International Collaborative Gaucher Group (ICGG) Gaucher Disease Registry & Pregnancy Sub-registry
May 5, 2023 updated by: Genzyme, a Sanofi Company
Gaucher Disease Registry Protocol
The ICGG Gaucher Registry is an ongoing, international multi-center, strictly observational program that tracks the routine clinical outcomes for patients with Gaucher 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 understanding of the variability, progression, identification, and natural history of Gaucher disease, with the ultimate goal of better guiding and assessing therapeutic intervention.
- To assist the Gaucher medical community with the development of recommendations for monitoring patients, and to provide reports on patient outcomes, to optimize patient care.
- To characterize the Gaucher disease population.
- To evaluate the long-term effectiveness of imiglucerase and of eliglustat.
Gaucher Pregnancy Sub-registry: The primary objective of this Sub-registry is to track pregnancy outcomes, including complications and infant growth, in all women with Gaucher disease during pregnancy, regardless of whether they receive disease-specific therapy. No experimental intervention is given; thus a patient will undergo clinical assessments and receive standard of care treatment as determined by the patient's physician.If a patient consents to this Sub-registry, information about the patient's medical and obstetric history, pregnancy, and birth will be collected, and, if a patient consents to data collection for her infant, data on infant growth through month 36 postpartum will be collected.
Study Overview
Status
Recruiting
Study Type
Observational
Enrollment (Anticipated)
12000
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Contact
- Name: Trial Transparency email recommended (Toll free number for US & Canada)
- Phone Number: 1 then # 800-633-1610
- Email: Contact-US@sanofi.com
Study Contact Backup
- Name: ICGG Gaucher Registry HelpLine
- Phone Number: +1(617) 252-7500
Study Locations
-
-
-
Buenos Aires, Argentina, 1425
- Recruiting
- Investigational Site Number :032012
-
Corrientes, Argentina, 3400
- Completed
- Investigational Site Number :154176
-
Córdoba, Argentina, X5004FHP
- Recruiting
- Investigational Site Number :032011
-
-
Buenos Aires
-
Caba, Buenos Aires, Argentina, 1425
- Recruiting
- Investigational Site Number :150661
-
La Plata, Buenos Aires, Argentina, 1900
- Recruiting
- Investigational Site Number :032003
-
Sarandi, Buenos Aires, Argentina, 1123
- Recruiting
- Investigational Site Number :151085
-
-
-
-
New South Wales
-
Camperdown, New South Wales, Australia, 2050
- Terminated
- Investigational Site Number :151431
-
Westmead, New South Wales, Australia, 2145
- Recruiting
- Investigational Site Number :151069
-
-
Queensland
-
Douglas, Queensland, Australia, 4814
- Recruiting
- Investigational Site Number :151491
-
Herston, Queensland, Australia, 4006
- Recruiting
- Investigational Site Number :151010
-
-
South Australia
-
Adelaide, South Australia, Australia, 5000
- Recruiting
- Investigational Site Number :151002
-
-
Tasmania
-
Hobart, Tasmania, Australia, 7000
- Recruiting
- Investigational Site Number :151725
-
-
Victoria
-
Clayton, Victoria, Australia, 3168
- Recruiting
- Investigational Site Number :151011
-
Parkville, Victoria, Australia, 3050
- Recruiting
- Investigational Site Number :151008
-
-
Western Australia
-
Murdoch, Western Australia, Australia, 6150
- Recruiting
- Investigational Site Number :151015
-
-
-
-
-
Antwerpen, Belgium, 2020
- Recruiting
- Investigational Site Number :150722
-
Brugge, Belgium, B-8000
- Terminated
- Investigational Site Number :151156
-
Brussel, Belgium, 1090
- Recruiting
- Investigational Site Number :150944
-
Brussel, Belgium, 1090
- Recruiting
- Investigational Site Number :152345
-
Bruxelles, Belgium, 1020
- Recruiting
- Investigational Site Number :151796
-
Bruxelles, Belgium, 1200
- Recruiting
- Investigational Site Number :150517
-
Edegem, Belgium, 2650
- Recruiting
- Investigational Site Number :154285
-
Gent, Belgium, 9000
- Completed
- Investigational Site Number :151134
-
Leuven, Belgium, 3000
- Recruiting
- Investigational Site Number :151668
-
-
-
-
-
Campinas, Brazil, 13083-220
- Recruiting
- Investigational Site Number :076005
-
Rio de Janeiro, Brazil, 20211030
- Recruiting
- Investigational Site Number :076006
-
São Paulo, Brazil, 04020-041
- Recruiting
- Investigational Site Number :076002
-
São Paulo, Brazil, 05403-000
- Recruiting
- Investigational Site Number :076008
-
-
Paraná
-
Curitiba, Paraná, Brazil, 80060-150
- Recruiting
- Investigational Site Number :076010
-
-
Rio Grande Do Sul
-
Porto Alegre, Rio Grande Do Sul, Brazil, 90035-003
- Recruiting
- Investigational Site Number :076007
-
-
São Paulo
-
Campinas, São Paulo, Brazil, 13059900
- Recruiting
- Investigational Site Number :076012
-
Ribeirao Preto, São Paulo, Brazil, 14048-900
- Recruiting
- Investigational Site Number :076001
-
-
-
-
-
Sofia, Bulgaria, 1680
- Recruiting
- Investigational Site Number :100001
-
Sofia, Bulgaria
- Recruiting
- Investigational Site Number :151164
-
-
-
-
Alberta
-
Calgary, Alberta, Canada, T2M 0L6
- Recruiting
- Investigational Site Number :124014
-
Edmonton, Alberta, Canada, TG62B7
- Recruiting
- Investigational Site Number :124002
-
-
British Columbia
-
Vancouver, British Columbia, Canada, V5Z1M9
- Recruiting
- Investigational Site Number :124010
-
Vancouver, British Columbia, Canada, V6H3N1
- Recruiting
- Investigational Site Number :124011
-
-
Manitoba
-
Winnipeg, Manitoba, Canada, R3A1R9
- Recruiting
- Investigational Site Number :124019
-
-
New Brunswick
-
Moncton, New Brunswick, Canada, E1C2Z3
- Recruiting
- Investigational Site Number :124022
-
-
Ontario
-
Kingston, Ontario, Canada, K7L 2V7
- Recruiting
- Investigational Site Number :124024
-
Kingston, Ontario, Canada, K7L2V7
- Terminated
- Investigational Site Number :124005
-
London, Ontario, Canada, N6A 5W9
- Recruiting
- Investigational Site Number :124021
-
Toronto, Ontario, Canada, M5G 1X8
- Recruiting
- Investigational Site Number :124006
-
Toronto, Ontario, Canada, M5G1X5
- Recruiting
- Investigational Site Number :124013
-
-
Quebec
-
Sherbrooke, Quebec, Canada, J1H5N4
- Recruiting
- Investigational Site Number :124001
-
-
-
-
-
Beijing, China, 100045
- Terminated
- Investigational Site Number :151787
-
Beijing, China
- Terminated
- Investigational Site Number :156003
-
Guangzhou, China, 510080
- Terminated
- Investigational Site Number :152357
-
Hangzhou, China
- Terminated
- Investigational Site Number :152358
-
Hangzhou, China
- Terminated
- Investigational Site Number :152359
-
Shanghai, China, 200092
- Terminated
- Investigational Site Number :152356
-
Shanghai, China
- Terminated
- Investigational Site Number :150899
-
-
-
-
-
Zagreb, Croatia, 10000
- Recruiting
- Investigational Site Number :1910001
-
Zagreb, Croatia, 10000
- Recruiting
- Investigational Site Number :1910002
-
Zagreb, Croatia, 10000
- Recruiting
- Investigational Site Number :1910003
-
-
-
-
-
Praha 2, Czechia, 12808
- Recruiting
- Investigational Site Number :150809
-
-
-
-
-
København Ø, Denmark, 2100
- Recruiting
- Investigational Site Number :151229
-
-
-
-
-
Santo Domingo, Dominican Republic, 10101
- Recruiting
- Investigational Site Number :2140001
-
-
-
-
-
Alexandria, Egypt
- Recruiting
- Investigational Site Number :5748
-
Ash Sharqia, Egypt
- Recruiting
- Investigational Site Number :9889
-
Assuit, Egypt
- Recruiting
- Investigational Site Number :9888
-
Cairo, Egypt
- Recruiting
- Investigational Site Number :8345
-
Cairo, Egypt
- Recruiting
- Investigational Site Number :9829
-
Cairo, Egypt
- Recruiting
- Investigational Site Number :9830
-
-
-
-
-
ANGERS Cedex 01, France, 49033
- Recruiting
- Investigational Site Number :250015
-
BRON Cedex, France, 69677
- Recruiting
- Investigational Site Number :250014
-
Besancon, France, 25030
- Recruiting
- Investigational Site Number :250004
-
Bordeaux, France, 33000
- Recruiting
- Investigational Site Number :250008
-
Brest, France, 29200
- Recruiting
- Investigational Site Number :250005
-
CLICHY Cedex, France, 92110
- Recruiting
- Investigational Site Number :250001
-
Caen, France, 14000
- Recruiting
- Investigational Site Number :250002
-
Garches, France, 92380
- Recruiting
- Investigational Site Number :250003
-
LYON Cedex 03, France, 69437
- Recruiting
- Investigational Site Number :250009
-
MARSEILLE cdx 08, France, 13285
- Terminated
- Investigational Site Number :250011
-
Montpellier, France, 34059
- Recruiting
- Investigational Site Number :2500013
-
NANTES Cedex 1, France, 44093
- Recruiting
- Investigational Site Number :250006
-
Paris, France, 75013
- Terminated
- Investigational Site Number :250007
-
Paris, France, 75015
- Recruiting
- Investigational Site Number :250010
-
Rennes Cedex 2, France, 35056
- Recruiting
- Investigational Site Number :250012
-
Strasbourg, France, 67091
- Recruiting
- Investigational Site Number :250018
-
TOURS Cedex 9, France, 37044
- Recruiting
- Investigational Site Number :250016
-
Toulouse, France, 31027
- Recruiting
- Investigational Site Number :250017
-
-
-
-
-
Berlin, Germany, 13353
- Recruiting
- Investigational Site Number :151686
-
Düsseldorf, Germany, 40225
- Recruiting
- Investigational Site Number :150783
-
Gießen, Germany, 35392
- Recruiting
- Investigational Site Number :154150
-
Hamburg, Germany, 20099
- Recruiting
- Investigational Site Number :151369
-
Hamburg, Germany, 20246
- Recruiting
- Investigational Site Number :151368
-
Mainz, Germany, 55131
- Recruiting
- Investigational Site Number :150031
-
Oberhausen, Germany, 46045
- Recruiting
- Investigational Site Number :150432
-
-
-
-
-
Athens, Greece, 11527
- Recruiting
- Investigational Site Number :300002
-
Athens, Greece
- Recruiting
- Investigational Site Number :300004
-
Patras, Greece, 26500
- Recruiting
- Investigational Site Number :300003
-
Thessaloniki, Greece, 54642
- Recruiting
- Investigational Site Number :300001
-
-
-
-
-
Budapest, Hungary, H-1097
- Recruiting
- Investigational Site Number :3480006
-
Létavértes, Hungary, 4281
- Recruiting
- Investigational Site Number :3480003
-
Nyíregyháza, Hungary, 4400
- Recruiting
- Investigational Site Number :3480007
-
Székesfehérvár, Hungary, 8000
- Recruiting
- Investigational Site Number :3480008
-
-
-
-
-
Ahmedabad, India, 380015
- Recruiting
- Investigational Site Number :356003
-
Jaipur, India, 302015
- Recruiting
- Investigational Site Number :356004
-
Kolkata, India, 700017
- Recruiting
- Investigational Site Number :356001
-
Mumbai, India, 400025
- Recruiting
- Investigational Site Number :356005
-
Secunderabad, India, 500026
- Recruiting
- Investigational Site Number :3560006
-
Vellore, India, 632004
- Recruiting
- Investigational Site Number :356002
-
-
-
-
-
Jakarta, Indonesia
- Recruiting
- Investigational Site Number :360001
-
-
-
-
-
Haifa, Israel
- Recruiting
- Investigational Site Number :3760003
-
Jerusalem, Israel
- Recruiting
- Investigational Site Number :3760001
-
Petach-Tikva, Israel
- Recruiting
- Investigational Site Number :3760002
-
-
-
-
-
Catania, Italy, 95100
- Recruiting
- Investigational Site Number :380008
-
Firenze, Italy, 50134
- Completed
- Investigational Site Number :380009
-
Genova, Italy, 16147
- Recruiting
- Investigational Site Number :380006
-
Milano, Italy, 20122
- Recruiting
- Investigational Site Number :380004
-
Modena, Italy, 41126
- Recruiting
- Investigational Site Number :380005
-
Monza, Italy
- Recruiting
- Investigational Site Number :380011
-
Monza (MB), Italy, 20900
- Recruiting
- Investigational Site Number :380002
-
Napoli, Italy
- Recruiting
- Investigational Site Number :380010
-
Padova, Italy, 35128
- Recruiting
- Investigational Site Number :380003
-
Roma, Italy, 00161
- Recruiting
- Investigational Site Number :380007
-
Udine, Italy
- Recruiting
- Investigational Site Number :380001
-
-
-
-
-
Chiba, Japan, 266-0007
- Recruiting
- Investigational Site Number :153217
-
-
Osaka
-
Abeno-ku, Osaka, Japan, 545-8585
- Completed
- Investigational Site Number :153205
-
-
Tokyo
-
Setagaya, Tokyo, Japan, 157-0074
- Recruiting
- Investigational Site Number :153204
-
-
Tottori
-
Yonago-shi, Tottori, Japan, 683-8504
- Recruiting
- Investigational Site Number :153203
-
-
-
-
-
Amman, Jordan, 261
- Recruiting
- Investigational Site Number :4000001
-
-
-
-
-
Daejeon, Korea, Republic of
- Recruiting
- Investigational Site Number :153191
-
Jeonnam, Korea, Republic of
- Recruiting
- Investigational Site Number :153225
-
Seoul, Korea, Republic of, 03080
- Recruiting
- Investigational Site Number :153180
-
Seoul, Korea, Republic of, 03080
- Recruiting
- Investigational Site Number :153196
-
Seoul, Korea, Republic of, 04401
- Recruiting
- Investigational Site Number :153192
-
Seoul, Korea, Republic of, 05505
- Recruiting
- Investigational Site Number :151149
-
Seoul, Korea, Republic of
- Recruiting
- Investigational Site Number :153212
-
Seoul, Korea, Republic of
- Recruiting
- Investigational Site Number :153222
-
Yangsan, Korea, Republic of, 50612
- Recruiting
- Investigational Site Number :154148
-
Yangsan, Korea, Republic of, 50612
- Terminated
- Investigational Site Number :154208
-
-
Gyeongsangnam-do
-
Yangsan-si, Gyeongsangnam-do, Korea, Republic of, 50612
- Recruiting
- Investigational Site Number :153178
-
-
-
-
-
Kuwait, Kuwait
- Recruiting
- Investigational Site Number :154225
-
-
-
-
-
Beirut, Lebanon
- Recruiting
- Investigational Site Number :4220004
-
Beirut, Lebanon
- Recruiting
- Investigational Site Number :422001
-
Douris, Lebanon
- Recruiting
- Investigational Site Number :422006
-
Hazmieh, Lebanon
- Recruiting
- Investigational Site Number :4220002
-
-
-
-
-
Vilnius, Lithuania
- Recruiting
- Investigational Site Number :154237
-
-
-
-
-
Jalan Pahang, Malaysia, 50586
- Recruiting
- Investigational Site Number :458001
-
Kuala Lumpur, Malaysia, 59100
- Recruiting
- Investigational Site Number :458002
-
-
-
-
-
Amsterdam, Netherlands
- Completed
- Investigational Site Number :150149
-
Amsterdam, Netherlands
- Terminated
- Investigational Site Number :151171
-
-
-
-
-
Karachi, Pakistan
- Recruiting
- Investigational Site Number :154264
-
Lahore, Pakistan
- Recruiting
- Investigational Site Number :154266
-
-
-
-
-
Arequipa, Peru
- Recruiting
- Investigational Site Number :604001
-
Callao, Peru, Callao02
- Recruiting
- Investigational Site Number :604002
-
-
-
-
-
Manila, Philippines, 1000
- Recruiting
- Investigational Site Number :608001
-
-
-
-
-
Warszawa, Poland, 04-730
- Recruiting
- Investigational Site Number :150575
-
-
-
-
-
Coimbra, Portugal
- Recruiting
- Investigational Site Number :620003
-
Lisboa, Portugal, 1649-035
- Recruiting
- Investigational Site Number :620004
-
Ponta Delgada, Portugal, 9500-370
- Recruiting
- Investigational Site Number :620009
-
-
-
-
-
Cluj Napoca, Romania, 400370
- Completed
- Investigational Site Number :151188
-
Cluj Napoca, Romania, 400370
- Recruiting
- Investigational Site Number :642002
-
Cluj-Napoca, Romania, 400006
- Recruiting
- Investigational Site Number :642001
-
-
-
-
-
Moscow, Russian Federation
- Recruiting
- Investigational Site Number :643001
-
sait-Petersburg, Russian Federation
- Recruiting
- Investigational Site Number :643002
-
-
-
-
-
Al Mubarraz, Saudi Arabia, 36424
- Recruiting
- Investigational Site Number :682002
-
Jeddah, Saudi Arabia
- Recruiting
- Investigational Site Number :682003
-
Makkah, Saudi Arabia, 21955
- Recruiting
- Investigational Site Number :682004
-
-
-
-
-
Belgrade, Serbia, 11000
- Recruiting
- Investigational Site Number :688001
-
Belgrade, Serbia, 11000
- Recruiting
- Investigational Site Number :688002
-
Belgrade, Serbia, 11000
- Recruiting
- Investigational Site Number :688003
-
Belgrade, Serbia, 11000
- Recruiting
- Investigational Site Number :688005
-
Nis, Serbia
- Recruiting
- Investigational Site Number :688004
-
-
-
-
-
Singapore, Singapore, 229899
- Recruiting
- Investigational Site Number :702001
-
-
-
-
-
Bratislava, Slovakia, 83340
- Recruiting
- Investigational Site Number :703001
-
-
-
-
-
Johannesburg, South Africa, 2000
- Recruiting
- Investigational Site Number :154138
-
Johannesburg, South Africa
- Recruiting
- Investigational Site Number :710-003
-
Sandton, South Africa, 2196
- Recruiting
- Investigational Site Number :150346
-
-
-
-
-
Barcelona, Spain, 08035
- Recruiting
- Investigational Site Number :153237
-
Barcelona, Spain, 08035
- Recruiting
- Investigational Site Number :154147
-
La Coruña, Spain, 15006
- Completed
- Investigational Site Number :154146
-
Madrid, Spain, 28007
- Recruiting
- Investigational Site Number :154163
-
Madrid, Spain, 28007
- Recruiting
- Investigational Site Number :154236
-
Madrid, Spain, 28034
- Recruiting
- Investigational Site Number :151524
-
Madrid, Spain, 28040
- Terminated
- Investigational Site Number :153251
-
Madrid, Spain, 28041
- Recruiting
- Investigational Site Number :154199
-
Madrid, Spain, 28046
- Recruiting
- Investigational Site Number :154160
-
Madrid, Spain, 28922
- Recruiting
- Investigational Site Number :154119
-
Sabadell, Spain, 08208
- Recruiting
- Investigational Site Number :154145
-
Santiago de Compostela, Spain, 15706
- Recruiting
- Investigational Site Number :154222
-
Sevilla, Spain, 41013
- Recruiting
- Investigational Site Number :153215
-
Sevilla, Spain, 41013
- Recruiting
- Investigational Site Number :153219
-
Valencia, Spain, 46009
- Recruiting
- Investigational Site Number :154205
-
Vigo, Spain, 36200
- Recruiting
- Investigational Site Number :153213
-
Zaragoza, Spain, 50006
- Recruiting
- Investigational Site Number :154238
-
Zaragoza, Spain, 50009
- Recruiting
- Investigational Site Number :153216
-
-
-
-
-
Borås, Sweden, 50182
- Recruiting
- Investigational Site Number :154095
-
Malmö, Sweden, 20502
- Active, not recruiting
- Investigational Site Number :151726
-
Norrköping, Sweden, 60182
- Recruiting
- Investigational Site Number :154092
-
-
-
-
-
Bern, Switzerland, 3010
- Recruiting
- Investigational Site Number :157045
-
-
-
-
-
Chiayi, Taiwan
- Recruiting
- Investigational Site Number :154093
-
Hualien, Taiwan
- Recruiting
- Investigational Site Number :152152
-
Kaohsiung, Taiwan, 807
- Recruiting
- Investigational Site Number :152244
-
Kaohsiung Hsien,, Taiwan
- Recruiting
- Investigational Site Number :152246
-
New Taipei City, Taiwan, 23142
- Recruiting
- Investigational Site Number :151534
-
Putz City, Chia-yi, Taiwan
- Recruiting
- Investigational Site Number :151500
-
Taichung, Taiwan, 00407
- Recruiting
- Investigational Site Number :151181
-
Taichung City, Taiwan, 404
- Recruiting
- Investigational Site Number :151179
-
Tainan, Taiwan, 70403
- Recruiting
- Investigational Site Number :153248
-
Tainan, Taiwan
- Recruiting
- Investigational Site Number :152153
-
Taipei, Taiwan, 100
- Recruiting
- Investigational Site Number :151182
-
Taipei, Taiwan, 10449
- Recruiting
- Investigational Site Number :152321
-
Taipei, Taiwan
- Recruiting
- Investigational Site Number :152226
-
Tao Yuan County, Taiwan
- Recruiting
- Investigational Site Number :153158
-
-
-
-
-
Bangkok, Thailand, 10400
- Recruiting
- Investigational Site Number :764003
-
Bangkok, Thailand, 10700
- Recruiting
- Investigational Site Number :764004
-
Bangkok, Thailand
- Recruiting
- Investigational Site Number :764001
-
Khon Kaen, Thailand, 40002
- Recruiting
- Investigational Site Number :764005
-
-
-
-
-
Adana, Turkey, 01330
- Recruiting
- Investigational Site Number :792006
-
Ankara, Turkey, 06100
- Recruiting
- Investigational Site Number :792005
-
Ankara, Turkey
- Recruiting
- Investigational Site Number :792001
-
Ankara, Turkey
- Recruiting
- Investigational Site Number :792002
-
Istanbul, Turkey
- Recruiting
- Investigational Site Number :792004
-
Izmir, Turkey
- Recruiting
- Investigational Site Number :792007
-
Malatya, Turkey
- Recruiting
- Investigational Site Number :792003
-
-
-
-
-
Dubai, United Arab Emirates
- Recruiting
- Investigational Site Number :7840001
-
-
-
-
-
Birmingham, United Kingdom, B152TH
- Recruiting
- Investigational Site Number :152287
-
Birmingham, United Kingdom, B46NH
- Recruiting
- Investigational Site Number :154240
-
London, United Kingdom, NW32QG
- Recruiting
- Investigational Site Number :151123
-
London, United Kingdom, WC1N3BG
- Recruiting
- Investigational Site Number :151646
-
London, United Kingdom, WC1N3JH
- Recruiting
- Investigational Site Number :150303
-
Manchester, United Kingdom, m139wl
- Recruiting
- Investigational Site Number :150455
-
Salford, United Kingdom, M6 8HD
- Recruiting
- Investigational Site Number :152361
-
-
Cambridgeshire
-
Cambridge, Cambridgeshire, United Kingdom, CB50QQ
- Recruiting
- Investigational Site Number :151340
-
-
-
-
Arizona
-
Phoenix, Arizona, United States, 85013
- Recruiting
- Phoenix Children's Hospital-Site Number:840003
-
Tucson, Arizona, United States, 85724
- Recruiting
- University of Arizona-Site Number:840015
-
-
Arkansas
-
Little Rock, Arkansas, United States, 72202
- Recruiting
- Arkansas Childrens Hospital-Site Number:840109
-
Little Rock, Arkansas, United States, 72205
- Recruiting
- University of Arkansas for Medical Sciences-Site Number:840113
-
-
California
-
Beverly Hills, California, United States, 90211
- Recruiting
- Tower Hematology/Oncology Medical Group-Site Number:840056
-
Irvine, California, United States
- Recruiting
- University of California at Irvine-Site Number:840036
-
Los Angeles, California, United States, 90027
- Recruiting
- Southern California Permanente Medical Group-Site Number:840108
-
Los Angeles, California, United States, 90033
- Recruiting
- USC Health Sciences Center Dept of Genetics-Site Number:840082
-
Orange, California, United States, 92868
- Recruiting
- Children's Hospital of Orange County-Site Number:840074
-
Palo Alto, California, United States, 94305
- Recruiting
- Stanford Unviersity MC Dept of Genetics-Site Number:840022
-
Sacramento, California, United States, 95817
- Recruiting
- UC Davis MIND Institute-Site Number:840010
-
Sacramento, California, United States, 95825
- Completed
- Kaiser Permanente Hospital-Site Number:840042
-
San Bernardino, California, United States, 92408
- Recruiting
- Loma Linda University Children's Hospital-Site Number:840117
-
San Diego, California, United States, 92093
- Completed
- University of California at San Diego-Site Number:840007
-
San Francisco, California, United States
- Recruiting
- University of California at San Francisco-Site Number:840051
-
-
Colorado
-
Aurora, Colorado, United States, 80045
- Recruiting
- University of Colorado at Denver Genetics-Site Number:840068
-
-
Connecticut
-
New Haven, Connecticut, United States, 06520
- Recruiting
- Yale-Site Number:840047
-
-
District of Columbia
-
Washington, District of Columbia, United States, 20010
- Recruiting
- Children's National Medical Center-Site Number:840067
-
-
Florida
-
Coral Springs, Florida, United States, 33065
- Recruiting
- University Hematology Oncology-Site Number:840075
-
Jacksonville, Florida, United States, 32207
- Recruiting
- University of Florida Pediatrics Genetics-Site Number:840121
-
Jacksonville, Florida, United States, 32216
- Recruiting
- The Atwal Clinic-Site Number:840112
-
Miami, Florida, United States, 33136
- Recruiting
- University Of Miami SOM-Site Number:840006
-
Tampa, Florida, United States, 33606
- Recruiting
- University of Florida-Genetics-Site Number:840096
-
Tampa, Florida, United States, 33612
- Recruiting
- Pediatric Endocrine Associates-Site Number:840017
-
-
Georgia
-
Decatur, Georgia, United States, 30033
- Recruiting
- Emory University School of Medicine- Human Genetics-Site Number:840060
-
-
Illinois
-
Chicago, Illinois, United States, 60611
- Recruiting
- Ann and Robert Lurie Children's Hospital-Site Number:840011
-
Chicago, Illinois, United States, 60611
- Recruiting
- Ann and Robert Lurie Children's Hospital-Site Number:840013
-
Chicago, Illinois, United States, 60612
- Recruiting
- Rush University Medical Center Genetics-Site Number:840079
-
-
Indiana
-
Indianapolis, Indiana, United States, 46202
- Recruiting
- Indianapolis University School of Medicine-Site Number:840027
-
-
Iowa
-
Iowa City, Iowa, United States, 52242
- Recruiting
- University of Iowa-Site Number:840032
-
-
Kentucky
-
Louisville, Kentucky, United States, 40202
- Recruiting
- University of Louisville- Genetics-Site Number:840030
-
-
Louisiana
-
New Orleans, Louisiana, United States, 70112
- Completed
- Tulane University Medical Center-Site Number:840001
-
New Orleans, Louisiana, United States, 70121
- Recruiting
- Ochsner Medical Center-Site Number:840120
-
-
Maine
-
Portland, Maine, United States, 04102
- Recruiting
- Maine Medical Center Pediatrics-Site Number:840064
-
-
Maryland
-
Baltimore, Maryland, United States
- Recruiting
- John Hopkins-Site Number:840044
-
-
Massachusetts
-
Boston, Massachusetts, United States, 02114
- Recruiting
- Massachusetts General Hospital-Genetics-Site Number:840062
-
Boston, Massachusetts, United States, 2115
- Recruiting
- Boston Children's Hospital-Site Number:840092
-
-
Michigan
-
Ann Arbor, Michigan, United States, 48109
- Recruiting
- University of Michigan Pediatrics-Site Number:840107
-
Detroit, Michigan, United States, 48201
- Recruiting
- Children's Hospital of Michigan-Site Number:840066
-
Grand Rapids, Michigan, United States, 49525
- Recruiting
- Infusion Associates-Site Number:840050
-
-
Minnesota
-
Minneapolis, Minnesota, United States, 55404
- Recruiting
- Children's Health Care-Site Number:840114
-
Minneapolis, Minnesota, United States, 55404
- Recruiting
- Children's Hospital and Clinics of Minnesota-Site Number:840046
-
Minneapolis, Minnesota, United States, 55455
- Recruiting
- University of Minnesota Medical Center Pediatrics-Site Number:840076
-
-
Missouri
-
Columibia, Missouri, United States, 65212
- Recruiting
- University of Missouri Health System Department of Genetics-Site Number:840031
-
Kansas City, Missouri, United States, 64108
- Completed
- Children's Mercy Hospital- Pediatrics-Site Number:840063
-
Saint Louis, Missouri, United States, 63110
- Recruiting
- Washington University of St. Louis-Site Number:840100
-
-
Nebraska
-
Omaha, Nebraska, United States, 68198
- Recruiting
- University of Nebraska Medical Center- Pediatrics-Site Number:840084
-
-
Nevada
-
Las Vegas, Nevada, United States, 89109
- Recruiting
- Children's Specialty Center of Nevada-Site Number:840008
-
-
New Jersey
-
Camden, New Jersey, United States
- Terminated
- Cooper Health Center-Site Number:840098
-
Hackensack, New Jersey, United States, 07601
- Recruiting
- Joseph M. Sanzari Children's Hospital-Site Number:840101
-
Morristown, New Jersey, United States, 07960
- Recruiting
- Atlantic Health System-Site Number:840099
-
New Brunswick, New Jersey, United States, 08901
- Recruiting
- St. Peter's University Hospital-Site Number:840016
-
Paterson, New Jersey, United States, 07503
- Recruiting
- St. Joseph's Children's Hospital-Site Number:840057
-
-
New York
-
Manhasset, New York, United States, 11030
- Recruiting
- Northwell Health-Site Number:840102
-
New York, New York, United States, 10016
- Recruiting
- New York University School Of Medicine-Site Number:840040
-
New York, New York, United States, 10029
- Recruiting
- Mt. Sinai School of Medicine-Site Number:840005
-
New York, New York, United States, 00000
- Recruiting
- -Site Number:840026
-
New York, New York, United States, 10029-7494
- Recruiting
- Metropolitan Hospital Center-Site Number:840110
-
Rochester, New York, United States, 14642
- Recruiting
- University of Rochester Medical Center SOM-Site Number:840105
-
Syracuse, New York, United States, 13210
- Recruiting
- State University of New York-Site Number:840052
-
Valhalla, New York, United States, 10595
- Recruiting
- New York Medical College-Site Number:840039
-
-
North Carolina
-
Charlotte, North Carolina, United States, 28232
- Recruiting
- Carolinas Medical Center Hospital-Site Number:840065
-
Durham, North Carolina, United States, 27710
- Recruiting
- Duke University Medical Center Genetics Dept-Site Number:840037
-
-
Ohio
-
Cleveland, Ohio, United States, 44106
- Recruiting
- UHCMC-Site Number:840119
-
Cleveland, Ohio, United States, 44195
- Recruiting
- Cleveland Clinic Foundation Pediatrics-Site Number:840048
-
Columbus, Ohio, United States, 43205-2664
- Recruiting
- Nationwide Children's Hospital-Site Number:840091
-
Dublin, Ohio, United States, 43017
- Recruiting
- LSD Data Registry Site LLC-Site Number:840094
-
-
Oregon
-
Portland, Oregon, United States, 97239
- Recruiting
- Oregon Health & Science University-Site Number:840080
-
-
Pennsylvania
-
Philadelphia, Pennsylvania, United States, 19104
- Recruiting
- Children's Hospital of Philadelphia HUP Medical Genetics-Site Number:840089
-
Philadelphia, Pennsylvania, United States
- Recruiting
- Children's Hospital of Philadelphia-Site Number:840034
-
Pittsburgh, Pennsylvania, United States, 15224
- Recruiting
- University of Pittsburgh-Site Number:840023
-
-
Rhode Island
-
Providence, Rhode Island, United States, 02903
- Recruiting
- Rhode Island Hospital Genetics-Site Number:840053
-
-
South Carolina
-
Greenville, South Carolina, United States, 29605
- Recruiting
- Greenwood Genetcs-Site Number:840055
-
-
Tennessee
-
Nashville, Tennessee, United States, 38232
- Recruiting
- Vanderbilt University Hospital-Pediatrics-Site Number:840049
-
-
Texas
-
Dallas, Texas, United States, 75226
- Recruiting
- Baylor Research Center-Site Number:840058
-
Fort Worth, Texas, United States, 76104
- Recruiting
- Cook Children's Health Care System-Site Number:840024
-
-
Utah
-
Salt Lake City, Utah, United States, 84132
- Recruiting
- University of Utah Department of Medical Genetics-Site Number:840043
-
-
Virginia
-
Charlottesville, Virginia, United States, 22908
- Recruiting
- University of Virginia School of Medicine-Pediatrics-Site Number:840078
-
Fairfax, Virginia, United States, 22030
- Recruiting
- O&O Alpan, LLC-Site Number:840025
-
Norfolk, Virginia, United States, 23507
- Recruiting
- Children's Hospital of the Kings Daughters-Site Number:840072
-
-
Washington
-
Seattle, Washington, United States, 98105
- Recruiting
- Seattle Children's Hospital-Site Number:840028
-
Seattle, Washington, United States, 98195
- Recruiting
- University Of Washington Medical Center-Site Number:840059
-
Tacoma, Washington, United States, 98415
- Recruiting
- Multicare Health System ¿ Mary Bridge Childrens Hospital-Site Number:840115
-
-
Wisconsin
-
Milwaukee, Wisconsin, United States, 53226
- Recruiting
- Children's Hospital of Wisconsin-Pediatrics-Site Number:840054
-
-
-
-
-
Hanoi, Vietnam
- Recruiting
- Investigational Site Number :154212
-
Ho Chi Minh City, Vietnam, 700000
- Recruiting
- Investigational Site Number :704002
-
-
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.
Eligibility Criteria
Ages Eligible for Study
- Child
- Adult
- Older Adult
Accepts Healthy Volunteers
No
Sampling Method
Non-Probability Sample
Study Population
All patients with a confirmed diagnosis of Gaucher disease are eligible for inclusion in the ICGG Gaucher Registry.
For Gaucher Pregnancy Sub-registry: It is recommended that pregnancy data be collected on all eligible women regardless of infant enrollment. In the event of patients having multiple pregnancies, data collection for each individual pregnancy is encouraged for all women enrolled in this Sub-registry.
Description
Inclusion Criteria:
ICGG Gaucher Registry
- All patients with a confirmed diagnosis of Gaucher disease are eligible for inclusion in the Registry. Confirmed diagnosis is defined as a documented β-glucocerebrosidase deficiency and/or mutation in the β-glucocerebrosidase gene.
- For all patients, appropriate patient authorization will be obtained.
Gaucher Pregnancy Sub-registry:
- be enrolled in the ICGG Gaucher Registry.
- be pregnant, or have been pregnant with appropriate medical documentation available.
- provide a signed informed consent and authorization form(s) to participate in the Sub-Registry prior to any Sub-Registry-related data collection being performed.
Exclusion Criteria:
- No exclusion criteria for participation in the ICGG Gaucher Registry and Sub-registry.
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Observational Models: Cohort
- Time Perspectives: Prospective
Cohorts and Interventions
Group / Cohort |
|---|
|
Patients in ICGG Gaucher Registry
No experimental intervention is given.
A patient with Gaucher Disease will undergo clinical assessments and receive standard of care treatment as determined by the patient's physician.
|
|
Pregnant women with confirmed diagnosis of Gaucher disease
No experimental intervention is given.
Pregnant women with confirmed diagnosis of Gaucher disease who are participating in the ICGG Gaucher Registry and consented to participate in the Gaucher Pregnancy Sub-registry, regardless of whether she is receiving disease-specific therapy and irrespective of the commercial product with which she may be treated.
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Time Frame |
|---|---|
|
ICGG Gaucher Registry: To provide the Gaucher medical community with recommendations for monitoring patients and to provide reports on patient outcomes to help optimize patient care
Time Frame: 42 years
|
42 years
|
|
Gaucher Pregnancy Sub-registry: To track pregnancy outcomes, including complications and infant growth, in all women with Gaucher disease during pregnancy, regardless of whether they receive disease-specific therapy, such as ERT with imiglucerase
Time Frame: 42 years
|
42 years
|
Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Investigators
- Study Director: Study Director, Genzyme, a Sanofi Company
Publications and helpful links
The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.
General Publications
- El-Beshlawy A, Tylki-Szymanska A, Vellodi A, Belmatoug N, Grabowski GA, Kolodny EH, Batista JL, Cox GF, Mistry PK. Long-term hematological, visceral, and growth outcomes in children with Gaucher disease type 3 treated with imiglucerase in the International Collaborative Gaucher Group Gaucher Registry. Mol Genet Metab. 2017 Jan-Feb;120(1-2):47-56. doi: 10.1016/j.ymgme.2016.12.001. Epub 2016 Dec 6.
- Mistry PK, Batista JL, Andersson HC, Balwani M, Burrow TA, Charrow J, Kaplan P, Khan A, Kishnani PS, Kolodny EH, Rosenbloom B, Scott CR, Weinreb N. Transformation in pretreatment manifestations of Gaucher disease type 1 during two decades of alglucerase/imiglucerase enzyme replacement therapy in the International Collaborative Gaucher Group (ICGG) Gaucher Registry. Am J Hematol. 2017 Sep;92(9):929-939. doi: 10.1002/ajh.24801. Epub 2017 Jul 7.
- Weinreb NJ, Goldblatt J, Villalobos J, Charrow J, Cole JA, Kerstenetzky M, vom Dahl S, Hollak C. Long-term clinical outcomes in type 1 Gaucher disease following 10 years of imiglucerase treatment. J Inherit Metab Dis. 2013 May;36(3):543-53. doi: 10.1007/s10545-012-9528-4. Epub 2012 Sep 14. Erratum In: J Inherit Metab Dis. 2014 Jan;37(1):147.
- Rosenbloom BE, Cappellini MD, Weinreb NJ, Dragosky M, Revel-Vilk S, Batista JL, Sekulic D, Mistry PK. Cancer risk and gammopathies in 2123 adults with Gaucher disease type 1 in the International Gaucher Group Gaucher Registry. Am J Hematol. 2022 Oct;97(10):1337-1347. doi: 10.1002/ajh.26675. Epub 2022 Aug 24.
- Weinreb NJ, Camelo JS Jr, Charrow J, McClain MR, Mistry P, Belmatoug N; International Collaborative Gaucher Group (ICGG) Gaucher Registry (NCT00358943) investigators. Gaucher disease type 1 patients from the ICGG Gaucher Registry sustain initial clinical improvements during twenty years of imiglucerase treatment. Mol Genet Metab. 2021 Feb;132(2):100-111. doi: 10.1016/j.ymgme.2020.12.295. Epub 2021 Jan 8.
- Weinreb NJ, Kaplan P. The history and accomplishments of the ICGG Gaucher registry. Am J Hematol. 2015 Jul;90 Suppl 1:S2-5. doi: 10.1002/ajh.24054. No abstract available.
- Buyers RP, Collin-Histed T. Foreword. Am J Hematol. 2015 Jul;90 Suppl 1:S1. doi: 10.1002/ajh.24053. No abstract available.
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 Major Dates
Study Start (Actual)
April 1, 1991
Primary Completion (Anticipated)
January 31, 2034
Study Completion (Anticipated)
January 31, 2034
Study Registration Dates
First Submitted
July 12, 2006
First Submitted That Met QC Criteria
July 31, 2006
First Posted (Estimate)
August 1, 2006
Study Record Updates
Last Update Posted (Actual)
May 8, 2023
Last Update Submitted That Met QC Criteria
May 5, 2023
Last Verified
May 5, 2023
More Information
Terms related to this study
Additional Relevant MeSH Terms
- Metabolic Diseases
- Brain Diseases
- Central Nervous System Diseases
- Nervous System Diseases
- Nutrition Disorders
- Genetic Diseases, Inborn
- Malnutrition
- Metabolism, Inborn Errors
- Lysosomal Storage Diseases
- Lipid Metabolism Disorders
- Brain Diseases, Metabolic
- Brain Diseases, Metabolic, Inborn
- Sphingolipidoses
- Lysosomal Storage Diseases, Nervous System
- Lipid Metabolism, Inborn Errors
- Deficiency Diseases
- Gaucher Disease
- Lipidoses
Other Study ID Numbers
- DIREGC07009
- ICGG Gaucher Registry (Other Identifier: Sanofi)
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
Clinical Trials on Gaucher Disease
-
KemPharm Denmark A/STerminatedGaucher Disease, Type 1 | Gaucher Disease, Type 3India
-
AVROBIOWithdrawn
-
Amicus TherapeuticsCompletedGaucher Disease | Gaucher Disease, Type 1 | Type 1 Gaucher DiseaseUnited Kingdom, United States
-
Genzyme, a Sanofi CompanyActive, not recruitingGaucher Disease Type 1 | Gaucher Disease Type 3Germany, United States, Japan, United Kingdom
-
Baylor Research InstituteTexas Scottish Rite Hospital for ChildrenWithdrawnGaucher Disease Type 1 | Gaucher Disease Type 3United States
-
CANbridge (Suzhou) Bio-pharma Co., Ltd.RecruitingGaucher Disease, Type 1 | Gaucher Disease, Type 3China
-
Cambridge University Hospitals NHS Foundation TrustMedical Research Council; National Institute for Health Research, United KingdomRecruitingGaucher Disease, Type III | Gaucher Disease, Type IUnited Kingdom
-
Amicus TherapeuticsCompletedGaucher Disease | Gaucher Disease, Type 1 | Type 1 Gaucher DiseaseUnited States
-
Amicus TherapeuticsCompletedGaucher Disease | Gaucher Disease, Type 1 | Type 1 Gaucher DiseaseUnited Kingdom, Israel, South Africa, United States
-
Freeline TherapeuticsRecruitingGaucher Disease, Type 1Spain, Israel, United States, United Kingdom, Brazil, Germany, Paraguay