Gaucher Disease Clinical Trial
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: - 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.
Status | Recruiting |
Enrollment | 12000 |
Est. completion date | January 31, 2034 |
Est. primary completion date | January 31, 2034 |
Accepts healthy volunteers | No |
Gender | All |
Age group | N/A and older |
Eligibility | 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 ß-glucocerebrosidase deficiency and/or mutation in the ß-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. |
Country | Name | City | State |
---|---|---|---|
Argentina | Investigational Site Number : 032012 | Buenos Aires | |
Argentina | Investigational Site Number : 150661 | Caba | Buenos Aires |
Argentina | Investigational Site Number : 032011 | Córdoba | |
Argentina | Investigational Site Number : 154176 | Corrientes | |
Argentina | Investigational Site Number : 032003 | La Plata | Buenos Aires |
Argentina | Investigational Site Number : 151085 | Sarandi | Buenos Aires |
Australia | Investigational Site Number : 151002 | Adelaide | South Australia |
Australia | Investigational Site Number : 151011 | Clayton | Victoria |
Australia | Investigational Site Number : 151491 | Douglas | Queensland |
Australia | Investigational Site Number : 151010 | Herston | Queensland |
Australia | Investigational Site Number : 151725 | Hobart | Tasmania |
Australia | Investigational Site Number : 151015 | Murdoch | Western Australia |
Australia | Investigational Site Number : 151008 | Parkville | Victoria |
Australia | Investigational Site Number : 151069 | Westmead | New South Wales |
Belgium | Investigational Site Number : 150722 | Antwerpen | |
Belgium | Investigational Site Number : 150944 | Brussel | |
Belgium | Investigational Site Number : 152345 | Brussel | |
Belgium | Investigational Site Number : 150517 | Bruxelles | |
Belgium | Investigational Site Number : 151796 | Bruxelles | |
Belgium | Investigational Site Number : 154285 | Edegem | |
Belgium | Investigational Site Number : 151134 | Gent | |
Belgium | Investigational Site Number : 151668 | Leuven | |
Brazil | Faculdade De Medicina - Unicamp Site Number : 076005 | Campinas | |
Brazil | PUC Campinas - Sociedade Campineira de Educaçao e Instruçao Site Number : 076012 | Campinas | São Paulo |
Brazil | Hospital das Clínicas da Universidade Federal do Paraná - HCUFPR Site Number : 076010 | Curitiba | Paraná |
Brazil | Hospital De Clinicas De Porto Alegre Site Number : 076007 | Porto Alegre | Rio Grande Do Sul |
Brazil | Faculdade de Medicina de Ribeirao Preto - USP Site Number : 076001 | Ribeirao Preto | São Paulo |
Brazil | HEMORIO - Instituto Estadual de Hematologia Arthur de Siqueira Cavalcanti Site Number : 076006 | Rio de Janeiro | |
Brazil | Hospital das Clínicas da Faculdade de Medicina da USP Site Number : 076008 | São Paulo | |
Brazil | Instituto de Genética e Erros Inatos do Metabolismo - IGEIM Site Number : 076002 | São Paulo | |
Bulgaria | Investigational Site Number : 100001 | Sofia | |
Bulgaria | Investigational Site Number : 151164 | Sofia | |
Canada | Investigational Site Number : 124014 | Calgary | Alberta |
Canada | Investigational Site Number : 124002 | Edmonton | Alberta |
Canada | Investigational Site Number : 124024 | Kingston | Ontario |
Canada | Investigational Site Number : 124021 | London | Ontario |
Canada | Investigational Site Number : 124022 | Moncton | New Brunswick |
Canada | Investigational Site Number : 124001 | Sherbrooke | Quebec |
Canada | Investigational Site Number : 124006 | Toronto | Ontario |
Canada | Investigational Site Number : 124013 | Toronto | Ontario |
Canada | Investigational Site Number : 124010 | Vancouver | British Columbia |
Canada | Investigational Site Number : 124011 | Vancouver | British Columbia |
Canada | Investigational Site Number : 124019 | Winnipeg | Manitoba |
Croatia | Investigational Site Number : 1910001 | Zagreb | |
Croatia | Investigational Site Number : 1910002 | Zagreb | |
Croatia | Investigational Site Number : 1910003 | Zagreb | |
Czechia | Investigational Site Number : 150809 | Praha 2 | |
Denmark | Investigational Site Number : 151229 | København Ø | |
Dominican Republic | Investigational Site Number : 2140001 | Santo Domingo | |
Egypt | Investigational Site Number : 5748 | Alexandria | |
Egypt | Investigational Site Number : 9889 | Ash Sharqia | |
Egypt | Investigational Site Number : 9888 | Assuit | |
Egypt | Investigational Site Number : 8345 | Cairo | |
Egypt | Investigational Site Number : 9829 | Cairo | |
Egypt | Investigational Site Number : 9830 | Cairo | |
France | Investigational Site Number : 250015 | ANGERS Cedex 01 | |
France | Investigational Site Number : 250004 | Besancon | |
France | Investigational Site Number : 250008 | Bordeaux | |
France | Investigational Site Number : 250005 | Brest | |
France | Investigational Site Number : 250014 | BRON Cedex | |
France | Investigational Site Number : 250002 | Caen | |
France | Investigational Site Number : 250001 | CLICHY Cedex | |
France | Investigational Site Number : 250003 | Garches | |
France | Investigational Site Number : 250009 | LYON Cedex 03 | |
France | Investigational Site Number : 2500013 | Montpellier | |
France | Investigational Site Number : 250006 | NANTES Cedex 1 | |
France | Investigational Site Number : 250010 | Paris | |
France | Investigational Site Number : 250012 | Rennes Cedex 2 | |
France | Investigational Site Number : 250018 | Strasbourg | |
France | Investigational Site Number : 250017 | Toulouse | |
France | Investigational Site Number : 250016 | TOURS Cedex 9 | |
Germany | Investigational Site Number : 151686 | Berlin | |
Germany | Investigational Site Number : 150783 | Düsseldorf | |
Germany | Investigational Site Number : 154150 | Gießen | |
Germany | Investigational Site Number : 151368 | Hamburg | |
Germany | Investigational Site Number : 151369 | Hamburg | |
Germany | Investigational Site Number : 150031 | Mainz | |
Germany | Investigational Site Number : 150432 | Oberhausen | |
Greece | Investigational Site Number : 300002 | Athens | |
Greece | Investigational Site Number : 300004 | Athens | |
Greece | Investigational Site Number : 300003 | Patras | |
Greece | Investigational Site Number : 300001 | Thessaloniki | |
Hong Kong | Investigational Site Number : 344001 | Kowloon | |
Hungary | Investigational Site Number : 3480006 | Budapest | |
Hungary | Investigational Site Number : 3480003 | Létavértes | |
Hungary | Investigational Site Number : 3480007 | Nyíregyháza | |
Hungary | Investigational Site Number : 3480008 | Székesfehérvár | |
India | Investigational Site Number : 356003 | Ahmedabad | |
India | Investigational Site Number : 356004 | Jaipur | |
India | Investigational Site Number : 356001 | Kolkata | |
India | Investigational Site Number : 356005 | Mumbai | |
India | Investigational Site Number : 3560006 | Secunderabad | |
India | Investigational Site Number : 356002 | Vellore | |
Indonesia | Investigational Site Number : 360001 | Jakarta | |
Israel | Investigational Site Number : 3760003 | Haifa | |
Israel | Investigational Site Number : 3760001 | Jerusalem | |
Israel | Investigational Site Number : 3760002 | Petach-Tikva | |
Italy | Investigational Site Number : 380008 | Catania | |
Italy | Investigational Site Number : 380009 | Firenze | |
Italy | Investigational Site Number : 380006 | Genova | |
Italy | Investigational Site Number : 380004 | Milano | |
Italy | Investigational Site Number : 380005 | Modena | |
Italy | Investigational Site Number : 380011 | Monza | |
Italy | Investigational Site Number : 380002 | Monza (MB) | |
Italy | Investigational Site Number : 380010 | Napoli | |
Italy | Investigational Site Number : 380003 | Padova | |
Italy | Investigational Site Number : 380007 | Roma | |
Italy | Investigational Site Number : 380001 | Udine | |
Japan | Investigational Site Number : 153205 | Abeno-ku | Osaka |
Japan | Investigational Site Number : 153217 | Chiba | |
Japan | Investigational Site Number : 153204 | Setagaya | Tokyo |
Japan | Investigational Site Number : 153203 | Yonago-shi | Tottori |
Jordan | Investigational Site Number : 4000001 | Amman | |
Korea, Republic of | Investigational Site Number : 153191 | Daejeon | |
Korea, Republic of | Investigational Site Number : 153225 | Jeonnam | |
Korea, Republic of | Investigational Site Number : 151149 | Seoul | |
Korea, Republic of | Investigational Site Number : 153180 | Seoul | |
Korea, Republic of | Investigational Site Number : 153192 | Seoul | |
Korea, Republic of | Investigational Site Number : 153196 | Seoul | |
Korea, Republic of | Investigational Site Number : 153212 | Seoul | |
Korea, Republic of | Investigational Site Number : 153222 | Seoul | |
Korea, Republic of | Investigational Site Number : 154148 | Yangsan | Gyeongsangnam-do |
Korea, Republic of | Investigational Site Number : 153178 | Yangsan-si | Gyeongsangnam-do |
Kuwait | Investigational Site Number : 154225 | Kuwait | |
Lebanon | Investigational Site Number : 4220004 | Beirut | |
Lebanon | Investigational Site Number : 422001 | Beirut | |
Lebanon | Investigational Site Number : 422006 | Douris | |
Lebanon | Investigational Site Number : 4220002 | Hazmieh | |
Lithuania | Investigational Site Number : 154237 | Vilnius | |
Malaysia | Investigational Site Number : 458001 | Jalan Pahang | |
Malaysia | Investigational Site Number : 458002 | Kuala Lumpur | |
Netherlands | Investigational Site Number : 150149 | Amsterdam | |
Pakistan | Investigational Site Number : 154264 | Karachi | |
Pakistan | Investigational Site Number : 154266 | Lahore | |
Peru | Investigational Site Number : 604001 | Arequipa | |
Peru | Investigational Site Number : 604002 | Callao | |
Philippines | Investigational Site Number : 608001 | Manila | |
Poland | Investigational Site Number : 150575 | Warszawa | |
Portugal | Investigational Site Number : 620003 | Coimbra | |
Portugal | Investigational Site Number : 620004 | Lisboa | |
Portugal | Investigational Site Number : 620009 | Ponta Delgada | |
Romania | Investigational Site Number : 151188 | Cluj Napoca | |
Romania | Investigational Site Number : 642002 | Cluj Napoca | |
Romania | Investigational Site Number : 642001 | Cluj-Napoca | |
Russian Federation | Investigational Site Number : 643001 | Moscow | |
Russian Federation | Investigational Site Number : 643002 | sait-Petersburg | |
Saudi Arabia | Investigational Site Number : 682002 | Al Mubarraz | |
Saudi Arabia | Investigational Site Number : 682003 | Jeddah | |
Saudi Arabia | Investigational Site Number : 682004 | Makkah | |
Serbia | Investigational Site Number : 688001 | Belgrade | |
Serbia | Investigational Site Number : 688002 | Belgrade | |
Serbia | Investigational Site Number : 688003 | Belgrade | |
Serbia | Investigational Site Number : 688005 | Belgrade | |
Serbia | Investigational Site Number : 688004 | Nis | |
Singapore | Investigational Site Number : 702001 | Singapore | |
Slovakia | Investigational Site Number : 703001 | Bratislava | |
South Africa | Investigational Site Number : 154138 | Johannesburg | |
South Africa | Investigational Site Number : 710-003 | Johannesburg | |
South Africa | Investigational Site Number : 150346 | Sandton | |
Spain | Investigational Site Number : 153237 | Barcelona | |
Spain | Investigational Site Number : 154147 | Barcelona | |
Spain | Investigational Site Number : 154146 | La Coruña | |
Spain | Investigational Site Number : 151524 | Madrid | |
Spain | Investigational Site Number : 154119 | Madrid | |
Spain | Investigational Site Number : 154160 | Madrid | |
Spain | Investigational Site Number : 154163 | Madrid | |
Spain | Investigational Site Number : 154199 | Madrid | |
Spain | Investigational Site Number : 154236 | Madrid | |
Spain | Investigational Site Number : 154145 | Sabadell | |
Spain | Investigational Site Number : 154222 | Santiago de Compostela | |
Spain | Investigational Site Number : 153215 | Sevilla | |
Spain | Investigational Site Number : 153219 | Sevilla | |
Spain | Investigational Site Number : 154205 | Valencia | |
Spain | Investigational Site Number : 153213 | Vigo | |
Spain | Investigational Site Number : 153216 | Zaragoza | |
Spain | Investigational Site Number : 154238 | Zaragoza | |
Sweden | Investigational Site Number : 154095 | Borås | |
Sweden | Investigational Site Number : 151726 | Malmö | |
Sweden | Investigational Site Number : 154092 | Norrköping | |
Switzerland | Investigational Site Number : 157045 | Bern | |
Taiwan | Investigational Site Number : 154093 | Chiayi | |
Taiwan | Investigational Site Number : 152152 | Hualien | |
Taiwan | Investigational Site Number : 152244 | Kaohsiung | |
Taiwan | Investigational Site Number : 152246 | Kaohsiung Hsien, | |
Taiwan | Investigational Site Number : 151534 | New Taipei City | |
Taiwan | Investigational Site Number : 151500 | Putz City, Chia-yi | |
Taiwan | Investigational Site Number : 151181 | Taichung | |
Taiwan | Investigational Site Number : 151179 | Taichung City | |
Taiwan | Investigational Site Number : 152153 | Tainan | |
Taiwan | Investigational Site Number : 153248 | Tainan | |
Taiwan | Investigational Site Number : 151182 | Taipei | |
Taiwan | Investigational Site Number : 152226 | Taipei | |
Taiwan | Investigational Site Number : 152321 | Taipei | |
Taiwan | Investigational Site Number : 153158 | Tao Yuan County | |
Thailand | Investigational Site Number : 764001 | Bangkok | |
Thailand | Investigational Site Number : 764003 | Bangkok | |
Thailand | Investigational Site Number : 764004 | Bangkok | |
Thailand | Investigational Site Number : 764005 | Khon Kaen | |
Turkey | Investigational Site Number : 792006 | Adana | |
Turkey | Investigational Site Number : 792001 | Ankara | |
Turkey | Investigational Site Number : 792002 | Ankara | |
Turkey | Investigational Site Number : 792005 | Ankara | |
Turkey | Investigational Site Number : 792004 | Istanbul | |
Turkey | Investigational Site Number : 792007 | Izmir | |
Turkey | Investigational Site Number : 792003 | Malatya | |
United Arab Emirates | Investigational Site Number : 7840001 | Dubai | |
United Kingdom | Investigational Site Number : 152287 | Birmingham | |
United Kingdom | Investigational Site Number : 154240 | Birmingham | |
United Kingdom | Investigational Site Number : 151340 | Cambridge | Cambridgeshire |
United Kingdom | Investigational Site Number : 150303 | London | |
United Kingdom | Investigational Site Number : 151123 | London | |
United Kingdom | Investigational Site Number : 151646 | London | |
United Kingdom | Investigational Site Number : 150455 | Manchester | |
United Kingdom | Investigational Site Number : 152361 | Salford | |
United States | University of Michigan Pediatrics Site Number : 840107 | Ann Arbor | Michigan |
United States | University of Colorado at Denver Genetics Site Number : 840068 | Aurora | Colorado |
United States | John Hopkins Site Number : 840044 | Baltimore | Maryland |
United States | Tower Hematology/Oncology Medical Group Site Number : 840056 | Beverly Hills | California |
United States | Boston Children's Hospital Site Number : 840092 | Boston | Massachusetts |
United States | Massachusetts General Hospital-Genetics Site Number : 840062 | Boston | Massachusetts |
United States | Carolinas Medical Center Hospital Site Number : 840065 | Charlotte | North Carolina |
United States | University of Virginia School of Medicine-Pediatrics Site Number : 840078 | Charlottesville | Virginia |
United States | Ann and Robert Lurie Children's Hospital Site Number : 840011 | Chicago | Illinois |
United States | Ann and Robert Lurie Children's Hospital Site Number : 840013 | Chicago | Illinois |
United States | Rush University Medical Center Genetics Site Number : 840079 | Chicago | Illinois |
United States | Cleveland Clinic Foundation Pediatrics Site Number : 840048 | Cleveland | Ohio |
United States | UHCMC Site Number : 840119 | Cleveland | Ohio |
United States | Nationwide Children's Hospital Site Number : 840091 | Columbus | Ohio |
United States | University of Missouri Health System Department of Genetics Site Number : 840031 | Columibia | Missouri |
United States | University Hematology Oncology Site Number : 840075 | Coral Springs | Florida |
United States | Baylor Research Center Site Number : 840058 | Dallas | Texas |
United States | Emory University School of Medicine- Human Genetics Site Number : 840060 | Decatur | Georgia |
United States | Children's Hospital of Michigan Site Number : 840066 | Detroit | Michigan |
United States | LSD Data Registry Site LLC Site Number : 840094 | Dublin | Ohio |
United States | Duke University Medical Center Genetics Dept Site Number : 840037 | Durham | North Carolina |
United States | O&O Alpan, LLC Site Number : 840025 | Fairfax | Virginia |
United States | Cook Children's Health Care System Site Number : 840024 | Fort Worth | Texas |
United States | Infusion Associates Site Number : 840050 | Grand Rapids | Michigan |
United States | Greenwood Genetcs Site Number : 840055 | Greenville | South Carolina |
United States | Joseph M. Sanzari Children's Hospital Site Number : 840101 | Hackensack | New Jersey |
United States | Indianapolis University School of Medicine Site Number : 840027 | Indianapolis | Indiana |
United States | University of Iowa Site Number : 840032 | Iowa City | Iowa |
United States | University of California at Irvine Site Number : 840036 | Irvine | California |
United States | The Atwal Clinic Site Number : 840112 | Jacksonville | Florida |
United States | University of Florida Pediatrics Genetics Site Number : 840121 | Jacksonville | Florida |
United States | Children's Mercy Hospital- Pediatrics Site Number : 840063 | Kansas City | Missouri |
United States | Children's Specialty Center of Nevada Site Number : 840008 | Las Vegas | Nevada |
United States | Arkansas Childrens Hospital Site Number : 840109 | Little Rock | Arkansas |
United States | University of Arkansas for Medical Sciences Site Number : 840113 | Little Rock | Arkansas |
United States | Southern California Permanente Medical Group Site Number : 840108 | Los Angeles | California |
United States | USC Health Sciences Center Dept of Genetics Site Number : 840082 | Los Angeles | California |
United States | University of Louisville- Genetics Site Number : 840030 | Louisville | Kentucky |
United States | Northwell Health Site Number : 840102 | Manhasset | New York |
United States | University Of Miami SOM Site Number : 840006 | Miami | Florida |
United States | Children's Hospital of Wisconsin-Pediatrics Site Number : 840054 | Milwaukee | Wisconsin |
United States | Children's Health Care Site Number : 840114 | Minneapolis | Minnesota |
United States | Children's Hospital and Clinics of Minnesota Site Number : 840046 | Minneapolis | Minnesota |
United States | University of Minnesota Medical Center Pediatrics Site Number : 840076 | Minneapolis | Minnesota |
United States | Atlantic Health System Site Number : 840099 | Morristown | New Jersey |
United States | Vanderbilt University Hospital-Pediatrics Site Number : 840049 | Nashville | Tennessee |
United States | St. Peter's University Hospital Site Number : 840016 | New Brunswick | New Jersey |
United States | Yale Site Number : 840047 | New Haven | Connecticut |
United States | Ochsner Medical Center Site Number : 840120 | New Orleans | Louisiana |
United States | Tulane University Medical Center Site Number : 840001 | New Orleans | Louisiana |
United States | Metropolitan Hospital Center Site Number : 840110 | New York | New York |
United States | Mt. Sinai School of Medicine Site Number : 840005 | New York | New York |
United States | New York University School Of Medicine Site Number : 840040 | New York | New York |
United States | Site Number : 840026 | New York | New York |
United States | Children's Hospital of the Kings Daughters Site Number : 840072 | Norfolk | Virginia |
United States | University of Nebraska Medical Center- Pediatrics Site Number : 840084 | Omaha | Nebraska |
United States | Children's Hospital of Orange County Site Number : 840074 | Orange | California |
United States | Stanford Unviersity MC Dept of Genetics Site Number : 840022 | Palo Alto | California |
United States | St. Joseph's Children's Hospital Site Number : 840057 | Paterson | New Jersey |
United States | Children's Hospital of Philadelphia HUP Medical Genetics Site Number : 840089 | Philadelphia | Pennsylvania |
United States | Children's Hospital of Philadelphia Site Number : 840034 | Philadelphia | Pennsylvania |
United States | Phoenix Children's Hospital Site Number : 840003 | Phoenix | Arizona |
United States | University of Pittsburgh Site Number : 840023 | Pittsburgh | Pennsylvania |
United States | Maine Medical Center Pediatrics Site Number : 840064 | Portland | Maine |
United States | Oregon Health & Science University Site Number : 840080 | Portland | Oregon |
United States | Rhode Island Hospital Genetics Site Number : 840053 | Providence | Rhode Island |
United States | University of Rochester Medical Center SOM Site Number : 840105 | Rochester | New York |
United States | Kaiser Permanente Hospital Site Number : 840042 | Sacramento | California |
United States | UC Davis MIND Institute Site Number : 840010 | Sacramento | California |
United States | Washington University of St. Louis Site Number : 840100 | Saint Louis | Missouri |
United States | University of Utah Department of Medical Genetics Site Number : 840043 | Salt Lake City | Utah |
United States | Loma Linda University Children's Hospital Site Number : 840117 | San Bernardino | California |
United States | University of California at San Diego Site Number : 840007 | San Diego | California |
United States | University of California at San Francisco Site Number : 840051 | San Francisco | California |
United States | Seattle Children's Hospital Site Number : 840028 | Seattle | Washington |
United States | University Of Washington Medical Center Site Number : 840059 | Seattle | Washington |
United States | State University of New York Site Number : 840052 | Syracuse | New York |
United States | Multicare Health System ¿ Mary Bridge Childrens Hospital Site Number : 840115 | Tacoma | Washington |
United States | Pediatric Endocrine Associates Site Number : 840017 | Tampa | Florida |
United States | University of Florida-Genetics Site Number : 840096 | Tampa | Florida |
United States | University of Arizona Site Number : 840015 | Tucson | Arizona |
United States | New York Medical College Site Number : 840039 | Valhalla | New York |
United States | Children's National Medical Center Site Number : 840067 | Washington | District of Columbia |
Vietnam | Investigational Site Number : 154212 | Hanoi | |
Vietnam | Investigational Site Number : 704002 | Ho Chi Minh City |
Lead Sponsor | Collaborator |
---|---|
Genzyme, a Sanofi Company |
United States, Vietnam, Argentina, Australia, Belgium, Brazil, Bulgaria, Canada, Croatia, Czechia, Denmark, Dominican Republic, Egypt, France, Germany, Greece, Hong Kong, Hungary, India, Indonesia, Israel, Italy, Japan, Jordan, Korea, Republic of, Kuwait, Lebanon, Lithuania, Malaysia, Netherlands, Pakistan, Peru, Philippines, Poland, Portugal, Romania, Russian Federation, Saudi Arabia, Serbia, Singapore, Slovakia, South Africa, Spain, Sweden, Switzerland, Taiwan, Thailand, Turkey, United Arab Emirates, United Kingdom,
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. — View Citation
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. — View Citation
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. — View Citation
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | 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 | 42 years | ||
Primary | 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 | 42 years |
Status | Clinical Trial | Phase | |
---|---|---|---|
Withdrawn |
NCT04189601 -
Complement Activation in the Lysosomal Storage Disorders
|
||
Completed |
NCT04430881 -
A National Study in Patients With Unexplained Splenomegaly
|
||
Completed |
NCT02536911 -
A Study of the Effects of Hepatic Impairment on the Pharmacokinetics and Tolerability of Eliglustat Tartrate
|
Phase 1 | |
Completed |
NCT02536937 -
A Study of the Effects of Renal Impairment on the Pharmacokinetics and Tolerability of Eliglustat Tartrate
|
Phase 1 | |
Completed |
NCT01411228 -
A Multicenter Extension Study of Taliglucerase Alfa in Pediatric Subjects With Gaucher Disease
|
Phase 3 | |
Terminated |
NCT04094181 -
A Study of VPRIV in Participants With Gaucher Disease Previously Treated With Other Enzyme Replacement Therapies or Substrate Reduction Therapies
|
||
Completed |
NCT00391625 -
Open-Label Extension Study Evaluating Long Term Safety in Patients With Type 1 Gaucher Disease Receiving DRX008A (ERT)
|
Phase 1/Phase 2 | |
Completed |
NCT03625882 -
Survey Study for Velaglucerase Alfa (VPRIV) in Japan
|
||
Active, not recruiting |
NCT05526664 -
Omics Gaucher Study: Multiomic Approach
|
||
Completed |
NCT02536755 -
Phase 3b Study to Evaluate Skeletal Response to Eliglustat in Adult Patients Who Completed Phase 2 or Phase 3 Studies
|
Phase 3 | |
Recruiting |
NCT01344096 -
Thrombocytopathy in Gaucher Disease Patients
|
N/A | |
Completed |
NCT01881633 -
A Study of the Tolerability, Safety, and Pharmacokinetics of ISU302 in Healthy Volunteers
|
Phase 1 | |
Recruiting |
NCT06116071 -
Biomarkers Related to Bone in Pediatric Gaucher Disease
|
||
Recruiting |
NCT01951989 -
Intra-monocyte Imiglucerase Kinetics in Gaucher Disease
|
Phase 2 | |
Completed |
NCT00258778 -
Phase I Single Dose-Escalation Safety Study of Human Glucocerebrosidase (prGCD)
|
Phase 1 | |
Recruiting |
NCT04388969 -
World Data on Ambroxol for Patients With GD and GBA Related PD
|
||
Recruiting |
NCT05992532 -
GammaGA: Prevalence of Acid Sphingomyelinase Deficiency Disease (ASMD) and Gaucher Disease in Patients With Monoclonal Gammopathies and/or Multiple Myeloma
|
||
Terminated |
NCT04145037 -
Lentiviral Vector Gene Therapy - The Guard1 Trial of AVR-RD-02 for Subjects With Type 1 Gaucher Disease
|
Phase 1/Phase 2 | |
Completed |
NCT00302146 -
Positron Emission Tomography (PET) Imaging in People With Gaucher Mutations
|
||
Active, not recruiting |
NCT02605603 -
SRT in Comparison to ERT on Immune Aspects and Bone Involvement in Gaucher Disease
|