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RECRUITING

NCT00231400

Pompe Disease Registry Protocol

Sponsor: Genzyme, a Sanofi Company

View on ClinicalTrials.gov

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.

Official title: Pompe Disease Registry

Key Details

Gender

All

Age Range

Any - Any

Study Type

OBSERVATIONAL

Enrollment

2000

Start Date

2004-09-15

Completion Date

2034-01-31

Last Updated

2026-04-09

Healthy Volunteers

Conditions

Glycogen Storage Disease Type II Pompe Disease

Locations (271)

University of Alabama at Birmingham- Site Number : 840106

Birmingham, Alabama, United States

Barrow Neurological Institute- Site Number : 840087

Phoenix, Arizona, United States

Phoenix Children's Hospital- Site Number : 840003

Phoenix, Arizona, United States

University of Arizona- Site Number : 840015

Tucson, Arizona, United States

Arkansas Children's Hospital- Site Number : 840109

Little Rock, Arkansas, United States

University of Arkansas for Medical Sciences- Site Number : 840113

Little Rock, Arkansas, United States

University of California at Irvine- Site Number : 840036

Irvine, California, United States

Loma Linda University- Genetics- Site Number : 840070

Loma Linda, California, United States

Southern California Permanente Medical Group- Site Number : 840108

Los Angeles, California, United States

USC Health Sciences Center Dept of Genetics- Site Number : 840082

Los Angeles, California, United States

UCSF Benioff Children's Hospital - Oakland- Site Number : 840029

Oakland, California, United States

Children's Hospital of Orange County- Site Number : 840074

Orange, California, United States

UC Davis MIND Institute- Site Number : 840010

Sacramento, California, United States

University of California at San Diego- Site Number : 840007

San Diego, California, United States

University of California San Francisco - Parnassus Campus- Site Number : 840051

San Francisco, California, United States

Stanford University Pediatrics- Site Number : 840021

Stanford, California, United States

Children's Hospital Colorado - Aurora- Site Number : 840069

Aurora, Colorado, United States

Yale University School of Medicine- Site Number : 840047

New Haven, Connecticut, United States

Children's National Medical Center - Washington- Site Number : 840067

Washington D.C., District of Columbia, United States

University of Florida College of Medicine- Site Number : 840012

Gainesville, Florida, United States

University of Florida Pediatrics Genetics- Site Number : 840121

Jacksonville, Florida, United States

University Of Miami SOM- Site Number : 840006

Miami, Florida, United States

University of Florida-Genetics- Site Number : 840096

Tampa, Florida, United States

Emory University School of Medicine- Human Genetics- Site Number : 840060

Decatur, Georgia, United States

University of Colorado at Denver Genetics- Site Number : 840068

Aurora, Illinois, United States

Ann & Robert H. Lurie Children's Hospital of Chicago- Site Number : 840011

Chicago, Illinois, United States

Ann & Robert H. Lurie Children's Hospital of Chicago- Site Number : 840013

Chicago, Illinois, United States

Rush University Medical Center Genetics- Site Number : 840079

Chicago, Illinois, United States

Indianapolis University School of Medicine- Site Number : 840027

Indianapolis, Indiana, United States

University of Iowa- Site Number : 840032

Iowa City, Iowa, United States

University Of Kansas Medical Center- Site Number : 840020

Kansas City, Kansas, United States

University of Louisville - Health Sciences Center- Site Number : 840030

Louisville, Kentucky, United States

Maine Medical Partners Pediatric Specialty Care- Site Number : 840064

Portland, Maine, United States

John Hopkins- Site Number : 840044

Baltimore, Maryland, United States

Massachusetts General Hospital-Genetics- Site Number : 840062

Boston, Massachusetts, United States

Boston Children's Hospital - PIN- Site Number : 840092

Boston, Massachusetts, United States

Baystate Medical Center- Site Number : 840002

Springfield, Massachusetts, United States

University of Michigan Pediatrics- Site Number : 840107

Ann Arbor, Michigan, United States

Children's Hospital of Michigan- Site Number : 840066

Detroit, Michigan, United States

Spectrum for Health- Site Number : 840019

Grand Rapids, Michigan, United States

Infusion Associates - Grand Rapids- Site Number : 840050

Grand Rapids, Michigan, United States

Children's Hospitals and Clinics of Minnesota- Site Number : 840046

Minneapolis, Minnesota, United States

Children's Hospitals and Clinics of Minnesota- Site Number : 840114

Minneapolis, Minnesota, United States

University of Minnesota Medical Center Pediatrics- Site Number : 840035

Minneapolis, Minnesota, United States

University of Minnesota Medical Center Pediatrics- Site Number : 840076

Minneapolis, Minnesota, United States

University of Missouri Health System Department of Genetics- Site Number : 840031

Columbia, Missouri, United States

Children's Mercy Adelle Hall Campus- Site Number : 840063

Kansas City, Missouri, United States

Washington University- Site Number : 840100

St Louis, Missouri, United States

University of Nebraska Medical Center- Site Number : 840084

Omaha, Nebraska, United States

Children's Specialty Center - Las Vegas- Site Number : 840008

Las Vegas, Nevada, United States

Cooper Health Center- Site Number : 840098

Camden, New Jersey, United States

Hackensack Meridian Health - Hackensack University Medical Center- Site Number : 840101

Hackensack, New Jersey, United States

Atlantic Health System- Site Number : 840099

Morristown, New Jersey, United States

Saint Peter's University Hospital - New Brunswick- Site Number : 840016

New Brunswick, New Jersey, United States

St. Joseph's Children's Hospital- Site Number : 840057

Paterson, New Jersey, United States

Northwell Health - Site Number : 840102

Manhasset, New York, United States

New York University School Of Medicine- Site Number : 840040

New York, New York, United States

Metropolitan Hospital Center- Site Number : 840110

New York, New York, United States

Mt. Sinai School of Medicine- Site Number : 840005

New York, New York, United States

University of Rochester Medical Center- Site Number : 840105

Rochester, New York, United States

State University of New York- Site Number : 840052

Syracuse, New York, United States

New York Medical College- Site Number : 840039

Valhalla, New York, United States

Carolinas Medical Center Hospital- Site Number : 840065

Charlotte, North Carolina, United States

Duke University Medical Center Genetics Dept- Site Number : 840037

Durham, North Carolina, United States

University of Cincinnati Medical Center- Site Number : 840124

Cincinnati, Ohio, United States

Cincinnati Children's Hospital Medical Center (Cincinnati Children's Hospital)- Site Number : 840041

Cincinnati, Ohio, United States

University Hospitals Cleveland Medical Center- Site Number : 840119

Cleveland, Ohio, United States

Cleveland Clinic - Cleveland- Site Number : 840048

Cleveland, Ohio, United States

Nationwide Children's Hospital- Site Number : 840091

Columbus, Ohio, United States

LSD Data Registry Site LLC- Site Number : 840094

Dublin, Ohio, United States

Oregon Health & Science University (OHSU)- Site Number : 840080

Portland, Oregon, United States

Oregon Health & Science University (OHSU)- Site Number : 840095

Portland, Oregon, United States

Oregon Health and Scinece University- Site Number : 840038

Portland, Oregon, United States

Penn Hershey Medical Center- Site Number : 840061

Hershey, Pennsylvania, United States

Penn State Health Milton South Hershey Medical Center- Site Number : 840126

Hershey, Pennsylvania, United States

Children's Hospital of Philadelphia HUP Medical Genetics- Site Number : 840089

Philadelphia, Pennsylvania, United States

Children's Hospital of Philadelphia- Site Number : 840034

Philadelphia, Pennsylvania, United States

UPMC Children's Hospital of Pittsburgh- Site Number : 840014

Pittsburgh, Pennsylvania, United States

UPMC Children's Hospital of Pittsburgh- Site Number : 840023

Pittsburgh, Pennsylvania, United States

Rhode Island Hospital Genetics- Site Number : 840053

Providence, Rhode Island, United States

Greenwood Genetic Center - Greenville- Site Number : 840055

Greenville, South Carolina, United States

Vanderbilt University Medical Center Diagnostic Lab- Site Number : 840049

Nashville, Tennessee, United States

Baylor Institute of Metabolic Disease- Site Number : 840058

Dallas, Texas, United States

Cook Children's Health Care System- Site Number : 840024

Fort Worth, Texas, United States

University of Utah Department of Medical Genetics- Site Number : 840043

Salt Lake City, Utah, United States

University of Virginia School of Medicine-Pediatrics- Site Number : 840078

Charlottesville, Virginia, United States

O & O Alpan- Site Number : 840025

Fairfax, Virginia, United States

Children's Hospital of The King's Daughters- Site Number : 840072

Norfolk, Virginia, United States

Seattle Children's Hospital- Site Number : 840028

Seattle, Washington, United States

University Of Washington Medical Center- Site Number : 840059

Seattle, Washington, United States

Mary Bridge Children's Hospital & Health Center- Site Number : 840115

Tacoma, Washington, United States

Children's Hospital of Wisconsin-Pediatrics- Site Number : 840054

Milwaukee, Wisconsin, United States

Investigational Site Number : 153195

Caba, Buenos Aires, Buenos Aires, Argentina

Investigational Site Number : 152218

CABA, Buenos Aires, Argentina

Investigational Site Number : 153194

Buenos Aires, Argentina

Investigational Site Number : 153032

Buenos Aires, Argentina

Investigational Site Number : 036013

Westmead, New South Wales, Australia

Investigational Site Number : 151069

Westmead, New South Wales, Australia

Investigational Site Number : 154087

Brisbane, Queensland, Australia

Investigational Site Number : 152096

Brisbane, Queensland, Australia

Investigational Site Number : 151015

Murdoch, Western Australia, Australia

Investigational Site Number : 150722

Antwerp, Belgium

Investigational Site Number : 154298

Bruges, Belgium

Investigational Site Number : 150684

Brussels, Belgium

Investigational Site Number : 056010

Brussels, Belgium

Investigational Site Number : 152072

Edegem, Belgium

Investigational Site Number : 152295

Ghent, Belgium

Investigational Site Number : 153179

Gosselies, Belgium

Investigational Site Number : 151668

Leuven, Belgium

Investigational Site Number : 152126

Leuven, Belgium

Hospital Universitario Walter Cantidio- Site Number : 076011

Fortaleza, Ceará, Brazil

Hospital Geral Universitario de Cuiaba- Site Number : 076003

Cuiabá, Mato Grosso, Brazil

Hospital de Clinicas de Porto Alegre- Site Number : 076008

Porto Alegre, Rio Grande do Sul, Brazil

Hospital das Clinicas da Faculdade de Medicina de Ribeirao Preto- Site Number : 076009

Ribeirão Preto, São Paulo, Brazil

Instituto Fernandes Figueira - FIOCRUZ- Site Number : 076001

Rio de Janeiro, Brazil

Instituto de Genética e Erros Inatos do Metabolismo- Site Number : 076002

São Paulo, Brazil

Hospital das Clinicas da Faculdade de Medicina da Universidade de Sao Paulo- Site Number : 076004

São Paulo, Brazil

Hospital das Clinicas da Faculdade de Medicina da Universidade de Sao Paulo- Site Number : 076007

São Paulo, Brazil

Investigational Site Number : 100001

Sofia, Bulgaria

Investigational Site Number : 124014

Calgary, Alberta, Canada

Investigational Site Number : 124002

Edmonton, Alberta, Canada

Investigational Site Number : 124007

Lethbridge, Alberta, Canada

Investigational Site Number : 124010

Vancouver, British Columbia, Canada

Investigational Site Number : 124011

Vancouver, British Columbia, Canada

Investigational Site Number : 124019

Winnipeg, Manitoba, Canada

Investigational Site Number : 124022

Moncton, New Brunswick, Canada

Investigational Site Number : 124021

London, Ontario, Canada

Investigational Site Number : 124018

Ottawa, Ontario, Canada

Investigational Site Number : 124006

Toronto, Ontario, Canada

Investigational Site Number : 124004

Montreal, Quebec, Canada

Investigational Site Number : 124001

Sherbrooke, Quebec, Canada

Investigational Site Number : 152002

Punta Arenas, Chile

Investigational Site Number : 152001

Santiago, Chile

Investigational Site Number : 152380

Beijing, China

Investigational Site Number : 153202

Beijing, China

Investigational Site Number : 153203

Beijing, China

Investigational Site Number : 152379

Guangzhou, China

Investigational Site Number : 152381

Jinan, China

Investigational Site Number : 157250

Jinan, China

Investigational Site Number : 152382

Shanghai, China

Investigational Site Number : 152356

Shanghai, China

Investigational Site Number : 170001

Bogotá, Colombia

Investigational Site Number : 1910001

Zagreb, Croatia

Investigational Site Number : 1910002

Zagreb, Croatia

Investigational Site Number : 150809

Prague, Czechia

Investigational Site Number : 151229

Copenhagen, Denmark

Investigational Site Number : 250010

Bordeaux, France

Investigational Site Number : 250012

Brest, France

Investigational Site Number : 250003

Bron, France

Investigational Site Number : 250007

Bron, France

Investigational Site Number : 250009

Caen, France

Investigational Site Number : 250015

La Tronche, France

Investigational Site Number : 250008

Lille, France

Investigational Site Number : 250002

Marseille, France

Investigational Site Number : 250017

Nancy, France

Investigational Site Number : 250019

Nantes, France

Investigational Site Number : 250005

Nice, France

Investigational Site Number : 250011

Nîmes, France

Investigational Site Number : 250004

Paris, France

Investigational Site Number : 250001

Paris, France

Investigational Site Number : 250018

Saint-Priest-en-Jarez, France

Investigational Site Number : 250016

Strasbourg, France

Investigational Site Number : 250014

Toulouse, France

Investigational Site Number : 250006

Tours, France

Investigational Site Number : 25013

Vannes, France

Investigational Site Number : 152041

Berlin, Germany

Investigational Site Number : 151686

Berlin, Germany

Investigational Site Number : 152293

Berlin, Germany

Investigational Site Number : 152234

Bochum, Germany

Investigational Site Number : 152200

Bonn, Germany

Investigational Site Number : 152108

Freiburg im Breisgau, Germany

Investigational Site Number : 154150

Giessen, Germany

Investigational Site Number : 152045

Halle, Germany

Investigational Site Number : 154215

Hamburg, Germany

Investigational Site Number : 150031

Mainz, Germany

Investigational Site Number : 152075

Munich, Germany

Investigational Site Number : 152299

Münster, Germany

Investigational Site Number : 300001

Thessaloniki, Greece

Investigational Site Number : 344001

Kowloon, Hong Kong

Investigational Site Number : 3480009

Debrecen, Hungary

Investigational Site Number : 3480007

Nyiregyháza, Hungary

Investigational Site Number : 3480011

Pécs, Hungary

Investigational Site Number : 356003

Ahmedabad, India

Investigational Site Number : 356001

Kolkata, India

Investigational Site Number : 3560004

Secunderabad, India

Investigational Site Number : 356002

Vellore, India

Investigational Site Number : 360001

Jakarta, Indonesia

Investigational Site Number : 3760002

Beersheba, Israel

Investigational Site Number : 3760001

Haifa, Israel

Investigational Site Number : 3760003

Tel Aviv, Israel

Investigational Site Number : 380020

Gussago, Brescia, Italy

Investigational Site Number : 380014

Cona, Ferrara, Italy

Investigational Site Number : 380005

Florence, Firenze, Italy

Investigational Site Number : 380007

Milan, Milano, Italy

Investigational Site Number : 380011

Padua, Padova, Italy

Investigational Site Number : 380015

Rome, Roma, Italy

Investigational Site Number : 380008

Brescia, Italy

Investigational Site Number : 380006

Cagliari, Italy

Investigational Site Number : 380013

Messina, Italy

Investigational Site Number : 380016

Milan, Italy

Investigational Site Number : 380009

Monza, Italy

Investigational Site Number : 380019

Naples, Italy

Investigational Site Number : 380002

Padova, Italy

Investigational Site Number : 380003

Pavia, Italy

Investigational Site Number : 157218

Pisa, Italy

Investigational Site Number : 380012

Roma, Italy

Investigational Site Number : 380017

Torino, Italy

Investigational Site Number : 380001

Udine, Italy

Investigational Site Number : 392105

Ginowan, Okinawa, Japan

Investigational Site Number : 392106

Kyoto, Japan

Investigational Site Number : 4000001

Amman, Jordan

Investigational Site Number : 154225

Kuwait City, Kuwait

Investigational Site Number : 4220004

Beirut, Lebanon

Investigational Site Number : 458001

Kuala Lumpur, Malaysia

Investigational Site Number : 458002

Kuala Lumpur, Malaysia

Investigational Site Number : 152029

Rotterdam, Netherlands

Investigational Site Number : 154264

Karachi, Pakistan

Investigational Site Number : 154266

Lahore, Pakistan

Investigational Site Number : 608001

City of Muntinlupa, Philippines

Investigational Site Number : 150575

Warsaw, Poland

Investigational Site Number : 620008

Coimbra, Portugal

Investigational Site Number : 620006

Lisbon, Portugal

Investigational Site Number : 154099

Bucharest, Romania

Investigational Site Number : 643002

Moscow, Russia

Investigational Site Number : 682001

Al Qaţīf, Saudi Arabia

Investigational Site Number : 682004

Mecca, Saudi Arabia

Investigational Site Number : 6880001

Belgrade, Serbia

Investigational Site Number : 702001

Singapore, Singapore

Investigational Site Number : 703001

Bratislava, Slovakia

Investigational Site Number : 153178

Yangsan, Gyeongsangnam-do, South Korea

Investigational Site Number : 153180