Pompe Disease Registry Protocol

Part of paid clinical trials in Birmingham, Alabama.

Sponsor
Genzyme, a Sanofi Company
Study ID
NCT00231400
Status
Recruiting
Apply to this trial

Conditions

Eligibility Criteria

Sex
ALL
Age
N/A - N/A
Healthy Volunteers
Not accepted

Study Details

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.

Key Dates

Start date
Sep 15, 2004
Status verified
Apr 2026
Primary completion
Jan 31, 2034
Completion
Jan 31, 2034

Study Design

Enrollment
2,000 participants (estimated)

Primary Outcome Measure

Understanding of the variability, progression , identification and natural history of the manifestations of Pompe disease [ Time Frame: maximum 30 years ]

Central Contacts

  • Trial Transparency email recommended (Toll free number for US & Canada)
    800-633-1610
    [email protected]
  • Pompe Registry HelpLine
    617-591-5500

Locations (93)

FacilityCityStateZIPSite coordinators
University of Alabama at Birmingham- Site Number : 840106BirminghamAlabama35233-
Barrow Neurological Institute- Site Number : 840087PhoenixArizona85013-
Phoenix Children's Hospital- Site Number : 840003PhoenixArizona85016-
University of Arizona- Site Number : 840015TucsonArizona85724-
Arkansas Children's Hospital- Site Number : 840109Little RockArkansas72202-
University of Arkansas for Medical Sciences- Site Number : 840113Little RockArkansas72205-
University of California at Irvine- Site Number : 840036IrvineCalifornia90211-
Loma Linda University- Genetics- Site Number : 840070Loma LindaCalifornia92354-
Southern California Permanente Medical Group- Site Number : 840108Los AngelesCalifornia90027-
USC Health Sciences Center Dept of Genetics- Site Number : 840082Los AngelesCalifornia90033-
UCSF Benioff Children's Hospital - Oakland- Site Number : 840029OaklandCalifornia94609-
Children's Hospital of Orange County- Site Number : 840074OrangeCalifornia92868-
UC Davis MIND Institute- Site Number : 840010SacramentoCalifornia95817-
University of California at San Diego- Site Number : 840007San DiegoCalifornia92093-
University of California San Francisco - Parnassus Campus- Site Number : 840051San FranciscoCalifornia94143-
Stanford University Pediatrics- Site Number : 840021StanfordCalifornia94305-
Children's Hospital Colorado - Aurora- Site Number : 840069AuroraColorado80045-
Yale University School of Medicine- Site Number : 840047New HavenConnecticut06510-
Children's National Medical Center - Washington- Site Number : 840067Washington D.C.District of Columbia20010-
University of Florida College of Medicine- Site Number : 840012GainesvilleFlorida32610-
University of Florida Pediatrics Genetics- Site Number : 840121JacksonvilleFlorida32207-
University Of Miami SOM- Site Number : 840006MiamiFlorida33136-
University of Florida-Genetics- Site Number : 840096TampaFlorida33606-
Emory University School of Medicine- Human Genetics- Site Number : 840060DecaturGeorgia30033-
University of Colorado at Denver Genetics- Site Number : 840068AuroraIllinois80045-
Ann & Robert H. Lurie Children's Hospital of Chicago- Site Number : 840011ChicagoIllinois60611-
Ann & Robert H. Lurie Children's Hospital of Chicago- Site Number : 840013ChicagoIllinois60611-
Rush University Medical Center Genetics- Site Number : 840079ChicagoIllinois60612-
Indianapolis University School of Medicine- Site Number : 840027IndianapolisIndiana46202-
University of Iowa- Site Number : 840032Iowa CityIowa52242-
University Of Kansas Medical Center- Site Number : 840020Kansas CityKansas66160-
University of Louisville - Health Sciences Center- Site Number : 840030LouisvilleKentucky40202-
Maine Medical Partners Pediatric Specialty Care- Site Number : 840064PortlandMaine04102-
John Hopkins- Site Number : 840044BaltimoreMaryland21205-
Boston Children's Hospital - PIN- Site Number : 840092BostonMassachusetts02115-
Massachusetts General Hospital-Genetics- Site Number : 840062BostonMassachusetts02114-
Baystate Medical Center- Site Number : 840002SpringfieldMassachusetts01199-
University of Michigan Pediatrics- Site Number : 840107Ann ArborMichigan48109-
Children's Hospital of Michigan- Site Number : 840066DetroitMichigan48201-
Corewell Health - Helen DeVos Children's Hospital (HDVCH)- Site Number : 840019Grand RapidsMichigan49503-
Infusion Associates - Grand Rapids- Site Number : 840050Grand RapidsMichigan49525-
Children's Hospitals and Clinics of Minnesota- Site Number : 840046MinneapolisMinnesota55404-
Children's Hospitals and Clinics of Minnesota- Site Number : 840114MinneapolisMinnesota55404-
M Health Fairview University of Minnesota Medical Center - West Bank- Site Number : 840035MinneapolisMinnesota55454-
M Health Fairview University of Minnesota Medical Center - West Bank- Site Number : 840076MinneapolisMinnesota55454-
University of Missouri Health System Department of Genetics- Site Number : 840031ColumbiaMissouri65212-
Children's Mercy Adelle Hall Campus- Site Number : 840063Kansas CityMissouri64108-
Washington University- Site Number : 840100St LouisMissouri63110-
University of Nebraska Medical Center- Site Number : 840084OmahaNebraska68198-
Children's Specialty Center - Las Vegas- Site Number : 840008Las VegasNevada89109-
Cooper Health Center- Site Number : 840098CamdenNew Jersey08103-
Hackensack Meridian Health - Hackensack University Medical Center- Site Number : 840101HackensackNew Jersey07601-
Atlantic Health System- Site Number : 840099MorristownNew Jersey07962-
Saint Peter's University Hospital - New Brunswick- Site Number : 840016New BrunswickNew Jersey08901-
St. Joseph's Children's Hospital- Site Number : 840057PatersonNew Jersey07503-
NYU Langone Health - Perlmutter Cancer Center - Huntington- Site Number : 840040HuntingtonNew York11743-
Northwell Health- Site Number : 840102New Hyde ParkNew York11042-
Columbia University Irving Medical Center- Site Number : 840125New YorkNew York10032-
Metropolitan Hospital Center- Site Number : 840110New YorkNew York10029-
Mt. Sinai School of Medicine- Site Number : 840005New YorkNew York10029-
University of Rochester Medical Center- Site Number : 840105RochesterNew York14642-
SUNY Upstate Medical University - Site Number : 840052SyracuseNew York13210-
New York Medical College- Site Number : 840039ValhallaNew York10595-
Carolinas Medical Center Hospital- Site Number : 840065CharlotteNorth Carolina28232-
Duke University Medical Center Genetics Dept- Site Number : 840037DurhamNorth Carolina27710-
Cincinnati Children's Hospital Medical Center (Cincinnati Children's Hospital)- Site Number : 840041CincinnatiOhio45229-
University of Cincinnati Medical Center- Site Number : 840124CincinnatiOhio45219-
Cleveland Clinic - Cleveland- Site Number : 840048ClevelandOhio44195-
University Hospitals Cleveland Medical Center- Site Number : 840119ClevelandOhio44106-
Nationwide Children's Hospital- Site Number : 840091ColumbusOhio43205-
LSD Data Registry Site LLC- Site Number : 840094DublinOhio43017-
Oregon Health & Science University (OHSU)- Site Number : 840080PortlandOregon97239-
Oregon Health & Science University (OHSU)- Site Number : 840095PortlandOregon97239-
Oregon Health and Scinece University- Site Number : 840038PortlandOregon97239-
Penn Hershey Medical Center- Site Number : 840061HersheyPennsylvania17033-
Penn State Health Milton South Hershey Medical Center- Site Number : 840126HersheyPennsylvania17033-
Children's Hospital of Philadelphia HUP Medical Genetics- Site Number : 840089PhiladelphiaPennsylvania19104-
Children's Hospital of Philadelphia- Site Number : 840034PhiladelphiaPennsylvania19104-
UPMC Children's Hospital of Pittsburgh- Site Number : 840014PittsburghPennsylvania15224-
UPMC Children's Hospital of Pittsburgh- Site Number : 840023PittsburghPennsylvania15224-
Rhode Island Hospital Genetics- Site Number : 840053ProvidenceRhode Island02903-
Greenwood Genetic Center - Greenville- Site Number : 840055GreenvilleSouth Carolina29605-
Vanderbilt University Medical Center Diagnostic Lab- Site Number : 840049NashvilleTennessee37212-
Baylor Institute of Metabolic Disease- Site Number : 840058DallasTexas75226-
Cook Children's Health Care System- Site Number : 840024Fort WorthTexas76104-
University of Utah Department of Medical Genetics- Site Number : 840043Salt Lake CityUtah84132-
University of Virginia School of Medicine-Pediatrics- Site Number : 840078CharlottesvilleVirginia22908-
O & O Alpan- Site Number : 840025FairfaxVirginia22030-
Children's Hospital of The King's Daughters- Site Number : 840072NorfolkVirginia23507-
Seattle Children's Hospital- Site Number : 840028SeattleWashington98105-
University Of Washington Medical Center- Site Number : 840059SeattleWashington98195-
Mary Bridge Children's Hospital & Health Center- Site Number : 840115TacomaWashington98405-
Children's Hospital of Wisconsin-Pediatrics- Site Number : 840054MilwaukeeWisconsin53226-

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