Mucopolysaccharidosis I (MPS I) Registry

Status: Recruiting
Location: See all (179) locations...
Study Type: Observational
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

Eligibility
Participation Requirements
Sex: All
Healthy Volunteers: f
View:

• 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

Locations
United States
Arkansas
Arkansas Childrens Hospital- Site Number : 840109
RECRUITING
Little Rock
Arizona
Phoenix Children's Hospital- Site Number : 840003
RECRUITING
Phoenix
California
Southern California Permanente Medical Group- Site Number : 840108
RECRUITING
Los Angeles
USC Health Sciences Center Dept of Genetics- Site Number : 840082
RECRUITING
Los Angeles
Children's Hospital Oakland- Site Number : 840029
COMPLETED
Oakland
Children's Hospital of Orange County- Site Number : 840074
RECRUITING
Orange
Stanford Unviersity MC Dept of Genetics- Site Number : 840022
RECRUITING
Palo Alto
UC Davis MIND Institute- Site Number : 840010
RECRUITING
Sacramento
Loma Linda University Children's Hospital- Site Number : 840117
RECRUITING
San Bernardino
University of California at San Diego- Site Number : 840007
COMPLETED
San Diego
University of California at San Francisco- Site Number : 840051
RECRUITING
San Francisco
Colorado
Children's Hospital IMD Clinic- Site Number : 840069
COMPLETED
Aurora
University of Colorado at Denver Genetics Site Number : 840068
RECRUITING
Aurora
Connecticut
Yale - Site Number : 840047
RECRUITING
New Haven
Washington, D.c.
Children's National Medical Center- Site Number : 840067
RECRUITING
Washington D.c.
Florida
University of Florida Dept of Genetics- Site Number : 840083
RECRUITING
Gainesville
The Atwal Clinic- Site Number : 840112
RECRUITING
Jacksonville
University of Florida Pediatrics Genetics- Site Number : 840121
RECRUITING
Jacksonville
University Of Miami SOM- Site Number : 840006
RECRUITING
Miami
Pediatric Endocrine Associates- Site Number : 840017
RECRUITING
Tampa
Georgia
Emory University School of Medicine- Human Genetics- Site Number : 840060
RECRUITING
Decatur
Iowa
University of Iowa- Site Number : 840032
RECRUITING
Iowa City
Illinois
Ann and Robert Lurie Children's Hospital- Site Number : 840011
RECRUITING
Chicago
Rush University Medical Center Genetics- Site Number : 840079
RECRUITING
Chicago
Indiana
Indianapolis University School of Medicine- Site Number : 840027
RECRUITING
Indianapolis
Kentucky
University of Louisville- Genetics- Site Number : 840030
RECRUITING
Louisville
Louisiana
Ochsner Medical Center - Site Number : 840120
RECRUITING
New Orleans
Massachusetts
Boston Children's Hospital- Site Number : 840093
RECRUITING
Boston
Massachusetts General Hospital-Genetics- Site Number : 840062
RECRUITING
Boston
Baystate Health- Site Number : 840002
RECRUITING
Springfield
Maryland
John Hopkins- Site Number : 840044
RECRUITING
Baltimore
Michigan
University of Michigan Pediatrics- Site Number : 840107
RECRUITING
Ann Arbor
Children's Hospital of Michigan- Site Number : 840066
RECRUITING
Detroit
Minnesota
Children's Health Care- Site Number : 840114
RECRUITING
Minneapolis
University of Minnesota Medical Center Pediatrics- Site Number : 840076
RECRUITING
Minneapolis
Missouri
Washington University of St. Louis- Site Number : 840100
RECRUITING
St Louis
North Carolina
Duke University Medical Center Genetics Dept- Site Number : 840037
RECRUITING
Durham
Nebraska
University of Nebraska Medical Center- Pediatrics- Site Number : 840084
RECRUITING
Omaha
New Jersey
Joseph M. Sanzari Children's Hospital- Site Number : 840101
RECRUITING
Hackensack
Atlantic Health System- Site Number : 840099
RECRUITING
Morristown
St. Peter's University Hospital- Site Number : 840016
RECRUITING
New Brunswick
St. Joseph's Children's Hospital- Site Number : 840057
RECRUITING
Paterson
Nevada
Children's Specialty Center of Nevada- Site Number : 840008
RECRUITING
Las Vegas
New York
Northwell Health- Site Number : 840102
RECRUITING
Manhasset
Metropolitan Hospital Center- Site Number : 840110
RECRUITING
New York
New York University School Of Medicine- Site Number : 840040
RECRUITING
New York
University of Rochester Medical Center SOM- Site Number : 840105
RECRUITING
Rochester
State University of New York- Site Number : 840052
RECRUITING
Syracuse
Ohio
Cincinnati Children's Hospital-Genetics- Site Number : 840041
RECRUITING
Cincinnati
UHCMC- Site Number : 840119
RECRUITING
Cleveland
Nationwide Children's Hospital- Site Number : 840091
RECRUITING
Columbus
LSD Data Registry Site LLC- Site Number : 840094
RECRUITING
Dublin
Oregon
Oregon Health and Science University- Site Number : 840080
RECRUITING
Portland
Pennsylvania
Children's Hospital of Philadelphia HUP Medical Genetics- Site Number : 840089
RECRUITING
Philadelphia
Children's Hospital of Philadelphia- Site Number : 840034
RECRUITING
Philadelphia
University of Pittsburgh- Site Number : 840023
RECRUITING
Pittsburgh
Rhode Island
Rhode Island Hospital Genetics- Site Number : 840053
RECRUITING
Providence
South Carolina
Greenwood Genetcs- Site Number : 840055
RECRUITING
Greenville
Texas
Baylor Research Center- Site Number : 840058
RECRUITING
Dallas
Utah
University of Utah Health Hospital- Site Number : 840092
RECRUITING
Salt Lake City
Virginia
University of Virginia School of Medicine-Pediatrics- Site Number : 840078
RECRUITING
Charlottesville
Children's Hospital of the Kings Daughters- Site Number : 840072
ACTIVE_NOT_RECRUITING
Norfolk
Washington
Seattle Children's Hospital- Site Number : 840028
RECRUITING
Seattle
University Of Washington Medical Center- Site Number : 840059
RECRUITING
Seattle
Multicare Health System ¿ Mary Bridge Childrens Hospital- Site Number : 840115
RECRUITING
Tacoma
Wisconsin
Children's Hospital of Wisconsin-Pediatrics- Site Number : 840054
RECRUITING
Milwaukee
Other Locations
Argentina
Investigational Site Number : 153130
RECRUITING
Buenos Aires
Investigational Site Number : 153032
RECRUITING
Ciudad Autónoma De Buenos Aires
Australia
Investigational Site Number : 152096
RECRUITING
Brisbane
Investigational Site Number : 151069
RECRUITING
Westmead
Belgium
Investigational Site Number : 150517
RECRUITING
Brussels
Investigational Site Number : 150944
RECRUITING
Brussels
Investigational Site Number : 151668
RECRUITING
Leuven
Investigational Site Number : 154008
RECRUITING
Roeselare
Brazil
Hospital e Maternidade Celso Pierro - PUC-Campinas- Site Number : 076007
RECRUITING
Campinas
Centro de Pesquisa Clínica Prédio 21, 2o andar Hospital de Clínicas de Porto Alegre- Site Number : 076001
RECRUITING
Porto Alegre
Instituto Fernandes Figueira - FIOCRUZ- Site Number : 076004
RECRUITING
Rio De Janeiro
Instituto de Genética e Erros Inatos do Metabolismo - IGEIM- Site Number : 076002
RECRUITING
São Paulo
Canada
Investigational Site Number : 124014
RECRUITING
Calgary
Investigational Site Number : 124002
RECRUITING
Edmonton
Investigational Site Number : 124021
RECRUITING
London
Investigational Site Number : 124022
RECRUITING
Moncton
Investigational Site Number : 124018
RECRUITING
Montreal
Investigational Site Number : 124001
RECRUITING
Sherbrooke
Investigational Site Number : 124006
RECRUITING
Toronto
Investigational Site Number : 124011
RECRUITING
Vancouver
Investigational Site Number : 124019
RECRUITING
Winnipeg
Chile
Investigational Site Number : 152003
RECRUITING
Los Ángeles
Investigational Site Number : 152001
RECRUITING
Talcahuano
Colombia
Investigational Site Number : 170001
RECRUITING
Bogotá
Croatia
Investigational Site Number : 1910001
RECRUITING
Zagreb
Denmark
Investigational Site Number : 151229
RECRUITING
København Ø
Egypt
Investigational Site Number : 818001
RECRUITING
Cairo
Investigational Site Number : 818002
RECRUITING
Cairo
France
Investigational Site Number : 250004
RECRUITING
Bordeaux
Investigational Site Number : 250001
RECRUITING
Bron
Investigational Site Number : 250002
COMPLETED
Marseille
Investigational Site Number : 250003
RECRUITING
Paris
Investigational Site Number : 250005
RECRUITING
Paris
Investigational Site Number : 250006
COMPLETED
Vandœuvre-lès-nancy
Germany
Investigational Site Number : 151368
RECRUITING
Hamburg
Investigational Site Number : 150031
RECRUITING
Mainz
Hong Kong Special Administrative Region
Investigational Site Number : 344001
RECRUITING
Kowloon
Hungary
Investigational Site Number : 3480005
RECRUITING
Pécs
India
Investigational Site Number : 356003
RECRUITING
Ahmedabad
Investigational Site Number : 356001
RECRUITING
Kolkata
Investigational Site Number : 3560004
RECRUITING
Secunderabad
Investigational Site Number : 356002
RECRUITING
Vellore
Indonesia
Investigational Site Number : 360001
RECRUITING
Jakarta
Ireland
Investigational Site Number : 154161
RECRUITING
Dublin
Italy
Investigational Site Number : 380002
COMPLETED
Ancona
Investigational Site Number : 380004
RECRUITING
Cusano Milanino
Investigational Site Number : 380009
RECRUITING
Florence
Investigational Site Number : 380003
RECRUITING
Genova
Investigational Site Number : 380007
COMPLETED
Milan
Investigational Site Number : 380005
RECRUITING
Napoli
Investigational Site Number : 380008
RECRUITING
Padua
Investigational Site Number : 380006
RECRUITING
Roma
Investigational Site Number : 380001
RECRUITING
Udine
Japan
Investigational Site Number : 392102
RECRUITING
Nangoku-shi
Investigational Site Number : 392101
RECRUITING
Tokyo
Kuwait
Investigational Site Number : 154225
RECRUITING
Kuwait City
Lebanon
Investigational Site Number : 4220004
RECRUITING
Beirut
Malaysia
Investigational Site Number : 458001
RECRUITING
Jalan Pahang
Investigational Site Number : 458002
RECRUITING
Kuala Lumpur
Netherlands
Investigational Site Number : 151171
COMPLETED
Amsterdam
Investigational Site Number : 152029
RECRUITING
Rotterdam
Pakistan
Investigational Site Number : 154264
RECRUITING
Lahore
Investigational Site Number : 154266
RECRUITING
Lahore
Philippines
Investigational Site Number : 608001
RECRUITING
Manila
Poland
Investigational Site Number : 150575
RECRUITING
Warsaw
Portugal
Investigational Site Number : 6200002
RECRUITING
Coimbra
Republic of Korea
Investigational Site Number : 154286
RECRUITING
Changwon-si
Investigational Site Number : 153191
RECRUITING
Daejeon
Investigational Site Number : 153225
RECRUITING
Jeonnam
Investigational Site Number : 151149
RECRUITING
Seoul
Investigational Site Number : 153180
RECRUITING
Seoul
Investigational Site Number : 153192
RECRUITING
Seoul
Investigational Site Number : 153196
RECRUITING
Seoul
Investigational Site Number : 153212
RECRUITING
Seoul
Investigational Site Number : 153222
RECRUITING
Seoul
Investigational Site Number : 153178
RECRUITING
Yangsan
Investigational Site Number : 154148
RECRUITING
Yangsan
Romania
Investigational Site Number : 154177
RECRUITING
Cluj-napoca
Russian Federation
Investigational Site Number : 643001
RECRUITING
Moscow
Investigational Site Number : 643002
RECRUITING
Moscow
Saudi Arabia
Investigational Site Number : 682001
RECRUITING
Al Qaţīf
Investigational Site Number : 682004
RECRUITING
Makkah Al Mukarramah
Singapore
Investigational Site Number : 702001
RECRUITING
Singapore
Slovakia
Investigational Site Number : 703001
RECRUITING
Bratislava
Sweden
Investigational Site Number : 7520001
RECRUITING
Stockholm
Taiwan
Investigational Site Number : 154093
RECRUITING
Chiayi City
Investigational Site Number : 152152
RECRUITING
Hualien City
Investigational Site Number : 152244
RECRUITING
Kaohsiung City
Investigational Site Number : 152246
RECRUITING
Kaohsiung City
Investigational Site Number : 151534
RECRUITING
New Taipei City
Investigational Site Number : 151500
RECRUITING
Putz City, Chia-yi
Investigational Site Number : 151179
RECRUITING
Taichung
Investigational Site Number : 151181
RECRUITING
Taichung
Investigational Site Number : 152153
RECRUITING
Tainan City
Investigational Site Number : 153248
RECRUITING
Tainan City
Investigational Site Number : 151182
RECRUITING
Taipei
Investigational Site Number : 152321
RECRUITING
Taipei
Investigational Site Number : 153158
RECRUITING
Taoyuan
Thailand
Investigational Site Number : 764001
RECRUITING
Bangkok
Investigational Site Number : 764002
COMPLETED
Bangkok
Investigational Site Number : 764003
RECRUITING
Bangkok
Investigational Site Number : 764004
RECRUITING
Bangkok
Investigational Site Number : 764005
RECRUITING
Khon Kaen
United Kingdom
Investigational Site Number : 152287
RECRUITING
Birmingham
Investigational Site Number : 154240
RECRUITING
Birmingham
Investigational Site Number : 152156
RECRUITING
Glasgow
Investigational Site Number : 150303
RECRUITING
London
Investigational Site Number : 151123
RECRUITING
London
Investigational Site Number : 151646
RECRUITING
London
Investigational Site Number : 150455
RECRUITING
Manchester
Investigational Site Number : 152361
RECRUITING
Salford
Viet Nam
Investigational Site Number : 154212
RECRUITING
Hanoi
Investigational Site Number : 704002
RECRUITING
Ho Chi Minh City
Contact Information
Primary
For site information, send an email with site number to
Contact-Us@sanofi.com
Backup
MPS I Registry HelpLine
help@MPSIRegistry.com
617-591-5500
Time Frame
Start Date: 2003-11-20
Estimated Completion Date: 2034-01-31
Participants
Target number of participants: 1500
Sponsors
Leads: Genzyme, a Sanofi Company

This content was sourced from clinicaltrials.gov