International Collaborative Gaucher Group (ICGG) Gaucher Disease Registry & Pregnancy Sub-registry

Gaucher Disease Clinical Trial

Official title:

Gaucher Disease Registry Protocol

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT00358943
• Other study ID #: DIREGC07009
• Secondary ID: ICGG Gaucher Reg
• Status: Recruiting
• Start date: April 1, 1991
• Est. completion date: January 31, 2034

Study information

• Verified date: April 2024
• Source: Sanofi
• Contact: Trial Transparency email recommended (Toll free number for US &
• Phone: 800-633-1610
• Email: Contact-Us[@]sanofi.com
• Is FDA regulated: No
• Study type: Observational [Patient Registry]

Clinical 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. 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.

Recruitment information / eligibility

• 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.

Related Conditions & MeSH terms

• Cerebroside Lipidosis Syndrome
• Deficiency Diseases
• Gaucher Disease
• Glucocerebrosidase Deficiency Disease
• Glucosylceramide Beta-Glucosidase Deficiency Disease

Locations

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

Sponsors (1)

Lead Sponsor	Collaborator
Genzyme, a Sanofi Company

Countries where clinical trial is conducted

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, 

References & Publications (3)

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

Outcome

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