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ClinicalTrials.gov public records Last synced Oct 21, 2026, 1:28 AM EDT

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Showing 1–24 of 38 matching trials from the live ClinicalTrials.gov search.
Filters: Intervention: Imiglucerase Clear all
Completed Phase 1 Interventional

Phase I Study of Retrovirally Mediated Transfer of the Human Glucocerebrosidase Gene Into Peripheral Blood Stem Cells for Autologous Transplantation in Patients With Type I Gaucher Disease

NCT00004294
View directory record Official record
Conditions
Gaucher's Disease
Interventions
human glucocerebrosidase gene into autologous peripheral blood stem cells
Genetic
Lead sponsor
National Center for Research Resources (NCRR)
NIH
Eligibility
18 Years to 65 Years
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1999
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Oct 21, 2026, 1:28 AM EDT
Completed Phase 4 Interventional

A Multicenter Study of the Efficacy of Cerezyme in Testing Skeletal Disease in Patients With Type I Gaucher Disease.

NCT00365131
View directory record Official record
Conditions
Gaucher Disease Type I, Cerebroside Lipidosis Syndrome, Clucocerebrosidase Deficiency Disease, Glucosylceramide Beta-Glucosidase Deficiency Disease, Gaucher Disease, Non-Neuronopathic Form
Interventions
Cerezyme (imiglucerase for injection)
Drug
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
10 Years to 65 Years
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1997 – 2004
U.S. locations
1
States / cities
Coral Springs, Florida
Source: ClinicalTrials.gov public record
Updated Mar 5, 2015 · Synced Oct 21, 2026, 1:28 AM EDT
Completed Phase 3 Interventional Results available

A Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease Who Have Reached Therapeutic Goals With Enzyme Replacement Therapy (ENCORE)

NCT00943111
View directory record Official record
Conditions
Gaucher Disease, Type 1
Interventions
Eliglustat tartrate, Imiglucerase
Drug
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
18 Years and older
Enrollment
160 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2015
U.S. locations
17
States / cities
Beverly Hills, California • San Francisco, California • Aurora, Colorado + 13 more
Source: ClinicalTrials.gov public record
Updated Nov 25, 2016 · Synced Oct 21, 2026, 1:28 AM EDT
Not listed No phase listed Observational Accepts healthy volunteers

Biomarker Analysis for GBA Associated Parkinson's Disease

NCT03811496
View directory record Official record
Conditions
Parkinson Disease, Gaucher Disease
Interventions
Not listed
Lead sponsor
Lysosomal and Rare Disorders Research and Treatment Center, Inc.
Other
Eligibility
50 Years and older
Enrollment
100 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2018 – 2020
U.S. locations
1
States / cities
Fairfax, Virginia
Source: ClinicalTrials.gov public record
Updated Jan 23, 2019 · Synced Oct 21, 2026, 1:28 AM EDT
Enrolling by invitation No phase listed Observational Accepts healthy volunteers

Genetic Studies of Lysosomal Storage Disorders

NCT00001215
View directory record Official record
Conditions
Lysosomal Storage Disorders, Gaucher Disease, Parkinson Disease
Interventions
Not listed
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
1 Week to 110 Years
Enrollment
1,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
Started 1995
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 22, 2026 · Synced Oct 21, 2026, 1:28 AM EDT
Completed Phase 4 Interventional Results available

Study of the Effect of Velaglucerase Alfa (VPRIV®) on Bone-related Pathology in Treatment-naïve Participants With Type 1 Gaucher Disease

NCT02574286
View directory record Official record
Conditions
Gaucher Disease
Interventions
Velaglucerase alfa, Vitamin D
Drug · Dietary Supplement
Lead sponsor
Shire
Industry
Eligibility
18 Years to 70 Years
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2020
U.S. locations
8
States / cities
Beverly Hills, California • Los Angeles, California • Atlanta, Georgia + 5 more
Source: ClinicalTrials.gov public record
Updated Feb 1, 2022 · Synced Oct 21, 2026, 1:28 AM EDT
Active, not recruiting Phase 1Phase 2 Interventional

Phase 1/2 Clinical Trial of PR001 in Infants With Type 2 Gaucher Disease (PROVIDE)

NCT04411654
View directory record Official record
Conditions
Gaucher Disease, Type 2
Interventions
LY3884961, Methylprednisolone, Sirolimus, Prednisone
Genetic · Drug
Lead sponsor
Prevail Therapeutics
Industry
Eligibility
0 Months to 24 Months
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2027
U.S. locations
5
States / cities
Oakland, California • Minneapolis, Minnesota • New York, New York + 2 more
Source: ClinicalTrials.gov public record
Updated Aug 19, 2026 · Synced Oct 21, 2026, 1:28 AM EDT
Completed Phase 3 Interventional Results available

Plant Cell Expressed Recombinant Human Glucocerebrosidase Extension Trial

NCT00705939
View directory record Official record
Conditions
Gaucher Disease
Interventions
Taliglucerase alfa
Drug
Lead sponsor
Pfizer
Industry
Eligibility
18 Years and older
Enrollment
45 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2013
U.S. locations
2
States / cities
Decatur, Georgia • New York, New York
Source: ClinicalTrials.gov public record
Updated Oct 4, 2018 · Synced Oct 21, 2026, 1:28 AM EDT
Active, not recruiting Phase 2 Interventional

Efficacy, Safety, Tolerability, Pharmacodynamics, and Pharmacokinetics of BIA 28-6156 in GBA-PD

NCT05819359
View directory record Official record
Conditions
Parkinson's Disease
Interventions
BIA 28-6156 10 mg, BIA 28-6156 60 mg, Placebo
Drug
Lead sponsor
Bial R&D Investments, S.A.
Industry
Eligibility
35 Years to 80 Years
Enrollment
237 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2026
U.S. locations
43
States / cities
Phoenix, Arizona • La Jolla, California • Los Angeles, California + 30 more
Source: ClinicalTrials.gov public record
Updated May 13, 2026 · Synced Oct 21, 2026, 1:28 AM EDT
Completed Phase 1 Interventional

PEG-Glucocerebrosidase for the Treatment of Gaucher Disease

NCT00001410
View directory record Official record
Conditions
Gaucher's Disease
Interventions
Lysodase
Drug
Lead sponsor
National Institute of Mental Health (NIMH)
NIH
Eligibility
Not listed
Enrollment
18 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1993 – 2001
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 4, 2008 · Synced Oct 21, 2026, 1:28 AM EDT
Withdrawn Phase 4 Interventional

The Effect of Velaglucerase Alfa (Vpriv) on Skeletal Development in Pediatric Gaucher Disease

NCT02528617
View directory record Official record
Conditions
Gaucher Disease Type 1, Gaucher Disease Type 3
Interventions
Velaglucerase alfa
Drug
Lead sponsor
Baylor Research Institute
Other
Eligibility
4 Years to 14 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2017
U.S. locations
1
States / cities
Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Mar 2, 2018 · Synced Oct 21, 2026, 1:28 AM EDT
Completed Phase 2Phase 3 Interventional Results available

Study of GA-GCB Enzyme Replacement Therapy in Type 1 Gaucher Disease Patients Previously Treated With Imiglucerase

NCT00478647
View directory record Official record
Conditions
Gaucher Disease
Interventions
GA-GCB (velaglucerase alfa)
Biological
Lead sponsor
Shire
Industry
Eligibility
2 Years and older
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2009
U.S. locations
11
States / cities
Los Angeles, California • Oakland, California • Decatur, Georgia + 8 more
Source: ClinicalTrials.gov public record
Updated Jun 10, 2021 · Synced Oct 21, 2026, 1:28 AM EDT
Completed Phase 2 Interventional

Bone Response to Enzyme Replacement in Gaucher's Disease

NCT00001416
View directory record Official record
Conditions
Gaucher's Disease
Interventions
CEREDASE™
Drug
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
Not listed
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1993 – 2000
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 22, 2016 · Synced Oct 21, 2026, 1:28 AM EDT
Completed Phase 3 Interventional Results available

A Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease (ENGAGE)

NCT00891202
View directory record Official record
Conditions
Gaucher Disease, Type 1
Interventions
Eliglustat tartrate, Placebo
Drug
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
16 Years and older
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2016
U.S. locations
6
States / cities
San Francisco, California • New Haven, Connecticut • Decatur, Georgia + 2 more
Source: ClinicalTrials.gov public record
Updated Mar 3, 2017 · Synced Oct 21, 2026, 1:28 AM EDT
Completed Phase 3 Interventional Results available

Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) ERT Compared With Imiglucerase in Type I Gaucher Disease

NCT00553631
View directory record Official record
Conditions
Gaucher Disease, Type 1
Interventions
velaglucerase alfa, imiglucerase
Biological
Lead sponsor
Shire
Industry
Eligibility
2 Years and older
Enrollment
34 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2009
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Jun 8, 2021 · Synced Oct 21, 2026, 1:28 AM EDT
Active, not recruiting No phase listed Observational

Genotypic Influences on Network Progression in Parkinson's Disease

NCT04228172
View directory record Official record
Conditions
Parkinson's Disease
Interventions
DNA/GeneticTesting, FDG PET scan, MRI scan, Clinical and neuropsychological assessments
Genetic · Radiation · Other
Lead sponsor
Northwell Health
Other
Eligibility
40 Years to 75 Years
Enrollment
32 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2026
U.S. locations
1
States / cities
Manhasset, New York
Source: ClinicalTrials.gov public record
Updated Apr 13, 2026 · Synced Oct 21, 2026, 1:28 AM EDT
Active, not recruiting Phase 2 Interventional

Venglustat in Combination With Cerezyme in Adult Patients With Gaucher Disease Type 3 With Venglustat Monotherapy Extension

NCT02843035
View directory record Official record
Conditions
Gaucher Disease Type 1, Gaucher Disease Type 3
Interventions
venglustat (GZ402671), imiglucerase
Drug
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
18 Years and older
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2026
U.S. locations
3
States / cities
New Haven, Connecticut • Dallas, Texas • Fairfax, Virginia
Source: ClinicalTrials.gov public record
Updated Jul 10, 2026 · Synced Oct 21, 2026, 1:28 AM EDT
Completed No phase listed Observational

High Order Spectral Analysis of Local Field Potential Data on a Subgroup of Parkinson's Disease Patients Who Are Carriers of Mutations in the Glucocerebrosidase (GBA) Gene Undergoing DBS Electrode Placement

NCT04268030
View directory record Official record
Conditions
Parkinson Disease
Interventions
collection of LFPs
Other
Lead sponsor
Rush University Medical Center
Other
Eligibility
30 Years to 80 Years
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020
U.S. locations
1
States / cities
Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Jul 9, 2021 · Synced Oct 21, 2026, 1:28 AM EDT
Terminated Phase 2 Interventional Results available

A Global Study to Assess the Drug Dynamics, Efficacy, and Safety of Venglustat (GZ/SAR402671) in Parkinson's Disease Patients Carrying a Glucocerebrosidase (GBA) Gene Mutation

NCT02906020
View directory record Official record
Conditions
Parkinson's Disease
Interventions
venglustat GZ/SAR402671, Placebo
Drug
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
18 Years to 80 Years
Enrollment
273 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2021
U.S. locations
19
States / cities
Scottsdale, Arizona • La Jolla, California • Palo Alto, California + 10 more
Source: ClinicalTrials.gov public record
Updated May 24, 2022 · Synced Oct 21, 2026, 1:28 AM EDT
Completed No phase listed Observational

Positron Emission Tomography (PET) Imaging in People With Gaucher Mutations

NCT00302146
View directory record Official record
Conditions
Parkinson Disease, Gaucher Disease
Interventions
15-0 H20
Drug
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
18 Years to 110 Years
Enrollment
64 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2006
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 28, 2026 · Synced Oct 21, 2026, 1:28 AM EDT
Completed Phase 3 Interventional Results available

A Phase III Trial to Assess the Safety and Efficacy of Plant Cell Expressed GCD in Patients With Gaucher Disease

NCT00376168
View directory record Official record
Conditions
Gaucher Disease
Interventions
Plant cell expressed recombinant glucocerebrosidase (prGCD)
Drug
Lead sponsor
Pfizer
Industry
Eligibility
18 Years and older
Enrollment
32 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2009
U.S. locations
3
States / cities
Coral Springs, Florida • Decatur, Georgia • New York, New York
Source: ClinicalTrials.gov public record
Updated Oct 4, 2018 · Synced Oct 21, 2026, 1:28 AM EDT
Recruiting No phase listed Observational

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

NCT00358943
View directory record Official record
Conditions
Gaucher Disease, Cerebroside Lipidosis Syndrome, Glucocerebrosidase Deficiency Disease, Glucosylceramide Beta-Glucosidase Deficiency Disease
Interventions
Not listed
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
Not listed
Enrollment
12,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1991 – 2034
U.S. locations
86
States / cities
Phoenix, Arizona • Tucson, Arizona • Little Rock, Arkansas + 65 more
Source: ClinicalTrials.gov public record
Updated Oct 14, 2026 · Synced Oct 21, 2026, 1:28 AM EDT
Completed No phase listed Observational

Validating a New Severity Score System for Adults With Type 1 Gaucher Disease (GD1)

NCT01136304
View directory record Official record
Conditions
Gaucher Disease
Interventions
Imiglucerase
Drug
Lead sponsor
University Research Foundation for Lysosomal Storage Diseases, Inc.
Other
Eligibility
18 Years and older
Enrollment
173 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2013
U.S. locations
2
States / cities
Beverly Hills, California • Coral Springs, Florida
Source: ClinicalTrials.gov public record
Updated Jun 1, 2015 · Synced Oct 21, 2026, 1:28 AM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

Gene Therapy for Gaucher's and Fabry Disease Using Viruses and Blood-Forming Cells

NCT00001234
View directory record Official record
Conditions
Gaucher's Disease
Interventions
human glucocerebrosidase cDNA
Genetic
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
Not listed
Enrollment
120 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
1988 – 2002
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 4, 2008 · Synced Oct 21, 2026, 1:28 AM EDT