Number : 380006
city: Modena
country: Italy
facility: Investigational Site Number : 380003
city: Monza (MB)
country: Italy
facility: Investigational Site Number : 380010
city: Napoli
country: Italy
facility: Investigational Site Number : 380008
city: Rimini
country: Italy
facility: Investigational Site Number : 380007
city: Roma
country: Italy
facility: Investigational Site Number : 380012
city: Torino
country: Italy
facility: Investigational Site Number : 380011
city: Udine
country: Italy
facility: Investigational Site Number : 380001
city: Vittoria (RG)
country: Italy
facility: Investigational Site Number : 380014
city: Nangoku-shi
country: Japan
facility: Investigational Site Number : 153201
state: Kochi
city: Abeno-ku
country: Japan
facility: Investigational Site Number : 153205
state: Osaka
city: Chiba
country: Japan
facility: Investigational Site Number : 153217
city: Kobe
country: Japan
facility: Investigational Site Number : 392007
city: Niigata
country: Japan
facility: Investigational Site Number : 392006
city: Tokyo
country: Japan
facility: Investigational Site Number : 153204
city: Seoul
country: Korea, Republic of
facility: Investigational Site Number : 151149
state: Gyeonggi-do
city: Yangsan-si
country: Korea, Republic of
facility: Investigational Site Number : 153178
state: Gyeongsangnam-do
city: Yangsan
country: Korea, Republic of
facility: Investigational Site Number : 154148
state: Gyeongsangnam-do
city: Yangsan
country: Korea, Republic of
facility: Investigational Site Number : 154208
state: Gyeongsangnam-do
city: Busan
country: Korea, Republic of
facility: Investigational Site Number : 154246
city: Daejeon
country: Korea, Republic of
facility: Investigational Site Number : 153191
city: Jeju-si
country: Korea, Republic of
facility: Investigational Site Number : 153228
city: Jeonnam
country: Korea, Republic of
facility: Investigational Site Number : 153225
city: Seoul
country: Korea, Republic of
facility: Investigational Site Number : 153180
city: Seoul
country: Korea, Republic of
facility: Investigational Site Number : 153196
city: Seoul
country: Korea, Republic of
facility: Investigational Site Number : 153192
city: Seoul
country: Korea, Republic of
facility: Investigational Site Number : 153188
city: Seoul
country: Korea, Republic of
facility: Investigational Site Number : 153212
city: Seoul
country: Korea, Republic of
facility: Investigational Site Number : 153222
city: Suwon-si
country: Korea, Republic of
facility: Investigational Site Number : 154273
city: Kuwait
country: Kuwait
facility: Investigational Site Number : 154225
city: Vilnius
country: Lithuania
facility: Investigational Site Number : 154244
city: Vilnius
country: Lithuania
facility: Investigational Site Number : 154652
city: Jalan Pahang
country: Malaysia
facility: Investigational Site Number : 458001
city: Kuala Lumpur
country: Malaysia
facility: Investigational Site Number : 458002
city: Amsterdam
country: Netherlands
facility: Investigational Site Number : 151171
city: Amsterdam
country: Netherlands
facility: Investigational Site Number : 153004
city: Bergen
country: Norway
facility: Investigational Site Number : 153141
city: Callao
country: Peru
facility: Investigational Site Number : 604002
city: Manila
country: Philippines
facility: Investigational Site Number : 608001
city: Warszawa
country: Poland
facility: Investigational Site Number : 150575
city: Lisboa
country: Portugal
facility: Investigational Site Number : 620005
city: Porto
country: Portugal
facility: Investigational Site Number : 620001
city: Bucuresti
country: Romania
facility: Investigational Site Number : 154099
city: Bucuresti
country: Romania
facility: Investigational Site Number : 154164
city: Cluj-napoca
country: Romania
facility: Investigational Site Number : 153081
city: Moscow
country: Russian Federation
facility: Investigational Site Number : 643001
city: Nizhny Novgorod
country: Russian Federation
facility: Investigational Site Number : 643003
city: Saint-Peterburg
country: Russian Federation
facility: Investigational Site Number : 643002
city: Al Mubarraz
country: Saudi Arabia
facility: Investigational Site Number : 682002
city: Novi Sad
country: Serbia
facility: Investigational Site Number : 6880001
city: Singapore
country: Singapore
facility: Investigational Site Number : 702001
city: Bratislava
country: Slovakia
facility: Investigational Site Number : 703001
city: Slovenj Gradec
country: Slovenia
facility: Investigational Site Number : 705001
city: A Coruña
country: Spain
facility: Investigational Site Number : 154146
city: A Coruña
country: Spain
facility: Investigational Site Number : 154159
city: Barakaldo
country: Spain
facility: Investigational Site Number : 154135
city: Barcelona
country: Spain
facility: Investigational Site Number : 153067
city: Barcelona
country: Spain
facility: Investigational Site Number : 153237
city: Barcelona
country: Spain
facility: Investigational Site Number : 154147
city: Barcelona
country: Spain
facility: Investigational Site Number : 154204
city: Barcelona
country: Spain
facility: Investigational Site Number : 153077
city: Barcelona
country: Spain
facility: Investigational Site Number : 154123
city: Tarragona
country: Spain
facility: Investigational Site Number : 154136
city: Vigo
country: Spain
facility: Investigational Site Number : 153213
city: Göteborg
country: Sweden
facility: Investigational Site Number : 153050
city: Stockholm
country: Sweden
facility: Investigational Site Number : 151484
city: Chiayi
country: Taiwan
facility: Investigational Site Number : 154093
city: Hualien
country: Taiwan
facility: Investigational Site Number : 152152
city: Kaohsiung City
country: Taiwan
facility: Investigational Site Number : 153088
city: Kaohsiung City
country: Taiwan
facility: Investigational Site Number : 152246
city: Kaohsiung
country: Taiwan
facility: Investigational Site Number : 152244
city: New Taipei City
country: Taiwan
facility: Investigational Site Number : 151534
city: Putz City, Chia-yi
country: Taiwan
facility: Investigational Site Number : 151500
city: Taichung
country: Taiwan
facility: Investigational Site Number : 151179
city: Taichung
country: Taiwan
facility: Investigational Site Number : 151181
city: Tainan City
country: Taiwan
facility: Investigational Site Number : 153248
city: Tainan City
country: Taiwan
facility: Investigational Site Number : 152153
city: Taipei City
country: Taiwan
facility: Investigational Site Number : 151182
city: Taipei City
country: Taiwan
facility: Investigational Site Number : 152226
city: Taipei
country: Taiwan
facility: Investigational Site Number : 152321
city: Taoyuan City
country: Taiwan
facility: Investigational Site Number : 153158
city: Bangkok
country: Thailand
facility: Investigational Site Number : 764003
city: Bangkok
country: Thailand
facility: Investigational Site Number : 764004
city: Bangkok
country: Thailand
facility: Investigational Site Number : 764001
city: Khon Kaen
country: Thailand
facility: Investigational Site Number : 764005
city: Cambridge
country: United Kingdom
facility: Investigational Site Number : 151340
state: Cambridgeshire
city: Belfast
country: United Kingdom
facility: Investigational Site Number : 154196
city: Birmingham
country: United Kingdom
facility: Investigational Site Number : 152287
city: London
country: United Kingdom
facility: Investigational Site Number : 151646
city: London
country: United Kingdom
facility: Investigational Site Number : 151123
city: London
country: United Kingdom
facility: Investigational Site Number : 150303
city: Manchester
country: United Kingdom
facility: Investigational Site Number : 150455
city: Salford
country: United Kingdom
facility: Investigational Site Number : 152361
city: Hanoi
country: Vietnam
facility: Investigational Site Number : 154212
Study officials:
affiliation: Genzyme, a Sanofi Company
name: Study Director
role: STUDY_DIRECTOR
References:
citation: 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.
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.
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.
citation: Wanner C, Feldt-Rasmussen U, Ortiz A. Plain language summary of a study looking at heart muscle thickness and kidney function in women with Fabry disease who received agalsidase beta treatment. Future Cardiol. 2022 Sep;18(10):755-763. doi: 10.2217/fca-2022-0047. Epub 2022 Sep 8.
citation: Ortiz A, Abiose A, Bichet DG, Cabrera G, Charrow J, Germain DP, Hopkin RJ, Jovanovic A, Linhart A, Maruti SS, Mauer M, Oliveira JP, Patel MR, Politei J, Waldek S, Wanner C, Yoo HW, Warnock DG. Time to treatment benefit for adult patients with Fabry disease receiving agalsidase beta: data from the Fabry Registry. J Med Genet. 2016 Jul;53(7):495-502. doi: 10.1136/jmedgenet-2015-103486. Epub 2016 Mar 18.
citation: Germain DP, Charrow J, Desnick RJ, Guffon N, Kempf J, Lachmann RH, Lemay R, Linthorst GE, Packman S, Scott CR, Waldek S, Warnock DG, Weinreb NJ, Wilcox WR. Ten-year outcome of enzyme replacement therapy with agalsidase beta in patients with Fabry disease. J Med Genet. 2015 May;52(5):353-8. doi: 10.1136/jmedgenet-2014-102797. Epub 2015 Mar 20.