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Pompe Disease Registry Protocol
Sponsor: Genzyme, a Sanofi Company
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
2026-10-28
Healthy Volunteers
No
Locations (282)
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
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
University of California Irvine Medical Center- Site Number : 840036
Orange, California, United States
Uc Davis Mind Institute- Site Number : 840010
Sacramento, California, United States
Kaiser Permanente Sacramento Medical Center- Site Number : 840042
Sacramento, California, United States
Loma Linda University Children'S Hospital- Site Number : 840117
San Bernardino, 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 Medical Center- Site Number : 840021
Stanford, California, United States
Children's Hospital Colorado - Aurora- Site Number : 840069
Aurora, Colorado, United States
University Of Colorado At Denver Genetics- Site Number : 840068
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 Miami- Site Number : 840006
Coral Gables, Florida, 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
The Atwal Clinic- Site Number : 840112
Jacksonville, 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
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
Tulane University School of Medicine- Site Number : 840001
New Orleans, Louisiana, United States
Ochsner Medical Center - Jefferson Highway- Site Number : 840120
New Orleans, Louisiana, 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- 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
Corewell Health - Helen DeVos Children's Hospital (HDVCH)- 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
M Health Fairview University of Minnesota Medical Center - West Bank- Site Number : 840035
Minneapolis, Minnesota, United States
M Health Fairview University of Minnesota Medical Center - West Bank- 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
Shodair Children'S Hospital- Site Number : 840090
Helena, Montana, 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 Regional Medical Center - Paterson- Site Number : 840057
Paterson, New Jersey, United States
NYU Langone Health - Perlmutter Cancer Center - Huntington- Site Number : 840040
Huntington, New York, United States
Northwell Health- Site Number : 840102
New Hyde Park, New York, United States
Icahn School of Medicine at Mount Sinai- Site Number : 840005
New York, New York, United States
Metropolitan Hospital Center- Site Number : 840110
New York, New York, United States
Columbia University Irving Medical Center- Site Number : 840125
New York, New York, United States
University of Rochester Medical Center- Site Number : 840105
Rochester, New York, United States
SUNY Upstate Medical University - Site Number : 840052
Syracuse, New York, United States
New York Medical College- Site Number : 840039
Valhalla, New York, United States
Atrium Health 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
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 : 840038
Portland, Oregon, 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
Penn State Health Milton South Hershey Medical Center- Site Number : 840061
Hershey, Pennsylvania, United States
Children'S Hospital Of Philadelphia Hup Medical Genetics- Site Number : 840089
Philadelphia, Pennsylvania, United States
The 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 - Medical Laboratories- Site Number : 840049
Nashville, Tennessee, United States
Baylor Institute of Metabolic Disease- Site Number : 840058
Dallas, Texas, United States
Cook Children's Medical Center- Site Number : 840024
Fort Worth, Texas, United States
University of Utah- Site Number : 840043
Salt Lake City, Utah, United States
The University of Vermont Medical Center- Site Number : 840129
South Burlington, Vermont, 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 Wisconsin- Site Number : 840054
Milwaukee, Wisconsin, United States
Investigational Site Number : 153195
Caba, Buenos Aires, Buenos Aires, Argentina
Investigational Site Number : 153194
Buenos Aires, Argentina