Working...
ClinicalTrials.gov
ClinicalTrials.gov Menu
Trial record 35 of 103 for:    Pompe Disease

Pompe Disease Registry

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT00231400
Recruitment Status : Recruiting
First Posted : October 4, 2005
Last Update Posted : June 18, 2019
Sponsor:
Information provided by (Responsible Party):
Sanofi ( Genzyme, a Sanofi Company )

Brief Summary:

The Pompe Registry is an ongoing, international multi-center, strictly observational program that tracks the routine clinical outcomes for patients with Pompe disease, irrespective of treatment status. No experimental intervention is involved; patients in the Registry undergo clinical assessments and receive care as determined by the patient's treating physician.

The objectives of the Registry are:

  • To enhance the understanding of the variability, progression, and natural history of the key manifestations of Pompe disease;
  • To assist the Pompe medical community with the development of recommendations for monitoring patients and reports on patient outcomes to help optimize patient care;
  • To characterize and describe the Pompe disease population as a whole; and
  • To evaluate the long-term effectiveness and safety of available treatment options including ERT(Enzyme Replacement Therapy) with Myozyme®.

Condition or disease
Glycogen Storage Disease Type II Pompe Disease

Layout table for study information
Study Type : Observational
Estimated Enrollment : 2000 participants
Observational Model: Cohort
Time Perspective: Other
Official Title: Pompe Disease Registry
Actual Study Start Date : August 21, 2004
Estimated Primary Completion Date : January 31, 2034
Estimated Study Completion Date : January 31, 2034





Primary Outcome Measures :
  1. Understanding of the variability, progression , identification and natural history of the manifestations of Pompe disease [ Time Frame: 15 Years ]


Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Layout table for eligibility information
Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Patients diagnosed with Pompe disease
Criteria

Inclusion Criteria:

  • Patient must have a confirmed diagnosis of Pompe disease, documented by GAA(Glucosidase Alpha Acid) enzyme deficiency or GAA gene mutation

Exclusion Criteria:

  • There are no exclusion criteria

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00231400


Contacts
Layout table for location contacts
Contact: Trial Transparency email recommended (Toll free number for US & Canada) 800-633-1610 ext 1 then # Contact-Us@sanofi.com
Contact: Medical Information 617-252-7832 medinfo@genzyme.com

  Hide Study Locations
Locations
Layout table for location information
United States, Alabama
Investigational Site Number 840106 Recruiting
Birmingham, Alabama, United States, 35233
United States, Arkansas
Investigational Site Number 840109 Recruiting
Little Rock, Arkansas, United States, 72202
Investigational Site Number 840113 Recruiting
Little Rock, Arkansas, United States, 72205
United States, California
Investigational Site Number 840108 Recruiting
Los Angeles, California, United States, 90027
Investigational Site Number 840042 Recruiting
Sacramento, California, United States, 95825
United States, Connecticut
Investigational Site Number 840047 Recruiting
New Haven, Connecticut, United States, 06520
United States, District of Columbia
Investigational Site Number 840067 Recruiting
Washington, District of Columbia, United States, 20010
United States, Florida
Investigational Site Number 840012 Recruiting
Gainesville, Florida, United States, 32610
Investigational Site Number 840112 Recruiting
Jacksonville, Florida, United States, 32216
Investigational Site Number 840096 Recruiting
Tampa, Florida, United States, 33606
United States, Georgia
Investigational Site Number 840060 Recruiting
Decatur, Georgia, United States, 30033
United States, Illinois
Investigational Site Number 840011 Recruiting
Chicago, Illinois, United States, 60611
Investigational Site Number 840013 Recruiting
Chicago, Illinois, United States, 60611
Investigational Site Number 840079 Recruiting
Chicago, Illinois, United States, 60612
United States, Indiana
Investigational Site Number 840027 Recruiting
Indianapolis, Indiana, United States, 46202
United States, Kansas
Investigational Site Number 840020 Recruiting
Kansas City, Kansas, United States, 66160
United States, Kentucky
Investigational Site Number 840030 Recruiting
Louisville, Kentucky, United States, 40202
United States, Maine
Investigational Site Number 840064 Active, not recruiting
Portland, Maine, United States, 04102
United States, Maryland
Investigational Site Number 840044 Recruiting
Baltimore, Maryland, United States
United States, Massachusetts
Investigational Site Number 840062 Recruiting
Boston, Massachusetts, United States, 02114
Investigational Site Number 840092 Recruiting
Boston, Massachusetts, United States, 02115
Investigational Site Number 840002 Recruiting
Springfield, Massachusetts, United States, 01199
United States, Michigan
Investigational Site Number 840107 Recruiting
Ann Arbor, Michigan, United States, 48109
Investigational Site Number 840019 Recruiting
Grand Rapids, Michigan, United States, 49503
Investigational Site Number 840050 Recruiting
Grand Rapids, Michigan, United States, 49525
United States, Minnesota
Investigational Site Number 840046 Recruiting
Minneapolis, Minnesota, United States, 55404
Investigational Site Number 840114 Recruiting
Minneapolis, Minnesota, United States, 55404
United States, Missouri
Investigational Site Number 840031 Recruiting
Columibia, Missouri, United States, 65212
Investigational Site Number 840063 Completed
Kansas City, Missouri, United States, 64108
Investigational Site Number 840100 Recruiting
Saint Louis, Missouri, United States, 63110
United States, Montana
Investigational Site Number 840090 Recruiting
Helena, Montana, United States, 59601
United States, Nebraska
Investigational Site Number 840084 Recruiting
Omaha, Nebraska, United States, 68198
United States, New Jersey
Investigational Site Number 840098 Terminated
Camden, New Jersey, United States
Investigational Site Number 840101 Recruiting
Hackensack, New Jersey, United States, 07601
Investigational Site Number 840099 Recruiting
Morristown, New Jersey, United States, 07960
Investigational Site Number 840016 Recruiting
New Brunswick, New Jersey, United States, 08901
Investigational Site Number 840057 Recruiting
Paterson, New Jersey, United States, 07503
United States, New York
Investigational Site Number 840102 Recruiting
Manhasset, New York, United States, 11030
Investigational Site Number 840040 Recruiting
New York, New York, United States, 10016
Investigational Site Number 840005 Recruiting
New York, New York, United States, 10029
Investigational Site Number 840110 Recruiting
New York, New York, United States, 10029
Investigational Site Number 840105 Recruiting
Rochester, New York, United States, 14642
Investigational Site Number 840052 Recruiting
Syracuse, New York, United States, 13210
Investigational Site Number 840039 Recruiting
Valhalla, New York, United States, 10595
United States, North Carolina
Investigational Site Number 840065 Recruiting
Charlotte, North Carolina, United States, 28232
Investigational Site Number 840037 Recruiting
Durham, North Carolina, United States, 27710
United States, Ohio
Investigational Site Number 840041 Recruiting
Cincinnati, Ohio, United States
Investigational Site Number 840048 Recruiting
Cleveland, Ohio, United States, 44195
Investigational Site Number 840094 Recruiting
Dublin, Ohio, United States, 43017
United States, Oregon
Investigational Site Number 840080 Recruiting
Portland, Oregon, United States, 97201
United States, Pennsylvania
Investigational Site Number 840061 Recruiting
Hershey, Pennsylvania, United States, 17033
Investigational Site Number 840034 Recruiting
Philadelphia, Pennsylvania, United States
Investigational Site Number 840014 Recruiting
Pittsburgh, Pennsylvania, United States, 15213
Investigational Site Number 840023 Recruiting
Pittsburgh, Pennsylvania, United States, 15224
United States, Rhode Island
Investigational Site Number 840053 Recruiting
Providence, Rhode Island, United States, 02903
United States, South Carolina
Investigational Site Number 840055 Recruiting
Greenville, South Carolina, United States, 29605
United States, Tennessee
Investigational Site Number 840049 Recruiting
Nashville, Tennessee, United States, 38232
United States, Texas
Investigational Site Number 840058 Recruiting
Dallas, Texas, United States, 75226
Investigational Site Number 840024 Recruiting
Fort Worth, Texas, United States, 76104
United States, Virginia
Investigational Site Number 840078 Recruiting
Charlottesville, Virginia, United States, 22908
Investigational Site Number 840025 Recruiting
Fairfax, Virginia, United States, 22030
Investigational Site Number 840072 Recruiting
Norfolk, Virginia, United States, 23507
United States, Washington
Investigational Site Number 840115 Recruiting
Tacoma, Washington, United States, 98415
United States, Wisconsin
Investigational Site Number 840054 Recruiting
Milwaukee, Wisconsin, United States, 53226
Argentina
Investigational Site Number 152218 Recruiting
Caba, Argentina, 1424
Investigational Site Number 032004 Recruiting
Caba, Argentina, CPC1181ACH
Investigational Site Number 153194 Recruiting
Caba, Argentina, CPC1181ACH
Investigational Site Number 153032 Recruiting
Ciudad Autónoma de Buenos Aires, Argentina, 1425
Australia
Investigational Site Number 151002 Recruiting
Adelaide, Australia, 5000
Investigational Site Number 152096 Terminated
Brisbane, Australia, 4101
Investigational Site Number 154087 Active, not recruiting
Herston, Australia, 4006
Investigational Site Number 036014 Recruiting
Murdoch, Australia, 6150
Investigational Site Number 036013 Recruiting
Westmead, Australia, 2145
Investigational Site Number 151069 Recruiting
Westmead, Australia, 2145
Belgium
Investigational Site Number 150722 Recruiting
Antwerpen, Belgium, 2020
Investigational Site Number 150684 Recruiting
Bruxelles, Belgium, 1070
Investigational Site Number 152072 Recruiting
Edegem, Belgium, 2650
Investigational Site Number 152295 Recruiting
Gent, Belgium, 9000
Investigational Site Number 153179 Recruiting
Gosselies, Belgium, 6041
Investigational Site Number 151668 Recruiting
Leuven, Belgium, 3000
Investigational Site Number 152126 Recruiting
Leuven, Belgium, 3000
Brazil
Investigational Site Number 076011 Recruiting
Fortaleza, Brazil, 60430-370
Investigational Site Number 076008 Recruiting
Porto Alegre, Brazil, 90035-003
Investigational Site Number 076009 Recruiting
Ribeirão Preto, Brazil, 14048-900
Investigational Site Number 076001 Recruiting
Rio de Janeiro, Brazil, 22250020
Investigational Site Number 076002 Recruiting
São Paulo, Brazil, 04020-041
Investigational Site Number 076004 Recruiting
São Paulo, Brazil, 05403-000
Investigational Site Number 076007 Recruiting
São Paulo, Brazil, 05403-000
Canada
Investigational Site Number 124020 Terminated
Halifax, Canada, B3K6R8
Investigational Site Number 124-022 Recruiting
Moncton, Canada, E1C2Z3
Investigational Site Number 124004 Active, not recruiting
Montreal, Canada, H3A2B4
Chile
Investigational Site Number 152002 Recruiting
Punta Arenas, Chile
Investigational Site Number 152001 Recruiting
Santiago, Chile
China
Investigational Site Number 153202 Recruiting
Beijing, China
Investigational Site Number 153203 Recruiting
Beijing, China
Colombia
Investigational Site Number 170001 Recruiting
Bogota, Colombia
Denmark
Investigational Site Number 151229 Recruiting
København Ø, Denmark, 2100
France
Investigational Site Number 250010 Recruiting
Bordeaux, France, 33000
Investigational Site Number 250012 Recruiting
Brest, France, 29200
Investigational Site Number 250003 Recruiting
BRON Cedex, France, 69677
Investigational Site Number 250007 Recruiting
BRON Cedex, France, 69677
Investigational Site Number 250009 Recruiting
Caen, France, 14000
Investigational Site Number 250015 Recruiting
GRENOBLE cedex, France, 38043
Investigational Site Number 250008 Recruiting
Lille Cedex, France, 59037
Investigational Site Number 250002 Recruiting
Marseille Cedex 05, France, 13385
Investigational Site Number 250017 Recruiting
NANCY Cedex, France, 54035
Investigational Site Number 250005 Recruiting
Nice cedex 1, France, 06001
Investigational Site Number 250011 Recruiting
NIMES Cedex 9, France, 30029
Investigational Site Number 250001 Recruiting
PARIS Cedex 13, France, 75013
Investigational Site Number 250004 Recruiting
Paris, France, 75015
Investigational Site Number 250018 Recruiting
St Etienne, France, 42055
Investigational Site Number 250016 Recruiting
Strasbourg Cedex 2, France, 67098
Investigational Site Number 250014 Recruiting
Toulouse, France, 31200
Investigational Site Number 250006 Recruiting
Tours, France, 37044
Investigational Site Number 25013 Recruiting
Vannes, France, 56000
Germany
Investigational Site Number 154150 Recruiting
Gießen, Germany, 35392
Investigational Site Number 154215 Recruiting
Hamburg, Germany, 20354
Greece
Investigational Site Number 300001 Recruiting
Thessaloniki, Greece, 54642
Hong Kong
Investigational Site Number 344001 Recruiting
Kowloon, Hong Kong
Hungary
Investigational Site Number 154256 Recruiting
Nyíregyháza, Hungary, 4400
Investigational Site Number 154166 Recruiting
Székesfehérvár, Hungary, 8000
India
Investigational Site Number 356001 Recruiting
Kolkata, India, 700017
Italy
Investigational Site Number 380008 Recruiting
Brescia, Italy, 25123
Investigational Site Number 380006 Recruiting
Cagliari, Italy, 09126
Investigational Site Number 380014 Recruiting
Ferrara, Italy, 44124
Investigational Site Number 380005 Recruiting
Firenze, Italy, 50141
Investigational Site Number 152013 Recruiting
Genova, Italy, 16147
Investigational Site Number 380004 Recruiting
Genova, Italy, 16147
Investigational Site Number 152191 Recruiting
Messina, Italy, 98125
Investigational Site Number 380013 Recruiting
Messina, Italy, 98125
Investigational Site Number 380016 Recruiting
Milano, Italy, 20122
Investigational Site Number 380007 Recruiting
Milano, Italy, 20133
Investigational Site Number 380009 Recruiting
Monza, Italy
Investigational Site Number 380010 Recruiting
Napoli, Italy
Investigational Site Number 380019 Recruiting
Napoli, Italy
Investigational Site Number 380002 Recruiting
Padova, Italy, 35128
Investigational Site Number 380011 Recruiting
Padova, Italy
Investigational Site Number 380003 Recruiting
Pavia, Italy, 27100
Investigational Site Number 380015 Recruiting
Roma, Italy, 00165
Investigational Site Number 380012 Recruiting
Roma, Italy
Investigational Site Number 380017 Recruiting
Torino, Italy, 10126
Investigational Site Number 380001 Recruiting
Udine, Italy, 33100
Japan
Investigational Site Number 392105 Recruiting
Ginowan, Japan
Investigational Site Number 392106 Recruiting
Sakyo-ku, Japan
Korea, Republic of
Investigational Site Number 153178 Recruiting
Busan, Korea, Republic of
Investigational Site Number 153191 Recruiting
Daejeon, Korea, Republic of
Investigational Site Number 153228 Completed
Jeju-si, Korea, Republic of
Investigational Site Number 153225 Recruiting
Jeonnam, Korea, Republic of
Investigational Site Number 151149 Recruiting
Seoul, Korea, Republic of
Investigational Site Number 153176 Recruiting
Seoul, Korea, Republic of
Investigational Site Number 153180 Recruiting
Seoul, Korea, Republic of
Investigational Site Number 153188 Recruiting
Seoul, Korea, Republic of
Investigational Site Number 153192 Recruiting
Seoul, Korea, Republic of
Investigational Site Number 153196 Recruiting
Seoul, Korea, Republic of
Investigational Site Number 153212 Recruiting
Seoul, Korea, Republic of
Investigational Site Number 153222 Recruiting
Seoul, Korea, Republic of
Investigational Site Number 154208 Terminated
Yangsan, Korea, Republic of, 50612
Kuwait
Investigational Site Number 154225 Recruiting
Kuwait, Kuwait
Malaysia
Investigational Site Number 458001 Recruiting
Jalan Pahang, Malaysia, 50586
Investigational Site Number 458002 Recruiting
Kuala Lumpur, Malaysia, 59100
Netherlands
Investigational Site Number 152029 Recruiting
Rotterdam, Netherlands, 3015GD
Philippines
Investigational Site Number 608001 Recruiting
Manila, Philippines, 1000
Poland
Investigational Site Number 150575 Recruiting
Warszawa, Poland, 04-730
Portugal
Investigational Site Number 620008 Recruiting
Coimbra, Portugal, 3000-075
Investigational Site Number 620006 Recruiting
Lisboa, Portugal, 1649-035
Russian Federation
Investigational Site Number 643002 Recruiting
Moscow, Russian Federation, 117218
Investigational Site Number 643001 Recruiting
Moscow, Russian Federation, 119991
Serbia
Investigational Site Number 6880001 Recruiting
Belgrade, Serbia, 11000
Singapore
Investigational Site Number 702001 Recruiting
Singapore, Singapore, 229899
Slovakia
Investigational Site Number 703001 Recruiting
Bratislava, Slovakia, 83340
Taiwan
Investigational Site Number 154093 Recruiting
Chiayi, Taiwan
Investigational Site Number 152152 Recruiting
Hualien, Taiwan
Investigational Site Number 152246 Recruiting
Kaohsiung Hsien,, Taiwan
Investigational Site Number 152244 Recruiting
Kaohsiung, Taiwan, 807
Investigational Site Number 151534 Recruiting
New Taipei City, Taiwan, 23142
Investigational Site Number 151500 Recruiting
Putz City, Chia-yi, Taiwan
Investigational Site Number 151179 Recruiting
Taichung City, Taiwan, 404
Investigational Site Number 151181 Recruiting
Taichung, Taiwan, 00407
Investigational Site Number 152153 Recruiting
Tainan, Taiwan
Investigational Site Number 153248 Recruiting
Tainan, Taiwan
Investigational Site Number 151182 Recruiting
Taipei, Taiwan, 100
Investigational Site Number 152321 Recruiting
Taipei, Taiwan, 10449
Investigational Site Number 152226 Recruiting
Taipei, Taiwan
Investigational Site Number 153158 Recruiting
Tao Yuan County, Taiwan
Thailand
Investigational Site Number 764002 Completed
Bangkok, Thailand, 10400
Investigational Site Number 764003 Recruiting
Bangkok, Thailand, 10400
Investigational Site Number 764001 Recruiting
Bangkok, Thailand
Investigational Site Number 764004 Recruiting
Bangkok, Thailand
United Arab Emirates
investigational site number TBC Recruiting
Dubai, United Arab Emirates
United Kingdom
Investigational Site Number 1232456 Recruiting
Birmingham, United Kingdom, B46NH
Sponsors and Collaborators
Genzyme, a Sanofi Company
Investigators
Layout table for investigator information
Study Director: Medical Monitor Genzyme, a Sanofi Company

Publications:
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Layout table for additonal information
Responsible Party: Genzyme, a Sanofi Company
ClinicalTrials.gov Identifier: NCT00231400     History of Changes
Other Study ID Numbers: Pompe Registry
DIREGC07005 ( Other Identifier: Sanofi )
First Posted: October 4, 2005    Key Record Dates
Last Update Posted: June 18, 2019
Last Verified: June 2019

Keywords provided by Sanofi ( Genzyme, a Sanofi Company ):
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:
Layout table for MeSH terms
Glycogen Storage Disease Type II
Glycogen Storage Disease
Lysosomal Storage Diseases, Nervous System
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Lysosomal Storage Diseases
Metabolic Diseases
Metabolism, Inborn Errors
Carbohydrate Metabolism, Inborn Errors