Mucopolysaccharidosis I (MPS I) Registry

Recruiting

• Conditions: Mucopolysaccharidosis I (MPS I)

• Registration Number: NCT00144794
• Lead Sponsor: Genzyme, a Sanofi Company

Brief Summary

The Mucopolysaccharidosis I (MPS I) Registry is an ongoing, observational database that tracks the outcomes of patients with MPS I. The data collected by the MPS I Registry will provide information to better characterize the natural history and progression of MPS I as well as the clinical responses of patients receiving enzyme replacement therapy, such as Aldurazyme (Recombinant Human Alpha-L-Iduronidase), or other treatment modalities.

The objectives of the Registry are:

* To evaluate the long-term effectiveness and safety of Aldurazyme® (laronidase)

* To characterize and describe the MPS I population as a whole, including the variability, progression, and natural history of MPS I

* To help the MPS I medical community with the development of recommendations for monitoring patients and reports on patient outcomes to optimize patient care

Detailed Description

The MPS I Registry is an international program; in addition to the central contact information provided under the "Location" heading, patients may contact:

* In Asia-Pacific - Vivian Liu, +65-6431-2548, Vivian.liu@genzyme.com

* In Europe - +31-35-699-1232, europe@mpsiregistry.com

* In Latin America - +617-591-5500, help@mpsiregistry.com

* In North America - +617-591-5500, help@mpsiregistry.com

Study Timeline

• Study Start: 2003-11-20
• Study First Submitted: 2005-09-02
• Study First Submitted QC: 2005-09-02
• Study First Posted: 2005-09-05
• Status Verified: 2024-11
• Last Update Submitted: 2024-11-19
• Last Update Posted: 2024-11-20
• Primary Completion: 2034-01-31
• Study Completion: 2034-01-31

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 1500

Inclusion Criteria

• All patients with a confirmed diagnosis of MPS I are eligible for inclusion. Confirmed diagnosis is defined as: A. documented biochemical evidence of a deficiency in alpha (a)-L-iduronidase enzyme activity and/or B. mutation(s) in the gene coding for a-L-iduronidase, or measurable clinical signs and symptoms of MPS I
• For all patients there should be a completed patient authorization form

Exclusion Criteria

• No exclusion criteria for participation in the MPS I Registry. NOTE: Registry participation does not exclude participation in other clinical studies.

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
To evaluate the long-term effectiveness of Aldurazyme	Approximately 17 Years

Trial Locations

Locations (180)

Investigational Site Number : 392102; 🇯🇵 Nangoku-shi, Kochi, Japan

Investigational Site Number : 392101; 🇯🇵 Tokyo, Japan

Investigational Site Number : 703001; 🇸🇰 Bratislava, Slovakia

Investigational Site Number : 7520001; 🇸🇪 Stockholm, Sweden

Investigational Site Number : 154093; 🇨🇳 Chiayi, Taiwan

Investigational Site Number : 152152; 🇨🇳 Hualien, Taiwan

Investigational Site Number : 152246; 🇨🇳 Kaohsiung City, Taiwan

Investigational Site Number : 152244; 🇨🇳 Kaohsiung, Taiwan

Investigational Site Number : 151534; 🇨🇳 New Taipei City, Taiwan

Investigational Site Number : 151500; 🇨🇳 Putz City, Chia-yi, Taiwan

Investigational Site Number : 151181; 🇨🇳 Taichung, Taiwan

Investigational Site Number : 151179; 🇨🇳 Taichung, Taiwan

Investigational Site Number : 153248; 🇨🇳 Tainan City, Taiwan

Investigational Site Number : 152153; 🇨🇳 Tainan City, Taiwan

Investigational Site Number : 151182; 🇨🇳 Taipei City, Taiwan

Investigational Site Number : 152321; 🇨🇳 Taipei, Taiwan

Investigational Site Number : 153158; 🇨🇳 Taoyuan City, Taiwan

Investigational Site Number : 764002; 🇹🇭 Bangkok, Thailand

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 : 152287; 🇬🇧 Birmingham, United Kingdom

Investigational Site Number : 154240; 🇬🇧 Birmingham, United Kingdom

Investigational Site Number : 152156; 🇬🇧 Glasgow, United Kingdom

Investigational Site Number : 151646; 🇬🇧 London, United Kingdom

Investigational Site Number : 151123; 🇬🇧 London, United Kingdom

Investigational Site Number : 150303; 🇬🇧 London, United Kingdom

Investigational Site Number : 150455; 🇬🇧 Manchester, United Kingdom

Investigational Site Number : 152361; 🇬🇧 Salford, United Kingdom

Investigational Site Number : 154212; 🇻🇳 Hanoi, Vietnam

Investigational Site Number : 704002; 🇻🇳 Ho Chi Minh City, Vietnam

Investigational Site Number : 380006; 🇮🇹 Roma, Italy

Investigational Site Number : 380001; 🇮🇹 Udine, Italy

Investigational Site Number : 250001; 🇫🇷 BRON Cedex, France

Investigational Site Number : 250002; 🇫🇷 Marseille Cedex 05, France

Investigational Site Number : 250005; 🇫🇷 Paris Cedex 12, France

Investigational Site Number : 250003; 🇫🇷 Paris, France

Investigational Site Number : 250006; 🇫🇷 Vandœuvre-lès-nancy, France

Investigational Site Number : 151368; 🇩🇪 Hamburg, Germany

Investigational Site Number : 150031; 🇩🇪 Mainz, Germany

Investigational Site Number : 344001; 🇭🇰 Kowloon, Hong Kong

Investigational Site Number : 3480005; 🇭🇺 Pécs, 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 : 154161; 🇮🇪 Dublin, Ireland

Investigational Site Number : 380002; 🇮🇹 Ancona, Italy

Investigational Site Number : 380004; 🇮🇹 Cusano Milanino, Italy

Investigational Site Number : 380009; 🇮🇹 Firenze, Italy

Investigational Site Number : 380003; 🇮🇹 Genova, Italy

Investigational Site Number : 380007; 🇮🇹 Milano, Italy

Investigational Site Number : 380005; 🇮🇹 Napoli, Italy

Instituto de Genética e Erros Inatos do Metabolismo - IGEIM- Site Number : 076002; 🇧🇷 São Paulo, Brazil

Phoenix Children's Hospital- Site Number : 840003; 🇺🇸 Phoenix, Arizona, United States

Arkansas Childrens Hospital- Site Number : 840109; 🇺🇸 Little Rock, Arkansas, 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

Children's Hospital Oakland- Site Number : 840029; 🇺🇸 Oakland, California, United States

Children's Hospital of Orange County- Site Number : 840074; 🇺🇸 Orange, California, United States

Stanford Unviersity MC Dept of Genetics- Site Number : 840022; 🇺🇸 Palo Alto, California, United States

UC Davis MIND Institute- Site Number : 840010; 🇺🇸 Sacramento, California, United States

Loma Linda University Children's Hospital- Site Number : 840117; 🇺🇸 San Bernardino, California, United States

University of California at San Diego- Site Number : 840007; 🇺🇸 San Diego, California, United States

University of California at San Francisco- Site Number : 840051; 🇺🇸 San Francisco, California, United States

Children's Hospital IMD Clinic- Site Number : 840069; 🇺🇸 Aurora, Colorado, United States

Yale - Site Number : 840047; 🇺🇸 New Haven, Connecticut, United States

Children's National Medical Center- Site Number : 840067; 🇺🇸 Washington, District of Columbia, 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

The Atwal Clinic- Site Number : 840112; 🇺🇸 Jacksonville, Florida, United States

University Of Miami SOM- Site Number : 840006; 🇺🇸 Miami, Florida, United States

Pediatric Endocrine Associates- Site Number : 840017; 🇺🇸 Tampa, Florida, United States

Emory University School of Medicine- Human Genetics- Site Number : 840060; 🇺🇸 Decatur, Georgia, United States

Ann and Robert Lurie Children's Hospital- Site Number : 840011; 🇺🇸 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 Louisville- Genetics- Site Number : 840030; 🇺🇸 Louisville, Kentucky, United States

Ochsner Medical Center - Site Number : 840120; 🇺🇸 New Orleans, Louisiana, 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- Site Number : 840093; 🇺🇸 Boston, Massachusetts, United States

Baystate Health- 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

Children's Health Care- Site Number : 840114; 🇺🇸 Minneapolis, Minnesota, United States

University of Minnesota Medical Center Pediatrics- Site Number : 840076; 🇺🇸 Minneapolis, Minnesota, United States

Washington University of St. Louis- Site Number : 840100; 🇺🇸 Saint Louis, Missouri, United States

University of Nebraska Medical Center- Pediatrics- Site Number : 840084; 🇺🇸 Omaha, Nebraska, United States

Children's Specialty Center of Nevada- Site Number : 840008; 🇺🇸 Las Vegas, Nevada, 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

St. Peter's University Hospital- Site Number : 840016; 🇺🇸 New Brunswick, New Jersey, United States

St. Joseph's Children's Hospital- Site Number : 840057; 🇺🇸 Paterson, New Jersey, United States

Northwell Health- Site Number : 840102; 🇺🇸 Manhasset, New York, United States

New York University School Of Medicine- Site Number : 840040; 🇺🇸 New York, New York, United States

Metropolitan Hospital Center- Site Number : 840110; 🇺🇸 New York, New York, United States

University of Rochester Medical Center SOM- Site Number : 840105; 🇺🇸 Rochester, New York, United States

Investigational Site Number : 380008; 🇮🇹 Padova, Italy

State University of New York- Site Number : 840052; 🇺🇸 Syracuse, New York, United States

Duke University Medical Center Genetics Dept- Site Number : 840037; 🇺🇸 Durham, North Carolina, United States

Cincinnati Children's Hospital-Genetics- Site Number : 840041; 🇺🇸 Cincinnati, Ohio, United States

UHCMC- Site Number : 840119; 🇺🇸 Cleveland, Ohio, United States

Nationwide Children's Hospital- Site Number : 840091; 🇺🇸 Columbus, Ohio, United States

LSD Data Registry Site LLC- Site Number : 840094; 🇺🇸 Dublin, Ohio, United States

Oregon Health and Science University- Site Number : 840080; 🇺🇸 Portland, Oregon, 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

University of Pittsburgh- Site Number : 840023; 🇺🇸 Pittsburgh, Pennsylvania, United States

Rhode Island Hospital Genetics- Site Number : 840053; 🇺🇸 Providence, Rhode Island, United States

Greenwood Genetcs- Site Number : 840055; 🇺🇸 Greenville, South Carolina, United States

Baylor Research Center- Site Number : 840058; 🇺🇸 Dallas, Texas, United States

University of Utah Health Hospital- Site Number : 840092; 🇺🇸 Salt Lake City, Utah, United States

University of Virginia School of Medicine-Pediatrics- Site Number : 840078; 🇺🇸 Charlottesville, Virginia, United States

Children's Hospital of the Kings 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

Multicare Health System ¿ Mary Bridge Childrens Hospital- Site Number : 840115; 🇺🇸 Tacoma, Washington, United States

Children's Hospital of Wisconsin-Pediatrics- Site Number : 840054; 🇺🇸 Milwaukee, Wisconsin, United States

Centro de Pesquisa Clínica Prédio 21, 2o andar Hospital de Clínicas de Porto Alegre- Site Number : 076001; 🇧🇷 Porto Alegre, Rio Grande Do Sul, Brazil

Hospital e Maternidade Celso Pierro - PUC-Campinas- Site Number : 076007; 🇧🇷 Campinas, São Paulo, Brazil

Instituto Fernandes Figueira - FIOCRUZ- Site Number : 076004; 🇧🇷 Rio de Janeiro, Brazil

University of Colorado at Denver Genetics Site Number : 840068; 🇺🇸 Aurora, Colorado, United States

Investigational Site Number : 153032; 🇦🇷 Ciudad Autónoma de Buenos Aires, Buenos Aires, Argentina

Investigational Site Number : 153130; 🇦🇷 Buenos Aires, Argentina

Investigational Site Number : 151069; 🇦🇺 Westmead, New South Wales, Australia

Investigational Site Number : 152096; 🇦🇺 Brisbane, Queensland, Australia

Investigational Site Number : 150944; 🇧🇪 Brussels, Belgium

Investigational Site Number : 150517; 🇧🇪 Brussels, Belgium

Investigational Site Number : 151668; 🇧🇪 Leuven, Belgium

Investigational Site Number : 154008; 🇧🇪 Roeselare, Belgium

Investigational Site Number : 124014; 🇨🇦 Calgary, Alberta, Canada

Investigational Site Number : 124002; 🇨🇦 Edmonton, Alberta, 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 : 124021; 🇨🇦 London, Ontario, Canada

Investigational Site Number : 124006; 🇨🇦 Toronto, Ontario, Canada

Investigational Site Number : 124018; 🇨🇦 Montreal, Quebec, Canada

Investigational Site Number : 124001; 🇨🇦 Sherbrooke, Quebec, Canada

Investigational Site Number : 152003; 🇨🇱 Los Angeles, Chile

Investigational Site Number : 152001; 🇨🇱 Talcahuano, Chile

Investigational Site Number : 170001; 🇨🇴 Bogotá, Colombia

Investigational Site Number : 1910001; 🇭🇷 Zagreb, Croatia

Investigational Site Number : 150659; 🇨🇿 Praha 2, Czechia

Investigational Site Number : 151229; 🇩🇰 København Ø, Denmark

Investigational Site Number : 818002; 🇪🇬 Cairo, Egypt

Investigational Site Number : 818001; 🇪🇬 Cairo, Egypt

Investigational Site Number : 250004; 🇫🇷 Bordeaux, France

Investigational Site Number : 151149; 🇰🇷 Seoul, Gyeonggi-do, Korea, Republic of

Investigational Site Number : 154286; 🇰🇷 Changwon-si, Gyeongsangnam-do, Korea, Republic of

Investigational Site Number : 153178; 🇰🇷 Yangsan-si, Gyeongsangnam-do, Korea, Republic of

Investigational Site Number : 154148; 🇰🇷 Yangsan, Gyeongsangnam-do, Korea, Republic of

Investigational Site Number : 153191; 🇰🇷 Daejeon, Korea, Republic of

Investigational Site Number : 153225; 🇰🇷 Jeonnam, Korea, Republic of

Investigational Site Number : 153180; 🇰🇷 Seoul, Korea, Republic of

Investigational Site Number : 153196; 🇰🇷 Seoul, Korea, Republic of

Investigational Site Number : 153192; 🇰🇷 Seoul, Korea, Republic of

Investigational Site Number : 153212; 🇰🇷 Seoul, Korea, Republic of

Investigational Site Number : 153222; 🇰🇷 Seoul, Korea, Republic of

Investigational Site Number : 154225; 🇰🇼 Kuwait, Kuwait

Investigational Site Number : 4220004; 🇱🇧 Beirut, Lebanon

Investigational Site Number : 458001; 🇲🇾 Jalan Pahang, Malaysia

Investigational Site Number : 458002; 🇲🇾 Kuala Lumpur, Malaysia

Investigational Site Number : 151171; 🇳🇱 Amsterdam, Netherlands

Investigational Site Number : 152029; 🇳🇱 Rotterdam, Netherlands

Investigational Site Number : 154264; 🇵🇰 Lahore, Pakistan

Investigational Site Number : 154266; 🇵🇰 Lahore, Pakistan

Investigational Site Number : 608001; 🇵🇭 Manila, Philippines

Investigational Site Number : 150575; 🇵🇱 Warszawa, Poland

Investigational Site Number : 6200002; 🇵🇹 Coimbra, Portugal

Investigational Site Number : 154177; 🇷🇴 Cluj Napoca, Romania

Investigational Site Number : 643001; 🇷🇺 Moscow, Russian Federation

Investigational Site Number : 643002; 🇷🇺 Moscow, Russian Federation

Investigational Site Number : 682004; 🇸🇦 Makkah, Saudi Arabia

Investigational Site Number : 682001; 🇸🇦 Qatif, Saudi Arabia

Investigational Site Number : 702001; 🇸🇬 Singapore, Singapore