Massive database tracks rare disease patients worldwide

NCT ID NCT00144794

Completed Knowledge-focused Sponsor: Genzyme, a Sanofi Company Source: ClinicalTrials.gov ↗

Summary

This study created a large international registry to track people with Mucopolysaccharidosis I (MPS I), a rare genetic disorder. It followed 1,406 patients to better understand how the disease naturally progresses and how treatments like enzyme replacement therapy work over the long term. The goal was to collect real-world information to help doctors improve care for people with MPS I.

This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes NO responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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Contacts and locations

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Locations

Ann & Robert H. Lurie Children's Hospital of Chicago- Site Number : 840011

Chicago, Illinois, 60611, United States
Arkansas Children's Hospital- Site Number : 840109

Little Rock, Arkansas, 72202, United States
Baylor Institute of Metabolic Disease- Site Number : 840058

Dallas, Texas, 75226, United States
Baystate Medical Center- Site Number : 840002

Springfield, Massachusetts, 01199, United States
Boston Children's Hospital - PIN- Site Number : 840093

Boston, Massachusetts, 2115, United States
Children's Hospital Colorado - Aurora- Site Number : 840069

Aurora, Colorado, 80045, United States
Children's Hospital of Orange County- Site Number : 840074

Orange, California, 92868, United States
Children's Hospital of Philadelphia HUP Medical Genetics- Site Number : 840089

Philadelphia, Pennsylvania, 19104, United States
Children's Hospital of Philadelphia- Site Number : 840034

Philadelphia, Pennsylvania, United States
Children's Hospital of The King's Daughters- Site Number : 840072

Norfolk, Virginia, 23507, United States
Children's Hospital of Wisconsin-Pediatrics- Site Number : 840054

Milwaukee, Wisconsin, 53226, United States
Children's National Medical Center - Washington- Site Number : 840067

Washington D.C., District of Columbia, 20010, United States
Children's Specialty Center - Las Vegas- Site Number : 840008

Las Vegas, Nevada, 89109, United States
Cincinnati Children's Hospital-Genetics- Site Number : 840041

Cincinnati, Ohio, United States
Duke University Medical Center Genetics Dept- Site Number : 840037

Durham, North Carolina, 27710, United States
Emory University School of Medicine- Human Genetics- Site Number : 840060

Decatur, Georgia, 30033, United States
Greenwood Genetic Center - Greenville- Site Number : 840055

Greenville, South Carolina, 29605, United States
Hospital e Maternidade Celso Pierro - PUC-Campinas- Site Number : 076007

Campinas, São Paulo, 13034-685, Brazil
Indianapolis University School of Medicine- Site Number : 840027

Indianapolis, Indiana, 46202, United States
Instituto Fernandes Figueira - FIOCRUZ- Site Number : 076004

Rio de Janeiro, 22250-020, Brazil
Instituto de Genética e Erros Inatos do Metabolismo- Site Number : 076002

São Paulo, 04020-041, Brazil
Investigational Site Number : 124001

Sherbrooke, Quebec, J1K 2R1, Canada
Investigational Site Number : 124002

Edmonton, Alberta, T6G 2B7, Canada
Investigational Site Number : 124006

Toronto, Ontario, M5G 1X8, Canada
Investigational Site Number : 124011

Vancouver, British Columbia, V6H 3N1, Canada
Investigational Site Number : 124014

Calgary, Alberta, T2E 7Z4, Canada
Investigational Site Number : 124018

Ottawa, Ontario, K1K 4L2, Canada
Investigational Site Number : 124019

Winnipeg, Manitoba, R3A 1R9, Canada
Investigational Site Number : 124021

London, Ontario, N6A 5A5, Canada
Investigational Site Number : 150031

Mainz, 55131, Germany
Investigational Site Number : 150303

London, London, City of, WC1N 3JH, United Kingdom
Investigational Site Number : 150455

Manchester, Missouri, M13 9WL, United Kingdom
Investigational Site Number : 150575

Warsaw, 04-730, Poland
Investigational Site Number : 150659

Prague, 12808, Czechia
Investigational Site Number : 150944

Brussels, 1090, Belgium
Investigational Site Number : 151069

Westmead, New South Wales, 2145, Australia
Investigational Site Number : 151123

London, England, NW3 2QG, United Kingdom
Investigational Site Number : 151171

Amsterdam, Netherlands
Investigational Site Number : 151179

Taichung, 404, Taiwan
Investigational Site Number : 151182

Taipei, 100, Taiwan
Investigational Site Number : 151229

Copenhagen, 2100, Denmark
Investigational Site Number : 151368

Hamburg, 20246, Germany
Investigational Site Number : 151500

Putz City, Chia-yi, Taiwan
Investigational Site Number : 151646

London, London, City of, WC1N 3BG, United Kingdom
Investigational Site Number : 151668

Leuven, 3000, Belgium
Investigational Site Number : 152001

Talcahuano, Región del Biobío, 2687000, Chile
Investigational Site Number : 152003

Los Ángeles, Chile
Investigational Site Number : 152029

Rotterdam, 3015 CE, Netherlands
Investigational Site Number : 152153

Tainan, 710, Taiwan
Investigational Site Number : 152156

Glasgow, Glasgow City, G51 4TF, United Kingdom
Investigational Site Number : 152246

Kaohsiung City, 833, Taiwan
Investigational Site Number : 152321

Taipei, 104, Taiwan
Investigational Site Number : 152361

Salford, Manchester, M6 8HD, United Kingdom
Investigational Site Number : 153032

Buenos Aires, 1192, Argentina
Investigational Site Number : 153130

San Luis, 5700, Argentina
Investigational Site Number : 153191

Daejeon, South Korea
Investigational Site Number : 153212

Seoul, South Korea
Investigational Site Number : 153222

Seoul, South Korea
Investigational Site Number : 153225

Jeonnam, South Korea
Investigational Site Number : 154008

Roeselare, 8800, Belgium
Investigational Site Number : 154093

Chiayi City, Taiwan
Investigational Site Number : 154161

Dublin, D01 F772, Ireland
Investigational Site Number : 154177

Cluj-Napoca, 400006, Romania
Investigational Site Number : 154212

Hanoi, Vietnam
Investigational Site Number : 154225

Kuwait City, Kuwait
Investigational Site Number : 154240

Birmingham, England, B4 6NH, United Kingdom
Investigational Site Number : 154264

Lahore, Pakistan
Investigational Site Number : 154266

Lahore, Pakistan
Investigational Site Number : 170001

Bogotá, Colombia
Investigational Site Number : 250001

Bron, 69677, France
Investigational Site Number : 250002

Marseille, 13885, France
Investigational Site Number : 250003

Paris, 75015, France
Investigational Site Number : 250004

Bordeaux, 33076, France
Investigational Site Number : 250006

Vandœuvre-lès-Nancy, 54511, France
Investigational Site Number : 344001

Kowloon, Hong Kong
Investigational Site Number : 356001

Kolkata, 700017, India
Investigational Site Number : 356002

Vellore, 632004, India
Investigational Site Number : 356003

Ahmedabad, 380015, India
Investigational Site Number : 360001

Jakarta, Indonesia
Investigational Site Number : 380002

Ancona, 60126, Italy
Investigational Site Number : 380003

Genova, 16147, Italy
Investigational Site Number : 380004

Cusano Milanino, Milano, 20095, Italy
Investigational Site Number : 380005

Naples, Italy
Investigational Site Number : 380006

Rome, Roma, 00165, Italy
Investigational Site Number : 380007

Milan, Milano, 20142, Italy
Investigational Site Number : 380008

Padua, 35128, Italy
Investigational Site Number : 392101

Tokyo, 157-8535, Japan
Investigational Site Number : 392102

Nankoku, Kochi, 783-8505, Japan
Investigational Site Number : 4220004

Beirut, 11072210, Lebanon
Investigational Site Number : 458001

Kuala Lumpur, 50586, Malaysia
Investigational Site Number : 458002

Kuala Lumpur, 59100, Malaysia
Investigational Site Number : 608001

Manila, 1000, Philippines
Investigational Site Number : 6200002

Coimbra, 3000-075, Portugal
Investigational Site Number : 643001

Moscow, 119571, Russia
Investigational Site Number : 643002

Moscow, 125412, Russia
Investigational Site Number : 682001

Al Qaţīf, Saudi Arabia
Investigational Site Number : 682004

Mecca, 24246, Saudi Arabia
Investigational Site Number : 703001

Bratislava, 833 40, Slovakia
Investigational Site Number : 7520001

Stockholm, 171 76, Sweden
Investigational Site Number : 764001

Bangkok, Thailand
Investigational Site Number : 764002

Bangkok, 10400, Thailand
Investigational Site Number : 764003

Bangkok, 10400, Thailand
Investigational Site Number : 764004

Bangkok, 10700, Thailand
Investigational Site Number : 818001

Cairo, Egypt
Investigational Site Number : 818002

Cairo, 11231, Egypt
John Hopkins- Site Number : 840044

Baltimore, Maryland, United States
Joseph M. Sanzari Children's Hospital- Site Number : 840101

Hackensack, New Jersey, 07601, United States
Massachusetts General Hospital-Genetics- Site Number : 840062

Boston, Massachusetts, 02114, United States
Nationwide Children's Hospital- Site Number : 840091

Columbus, Ohio, 43205, United States
New York University School Of Medicine- Site Number : 840040

New York, New York, 10016, United States
Northwell Health - Site Number : 840102

Manhasset, New York, 11030, United States
Oregon Health & Science University (OHSU)- Site Number : 840080

Portland, Oregon, 97239, United States
Pediatric Endocrine Associates- Site Number : 840017

Tampa, Florida, 33612, United States
Phoenix Children's Hospital- Site Number : 840003

Phoenix, Arizona, 85016, United States
Private Practice - Dr. Fabiano Klaus- Site Number : 076001

Porto Alegre, Rio Grande do Sul, 90035-002, Brazil
Rhode Island Hospital Genetics- Site Number : 840053

Providence, Rhode Island, 02903, United States
Rush University Medical Center Genetics- Site Number : 840079

Chicago, Illinois, 60612, United States
Saint Peter's University Hospital - New Brunswick- Site Number : 840016

New Brunswick, New Jersey, 08901, United States
Seattle Children's Hospital- Site Number : 840028

Seattle, Washington, 98105, United States
Southern California Permanente Medical Group- Site Number : 840108

Los Angeles, California, 90027, United States
Stanford Unviersity MC Dept of Genetics- Site Number : 840022

Palo Alto, California, 94305, United States
State University of New York- Site Number : 840052

Syracuse, New York, 13210, United States
UC Davis MIND Institute- Site Number : 840010

Sacramento, California, 95817, United States
UCSF Benioff Children's Hospital - Oakland- Site Number : 840029

Oakland, California, 94609, United States
USC Health Sciences Center Dept of Genetics- Site Number : 840082

Los Angeles, California, 90033, United States
University Of Miami SOM- Site Number : 840006

Miami, Florida, 33136, United States
University Of Washington Medical Center- Site Number : 840059

Seattle, Washington, 98195, United States
University of California at San Diego- Site Number : 840007

San Diego, California, 92093, United States
University of California at San Francisco- Site Number : 840051

San Francisco, California, United States
University of Colorado at Denver Genetics Site Number : 840068

Aurora, Colorado, 80045, United States
University of Florida Dept of Genetics- Site Number : 840083

Gainesville, Florida, 32610, United States
University of Iowa- Site Number : 840032

Iowa City, Iowa, 52242, United States
University of Louisville- Genetics- Site Number : 840030

Louisville, Kentucky, 40202, United States
University of Michigan Pediatrics- Site Number : 840107

Ann Arbor, Michigan, 48109, United States
University of Minnesota Medical Center Pediatrics- Site Number : 840076

Minneapolis, Minnesota, 55455, United States
University of Nebraska Medical Center- Site Number : 840084

Omaha, Nebraska, 68198, United States
University of Pittsburgh- Site Number : 840023

Pittsburgh, Pennsylvania, 15224, United States
University of Utah Health Hospital- Site Number : 840092

Salt Lake City, Utah, 84132, United States
University of Virginia School of Medicine-Pediatrics- Site Number : 840078

Charlottesville, Virginia, 22908, United States
Washington University- Site Number : 840100

St Louis, Missouri, 63110, United States
Yale University School of Medicine- Site Number : 840047

New Haven, Connecticut, 06510, United States

Conditions

Explore the condition pages connected to this study.

MUCOPOLYSACCHARIDOSIS I (MPS I)