ClinicalTrials.gg
RecruitingNCT00358943Updated Oct 14, 2026

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

An observational study in Gaucher Disease, Cerebroside Lipidosis Syndrome and Glucocerebrosidase Deficiency Disease, sponsored by Genzyme, a Sanofi Company. Recruiting at 317 sites in 52 countries. Per ClinicalTrials.gov, last updated 2026-10-14.

Sponsored by Genzyme, a Sanofi Company (part of Sanofi) · Observational

From the registry’s dates

  • Started Apr 1991; still recruiting 35 years 7 months later.
Updated Oct 14, 2026Site recruiting status changed48 sites added+1 moreGo to Updates ↓
Study type
Observational
Model
Cohort
Time perspective
Prospective
Enrollment
12,000
Sex
All
01

Study 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.

02

Conditions studied

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

Browse trials for

Keywords

  • Gaucher Disease
  • Glucocerebrosidase Deficiency Disease
03

In context

Gaucher Disease

177 studies on the registry are indexed under Gaucher Disease; 41 are open to participants now.

This study's planned enrollment of 12,000 is above the median of 63 across 67 observational studies indexed under Gaucher Disease.

Browse Gaucher Disease studies →

Lead sponsor

Genzyme, a Sanofi Company is the lead sponsor of 308 studies on the registry; 5 are open to participants now.

Of its 25 completed or terminated interventional studies of FDA-regulated products, 19 (76%) have results posted.

First submitted before Genzyme became part of Sanofi.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
Child (0–17), Adult (18–64), Older adult (65+)
Sexes eligible
All
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.

Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.

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 and authorization form(s) to participate in the Sub-Registry prior to any Sub-Registry-related data collection being performed.

Exclusion criteria

Exclusion Criteria:

- No exclusion criteria for participation in the ICGG Gaucher Registry and Sub-registry.

Terms in these criteria, in plain language(1)

Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.

Informed consent

Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.

You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.

05

Study design

Observational model
Cohort
Time perspective
Prospective
Enrollment
12,000 participants (estimated)
Target follow-up
12 Months
Patient registry
Yes

Groups and cohorts

  • 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.

06

What researchers measure

Primary outcomes

  1. 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

  2. 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

07

Study locations

238 of 317 sites recruiting
  • Phoenix Children's Hospital- Site Number : 840003
    Phoenix, Arizona 85016, United States
    Recruiting
  • University of Arizona- Site Number : 840015
    Tucson, Arizona 85724, United States
    Recruiting
  • Arkansas Children's Hospital- Site Number : 840109
    Little Rock, Arkansas 72202, United States
    Recruiting
  • University of Arkansas for Medical Sciences- Site Number : 840113
    Little Rock, Arkansas 72205, United States
    Recruiting
  • Tower Hematology Oncology Medical Group- Site Number : 840056
    Beverly Hills, California 90211, United States
    Recruiting
  • Southern California Permanente Medical Group- Site Number : 840108
    Los Angeles, California 90027, United States
    Recruiting
  • Usc Health Sciences Center Dept Of Genetics- Site Number : 840082
    Los Angeles, California 90033, United States
    Completed

    Not enrolling at this site — choose another above.

  • Children's Hospital of Orange County- Site Number : 840074
    Orange, California 92868, United States
    Recruiting
  • University of California Irvine Medical Center- Site Number : 840036
    Orange, California 92868, United States
    Completed

    Not enrolling at this site — choose another above.

  • Uc Davis Mind Institute- Site Number : 840010
    Sacramento, California 95817, United States
    Recruiting
  • Kaiser Permanente Sacramento Medical Center- Site Number : 840042
    Sacramento, California 95825, United States
    Completed

    Not enrolling at this site — choose another above.

  • University of California at San Diego- Site Number : 840007
    San Diego, California 92123, United States
    Completed

    Not enrolling at this site — choose another above.

  • University of California San Francisco - Parnassus Campus- Site Number : 840051
    San Francisco, California 94143, United States
    Recruiting
  • Stanford University Medical Center- Site Number : 840022
    Stanford, California 94305, United States
    Recruiting
  • University Of Colorado At Denver Genetics- Site Number : 840068
    Aurora, Colorado 80045, United States
    Recruiting
  • Yale University School of Medicine- Site Number : 840047
    New Haven, Connecticut 06510, United States
    Recruiting
  • Children's National Medical Center - Washington- Site Number : 840067
    Washington D.C., District of Columbia 20010, United States
    Recruiting
  • Investigational Site Number : 250012
    Washington D.C., District of Columbia 20037-3201, United States
    Recruiting
  • University of Miami- Site Number : 840006
    Coral Gables, Florida 33124, United States
    Recruiting
  • University of Miami Sylvester Comprehensive Cancer Center - Coral Springs- Site Number : 840075
    Coral Springs, Florida 33065, United States
    Recruiting
  • University Of Florida Pediatrics Genetics- Site Number : 840121
    Jacksonville, Florida 32207, United States
    Recruiting
  • The Atwal Clinic- Site Number : 840112
    Jacksonville, Florida 32216, United States
    Completed

    Not enrolling at this site — choose another above.

  • University Of Florida-Genetics- Site Number : 840096
    Tampa, Florida 33606, United States
    Recruiting
  • Pediatric Endocrine Association- Site Number : 840017
    Tampa, Florida 33607, United States
    Completed

    Not enrolling at this site — choose another above.

  • Emory University School Of Medicine- Human Genetics- Site Number : 840060
    Decatur, Georgia 30033, United States
    Recruiting
  • Ann & Robert H. Lurie Children's Hospital of Chicago- Site Number : 840011
    Chicago, Illinois 60611, United States
    Recruiting
  • Ann & Robert H. Lurie Children's Hospital of Chicago- Site Number : 840013
    Chicago, Illinois 60611, United States
    Recruiting
  • Rush University Medical Center Genetics- Site Number : 840079
    Chicago, Illinois 60612, United States
    Recruiting
  • Indianapolis University School Of Medicine- Site Number : 840027
    Indianapolis, Indiana 46202, United States
    Recruiting
  • University of Iowa- Site Number : 840032
    Iowa City, Iowa 52242, United States
    Recruiting
  • University of Louisville - Health Sciences Center- Site Number : 840030
    Louisville, Kentucky 40202, United States
    Completed

    Not enrolling at this site — choose another above.

  • Tulane University School of Medicine- Site Number : 840001
    New Orleans, Louisiana 70112, United States
    Completed

    Not enrolling at this site — choose another above.

  • Maine Medical Partners Pediatric Specialty Care- Site Number : 840064
    Portland, Maine 04102, United States
    Recruiting
  • John Hopkins- Site Number : 840044
    Baltimore, Maryland 21205, United States
    Completed

    Not enrolling at this site — choose another above.

  • Massachusetts General Hospital-Genetics- Site Number : 840062
    Boston, Massachusetts 02114, United States
    Recruiting
  • Boston Children's Hospital- Site Number : 840092
    Boston, Massachusetts 02115, United States
    Recruiting
  • University Of Michigan Pediatrics- Site Number : 840107
    Ann Arbor, Michigan 48109, United States
    Recruiting
  • Children's Hospital of Michigan- Site Number : 840066
    Detroit, Michigan 48201, United States
    Recruiting
  • Corewell Health - Helen DeVos Children's Hospital (HDVCH)- Site Number : 840019
    Grand Rapids, Michigan 49503, United States
    Completed

    Not enrolling at this site — choose another above.

  • Infusion Associates - Grand Rapids- Site Number : 840050
    Grand Rapids, Michigan 49525, United States
    Completed

    Not enrolling at this site — choose another above.

  • Children's Hospitals and Clinics of Minnesota- Site Number : 840046
    Minneapolis, Minnesota 55404, United States
    Recruiting
  • Children's Hospitals and Clinics of Minnesota- Site Number : 840114
    Minneapolis, Minnesota 55404, United States
    Recruiting
  • M Health Fairview University of Minnesota Medical Center - West Bank- Site Number : 840076
    Minneapolis, Minnesota 55454, United States
    Recruiting
  • University Of Missouri Health System Department Of Genetics- Site Number : 840031
    Columbia, Missouri 65212, United States
    Recruiting
  • Children's Mercy Adelle Hall Campus- Site Number : 840063
    Kansas City, Missouri 64108, United States
    Completed

    Not enrolling at this site — choose another above.

  • Washington University- Site Number : 840100
    St Louis, Missouri 63110, United States
    Recruiting
  • University of Nebraska Medical Center- Site Number : 840084
    Omaha, Nebraska 68198, United States
    Recruiting
  • Children's Specialty Center - Las Vegas- Site Number : 840008
    Las Vegas, Nevada 89109, United States
    Completed

    Not enrolling at this site — choose another above.

  • Cooper Health Center- Site Number : 840098
    Camden, New Jersey 08103, United States
    Terminated

    Not enrolling at this site — choose another above.

  • Joseph M. Sanzari Children'S Hospital- Site Number : 840101
    Hackensack, New Jersey 07601, United States
    Recruiting
  • Atlantic Health System- Site Number : 840099
    Morristown, New Jersey 07962, United States
    Recruiting
  • Saint Peter's University Hospital - New Brunswick- Site Number : 840016
    New Brunswick, New Jersey 08901, United States
    Recruiting
  • St. Joseph's Regional Medical Center - Paterson- Site Number : 840057
    Paterson, New Jersey 07503, United States
    Recruiting
  • NYU Langone Health - Perlmutter Cancer Center - Huntington- Site Number : 840040
    Huntington, New York 11743, United States
    Recruiting
  • Northwell Health- Site Number : 840102
    New Hyde Park, New York 11042, United States
    Recruiting
  • Icahn School of Medicine at Mount Sinai- Site Number : 840005
    New York, New York 10029, United States
    Recruiting
  • Metropolitan Hospital Center- Site Number : 840110
    New York, New York 10029, United States
    Recruiting
  • Columbia University Irving Medical Center- Site Number : 840125
    New York, New York 10032, United States
    Recruiting
  • University of Rochester Medical Center- Site Number : 840105
    Rochester, New York 14642, United States
    Recruiting
  • SUNY Upstate Medical University - Site Number : 840052
    Syracuse, New York 13210, United States
    Recruiting
  • New York Medical College- Site Number : 840039
    Valhalla, New York 10595, United States
    Recruiting
  • Atrium Health Carolinas Medical Center Hospital- Site Number : 840065
    Charlotte, North Carolina 28203, United States
    Recruiting
  • Duke University Medical Center Genetics Dept- Site Number : 840037
    Durham, North Carolina 27710, United States
    Recruiting
  • Cincinnati Children's Hospital Medical Center- Site Number : 840026
    Cincinnati, Ohio 5229, United States
    Recruiting
  • University Hospitals Cleveland Medical Center- Site Number : 840119
    Cleveland, Ohio 44106, United States
    Recruiting
  • Cleveland Clinic - Cleveland- Site Number : 840048
    Cleveland, Ohio 44195, United States
    Recruiting
  • Nationwide Children's Hospital- Site Number : 840091
    Columbus, Ohio 43205, United States
    Recruiting
  • Lsd Data Registry Site Llc- Site Number : 840094
    Dublin, Ohio 43017, United States
    Recruiting
  • Oregon Health & Science University (OHSU)- Site Number : 840080
    Portland, Oregon 97239, United States
    Recruiting
  • Penn State Health Milton South Hershey Medical Center- Site Number : 840126
    Hershey, Pennsylvania 17033, United States
    Recruiting
  • Children'S Hospital Of Philadelphia Hup Medical Genetics- Site Number : 840089
    Philadelphia, Pennsylvania 19104, United States
    Recruiting
  • The Children's Hospital of Philadelphia- Site Number : 840034
    Philadelphia, Pennsylvania 19104, United States
    Recruiting
  • UPMC Children's Hospital of Pittsburgh- Site Number : 840023
    Pittsburgh, Pennsylvania 15224, United States
    Recruiting
  • Rhode Island Hospital Genetics- Site Number : 840053
    Providence, Rhode Island 02903, United States
    Completed

    Not enrolling at this site — choose another above.

  • Greenwood Genetic Center - Greenville- Site Number : 840055
    Greenville, South Carolina 29605, United States
    Recruiting
  • Vanderbilt University Medical Center - Medical Laboratories- Site Number : 840049
    Nashville, Tennessee 37228, United States
    Recruiting
  • Baylor Institute of Metabolic Disease- Site Number : 840058
    Dallas, Texas 75226, United States
    Completed

    Not enrolling at this site — choose another above.

  • Cook Children's Medical Center- Site Number : 840024
    Fort Worth, Texas 76104, United States
    Completed

    Not enrolling at this site — choose another above.

  • University of Utah- Site Number : 840043
    Salt Lake City, Utah 84112, United States
    Recruiting
  • University Of Virginia School Of Medicine-Pediatrics- Site Number : 840078
    Charlottesville, Virginia 22908, United States
    Completed

    Not enrolling at this site — choose another above.

  • O & O Alpan- Site Number : 840025
    Fairfax, Virginia 22030, United States
    Recruiting
  • Children's Hospital of The King's Daughters- Site Number : 840072
    Norfolk, Virginia 23507, United States
    Completed

    Not enrolling at this site — choose another above.

  • Seattle Children's Hospital- Site Number : 840028
    Seattle, Washington 98105, United States
    Recruiting
  • University of Washington Medical Center- Site Number : 840059
    Seattle, Washington 98195, United States
    Completed

    Not enrolling at this site — choose another above.

  • Mary Bridge Children's Hospital & Health Center- Site Number : 840115
    Tacoma, Washington 98405, United States
    Recruiting
  • Children's Wisconsin- Site Number : 840054
    Milwaukee, Wisconsin 53226, United States
    Recruiting
  • Investigational Site Number : 032003
    La Plata, Buenos Aires 1900, Argentina
    Completed

    Not enrolling at this site — choose another above.

  • Investigational Site Number : 150661
    Munro, Buenos Aires 1605, Argentina
    Completed

    Not enrolling at this site — choose another above.

  • Investigational Site Number : 604002
    Buenos Aires, 1107, Argentina
    Completed

    Not enrolling at this site — choose another above.

  • Investigational Site Number : 032012
    Buenos Aires, 1425, Argentina
    Completed

    Not enrolling at this site — choose another above.

  • Investigational Site Number : 032014
    Buenos Aires, 1428, Argentina
    Recruiting
  • Investigational Site Number : 154176
    Corrientes, 3400, Argentina
    Completed

    Not enrolling at this site — choose another above.

  • Investigational Site Number : 032011
    Córdoba, 5004, Argentina
    Completed

    Not enrolling at this site — choose another above.

  • Investigational Site Number : 151085
    Sarandí, 1123, Argentina
    Completed

    Not enrolling at this site — choose another above.

  • Investigational Site Number : 151431
    Sydney, New South Wales 2050, Australia
    Terminated

    Not enrolling at this site — choose another above.

  • Investigational Site Number : 151069
    Westmead, New South Wales 2145, Australia
    Recruiting
  • Investigational Site Number : 151010
    Brisbane, Queensland 4029, Australia
    Completed

    Not enrolling at this site — choose another above.

  • Investigational Site Number : 151491
    Townsville, Queensland 4814, Australia
    Completed

    Not enrolling at this site — choose another above.

  • Investigational Site Number : 151002
    Adelaide, South Australia 5000, Australia
    Recruiting
  • Investigational Site Number : 151725
    Hobart, Tasmania 7000, Australia
    Recruiting

Showing the first 100 of 317 sites across 52 countries. Choose a country and city above to see the rest.

08

References and documents

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 28040394 ↗
  • 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. PubMed 28569047 ↗
  • 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. PubMed 22976765 ↗
  • Ain 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 41527340 ↗
  • 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. PubMed 36054609 ↗
  • 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. PubMed 33485799 ↗
  • 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. PubMed 26096743 ↗
  • Buyers RP, Collin-Histed T. Foreword. Am J Hematol. 2015 Jul;90 Suppl 1:S1. doi: 10.1002/ajh.24053. No abstract available. PubMed 26096740 ↗
09

Timeline

1 registry update since Sep 25, 2026
ElapsedEstimatedEstimated, not yet reached

Started Apr 1991 (actual). Primary completion is estimated for Jan 2034, 513 months after the start.

How these figures are worked out, and which studies are left out of them.

Registered
Jul 12, 2006
First posted
Aug 1, 2006
StartActual
Apr 1, 1991
Primary completionEst.
Jan 31, 2034
CompletionEst.
Jan 31, 2034
Last update
Oct 14, 2026
What these dates mean
Start.
When the first participant enrolled; while estimated, when the study expects to open to recruitment.
Primary completion.
When the last participant was examined or treated to collect data for the study's main question, its primary outcome. A study that stopped early gives the date its data collection stopped.
Completion.
When the last participant's last visit took place, for every outcome, secondary ones and side effects included: the end of the study. Often later than primary completion, sometimes the same day.
Actual and Est.
Actual means the registry says the date has happened. Est. (estimated) is a date the sponsor plans or expects. It can still move, and the sponsor is expected to change it to actual once it has passed.
Sites
48 sites added, 49 sites removed. 11 sites changed recruiting status (11 now completed)
Show 11 sites
  • Children's Specialty Center - Las Vegas- Site Number : 840008 · Las Vegas, United StatesRecruiting→Completed
  • Investigational Site Number : 150661 · Munro, ArgentinaRecruiting→Completed
  • Investigational Site Number : 604002 · Buenos Aires, ArgentinaRecruiting→Completed
  • Investigational Site Number : 150944 · Brussels, BelgiumRecruiting→Completed
  • Investigational Site Number : 124002 · Edmonton, CanadaRecruiting→Completed
  • Investigational Site Number : 124022 · Moncton, CanadaRecruiting→Completed
  • Investigational Site Number : 124024 · Kingston, CanadaRecruiting→Completed
  • Investigational Site Number : 250006 · Nantes, FranceRecruiting→Completed
  • Investigational Site Number : 250016 · Tours, FranceRecruiting→Completed
  • Investigational Site Number : 422001 · Beirut, LebanonRecruiting→Completed
  • Investigational Site Number : 642001 · Cluj-Napoca, RomaniaRecruiting→Completed
Show 48 added (32 United States, 7 Spain, 3 Taiwan, 2 Italy, 1 Argentina, 1 Egypt, 1 Poland, 1 Russia)
  • Usc Health Sciences Center Dept Of Genetics- Site Number : 840082 · Los Angeles, United States
  • University of California Irvine Medical Center- Site Number : 840036 · Orange, United States
  • Uc Davis Mind Institute- Site Number : 840010 · Sacramento, United States
  • University Of Colorado At Denver Genetics- Site Number : 840068 · Aurora, United States
  • University of Miami- Site Number : 840006 · Coral Gables, United States
  • University of Miami Sylvester Comprehensive Cancer Center - Coral Springs- Site Number : 840075 · Coral Springs, United States
  • University Of Florida Pediatrics Genetics- Site Number : 840121 · Jacksonville, United States
  • University Of Florida-Genetics- Site Number : 840096 · Tampa, United States
  • Pediatric Endocrine Association- Site Number : 840017 · Tampa, United States
  • Emory University School Of Medicine- Human Genetics- Site Number : 840060 · Decatur, United States
  • Indianapolis University School Of Medicine- Site Number : 840027 · Indianapolis, United States
  • Boston Children's Hospital- Site Number : 840092 · Boston, United States
  • University Of Michigan Pediatrics- Site Number : 840107 · Ann Arbor, United States
  • Corewell Health - Helen DeVos Children's Hospital (HDVCH)- Site Number : 840019 · Grand Rapids, United States
  • M Health Fairview University of Minnesota Medical Center - West Bank- Site Number : 840076 · Minneapolis, United States
  • University Of Missouri Health System Department Of Genetics- Site Number : 840031 · Columbia, United States
  • Joseph M. Sanzari Children'S Hospital- Site Number : 840101 · Hackensack, United States
  • St. Joseph's Regional Medical Center - Paterson- Site Number : 840057 · Paterson, United States
  • NYU Langone Health - Perlmutter Cancer Center - Huntington- Site Number : 840040 · Huntington, United States
  • Northwell Health- Site Number : 840102 · New Hyde Park, United States
  • Icahn School of Medicine at Mount Sinai- Site Number : 840005 · New York, United States
  • SUNY Upstate Medical University - Site Number : 840052 · Syracuse, United States
  • Atrium Health Carolinas Medical Center Hospital- Site Number : 840065 · Charlotte, United States
  • Lsd Data Registry Site Llc- Site Number : 840094 · Dublin, United States
  • Children'S Hospital Of Philadelphia Hup Medical Genetics- Site Number : 840089 · Philadelphia, United States
  • The Children's Hospital of Philadelphia- Site Number : 840034 · Philadelphia, United States
  • Vanderbilt University Medical Center - Medical Laboratories- Site Number : 840049 · Nashville, United States
  • Cook Children's Medical Center- Site Number : 840024 · Fort Worth, United States
  • University of Utah- Site Number : 840043 · Salt Lake City, United States
  • University Of Virginia School Of Medicine-Pediatrics- Site Number : 840078 · Charlottesville, United States
  • …and 18 more on the registry record
Show 49 removed
  • University of California at Irvine- Site Number : 840036 · Irvine, United States
  • USC Health Sciences Center Dept of Genetics- Site Number : 840082 · Los Angeles, United States
  • UC Davis MIND Institute- Site Number : 840010 · Sacramento, United States
  • University Hematology Oncology- Site Number : 840075 · Coral Springs, United States
  • University of Florida Pediatrics Genetics- Site Number : 840121 · Jacksonville, United States
  • University Of Miami SOM- Site Number : 840006 · Miami, United States
  • University of Florida-Genetics- Site Number : 840096 · Tampa, United States
  • Pediatric Endocrine Associates- Site Number : 840017 · Tampa, United States
  • Emory University School of Medicine- Human Genetics- Site Number : 840060 · Decatur, United States
  • University of Colorado at Denver Genetics- Site Number : 840068 · Aurora, United States
  • Indianapolis University School of Medicine- Site Number : 840027 · Indianapolis, United States
  • Boston Children's Hospital - PIN- Site Number : 840092 · Boston, United States
  • University of Michigan Pediatrics- Site Number : 840107 · Ann Arbor, United States
  • Spectrum for Health- Site Number : 840019 · Grand Rapids, United States
  • University of Minnesota Medical Center Pediatrics- Site Number : 840076 · Minneapolis, United States
  • University of Missouri Health System Department of Genetics- Site Number : 840031 · Columbia, United States
  • Joseph M. Sanzari Children's Hospital- Site Number : 840101 · Hackensack, United States
  • St. Joseph's Children's Hospital- Site Number : 840057 · Paterson, United States
  • Northwell Health - Site Number : 840102 · Manhasset, United States
  • New York University School Of Medicine- Site Number : 840040 · New York, United States
  • Mt. Sinai School of Medicine- Site Number : 840005 · New York, United States
  • State University of New York- Site Number : 840052 · Syracuse, United States
  • Carolinas Medical Center Hospital- Site Number : 840065 · Charlotte, United States
  • LSD Data Registry Site LLC- Site Number : 840094 · Dublin, United States
  • Children's Hospital of Philadelphia HUP Medical Genetics- Site Number : 840089 · Philadelphia, United States
  • Children's Hospital of Philadelphia- Site Number : 840034 · Philadelphia, United States
  • Vanderbilt University Medical Center Diagnostic Lab- Site Number : 840049 · Nashville, United States
  • Cook Children's Health Care System- Site Number : 840024 · Fort Worth, United States
  • University of Utah Department of Medical Genetics- Site Number : 840043 · Salt Lake City, United States
  • University of Virginia School of Medicine-Pediatrics- Site Number : 840078 · Charlottesville, United States
  • …and 19 more on the registry record
Oct 14, 2026
Show all 1 update
  1. Oct 14, 2026
    48 sites added, 49 sites removed. 11 sites changed recruiting status (11 now completed)
    Show 11 sites
    • Children's Specialty Center - Las Vegas- Site Number : 840008 · Las Vegas, United StatesRecruiting→Completed
    • Investigational Site Number : 150661 · Munro, ArgentinaRecruiting→Completed
    • Investigational Site Number : 604002 · Buenos Aires, ArgentinaRecruiting→Completed
    • Investigational Site Number : 150944 · Brussels, BelgiumRecruiting→Completed
    • Investigational Site Number : 124002 · Edmonton, CanadaRecruiting→Completed
    • Investigational Site Number : 124022 · Moncton, CanadaRecruiting→Completed
    • Investigational Site Number : 124024 · Kingston, CanadaRecruiting→Completed
    • Investigational Site Number : 250006 · Nantes, FranceRecruiting→Completed
    • Investigational Site Number : 250016 · Tours, FranceRecruiting→Completed
    • Investigational Site Number : 422001 · Beirut, LebanonRecruiting→Completed
    • Investigational Site Number : 642001 · Cluj-Napoca, RomaniaRecruiting→Completed
    Show 48 added (32 United States, 7 Spain, 3 Taiwan, 2 Italy, 1 Argentina, 1 Egypt, 1 Poland, 1 Russia)
    • Usc Health Sciences Center Dept Of Genetics- Site Number : 840082 · Los Angeles, United States
    • University of California Irvine Medical Center- Site Number : 840036 · Orange, United States
    • Uc Davis Mind Institute- Site Number : 840010 · Sacramento, United States
    • University Of Colorado At Denver Genetics- Site Number : 840068 · Aurora, United States
    • University of Miami- Site Number : 840006 · Coral Gables, United States
    • University of Miami Sylvester Comprehensive Cancer Center - Coral Springs- Site Number : 840075 · Coral Springs, United States
    • University Of Florida Pediatrics Genetics- Site Number : 840121 · Jacksonville, United States
    • University Of Florida-Genetics- Site Number : 840096 · Tampa, United States
    • Pediatric Endocrine Association- Site Number : 840017 · Tampa, United States
    • Emory University School Of Medicine- Human Genetics- Site Number : 840060 · Decatur, United States
    • Indianapolis University School Of Medicine- Site Number : 840027 · Indianapolis, United States
    • Boston Children's Hospital- Site Number : 840092 · Boston, United States
    • University Of Michigan Pediatrics- Site Number : 840107 · Ann Arbor, United States
    • Corewell Health - Helen DeVos Children's Hospital (HDVCH)- Site Number : 840019 · Grand Rapids, United States
    • M Health Fairview University of Minnesota Medical Center - West Bank- Site Number : 840076 · Minneapolis, United States
    • University Of Missouri Health System Department Of Genetics- Site Number : 840031 · Columbia, United States
    • Joseph M. Sanzari Children'S Hospital- Site Number : 840101 · Hackensack, United States
    • St. Joseph's Regional Medical Center - Paterson- Site Number : 840057 · Paterson, United States
    • NYU Langone Health - Perlmutter Cancer Center - Huntington- Site Number : 840040 · Huntington, United States
    • Northwell Health- Site Number : 840102 · New Hyde Park, United States
    • Icahn School of Medicine at Mount Sinai- Site Number : 840005 · New York, United States
    • SUNY Upstate Medical University - Site Number : 840052 · Syracuse, United States
    • Atrium Health Carolinas Medical Center Hospital- Site Number : 840065 · Charlotte, United States
    • Lsd Data Registry Site Llc- Site Number : 840094 · Dublin, United States
    • Children'S Hospital Of Philadelphia Hup Medical Genetics- Site Number : 840089 · Philadelphia, United States
    • The Children's Hospital of Philadelphia- Site Number : 840034 · Philadelphia, United States
    • Vanderbilt University Medical Center - Medical Laboratories- Site Number : 840049 · Nashville, United States
    • Cook Children's Medical Center- Site Number : 840024 · Fort Worth, United States
    • University of Utah- Site Number : 840043 · Salt Lake City, United States
    • University Of Virginia School Of Medicine-Pediatrics- Site Number : 840078 · Charlottesville, United States
    • …and 18 more on the registry record
    Show 49 removed
    • University of California at Irvine- Site Number : 840036 · Irvine, United States
    • USC Health Sciences Center Dept of Genetics- Site Number : 840082 · Los Angeles, United States
    • UC Davis MIND Institute- Site Number : 840010 · Sacramento, United States
    • University Hematology Oncology- Site Number : 840075 · Coral Springs, United States
    • University of Florida Pediatrics Genetics- Site Number : 840121 · Jacksonville, United States
    • University Of Miami SOM- Site Number : 840006 · Miami, United States
    • University of Florida-Genetics- Site Number : 840096 · Tampa, United States
    • Pediatric Endocrine Associates- Site Number : 840017 · Tampa, United States
    • Emory University School of Medicine- Human Genetics- Site Number : 840060 · Decatur, United States
    • University of Colorado at Denver Genetics- Site Number : 840068 · Aurora, United States
    • Indianapolis University School of Medicine- Site Number : 840027 · Indianapolis, United States
    • Boston Children's Hospital - PIN- Site Number : 840092 · Boston, United States
    • University of Michigan Pediatrics- Site Number : 840107 · Ann Arbor, United States
    • Spectrum for Health- Site Number : 840019 · Grand Rapids, United States
    • University of Minnesota Medical Center Pediatrics- Site Number : 840076 · Minneapolis, United States
    • University of Missouri Health System Department of Genetics- Site Number : 840031 · Columbia, United States
    • Joseph M. Sanzari Children's Hospital- Site Number : 840101 · Hackensack, United States
    • St. Joseph's Children's Hospital- Site Number : 840057 · Paterson, United States
    • Northwell Health - Site Number : 840102 · Manhasset, United States
    • New York University School Of Medicine- Site Number : 840040 · New York, United States
    • Mt. Sinai School of Medicine- Site Number : 840005 · New York, United States
    • State University of New York- Site Number : 840052 · Syracuse, United States
    • Carolinas Medical Center Hospital- Site Number : 840065 · Charlotte, United States
    • LSD Data Registry Site LLC- Site Number : 840094 · Dublin, United States
    • Children's Hospital of Philadelphia HUP Medical Genetics- Site Number : 840089 · Philadelphia, United States
    • Children's Hospital of Philadelphia- Site Number : 840034 · Philadelphia, United States
    • Vanderbilt University Medical Center Diagnostic Lab- Site Number : 840049 · Nashville, United States
    • Cook Children's Health Care System- Site Number : 840024 · Fort Worth, United States
    • University of Utah Department of Medical Genetics- Site Number : 840043 · Salt Lake City, United States
    • University of Virginia School of Medicine-Pediatrics- Site Number : 840078 · Charlottesville, United States
    • …and 19 more on the registry record
    + 1 other change: verification date

From the registry record's own update history. This site started tracking changes on Sep 25, 2026; for anything earlier, see the record history on ClinicalTrials.gov ↗

10

Registry details

Key details

Study ID
NCT00358943
Responsible party
Sponsor
First posted
Aug 1, 2006
Start date
Apr 1, 1991
Primary completion
Jan 31, 2034 (estimated)
Completion
Jan 31, 2034 (estimated)
Last update
Oct 14, 2026

Study contacts

Trial Transparency email recommended (Toll free number for US & Canada)
Contact
contact-us@sanofi.com
800-633-1610 ext. option 6
ICGG Gaucher Registry HelpLine
Contact
+1(617) 252-7500
Study Director
study director · Genzyme, a Sanofi Company

Oversight

Data monitoring committee
No
View the source record on ClinicalTrials.gov ↗

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