Clinical trial NCT00231400: Pompe Disease Registry Protocol
Official title: Pompe Disease Registry
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Glycogen Storage Disease Type II
Pompe Disease
Keywords: Glycogen Storage Disease Type II (GSD-II)
GSD-II
Pompe Disease
Pompe Disease (late-onset)
Acid Maltase Deficiency Disease
Glycogenosis II
Lead sponsor: Sanofi
Sponsor class: INDUSTRY
First posted: 2005-10-04
Enrollment: 2000
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: OTHER
Sampling method: NON_PROBABILITY_SAMPLE
Study population: All patients with a confirmed diagnosis of Pompe disease are eligible for inclusion in the Registry.

Brief summary: The Pompe Registry is a global, multicenter, international, longitudinal, observational, and voluntary program for patients with Pompe disease, designed to track the disease's natural history and outcomes in patients, both treated and not. Data from the Registry are also used to fulfill various global regulatory commitments, to support product development/reimbursement, and for other research and non-research related purposes.

The objectives of the Registry are:

* To enhance understanding of the variability, progression, identification, and natural history of Pompe disease, with the ultimate goal of better guiding and assessing therapeutic intervention.
* To assist the Pompe medical community with the development of recommendations for monitoring patients, and to provide reports on patient outcomes, to optimize patient care.
* To characterize the Pompe disease population.
* To evaluate the long-term effectiveness of alglucosidase alfa.

Detailed description: Study Design Time Perspective: Retrospective and Prospective

Eligibility criteria: Inclusion Criteria:

All patients with a confirmed diagnosis of Pompe disease who have signed the informed consent and authorization form(s) are eligible for inclusion. Confirmed diagnosis is defined as documented GAA enzyme deficiency from blood, skin, or muscle tissue and/or documentation of 2 GAA gene mutations.

Exclusion Criteria:

There are no exclusion criteria in this Registry

Primary outcomes:
measure: Understanding of the variability, progression , identification and natural history of the manifestations of Pompe disease
timeFrame: maximum 30 years
Secondary outcomes:
Other outcomes:
Locations:
city: Birmingham
country: United States
facility: University Alabama at Birmingham- Site Number : 840106
state: Alabama
city: Phoenix
country: United States
facility: Barrow Neurological Institute- Site Number : 840087
state: Arizona
city: Phoenix
country: United States
facility: Phoenix Children's Hospital- Site Number : 840003
state: Arizona
city: Tucson
country: United States
facility: University of Arizona- Site Number : 840015
state: Arizona
city: Little Rock
country: United States
facility: Arkansas Childrens Hospital- Site Number : 840109
state: Arkansas
city: Little Rock
country: United States
facility: University of Arkansas for Medical Sciences- Site Number : 840113
state: Arkansas
city: Irvine
country: United States
facility: University of California at Irvine- Site Number : 840036
state: California
city: Loma Linda
country: United States
facility: Loma Linda University- Genetics- Site Number : 840070
state: California
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: 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: Stanford
country: United States
facility: Stanford University Pediatrics- Site Number : 840021
state: California
city: Aurora
country: United States
facility: Children's Hospital Colorado - Aurora- Site Number : 840069
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 College of Medicine- Site Number : 840012
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: University of Florida-Genetics- Site Number : 840096
state: Florida
city: Decatur
country: United States
facility: Emory University School of Medicine- Human Genetics- Site Number : 840060
state: Georgia
city: Aurora
country: United States
facility: University of Colorado at Denver Genetics- Site Number : 840068
state: Illinois
city: Chicago
country: United States
facility: Ann and Robert Lurie Children's Hospital- Site Number : 840011
state: Illinois
city: Chicago
country: United States
facility: Ann and Robert Lurie Children's Hospital- Site Number : 840013
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: Kansas City
country: United States
facility: University of Kansas Medical Center- Site Number : 840020
state: Kansas
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: Portland
country: United States
facility: Maine Medical Center Pediatrics- Site Number : 840064
state: Maine
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 : 840092
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: Grand Rapids
country: United States
facility: Spectrum for Health- Site Number : 840019
state: Michigan
city: Grand Rapids
country: United States
facility: Infusion Associates- Site Number : 840050
state: Michigan
city: Minneapolis
country: United States
facility: Children's Health Care- Site Number : 840114
state: Minnesota
city: Minneapolis
country: United States
facility: Children's Hospital and Clinics of Minnesota- Site Number : 840046
state: Minnesota
city: Minneapolis
country: United States
facility: University of Minnesota Medical Center Pediatrics- Site Number : 840035
state: Minnesota
city: Minneapolis
country: United States
facility: University of Minnesota Medical Center Pediatrics- Site Number : 840076
state: Minnesota
city: Columbia
country: United States
facility: University of Missouri Health System Department of Genetics- Site Number : 840031
state: Missouri
city: Kansas City
country: United States
facility: Children's Mercy Hospital- Pediatrics- Site Number : 840063
state: Missouri
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: Camden
country: United States
facility: Cooper Health Center- Site Number : 840098
state: New Jersey
city: Hackensack
country: United States
facility: Hackensack University Medical Center- 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: New York
country: United States
facility: Mt. Sinai School of Medicine- Site Number : 840005
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: Valhalla
country: United States
facility: New York Medical College- Site Number : 840039
state: New York
city: Charlotte
country: United States
facility: Carolinas Medical Center Hospital- Site Number : 840065
state: North Carolina
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: University Hospitals Cleveland Medical Center- Site Number : 840119
state: Ohio
city: Cleveland
country: United States
facility: Cleveland Clinic Foundation Pediatrics- Site Number : 840048
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 & Sciences University- Site Number : 840080
state: Oregon
city: Portland
country: United States
facility: Oregon Health and Science University- Site Number : 840095
state: Oregon
city: Portland
country: United States
facility: Oregon Health and Scinece University- Site Number : 840038
state: Oregon
city: Hershey
country: United States
facility: Penn Hershey Medical Center- Site Number : 840061
state: Pennsylvania
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: UPMC- Site Number : 840014
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: Nashville
country: United States
facility: Vanderbilt University Hospital-Pediatrics- Site Number : 840049
state: Tennessee
city: Dallas
country: United States
facility: Baylor Research Center- Site Number : 840058
state: Texas
city: Fort Worth
country: United States
facility: Cook Children's Health Care System- Site Number : 840024
state: Texas
city: Salt Lake City
country: United States
facility: University of Utah Department of Medical Genetics- Site Number : 840043
state: Utah
city: Charlottesville
country: United States
facility: University of Virginia School of Medicine-Pediatrics- Site Number : 840078
state: Virginia
city: Fairfax
country: United States
facility: O&O Alpan, LLC- Site Number : 840025
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: Caba
country: Argentina
facility: Investigational Site Number : 152218
state: Buenos Aires
city: Caba
country: Argentina
facility: Investigational Site Number : 153194
state: Buenos Aires
city: Ciudad Autónoma de Buenos Aires
country: Argentina
facility: Investigational Site Number : 153032
state: Buenos Aires
city: Westmead
country: Australia
facility: Investigational Site Number : 036013
state: New South Wales
city: Westmead
country: Australia
facility: Investigational Site Number : 151069
state: New South Wales
city: Brisbane
country: Australia
facility: Investigational Site Number : 154087
state: Queensland
city: Brisbane
country: Australia
facility: Investigational Site Number : 152096
state: Queensland
city: Adelaide
country: Australia
facility: Investigational Site Number : 151002
state: South Australia
city: Murdoch
country: Australia
facility: Investigational Site Number : 151015
state: Western Australia
city: Antwerp
country: Belgium
facility: Investigational Site Number : 150722
city: Bruges
country: Belgium
facility: Investigational Site Number : 154298
city: Brussels
country: Belgium
facility: Investigational Site Number : 056010
city: Bruxelles
country: Belgium
facility: Investigational Site Number : 150684
city: Edegem
country: Belgium
facility: Investigational Site Number : 152072
city: Gent
country: Belgium
facility: Investigational Site Number : 152295
city: Gosselies
country: Belgium
facility: Investigational Site Number : 153179
city: Leuven
country: Belgium
facility: Investigational Site Number : 151668
city: Leuven
country: Belgium
facility: Investigational Site Number : 152126
city: Fortaleza
country: Brazil
facility: Hospital Universitario Walter Cantidio- Site Number : 076011
state: Ceará
city: Cuiabá
country: Brazil
facility: Hospital Geral e Maternidade de Cuiabá- Site Number : 076003
state: Mato Grosso
city: Porto Alegre
country: Brazil
facility: Hospital de Clinicas de Porto Alegre- Site Number : 076008
state: Rio Grande Do Sul
city: Ribeirão Preto
country: Brazil
facility: Hospital das Clinicas da Faculdade de Medicina de Ribeirao Preto- Site Number : 076009
state: São Paulo
city: Rio de Janeiro
country: Brazil
facility: Instituto Fernandes Figueira - FIOCRUZ- Site Number : 076001
city: São Paulo
country: Brazil
facility: Instituto de Genética e Erros Inatos do Metabolismo- Site Number : 076002
city: São Paulo
country: Brazil
facility: Núcleo de Gestão em Pesquisa (NGP) - UNIFESP- Site Number : 076012
city: São Paulo
country: Brazil
facility: Hospital das Clinicas da Faculdade de Medicina da Universidade de Sao Paulo- Site Number : 076004
city: São Paulo
country: Brazil
facility: Hospital das Clinicas FMUSP- Site Number : 076007
city: Sofia
country: Bulgaria
facility: Investigational Site Number : 100001
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 : 124010
state: British Columbia
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: Hamilton
country: Canada
facility: Investigational Site Number : 124007
state: Ontario
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 : 124004
state: Quebec
city: Montreal
country: Canada
facility: Investigational Site Number : 124018
state: Quebec
city: Sherbrooke
country: Canada
facility: Investigational Site Number : 124001
state: Quebec
city: Punta Arenas
country: Chile
facility: Investigational Site Number : 152002
city: Santiago
country: Chile
facility: Investigational Site Number : 152001
city: Beijing
country: China
facility: Investigational Site Number : 152380
city: Beijing
country: China
facility: Investigational Site Number : 153202
city: Beijing
country: China
facility: Investigational Site Number : 153203
city: Guangzhou
country: China
facility: Investigational Site Number : 152379
city: Jinan
country: China
facility: Investigational Site Number : 152381
city: Shanghai
country: China
facility: Investigational Site Number : 152356
city: Shanghai
country: China
facility: Investigational Site Number : 152382
city: Bogota
country: Colombia
facility: Investigational Site Number : 170001
city: Zagreb
country: Croatia
facility: Investigational Site Number : 1910001
city: Zagreb
country: Croatia
facility: Investigational Site Number : 1910002
city: Praha 2
country: Czechia
facility: Investigational Site Number : 150809
city: København Ø
country: Denmark
facility: Investigational Site Number : 151229
city: Bordeaux
country: France
facility: Investigational Site Number : 250010
city: Brest
country: France
facility: Investigational Site Number : 250012
city: BRON Cedex
country: France
facility: Investigational Site Number : 250003
city: BRON Cedex
country: France
facility: Investigational Site Number : 250007
city: Caen
country: France
facility: Investigational Site Number : 250009
city: La Tronche
country: France
facility: Investigational Site Number : 250015
city: Lille
country: France
facility: Investigational Site Number : 250008
city: Marseille Cedex 05
country: France
facility: Investigational Site Number : 250002
city: Nancy
country: France
facility: Investigational Site Number : 250017
city: Nantes
country: France
facility: Investigational Site Number : 250019
city: Nice
country: France
facility: Investigational Site Number : 250005
city: Nîmes
country: France
facility: Investigational Site Number : 250011
city: Paris
country: France
facility: Investigational Site Number : 250004
city: Paris
country: France
facility: Investigational Site Number : 250001
city: St-priest-en-jarez
country: France
facility: Investigational Site Number : 250018
city: Strasbourg
country: France
facility: Investigational Site Number : 250016
city: Toulouse
country: France
facility: Investigational Site Number : 250014
city: Tours
country: France
facility: Investigational Site Number : 250006
city: Vannes Cedex
country: France
facility: Investigational Site Number : 25013
city: Berlin
country: Germany
facility: Investigational Site Number : 152041
city: Berlin
country: Germany
facility: Investigational Site Number : 151686
city: Berlin
country: Germany
facility: Investigational Site Number : 152293
city: Bochum
country: Germany
facility: Investigational Site Number : 152234
city: Bonn
country: Germany
facility: Investigational Site Number : 152200
city: Freiburg
country: Germany
facility: Investigational Site Number : 152108
city: Gießen
country: Germany
facility: Investigational Site Number : 154150
city: Halle (Saale)
country: Germany
facility: Investigational Site Number : 152045
city: Hamburg
country: Germany
facility: Investigational Site Number : 154215
city: Mainz
country: Germany
facility: Investigational Site Number : 150031
city: München
country: Germany
facility: Investigational Site Number : 152075
city: Münster
country: Germany
facility: Investigational Site Number : 152299
city: Thessaloniki
country: Greece
facility: Investigational Site Number : 300001
city: Kowloon
country: Hong Kong
facility: Investigational Site Number : 344001
city: Debrecen
country: Hungary
facility: Investigational Site Number : 3480009
city: Nyíregyháza
country: Hungary
facility: Investigational Site Number : 3480007
city: Pécs
country: Hungary
facility: Investigational Site Number : 3480011
city: Székesfehérvár
country: Hungary
facility: Investigational Site Number : 3480012
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: Beer Sheva
country: Israel
facility: Investigational Site Number : 3760002
city: Haifa
country: Israel
facility: Investigational Site Number : 3760001
city: Tel Aviv
country: Israel
facility: Investigational Site Number : 3760003
city: Milan
country: Italy
facility: Investigational Site Number : 380007
state: Milano
city: Padua
country: Italy
facility: Investigational Site Number : 380011
state: Padova
city: Brescia
country: Italy
facility: Investigational Site Number : 380008
city: Cagliari
country: Italy
facility: Investigational Site Number : 380006
city: Ferrara
country: Italy
facility: Investigational Site Number : 380014
city: Firenze
country: Italy
facility: Investigational Site Number : 380005
city: Genova
country: Italy
facility: Investigational Site Number : 380004
city: Messina
country: Italy
facility: Investigational Site Number : 380013
city: Milano
country: Italy
facility: Investigational Site Number : 380016
city: Monza
country: Italy
facility: Investigational Site Number : 380009
city: Napoli
country: Italy
facility: Investigational Site Number : 380010
city: Napoli
country: Italy
facility: Investigational Site Number : 380019
city: Padova
country: Italy
facility: Investigational Site Number : 380002
city: Pavia
country: Italy
facility: Investigational Site Number : 380003
city: Roma
country: Italy
facility: Investigational Site Number : 380015
city: Roma
country: Italy
facility: Investigational Site Number : 380012
city: Torino
country: Italy
facility: Investigational Site Number : 380017
city: Udine
country: Italy
facility: Investigational Site Number : 380001
city: Sakyo-ku
country: Japan
facility: Investigational Site Number : 392106
state: Kyoto
city: Ginowan
country: Japan
facility: Investigational Site Number : 392105
state: Okinawa
city: Amman
country: Jordan
facility: Investigational Site Number : 4000001
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: Seoul
country: Korea, Republic of
facility: Investigational Site Number : 153180
state: Seoul-teukbyeolsi
city: Seoul
country: Korea, Republic of
facility: Investigational Site Number : 153196
state: Seoul-teukbyeolsi
city: Seoul
country: Korea, Republic of
facility: Investigational Site Number : 153176
state: Seoul-teukbyeolsi
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 : 153192
city: Seoul
country: Korea, Republic of
facility: Investigational Site Number : 151149
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: Beirut
country: Lebanon
facility: Investigational Site Number : 4220002
city: Jalan Pahang
country: Malaysia
facility: Investigational Site Number : 458001
city: Kuala Lumpur
country: Malaysia
facility: Investigational Site Number : 458002
city: Rotterdam
country: Netherlands
facility: Investigational Site Number : 152029
city: Karachi
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 : 620008
city: Lisboa
country: Portugal
facility: Investigational Site Number : 620006
city: Bucuresti
country: Romania
facility: Investigational Site Number : 154099
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: Belgrade
country: Serbia
facility: Investigational Site Number : 6880001
city: Singapore
country: Singapore
facility: Investigational Site Number : 702001
city: Bratislava
country: Slovakia
facility: Investigational Site Number : 703001
city: Chiayi
country: Taiwan
facility: Investigational Site Number : 154093
city: Hualian
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: Taipei
country: Taiwan
facility: Investigational Site Number : 152226
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: Cambridge
country: United Kingdom
facility: Investigational Site Number : 151340
state: Cambridgeshire
city: Birmingham
country: United Kingdom
facility: Investigational Site Number : 152287
state: England
city: London
country: United Kingdom
facility: Investigational Site Number : 151646
state: London, City Of
city: London
country: United Kingdom
facility: Investigational Site Number : 150303
state: London, City Of
city: Birmingham
country: United Kingdom
facility: Investigational Site Number : 154240
city: Cardiff
country: United Kingdom
facility: Investigational Site Number : 152334
city: Glasgow
country: United Kingdom
facility: Investigational Site Number : 152156
city: London
country: United Kingdom
facility: Investigational Site Number : 151123
city: Manchester
country: United Kingdom
facility: Investigational Site Number : 150455
city: Newcastle
country: United Kingdom
facility: Investigational Site Number : 152087
city: Salford
country: United Kingdom
facility: Investigational Site Number : 150964
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: Study Director
role: STUDY_DIRECTOR
References:
citation: Kishnani PS, Amartino HM, Lindberg C, Miller TM, Wilson A, Keutzer J. Methods of diagnosis of patients with Pompe disease: Data from the Pompe Registry. Mol Genet Metab. 2014 Sep-Oct;113(1-2):84-91. doi: 10.1016/j.ymgme.2014.07.014. Epub 2014 Jul 16.
citation: Kishnani PS, Amartino HM, Lindberg C, Miller TM, Wilson A, Keutzer J; Pompe Registry Boards of Advisors. Timing of diagnosis of patients with Pompe disease: data from the Pompe registry. Am J Med Genet A. 2013 Oct;161A(10):2431-43. doi: 10.1002/ajmg.a.36110. Epub 2013 Aug 30.
citation: Byrne BJ, Kishnani PS, Case LE, Merlini L, Muller-Felber W, Prasad S, van der Ploeg A. Pompe disease: design, methodology, and early findings from the Pompe Registry. Mol Genet Metab. 2011 May;103(1):1-11. doi: 10.1016/j.ymgme.2011.02.004. Epub 2011 Feb 11.
citation: Zhao Y, Wang Z, Lu J, Gu X, Huang Y, Qiu Z, Wei Y, Yan C. Characteristics of Pompe disease in China: a report from the Pompe registry. Orphanet J Rare Dis. 2019 Apr 3;14(1):78. doi: 10.1186/s13023-019-1054-0.