Gaucher DiseaseNCT00358943
International Collaborative Gaucher Group (ICGG) Gaucher Disease Registry & Pregnancy Sub-registry
An observational study of Gaucher Disease and Cerebroside Lipidosis Syndrome, run by Genzyme, a Sanofi Company at 317 sites in 52 countries.
Trial summary
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. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Gaucher Disease, Cerebroside Lipidosis Syndrome, Glucocerebrosidase Deficiency Disease
- How many people
- 12,000 participants
- Where
- 317 sitesin 52 countries
- Study dates
- 1 Apr 1991to 31 Jan 2034
- How long will I take part?
- 12 Months
- Phase
- Observational
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
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.
Who may be able to join
- 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.
Who cannot join
- No exclusion criteria for participation in the ICGG Gaucher Registry and Sub-registry.
Trial locations and contacts
317 sites in 52 countries. Contact the study team or a site near you to ask about taking part.
Trial Transparency email recommended (Toll free number for US & Canada) contact
ICGG Gaucher Registry HelpLine contact
317 sites in 52 countries
United States86 sites, 66 recruiting
- Recruiting
University Of Michigan Pediatrics- Site Number : 840107
Ann Arbor, Michigan, 48109
- Recruiting
University Of Colorado At Denver Genetics- Site Number : 840068
Aurora, Colorado, 80045
- Completed
John Hopkins- Site Number : 840044
Baltimore, Maryland, 21205
- Recruiting
Tower Hematology Oncology Medical Group- Site Number : 840056
Beverly Hills, California, 90211
- Recruiting
Massachusetts General Hospital-Genetics- Site Number : 840062
Boston, Massachusetts, 02114
- Recruiting
Boston Children's Hospital- Site Number : 840092
Boston, Massachusetts, 02115
- Terminated
Cooper Health Center- Site Number : 840098
Camden, New Jersey, 08103
- Recruiting
Atrium Health Carolinas Medical Center Hospital- Site Number : 840065
Charlotte, North Carolina, 28203
- Completed
University Of Virginia School Of Medicine-Pediatrics- Site Number : 840078
Charlottesville, Virginia, 22908
- Recruiting
Ann & Robert H. Lurie Children's Hospital of Chicago- Site Number : 840011
Chicago, Illinois, 60611
- Recruiting
Ann & Robert H. Lurie Children's Hospital of Chicago- Site Number : 840013
Chicago, Illinois, 60611
- Recruiting
Rush University Medical Center Genetics- Site Number : 840079
Chicago, Illinois, 60612
- Recruiting
Cincinnati Children's Hospital Medical Center- Site Number : 840026
Cincinnati, Ohio, 5229
- Recruiting
University Hospitals Cleveland Medical Center- Site Number : 840119
Cleveland, Ohio, 44106
- Recruiting
Cleveland Clinic - Cleveland- Site Number : 840048
Cleveland, Ohio, 44195
- Recruiting
University Of Missouri Health System Department Of Genetics- Site Number : 840031
Columbia, Missouri, 65212
- Recruiting
Nationwide Children's Hospital- Site Number : 840091
Columbus, Ohio, 43205
- Recruiting
University of Miami- Site Number : 840006
Coral Gables, Florida, 33124
- Recruiting
University of Miami Sylvester Comprehensive Cancer Center - Coral Springs- Site Number : 840075
Coral Springs, Florida, 33065
- Completed
Baylor Institute of Metabolic Disease- Site Number : 840058
Dallas, Texas, 75226
- Recruiting
Emory University School Of Medicine- Human Genetics- Site Number : 840060
Decatur, Georgia, 30033
- Recruiting
Children's Hospital of Michigan- Site Number : 840066
Detroit, Michigan, 48201
- Recruiting
Lsd Data Registry Site Llc- Site Number : 840094
Dublin, Ohio, 43017
- Recruiting
Duke University Medical Center Genetics Dept- Site Number : 840037
Durham, North Carolina, 27710
- Recruiting
O & O Alpan- Site Number : 840025
Fairfax, Virginia, 22030
- Completed
Cook Children's Medical Center- Site Number : 840024
Fort Worth, Texas, 76104
- Completed
Corewell Health - Helen DeVos Children's Hospital (HDVCH)- Site Number : 840019
Grand Rapids, Michigan, 49503
- Completed
Infusion Associates - Grand Rapids- Site Number : 840050
Grand Rapids, Michigan, 49525
- Recruiting
Greenwood Genetic Center - Greenville- Site Number : 840055
Greenville, South Carolina, 29605
- Recruiting
Joseph M. Sanzari Children'S Hospital- Site Number : 840101
Hackensack, New Jersey, 07601
- Recruiting
Penn State Health Milton South Hershey Medical Center- Site Number : 840126
Hershey, Pennsylvania, 17033
- Recruiting
NYU Langone Health - Perlmutter Cancer Center - Huntington- Site Number : 840040
Huntington, New York, 11743
- Recruiting
Indianapolis University School Of Medicine- Site Number : 840027
Indianapolis, Indiana, 46202
- Recruiting
University of Iowa- Site Number : 840032
Iowa City, Iowa, 52242
- Recruiting
University Of Florida Pediatrics Genetics- Site Number : 840121
Jacksonville, Florida, 32207
- Completed
The Atwal Clinic- Site Number : 840112
Jacksonville, Florida, 32216
- Completed
Children's Mercy Adelle Hall Campus- Site Number : 840063
Kansas City, Missouri, 64108
- Completed
Children's Specialty Center - Las Vegas- Site Number : 840008
Las Vegas, Nevada, 89109
- Recruiting
Arkansas Children's Hospital- Site Number : 840109
Little Rock, Arkansas, 72202
- Recruiting
University of Arkansas for Medical Sciences- Site Number : 840113
Little Rock, Arkansas, 72205
- Recruiting
Southern California Permanente Medical Group- Site Number : 840108
Los Angeles, California, 90027
- Completed
Usc Health Sciences Center Dept Of Genetics- Site Number : 840082
Los Angeles, California, 90033
- Completed
University of Louisville - Health Sciences Center- Site Number : 840030
Louisville, Kentucky, 40202
- Recruiting
Children's Wisconsin- Site Number : 840054
Milwaukee, Wisconsin, 53226
- Recruiting
Children's Hospitals and Clinics of Minnesota- Site Number : 840046
Minneapolis, Minnesota, 55404
- Recruiting
Children's Hospitals and Clinics of Minnesota- Site Number : 840114
Minneapolis, Minnesota, 55404
- Recruiting
M Health Fairview University of Minnesota Medical Center - West Bank- Site Number : 840076
Minneapolis, Minnesota, 55454
- Recruiting
Atlantic Health System- Site Number : 840099
Morristown, New Jersey, 07962
- Recruiting
Vanderbilt University Medical Center - Medical Laboratories- Site Number : 840049
Nashville, Tennessee, 37228
- Recruiting
Saint Peter's University Hospital - New Brunswick- Site Number : 840016
New Brunswick, New Jersey, 08901
- Recruiting
Yale University School of Medicine- Site Number : 840047
New Haven, Connecticut, 06510
- Recruiting
Northwell Health- Site Number : 840102
New Hyde Park, New York, 11042
- Completed
Tulane University School of Medicine- Site Number : 840001
New Orleans, Louisiana, 70112
- Recruiting
Icahn School of Medicine at Mount Sinai- Site Number : 840005
New York, New York, 10029
- Recruiting
Metropolitan Hospital Center- Site Number : 840110
New York, New York, 10029
- Recruiting
Columbia University Irving Medical Center- Site Number : 840125
New York, New York, 10032
- Completed
Children's Hospital of The King's Daughters- Site Number : 840072
Norfolk, Virginia, 23507
- Recruiting
University of Nebraska Medical Center- Site Number : 840084
Omaha, Nebraska, 68198
- Recruiting
Children's Hospital of Orange County- Site Number : 840074
Orange, California, 92868
- Completed
University of California Irvine Medical Center- Site Number : 840036
Orange, California, 92868
- Recruiting
St. Joseph's Regional Medical Center - Paterson- Site Number : 840057
Paterson, New Jersey, 07503
- Recruiting
Children'S Hospital Of Philadelphia Hup Medical Genetics- Site Number : 840089
Philadelphia, Pennsylvania, 19104
- Recruiting
The Children's Hospital of Philadelphia- Site Number : 840034
Philadelphia, Pennsylvania, 19104
- Recruiting
Phoenix Children's Hospital- Site Number : 840003
Phoenix, Arizona, 85016
- Recruiting
UPMC Children's Hospital of Pittsburgh- Site Number : 840023
Pittsburgh, Pennsylvania, 15224
- Recruiting
Maine Medical Partners Pediatric Specialty Care- Site Number : 840064
Portland, Maine, 04102
- Recruiting
Oregon Health & Science University (OHSU)- Site Number : 840080
Portland, Oregon, 97239
- Completed
Rhode Island Hospital Genetics- Site Number : 840053
Providence, Rhode Island, 02903
- Recruiting
University of Rochester Medical Center- Site Number : 840105
Rochester, New York, 14642
- Recruiting
Uc Davis Mind Institute- Site Number : 840010
Sacramento, California, 95817
- Completed
Kaiser Permanente Sacramento Medical Center- Site Number : 840042
Sacramento, California, 95825
- Recruiting
University of Utah- Site Number : 840043
Salt Lake City, Utah, 84112
- Completed
University of California at San Diego- Site Number : 840007
San Diego, California, 92123
- Recruiting
University of California San Francisco - Parnassus Campus- Site Number : 840051
San Francisco, California, 94143
- Recruiting
Seattle Children's Hospital- Site Number : 840028
Seattle, Washington, 98105
- Completed
University of Washington Medical Center- Site Number : 840059
Seattle, Washington, 98195
- Recruiting
Washington University- Site Number : 840100
St Louis, Missouri, 63110
- Recruiting
Stanford University Medical Center- Site Number : 840022
Stanford, California, 94305
- Recruiting
SUNY Upstate Medical University - Site Number : 840052
Syracuse, New York, 13210
- Recruiting
Mary Bridge Children's Hospital & Health Center- Site Number : 840115
Tacoma, Washington, 98405
- Recruiting
University Of Florida-Genetics- Site Number : 840096
Tampa, Florida, 33606
- Completed
Pediatric Endocrine Association- Site Number : 840017
Tampa, Florida, 33607
- Recruiting
University of Arizona- Site Number : 840015
Tucson, Arizona, 85724
- Recruiting
New York Medical College- Site Number : 840039
Valhalla, New York, 10595
- Recruiting
Children's National Medical Center - Washington- Site Number : 840067
Washington D.C., District of Columbia, 20010
- Recruiting
Investigational Site Number : 250012
Washington D.C., District of Columbia, 20037-3201
Spain17 sites, 15 recruiting
- Completed
Investigational Site Number : 154146
A Coruña, Galicia [Galicia], 15006
- Recruiting
Investigational Site Number : 154119
Alcorcón, Madrid, 28922
- Recruiting
Investigational Site Number : 153237
Barcelona, Catalunya [Cataluña], 08035
- Recruiting
Investigational Site Number : 154147
Barcelona, Catalunya [Cataluña], 08035
- Recruiting
Investigational Site Number : 151524
Madrid, Madrid, Comunidad de, 28034
- Recruiting
Investigational Site Number : 154160
Madrid, Madrid, Comunidad de, 28046
- Recruiting
Investigational Site Number : 154163
Madrid, 28007
- Recruiting
Investigational Site Number : 154236
Madrid, 28007
- Terminated
Investigational Site Number : 153251
Madrid, 28040
- Recruiting
Investigational Site Number : 154199
Madrid, 28041
- Recruiting
Investigational Site Number : 154145
Sabadell, 08208
- Recruiting
Investigational Site Number : 153215
Seville, Andalusia, 41013
- Recruiting
Investigational Site Number : 153219
Seville, Andalusia, 41013
- Recruiting
Investigational Site Number : 154205
Valencia, 46026
- Recruiting
Investigational Site Number : 153213
Vigo, Pontevedra [Pontevedra], 36200
- Recruiting
Investigational Site Number : 154238
Zaragoza, 50006
- Recruiting
Investigational Site Number : 153216
Zaragoza, 50009
France16 sites, 8 recruiting
- Completed
Investigational Site Number : 250004
Besançon, 25000
- Recruiting
Investigational Site Number : 250008
Bordeaux, 33000
- Recruiting
Investigational Site Number : 250005
Brest, 29200
- Recruiting
Investigational Site Number : 250014
Bron, 69677
- Completed
Investigational Site Number : 250002
Caen, 14033
- Recruiting
Investigational Site Number : 250001
Clichy, 92110
- Recruiting
Investigational Site Number : 250003
Garches, 92380
- Completed
Investigational Site Number : 250009
Lyon, 69003
- Terminated
Investigational Site Number : 250011
Marseille, 13008
- Recruiting
Investigational Site Number : 2500013
Montpellier, 34295
- Completed
Investigational Site Number : 250006
Nantes, 44093
- Terminated
Investigational Site Number : 250007
Paris, 75013
- Recruiting
Investigational Site Number : 250010
Paris, 75015
- Recruiting
Investigational Site Number : 250018
Strasbourg, 67000
- Completed
Investigational Site Number : 250017
Toulouse, 31027
- Completed
Investigational Site Number : 250016
Tours, 37044
Taiwan14 sites, 13 recruiting
- Recruiting
Investigational Site Number : 154093
Chia-Yi City, 60002
- Recruiting
Investigational Site Number : 152152
Hualien City, 970
- Completed
Investigational Site Number : 152244
Kaohsiung City, 80756
- Recruiting
Investigational Site Number : 152246
Kaohsiung City, 833
- Recruiting
Investigational Site Number : 151534
New Taipei City, 231
- Recruiting
Investigational Site Number : 151500
Putz City, Chia-Yi
- Recruiting
Investigational Site Number : 151179
Taichung, 404
- Recruiting
Investigational Site Number : 151181
Taichung, 411
- Recruiting
Investigational Site Number : 153248
Tainan, 704
- Recruiting
Investigational Site Number : 152153
Tainan, 710
- Recruiting
Investigational Site Number : 151182
Taipei, 100
- Recruiting
Investigational Site Number : 152321
Taipei, 104
- Recruiting
Investigational Site Number : 152226
Taipei, 112
- Recruiting
Investigational Site Number : 153158
Taoyuan City, 333
Canada12 sites, 7 recruiting
- Recruiting
Investigational Site Number : 124014
Calgary, Alberta, T2E 7Z4
- Completed
Investigational Site Number : 124002
Edmonton, Alberta, T6G 2B7
- Completed
Investigational Site Number : 124024
Kingston, Ontario, K7L 2V7
- Terminated
Investigational Site Number : 124005
Kingston, Ontario, K7L 4X3
- Recruiting
Investigational Site Number : 124021
London, Ontario, N6A 5A5
- Completed
Investigational Site Number : 124022
Moncton, New Brunswick, E1C 2Z3
- Recruiting
Investigational Site Number : 124001
Sherbrooke, Quebec, J1K 2R1
- Recruiting
Investigational Site Number : 124013
Toronto, Ontario, M5G 1X5
- Recruiting
Investigational Site Number : 124006
Toronto, Ontario, M5G 1X8
- Recruiting
Investigational Site Number : 124010
Vancouver, British Columbia, V5Z 1M9
- Completed
Investigational Site Number : 124011
Vancouver, British Columbia, V6H 3N1
- Recruiting
Investigational Site Number : 124019
Winnipeg, Manitoba, R3A 1R9
Italy12 sites, 9 recruiting
- Recruiting
Investigational Site Number : 380005
Baggiovara, Modena, 41126
- Recruiting
Investigational Site Number : 380008
Catania, 95100
- Completed
Investigational Site Number : 380009
Florence, Firenze, 50134
- Recruiting
Investigational Site Number : 380006
Genoa, Genova, 16164
- Recruiting
Investigational Site Number : 380004
Milan, Milano, 20122
- Completed
Investigational Site Number : 380002
Monza, 20900
- Recruiting
Investigational Site Number : 380011
Monza
- Completed
Investigational Site Number : 380010
Naples
- Recruiting
Investigational Site Number : 380003
Padova, 35128
- Recruiting
Investigational Site Number : 380007
Rome, Roma, 00161
- Recruiting
Investigational Site Number : 380014
Sassari, Sardinia, 07100
- Recruiting
Investigational Site Number : 380001
Udine, 33100
China10 sites, 3 recruiting
- Recruiting
Investigational Site Number : 1560001
Beijing, 100045
- Terminated
Investigational Site Number : 156003
Beijing, 100730
- Terminated
Investigational Site Number : 152357
Guangzhou, 510080
- Recruiting
Investigational Site Number : 1560002
Guangzhou, 510080
- Terminated
Investigational Site Number : 152358
Hangzhou, 310003
- Terminated
Investigational Site Number : 152359
Hangzhou
- Recruiting
Investigational Site Number : 157252
Nanjing, 210008
- Terminated
Investigational Site Number : 151787
Shanghai, 200040
- Terminated
Investigational Site Number : 152356
Shanghai, 200092
- Terminated
Investigational Site Number : 150899
Shanghai, 200127
Australia9 sites, 6 recruiting
- Recruiting
Investigational Site Number : 151002
Adelaide, South Australia, 5000
- Completed
Investigational Site Number : 151010
Brisbane, Queensland, 4029
- Recruiting
Investigational Site Number : 151011
Clayton, Victoria, 3168
- Recruiting
Investigational Site Number : 151725
Hobart, Tasmania, 7000
- Recruiting
Investigational Site Number : 151015
Murdoch, Western Australia, 6150
- Recruiting
Investigational Site Number : 151008
Parkville, Victoria, 3050
- Terminated
Investigational Site Number : 151431
Sydney, New South Wales, 2050
- Completed
Investigational Site Number : 151491
Townsville, Queensland, 4814
- Recruiting
Investigational Site Number : 151069
Westmead, New South Wales, 2145
Belgium9 sites, 6 recruiting
- Recruiting
Investigational Site Number : 150722
Antwerp, 2020
- Terminated
Investigational Site Number : 151156
Bruges, 8000
- Recruiting
Investigational Site Number : 151796
Brussels, 1020
- Completed
Investigational Site Number : 150944
Brussels, 1090
- Recruiting
Investigational Site Number : 152345
Brussels, 1090
- Recruiting
Investigational Site Number : 150517
Brussels, 1200
- Recruiting
Investigational Site Number : 154285
Edegem, 2650
- Completed
Investigational Site Number : 151134
Ghent, 9000
- Recruiting
Investigational Site Number : 151668
Leuven, 3000
Argentina8 sites, 1 recruiting
- Completed
Investigational Site Number : 604002
Buenos Aires, 1107
- Completed
Investigational Site Number : 032012
Buenos Aires, 1425
- Recruiting
Investigational Site Number : 032014
Buenos Aires, 1428
- Completed
Investigational Site Number : 032011
Córdoba, 5004
- Completed
Investigational Site Number : 154176
Corrientes, 3400
- Completed
Investigational Site Number : 032003
La Plata, Buenos Aires, 1900
- Completed
Investigational Site Number : 150661
Munro, Buenos Aires, 1605
- Completed
Investigational Site Number : 151085
Sarandí, 1123
United Kingdom8 sites, 8 recruiting
- Recruiting
Investigational Site Number : 152287
Birmingham, England, B15 2TH
- Recruiting
Investigational Site Number : 154240
Birmingham, England, B4 6NH
- Recruiting
Investigational Site Number : 151340
Cambridge, Cambridgeshire, CB2 2QQ
- Recruiting
Investigational Site Number : 151123
London, England, NW3 2QG
- Recruiting
Investigational Site Number : 151646
London, London, City of, WC1N 3BG
- Recruiting
Investigational Site Number : 150303
London, London, City of, WC1N 3JH
- Recruiting
Investigational Site Number : 150455
Manchester, M13 9wl
- Recruiting
Investigational Site Number : 152361
Salford, Manchester, M6 8HD
Brazil7 sites, 6 recruiting
- Recruiting
Hospital de Clinicas da Universidade Federal do Parana- Site Number : 076010
Curitiba, Paraná, 80060-900
- Recruiting
Universidade Estadual de Ciencias da Saude de Alagoas- Site Number : 076005
Maceió, Alagoas, 57010-300
- Recruiting
Hospital de Clinicas de Porto Alegre- Site Number : 076007
Porto Alegre, Rio Grande do Sul, 90035903
- Recruiting
Hospital das Clinicas da Faculdade de Medicina de Ribeirao Preto- Site Number : 076001
Ribeirão Preto, São Paulo, 14049-900
- Recruiting
HEMORIO- Site Number : 076006
Rio de Janeiro, 20211-030
- Recruiting
Instituto de Genética e Erros Inatos do Metabolismo- Site Number : 076002
São Paulo, 04020-041
- Completed
Hospital das Clinicas da Faculdade de Medicina da Universidade de Sao Paulo- Site Number : 076008
São Paulo, 05403-000
Egypt7 sites, 7 recruiting
- Recruiting
Investigational Site Number : 818007
Al Mansurah
- Recruiting
Investigational Site Number : 5748
Alexandria
- Recruiting
Investigational Site Number : 9889
Ash Sharqia
- Recruiting
Investigational Site Number : 9888
Asyut, 2074020
- Recruiting
Investigational Site Number : 8345
Cairo, 11432
- Recruiting
Investigational Site Number : 9829
Cairo, 11566
- Recruiting
Investigational Site Number : 9830
Cairo
Germany7 sites, 5 recruiting
- Recruiting
Investigational Site Number : 151686
Berlin, 13353
- Recruiting
Investigational Site Number : 150783
Düsseldorf, 40225
- Recruiting
Investigational Site Number : 154150
Giessen, 35392
- Recruiting
Investigational Site Number : 151369
Hamburg, 20099
- Completed
Investigational Site Number : 151368
Hamburg, 20246
- Recruiting
Investigational Site Number : 150031
Mainz, 55131
- Completed
Investigational Site Number : 150432
Oberhausen, 46045
South Korea7 sites, 6 recruiting
- Recruiting
Investigational Site Number : 153191
Daejeon
- Recruiting
Investigational Site Number : 153180
Seoul, Seoul-teukbyeolsi, 03080
- Recruiting
Investigational Site Number : 153196
Seoul, Seoul-teukbyeolsi, 03080
- Recruiting
Investigational Site Number : 151149
Seoul, Seoul-teukbyeolsi, 05505
- Recruiting
Investigational Site Number : 153222
Seoul, Seoul-teukbyeolsi, 06973
- Recruiting
Investigational Site Number : 153212
Seoul
- Terminated
Investigational Site Number : 154208
Yangsan, Gyeongsangnam-do, 50612
Turkey (Türkiye)7 sites, 5 recruiting
- Recruiting
Investigational Site Number : 792006
Adana, 01330
- Recruiting
Investigational Site Number : 792001
Ankara, 06100
- Completed
Investigational Site Number : 792002
Ankara, 06230
- Recruiting
Investigational Site Number : 792005
Ankara, 06620
- Recruiting
Investigational Site Number : 792004
Istanbul, 34093
- Recruiting
Investigational Site Number : 792007
Izmir
- Completed
Investigational Site Number : 792003
Malatya, 44280
India6 sites, 5 recruiting
- Recruiting
Investigational Site Number : 356003
Ahmedabad, 380015
- Completed
Investigational Site Number : 356004
Jaipur, 302015
- Recruiting
Investigational Site Number : 356001
Kolkata, 700017
- Recruiting
Investigational Site Number : 356005
Mumbai, 400025
- Recruiting
Investigational Site Number : 3560006
Secunderabad, 500026
- Recruiting
Investigational Site Number : 356002
Vellore, 632004
Serbia5 sites, 5 recruiting
- Recruiting
Investigational Site Number : 688001
Belgrade, 11000
- Recruiting
Investigational Site Number : 688002
Belgrade, 11000
- Recruiting
Investigational Site Number : 688005
Belgrade, 11000
- Recruiting
Investigational Site Number : 688003
Belgrade, 11080
- Recruiting
Investigational Site Number : 688004
Niš, 18000
Greece4 sites, 4 recruiting
- Recruiting
Investigational Site Number : 300002
Athens, 115 27
- Recruiting
Investigational Site Number : 300004
Athens, 115 27
- Recruiting
Investigational Site Number : 300003
Pátrai, 26500
- Recruiting
Investigational Site Number : 300001
Thessaloniki, 546 42
Hungary4 sites, 4 recruiting
- Recruiting
Investigational Site Number : 3480006
Budapest, H-1097
- Recruiting
Investigational Site Number : 3480003
Létavértes, 4281
- Recruiting
Investigational Site Number : 3480007
Nyiregyháza, 4400
- Recruiting
Investigational Site Number : 3480008
Székesfehérvár, 8000
Japan4 sites, 2 recruiting
- Recruiting
Investigational Site Number : 153217
Chiba, 266-0007
- Completed
Investigational Site Number : 153205
Osaka, 545-8586
- Recruiting
Investigational Site Number : 153204
Tokyo, 157-8535
- Completed
Investigational Site Number : 153203
Yonago, Tottori, 683-8504
Switzerland4 sites, 4 recruiting
- Recruiting
Investigational Site Number : 157237
Aarau, 5001
- Recruiting
Investigational Site Number : 157045
Bern, 3010
- Recruiting
Investigational Site Number : 157244
Lausanne, 1011
- Recruiting
Investigational Site Number : 157241
Zurich, 8057
Thailand4 sites, 4 recruiting
- Recruiting
Investigational Site Number : 764003
Bangkok, 10400
- Recruiting
Investigational Site Number : 764004
Bangkok, 10700
- Recruiting
Investigational Site Number : 764001
Bangkok
- Recruiting
Investigational Site Number : 764005
Khon Kaen, 40002
Croatia3 sites, 3 recruiting
- Recruiting
Investigational Site Number : 1910001
Zagreb, 10000
- Recruiting
Investigational Site Number : 1910002
Zagreb, 10000
- Recruiting
Investigational Site Number : 1910003
Zagreb, 10000
Israel3 sites, 3 recruiting
- Recruiting
Investigational Site Number : 3760003
Haifa, 3109601
- Recruiting
Investigational Site Number : 3760001
Jerusalem, 9103102
- Recruiting
Investigational Site Number : 3760002
Petah Tikva, 4941492
Portugal3 sites, 3 recruiting
- Recruiting
Investigational Site Number : 620003
Coimbra, 3040-081
- Recruiting
Investigational Site Number : 620004
Lisbon, 1649-035
- Recruiting
Investigational Site Number : 620009
Ponta Delgada, 9500-370
Romania3 sites, 1 recruiting
- Completed
Investigational Site Number : 151188
Cluj-Napoca, 400006
- Completed
Investigational Site Number : 642001
Cluj-Napoca, 400006
- Recruiting
Investigational Site Number : 642002
Cluj-Napoca, 400006
Saudi Arabia3 sites, 2 recruiting
- Recruiting
Investigational Site Number : 682002
Al-Hasa, 31982
- Completed
Investigational Site Number : 682003
Jeddah
- Recruiting
Investigational Site Number : 682004
Mecca, 24246
South Africa3 sites, 3 recruiting
- Recruiting
Investigational Site Number : 154138
Durban, 4001
- Recruiting
Investigational Site Number : 150346
Johannesburg, 2057
- Recruiting
Investigational Site Number : 710-003
Johannesburg, 2193
Sweden3 sites, 1 recruiting
- Completed
Investigational Site Number : 154095
Borås, 501 82
- Active, not recruiting
Investigational Site Number : 151726
Malmö, 205 02
- Recruiting
Investigational Site Number : 154092
Norrköping, 601 82
Bulgaria2 sites, 2 recruiting
- Recruiting
Investigational Site Number : 151164
Sofia, 1606
- Recruiting
Investigational Site Number : 100001
Sofia, 1680
Lebanon2 sites, 1 recruiting
- Completed
Investigational Site Number : 422001
Beirut, 166830
- Recruiting
Investigational Site Number : 4220004
Beirut
Malaysia2 sites, 2 recruiting
- Recruiting
Investigational Site Number : 458001
Kuala Lumpur, 50586
- Recruiting
Investigational Site Number : 458002
Kuala Lumpur, 59100
Netherlands2 sites
- Completed
Investigational Site Number : 150149
Amsterdam, 1105 AZ
- Terminated
Investigational Site Number : 151171
Amsterdam, 1105 AZ
Pakistan2 sites, 2 recruiting
- Recruiting
Investigational Site Number : 154264
Karachi
- Recruiting
Investigational Site Number : 154266
Lahore
Russia2 sites, 2 recruiting
- Recruiting
Investigational Site Number : 643001
Moscow
- Recruiting
Investigational Site Number : 643002
Sait-Petersburg
Vietnam2 sites, 2 recruiting
- Recruiting
Investigational Site Number : 154212
Hanoi
- Recruiting
Investigational Site Number : 704002
Ho Chi Minh City, 70000
Colombia1 site, 1 recruiting
- Recruiting
Investigational Site Number : 170004
Bogotá
Czechia1 site, 1 recruiting
- Recruiting
Investigational Site Number : 150809
Prague, 12808
Denmark1 site, 1 recruiting
- Recruiting
Investigational Site Number : 151229
Copenhagen, 2100
Dominican Republic1 site, 1 recruiting
- Recruiting
Investigational Site Number : 2140001
Santo Domingo, 10101
Hong Kong1 site, 1 recruiting
- Recruiting
Investigational Site Number : 344001
Kowloon
Indonesia1 site, 1 recruiting
- Recruiting
Investigational Site Number : 360001
Jakarta
Jordan1 site, 1 recruiting
- Recruiting
Investigational Site Number : 4000001
Amman, 11941
Lithuania1 site, 1 recruiting
- Recruiting
Investigational Site Number : 154237
Vilnius, 08661
Philippines1 site, 1 recruiting
- Recruiting
Investigational Site Number : 608001
City of Muntinlupa, 1000
Poland1 site, 1 recruiting
- Recruiting
Investigational Site Number : 150575
Warsaw, Masovian Voivodeship, 04-730
Singapore1 site, 1 recruiting
- Recruiting
Investigational Site Number : 702001
Singapore, 229899
Slovakia1 site
- Completed
Investigational Site Number : 703001
Bratislava, 833 40
United Arab Emirates1 site, 1 recruiting
- Recruiting
Investigational Site Number : 7840001
Dubai
About this study
Official title: 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:
Read more
- 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.
- Conditions
- Gaucher Disease, Cerebroside Lipidosis Syndrome, Glucocerebrosidase Deficiency Disease, Glucosylceramide Beta-Glucosidase Deficiency Disease
- Keywords
- Gaucher Disease, Glucocerebrosidase Deficiency Disease
- Medical subject headings
- Gaucher Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| 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 being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: 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
Measured: 42 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Aug 2006 (estimated)
- Study start1 Apr 1991
- Primary completion31 Jan 2034 (estimated)
- Study completion31 Jan 2034 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (8)Papers the sponsor has linked to this study.
- Other trials for Gaucher DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 12,000 participants (estimated)
- Target duration
- 12 Months
Sponsor and investigators
- Lead sponsor
- Genzyme, a Sanofi Company
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Study Director, Study director, Genzyme, a Sanofi Company
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00358943
- Sponsor's protocol ID
- DIREGC07009
- Other IDs
- ICGG Gaucher Registry, U1111-1295-5844
- Status last verified
- Oct 2026
- First submitted
- 12 Jul 2006
- Last updated
- 14 Oct 2026
- Results posted
- No
- Data monitoring committee
- No
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.
PubMed 28040394Mistry 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.
PubMed 28569047Weinreb 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.
PubMed 22976765Ain NU, Vaishnaw M, Mistry PK. Natural-History Mapping of Lysosomal Storage Disorders (LSDs): Gaucher Disease as a Model for Precision Care. J Inherit Metab Dis. 2026 Jan;49(1):e70128. doi: 10.1002/jimd.70128.
PubMed 41527340Rosenbloom 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.
PubMed 36054609Weinreb 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.
PubMed 33485799Weinreb 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.
PubMed 26096743Buyers RP, Collin-Histed T. Foreword. Am J Hematol. 2015 Jul;90 Suppl 1:S1. doi: 10.1002/ajh.24053. No abstract available.
PubMed 26096740