Fabry DiseaseNCT00196742
Fabry Disease Registry & Pregnancy Sub-registry
An observational study of Fabry Disease, run by Genzyme, a Sanofi Company at 284 sites in 44 countries.
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. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Fabry Disease
- How many people
- 9,000 participants
- Where
- 284 sitesin 44 countries
- Study dates
- 31 Jul 2001to 31 Jan 2034
- How long will I take part?
- 52 Weeks
- Phase
- Observational
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 Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
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.
Who may be able to join
- 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.
Who cannot join
None listed.
Trial locations and contacts
284 sites in 44 countries. Contact the study team or a site near you to ask about taking part.
Fabry Registry HelpLine contact
284 sites in 44 countries
United States91 sites, 74 recruiting
- Recruiting
University Of Michigan Pediatrics- Site Number : 840107
Ann Arbor, Michigan, 48109
- Recruiting
University Of Colorado At Denver Genetics- Site Number : 840068
Aurora, Colorado, 80045
- Completed
John Hopkins- Site Number : 840044
Baltimore, Maryland, 21205
- Recruiting
Ochsner Health Center - Baton Rouge- Site Number : 840120
Baton Rouge, Louisiana, 70809
- Completed
University Of Alabama Birmingham- Nephrology- Site Number : 840018
Birmingham, Alabama, 35233
- Recruiting
University Of Alabama Birmingham- Nephrology- Site Number : 840073
Birmingham, Alabama, 35233
- Recruiting
Massachusetts General Hospital-Genetics- Site Number : 840062
Boston, Massachusetts, 02114
- Recruiting
Boston Children's Hospital- Site Number : 840092
Boston, Massachusetts, 02115
- Recruiting
Brigham & Women's Hospital- Site Number : 840103
Boston, Massachusetts, 02115
- Recruiting
Atrium Health - Carolinas Medical Center- Site Number : 840065
Charlotte, North Carolina, 28203
- Completed
University Of Virginia School Of Medicine-Pediatrics- Site Number : 840078
Charlottesville, Virginia, 22908
- Recruiting
Ann & Robert H. Lurie Children's Hospital of Chicago- Site Number : 840013
Chicago, Illinois, 60611
- Recruiting
Rush University Medical Center Genetics- Site Number : 840079
Chicago, Illinois, 60612
- Recruiting
Cincinnati Children's Hospital Medical Center (Cincinnati Children's Hospital)- Site Number : 840033
Cincinnati, Ohio, 45229
- Recruiting
University Hospitals Cleveland Medical Center- Site Number : 840119
Cleveland, Ohio, 44106
- Recruiting
Cleveland Clinic - Cleveland- Site Number : 840048
Cleveland, Ohio, 44195
- Recruiting
University Of Missouri Health System Department Of Genetics- Site Number : 840031
Columbia, Missouri, 65212
- Recruiting
Nationwide Children's Hospital- Site Number : 840091
Columbus, Ohio, 43205
- Active, not recruiting
Ohio State University Department Of Genetics- Site Number : 840097
Columbus, Ohio, 43210
- Recruiting
University of Miami- Site Number : 840006
Coral Gables, Florida, 33124
- Recruiting
University of Miami Sylvester Comprehensive Cancer Center - Coral Springs- Site Number : 840075
Coral Springs, Florida, 33065
- Recruiting
Baylor Institute of Metabolic Disease- Site Number : 840058
Dallas, Texas, 75226
- Recruiting
Geisinger Medical Center- Site Number : 840111
Danville, Pennsylvania, 17822
- Recruiting
Emory University School Of Medicine- Human Genetics- Site Number : 840060
Decatur, Georgia, 30033
- Recruiting
Children's Hospital of Michigan- Site Number : 840066
Detroit, Michigan, 48201
- Recruiting
Lsd Data Registry Site Llc- Site Number : 840094
Dublin, Ohio, 43017
- Recruiting
Duke University Medical Center Genetics Dept- Site Number : 840045
Durham, North Carolina, 27710
- Recruiting
O & O Alpan- Site Number : 840025
Fairfax, Virginia, 22030
- Recruiting
Cook Children's Medical Center- Site Number : 840024
Fort Worth, Texas, 76104
- Recruiting
University Of Florida Dept Of Genetics- Site Number : 840083
Gainesville, Florida, 32610
- Completed
Corewell Health - Helen DeVos Children's Hospital (HDVCH)- Site Number : 840019
Grand Rapids, Michigan, 49503
- Recruiting
Infusion Associates - Grand Rapids- Site Number : 840050
Grand Rapids, Michigan, 49525
- Recruiting
Greenwood Genetic Center - Greenville- Site Number : 840055
Greenville, South Carolina, 29605
- Recruiting
Joseph M. Sanzari Children'S Hospital- Site Number : 840101
Hackensack, New Jersey, 07601
- Completed
Shodair Children'S Hospital- Site Number : 840090
Helena, Montana, 59601
- Recruiting
Penn State University Medical Center-Neurology- Site Number : 840104
Hershey, Pennsylvania, 17033
- Recruiting
NYU Langone Health - Perlmutter Cancer Center - Huntington- Site Number : 840040
Huntington, New York, 11743
- Recruiting
Indianapolis University School Of Medicine- Site Number : 840027
Indianapolis, Indiana, 46202
- Recruiting
University of Iowa- Site Number : 840032
Iowa City, Iowa, 52242
- Recruiting
University Of Florida Pediatrics Genetics- Site Number : 840121
Jacksonville, Florida, 32207
- Recruiting
University of Kansas Medical Center- Site Number : 840071
Kansas City, Kansas, 66160
- Recruiting
Children's Specialty Center - Las Vegas- Site Number : 840008
Las Vegas, Nevada, 89109
- Recruiting
Arkansas Children's Hospital- Site Number : 840109
Little Rock, Arkansas, 72202
- Recruiting
University of Arkansas for Medical Sciences- Site Number : 840113
Little Rock, Arkansas, 72205
- Recruiting
Southern California Permanente Medical Group- Site Number : 840108
Los Angeles, California, 90027
- Completed
Usc Health Sciences Center Dept Of Genetics- Site Number : 840082
Los Angeles, California, 90033
- Recruiting
University Of California Los Angeles- Site Number : 840088
Los Angeles, California, 90095
- Completed
University of Louisville - Health Sciences Center- Site Number : 840030
Louisville, Kentucky, 40202
- Recruiting
Children's Wisconsin- Site Number : 840054
Milwaukee, Wisconsin, 53226
- Completed
Children's Hospitals and Clinics of Minnesota- Site Number : 840046
Minneapolis, Minnesota, 55404
- Recruiting
Children's Hospitals and Clinics of Minnesota- Site Number : 840114
Minneapolis, Minnesota, 55404
- Recruiting
M Health Fairview University of Minnesota Medical Center - West Bank- Site Number : 840076
Minneapolis, Minnesota, 55454
- Recruiting
Atlantic Health System- Site Number : 840099
Morristown, New Jersey, 07962
- Recruiting
Vanderbilt University Medical Center - Medical Laboratories- Site Number : 840049
Nashville, Tennessee, 37228
- Recruiting
Saint Peter's University Hospital - New Brunswick- Site Number : 840016
New Brunswick, New Jersey, 08901
- Recruiting
Yale University School of Medicine- Site Number : 840047
New Haven, Connecticut, 06510
- Recruiting
Northwell Health- Site Number : 840102
New Hyde Park, New York, 11042
- Completed
Tulane University School of Medicine- Site Number : 840001
New Orleans, Louisiana, 70112
- Recruiting
Icahn School of Medicine at Mount Sinai- Site Number : 840080
New York, New York, 10029
- Recruiting
Metropolitan Hospital Center- Site Number : 840110
New York, New York, 10029
- Completed
Children's Hospital of The King's Daughters- Site Number : 840072
Norfolk, Virginia, 23507
- Completed
UCSF Benioff Children's Hospital - Oakland- Site Number : 840029
Oakland, California, 94609
- Recruiting
University of Nebraska Medical Center- Site Number : 840084
Omaha, Nebraska, 68198
- Recruiting
Children's Hospital of Orange County- Site Number : 840074
Orange, California, 92868
- Recruiting
University of California Irvine Medical Center- Site Number : 840036
Orange, California, 92868
- Recruiting
St. Joseph's Regional Medical Center - Paterson- Site Number : 840057
Paterson, New Jersey, 07503
- Recruiting
Children'S Hospital Of Philadelphia Hup Medical Genetics- Site Number : 840089
Philadelphia, Pennsylvania, 19104
- Recruiting
The Children's Hospital of Philadelphia- Site Number : 840034
Philadelphia, Pennsylvania, 19104
- Recruiting
Phoenix Children's Hospital- Site Number : 840003
Phoenix, Arizona, 85016
- Recruiting
UPMC Children's Hospital of Pittsburgh- Site Number : 840023
Pittsburgh, Pennsylvania, 15224
- Recruiting
Maine Medical Partners Pediatric Specialty Care- Site Number : 840064
Portland, Maine, 04102
- Terminated
Oregon Health & Science University (OHSU)- Site Number : 840038
Portland, Oregon, 97239
- Recruiting
Oregon Health & Science University (OHSU)- Site Number : 840116
Portland, Oregon, 97239
- Completed
Rhode Island Hospital Genetics- Site Number : 840053
Providence, Rhode Island, 02903
- Completed
University of Rochester Medical Center- Site Number : 840105
Rochester, New York, 14642
- Recruiting
Uc Davis Mind Institute- Site Number : 840010
Sacramento, California, 95817
- Completed
Kaiser Permanente Sacramento Medical Center- Site Number : 840042
Sacramento, California, 95825
- Recruiting
University of Utah- Site Number : 840043
Salt Lake City, Utah, 84112
- Completed
University of California at San Diego- Site Number : 840007
San Diego, California, 92123
- Recruiting
University of California San Francisco - Parnassus Campus- Site Number : 840051
San Francisco, California, 94143
- Recruiting
Seattle Children's Hospital- Site Number : 840028
Seattle, Washington, 98105
- Recruiting
University of Washington Medical Center- Site Number : 840059
Seattle, Washington, 98195
- Recruiting
Baystate Medical Center- Site Number : 840002
Springfield, Massachusetts, 01199
- Recruiting
Washington University- Site Number : 840100
St Louis, Missouri, 63110
- Recruiting
Stanford University Medical Center- Site Number : 840022
Stanford, California, 94305
- Recruiting
SUNY Upstate Medical University - Site Number : 840052
Syracuse, New York, 13210
- Recruiting
MultiCare Mary Bridge Children's Health Center- Site Number : 840115
Tacoma, Washington, 98405
- Recruiting
University Of Florida-Genetics- Site Number : 840096
Tampa, Florida, 33606
- Recruiting
University of Arizona- Site Number : 840015
Tucson, Arizona, 85724
- Recruiting
New York Medical College- Site Number : 840039
Valhalla, New York, 10595
- Recruiting
Children's National Medical Center - Washington- Site Number : 840067
Washington D.C., District of Columbia, 20010
Italy17 sites, 14 recruiting
- Recruiting
Investigational Site Number : 380003
Baggiovara, Modena, 41126
- Recruiting
Investigational Site Number : 380002
Bassano del Grappa, Vicenza, 36061
- Recruiting
Investigational Site Number : 380019
Bologna, 40138
- Recruiting
Investigational Site Number : 380004
Florence, Firenze, 50134
- Recruiting
Investigational Site Number : 380013
Florence, Firenze, 50139
- Completed
Investigational Site Number : 380009
Milan, Milano, 20142
- Completed
Investigational Site Number : 380006
Milan, Milano, 20162
- Recruiting
Investigational Site Number : 380010
Monza, 20900
- Recruiting
Investigational Site Number : 380008
Naples, 80131
- Recruiting
Investigational Site Number : 157282
Padua, Padova, 35128
- Recruiting
Investigational Site Number : 157300
Palermo, 90127
- Recruiting
Investigational Site Number : 380014
Ragusa, 97019
- Recruiting
Investigational Site Number : 157298
Reggio Calabria, 89125
- Completed
Investigational Site Number : 380007
Rimini, 47900
- Recruiting
Investigational Site Number : 380012
Rome, Roma, 00168
- Recruiting
Investigational Site Number : 380011
Turin, Piedmont, 10126
- Recruiting
Investigational Site Number : 380001
Udine, 33100
Taiwan15 sites, 13 recruiting
- Recruiting
Investigational Site Number : 154093
Chia-Yi City, 60002
- Recruiting
Investigational Site Number : 152152
Hualien City, 970
- Completed
Investigational Site Number : 152244
Kaohsiung City, 80756
- Completed
Investigational Site Number : 153088
Kaohsiung City, 813
- Recruiting
Investigational Site Number : 152246
Kaohsiung City, 833
- Recruiting
Investigational Site Number : 151534
New Taipei City, 231
- Recruiting
Investigational Site Number : 151500
Putz City, Chia-Yi, NaN
- Recruiting
Investigational Site Number : 151179
Taichung, 404
- Recruiting
Investigational Site Number : 151181
Taichung, 407
- Recruiting
Investigational Site Number : 153248
Tainan, 704
- Recruiting
Investigational Site Number : 152153
Tainan, 710
- Recruiting
Investigational Site Number : 151182
Taipei, 100
- Recruiting
Investigational Site Number : 152321
Taipei, 104
- Recruiting
Investigational Site Number : 152226
Taipei, 112
- Recruiting
Investigational Site Number : 153158
Taoyuan City, 333
Canada14 sites, 11 recruiting
- Recruiting
Investigational Site Number : 124014
Calgary, Alberta, T2E 7Z4
- Recruiting
Investigational Site Number : 124002
Edmonton, Alberta, T6G 2B7
- Recruiting
Investigational Site Number : 124008
Halifax, Nova Scotia, B3H 3A7
- Terminated
Investigational Site Number : 124005
Kingston, Ontario, K7L 4X3
- Recruiting
Investigational Site Number : 124021
London, Ontario, N6A 5A5
- Completed
Investigational Site Number : 124022
Moncton, New Brunswick, E1C 2Z3
- Recruiting
Investigational Site Number : 124013
Montreal, Quebec, H4J 1C5
- Recruiting
Investigational Site Number : 124001
Sherbrooke, Quebec, J1K 2R1
- Recruiting
Investigational Site Number : 124023
St. John's, Newfoundland and Labrador, A1B 3V6
- Recruiting
Investigational Site Number : 124006
Toronto, Ontario, M5G 1X8
- Recruiting
Investigational Site Number : 124009
Toronto, Ontario, M5T 3H7
- Recruiting
Investigational Site Number : 124010
Vancouver, British Columbia, V5Z 1M9
- Completed
Investigational Site Number : 124011
Vancouver, British Columbia, V6H 3N1
- Recruiting
Investigational Site Number : 124019
Winnipeg, Manitoba, R3A 1R9
South Korea14 sites, 10 recruiting
- Recruiting
Investigational Site Number : 154246
Busan, NaN
- Recruiting
Investigational Site Number : 153191
Daejeon, NaN
- Terminated
Investigational Site Number : 153228
Jeju City, Jeju-teukbyeoljachido, 63241
- Recruiting
Investigational Site Number : 153180
Seoul, Seoul-teukbyeolsi, 03080
- Recruiting
Investigational Site Number : 153196
Seoul, Seoul-teukbyeolsi, 03080
- Completed
Investigational Site Number : 153192
Seoul, Seoul-teukbyeolsi, 04401
- Recruiting
Investigational Site Number : 151149
Seoul, Seoul-teukbyeolsi, 05505
- Recruiting
Investigational Site Number : 153222
Seoul, Seoul-teukbyeolsi, 06973
- Recruiting
Investigational Site Number : 153188
Seoul, NaN
- Recruiting
Investigational Site Number : 153212
Seoul, NaN
- Recruiting
Investigational Site Number : 154273
Suwon, Gyeonggi-do, 16247
- Completed
Investigational Site Number : 153178
Yangsan, Gyeongsangnam-do, 50612
- Terminated
Investigational Site Number : 154208
Yangsan, Gyeongsangnam-do, 50612
- Recruiting
Investigational Site Number : 157176
Yangsan, Gyeongsangnam-do, 50612
Brazil12 sites, 8 recruiting
- Completed
Hospital e Maternidade Celso Pierro - PUC-Campinas- Site Number : 076013
Campinas, São Paulo, 13034-685
- Completed
Centro de Pesq Materno Infantil de Campinas- Site Number : 076009
Campinas, São Paulo, 13081-970
- Completed
Hospital Geral Universitario de Cuiaba- Site Number : 076006
Cuiabá, Mato Grosso, 78025-000
- Recruiting
Universidade Federal do Paraná- Site Number : 076004
Curitiba, Paraná, 80060-000
- Recruiting
Clínica Senhor do Bonfim- Site Number : 076008
Feira de Santana, Estado de Bahia, 44001-584
- Recruiting
Hospital Universitário Polydoro Ernani de São Thiago - UFSC- Site Number : 076012
Florianópolis, Santa Catarina, 88040-970
- Recruiting
Hospital Geral de Fortaleza - HGF- Site Number : 076016
Fortaleza, Ceará, 60150-160
- Recruiting
NPDM - Núcleo de Pesquisa e Desenvolvimento de Medicamentos- Site Number : 076007
Fortaleza, Ceará, 60430-275
- Completed
Serviço De Terapia Renal De Ourinhos- Site Number : 076011
Ourinhos, São Paulo, 19900280
- Recruiting
Instituto de Medicina Integral Professor Fernando Figueira- Site Number : 076005
Recife, Pernambuco, 50070-550
- Recruiting
Centro Saude Escola Joel Domingos Machado- Site Number : 076002
Ribeirão Preto, São Paulo, 14055-380
- Recruiting
Instituto de Genética e Erros Inatos do Metabolismo- Site Number : 076001
São Paulo, 04020-041
Spain11 sites, 8 recruiting
- Completed
Investigational Site Number : 154146
A Coruña, Galicia [Galicia], 15006
- Completed
Investigational Site Number : 154159
A Coruña, Galicia [Galicia], 15006
- Recruiting
Investigational Site Number : 154123
Barcelona, Catalunya [Cataluña], 08003
- Recruiting
Investigational Site Number : 153067
Barcelona, Catalunya [Cataluña], 08025
- Recruiting
Investigational Site Number : 153237
Barcelona, Catalunya [Cataluña], 08035
- Recruiting
Investigational Site Number : 154147
Barcelona, Catalunya [Cataluña], 08035
- Recruiting
Investigational Site Number : 154204
Barcelona, Catalunya [Cataluña], 08036
- Completed
Investigational Site Number : 154135
Bizkaia, Basque Country, 48903
- Recruiting
Investigational Site Number : 153077
L'Hospitalet de Llobregat, Catalunya [Cataluña], 08907
- Recruiting
Investigational Site Number : 154136
Tarragona, Tarragona [Tarragona], 43005
- Recruiting
Investigational Site Number : 153213
Vigo, Pontevedra [Pontevedra], 36200
Argentina10 sites, 5 recruiting
- Recruiting
Investigational Site Number : 153280
Berazategui, Buenos Aires, B
- Recruiting
Investigational Site Number : 604002
Buenos Aires, 1107
- Completed
Investigational Site Number : 153032
Buenos Aires, 1192
- Completed
Investigational Site Number : 153172
Buenos Aires, 1280
- Recruiting
Investigational Site Number : 153260
Caba, Buenos Aires, Buenos Aires, C1415EKV
- Completed
Investigational Site Number : 153194
Catamarca, Catamarca Province, 4703
- Completed
Investigational Site Number : 032005
Neuquén, 8300
- Recruiting
Investigational Site Number : 153250
San Isidro, Buenos Aires, 1642
- Completed
Investigational Site Number : 153130
San Luis, 5700
- Recruiting
Investigational Site Number : 032004
Santa Fe, 3000
Belgium8 sites, 8 recruiting
- Recruiting
Investigational Site Number : 150722
Antwerp, 2020
- Recruiting
Investigational Site Number : 153133
Antwerp, 2020
- Recruiting
Investigational Site Number : 153001
Brussels, 1070
- Recruiting
Investigational Site Number : 0560008
Brussels, 1090
- Recruiting
Investigational Site Number : 150517
Brussels, 1200
- Recruiting
Investigational Site Number : 151668
Leuven, 3000
- Recruiting
Investigational Site Number : 154275
Liège, 4000
- Recruiting
Investigational Site Number : 153100
Seraing, 4100
United Kingdom8 sites, 8 recruiting
- Recruiting
Investigational Site Number : 154196
Belfast, BT9 7AB
- Recruiting
Investigational Site Number : 152287
Birmingham, England, B15 2TH
- Recruiting
Investigational Site Number : 151340
Cambridge, Cambridgeshire, CB2 2QQ
- Recruiting
Investigational Site Number : 151123
London, England, NW3 2QG
- Recruiting
Investigational Site Number : 151646
London, London, City of, WC1N 3BG
- Recruiting
Investigational Site Number : 150303
London, London, City of, WC1N 3JH
- Recruiting
Investigational Site Number : 150455
Manchester, M13 9wl
- Recruiting
Investigational Site Number : 152361
Salford, Manchester, M6 8HD
China7 sites, 5 recruiting
- Terminated
Investigational Site Number : 153165
Beijing, 100730
- Recruiting
Investigational Site Number : 1560003
Hangzhou, 310003
- Recruiting
Investigational Site Number : 1560002
Jinan, 250001
- Recruiting
Investigational Site Number : 1560004
Nanjing, 210008
- Terminated
Investigational Site Number : 153163
Shanghai, 200025
- Recruiting
Investigational Site Number : 1560001
Shanghai, 200025
- Recruiting
Investigational Site Number : 157261
Shanghai, 200032
Australia6 sites, 6 recruiting
- Recruiting
Investigational Site Number : 151002
Adelaide, South Australia, 5000
- Recruiting
Investigational Site Number : 153063
Brisbane, Queensland, 4029
- Recruiting
Investigational Site Number : 153040
Parkville, Victoria, 3050
- Recruiting
Investigational Site Number : 153090
Perth, Western Australia, 6000
- Recruiting
Investigational Site Number : 036013
Westmead, New South Wales, 2145
- Recruiting
Investigational Site Number : 151069
Westmead, New South Wales, 2145
France6 sites, 6 recruiting
- Recruiting
Investigational Site Number : 250005
Bordeaux, 33076
- Recruiting
Investigational Site Number : 250002
Bron, 69677
- Recruiting
Investigational Site Number : 250001
Garches, 92380
- Recruiting
Investigational Site Number : 250007
Lille, 59037
- Recruiting
Investigational Site Number : 250004
Saint-Priest-en-Jarez, 42270
- Recruiting
Investigational Site Number : 250009
Strasbourg, 67000
Japan6 sites, 4 recruiting
- Recruiting
Investigational Site Number : 153217
Chiba, 266-0007
- Active, not recruiting
Investigational Site Number : 392007
Kobe, Hyōgo, 650-0017
- Recruiting
Investigational Site Number : 153201
Nankoku, Kochi, 783-8505
- Recruiting
Investigational Site Number : 392006
Niigata, 951-8520
- Completed
Investigational Site Number : 153205
Osaka, 545-8586
- Recruiting
Investigational Site Number : 153204
Tokyo, 157-8535
Chile5 sites, 1 recruiting
- Completed
Investigational Site Number : 152004
Antofagasta, NaN
- Recruiting
Investigational Site Number : 152001
Coquimbo, 1780000
- Completed
Investigational Site Number : 152003
Iquique, NaN
- Terminated
Investigational Site Number : 152002
Santiago, NaN
- Completed
Investigational Site Number : 152005
Vallenar, NaN
Germany4 sites, 4 recruiting
- Recruiting
Investigational Site Number : 153013
Berlin, 13353
- Recruiting
Investigational Site Number : 154150
Giessen, 35392
- Recruiting
Investigational Site Number : 153147
Münster, 48149
- Recruiting
Investigational Site Number : 153011
Würzburg, 97080
Hungary4 sites, 4 recruiting
- Recruiting
Investigational Site Number : 3480002
Budapest, 1212
- Recruiting
Investigational Site Number : 3480003
Létavértes, 4281
- Recruiting
Investigational Site Number : 3480005
Pécs, 7623
- Recruiting
Investigational Site Number : 3480004
Sopron, 9400
India4 sites, 4 recruiting
- Recruiting
Investigational Site Number : 356003
Ahmedabad, 380015
- Recruiting
Investigational Site Number : 356001
Kolkata, 700017
- Recruiting
Investigational Site Number : 3560004
Secunderabad, 500026
- Recruiting
Investigational Site Number : 356002
Vellore, 632004
Thailand4 sites, 4 recruiting
- Recruiting
Investigational Site Number : 764003
Bangkok, 10400
- Recruiting
Investigational Site Number : 764004
Bangkok, 10700
- Recruiting
Investigational Site Number : 764001
Bangkok, NaN
- Recruiting
Investigational Site Number : 764005
Khon Kaen, 40002
Romania3 sites, 2 recruiting
- Recruiting
Investigational Site Number : 154164
Brasov, 500326
- Recruiting
Investigational Site Number : 154099
Bucharest, 022328
- Completed
Investigational Site Number : 153081
Cluj-Napoca, 400006
Russia3 sites, 3 recruiting
- Recruiting
Investigational Site Number : 643001
Moscow, NaN
- Recruiting
Investigational Site Number : 643003
Nizhny Novgorod, 603155
- Recruiting
Investigational Site Number : 643002
Saint Petersburg, 197341
Bulgaria2 sites, 1 recruiting
- Completed
Investigational Site Number : 153169
Sofia, 1431
- Recruiting
Investigational Site Number : 100001
Sofia, 1680
Croatia2 sites, 1 recruiting
- Completed
Investigational Site Number : 1910001
Rijeka, 51000
- Recruiting
Investigational Site Number : 1910002
Zagreb, 10000
Denmark2 sites, 1 recruiting
- Completed
Investigational Site Number : 151229
Copenhagen, 2100
- Recruiting
Investigational Site Number : 153041
Copenhagen, 2100
Lithuania2 sites, 2 recruiting
- Recruiting
Investigational Site Number : 154244
Vilnius, 08406
- Recruiting
Investigational Site Number : 154652
Vilnius, 08661
Malaysia2 sites, 2 recruiting
- Recruiting
Investigational Site Number : 458001
Kuala Lumpur, 50586
- Recruiting
Investigational Site Number : 458002
Kuala Lumpur, 59100
Netherlands2 sites
- Terminated
Investigational Site Number : 151171
Amsterdam, 1105 AZ
- Terminated
Investigational Site Number : 153004
Amsterdam, 1105 AZ
Portugal2 sites, 2 recruiting
- Recruiting
Investigational Site Number : 620005
Lisbon, 1649-035
- Recruiting
Investigational Site Number : 620001
Porto, 4200-319
Slovakia2 sites, 1 recruiting
- Completed
Investigational Site Number : 703001
Bratislava, 833 40
- Recruiting
Investigational Site Number : 705001
Nové Zámky, 940 34
Sweden2 sites, 2 recruiting
- Recruiting
Investigational Site Number : 153050
Gothenburg, 413 45
- Recruiting
Investigational Site Number : 151484
Stockholm, 171 76
Colombia1 site, 1 recruiting
- Recruiting
Investigational Site Number : 170001
Bogotá, 110131
Czechia1 site, 1 recruiting
- Recruiting
Investigational Site Number : 153126
Prague, 128 08
Estonia1 site, 1 recruiting
- Recruiting
Investigational Site Number : 153106
Tartu, 50406
Finland1 site, 1 recruiting
- Recruiting
Investigational Site Number : 153024
Turku, 20521
Hong Kong1 site, 1 recruiting
- Recruiting
Investigational Site Number : 344001
Kowloon
Indonesia1 site, 1 recruiting
- Recruiting
Investigational Site Number : 360001
Jakarta, NaN
Norway1 site, 1 recruiting
- Recruiting
Investigational Site Number : 153141
Bergen, 5021
Peru1 site, 1 recruiting
- Recruiting
Investigational Site Number : 604001
La Victoria, 15033
Philippines1 site, 1 recruiting
- Recruiting
Investigational Site Number : 608001
City of Muntinlupa, 1000
Poland1 site, 1 recruiting
- Recruiting
Investigational Site Number : 150575
Warsaw, Masovian Voivodeship, 04-730
Saudi Arabia1 site, 1 recruiting
- Recruiting
Investigational Site Number : 682002
Al-Hasa, 31982
Serbia1 site, 1 recruiting
- Recruiting
Investigational Site Number : 6880001
Novi Sad, 21000
Singapore1 site, 1 recruiting
- Recruiting
Investigational Site Number : 702001
Singapore, 229899
Vietnam1 site, 1 recruiting
- Recruiting
Investigational Site Number : 154212
Hanoi, NaN
About this study
Official title: Fabry Disease Registry Protocol
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:
Read more
- 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.
- Conditions
- Fabry Disease
- Keywords
- alpha Galactosidase A, aGAL (alpha-galactosidase), Fabry, GL3 (globotriaosylceramide), Anderson-Fabry Disease, angiokeratomas, GLA deficiency (gene deficiency), errors in metabolism
- Medical subject headings
- Fabry Disease, Angiokeratoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| Patients with Fabry disease | No experimental intervention is given. A patient with Fabry Disease will undergo clinical assessments and receive standard of care treatment as determined by the patient's physician. | |
| Pregnant women with confirmed diagnosis of Fabry | No experimental intervention is given. Pregnant women with confirmed diagnosis of Fabry that are participating in the Fabry Registry and consented to participate in the Fabry Sub-registry, regardless of whether she is receiving disease-specific therapy (such as ERT with agalsidase beta) and irrespective of the commercial product with which she may be treated. |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 33 years
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.
Measured: 33 years
Secondary outcomes (1)
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.
Measured: 33 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Sep 2005 (estimated)
- Study start31 Jul 2001
- Primary completion31 Jan 2034 (estimated)
- Study completion31 Jan 2034 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (7)Papers the sponsor has linked to this study.
- 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
- Other
- Enrolment
- 9,000 participants (estimated)
- Target duration
- 52 Weeks
Sponsor and investigators
- Lead sponsor
- Genzyme, a Sanofi Company
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Study Director, Study director, Genzyme, a Sanofi Company
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00196742
- Sponsor's protocol ID
- DIREGC07006
- Other IDs
- AGAL19211, U1111-1294-8298
- Status last verified
- Oct 2026
- First submitted
- 13 Sep 2005
- Last updated
- 14 Oct 2026
- Results posted
- No
- Data monitoring committee
- No
Publications
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 Jul;6(4):492-503. doi: 10.1002/mgg3.389. Epub 2018 Apr 12.
PubMed 29649853Wilcox 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.
PubMed 28510034Hopkin 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.
PubMed 27510433Hopkin RJ, Cabrera GH, Jefferies JL, Yang M, Ponce E, Brand E, Feldt-Rasmussen U, Germain DP, Guffon N, Jovanovic A, Kantola I, Karaa A, Martins AM, Tondel C, Wilcox WR, Yoo HW, Burlina AP, Mauer M. Clinical outcomes among young patients with Fabry disease who initiated agalsidase beta treatment before 30 years of age: An analysis from the Fabry Registry. Mol Genet Metab. 2023 Feb;138(2):106967. doi: 10.1016/j.ymgme.2022.106967. Epub 2022 Nov 30.
PubMed 36709533Wanner 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.
PubMed 36073247Ortiz 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.
PubMed 26993266Germain 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.
PubMed 25795794