Pompe Disease Registry Protocol
NCT00231400
This study is recruiting at 74 US sites in 32 states, according to its ClinicalTrials.gov record last updated .
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.
Shortened from the sponsor's brief summary. View official record on ClinicalTrials.gov
Conditions studied
- Glycogen Storage Disease Type II
- Pompe Disease
Eligibility basics and study details
- Study type
- Observational
- Phase
- Not applicable
- Ages
- Any age
- Sex
- All
- Healthy volunteers
- Not accepted
- Sponsor
- Genzyme, a Sanofi Company
- Last updated on ClinicalTrials.gov
- June 23, 2026
These are the basic criteria only. The full inclusion and exclusion criteria are on the official record, and the study team decides who can take part.
US recruiting sites
Alabama · 1
- University of Alabama at Birmingham- Site Number : 840106 · Birmingham, AL
Arizona · 3
- Barrow Neurological Institute- Site Number : 840087 · Phoenix, AZ
- Phoenix Children's Hospital- Site Number : 840003 · Phoenix, AZ
- University of Arizona- Site Number : 840015 · Tucson, AZ
Arkansas · 2
- Arkansas Children's Hospital- Site Number : 840109 · Little Rock, AR
- University of Arkansas for Medical Sciences- Site Number : 840113 · Little Rock, AR
California · 6
- University of California at Irvine- Site Number : 840036 · Irvine, CA
- Southern California Permanente Medical Group- Site Number : 840108 · Los Angeles, CA
- Children's Hospital of Orange County- Site Number : 840074 · Orange, CA
- UC Davis MIND Institute- Site Number : 840010 · Sacramento, CA
- University of California San Francisco - Parnassus Campus- Site Number : 840051 · San Francisco, CA
- Stanford University Pediatrics- Site Number : 840021 · Stanford, CA
Connecticut · 1
- Yale University School of Medicine- Site Number : 840047 · New Haven, CT
District of Columbia · 1
- Children's National Medical Center - Washington- Site Number : 840067 · Washington D.C., DC
Florida · 4
- University of Miami- Site Number : 840006 · Coral Gables, FL
- University of Florida College of Medicine- Site Number : 840012 · Gainesville, FL
- University of Florida Pediatrics Genetics- Site Number : 840121 · Jacksonville, FL
- University of Florida-Genetics- Site Number : 840096 · Tampa, FL
Georgia · 1
- Emory University School of Medicine- Human Genetics- Site Number : 840060 · Decatur, GA
Illinois · 4
- University of Colorado at Denver Genetics- Site Number : 840068 · Aurora, IL
- Ann & Robert H. Lurie Children's Hospital of Chicago- Site Number : 840011 · Chicago, IL
- Ann & Robert H. Lurie Children's Hospital of Chicago- Site Number : 840013 · Chicago, IL
- Rush University Medical Center Genetics- Site Number : 840079 · Chicago, IL
Indiana · 1
- Indianapolis University School of Medicine- Site Number : 840027 · Indianapolis, IN
Iowa · 1
- University of Iowa- Site Number : 840032 · Iowa City, IA
Kansas · 1
- University of Kansas Medical Center- Site Number : 840020 · Kansas City, KS
Maine · 1
- Maine Medical Partners Pediatric Specialty Care- Site Number : 840064 · Portland, ME
Massachusetts · 3
- Boston Children's Hospital- Site Number : 840092 · Boston, MA
- Massachusetts General Hospital-Genetics- Site Number : 840062 · Boston, MA
- Baystate Medical Center- Site Number : 840002 · Springfield, MA
Michigan · 3
- University of Michigan Pediatrics- Site Number : 840107 · Ann Arbor, MI
- Children's Hospital of Michigan- Site Number : 840066 · Detroit, MI
- Infusion Associates - Grand Rapids- Site Number : 840050 · Grand Rapids, MI
Minnesota · 3
- Children's Hospitals and Clinics of Minnesota- Site Number : 840114 · Minneapolis, MN
- M Health Fairview University of Minnesota Medical Center - West Bank- Site Number : 840035 · Minneapolis, MN
- M Health Fairview University of Minnesota Medical Center - West Bank- Site Number : 840076 · Minneapolis, MN
Missouri · 2
- University of Missouri Health System Department of Genetics- Site Number : 840031 · Columbia, MO
- Washington University- Site Number : 840100 · St Louis, MO
Nebraska · 1
- University of Nebraska Medical Center- Site Number : 840084 · Omaha, NE
Nevada · 1
- Children's Specialty Center - Las Vegas- Site Number : 840008 · Las Vegas, NV
New Jersey · 4
- Hackensack Meridian Health - Hackensack University Medical Center- Site Number : 840101 · Hackensack, NJ
- Atlantic Health System- Site Number : 840099 · Morristown, NJ
- Saint Peter's University Hospital - New Brunswick- Site Number : 840016 · New Brunswick, NJ
- St. Joseph's Regional Medical Center - Paterson- Site Number : 840057 · Paterson, NJ
New York · 7
- NYU Langone Health - Perlmutter Cancer Center - Huntington- Site Number : 840040 · Huntington, NY
- Northwell Health- Site Number : 840102 · New Hyde Park, NY
- Columbia University Irving Medical Center- Site Number : 840125 · New York, NY
- Icahn School of Medicine at Mount Sinai- Site Number : 840005 · New York, NY
- Metropolitan Hospital Center- Site Number : 840110 · New York, NY
- SUNY Upstate Medical University - Site Number : 840052 · Syracuse, NY
- New York Medical College- Site Number : 840039 · Valhalla, NY
North Carolina · 2
- Carolinas Medical Center Hospital- Site Number : 840065 · Charlotte, NC
- Duke University Medical Center Genetics Dept- Site Number : 840037 · Durham, NC
Ohio · 5
- University of Cincinnati Medical Center- Site Number : 840124 · Cincinnati, OH
- Cleveland Clinic - Cleveland- Site Number : 840048 · Cleveland, OH
- University Hospitals Cleveland Medical Center- Site Number : 840119 · Cleveland, OH
- Nationwide Children's Hospital- Site Number : 840091 · Columbus, OH
- LSD Data Registry Site LLC- Site Number : 840094 · Dublin, OH
Oregon · 1
- Oregon Health & Science University (OHSU)- Site Number : 840080 · Portland, OR
Pennsylvania · 5
- Penn State Health Milton South Hershey Medical Center- Site Number : 840126 · Hershey, PA
- Children's Hospital of Philadelphia HUP Medical Genetics- Site Number : 840089 · Philadelphia, PA
- Children's Hospital of Philadelphia- Site Number : 840034 · Philadelphia, PA
- UPMC Children's Hospital of Pittsburgh- Site Number : 840014 · Pittsburgh, PA
- UPMC Children's Hospital of Pittsburgh- Site Number : 840023 · Pittsburgh, PA
South Carolina · 1
- Greenwood Genetic Center - Greenville- Site Number : 840055 · Greenville, SC
Tennessee · 1
- Vanderbilt University Medical Center Diagnostic Lab- Site Number : 840049 · Nashville, TN
Texas · 2
- Baylor Institute of Metabolic Disease- Site Number : 840058 · Dallas, TX
- Cook Children's Medical Center- Site Number : 840024 · Fort Worth, TX
Utah · 1
- University of Utah Department of Medical Genetics- Site Number : 840043 · Salt Lake City, UT
Virginia · 1
- O & O Alpan- Site Number : 840025 · Fairfax, VA
Washington · 3
- Seattle Children's Hospital- Site Number : 840028 · Seattle, WA
- University of Washington Medical Center- Site Number : 840059 · Seattle, WA
- Mary Bridge Children's Hospital & Health Center- Site Number : 840115 · Tacoma, WA
Wisconsin · 1
- Children's Wisconsin- Site Number : 840054 · Milwaukee, WI
Source: ClinicalTrials.gov, data processed . Recruiting US sites filtered by distance and site status; summaries shortened.