RecruitingNCT00196742

Fabry Disease Registry & Pregnancy Sub-registry

Fabry Disease Registry Protocol


Sponsor

Genzyme, a Sanofi Company

Enrollment

9,000 participants

Start Date

Jul 31, 2001

Study Type

OBSERVATIONAL

Conditions

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

Plain Language Summary

Simplified for easier understanding

This registry study is collecting long-term health data from people with Fabry disease — a rare inherited metabolic condition — including a sub-registry specifically for pregnant women with Fabry disease. **You may be eligible if...** - You have a confirmed diagnosis of Fabry disease (documented enzyme deficiency or known gene mutation) - For the pregnancy sub-registry: you are enrolled in the main Fabry Registry and are currently or have previously been pregnant - You have signed the informed consent form **You may NOT be eligible if...** - There are no exclusion criteria for either the main registry or the pregnancy sub-registry Talk to your doctor to see if this trial is right for you.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Locations(283)

University of Alabama Birmingham- Nephrology- Site Number : 840018

Birmingham, Alabama, United States

University of Alabama Birmingham- Nephrology- Site Number : 840073

Birmingham, Alabama, United States

Phoenix Children's Hospital- Site Number : 840003

Phoenix, Arizona, United States

University of Arizona- Site Number : 840015

Tucson, Arizona, United States

Arkansas Children's Hospital- Site Number : 840109

Little Rock, Arkansas, United States

University of Arkansas for Medical Sciences- Site Number : 840113

Little Rock, Arkansas, United States

University of California at Irvine- Site Number : 840036

Irvine, California, United States

Southern California Permanente Medical Group- Site Number : 840108

Los Angeles, California, United States

USC Health Sciences Center Dept of Genetics- Site Number : 840082

Los Angeles, California, United States

UCLA School Of Medicine- Site Number : 840088

Los Angeles, California, United States

UCSF Benioff Children's Hospital - Oakland- Site Number : 840029

Oakland, California, United States

Children's Hospital of Orange County- Site Number : 840074

Orange, California, United States

UC Davis MIND Institute- Site Number : 840010

Sacramento, California, United States

Kaiser Permanente Sacramento Medical Center- Site Number : 840042

Sacramento, California, United States

University of California at San Diego- Site Number : 840007

San Diego, California, United States

University of California San Francisco - Parnassus Campus- Site Number : 840051

San Francisco, California, United States

Stanford University Medical Center- Site Number : 840022

Stanford, California, United States

Yale University School of Medicine- Site Number : 840047

New Haven, Connecticut, United States

Children's National Medical Center - Washington- Site Number : 840067

Washington D.C., District of Columbia, United States

University Hematology Oncology- Site Number : 840075

Coral Springs, Florida, United States

University of Florida Dept of Genetics- Site Number : 840083

Gainesville, Florida, United States

University of Florida Pediatrics Genetics- Site Number : 840121

Jacksonville, Florida, United States

University Of Miami SOM- Site Number : 840006

Miami, Florida, United States

University of Florida-Genetics- Site Number : 840096

Tampa, Florida, 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

Aurora, Illinois, United States

Ann & Robert H. Lurie Children's Hospital of Chicago- Site Number : 840013

Chicago, Illinois, United States

Rush University Medical Center Genetics- Site Number : 840079

Chicago, Illinois, 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 Kansas Medical Center- Site Number : 840071

Kansas City, Kansas, United States

University of Louisville - Health Sciences Center- Site Number : 840030

Louisville, Kentucky, United States

Ochsner Health Center - Baton Rouge- Site Number : 840120

Baton Rouge, Louisiana, United States

Tulane University School of Medicine- Site Number : 840001

New Orleans, Louisiana, United States

Maine Medical Partners Pediatric Specialty Care- Site Number : 840064

Portland, Maine, United States

John Hopkins- Site Number : 840044

Baltimore, Maryland, United States

Massachusetts General Hospital-Genetics- Site Number : 840062

Boston, Massachusetts, United States

Boston Children's Hospital - PIN- Site Number : 840092

Boston, Massachusetts, United States

Brigham & Women's Hospital- Site Number : 840103

Boston, Massachusetts, United States

Baystate Medical Center- Site Number : 840002

Springfield, Massachusetts, United States

University of Michigan Pediatrics- Site Number : 840107

Ann Arbor, Michigan, United States

Children's Hospital of Michigan- Site Number : 840066

Detroit, Michigan, United States

Corewell Health - Helen DeVos Children's Hospital (HDVCH)- Site Number : 840019

Grand Rapids, Michigan, United States

Infusion Associates - Grand Rapids- Site Number : 840050

Grand Rapids, Michigan, United States

Children's Hospitals and Clinics of Minnesota- Site Number : 840046

Minneapolis, Minnesota, United States

Children's Hospitals and Clinics of Minnesota- Site Number : 840114

Minneapolis, Minnesota, United States

M Health Fairview University of Minnesota Medical Center - West Bank- Site Number : 840076

Minneapolis, Minnesota, United States

University of Missouri Health System Department of Genetics- Site Number : 840031

Columbia, Missouri, United States

Washington University- Site Number : 840100

St Louis, Missouri, United States

Shodair Children's Hospital- Site Number : 840090

Helena, Montana, United States

University of Nebraska Medical Center- Site Number : 840084

Omaha, Nebraska, United States

Children's Specialty Center - Las Vegas- Site Number : 840008

Las Vegas, Nevada, United States

Cooper Health Center- Site Number : 840098

Camden, New Jersey, United States

Joseph M. Sanzari Children's Hospital- Site Number : 840101

Hackensack, New Jersey, United States

Atlantic Health System- Site Number : 840099

Morristown, New Jersey, United States

Saint Peter's University Hospital - New Brunswick- Site Number : 840016

New Brunswick, New Jersey, United States

St. Joseph's Children's Hospital- Site Number : 840057

Paterson, New Jersey, United States

NYU Langone Health - Perlmutter Cancer Center - Huntington- Site Number : 840040

Huntington, New York, United States

Northwell Health- Site Number : 840102

New Hyde Park, New York, United States

Icahn School of Medicine at Mount Sinai- Site Number : 840080

New York, New York, United States

Metropolitan Hospital Center- Site Number : 840110

New York, New York, United States

University of Rochester Medical Center- Site Number : 840105

Rochester, New York, United States

SUNY Upstate Medical University - Site Number : 840052

Syracuse, New York, United States

New York Medical College- Site Number : 840039

Valhalla, New York, United States

Carolinas Medical Center- Site Number : 840065

Charlotte, North Carolina, United States

Duke University Medical Center Genetics Dept- Site Number : 840045

Durham, North Carolina, United States

Cincinnati Children's Hospital Medical Center (Cincinnati Children's Hospital)- Site Number : 840033

Cincinnati, Ohio, United States

University Hospitals Cleveland Medical Center- Site Number : 840119

Cleveland, Ohio, United States

Cleveland Clinic - Cleveland- Site Number : 840048

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

LSD Data Registry Site LLC- Site Number : 840094

Dublin, Ohio, United States

Oregon Health & Science University (OHSU)- Site Number : 840116

Portland, Oregon, United States

Oregon Health and Scinece University- Site Number : 840038

Portland, Oregon, United States

Geisinger Medical Center- Site Number : 840111

Danville, Pennsylvania, United States

Penn State University Medical Center-Neurology- Site Number : 840104

Hershey, Pennsylvania, 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

UPMC Children's Hospital of Pittsburgh- Site Number : 840023

Pittsburgh, Pennsylvania, United States

Rhode Island Hospital Genetics- Site Number : 840053

Providence, Rhode Island, United States

Greenwood Genetic Center - Greenville- Site Number : 840055

Greenville, South Carolina, United States

Vanderbilt University Medical Center Diagnostic Lab- Site Number : 840049

Nashville, Tennessee, United States

Baylor Institute of Metabolic Disease- Site Number : 840058

Dallas, Texas, United States

Cook Children's Health Care System- Site Number : 840024

Fort Worth, Texas, United States

University of Utah Department of Medical Genetics- Site Number : 840043

Salt Lake City, Utah, United States

University of Virginia School of Medicine-Pediatrics- Site Number : 840078

Charlottesville, Virginia, United States

O & O Alpan- Site Number : 840025

Fairfax, Virginia, United States

Children's Hospital of The King's Daughters- Site Number : 840072

Norfolk, Virginia, 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

Mary Bridge Children's Outpatient Center- Site Number : 840115

Tacoma, Washington, United States

Children's Hospital of Wisconsin-Pediatrics- Site Number : 840054

Milwaukee, Wisconsin, United States

Investigational Site Number : 153280

Berazategui, Buenos Aires, Argentina

Investigational Site Number : 153260

Caba, Buenos Aires, Buenos Aires, Argentina

Investigational Site Number : 153250

San Isidro, Buenos Aires, Argentina

Investigational Site Number : 153194

Catamarca, Catamarca Province, Argentina

Investigational Site Number : 604002

Buenos Aires, Argentina

Investigational Site Number : 153032

Buenos Aires, Argentina

Investigational Site Number : 153172

Buenos Aires, Argentina

Investigational Site Number : 153270

La Rioja Province, Argentina

Investigational Site Number : 032005

Neuquén, Argentina

Investigational Site Number : 153130

San Luis, Argentina

Investigational Site Number : 032004

Santa Fe, Argentina

Investigational Site Number : 036013

Westmead, New South Wales, Australia

Investigational Site Number : 151069

Westmead, New South Wales, Australia

Investigational Site Number : 153063

Brisbane, Queensland, Australia

Investigational Site Number : 151002

Adelaide, South Australia, Australia

Investigational Site Number : 153040

Parkville, Victoria, Australia

Investigational Site Number : 153090

Perth, Western Australia, Australia

Investigational Site Number : 150722

Antwerp, Belgium

Investigational Site Number : 153133

Antwerp, Belgium

Investigational Site Number : 153001

Brussels, Belgium

Investigational Site Number : 0560008

Brussels, Belgium

Investigational Site Number : 150517

Brussels, Belgium

Investigational Site Number : 151668

Leuven, Belgium

Investigational Site Number : 154275

Liège, Belgium

Investigational Site Number : 153100

Seraing, Belgium

NPDM - Núcleo de Pesquisa e Desenvolvimento de Medicamentos- Site Number : 076007

Fortaleza, Ceará, Brazil

Clínica Senhor do Bonfim- Site Number : 076008

Feira de Santana, Estado de Bahia, Brazil

Hospital Geral Universitario de Cuiaba- Site Number : 076006

Cuiabá, Mato Grosso, Brazil

Universidade Federal do Paraná- Site Number : 076004

Curitiba, Paraná, Brazil

Instituto de Medicina Integral Professor Fernando Figueira- Site Number : 076005

Recife, Pernambuco, Brazil

Hospital Universitário Polydoro Ernani de São Thiago - UFSC- Site Number : 076012

Florianópolis, Santa Catarina, Brazil

Hospital e Maternidade Celso Pierro - PUC-Campinas- Site Number : 076013

Campinas, São Paulo, Brazil

Centro de Pesq Materno Infantil de Campinas- Site Number : 076009

Campinas, São Paulo, Brazil

Serviço de Terapia Renal de Ourinhos- Site Number : 076011

Ourinhos, São Paulo, Brazil

Centro Saude Escola Joel Domingos Machado- Site Number : 076002

Ribeirão Preto, São Paulo, Brazil

Instituto de Genética e Erros Inatos do Metabolismo- Site Number : 076001

São Paulo, Brazil

Investigational Site Number : 153169

Sofia, Bulgaria

Investigational Site Number : 100001

Sofia, Bulgaria

Investigational Site Number : 124014

Calgary, Alberta, Canada

Investigational Site Number : 124002

Edmonton, Alberta, Canada

Investigational Site Number : 124010

Vancouver, British Columbia, Canada

Investigational Site Number : 124011

Vancouver, British Columbia, Canada

Investigational Site Number : 124019

Winnipeg, Manitoba, Canada

Investigational Site Number : 124022

Moncton, New Brunswick, Canada

Investigational Site Number : 124023

St. John's, Newfoundland and Labrador, Canada

Investigational Site Number : 124008

Halifax, Nova Scotia, Canada

Investigational Site Number : 124005

Kingston, Ontario, Canada

Investigational Site Number : 124021

London, Ontario, Canada

Investigational Site Number : 124006

Toronto, Ontario, Canada

Investigational Site Number : 124009

Toronto, Ontario, Canada

Investigational Site Number : 124013

Montreal, Quebec, Canada

Investigational Site Number : 124001

Sherbrooke, Quebec, Canada

Investigational Site Number : 152004

Antofagasta, Chile

Investigational Site Number : 152001

Coquimbo, Chile

Investigational Site Number : 152003

Iquique, Chile

Investigational Site Number : 152002

Santiago, Chile

Investigational Site Number : 152005

Vallenar, Chile

Investigational Site Number : 153165

Beijing, China

Investigational Site Number : 1560003

Hangzhou, China

Investigational Site Number : 1560002

Jinan, China

Investigational Site Number : 1560004

Nanjing, China

Investigational Site Number : 153163

Shanghai, China

Investigational Site Number : 1560001

Shanghai, China

Investigational Site Number : 170001

Bogotá, Colombia

Investigational Site Number : 1910001

Rijeka, Croatia

Investigational Site Number : 1910002

Zagreb, Croatia

Investigational Site Number : 153126

Prague, Czechia

Investigational Site Number : 151229

Copenhagen, Denmark

Investigational Site Number : 153041

Copenhagen, Denmark

Investigational Site Number : 153106

Tartu, Estonia

Investigational Site Number : 153024

Turku, Finland

Investigational Site Number : 250005

Bordeaux, France

Investigational Site Number : 250002

Bron, France

Investigational Site Number : 250001

Garches, France

Investigational Site Number : 250007

Lille, France

Investigational Site Number : 250004

Saint-Priest-en-Jarez, France

Investigational Site Number : 250009

Strasbourg, France

Investigational Site Number : 153013

Berlin, Germany

Investigational Site Number : 154150

Giessen, Germany

Investigational Site Number : 153147

Münster, Germany

Investigational Site Number : 153011

Würzburg, Germany

Investigational Site Number : 344001

Kowloon, Hong Kong

Investigational Site Number : 3480002

Budapest, Hungary

Investigational Site Number : 3480003

Létavértes, Hungary

Investigational Site Number : 3480005

Pécs, Hungary

Investigational Site Number : 3480004

Sopron, Hungary

Investigational Site Number : 356003

Ahmedabad, India

Investigational Site Number : 356001

Kolkata, India

Investigational Site Number : 3560004

Secunderabad, India

Investigational Site Number : 356002

Vellore, India

Investigational Site Number : 360001

Jakarta, Indonesia

Investigational Site Number : 380013

Florence, Firenze, Italy

Investigational Site Number : 380009

Milan, Milano, Italy

Investigational Site Number : 380006

Milan, Milano, Italy

Investigational Site Number : 380003

Baggiovara, Modena, Italy

Investigational Site Number : 380010

Monza, Monza E Brianza, Italy

Investigational Site Number : 157282

Padua, Padova, Italy

Investigational Site Number : 380012

Rome, Roma, Italy

Investigational Site Number : 380002

Bassano del Grappa, Italy

Investigational Site Number : 380019

Bologna, Italy

Investigational Site Number : 380004

Florence, Italy

Investigational Site Number : 380008

Naples, Italy

Investigational Site Number : 380008

Naples, Italy

Investigational Site Number : 380014

Ragusa, Italy

Investigational Site Number : 380007

Rimini, Italy

Investigational Site Number : 380011

Torino, Italy

Investigational Site Number : 380001

Udine, Italy

Investigational Site Number : 392007

Kobe, Hyōgo, Japan

Investigational Site Number : 153201

Nankoku, Kochi, Japan

Investigational Site Number : 153217

Chiba, Japan

Investigational Site Number : 392006

Niigata, Japan

Investigational Site Number : 153205

Osaka, Japan

Investigational Site Number : 153204

Tokyo, Japan

Investigational Site Number : 154244

Vilnius, Lithuania

Investigational Site Number : 154652

Vilnius, Lithuania

Investigational Site Number : 458001

Kuala Lumpur, Malaysia

Investigational Site Number : 458002

Kuala Lumpur, Malaysia

Investigational Site Number : 151171

Amsterdam, Netherlands

Investigational Site Number : 153004

Amsterdam, Netherlands

Investigational Site Number : 153141

Bergen, Norway

Investigational Site Number : 604001

Lima, Peru

Investigational Site Number : 608001

City of Muntinlupa, Philippines

Investigational Site Number : 150575

Warsaw, Poland

Investigational Site Number : 620005

Lisbon, Portugal

Investigational Site Number : 620001

Porto, Portugal

Investigational Site Number : 154164

Brasov, Romania

Investigational Site Number : 154099

Bucharest, Romania

Investigational Site Number : 153081

Cluj-Napoca, Romania

Investigational Site Number : 643001

Moscow, Russia

Investigational Site Number : 643003

Nizhny Novgorod, Russia

Investigational Site Number : 643002

Saint Petersburg, Russia

Investigational Site Number : 682002

Al-Hasa, Saudi Arabia

Investigational Site Number : 6880001

Novi Sad, Serbia

Investigational Site Number : 702001

Singapore, Singapore

Investigational Site Number : 703001

Bratislava, Slovakia

Investigational Site Number : 705001

Nové Zámky, Slovakia

Investigational Site Number : 154273

Suwon, Gyeonggi-do, South Korea

Investigational Site Number : 153178

Yangsan, Gyeongsangnam-do, South Korea

Investigational Site Number : 154208

Yangsan, Gyeongsangnam-do, South Korea

Investigational Site Number : 157176

Yangsan, Gyeongsangnam-do, South Korea

Investigational Site Number : 153228

Jeju City, Jeju-teukbyeoljachido, South Korea

Investigational Site Number : 153180

Seoul, Seoul-teukbyeolsi, South Korea

Investigational Site Number : 153196

Seoul, Seoul-teukbyeolsi, South Korea

Investigational Site Number : 153192

Seoul, Seoul-teukbyeolsi, South Korea

Investigational Site Number : 151149

Seoul, Seoul-teukbyeolsi, South Korea

Investigational Site Number : 153222

Seoul, Seoul-teukbyeolsi, South Korea

Investigational Site Number : 154246

Busan, South Korea

Investigational Site Number : 153191

Daejeon, South Korea

Investigational Site Number : 153188

Seoul, South Korea

Investigational Site Number : 153212

Seoul, South Korea

Investigational Site Number : 154146

A Coruña, A Coruña [La Coruña], Spain

Investigational Site Number : 154159

A Coruña, A Coruña [La Coruña], Spain

Investigational Site Number : 153237

Barcelona, Barcelona [Barcelona], Spain

Investigational Site Number : 154147

Barcelona, Barcelona [Barcelona], Spain

Investigational Site Number : 154135

Barakaldo, Basque Country, Spain

Investigational Site Number : 153067

Barcelona, Catalunya [Cataluña], Spain

Investigational Site Number : 154204

Barcelona, Catalunya [Cataluña], Spain

Investigational Site Number : 153077

L'Hospitalet de Llobregat, Catalunya [Cataluña], Spain

Investigational Site Number : 153213

Vigo, Pontevedra [Pontevedra], Spain

Investigational Site Number : 154136

Tarragona, Tarragona [Tarragona], Spain

Investigational Site Number : 154123

Barcelona, Spain

Investigational Site Number : 153050

Gothenburg, Sweden

Investigational Site Number : 151484

Stockholm, Sweden

Investigational Site Number : 151500

Chiayi City, Taiwan

Investigational Site Number : 154093

Chiayi City, Taiwan

Investigational Site Number : 152152

Hualien City, Taiwan

Investigational Site Number : 152244

Kaohsiung City, Taiwan

Investigational Site Number : 153088

Kaohsiung City, Taiwan

Investigational Site Number : 152246

Kaohsiung City, Taiwan

Investigational Site Number : 151534

New Taipei City, Taiwan

Investigational Site Number : 151179

Taichung, Taiwan

Investigational Site Number : 151181

Taichung, Taiwan

Investigational Site Number : 153248

Tainan, Taiwan

Investigational Site Number : 152153

Tainan, Taiwan

Investigational Site Number : 151182

Taipei, Taiwan

Investigational Site Number : 152321

Taipei, Taiwan

Investigational Site Number : 152226

Taipei, Taiwan

Investigational Site Number : 153158

Tao Yuan County, Taiwan

Investigational Site Number : 764003

Bangkok, Thailand

Investigational Site Number : 764004

Bangkok, Thailand

Investigational Site Number : 764001

Bangkok, Thailand

Investigational Site Number : 764005

Khon Kaen, Thailand

Investigational Site Number : 151340

Cambridge, Cambridgeshire, United Kingdom

Investigational Site Number : 152287

Birmingham, England, United Kingdom

Investigational Site Number : 151123

London, England, United Kingdom

Investigational Site Number : 151646

London, London, City of, United Kingdom

Investigational Site Number : 150303

London, London, City of, United Kingdom

Investigational Site Number : 152361

Salford, Manchester, United Kingdom

Investigational Site Number : 154196

Belfast, United Kingdom

Investigational Site Number : 150455

Manchester, United Kingdom

Investigational Site Number : 154212

Hanoi, Vietnam

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NCT00196742


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