Mucopolysaccharidoses (MPS) Clinical Trials

Find Mucopolysaccharidoses (MPS) Clinical Trials Near You

Mucopolysaccharidosis I (MPS I) Registry

Status: Completed
Location: See all (140) 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 Children's Hospital- Site Number : 840109
Little Rock
Arizona
Phoenix Children's Hospital- Site Number : 840003
Phoenix
California
Southern California Permanente Medical Group- Site Number : 840108
Los Angeles
USC Health Sciences Center Dept of Genetics- Site Number : 840082
Los Angeles
UCSF Benioff Children's Hospital - Oakland- Site Number : 840029
Oakland
Children's Hospital of Orange County- Site Number : 840074
Orange
Stanford Unviersity MC Dept of Genetics- Site Number : 840022
Palo Alto
UC Davis MIND Institute- Site Number : 840010
Sacramento
University of California at San Diego- Site Number : 840007
San Diego
University of California at San Francisco- Site Number : 840051
San Francisco
Colorado
Children's Hospital Colorado - Aurora- Site Number : 840069
Aurora
University of Colorado at Denver Genetics Site Number : 840068
Aurora
Connecticut
Yale University School of Medicine- Site Number : 840047
New Haven
Washington, D.c.
Children's National Medical Center - Washington- Site Number : 840067
Washington D.c.
Florida
University of Florida Dept of Genetics- Site Number : 840083
Gainesville
University Of Miami SOM- Site Number : 840006
Miami
Pediatric Endocrine Associates- Site Number : 840017
Tampa
Georgia
Emory University School of Medicine- Human Genetics- Site Number : 840060
Decatur
Iowa
University of Iowa- Site Number : 840032
Iowa City
Illinois
Ann & Robert H. Lurie Children's Hospital of Chicago- Site Number : 840011
Chicago
Rush University Medical Center Genetics- Site Number : 840079
Chicago
Indiana
Indianapolis University School of Medicine- Site Number : 840027
Indianapolis
Kentucky
University of Louisville- Genetics- Site Number : 840030
Louisville
Massachusetts
Boston Children's Hospital - PIN- Site Number : 840093
Boston
Massachusetts General Hospital-Genetics- Site Number : 840062
Boston
Baystate Medical Center- Site Number : 840002
Springfield
Maryland
John Hopkins- Site Number : 840044
Baltimore
Michigan
University of Michigan Pediatrics- Site Number : 840107
Ann Arbor
Minnesota
University of Minnesota Medical Center Pediatrics- Site Number : 840076
Minneapolis
Missouri
Washington University- Site Number : 840100
St Louis
North Carolina
Duke University Medical Center Genetics Dept- Site Number : 840037
Durham
Nebraska
University of Nebraska Medical Center- Site Number : 840084
Omaha
New Jersey
Joseph M. Sanzari Children's Hospital- Site Number : 840101
Hackensack
Saint Peter's University Hospital - New Brunswick- Site Number : 840016
New Brunswick
Nevada
Children's Specialty Center - Las Vegas- Site Number : 840008
Las Vegas
New York
Northwell Health - Site Number : 840102
Manhasset
New York University School Of Medicine- Site Number : 840040
New York
State University of New York- Site Number : 840052
Syracuse
Ohio
Cincinnati Children's Hospital-Genetics- Site Number : 840041
Cincinnati
Nationwide Children's Hospital- Site Number : 840091
Columbus
Oregon
Oregon Health & Science University (OHSU)- Site Number : 840080
Portland
Pennsylvania
Children's Hospital of Philadelphia HUP Medical Genetics- Site Number : 840089
Philadelphia
Children's Hospital of Philadelphia- Site Number : 840034
Philadelphia
University of Pittsburgh- Site Number : 840023
Pittsburgh
Rhode Island
Rhode Island Hospital Genetics- Site Number : 840053
Providence
South Carolina
Greenwood Genetic Center - Greenville- Site Number : 840055
Greenville
Texas
Baylor Institute of Metabolic Disease- Site Number : 840058
Dallas
Utah
University of Utah Health Hospital- Site Number : 840092
Salt Lake City
Virginia
University of Virginia School of Medicine-Pediatrics- Site Number : 840078
Charlottesville
Children's Hospital of The King's Daughters- Site Number : 840072
Norfolk
Washington
Seattle Children's Hospital- Site Number : 840028
Seattle
University Of Washington Medical Center- Site Number : 840059
Seattle
Wisconsin
Children's Hospital of Wisconsin-Pediatrics- Site Number : 840054
Milwaukee
Other Locations
Argentina
Investigational Site Number : 153032
Buenos Aires
Investigational Site Number : 153130
San Luis
Australia
Investigational Site Number : 151069
Westmead
Belgium
Investigational Site Number : 150944
Brussels
Investigational Site Number : 151668
Leuven
Investigational Site Number : 154008
Roeselare
Brazil
Hospital e Maternidade Celso Pierro - PUC-Campinas- Site Number : 076007
Campinas
Private Practice - Dr. Fabiano Klaus- Site Number : 076001
Porto Alegre
Instituto Fernandes Figueira - FIOCRUZ- Site Number : 076004
Rio De Janeiro
Instituto de Genética e Erros Inatos do Metabolismo- Site Number : 076002
São Paulo
Canada
Investigational Site Number : 124014
Calgary
Investigational Site Number : 124002
Edmonton
Investigational Site Number : 124021
London
Investigational Site Number : 124018
Ottawa
Investigational Site Number : 124001
Sherbrooke
Investigational Site Number : 124006
Toronto
Investigational Site Number : 124011
Vancouver
Investigational Site Number : 124019
Winnipeg
Chile
Investigational Site Number : 152003
Los Ángeles
Investigational Site Number : 152001
Talcahuano
Colombia
Investigational Site Number : 170001
Bogotá
Denmark
Investigational Site Number : 151229
Copenhagen
Egypt
Investigational Site Number : 818001
Cairo
Investigational Site Number : 818002
Cairo
France
Investigational Site Number : 250004
Bordeaux
Investigational Site Number : 250001
Bron
Investigational Site Number : 250002
Marseille
Investigational Site Number : 250003
Paris
Investigational Site Number : 250006
Vandœuvre-lès-nancy
Germany
Investigational Site Number : 151368
Hamburg
Investigational Site Number : 150031
Mainz
Hong Kong Special Administrative Region
Investigational Site Number : 344001
Kowloon
India
Investigational Site Number : 356003
Ahmedabad
Investigational Site Number : 356001
Kolkata
Investigational Site Number : 356002
Vellore
Indonesia
Investigational Site Number : 360001
Jakarta
Ireland
Investigational Site Number : 154161
Dublin
Italy
Investigational Site Number : 380002
Ancona
Investigational Site Number : 380004
Cusano Milanino
Investigational Site Number : 380003
Genova
Investigational Site Number : 380007
Milan
Investigational Site Number : 380005
Naples
Investigational Site Number : 380008
Padova
Investigational Site Number : 380006
Rome
Japan
Investigational Site Number : 392102
Nankoku
Investigational Site Number : 392101
Tokyo
Kuwait
Investigational Site Number : 154225
Kuwait City
Lebanon
Investigational Site Number : 4220004
Beirut
Malaysia
Investigational Site Number : 458001
Kuala Lumpur
Investigational Site Number : 458002
Kuala Lumpur
Netherlands
Investigational Site Number : 151171
Amsterdam
Investigational Site Number : 152029
Rotterdam
Pakistan
Investigational Site Number : 154264
Lahore
Investigational Site Number : 154266
Lahore
Philippines
Investigational Site Number : 608001
Manila
Poland
Investigational Site Number : 150575
Warsaw
Portugal
Investigational Site Number : 6200002
Coimbra
Republic of Korea
Investigational Site Number : 153191
Daejeon
Investigational Site Number : 153225
Jeonnam
Investigational Site Number : 153212
Seoul
Investigational Site Number : 153222
Seoul
Romania
Investigational Site Number : 154177
Cluj-napoca
Russian Federation
Investigational Site Number : 643001
Moscow
Investigational Site Number : 643002
Moscow
Saudi Arabia
Investigational Site Number : 682001
Al Qaţīf
Investigational Site Number : 682004
Mecca
Slovakia
Investigational Site Number : 703001
Bratislava
Sweden
Investigational Site Number : 7520001
Stockholm
Taiwan
Investigational Site Number : 154093
Chiayi City
Investigational Site Number : 152246
Kaohsiung City
Investigational Site Number : 151500
Putz City, Chia-yi
Investigational Site Number : 151179
Taichung
Investigational Site Number : 152153
Tainan
Investigational Site Number : 151182
Taipei
Investigational Site Number : 152321
Taipei
Thailand
Investigational Site Number : 764001
Bangkok
Investigational Site Number : 764002
Bangkok
Investigational Site Number : 764003
Bangkok
Investigational Site Number : 764004
Bangkok
United Kingdom
Investigational Site Number : 154240
Birmingham
Investigational Site Number : 152156
Glasgow
Investigational Site Number : 150303
London
Investigational Site Number : 151123
London
Investigational Site Number : 151646
London
Investigational Site Number : 150455
Manchester
Investigational Site Number : 152361
Salford
Viet Nam
Investigational Site Number : 154212
Hanoi
Time Frame
Start Date: 2003-11-20
Completion Date: 2024-12-31
Participants
Target number of participants: 1406
Sponsors
Leads: Genzyme, a Sanofi Company

This content was sourced from clinicaltrials.gov