Pompe Disease Registry

Status: Recruiting
Location: See all (271) locations...
Study Type: Observational
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.

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

• 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.

Locations
United States
Alabama
University Alabama at Birmingham- Site Number : 840106
RECRUITING
Birmingham
Arkansas
Arkansas Childrens Hospital- Site Number : 840109
RECRUITING
Little Rock
University of Arkansas for Medical Sciences- Site Number : 840113
RECRUITING
Little Rock
Arizona
Barrow Neurological Institute- Site Number : 840087
RECRUITING
Phoenix
Phoenix Children's Hospital- Site Number : 840003
RECRUITING
Phoenix
University of Arizona- Site Number : 840015
RECRUITING
Tucson
California
University of California at Irvine- Site Number : 840036
RECRUITING
Irvine
Loma Linda University- Genetics- Site Number : 840070
COMPLETED
Loma Linda
Southern California Permanente Medical Group- Site Number : 840108
RECRUITING
Los Angeles
USC Health Sciences Center Dept of Genetics- Site Number : 840082
COMPLETED
Los Angeles
Children's Hospital Oakland- Site Number : 840029
COMPLETED
Oakland
Children's Hospital of Orange County- Site Number : 840074
RECRUITING
Orange
UC Davis MIND Institute- Site Number : 840010
RECRUITING
Sacramento
University of California at San Diego- Site Number : 840007
COMPLETED
San Diego
University of California at San Francisco- Site Number : 840051
RECRUITING
San Francisco
Stanford University Pediatrics- Site Number : 840021
RECRUITING
Stanford
Colorado
Children's Hospital Colorado - Aurora- Site Number : 840069
COMPLETED
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 College of Medicine- Site Number : 840012
RECRUITING
Gainesville
University of Florida Pediatrics Genetics- Site Number : 840121
RECRUITING
Jacksonville
University Of Miami SOM- Site Number : 840006
RECRUITING
Miami
University of Florida-Genetics- Site Number : 840096
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
University of Colorado at Denver Genetics- Site Number : 840068
RECRUITING
Aurora
Ann and Robert Lurie Children's Hospital- Site Number : 840011
RECRUITING
Chicago
Ann and Robert Lurie Children's Hospital- Site Number : 840013
RECRUITING
Chicago
Rush University Medical Center Genetics- Site Number : 840079
RECRUITING
Chicago
Indiana
Indianapolis University School of Medicine- Site Number : 840027
RECRUITING
Indianapolis
Kansas
University of Kansas Medical Center- Site Number : 840020
RECRUITING
Kansas City
Kentucky
University of Louisville- Genetics- Site Number : 840030
COMPLETED
Louisville
Massachusetts
Boston Children's Hospital - PIN- Site Number : 840092
RECRUITING
Boston
Massachusetts General Hospital-Genetics- Site Number : 840062
RECRUITING
Boston
Baystate Health- Site Number : 840002
RECRUITING
Springfield
Maryland
John Hopkins- Site Number : 840044
COMPLETED
Baltimore
Maine
Maine Medical Center Pediatrics- Site Number : 840064
RECRUITING
Portland
Michigan
University of Michigan Pediatrics- Site Number : 840107
RECRUITING
Ann Arbor
Children's Hospital of Michigan- Site Number : 840066
RECRUITING
Detroit
Infusion Associates- Site Number : 840050
RECRUITING
Grand Rapids
Spectrum for Health- Site Number : 840019
COMPLETED
Grand Rapids
Minnesota
Children's Hospital and Clinics of Minnesota- Site Number : 840046
COMPLETED
Minneapolis
Children's Hospitals and Clinics of Minnesota- Site Number : 840114
RECRUITING
Minneapolis
University of Minnesota Medical Center Pediatrics- Site Number : 840035
RECRUITING
Minneapolis
University of Minnesota Medical Center Pediatrics- Site Number : 840076
RECRUITING
Minneapolis
Missouri
University of Missouri Health System Department of Genetics- Site Number : 840031
RECRUITING
Columbia
Children's Mercy Hospital- Pediatrics- Site Number : 840063
COMPLETED
Kansas City
Washington University of St. Louis- Site Number : 840100
RECRUITING
St Louis
North Carolina
Carolinas Medical Center Hospital- Site Number : 840065
RECRUITING
Charlotte
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
Cooper Health Center- Site Number : 840098
TERMINATED
Camden
Hackensack University Medical Center- 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
Mt. Sinai School of Medicine- Site Number : 840005
RECRUITING
New York
New York University School Of Medicine- Site Number : 840040
RECRUITING
New York
University of Rochester Medical Center SOM- Site Number : 840105
COMPLETED
Rochester
State University of New York- Site Number : 840052
RECRUITING
Syracuse
New York Medical College- Site Number : 840039
RECRUITING
Valhalla
Ohio
Cincinnati Children's Hospital-Genetics- Site Number : 840041
RECRUITING
Cincinnati
University of Cincinnati Medical Center- Site Number : 840124
RECRUITING
Cincinnati
Cleveland Clinic Foundation Pediatrics- Site Number : 840048
RECRUITING
Cleveland
University Hospitals Cleveland Medical Center- Site Number : 840119
RECRUITING
Cleveland
Nationwide Children's Hospital - PIN- Site Number : 840091
RECRUITING
Columbus
LSD Data Registry Site LLC- Site Number : 840094
RECRUITING
Dublin
Oregon
Oregon Health & Science University (OHSU)- Site Number : 840095
TERMINATED
Portland
Oregon Health & Sciences University- Site Number : 840080
RECRUITING
Portland
Oregon Health and Scinece University- Site Number : 840038
TERMINATED
Portland
Pennsylvania
Penn Hershey Medical Center- Site Number : 840061
COMPLETED
Hershey
Penn State Health Milton South Hershey Medical Center- Site Number : 840126
RECRUITING
Hershey
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
UPMC- Site Number : 840014
RECRUITING
Pittsburgh
Rhode Island
Rhode Island Hospital Genetics- Site Number : 840053
COMPLETED
Providence
South Carolina
Greenwood Genetcs- Site Number : 840055
RECRUITING
Greenville
Tennessee
Vanderbilt University Hospital-Pediatrics- Site Number : 840049
RECRUITING
Nashville
Texas
Baylor Research Center- Site Number : 840058
RECRUITING
Dallas
Cook Children's Health Care System- Site Number : 840024
RECRUITING
Fort Worth
Utah
University of Utah Department of Medical Genetics- Site Number : 840043
RECRUITING
Salt Lake City
Virginia
University of Virginia School of Medicine-Pediatrics- Site Number : 840078
COMPLETED
Charlottesville
O&O Alpan, LLC- Site Number : 840025
RECRUITING
Fairfax
Children's Hospital of the Kings Daughters- Site Number : 840072
COMPLETED
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 : 152218
RECRUITING
Caba
Investigational Site Number : 153194
ACTIVE_NOT_RECRUITING
Caba
Investigational Site Number : 153195
RECRUITING
Caba, Buenos Aires
Investigational Site Number : 153032
COMPLETED
Ciudad Autónoma De Buenos Aires
Australia
Investigational Site Number : 152096
TERMINATED
Brisbane
Investigational Site Number : 154087
COMPLETED
Brisbane
Investigational Site Number : 151015
RECRUITING
Murdoch
Investigational Site Number : 036013
RECRUITING
Westmead
Investigational Site Number : 151069
RECRUITING
Westmead
Belgium
Investigational Site Number : 150722
RECRUITING
Antwerp
Investigational Site Number : 154298
RECRUITING
Bruges
Investigational Site Number : 056010
RECRUITING
Brussels
Investigational Site Number : 150684
COMPLETED
Brussels
Investigational Site Number : 152072
RECRUITING
Edegem
Investigational Site Number : 152295
RECRUITING
Ghent
Investigational Site Number : 153179
RECRUITING
Gosselies
Investigational Site Number : 151668
RECRUITING
Leuven
Investigational Site Number : 152126
RECRUITING
Leuven
Brazil
Hospital Geral e Maternidade de Cuiabá- Site Number : 076003
RECRUITING
Cuiabá
Hospital Universitario Walter Cantidio- Site Number : 076011
RECRUITING
Fortaleza
Hospital de Clinicas de Porto Alegre- Site Number : 076008
RECRUITING
Porto Alegre
Hospital das Clinicas da Faculdade de Medicina de Ribeirao Preto- Site Number : 076009
RECRUITING
Ribeirão Preto
Instituto Fernandes Figueira - FIOCRUZ- Site Number : 076001
RECRUITING
Rio De Janeiro
Hospital das Clinicas da Faculdade de Medicina da Universidade de Sao Paulo- Site Number : 076004
RECRUITING
São Paulo
Hospital das Clinicas da Faculdade de Medicina da Universidade de Sao Paulo- Site Number : 076007
RECRUITING
São Paulo
Instituto de Genética e Erros Inatos do Metabolismo- Site Number : 076002
RECRUITING
São Paulo
Bulgaria
Investigational Site Number : 100001
RECRUITING
Sofia
Canada
Investigational Site Number : 124014
RECRUITING
Calgary
Investigational Site Number : 124002
RECRUITING
Edmonton
Investigational Site Number : 124007
RECRUITING
Hamilton
Investigational Site Number : 124021
RECRUITING
London
Investigational Site Number : 124022
RECRUITING
Moncton
Investigational Site Number : 124004
RECRUITING
Montreal
Investigational Site Number : 124018
RECRUITING
Montreal
Investigational Site Number : 124001
RECRUITING
Sherbrooke
Investigational Site Number : 124006
RECRUITING
Toronto
Investigational Site Number : 124010
RECRUITING
Vancouver
Investigational Site Number : 124011
COMPLETED
Vancouver
Investigational Site Number : 124019
RECRUITING
Winnipeg
Chile
Investigational Site Number : 152002
COMPLETED
Punta Arenas
Investigational Site Number : 152001
RECRUITING
Santiago
China
Investigational Site Number : 152380
TERMINATED
Beijing
Investigational Site Number : 153202
TERMINATED
Beijing
Investigational Site Number : 153203
COMPLETED
Beijing
Investigational Site Number : 152379
TERMINATED
Guangzhou
Investigational Site Number : 152381
TERMINATED
Jinan
Investigational Site Number : 157250
RECRUITING
Jinan
Investigational Site Number : 152356
TERMINATED
Shanghai
Investigational Site Number : 152382
TERMINATED
Shanghai
Colombia
Investigational Site Number : 170001
RECRUITING
Bogotá
Croatia
Investigational Site Number : 1910001
RECRUITING
Zagreb
Investigational Site Number : 1910002
RECRUITING
Zagreb
Denmark
Investigational Site Number : 151229
RECRUITING
København Ø
France
Investigational Site Number : 250010
COMPLETED
Bordeaux
Investigational Site Number : 250012
RECRUITING
Brest
Investigational Site Number : 250003
RECRUITING
Bron
Investigational Site Number : 250007
RECRUITING
Bron
Investigational Site Number : 250009
RECRUITING
Caen
Investigational Site Number : 250015
RECRUITING
La Tronche
Investigational Site Number : 250008
RECRUITING
Lille
Investigational Site Number : 250002
COMPLETED
Marseille
Investigational Site Number : 250017
RECRUITING
Nancy
Investigational Site Number : 250019
RECRUITING
Nantes
Investigational Site Number : 250005
RECRUITING
Nice
Investigational Site Number : 250011
RECRUITING
Nîmes
Investigational Site Number : 250001
RECRUITING
Paris
Investigational Site Number : 250004
RECRUITING
Paris
Investigational Site Number : 250018
RECRUITING
Saint-priest-en-jarez
Investigational Site Number : 250016
RECRUITING
Strasbourg
Investigational Site Number : 250014
RECRUITING
Toulouse
Investigational Site Number : 250006
RECRUITING
Tours
Investigational Site Number : 25013
RECRUITING
Vannes
Germany
Investigational Site Number : 151686
RECRUITING
Berlin
Investigational Site Number : 152041
COMPLETED
Berlin
Investigational Site Number : 152293
RECRUITING
Berlin
Investigational Site Number : 152234
RECRUITING
Bochum
Investigational Site Number : 152200
RECRUITING
Bonn
Investigational Site Number : 152108
RECRUITING
Freiburg Im Breisgau
Investigational Site Number : 154150
RECRUITING
Giessen
Investigational Site Number : 152045
RECRUITING
Halle
Investigational Site Number : 154215
RECRUITING
Hamburg
Investigational Site Number : 150031
RECRUITING
Mainz
Investigational Site Number : 152075
RECRUITING
München
Investigational Site Number : 152299
COMPLETED
Münster
Greece
Investigational Site Number : 300001
RECRUITING
Thessaloniki
Hong Kong Special Administrative Region
Investigational Site Number : 344001
RECRUITING
Kowloon
Hungary
Investigational Site Number : 3480009
RECRUITING
Debrecen
Investigational Site Number : 3480007
RECRUITING
Nyíregyháza
Investigational Site Number : 3480011
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
Israel
Investigational Site Number : 3760002
RECRUITING
Beersheba
Investigational Site Number : 3760001
RECRUITING
Haifa
Investigational Site Number : 3760003
COMPLETED
Tel Aviv
Italy
Investigational Site Number : 380008
RECRUITING
Brescia
Investigational Site Number : 380006
RECRUITING
Cagliari
Investigational Site Number : 380014
RECRUITING
Ferrara
Investigational Site Number : 380005
RECRUITING
Florence
Investigational Site Number : 380004
RECRUITING
Genova
Investigational Site Number : 380013
RECRUITING
Messina
Investigational Site Number : 380007
RECRUITING
Milan
Investigational Site Number : 380016
RECRUITING
Milan
Investigational Site Number : 380009
RECRUITING
Monza
Investigational Site Number : 380010
RECRUITING
Napoli
Investigational Site Number : 380019
RECRUITING
Napoli
Investigational Site Number : 380002
RECRUITING
Padua
Investigational Site Number : 380011
RECRUITING
Padua
Investigational Site Number : 380003
RECRUITING
Pavia
Investigational Site Number : 157218
RECRUITING
Pisa
Investigational Site Number : 380012
RECRUITING
Roma
Investigational Site Number : 380015
RECRUITING
Roma
Investigational Site Number : 380017
RECRUITING
Torino
Investigational Site Number : 380001
RECRUITING
Udine
Japan
Investigational Site Number : 392105
COMPLETED
Ginowan
Investigational Site Number : 392106
COMPLETED
Sakyo-ku
Jordan
Investigational Site Number : 4000001
RECRUITING
Amman
Kuwait
Investigational Site Number : 154225
COMPLETED
Kuwait City
Lebanon
Investigational Site Number : 4220004
RECRUITING
Beirut
Malaysia
Investigational Site Number : 458002
RECRUITING
Kuala Lumpur
Investigational Site Number : 458001
RECRUITING
Kuala Pahang
Netherlands
Investigational Site Number : 152029
RECRUITING
Rotterdam
Pakistan
Investigational Site Number : 154264
RECRUITING
Karachi
Investigational Site Number : 154266
RECRUITING
Lahore
Philippines
Investigational Site Number : 608001
RECRUITING
Manila
Poland
Investigational Site Number : 150575
RECRUITING
Warsaw
Portugal
Investigational Site Number : 620008
RECRUITING
Coimbra
Investigational Site Number : 620006
RECRUITING
Lisbon
Republic of Korea
Investigational Site Number : 153191
RECRUITING
Daejeon
Investigational Site Number : 151149
RECRUITING
Seoul
Investigational Site Number : 153176
RECRUITING
Seoul
Investigational Site Number : 153180
RECRUITING
Seoul
Investigational Site Number : 153192
COMPLETED
Seoul
Investigational Site Number : 153196
COMPLETED
Seoul
Investigational Site Number : 153212
RECRUITING
Seoul
Investigational Site Number : 153222
RECRUITING
Seoul
Investigational Site Number : 153178
COMPLETED
Yangsan
Romania
Investigational Site Number : 154099
RECRUITING
Bucharest
Russian Federation
Investigational Site Number : 643002
COMPLETED
Moscow
Saudi Arabia
Investigational Site Number : 682001
COMPLETED
Al Qaţīf
Investigational Site Number : 682004
RECRUITING
Mecca
Serbia
Investigational Site Number : 6880001
COMPLETED
Belgrade
Singapore
Investigational Site Number : 702001
RECRUITING
Singapore
Slovakia
Investigational Site Number : 703001
COMPLETED
Bratislava
Taiwan
Investigational Site Number : 151500
RECRUITING
Chiayi City
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 : 151179
RECRUITING
Taichung
Investigational Site Number : 151181
RECRUITING
Taichung
Investigational Site Number : 152153
RECRUITING
Tainan
Investigational Site Number : 153248
RECRUITING
Tainan
Investigational Site Number : 151182
RECRUITING
Taipei
Investigational Site Number : 152226
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 Arab Emirates
Investigational Site Number : 7840001
RECRUITING
Dubai
United Kingdom
Investigational Site Number : 152287
RECRUITING
Birmingham
Investigational Site Number : 154240
RECRUITING
Birmingham
Investigational Site Number : 151340
RECRUITING
Cambridge
Investigational Site Number : 152334
RECRUITING
Cardiff
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 : 152087
RECRUITING
Newcastle
Investigational Site Number : 150964
RECRUITING
Salford
Viet Nam
Investigational Site Number : 154212
RECRUITING
Hanoi
Investigational Site Number : 704002
RECRUITING
Ho Chi Minh City
Contact Information
Primary
Trial Transparency email recommended (Toll free number for US & Canada)
contact-us@sanofi.com
800-633-1610
Backup
Pompe Registry HelpLine
617-591-5500
Time Frame
Start Date: 2004-09-15
Estimated Completion Date: 2034-01-31
Participants
Target number of participants: 2000
Related Therapeutic Areas
Sponsors
Leads: Genzyme, a Sanofi Company

This content was sourced from clinicaltrials.gov