RECRUITING

Pompe Disease Registry Protocol

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Conditions

Glycogen Storage Disease Type IIPompe DiseaseGlycogen Storage Disease Type II (GSD-II)GSD-IIPompe Disease (late-onset)Acid Maltase Deficiency DiseaseGlycogenosis II

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

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

Quick Facts

Study Start:2004-09-15
Study Completion:2034-01-31
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:RECRUITING

Study ID

NCT00231400

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:Not specified
Sexes Eligible for Study:ALL
Accepts Healthy Volunteers:No
Standard Ages:CHILD, ADULT, OLDER_ADULT
Inclusion CriteriaExclusion Criteria
  1. Age 18 years or older
  2. Willing and able to provide informed consent
  3. Able to understand and follow study procedures
  4. Stable medical condition
  1. Pregnancy or breastfeeding
  2. Severe psychiatric disorders
  3. Active substance abuse
  4. Unstable medical conditions
  5. Inability to comply with study requirements

Contacts and Locations

Study Contact

Trial Transparency email recommended (Toll free number for US & Canada)
CONTACT
800-633-1610
Contact-Us@sanofi.com
Pompe Registry HelpLine
CONTACT
617-591-5500

Principal Investigator

Study Director
STUDY_DIRECTOR
Genzyme, a Sanofi Company

Study Locations (Sites)

University Alabama at Birmingham- Site Number : 840106
Birmingham, Alabama, 35233
United States
Barrow Neurological Institute- Site Number : 840087
Phoenix, Arizona, 85013
United States
Phoenix Children's Hospital- Site Number : 840003
Phoenix, Arizona, 85013
United States
University of Arizona- Site Number : 840015
Tucson, Arizona, 85724
United States
Arkansas Childrens Hospital- Site Number : 840109
Little Rock, Arkansas, 72202
United States
University of Arkansas for Medical Sciences- Site Number : 840113
Little Rock, Arkansas, 72205
United States
University of California at Irvine- Site Number : 840036
Irvine, California, 90211
United States
Loma Linda University- Genetics- Site Number : 840070
Loma Linda, California, 92354
United States
Southern California Permanente Medical Group- Site Number : 840108
Los Angeles, California, 90027
United States
USC Health Sciences Center Dept of Genetics- Site Number : 840082
Los Angeles, California, 90033
United States
Children's Hospital Oakland- Site Number : 840029
Oakland, California, 94609
United States
Children's Hospital of Orange County- Site Number : 840074
Orange, California, 92868
United States
UC Davis MIND Institute- Site Number : 840010
Sacramento, California, 95817
United States
Loma Linda University Children's Hospital- Site Number : 840117
San Bernardino, California, 92408
United States
University of California at San Diego- Site Number : 840007
San Diego, California, 92093
United States
University of California at San Francisco- Site Number : 840051
San Francisco, California
United States
Stanford University Pediatrics- Site Number : 840021
Stanford, California, 94305
United States
Children's Hospital IMD Clinic- Site Number : 840069
Aurora, Colorado, 80045
United States
Yale - Site Number : 840047
New Haven, Connecticut, 06520
United States
Children's National Medical Center- Site Number : 840067
Washington, District of Columbia, 20010
United States
University of Florida-Genetics- Site Number : 840012
Gainesville, Florida, 32610
United States
University of Florida Pediatrics Genetics- Site Number : 840121
Jacksonville, Florida, 32207
United States
The Atwal Clinic- Site Number : 840112
Jacksonville, Florida, 32216
United States
University Of Miami SOM- Site Number : 840006
Miami, Florida, 33136
United States
University of Florida-Genetics- Site Number : 840096
Tampa, Florida, 33606
United States
Emory University School of Medicine- Human Genetics- Site Number : 840060
Decatur, Georgia, 30033
United States
University of Colorado at Denver Genetics- Site Number : 840068
Aurora, Illinois, 80045
United States
Ann and Robert Lurie Children's Hospital- Site Number : 840011
Chicago, Illinois, 60611
United States
Ann and Robert Lurie Children's Hospital- Site Number : 840013
Chicago, Illinois, 60611
United States
Rush University Medical Center Genetics- Site Number : 840079
Chicago, Illinois, 60612
United States
Indianapolis University School of Medicine- Site Number : 840027
Indianapolis, Indiana, 46202
United States
University of Iowa- Site Number : 840032
Iowa City, Iowa, 52242
United States
University of Kansas Medical Center- Site Number : 840020
Kansas City, Kansas, 66160
United States
University of Louisville- Genetics- Site Number : 840030
Louisville, Kentucky, 40202
United States
Ochsner Medical Center - Site Number : 840120
New Orleans, Louisiana, 70121
United States
Maine Medical Center Pediatrics- Site Number : 840064
Portland, Maine, 04102
United States
John Hopkins- Site Number : 840044
Baltimore, Maryland
United States
Massachusetts General Hospital-Genetics- Site Number : 840062
Boston, Massachusetts, 02114
United States
Boston Children's Hospital- Site Number : 840092
Boston, Massachusetts, 2115
United States
Baystate Health- Site Number : 840002
Springfield, Massachusetts, 01199
United States
University of Michigan Pediatrics- Site Number : 840107
Ann Arbor, Michigan, 48109
United States
Children's Hospital of Michigan- Site Number : 840066
Detroit, Michigan, 48201
United States
Spectrum for Health- Site Number : 840019
Grand Rapids, Michigan, 49503
United States
Infusion Associates- Site Number : 840050
Grand Rapids, Michigan, 49525
United States
Children's Health Care- Site Number : 840114
Minneapolis, Minnesota, 55404
United States
Children's Hospital and Clinics of Minnesota- Site Number : 840046
Minneapolis, Minnesota, 55404
United States
University of Minnesota Medical Center Pediatrics- Site Number : 840035
Minneapolis, Minnesota, 55455
United States
University of Minnesota Medical Center Pediatrics- Site Number : 840076
Minneapolis, Minnesota, 55455
United States
University of Missouri Health System Department of Genetics- Site Number : 840031
Columibia, Missouri, 65212
United States
Children's Mercy Hospital- Pediatrics- Site Number : 840063
Kansas City, Missouri, 64108
United States
Washington University of St. Louis- Site Number : 840100
Saint Louis, Missouri, 63110
United States
University of Nebraska Medical Center- Pediatrics- Site Number : 840084
Omaha, Nebraska, 68198
United States
Children's Specialty Center of Nevada- Site Number : 840008
Las Vegas, Nevada, 89109
United States
Cooper Health Center- Site Number : 840098
Camden, New Jersey
United States
Hackensack University Medical Center- Site Number : 840101
Hackensack, New Jersey, 07601
United States
Atlantic Health System- Site Number : 840099
Morristown, New Jersey, 07962
United States
St. Peter's University Hospital- Site Number : 840016
New Brunswick, New Jersey, 08901
United States
St. Joseph's Children's Hospital- Site Number : 840057
Paterson, New Jersey, 07503
United States
Northwell Health- Site Number : 840102
Manhasset, New York, 11030
United States
New York University School Of Medicine- Site Number : 840040
New York, New York, 10016
United States
Metropolitan Hospital Center- Site Number : 840110
New York, New York, 10029-7494
United States
Mt. Sinai School of Medicine- Site Number : 840005
New York, New York, 10029
United States
University of Rochester Medical Center SOM- Site Number : 840105
Rochester, New York, 14642
United States
State University of New York- Site Number : 840052
Syracuse, New York, 13210
United States
New York Medical College- Site Number : 840039
Valhalla, New York, 10595
United States
Carolinas Medical Center Hospital- Site Number : 840065
Charlotte, North Carolina, 28232
United States
Duke University Medical Center Genetics Dept- Site Number : 840037
Durham, North Carolina, 27710
United States
Cincinnati Children's Hospital-Genetics- Site Number : 840041
Cincinnati, Ohio
United States
UHCMC- Site Number : 840119
Cleveland, Ohio, 44106
United States
Cleveland Clinic Foundation Pediatrics- Site Number : 840048
Cleveland, Ohio, 44195
United States
Nationwide Children's Hospital- Site Number : 840091
Columbus, Ohio, 43205-2664
United States
LSD Data Registry Site LLC- Site Number : 840094
Dublin, Ohio, 43017
United States
Oregon Health & Sciences University- Site Number : 840080
Portland, Oregon, 97201
United States
Oregon Health and Science University- Site Number : 840095
Portland, Oregon, 97239
United States
Oregon Health and Scinece University- Site Number : 840038
Portland, Oregon, 97239
United States
Penn Hershey Medical Center- Site Number : 840061
Hershey, Pennsylvania, 17033
United States
Children's Hospital of Philadelphia HUP Medical Genetics- Site Number : 840089
Philadelphia, Pennsylvania, 19104
United States
Children's Hospital of Philadelphia- Site Number : 840034
Philadelphia, Pennsylvania
United States
UPMC- Site Number : 840014
Pittsburgh, Pennsylvania, 15213
United States
University of Pittsburgh- Site Number : 840023
Pittsburgh, Pennsylvania, 15224
United States
Rhode Island Hospital Genetics- Site Number : 840053
Providence, Rhode Island, 02903
United States
Greenwood Genetcs- Site Number : 840055
Greenville, South Carolina, 29605
United States
Vanderbilt University Hospital-Pediatrics- Site Number : 840049
Nashville, Tennessee, 38232
United States
Baylor Research Center- Site Number : 840058
Dallas, Texas, 75226
United States
Cook Children's Health Care System- Site Number : 840024
Fort Worth, Texas, 76104
United States
University of Utah Department of Medical Genetics- Site Number : 840043
Salt Lake City, Utah, 84132
United States
University of Virginia School of Medicine-Pediatrics- Site Number : 840078
Charlottesville, Virginia, 22908
United States
O&O Alpan, LLC- Site Number : 840025
Fairfax, Virginia, 22030
United States
Children's Hospital of the Kings Daughters- Site Number : 840072
Norfolk, Virginia, 23507
United States
Seattle Children's Hospital- Site Number : 840028
Seattle, Washington, 98105
United States
University Of Washington Medical Center- Site Number : 840059
Seattle, Washington, 98195
United States
Multicare Health System ¿ Mary Bridge Childrens Hospital- Site Number : 840115
Tacoma, Washington, 98415
United States
Children's Hospital of Wisconsin-Pediatrics- Site Number : 840054
Milwaukee, Wisconsin, 53226
United States

Collaborators and Investigators

Sponsor: Genzyme, a Sanofi Company

  • Study Director, STUDY_DIRECTOR, Genzyme, a Sanofi Company

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2004-09-15
Study Completion Date2034-01-31

Study Record Updates

Study Start Date2004-09-15
Study Completion Date2034-01-31

Terms related to this study

Keywords Provided by Researchers

  • Glycogen Storage Disease Type II (GSD-II)
  • GSD-II
  • Pompe Disease
  • Pompe Disease (late-onset)
  • Acid Maltase Deficiency Disease
  • Glycogenosis II

Additional Relevant MeSH Terms

  • Glycogen Storage Disease Type II
  • Pompe Disease