Clinical trial NCT00144794: Mucopolysaccharidosis I (MPS I) Registry
Official title: Mucopolysaccharidosis I (MPS I) Registry
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Mucopolysaccharidosis I (MPS I)
Keywords: Hurler's Syndrome
Hurler-Scheie Syndrome
Scheie Syndrome
Lead sponsor: Sanofi
Sponsor class: INDUSTRY
First posted: 2005-09-05
Enrollment: 1500
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: OTHER
Sampling method: PROBABILITY_SAMPLE
Study population: All Patients with MPS I

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

Eligibility criteria: 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.

Primary outcomes:
measure: To evaluate the long-term effectiveness of Aldurazyme
timeFrame: Approximately 17 Years
Secondary outcomes:
Other outcomes:
Locations:
city: Phoenix
country: United States
facility: Phoenix Children's Hospital- Site Number : 840003
state: Arizona
city: Little Rock
country: United States
facility: Arkansas Childrens Hospital- Site Number : 840109
state: Arkansas
city: Los Angeles
country: United States
facility: Southern California Permanente Medical Group- Site Number : 840108
state: California
city: Los Angeles
country: United States
facility: USC Health Sciences Center Dept of Genetics- Site Number : 840082
state: California
city: Oakland
country: United States
facility: Children's Hospital Oakland- Site Number : 840029
state: California
city: Orange
country: United States
facility: Children's Hospital of Orange County- Site Number : 840074
state: California
city: Palo Alto
country: United States
facility: Stanford Unviersity MC Dept of Genetics- Site Number : 840022
state: California
city: Sacramento
country: United States
facility: UC Davis MIND Institute- Site Number : 840010
state: California
city: San Bernardino
country: United States
facility: Loma Linda University Children's Hospital- Site Number : 840117
state: California
city: San Diego
country: United States
facility: University of California at San Diego- Site Number : 840007
state: California
city: San Francisco
country: United States
facility: University of California at San Francisco- Site Number : 840051
state: California
city: Aurora
country: United States
facility: Children's Hospital IMD Clinic- Site Number : 840069
state: Colorado
city: Aurora
country: United States
facility: University of Colorado at Denver Genetics Site Number : 840068
state: Colorado
city: New Haven
country: United States
facility: Yale - Site Number : 840047
state: Connecticut
city: Washington
country: United States
facility: Children's National Medical Center- Site Number : 840067
state: District of Columbia
city: Gainesville
country: United States
facility: University of Florida Dept of Genetics- Site Number : 840083
state: Florida
city: Jacksonville
country: United States
facility: University of Florida Pediatrics Genetics- Site Number : 840121
state: Florida
city: Jacksonville
country: United States
facility: The Atwal Clinic- Site Number : 840112
state: Florida
city: Miami
country: United States
facility: University Of Miami SOM- Site Number : 840006
state: Florida
city: Tampa
country: United States
facility: Pediatric Endocrine Associates- Site Number : 840017
state: Florida
city: Decatur
country: United States
facility: Emory University School of Medicine- Human Genetics- Site Number : 840060
state: Georgia
city: Chicago
country: United States
facility: Ann and Robert Lurie Children's Hospital- Site Number : 840011
state: Illinois
city: Chicago
country: United States
facility: Rush University Medical Center Genetics- Site Number : 840079
state: Illinois
city: Indianapolis
country: United States
facility: Indianapolis University School of Medicine- Site Number : 840027
state: Indiana
city: Iowa City
country: United States
facility: University of Iowa- Site Number : 840032
state: Iowa
city: Louisville
country: United States
facility: University of Louisville- Genetics- Site Number : 840030
state: Kentucky
city: New Orleans
country: United States
facility: Ochsner Medical Center - Site Number : 840120
state: Louisiana
city: Baltimore
country: United States
facility: John Hopkins- Site Number : 840044
state: Maryland
city: Boston
country: United States
facility: Massachusetts General Hospital-Genetics- Site Number : 840062
state: Massachusetts
city: Boston
country: United States
facility: Boston Children's Hospital- Site Number : 840093
state: Massachusetts
city: Springfield
country: United States
facility: Baystate Health- Site Number : 840002
state: Massachusetts
city: Ann Arbor
country: United States
facility: University of Michigan Pediatrics- Site Number : 840107
state: Michigan
city: Detroit
country: United States
facility: Children's Hospital of Michigan- Site Number : 840066
state: Michigan
city: Minneapolis
country: United States
facility: Children's Health Care- Site Number : 840114
state: Minnesota
city: Minneapolis
country: United States
facility: University of Minnesota Medical Center Pediatrics- Site Number : 840076
state: Minnesota
city: Saint Louis
country: United States
facility: Washington University of St. Louis- Site Number : 840100
state: Missouri
city: Omaha
country: United States
facility: University of Nebraska Medical Center- Pediatrics- Site Number : 840084
state: Nebraska
city: Las Vegas
country: United States
facility: Children's Specialty Center of Nevada- Site Number : 840008
state: Nevada
city: Hackensack
country: United States
facility: Joseph M. Sanzari Children's Hospital- Site Number : 840101
state: New Jersey
city: Morristown
country: United States
facility: Atlantic Health System- Site Number : 840099
state: New Jersey
city: New Brunswick
country: United States
facility: St. Peter's University Hospital- Site Number : 840016
state: New Jersey
city: Paterson
country: United States
facility: St. Joseph's Children's Hospital- Site Number : 840057
state: New Jersey
city: Manhasset
country: United States
facility: Northwell Health- Site Number : 840102
state: New York
city: New York
country: United States
facility: New York University School Of Medicine- Site Number : 840040
state: New York
city: New York
country: United States
facility: Metropolitan Hospital Center- Site Number : 840110
state: New York
city: Rochester
country: United States
facility: University of Rochester Medical Center SOM- Site Number : 840105
state: New York
city: Syracuse
country: United States
facility: State University of New York- Site Number : 840052
state: New York
city: Durham
country: United States
facility: Duke University Medical Center Genetics Dept- Site Number : 840037
state: North Carolina
city: Cincinnati
country: United States
facility: Cincinnati Children's Hospital-Genetics- Site Number : 840041
state: Ohio
city: Cleveland
country: United States
facility: UHCMC- Site Number : 840119
state: Ohio
city: Columbus
country: United States
facility: Nationwide Children's Hospital- Site Number : 840091
state: Ohio
city: Dublin
country: United States
facility: LSD Data Registry Site LLC- Site Number : 840094
state: Ohio
city: Portland
country: United States
facility: Oregon Health and Science University- Site Number : 840080
state: Oregon
city: Philadelphia
country: United States
facility: Children's Hospital of Philadelphia HUP Medical Genetics- Site Number : 840089
state: Pennsylvania
city: Philadelphia
country: United States
facility: Children's Hospital of Philadelphia- Site Number : 840034
state: Pennsylvania
city: Pittsburgh
country: United States
facility: University of Pittsburgh- Site Number : 840023
state: Pennsylvania
city: Providence
country: United States
facility: Rhode Island Hospital Genetics- Site Number : 840053
state: Rhode Island
city: Greenville
country: United States
facility: Greenwood Genetcs- Site Number : 840055
state: South Carolina
city: Dallas
country: United States
facility: Baylor Research Center- Site Number : 840058
state: Texas
city: Salt Lake City
country: United States
facility: University of Utah Health Hospital- Site Number : 840092
state: Utah
city: Charlottesville
country: United States
facility: University of Virginia School of Medicine-Pediatrics- Site Number : 840078
state: Virginia
city: Norfolk
country: United States
facility: Children's Hospital of the Kings Daughters- Site Number : 840072
state: Virginia
city: Seattle
country: United States
facility: Seattle Children's Hospital- Site Number : 840028
state: Washington
city: Seattle
country: United States
facility: University Of Washington Medical Center- Site Number : 840059
state: Washington
city: Tacoma
country: United States
facility: Multicare Health System ¿ Mary Bridge Childrens Hospital- Site Number : 840115
state: Washington
city: Milwaukee
country: United States
facility: Children's Hospital of Wisconsin-Pediatrics- Site Number : 840054
state: Wisconsin
city: Ciudad Autónoma de Buenos Aires
country: Argentina
facility: Investigational Site Number : 153032
state: Buenos Aires
city: Buenos Aires
country: Argentina
facility: Investigational Site Number : 153130
city: Westmead
country: Australia
facility: Investigational Site Number : 151069
state: New South Wales
city: Brisbane
country: Australia
facility: Investigational Site Number : 152096
state: Queensland
city: Brussels
country: Belgium
facility: Investigational Site Number : 150944
city: Brussels
country: Belgium
facility: Investigational Site Number : 150517
city: Leuven
country: Belgium
facility: Investigational Site Number : 151668
city: Roeselare
country: Belgium
facility: Investigational Site Number : 154008
city: Porto Alegre
country: Brazil
facility: Centro de Pesquisa Clínica Prédio 21, 2o andar Hospital de Clínicas de Porto Alegre- Site Number : 076001
state: Rio Grande Do Sul
city: Campinas
country: Brazil
facility: Hospital e Maternidade Celso Pierro - PUC-Campinas- Site Number : 076007
state: São Paulo
city: Rio de Janeiro
country: Brazil
facility: Instituto Fernandes Figueira - FIOCRUZ- Site Number : 076004
city: São Paulo
country: Brazil
facility: Instituto de Genética e Erros Inatos do Metabolismo - IGEIM- Site Number : 076002
city: Calgary
country: Canada
facility: Investigational Site Number : 124014
state: Alberta
city: Edmonton
country: Canada
facility: Investigational Site Number : 124002
state: Alberta
city: Vancouver
country: Canada
facility: Investigational Site Number : 124011
state: British Columbia
city: Winnipeg
country: Canada
facility: Investigational Site Number : 124019
state: Manitoba
city: Moncton
country: Canada
facility: Investigational Site Number : 124022
state: New Brunswick
city: London
country: Canada
facility: Investigational Site Number : 124021
state: Ontario
city: Toronto
country: Canada
facility: Investigational Site Number : 124006
state: Ontario
city: Montreal
country: Canada
facility: Investigational Site Number : 124018
state: Quebec
city: Sherbrooke
country: Canada
facility: Investigational Site Number : 124001
state: Quebec
city: Los Angeles
country: Chile
facility: Investigational Site Number : 152003
city: Talcahuano
country: Chile
facility: Investigational Site Number : 152001
city: Bogotá
country: Colombia
facility: Investigational Site Number : 170001
city: Zagreb
country: Croatia
facility: Investigational Site Number : 1910001
city: Praha 2
country: Czechia
facility: Investigational Site Number : 150659
city: København Ø
country: Denmark
facility: Investigational Site Number : 151229
city: Cairo
country: Egypt
facility: Investigational Site Number : 818002
city: Cairo
country: Egypt
facility: Investigational Site Number : 818001
city: Bordeaux
country: France
facility: Investigational Site Number : 250004
city: BRON Cedex
country: France
facility: Investigational Site Number : 250001
city: Marseille Cedex 05
country: France
facility: Investigational Site Number : 250002
city: Paris Cedex 12
country: France
facility: Investigational Site Number : 250005
city: Paris
country: France
facility: Investigational Site Number : 250003
city: Vandœuvre-lès-nancy
country: France
facility: Investigational Site Number : 250006
city: Hamburg
country: Germany
facility: Investigational Site Number : 151368
city: Mainz
country: Germany
facility: Investigational Site Number : 150031
city: Kowloon
country: Hong Kong
facility: Investigational Site Number : 344001
city: Pécs
country: Hungary
facility: Investigational Site Number : 3480005
city: Ahmedabad
country: India
facility: Investigational Site Number : 356003
city: Kolkata
country: India
facility: Investigational Site Number : 356001
city: Secunderabad
country: India
facility: Investigational Site Number : 3560004
city: Vellore
country: India
facility: Investigational Site Number : 356002
city: Jakarta
country: Indonesia
facility: Investigational Site Number : 360001
city: Dublin
country: Ireland
facility: Investigational Site Number : 154161
city: Ancona
country: Italy
facility: Investigational Site Number : 380002
city: Cusano Milanino
country: Italy
facility: Investigational Site Number : 380004
city: Firenze
country: Italy
facility: Investigational Site Number : 380009
city: Genova
country: Italy
facility: Investigational Site Number : 380003
city: Milano
country: Italy
facility: Investigational Site Number : 380007
city: Napoli
country: Italy
facility: Investigational Site Number : 380005
city: Padova
country: Italy
facility: Investigational Site Number : 380008
city: Roma
country: Italy
facility: Investigational Site Number : 380006
city: Udine
country: Italy
facility: Investigational Site Number : 380001
city: Nangoku-shi
country: Japan
facility: Investigational Site Number : 392102
state: Kochi
city: Tokyo
country: Japan
facility: Investigational Site Number : 392101
city: Seoul
country: Korea, Republic of
facility: Investigational Site Number : 151149
state: Gyeonggi-do
city: Changwon-si
country: Korea, Republic of
facility: Investigational Site Number : 154286
state: Gyeongsangnam-do
city: Yangsan-si
country: Korea, Republic of
facility: Investigational Site Number : 153178
state: Gyeongsangnam-do
city: Yangsan
country: Korea, Republic of
facility: Investigational Site Number : 154148
state: Gyeongsangnam-do
city: Daejeon
country: Korea, Republic of
facility: Investigational Site Number : 153191
city: Jeonnam
country: Korea, Republic of
facility: Investigational Site Number : 153225
city: Seoul
country: Korea, Republic of
facility: Investigational Site Number : 153180
city: Seoul
country: Korea, Republic of
facility: Investigational Site Number : 153196
city: Seoul
country: Korea, Republic of
facility: Investigational Site Number : 153192
city: Seoul
country: Korea, Republic of
facility: Investigational Site Number : 153212
city: Seoul
country: Korea, Republic of
facility: Investigational Site Number : 153222
city: Kuwait
country: Kuwait
facility: Investigational Site Number : 154225
city: Beirut
country: Lebanon
facility: Investigational Site Number : 4220004
city: Jalan Pahang
country: Malaysia
facility: Investigational Site Number : 458001
city: Kuala Lumpur
country: Malaysia
facility: Investigational Site Number : 458002
city: Amsterdam
country: Netherlands
facility: Investigational Site Number : 151171
city: Rotterdam
country: Netherlands
facility: Investigational Site Number : 152029
city: Lahore
country: Pakistan
facility: Investigational Site Number : 154264
city: Lahore
country: Pakistan
facility: Investigational Site Number : 154266
city: Manila
country: Philippines
facility: Investigational Site Number : 608001
city: Warszawa
country: Poland
facility: Investigational Site Number : 150575
city: Coimbra
country: Portugal
facility: Investigational Site Number : 6200002
city: Cluj Napoca
country: Romania
facility: Investigational Site Number : 154177
city: Moscow
country: Russian Federation
facility: Investigational Site Number : 643001
city: Moscow
country: Russian Federation
facility: Investigational Site Number : 643002
city: Makkah
country: Saudi Arabia
facility: Investigational Site Number : 682004
city: Qatif
country: Saudi Arabia
facility: Investigational Site Number : 682001
city: Singapore
country: Singapore
facility: Investigational Site Number : 702001
city: Bratislava
country: Slovakia
facility: Investigational Site Number : 703001
city: Stockholm
country: Sweden
facility: Investigational Site Number : 7520001
city: Chiayi
country: Taiwan
facility: Investigational Site Number : 154093
city: Hualien
country: Taiwan
facility: Investigational Site Number : 152152
city: Kaohsiung City
country: Taiwan
facility: Investigational Site Number : 152246
city: Kaohsiung
country: Taiwan
facility: Investigational Site Number : 152244
city: New Taipei City
country: Taiwan
facility: Investigational Site Number : 151534
city: Putz City, Chia-yi
country: Taiwan
facility: Investigational Site Number : 151500
city: Taichung
country: Taiwan
facility: Investigational Site Number : 151181
city: Taichung
country: Taiwan
facility: Investigational Site Number : 151179
city: Tainan City
country: Taiwan
facility: Investigational Site Number : 153248
city: Tainan City
country: Taiwan
facility: Investigational Site Number : 152153
city: Taipei City
country: Taiwan
facility: Investigational Site Number : 151182
city: Taipei
country: Taiwan
facility: Investigational Site Number : 152321
city: Taoyuan City
country: Taiwan
facility: Investigational Site Number : 153158
city: Bangkok
country: Thailand
facility: Investigational Site Number : 764002
city: Bangkok
country: Thailand
facility: Investigational Site Number : 764003
city: Bangkok
country: Thailand
facility: Investigational Site Number : 764004
city: Bangkok
country: Thailand
facility: Investigational Site Number : 764001
city: Khon Kaen
country: Thailand
facility: Investigational Site Number : 764005
city: Birmingham
country: United Kingdom
facility: Investigational Site Number : 152287
city: Birmingham
country: United Kingdom
facility: Investigational Site Number : 154240
city: Glasgow
country: United Kingdom
facility: Investigational Site Number : 152156
city: London
country: United Kingdom
facility: Investigational Site Number : 151646
city: London
country: United Kingdom
facility: Investigational Site Number : 151123
city: London
country: United Kingdom
facility: Investigational Site Number : 150303
city: Manchester
country: United Kingdom
facility: Investigational Site Number : 150455
city: Salford
country: United Kingdom
facility: Investigational Site Number : 152361
city: Hanoi
country: Vietnam
facility: Investigational Site Number : 154212
city: Ho Chi Minh City
country: Vietnam
facility: Investigational Site Number : 704002
Study officials:
affiliation: Genzyme, a Sanofi Company
name: Medical Monitor
role: STUDY_DIRECTOR
References:
citation: Viskochil D, Muenzer J, Guffon N, Garin C, Munoz-Rojas MV, Moy KA, Hutchinson DT. Carpal tunnel syndrome in mucopolysaccharidosis I: a registry-based cohort study. Dev Med Child Neurol. 2017 Dec;59(12):1269-1275. doi: 10.1111/dmcn.13545. Epub 2017 Sep 11.
citation: Arn P, Bruce IA, Wraith JE, Travers H, Fallet S. Airway-related symptoms and surgeries in patients with mucopolysaccharidosis I. Ann Otol Rhinol Laryngol. 2015 Mar;124(3):198-205. doi: 10.1177/0003489414550154. Epub 2014 Sep 11.
citation: Beck M, Arn P, Giugliani R, Muenzer J, Okuyama T, Taylor J, Fallet S. The natural history of MPS I: global perspectives from the MPS I Registry. Genet Med. 2014 Oct;16(10):759-65. doi: 10.1038/gim.2014.25. Epub 2014 Mar 27.